MELANIE SWICK BUZZELLI

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Drug Pricing. H.R.3: Elijah E. Cummings Lower Drug Costs Now Act. FDA Patient-Focused Drug Development (PFDD) Draft Guidance. Importation Issues. FDA-2019-D-5743: Importation of Certain Food and Drug Administration-Approved Human Prescription Drugs, Including Biological Products, Under Section 801(d)(1)(B) of the Federal Food, Drug, and Cosmetic Act; Draft Guidance for Industry; Availability. H.R.5497/S.3133: Conditional Approval Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Drug Pricing. H.R.3: Elijah E. Cummings Lower Drug Costs Now Act. FDA Patient-Focused Drug Development (PFDD) Draft Guidance. Importation Issues. FDA-2019-D-5743: Importation of Certain Food and Drug Administration-Approved Human Prescription Drugs, Including Biological Products, Under Section 801(d)(1)(B) of the Federal Food, Drug, and Cosmetic Act; Draft Guidance for Industry; Availability. H.R.5497/S.3133: Conditional Approval Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Disease Day Resolution. NIH Rare Disease Day. FDA Rare Disease Day. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Individualized Therapies. Genetic Testing. Gene Therapy. Medical Foods. Public Law No: 111-148: The Patient Protection and Affordable Care Act. CMS-9916-P: Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2021; Notice Requirement for Non-Federal Governmental Plans. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. COVID-19 Related Issues. H.R.748/S.3548: Coronavirus Aid, Relief, and Economic Security Act (CARES) Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. Drug Pricing. S.2543: Prescription Drug Pricing Reduction Act (PDPRA) of 2019. H.R.3: Lower Drug Costs Now Act of 2019.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. FDA-2018-N-4171: Patient Engagement in Medical Device Clinical Trials. Docket No. FDA-2018-D-4455-0001: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data; Draft Guidance for Industry and Other Stakeholders. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. FR Document: 2019-01026 Citation: 84 FR 2340: Removal of Safe Harbor Protection for Rebates Involving Prescription Pharmaceuticals and Creation of New Safe Harbor Protection for Certain Point-of-Sale Reductions in Price on Prescription Pharmaceuticals and Certain Pharmacy Benefit Manager Service Fees.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. Drug Pricing. S.2543: Prescription Drug Pricing Reduction Act (PDPRA) of 2019. H.R.3: Lower Drug Costs Now Act of 2019.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Drug Pricing. H.R.3: Elijah E. Cummings Lower Drug Costs Now Act. FDA Patient-Focused Drug Development (PFDD) Draft Guidance. H.R.4712: Fairness in Orphan Drug Exclusivity Act.

Patent issues impacting patients with rare diseases. Section 101 of the Patent Act. H.R.3666: STRONGER Patents Act of 2019.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Drug Pricing. H.R.3: Elijah E. Cummings Lower Drug Costs Now Act. FDA Patient-Focused Drug Development (PFDD) Draft Guidance. H.R.4712: Fairness in Orphan Drug Exclusivity Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. FDA-2018-N-4171: Patient Engagement in Medical Device Clinical Trials. Docket No. FDA-2018-D-4455-0001: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data; Draft Guidance for Industry and Other Stakeholders. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. FR Document: 2019-01026 Citation: 84 FR 2340: Removal of Safe Harbor Protection for Rebates Involving Prescription Pharmaceuticals and Creation of New Safe Harbor Protection for Certain Point-of-Sale Reductions in Price on Prescription Pharmaceuticals and Certain Pharmacy Benefit Manager Service Fees.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. Drug Pricing. S.2543: Prescription Drug Pricing Reduction Act (PDPRA) of 2019. H.R.3: Lower Drug Costs Now Act of 2019.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. FDA-2018-N-4171: Patient Engagement in Medical Device Clinical Trials. Docket No. FDA-2018-D-4455-0001: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data; Draft Guidance for Industry and Other Stakeholders. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. FR Document: 2019-01026 Citation: 84 FR 2340: Removal of Safe Harbor Protection for Rebates Involving Prescription Pharmaceuticals and Creation of New Safe Harbor Protection for Certain Point-of-Sale Reductions in Price on Prescription Pharmaceuticals and Certain Pharmacy Benefit Manager Service Fees.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. Orphan Drug Tax Credit. H.R.1332: Fair Care Act of 2019. H.R.2296: FAIR Drug Pricing Act of 2019. H.R.987: Strengthening Health Care and Lowering Prescription Drug Costs Act. S.474: Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019. Rebate Rule Comments. FDA Natural History Studies for Drug Development. Docket No. FDA-2015-D-2818: Rare Diseases: Common Issues in Drug Development, Guidance For Industry; Draft Guidance. H.R.965/S.340: CREATES Act of 2019. Prescription Drug User Fee Act (PDUFA) Reauthorization.

