FDA orphan disease

On Wednesday, February 29, the world celebrated the Fifth Annual Rare Disease Day (made all the more special by the “rareness” of this day’s appearance in our 2012 leap year calendar!) But that of course wasn’t what made this day so particularly impactful.

This year the cause was strengthened with a sense of collaboration, as the FDA hosted its first Patient Advocacy Day, dedicated to the over 30 million American patients in the rare disease community. I was fortunate to be able to attend this event, which featured speakers from various offices within the FDA, including Dr. Gayatri R. Rao of the Office of Orphan Products Development, Dr. Anne Pariser from the Center for Drug Evaluation and Research, Dr. Markham Luke from the Center for Devices and Radiological Health and Dr. Richard Klein from the Office of Special Health Issues. Amid the alphabet soup of these various departments, the FDA helped to showcase just how the regulatory side works with and really–relies on–patients to move orphan products to the market in the most efficient way possible. The FDA works to do so in number of ways, including:

  • Running a robust patient representative program to provide advisory committees with the unique perspective of patients and family members affected by life-threatening diseases
  • Awarding grants to further the development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases
  • Assisting and supporting the research, development, regulation and approval of drug and biologic products
  • Collaborating with international regulatory agencies to address unmet medical needs of patients on a global scale

It was interesting to hear about how the pharmaceutical industry is encouraged to develop drugs for rare diseases. For example, if a company develops the first drug to successfully treat an orphan disease, that company obtains seven years of market exclusivity–a huge financial incentive to counteract a small drug market. Couple this with how cheap the research technology has become (running one genome sequence once cost a half a billion dollars, now the same costs less than $1, 000) and you’ve got a great recipe for speeding up this research.

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orphan diseases what are orphan diseases?

"Orphan disease" is a description assigned to one of the more than 6,000 rare diseases that affect relatively small numbers of patients.

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