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.

CMS Rule CMS-1711-P relating to Medicare and Medicaid Programs, "CY 2020 Home Health Prospective Payment System Rate Update, Home Health Value-Based Purchasing Model, Home Health Quality Reporting Requirements, and Home Infusion Therapy Requirements," all provisions. H Res 242 "Affirming the importance of the Orphan Drug Act," all provisions. HR 748 / S 3548 "CARES Act," all provisions pertaining to corporate tax, and CMS home infusion benefits. HR 19 "Lower Costs, More Cures Act of 2019," all provisions. HR 2113 Prescription Drug STAR Act, all provisions. HR 2296 More Efficient Tools to Realize Information for Consumers Act, all provisions. HR 3 "Elijah E. Cummings Lower Drug Costs Now Act," all provisions. Issues related to the implementation of PL 111-148 "Patient Protection and Affordable Care Act," all provisions pertaining to the Center for Medicare and Medicaid Innovation (CMMI) and federal programs. Issues related to the implementation of PL 97-414 "Orphan Drug Act," all provisions. Importation of Prescription Drugs Proposed Rule, Food and Drug Administration (FDA), [Docket No. FDA-2019-N-5711]. Issues relating to improved oversight regulatory requirements for 340B hospital reporting, excluding Federal Grantees. S 1391 "Fair Accountability and Innovative Research Drug Pricing Act of 2019, all provisions. S 474 Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019, all provisions. Issues relating to creating a cystinosis disease awareness day on May 7th. S 2543 Prescription Drug Pricing Reduction Act of 2019, all provisions in Amendment #4 Improving Metrics for Price Reporting for Orphan Drugs Act. CMS Rule CMS-5528-ANPRM relating to Medicare Program, "International Pricing Index Model for Medicare Part B Drugs," all provisions. S 1895 Lower Health Care Costs Act, all provisions.

Client expense: $1,280,000 · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), Executive Office of the President (EOP), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE, Treasury, Dept of

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; Newborn Screening legislation in general; Newborn Screening Saves Lives Reauthorization Act of 2019, H.R. 2507; Lower Health Care Costs Act, S. 1895; H.R. 4144, Ending the Diagnostic Odyssey Act of 2019; H.R. 534, Aidan's Law (newborn screening for ALD); H.R. 5062, Advancing Access to Precision Medicine Act; Kidney disease diagnosis, prevention, and care; H.R. 4712, Fairness in Orphan Drug Exclusivity Act.

Client expense: Not reported · Firm income: $60,000

Regulation CMS-1711-P "CY 2020 Home Health Prospective Payment System Rate Update, Home Health Value-Based Purchasing Model, Home Health Quality Reporting Requirements, and Home Infusion Therapy Requirements," all provisions. Issues related to the implementation of PL 111-148 "Patient Protection and Affordable Care Act," all provisions pertaining to the Center for Medicare and Medicaid Innovation (CMMI) and federal programs. Issues related to drug pricing transparency. HR 2296 FAIR Drug Pricing Act of 2019, all provisions. S 4743 Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019, all provisions. HR 2113 Prescription Drug STAR Act, all provisions. HR 2296 More Efficient Tools to Realize Information for Consumers (METRIC) Act, all provisions. Issues related to improved oversight regulatory requirements for 340B hospital reporting, excluding Federal Grantees. Issues related to the implementation of PL 97-414 "Orphan Drug Act," all provisions. H Res 242 Orphan Drug Act, all provisions. HR 1503 "Orange Book Transparency Act of 2019," provisions in Section 2 relating to the Orange Book. HR 107 To amend title XIX of Social Security Act, all provisions. Issues related to creating a cystinosis disease awareness day on May 7th. Office of the Inspector General (HHS) Rule OIG-0936-P relating to Fraud and Abuse, 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, all provisions. CMS Rule CMS-5528-ANPRM relating to Medicare Program, International Pricing Index Model for Medicare Part B Drugs, all provisions. Issues related to importation of pharmaceuticals. S 2543 Prescription Drug Pricing Reduction Act of 2019, all provisions in Amendment #4 Improving Metrics for Price Reporting for Orphan Drugs Act. HR 3 "Lower Drug Costs Now Act of 2019," all provisions. Issues related to the implementation of PL 115-97 (2017) "Tax Cuts and Jobs Act, all provisions. S 1895 Lower Health Care Costs Act, all provisions. HR 259 Medicaid Extenders Act of 2019, all provisions. HR 19 Lower Costs, More Cures Act of 2019, all provisions.

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; H.R. 2507, S. 2158 Newborn Screening Saves Lives Act Reauthorization; HR 4144, Ending the Diagnostic Odyssey Act; H.R. 5062, Advancing Access to Precision Medicine Act; H.R. 4712, Fairness in Orphan Drug Exclusivity Act

Client expense: $170,000 · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; Newborn Screening legislation in general; Newborn Screening Saves Lives Reauthorization Act of 2019, H.R. 2507; Lower Health Care Costs Act, S. 1895; H.R. 4144, Ending the Diagnostic Odyssey Act of 2019.

Client expense: Not reported · Firm income: $60,000

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

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.