Health issues impacting patients with rare diseases. H.R.2279/S.2546: Safe Step Act. H.R.4228: Rare disease Advancement, surveillance Research, and Education (RARE) Act of 2019. H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Docket No. FDA-2019-D-1264: Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Draft Guidance for Industry; Availability. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.4439: Creating Hope Reauthorization Act. Biosimilars. Genetic Testing. FDA User Fee Reauthorization (Legislation Not Yet Introduced). Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Section 1557 of the Patient Protection and Affordable Care Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance. Enrollment Practices. Copay Accumulator Adjustment Programs (CAAPs). CMS-2406-P/RIN 0938-AT41/: Methods for Assuring Access to Care; Exceptions for High Managed Care Penetration and Rate Reduction Threshold. Docket No. HHS-OCR-2019-0007: Nondiscrimination in Health and Health Education Programs or Activities. Docket No. FDA-2019-N-1619: List of Patient Preference-Sensitive Priorities; Establishment of a Public Docket; Request for Comments.

Health issues impacting patients with rare diseases. H.R.4228: Rare disease Advancement, Surveillance Research, and Education (RARE) Act of 2019. Rare Cancer. Step Therapy. H.R.2279/S.2546: Safe Step Act. Newborn Screening. H.R.2507/S.2158: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. Pediatric Research Equity Act (PREA). Biosimilars. Genetic Testing. Clinical Trials. Institutional Review Boards. Medical Devices. Medical Foods. H.R.2501: Medical Nutrition Equity Act of 2019. Public Law No: 111-148: The Patient Protection and Affordable Care Act. S.1895: Lower Health Care Costs Act. Surprise Medical Billing. Dr. Hahn's Confirmation.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). Advancing Access to Targeted Therapies Act (Legislation not yet introduced). H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. H.R.2507: Newborn Screening Saves Lives Reauthorization Act of 2019. H.R. 776/S.1173: Emergency Medical Services for Children Program Reauthorization Act of 2019. H.R.1379/S.560: Ensuring Lasting Smiles Act. H.R.2862: VACCINES Act of 2019. Copay Accumulator Adjustment Programs (CAAPs). The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). H.R.2501: Medical Nutrition Equity Act. H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect. H.R.986: Protecting Americans with Preexisting Conditions Act of 2019. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. S.1125: Protect Act. RIN 0938-ZB45: Patient Protection and Affordable Care Act; Increasing Consumer Choice Through the Sale of Individual Health Insurance Coverage Across State Lines Through Health Care Choice Compacts. H.R.1386/S.1905: ENROLL Act of 2019. H.R.2073/S.110: Medical Expense Savings Act. Guidance on Compliance with the Congressional Review Act.

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Medicare issues impacting patients with rare diseases. Medicare Part D Program. Section 1115 Waivers. Section 1332: State Innovation Waivers. CMS-2406-P2/RIN: 0938-AT41: Methods for Assuring Access to Covered Medicaid Services - Rescission. CMS-9936-NC2: Request for Information Regarding State Relief and Empowerment Waivers. S.J.Res.52: A joint resolution providing for congressional disapproval under chapter 8 of title 5, United States Code, of the rule submitted by the Secretary of the Treasury and the Secretary of Health and Human Services relating to "State Relief and Empowerment Waivers".

Medicare issues impacting patients with rare diseases. Medicare Part D Program. Section 1115 Waivers. Section 1332: State Innovation Waivers. CMS-2019-0100-0562: Medicare and Medicaid Programs: CY 2020 Home Health Prospective Payment System Rate Update; Home Health Value-Based Purchasing Model; Home Health Value-Based Purchasing Model; Home Health Quality Reporting Requirements; and Home Infusion Therapy Requirements. S.J.Res.52: A joint resolution providing for congressional disapproval under chapter 8 of title 5, United States Code, of the rule submitted by the Secretary of the Treasury and the Secretary of Health and Human Services relating to "State Relief and Empowerment Waivers".

