An FDA rule created to spur drug companies to develop treatments for rare diseases is being used far beyond its original scope, critics say.
[Would you believe that Crestor is an "orphan" drug?]
A new study from Johns Hopkins University School of Medicine questions whether some of the biggest drug companies and their blockbuster medications are taking advantage of a decades-old act meant to increase research, development and drug approval for people suffering from rare diseases.
The new study, published in the American Journal of Clinical Oncology,argues that while the ODA has fostered drug development for patients with rare cancers and other diseases, current data suggest that companies are "gaming the system to use the law for mainstream drugs."
The authors found what they deem "a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits."
The study contends that some big drug companies submit a drug for FDA approval with a narrow enough indication that would qualify it for orphan drug benefits. After FDA approval, however, the drug can be used more broadly.
For example, Rituxan (rituximab), which is made by Roche and was initially FDA approved for use in the treatment of follicular non-Hodgkin's lymphoma, is the No. 1 selling medication approved as an orphan drug.
"It is currently used to treat a wide variety of conditions, ranks as the 12th all-time bestselling medication in the United States, and generated over $3.7 billion in U.S. sales in 2014," the report states.
The researchers' concern is not only the "corporate welfare" that is being afforded to blockbuster drugs and highly profitable drug companies, but that "patients with rare cancers and other diseases may suffer due to dilution of the tax incentives and other benefits" offered by the rule to spur the development of niche drugs.