Overview of Legislation Benefiting the Rare Disease Community

Passed in the 113th Congress:

  • Gabriella Miller Kids First Research Act: Redirects $12.6 Million in funding from political conventions to the National Institutes of Health (NIH) Common Fund to support transformative research for childhood cancer, autism, Fragile X syndrome and other rare pediatric diseases. With the odds stacked against us, more than 70 organizations called on Congress and ensured this essential funding was in the 2015 omnibus appropriations bill.

  • National Pediatric Research Network Act: Authorizes the National Institutes of Health (NIH) to establish a network to provide support for research and training at up to 20 pediatric research consortia. Requires the Director of NIH to establish a data coordinating center for the consortia.
  • Achieving a Better Life Experience (ABLE) Act: Enables individuals with disabilities and families, to save for the future without jeopardizing necessary healthcare benefits by creating a tax-free savings account.  With unprecedented support, this bill passed the House 404 to 17 and the Senate 76 to 16.
  • Newborn Screening Saves Lives Reauthorization Act: Reauthorizes critical federal activities that assist states in improving and expanding newborn screening programs, supporting parent and provider newborn screening education, and ensuring laboratory quality and surveillance.
  • MD-CARE Act: Reauthorizes the Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act and expands the range of forms of muscular dystrophy included within the muscular dystrophy research program of the National Institutes of Health (NIH). Requires the research conducted to include cardiac and pulmonary function research and ensures the sharing of data between such centers.

Legislation that gained support in the 113th Congress:

  • Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network (MODDERN) CURES Act: The language to improve access to diagnostics and spur diagnostic innovation was included in Medicare Sustainable Growth (SGR) reform, passed by Congress earlier this year.  The dormant patent provision is designed to encourage the development of drugs to treat conditions with few or no treatment options and has gained significant support, leading to a companion bill in the Senate and potential for the provision to be included in the broader 21st Century Cures legislation.
  • Patients’ Access to Treatments Act: Prohibits health insurance plans from imposing cost-sharing requirements, including co-payment and co-insurance, applicable to prescription drugs in a specialty drug tier. This bill did not pass this Congress but gained significant support from both patient groups, industry and legislators.
  • FDA Safety Over Sequestration Act (FDA S.O.S.):  Amends the Balanced Budget and Emergency Deficit Control Act to exempt the user fees paid by industry from sequestration – mandatory across-the-board spending cuts. Support of this legislation enables Congress to ensure the FDA receives full revenues from user fees.  The legislation needs to pass to ensure the exemption becomes permanent.
  • Charles August Long Undiagnosed Diseases Research and Collaboration Network Act: Establishes a physician network to help individuals and military Service Members and Veterans who have unexplained symptoms and medical problems by establishing an undiagnosed diseases registry.  Representative Carter (R-TX) is committed to reintroducing this legislation early next year.

New Legislation:

  • Orphan Product Extensions Now Accelerating Cures & Treatments (OPEN ACT HR 5750): Incentivizes drug makers to “repurpose” major market drugs for rare diseases and pediatric cancers, which opens the door to the development of hundreds of safe, effective, and affordable treatments for rare disease patients. The Foundation was pleased to see our legislative proposal introduced this Congress by Rep. Bilirakis (R-FL) and backed by four bipartisan co-sponsors including Reps Butterfield (D-SC), McCaul (R-TX), Hastings (D-FL), and Quigley (D-IL).
  • Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act: Seeks to improve patient access to experimental drugs by clarifying FDA and industry policyRequires companies to establish Expanded Access policies for unapproved drugs and requires the FDA to finalize their guidance.