Health issues impacting patients with rare diseases. H.R.2279: Safe Step Act. Rare Disease Advancement, Research, and Education Act (Legislation not yet introduced). H.Res.161/S.Res.90: Expressing support for the designation of the last day of February each year as "Rare Disease Day". H.Res.242: Affirming the importance of the Orphan Drug Act, celebrating the over 750 new orphan therapies approved since its creation, and recognizing the need to continue supporting research and development for rare diseases. Pediatric subspecialty loan repayment reauthorization. Newborn Screening Saves Lives Act Reauthorization (Legislation not yet introduced). H.R.1379/S.560: Ensuring Lasting Smiles Act. CMS-9923-NC: Request for Information Regarding Grandfathered Group Health Plans and Grandfathered Group Health Insurance Coverage. H.R.1884: Protecting Pre-Existing Conditions and Making Health Care More Affordable Act of 2019. The Patient-Centered Outcomes Research Institute (PCORI) Reauthorization (Legislation not yet introduced). Medical Nutrition Equity Act (Legislation not yet introduced). H.R.1010: To provide that the rule entitled "Short-Term, Limited Duration Insurance" shall have no force or effect.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. H.R.1223/S.1509: Orphan Products Extension Now Accelerating Cures and Treatments Act of 2017. H.R.5062: Advancing Access to Precision Medicine Act.

Bipartisan Health Care Stabilization Act Medicare Part D Right to Try Act; H.R.5247, S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health Funding Food and Drug Administration Funding Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Advancing Care for Exceptional Kids Act; S.428 Pediatric Device Consortia Grant Program Congressionally Directed Medical Research Program Rare Disease Day Resolution; S.Res.423 Short-term, limited-duration health plans Association health plans OPEN Act; H.R.1223, S.1509 Advancing Access to Precision Medicine Act of 2018; H.R.5062 Restoring the Patient's Voice Act of 2017; H.R.2077

Pharmaceutical issues impacting patients with rare diseases. H.R.1223/S.1509: Orphan Products Extension Now Accelerating Cures and Treatments Act of 2017. Patient-focused drug development. Biological products marketplace. HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. H.R.1223/S.1509: Orphan Products Extension Now Accelerating Cures and Treatments Act of 2017. Accelerated Approval. S.3000: Patient Advocacy Transparency Act. HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs.

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Copay accumulator programs. Slowly progressive low-prevalence rare diseases. S.3388: Ensuring Coverage for Patients with Pre-Existing Conditions Act. Pediatric subspecialty loan repayment reauthorization. Medical expense deduction. Humanitarian Device Exemption (HDE) program. Risk adjustment payments.

Health issues impacting patients with rare diseases. Bipartisan Health Care Stabilization Act. H.R.5247/S.204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. S.428: Advancing Care for Exceptional Kids Act of 2017 or the ACE Kids Act of 2017. Congressionally Directed Medical Research Program. S.Res.423: A resolution designating February 28, 2018, as "Rare Disease Day". Short-term, limited-duration health plans. Association health plans. H.R.2077: Restoring the Patient's Voice Act of 2017.

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Pediatric subspecialty loan repayment reauthorization. Human gene therapy for rare diseases. Newborn Screening Saves Lives Act reauthorization. Neurological Conditions Surveillance System funding. H.R.7102: ENROLL Act of 2018.

Medicare Part D Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper Medical Nutrition Equity Act; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Medicaid waivers Section 1332 waivers Copay accumulator programs Patient-focused drug development Biological products marketplace Slowly progressive low-prevalence rare diseases HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Ensuring Coverage for Patients with Pre-Existing Conditions Act; S.3388 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Medical expense deduction Humanitarian Device Exemption (HDE) program Risk adjustment payments

Medicare Part D Affirming the importance of the Orphan Drug Act, applauding its lifesaving contributions over its 35-year history, and recognizing the need to continue supporting research and development for rare diseases; H.Res.1154 A resolution affirming the importance of the Orphan Drug Act, applauding the life-saving accomplishments of the Act during its 35-year history, and recognizing the need to continue support for research and development of new therapies for rare diseases; S.Res.690 Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Medical Nutrition Equity Act of 2017; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Section 1115 waivers Section 1332 waivers Copay accumulator programs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Patient-focused drug development Human gene therapy for rare diseases Rare disease early development and the role of pre-investigational new drug application meetings International pricing index model for Medicare part B drugs Newborn Screening Saves Lives Act reauthorization Neurological Conditions Surveillance System funding MODERN Labeling Act of 2018; S.3519 ENROLL Act of 2018; H.R.7102

