Current Initiatives & Issues Updates

NORD’s policy initiatives and alliances are conducted out of the Washington DC office.

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.

NORD Issues Updates

NORD closely follows and comments on legislation and policies that affect members of the rare disease community. The following Issues Updates are meant to provide a summary and NORD’s position on some of these issues.

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

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

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

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

Disability Insurance Waiting Period

Currently there exists a five month waiting period before anyone can receive disability assistance from the Social Security Administration, regardless of the cause of the disability.  For disabled individuals that do not have a medically valid reason to expect that their disability will improve, these five months are little more than a barrier to access. Read more

Reauthorization of the Prescription Drug User Fee Act (PDUFA V)

For the FDA, the Prescription Drug User Fee Act is one of the most important pieces of upcoming legislation.  PDUFA is considered a 'must-pass' bill and is renewed by Congress every 5 years. NORD has been working closely with FDA to address some of the special needs of the rare disease community as they relate to PDUFA. Read more

Reauthorization of the Medical Device User Fee Act (MDUFA III)

Similar to PDUFA V, the Medical Device User Fee Act authorizes the FDA to collect user fees from medical device makers to accelerate the review of their products and determine if they are safe and effective.  Like PDUFA, MDUFA is reauthorized on a regular basis by Congress and is an important part of ensuring that FDA has the resources it needs to fulfill its mission. 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