Client expense: $60,000 · Firm income: Not reported

Regulation CMS-1711-P "CY 2020 Home Health Prospective Payment System Rate Update; Home Health Value-Based Purchasing Model; Home Health Quality Reporting Requirements; and Home Infusion Therapy Requirements," all provisions. Issues related to the implementation of PL 111-148 "Patient Protection and Affordable Care Act," all provisions pertaining to the Center for Medicare and Medicaid Innovation (CMMI) and federal programs. Issues related to drug pricing transparency. HR 2296 FAIR Drug Pricing Act of 2019, all provisions. S 4743 Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019, all provisions. HR 2113 Prescription Drug STAR Act, all provisions. HR 2296 More Efficient Tools to Realize Information for Consumers (METRIC) Act, all provisions. Issues related to improved oversight regulatory requirements for 340B hospital reporting, excluding Federal Grantees. Issues related to the implementation of PL 97-414 "Orphan Drug Act," all provisions. H Res 242 Orphan Drug Act, all provisions. HR 1503 "Orange Book Transparency Act of 2019," provisions in Section 2 relating to the Orange Book. HR 107 To amend title XIX of Social Security Act, all provisions. Issues related to creating a cystinosis disease awareness day on May 7th. Office of the Inspector General (HHS) Rule OIG-0936-P, all provisions pertaining to Fraud and Abuse; Issues related to the 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. CMS Rule CMS-5528-ANPRM relating to Medicare Program, all provisions. Issues related to an International Pricing Index Model for Medicare Part B Drugs. Issues related to importation of pharmaceuticals. S 102 " Prescription Drug Price Relief Act of 2019," all provisions. HR 3 " Lower Drug Costs Now Act of 2019," all provisions.

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; H.R. 2507, S. 2158 Newborn Screening Saves Lives Act Reauthorization; HR 4144, Ending the Diagnostic Odyssey Act

Client expense: $180,000 · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

Issues related to the Orphan Drug Act H Res 242 "Affirming the importance of the Orphan Drug Act" The SPIKE Act; Prescription Drug Pricing Classification

Client expense: Not reported · Firm income: $10,000

Government targets: HOUSE OF REPRESENTATIVES, SENATE

Issues related to the Orphan Drug Act H Res 242 "Affirming the importance of the Orphan Drug Act" The SPIKE Act; Prescription Drug Pricing Classification

Client expense: Not reported · Firm income: $90,000

Government targets: HOUSE OF REPRESENTATIVES, SENATE

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.

Client expense: $90,000 · Firm income: Not reported

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; Newborn Screening legislation in general; Newborn Screening Saves Lives Reauthorization Act of 2019, H.R. 2507; Lower Health Care Costs Act, S. 1895.

Client expense: Not reported · Firm income: $60,000

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

Issues related to the implementation of PL 111-148 "Patient Protection and Affordable Care Act," all provisions pertaining to the Center for Medicare and Medicaid Innovation (CMMI) and federal programs. Issues related to drug pricing transparency. HR 2296 FAIR Drug Pricing Act of 2019, all provisions. S 4743 Stopping the Pharmaceutical Industry from Keeping drugs Expensive (SPIKE) Act of 2019, all provisions. HR 2113 Prescription Drug STAR Act, all provisions. HR 2296 More Efficient Tools to Realize Information for Consumers (METRIC) Act, all provisions. Issues pertaining to improved oversight regulatory requirements for 340B hospital reporting, excluding Federal Grantees. Issues related to the implementation of PL 97-414 "Orphan Drug Act," all provisions. H Res 242 Orphan Drug Act, all provisions. HR 1503 "Orange Book Transparency Act of 2019," provisions in Section 2 relating to the Orange Book. HR 107 To amend title XIX of Social Security Act, all provisions. Issues pertaining to creating a cystinosis disease awareness day on May 7th. Office of the Inspector General (HHS) Rule OIG-0936-P relating to Fraud and Abuse; 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. CMS Rule CMS-5528-ANPRM relating to Medicare Program; International Pricing Index Model for Medicare Part B Drugs. Issues pertaining to importation of pharmaceuticals.

Client expense: $680,000 · Firm income: Not reported

Issues related to the Orphan Drug Act H Res 242 "Affirming the importance of the Orphan Drug Act" The SPIKE Act; Prescription Drug Pricing Classification

Client expense: Not reported · Firm income: Not reported

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act; H.R. 2507, Newborn Screening Saves Lives Act Reauthorization

Client expense: $270,000 · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

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.

Client expense: $100,000 · Firm income: Not reported

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act.

Client expense: Not reported · Firm income: $60,000

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE

Issues related to the implementation of PL 111-148 "Patient Protection and Affordable Care Act," all provisions pertaining to the Center for Medicare and Medicaid Innovation (CMMI) and federal programs. Issues related to the implementation of PL 97-414 "Orphan Drug Act," all provisions. HR 1503 "Orange Book Transparency Act of 2019," all provisions. 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," all provisions.

Client expense: $1,380,000 · Firm income: Not reported

Government targets: HOUSE OF REPRESENTATIVES, Office of Policy Development, SENATE

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 DISEASE; ALL PROVISIONS OF THE RESOLUTION H.R. 1046: MEDICARE NEGOTIATION AND COMPETITIVE LICENSING ACT OF 2019; ALL PROVISION OF THE BILL S. 377: MEDICARE NEGOTIATION AND COMPETITIVE LICENSING ACT OF 2019; ALL PROVISION OF THE BILL GENERAL DISCUSSIONS REGARDING GENE THERAPY AND VALUE BASED CONTRACTING GENERAL DISCUSSIONS REGARDING NEWBORN SCREENING RE-AUTHORIZATION ACT GENERAL DISCUSSIONS REGARDING THE ORPHAN DRUG ACT GENERAL DISCUSSIONS REGARDING HEALTH CARE FINANCING

Client expense: $410,000 · Firm income: Not reported

Government targets: HOUSE OF REPRESENTATIVES, SENATE

Rare disease patient care, screening, and coverage issues in authorization and appropriations legislation; Orphan Drug Act; HRes 242, Affirming importance of Orphan Drug Act

Client expense: $280,000 · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), SENATE