Bipartisan Health Care Stabilization Act Medicare Part D Right to Try Act; H.R.5247, S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health Funding Food and Drug Administration Funding Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Advancing Care for Exceptional Kids Act; S.428 Pediatric Device Consortia Grant Program Congressionally Directed Medical Research Program Rare Disease Day Resolution; S.Res.423 Short-term, limited-duration health plans Association health plans OPEN Act; H.R.1223, S.1509 Advancing Access to Precision Medicine Act of 2018; H.R.5062 Restoring the Patient's Voice Act of 2017; H.R.2077

Medicare Part D Right to Try Act; S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration Appropriations Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Congressionally Directed Medical Research Program Short-term, limited-duration health plans Association health plans Accelerated Approval Medicaid waivers Inpatient prospective payment system (IPPS) proposed rule Patient Advocacy Transparency Act; S.3000 HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs

Bipartisan Health Care Stabilization Act Medicare Part D Right to Try Act; H.R.5247, S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health Funding Food and Drug Administration Funding Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Advancing Care for Exceptional Kids Act; S.428 Pediatric Device Consortia Grant Program Congressionally Directed Medical Research Program Rare Disease Day Resolution; S.Res.423 Short-term, limited-duration health plans Association health plans OPEN Act; H.R.1223, S.1509 Advancing Access to Precision Medicine Act of 2018; H.R.5062 Restoring the Patient's Voice Act of 2017; H.R.2077

Medicare Part D Affirming the importance of the Orphan Drug Act, applauding its lifesaving contributions over its 35-year history, and recognizing the need to continue supporting research and development for rare diseases; H.Res.1154 A resolution affirming the importance of the Orphan Drug Act, applauding the life-saving accomplishments of the Act during its 35-year history, and recognizing the need to continue support for research and development of new therapies for rare diseases; S.Res.690 Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Medical Nutrition Equity Act of 2017; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Section 1115 waivers Section 1332 waivers Copay accumulator programs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Patient-focused drug development Human gene therapy for rare diseases Rare disease early development and the role of pre-investigational new drug application meetings International pricing index model for Medicare part B drugs Newborn Screening Saves Lives Act reauthorization Neurological Conditions Surveillance System funding MODERN Labeling Act of 2018; S.3519 ENROLL Act of 2018; H.R.7102

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Copay accumulator programs. Slowly progressive low-prevalence rare diseases. S.3388: Ensuring Coverage for Patients with Pre-Existing Conditions Act. Pediatric subspecialty loan repayment reauthorization. Medical expense deduction. Humanitarian Device Exemption (HDE) program. Risk adjustment payments.

Health issues impacting patients with rare diseases. Bipartisan Health Care Stabilization Act. H.R.5247/S.204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. S.428: Advancing Care for Exceptional Kids Act of 2017 or the ACE Kids Act of 2017. Congressionally Directed Medical Research Program. S.Res.423: A resolution designating February 28, 2018, as "Rare Disease Day". Short-term, limited-duration health plans. Association health plans. H.R.2077: Restoring the Patient's Voice Act of 2017.

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Pediatric subspecialty loan repayment reauthorization. Human gene therapy for rare diseases. Newborn Screening Saves Lives Act reauthorization. Neurological Conditions Surveillance System funding. H.R.7102: ENROLL Act of 2018.

Medicare Part D Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper Medical Nutrition Equity Act; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Medicaid waivers Section 1332 waivers Copay accumulator programs Patient-focused drug development Biological products marketplace Slowly progressive low-prevalence rare diseases HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Ensuring Coverage for Patients with Pre-Existing Conditions Act; S.3388 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Medical expense deduction Humanitarian Device Exemption (HDE) program Risk adjustment payments

Health issues impacting patients with rare diseases. H.R.5247/S.204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Congressionally Directed Medical Research Program. Short-term, limited-duration health plans. Association health plans.

Pharmaceutical issues impacting patients with rare diseases. Orphan Drug Act. H.R.1223/S.1509: Orphan Products Extension Now Accelerating Cures and Treatments Act of 2017. H.R.5062: Advancing Access to Precision Medicine Act.

