RARE Act of 2018

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

Client expense: Not reported · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), Food & Drug Administration (FDA), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), Natl Institutes of Health (NIH), SENATE

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

Client expense: Not reported · Firm income: Not reported

Promote federal funding for mitochondrial disease research, FY19 Defense Appropriations Act (Title V - Health); National Institutes of Health funding for mitochondrial research; H.R. 5062, Advancing Access to Precision Medicine Act; H.R. 5115, RARE Act of 2018.

Client expense: Not reported · Firm income: $20,000

Government targets: HOUSE OF REPRESENTATIVES, SENATE

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

Client expense: Not reported · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), Food & Drug Administration (FDA), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), Natl Institutes of Health (NIH), SENATE

H.R. 5247: TRICKETT WENDLER, FRANK MONGIELLO, JORDAN MCLINN, AND MATTHEW BELLINA RIGHT TO TRY ACT OF 2018; ALL PROVISIONS OF THE BILL H.R. 5115: RARE dISEASE ADVANCEMENT, RESEARCH, AND EDUCATION (RARE) ACT OF 2018; ALL PROVISIONS OF THE BILL S. 204: TRICKETT WENDLER, FRANK MONGIELLO, JORDAN MCLINN, AND MATTHEW BELLINA RIGHT TO TRY ACT OF 2017; ALL PROVISIONS OF THE BILL GENERAL DISCUSSIONS REGARDING PRESERVATION OF ORPHAN DRUG ACT GENERAL DISCUSSIONS REGARDING FOOD AND DRUG ADMINISTRATION DISEASE EDUCATION GENERAL DISCUSSIONS REGARDING GENE THERAPY, RARE BRAIN DISORDER, FOOD AND DRUG ADMINISTRATION ADVISORY COMMITTEE MEETINGS AND PRESCRIPTION DRUG COST

Client expense: $110,000 · Firm income: Not reported

Government targets: HOUSE OF REPRESENTATIVES, SENATE

Promote federal funding for mitochondrial disease research, FY19 Defense Appropriations Act (Title V - Health); National Institutes of Health funding for mitochondrial research; H.R. 5062, Advancing Access to Precision Medicine Act; H.R. 5115, RARE Act of 2018.

Client expense: Not reported · Firm income: $20,000

Government targets: HOUSE OF REPRESENTATIVES, SENATE

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

Client expense: Not reported · Firm income: Not reported

Government targets: Centers For Medicare and Medicaid Services (CMS), Food & Drug Administration (FDA), HOUSE OF REPRESENTATIVES, Health & Human Services, Dept of (HHS), Natl Institutes of Health (NIH), SENATE