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Current Initiatives & Issues Updates

Visit this page to learn what is going on in rare disease legislation.  NORD provides advocacy on behalf of the entire rare disease community.  Read key testimony, letters, and position papers here.

NORD’s policy initiatives and alliances are conducted out of the Washington, D.C. office.


Below are policy and advocacy initiatives NORD is working on to help improve the lives of rare disease patients and their families.  Be heard and join us in making a difference.  We update this page frequently so please check back for updates.


Send a letter to your Congressperson in support of the Ensuring Access to Clinical Trials Act by clicking here.
In 2010, Congress passed the Improving Access to Clinical Trials Act, a law that allows patients with rare diseases to exempt up to $2,000 in compensation received for participating in clinical trials from counting towards their income eligibility for vital health benefits, including SSI and Medicaid. The Ensuring Access to Clinical Trials Act of 2015 (S.139 and H.R.209) would make this program permanent. It is currently set to sunset if it is not renewed this Congress. Eliminating all disincentives for rare disease patients to participate in clinical trials is paramount in ensuring that therapies reach the millions of rare disease patients that currently do not have an FDA-approved treatment. Ensuring patients are not penalized for participating in clinical trials is integral to their continued success.


Contact your senators and representatives (202-224-3121) and ask them to support H.R. 1600 from Reps. McKinley and Capps. This forbids the use of cost sharing on the highest prescription drug tier (usually the specialty tier) to surpass 110 percent of the next lowest tier. A Senate companion is to be introduced. Read more


Contact your senators (202-224-3121) and ask them to sign on to Senator Bob Casey (D-PA) and Senator Richard Burr's (R-NC) "Dear Colleague" letter that supports appropriate NIH funding for 2016. The NIH has been underfunded for the past 10 years, receiving small funding increases resulting in a loss of 21 percent of its purchasing power.


NORD Issues Updates

NORD closely follows and comments on legislation and policies that affect members of the rare disease community. The following issues updates (listed in alphabetical order) are meant to provide a summary and NORD’s position on some of these issues.

21st Century Cures

The 21st Century Cures initiative was launched in 2014 by the Energy & Commerce Committee of the U.S. House of Representatives. The purpose is to promote medical innovation and advance the discovery, development, and delivery of medical therapies and cures. Read more.

Advancing Care for Exceptional Kids Act of 2015 (ACE Kids Act)

The Advancing Care for Exceptional Kids Act of 2015 (ACE Kids Act) introduced by Sens. Charles Grassley (R-IA) and Michael Bennet (D-CO) (S. 298) and Reps. Joe Barton (R-TX) and Kathy Castor (D-FL)(H.R. 546) allows states to coordinate within their Medicaid programs for children with "medically complex conditions." Once enacted into law, it will improve coordination of care for children to reduce the burden on families; address problems with fragmented care across state lines; gather national data on complex conditions to help researchers improve treatments for rare diseases; potentially save an estimated $13 billion over its first 10 years, compared to the current system.

Children's Health Insurance (CHIP) Funding

NORD and 1,000+ organizations signed on to support a 4-year funding extension for the Children’s Health Insurance Program (CHIP). CHIP has played a critical role in reducing the number of uninsured children by more than 50 percent. If Congress fails to act now, over 8 million children will be at risk of having their health coverage disrupted.

Compassionate Allowances

When a US citizen applies for assistance from the Social Security Administration for a disability, they must prove that their disability will affect them long-term and that it is serious enough to prevent 'gainful employment.'  If their disability is caused by a particular disease, as is the case for many with rare diseases, the process may be expedited if the disease has been pre-approved for a 'compassionate allowance'.  NORD is working with SSA to expand the number of rare diseases that qualify for the Compassionate Allowances program. Read more

FDA Safety Over Sequestration Act (H.R. 1078)

Put forth by Rep. Lance, this is critical to ensure the expedient FDA review processes.  H.R. 1078 will exempt FDA user fees from sequestration.  User fees are used specifically for the approval of safe and effective drugs and devices. Unless Congress acts, sequestration will be back in 2016.  This will amend the Balanced Budget and Emergency Deficit Control Act of 1985 to exempt certain FDA user fees from sequestration.

Humanitarian Use Devices

Humanitarian Use Devices are similar to orphan drugs in that they are medical devices designed with small patient populations in mind.  NORD is looking to address some of the reasons why there aren't more humanitarian use devices in development because of the potential impact they may have in the lives of individuals with rare diseases. Read more

Medical Foods

Medical food is an essential to care for many people with rare diseases, particularly those with 'inborn errors of metabolism, but also others.  However, despite the necessity of medical foods for a number of rare diseases, many health benefits plans do not cover them.  In fact, there is a broad disparity between medical foods and other therapies within the context of the overall health care system. Read more

Off-Label Pharmaceutical Use

Because so few therapies exist that are specifically designed to treat rare diseases, many individuals with rare diseases have to make do with other therapies.  This is called 'off-label' use because the disease treated is not the disease that the therapy was designed for.  NORD is looking to address the barriers of access that people with rare diseases face when their physician recommends that they use a therapy off-label. Read more

Patient-Centered Outcomes Research Institute (PCORI)

One of the most promising new initiatives to come about from health reform was the consensus that public health is improved by knowing definitively what kind of care works and doesn't work in a given situation. The Patient-Centered Outcomes Research Institute will begin to systematically tackle questions related to standards of clinical care and will have an important impact on healthcare delivery in the United States, including for rare diseases. Read more

Rare Disease Congressional Caucus

This Caucus was established in 2010 to create a forum for members of Congress to help improve the lives of individuals with rare diseases. Read more.

Rare Disease Pediatric Review Voucher Program (PRV)

NORD has led the charge to renew the PRV with one of our most successful sign-on campaigns to date, having signed on 115 patient organizations. The program is designed to spur innovation in the rare pediatric disease drug development arena.  It is currently set to expire in March 2016 unless Congress acts. PRV was established under the Creating Hope Act of 2011 and was later included in the successfully enacted Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012. This program allows biopharmaceutical manufacturers to receive a voucher guaranteeing a 6-month priority review of another product, rare disease or not. The voucher can be sold to another company, and there is no limit on how many times it can be transferred. This voucher is a powerful economic incentive for pharmaceutical companies.  But if Congress does not permanently reauthorize the program, this crucial incentive will disappear. Read the Senate version of our sign on letter

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