Patient advocates call on FDA to develop policy that would speed development of new therapies for rare disease.
Rare disease patient advocates called on the U.S. Food and Drug Administration to remove regulatory uncertainty and allow for greater flexibility in how the agency reviews drugs for diseases afflicting small populations of patients. The call came during a two-day public hearing held by the agency as it seeks ways to spur drug development around diseases where markets may be too small to entice drugmakers to make the large investment needed to bring drugs to market for these conditions.
“No one is arguing against rigorous standards, it’s just the standards for rare diseases need to take into account the size of the patient population and the devastating nature of these diseases,” says Jonathan Jacoby, CEO of the RARE Project, a group that advocates for accelerated rare disease research.
There are about 7,000 diseases that are considered rare and treatments for only about 200 of them. A disease is considered to be rare if it afflicts 200,000 people or less. Though individually each of these diseases is rare, about 10 percent of the population suffers from a rare disease.
The FDA’s 2010 appropriations bill required the agency to convene an expert committee to review the ways the agency deals with therapies to treat people with rare diseases. As part of that effort, the expert committee held the hearings as a way to reach out to patients, their families, patient advocates, drugmakers and researchers for their input.
Frank Sasinowski, chairman of the National Organization for Rare Diseases, said in a statement at the hearing that the FDA in the 1980s put into place regulations in response to the AIDS crisis that provided for flexibility in applying standards to drugs in development to treat life-threatening and debilitating illnesses in order to speed the development, evaluation, and availability of new drugs.
“When these trials are conducted, sometimes with designs with which all parties may not be in full concurrence, including the FDA, great deference should be afforded the design of these trials and flexibility applied in the interpretation of their results,” says Sasinowski. “If such a principle were to be addressed and accepted by the FDA, much good would come of it.”

X