H.R. 6/ H.R. 34 The 21st Century Cures Act (H.R. 34) was signed into law by President Obama in December 2016. It provides $1.75 billion in additional annual funding of for the National Institutes of Health (NIH) and $110 million in new annual funding for the Food and Drug Administration (FDA). The legislation also contains provisions to enhance patient engagement in the drug development process, expand biomarker qualification, and improve workforce specialization, hiring and retention at the FDA. A slightly different version of the 21st Century Cures Act (H.R. 6) passed the House of Representatives in July 2015.

H.R. 971 / S. 1421 The Orphan Product Extensions Now, Accelerating Cures and Treatments (OPEN ACT) would incentivize the repurposing of potentially life-saving drugs for rare diseases and pediatric cancers. The legislation would establish a six-month marketing exclusivity extension for an approved drug or biological product when the repurposed therapy is approved to prevent, diagnose, or treat a new indication that is a rare disease. The OPEN ACT’s incentive structure is modeled on the Best Pharmaceuticals for Children Act (2002), which has led to more than 600 labeling changes for pediatric populations. The bill was passed by the House in 2015.

HR 1537 / S. 2030 The Advancing Hope Act was signed into law by President Obama in September 2016 and expands the priority review voucher program for rare pediatric diseases to include treatments for sickle cell disease and pediatric cancers. The priority review voucher provides an incentive for companies to develop treatments for rare, pediatric diseases. If a company successfully develops a drug in this class, it receives a voucher that can be used to expedite review for another drug at the FDA and may be redeemed or sold to another company.

H.R. 1608 The Lymphedema Treatment Act would designate certain lymphedema compression treatment items as durable medical equipment. The compression supplies used daily in lymphedema treatment are currently not covered by Medicare because they do not fit under any benefit category. The legislation would remedy the unintended gap in coverage and reduce total healthcare costs by decreasing the incidence of complications, co-morbidities and disabilities resulting from lymphedema.

H.R. 3381 / S. 1883 The Childhood Cancer STAR (Survivorship, Treatment, Access and Research) Act of 2016 would advance pediatric cancer research and child-focused cancer treatments, while also improving childhood cancer surveillance and providing resources for survivors and those impacted by childhood cancer. It would provide support to collect the medical specimens and information of children, adolescents and young adults with cancer to improve the understanding of these cancers and the effects of treatment. Funding for the national childhood cancer registry is reauthorized through FY2020 and is revised to require the Centers for Disease Control and Prevention (CDC) award grants to states to improve tracking of childhood cancers. The bill was passed by the House in 2015.

H.R. 209 / S. 139 The Ensuring Access to Clinical Trials Act of 2015 (EACT) was signed into law by President Obama in October 2015. The legislation maintains federal support for the participation of low-income Americans with rare diseases in clinical trials. The bill was accomplished through the renewal of the Improving Access to Clinical Trials Act (I-ACT) of 2009, which allows compensation for participation in clinical trials to those on Supplemental Security Income (SSI) and Medicaid. I-ACT directs that “the first $2,000 per year received by individuals who participate in clinical trials for rare diseases or conditions” are not counted against the individual’s eligibility for SSI and Medicaid benefits.

S. 1597 The Patient-Focused Impact Assessment Act of 2016 was signed into law by President Obama in December 2016 as a provision of the 21st Century Cures Act. The legislation enhances patient engagement in the medical product development process. The law enacts two targeted reforms to provide greater clarity and transparency to all stakeholders involved in development and to help demonstrate how the FDA is using the tools and authorities. The requirements include the development of a Patient-Focused Impact Assessment and issuance of Draft and Final Guidance to Patients and Industry on patient-focused drug development.

S. 2030 The Advancing Targeted Therapies for Rare Diseases Act of 2016 was signed into law by President Obama in December 2016 as a provision of the 21st Century Cures Act. It aims to help accelerate the development of targeted drugs to treat rare diseases, including Duchenne muscular dystrophy, cystic fibrosis, some cancers and other genetic diseases. The legislation allows innovators to use their personal data to support the approval of a targeted therapy and helps facilitate additional targeted therapies to treat patients with the same rare disease.

The bipartisan, bicameral Rare Disease Congressional Caucus is currently co-chaired by Representatives G.K. Butterfield (D-NC) and Leonard Lance (R-NJ) as well as Senators Orrin Hatch (R-UT) and Amy Klobuchar (D-MN). It works to advance legislation and education regarding specialized and uncommon health issues and diseases, while promoting patient advocacy. Rare Disease Legislative Advocates helps to coordinate quarterly Caucus briefings.