Attention Minnesota: the Food and Drug Administration wants your orphans.
In August, the University of Minnesota will host a FDA workshop where the agency will help area researchers, biotech firms and drug companies apply for “orphan drug” status, a fast-track approval program for drugs that treat rare diseases like Huntington’s disease, myoclonus, ALS (Lou Gehrig’s disease), Tourette’s syndrome and muscular dystrophy.
The FDA expects participants to complete their applications at the workshop, or within 30 days.
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Created by Congress in 1983, the orphan drug program aims to speed the commercialization of therapies to treat diseases affecting less than 200,000 people a year in the United States. For diseases afflicting more than 200,000 people a year, applicants must show a reasonable expectation that they can’t recover research and development costs solely through U.S. sales.
Since it takes a lot of time and money to develop a drug, and the potential pool of customers is so small, the program offers a 50 percent tax credit on clinical testing expenses and a seven-year period to exclusively sell the drug.
Since 1983, more than 200 orphan drugs have come to market. The first products sold by biotech giants Amgen, Genzyme and Genentech were orphan products.
The program is not without controversy, though. Some critics charge the law is too generous to companies, which charge high prices for their treatments. In 2004, Genzyme reportedly generated $800 million from the sale of an enzyme to treat Gaucher’s disease even though it affects fewer than 200,000 people. Treatment for Gaucher’s can cost up to $400,000 per adult a year, according to a report in The Lancet, a British medical journal.
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Critics also say the program is too slow. From 1983 to 1988, the FDA awarded 255 orphan-product designations, eventually approving 37 products for sale, according to the Lancet. By the end of 2006, the FDA had awarded 1,713 orphan-product designations and approved 304 orphan products.
Supporters, though, say the orphan program is still valuable to patients who need the treatments.
“The development of any new medication is a long, risky and costly undertaking, and drug companies are naturally impatient to recover their investment once the drug is marketed,” according to The Lancet paper. “There are many examples of orphan drugs that provide valuable treatment, but which have little prospect of commercial return” such as zinc acetate for Wilson’s disease.
“Although every effort should be made to prevent any unfair advantage from orphan-product legislation, changes that might stifle essential enthusiasm for development of rare-disease products should be avoided,” the report said. “Without the well-considered incentives of the Orphan Drug Act, development of drugs for many rare diseases might well not have taken place.”