NORD urges FDA to reduce regulatory uncertainty in developing medicines for rare disorders

Noting that only about 200 of the nearly 7,000 diseases classified as rare currently have treatments, the National Organization for Rare Disorders (NORD) today called for a Food and Drug Administration (FDA) statement of policy on regulation of therapies for rare disorders.

NORD also urged FDA to work with the research community to reduce regulatory uncertainty in the development of medicines for rare disorders.

"There are still about 5,800 rare disorders for which there are no FDA-approved therapies," said Frank J. Sasinowski, chairman of the NORD Board of Directors, in the opening address at a two-day public hearing sponsored by FDA.  "Perhaps most discouraging is that many affected with these rare disorders do not even see any research being conducted on their conditions."

Referring to the Orphan Drug Act of 1983, Sasinowski said that more than 350 new treatments for rare diseases have been approved by FDA since that law was enacted.  This is good progress, he said.  

"But to NORD and the patient community," he added, "it seems as if the low-hanging fruit have been harvested.  While much has been accomplished, much more remains to be done."

Sasinowski said that NORD recently hosted focus groups for four categories of stakeholders in the development or rare disease therapies or orphan drugs.  The four categories were patient leaders, academic research community, pharmaceutical industry, and financial investment community.

Based on their input, and findings from other events hosted by NORD over the past year including a summit in Washington DC, he said NORD sees a need for:

• A Statement of Policy from FDA explaining that, while orphan drugs are reviewed with the same standards for safety and effectiveness as other drugs, FDA also exercises its scientific judgment in taking into account the special challenges of developing treatments for very small patient populations.
• A reduction in regulatory uncertainty through measures that protect patient safety but also acknowledge that patients with devastating diseases who have no treatment options may not be able to wait for "the perfect clinical trial design".  

Sasinowski said NORD "commends the FDA on its stellar, worldwide leadership role on orphan product issues for the past 27 years."  He also said NORD stands ready to do all it can to assist FDA on matters related to the development of safe, effective treatments for the nearly 30 million Americans with rare diseases.

Sasinowski presented his remarks in the opening session of a two-day public hearing sponsored by FDA to gather input from patient leaders, researchers, industry and others on current procedures and recommendations for future improvements in FDA review processes related to rare diseases and orphan drugs.

The full text of Sasinowski's remarks is posted on the NORD website (www.rarediseases.org).