Bipartisan Health Care Stabilization Act Medicare Part D Right to Try Act; H.R.5247, S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health Funding Food and Drug Administration Funding Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Advancing Care for Exceptional Kids Act; S.428 Pediatric Device Consortia Grant Program Congressionally Directed Medical Research Program Rare Disease Day Resolution; S.Res.423 Short-term, limited-duration health plans Association health plans OPEN Act; H.R.1223, S.1509 Advancing Access to Precision Medicine Act of 2018; H.R.5062 Restoring the Patient's Voice Act of 2017; H.R.2077

Bipartisan Health Care Stabilization Act Medicare Part D Right to Try Act; H.R.5247, S.204 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health Funding Food and Drug Administration Funding Orphan Drug Act Medical Nutrition Equity Act; H.R.2587, S.1194 Advancing Care for Exceptional Kids Act; S.428 Pediatric Device Consortia Grant Program Congressionally Directed Medical Research Program Rare Disease Day Resolution; S.Res.423 Short-term, limited-duration health plans Association health plans OPEN Act; H.R.1223, S.1509 Advancing Access to Precision Medicine Act of 2018; H.R.5062 Restoring the Patient's Voice Act of 2017; H.R.2077

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Copay accumulator programs. Slowly progressive low-prevalence rare diseases. S.3388: Ensuring Coverage for Patients with Pre-Existing Conditions Act. Pediatric subspecialty loan repayment reauthorization. Medical expense deduction. Humanitarian Device Exemption (HDE) program. Risk adjustment payments.

Health issues impacting patients with rare diseases. H.R.2077: Restoring the Patient's Voice Act of 2017. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. Short-term, limited-duration health plans. Association health plans. Pediatric subspecialty loan repayment reauthorization. Human gene therapy for rare diseases. Newborn Screening Saves Lives Act reauthorization. Neurological Conditions Surveillance System funding. H.R.7102: ENROLL Act of 2018.

Health issues impacting patients with rare diseases. Bipartisan Health Care Stabilization Act. H.R.5247/S.204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018. H.R.5115: Rare Disease Advancement, Research, and Education (RARE) Act of 2018. H.R.2587/S.1194: S.1194 - Medical Nutrition Equity Act of 2017. S.428: Advancing Care for Exceptional Kids Act of 2017 or the ACE Kids Act of 2017. Congressionally Directed Medical Research Program. S.Res.423: A resolution designating February 28, 2018, as "Rare Disease Day". Short-term, limited-duration health plans. Association health plans. H.R.2077: Restoring the Patient's Voice Act of 2017.

Medicare Part D Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Senator Cassidy's "Ideas to Make Health Care Affordable Again" white paper Medical Nutrition Equity Act; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Medicaid waivers Section 1332 waivers Copay accumulator programs Patient-focused drug development Biological products marketplace Slowly progressive low-prevalence rare diseases HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Ensuring Coverage for Patients with Pre-Existing Conditions Act; S.3388 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Medical expense deduction Humanitarian Device Exemption (HDE) program Risk adjustment payments

Medicare Part D Affirming the importance of the Orphan Drug Act, applauding its lifesaving contributions over its 35-year history, and recognizing the need to continue supporting research and development for rare diseases; H.Res.1154 A resolution affirming the importance of the Orphan Drug Act, applauding the life-saving accomplishments of the Act during its 35-year history, and recognizing the need to continue support for research and development of new therapies for rare diseases; S.Res.690 Step therapy in Medicare Advantage plans Restoring the Patient's Voice Act of 2017; H.R.2077 Rare disease Advancement, Research, and Education Act of 2018; H.R.5115 National Institutes of Health funding Food and Drug Administration appropriations Orphan Drug Act Medical Nutrition Equity Act of 2017; H.R.2587, S.1194 Short-term, limited-duration health plans Association health plans Section 1115 waivers Section 1332 waivers Copay accumulator programs FY19 Labor, Health and Human Services, Education/Defense appropriations conference report; H.R.6157 Medicaid expansion Pediatric subspecialty loan repayment reauthorization Patient-focused drug development Human gene therapy for rare diseases Rare disease early development and the role of pre-investigational new drug application meetings International pricing index model for Medicare part B drugs Newborn Screening Saves Lives Act reauthorization Neurological Conditions Surveillance System funding MODERN Labeling Act of 2018; S.3519 ENROLL Act of 2018; H.R.7102