To Top

Dec. 10, 2015

TOPIC: Featured News, Advocacy, Get Involved

Be a Hero: Help NORD Empower the Rare Disease Community in Washington D.C. and in all 50 States.

Posted by Heidi Ross

2015 was a bellwether year for the NORD public policy team and some exciting growth was seen that could not have come at a better time; the next few years will be some of the most critical in rare disease public policy since the inception of the Orphan Drug Act in 1983.

Congress will consider legislation impacting all sectors of the health care system important to rare disease research and care; the FDA is seeking to increase the role of the patient voice in drug development; companies and researchers are looking for new ways to expand clinical trials to new disease; and states are taking up legislation that will decide what medical services should be covered by insurance and how much it will cost.

What’s Ahead for Rare Disease Policy at NORD:

Strengthening the Voice of the Rare Disease Community in Washington D.C.

The policy team represented the rare disease community on a variety of federal legislative and regulatory initiatives in 2015.

Our year began with work on the 21st Century Cures Act, which will provide critical reforms to the drug research and development process, such as ensuring greater patient access and participation in the FDA approval process, maintaining critical incentives for orphan drug development, and providing additional funding for research to the NIH. In July, 21st Century Cures passed out of the House of Representatives and is now under consideration in the Senate.   2016 will be a make or break year for this legislation.

In the summer, NORD began consulting with legislators on bills to reauthorize funding for the FDA (the Prescription Drug User Fee Act [PDUFA] and the Medical Device User Fee Act [MDUFA]). Both of these bills are likely to be considered in 2016.

As summer turned to fall, NORD, in collaboration with the Cystic Fibrosis Foundation, the Muscular Dystrophy Association, and many of our members successfully enacted the Ensuring Access to Clinical Trials Act. This law ensures that stipends for clinical trials do not count against participants’ enrollment in Medicaid or SSI.

NORD also helped spearhead the development of the Advancing Targeted Therapies for Rare Disease Act, which removes barriers in conducting clinical trials for certain genetic subgroups of rare diseases.  NORD coordinated a coalition letter of more than 100 rare disease groups in support of this bill.

Heading into 2016, the federal policy team will continue work on these important initiatives. In addition, NORD will be at the forefront of potential legislative efforts to amend the Orphan Drug Act.

Empowering the Rare Disease Community in Every State

2015 saw the launch of NORD’s Rare Action Network, a nationwide grassroots advocacy initiative that works with advocates to address state policies concerning rare diseases. Over the summer, Rare Action had its first ever “Road Tour” when NORD policy staff drove from Washington D.C. to Seattle to meet with advocates and legislators in 10 different states.

In October, NORD launched its first ever State Policy Progress Report at the NORD Summit in Washington D.C.  This report is the first comprehensive analysis of state policies affecting the rare disease community. With input from members and partners, our goal is to expand the report and publish a new version every year.

Consistent with the goals of the State Policy Progress report and Rare Action, by the end of the year, NORD drastically increased its involvement on state legislation. This year alone, NORD has become directly involved in the following legislation:

  • California: NORD supported SB 671, a bill to ensure prescriber communication on the dispensing of biosimilars.
  • Connecticut (HB 6580): Earlier this year, NORD helped advise on the passage of HB 6580, which creates a rare disease legislative task force. We are now in the process of helping recruit members for the task force and its first meeting should be by mid-January.
  • Illinois: NORD supported SB 455, a bill to ensure prescriber communication on the dispensing of biosimilars.
  • Massachusetts (SB 541): NORD is supporting the passage of SB 541, which would cap the amount of cost sharing a patient has to pay for their medication. This bill will be a major focus for Rare Action in 2016.
  • Pennsylvania (SB 841). NORD also supported a prescription cost sharing bill in PA. Separately, NORD is supporting local organizations in the creation of a rare disease caucus in the PA legislature.
  • North Carolina: Earlier this year North Carolina created its first-ever Rare Disease Advisory Council. NORD actively collaborated with local advocates on helping pass the legislation. The advisory council is being hosted by UNC-chapel hill. In November, the council held its first meeting and invited NORD to present on the findings of the State Policy Progress Report.

In 2016, the state policy team hopes to push its impact into more states. This process will begin with Rare Disease Day 2016 during which NORD and many of our state advocacy coalitions will be introducing new legislative initiatives to improve quality and access to care.

Thanks to the support of our partners and donors, we have launched new initiatives that empower the rare disease community in every state and strengthen the patient voice in Washington D.C.  Thank you for your continued support of NORD.


Your donations will help NORD to build upon the legislative activities on a state and federal level and greatly strengthen the voice of the rare disease community.

To make a donation please click here.