Rare Disease Report

Update on Rare Disease Policy Priorities for 2015

OCTOBER 10, 2015
Guest post by NORD
In partnership with the Cystic Fibrosis Foundation (CFF) and Muscular Dystrophy Association (MDA), NORD and its members led the charge in support of passage of the Ensuring Access to Clinical Trials Act (S.139/H.R.209), which was approved by the Senate in July and by the House on September 28. The bill was signed by President Obama on October 7.

This new law is extremely important for the rare disease community because it eliminates a barrier to clinical trial participation for rare disease patients. Specifically, the bill permanently reauthorizes a policy enacted in 2010 that allows patients to exempt up to $2,000 earned for participating in rare disease clinical trials from counting against their Medicaid and SSI income eligibility.

NORD and its advocacy partners presented the case that recruitment for rare disease clinical trials is challenging because of the small patient populations. At the same time, rare disease patients are highly motivated to participate in research because they have serious diseases with few or no treatment options.

Patients should not be at risk of losing their benefits and health insurance simply because they made a choice to participate in a rare disease clinical trial, NORD and its partners contended, and Congress agreed.

The 2010 legislation would have expired on Oct. 5, 2015, so it was extremely important for this issue to be addressed during this session of Congress. NORD and its members are grateful to all who supported NORD, CFF and MDA in this successful advocacy effort!

A Sampling of Other NORD Policy Objectives at This Time

The following is a brief sampling of other objectives of the NORD policy team at this time. To join NORD in advocacy, read about the Rare Action Network™ and sign up to join the network today.

Representing Rare Disease Patients in PDUFA/MDUFA Reauthorization Negotiations

The processes for reauthorizing the Prescription Drug User Fee Act (PDUFA) and the Medical Device User Fee Amendments (MDUFA) both officially began this summer with the FDA holding PDUFA and MDUFA Reauthorization Stakeholder Meetings. NORD was proud to present and serve on the patient panels at both stakeholder meetings. NORD’s testimony at the PDUFA meeting can be found here, and at the MDUFA meeting here.

NORD will continue to represent the rare disease patient in PDUFA and MDUFA discussions throughout the fall. This will include participating in monthly patient stakeholder meetings convened by FDA, as well as continuing discussions with NORD member organizations and other rare disease stakeholders to assure strong representation in this important process.

Ensuring that Rare Disease Policy Reforms Incorporated into the 21st Century Cures Act are also Included in the Senate’s Innovation for Healthier Americans Initiative

NORD assisted the House Energy and Commerce Committee throughout the 21st Century Cures Initiative by providing input from the patient perspective that ultimately was included in the final 21st Century Cures Act. Among these are the OPEN Act, a reauthorization of the Rare Pediatric Disease Priority Review Voucher program, the establishment of a public-private partnership to develop natural history data patient registries, and more. Also included is a critically important mandatory spending increase of $8.75 billion over five years for the National Institutes of Health (NIH).

NORD will be encouraging the Senate Health, Education, Labor and Pensions (HELP) Committee to include these important provisions in the Senate’s Innovation for Healthier Americans initiative. NORD has discussed various provisions with the HELP Committee this summer and will continue to advocate for their inclusion this fall.

This will also include advocating for some additional policy reforms to accelerate the pace of discovery, development and delivery of treatments for rare diseases.

Ensuring that NIH and FDA Remain Strong and Well-Funded

NORD is proud to be a founding member of the Alliance for a Stronger FDA, and it works with the Alliance in support of funding to keep this agency, which regulates products that represent approximately 1 of every 4 dollars of consumer spending, strong.

Similarly, NORD supported the Rally for Medical Research in Washington DC on September 16-17, and with its members continues to provide strong advocacy on behalf of NIH funding.  

Increasing State Policy Activity

Through its Rare Action Network, NORD is organizing state coalitions that are providing advocacy for rare disease patients at the state level. Currently, there are grassroots coalitions in 10 states: CT, FL, GA, MA, MI, OK, MA, PA, UT and TX. Additional coalitions will be joining the network over the next several months.

State-related policy has focused on issues such as “cap the co-pay” legislation, opposing unnecessary step-therapy requirements. NORD has also been evaluating state policies with respect to insurance coverage for medical foods, newborn screening, prescription drug cost-sharing and Medicaid eligibility.

Preparing for Rare Disease Day 2016

NORD will again this year host Rare Disease Day State House Events, and the policy agenda for RDD 2016 is being developed now, consistent with the findings of the evaluation of state policies mentioned above.

Watch for the launch of the redesigned national Rare Disease Day website hosted by NORD in November 2015!

Image 'Freedom of Speech' by Normal Rockwell courtesy of wikimedia commons.

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