Edition of 15 May 2017

Early this year, an investigative report published by Kaiser Health News revealed that financial incentives created by the Orphan Drug Act of the United States may be “manipulated by drugmakers to maximize profits and to protect niche markets for medicines already being taken by millions.” According to the article, the companies are able to legally do this due to the manner in which the Orphan Drug Act of the United States (US) was originally written. The article details that, 7 year exclusivity clause written into the Act can be a “potent pricing tool” and is used by many “repurposed drugs” to crank up the price. They found that seven of the top 10 drugs (ranked by annual sales) in the US are orphan drugs and have pointed out how it has to be turned out to be a big business. The examples provided in the article include:

Abbvie’s Humira: This drug was approved for rheumatoid arthritis to treat millions, but was later approved for juvenile rheumatoid arthritis and four more rare diseases. Humira is second best-selling drug in the US, and receives orphan drug benefits even though it may not be considered a “true orphan”.

Amgen Inc.’s Repatha: This drug won marketing approval and exclusive rights for an orphan disease (homozygous familial hypercholesterolemia) and for mass market (people with uncontrolled levels of LDL cholesterol) on the same day.

Depomed Inc.’s Gralise: A lawsuit was filed by the company against the Food and Drug Administration (FDA) as they were not granted exclusivity. The FDA wanted to know if Gralise was clinically superior to the mass market drug neuropentin (by Pfizer) which has the same active ingredient. The drug maker won because according to the law, they do not have to prove clinical superiority.

The Result
This investigation has led the Government Accountability Office of the US to “investigate the potential abuses of the Orphan Drug Act”. Although there is agreement on the importance of the Orphan Drug Act, 3 senators have raised the possibility of making legislative or regulatory changes in order to keep the initial intent of the Act.

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Following up
Nature Biotechnology published a comment on the effort by Kaiser Health News and the subsequent inquiry of the Orphan Drug Act. They also provide the example of Deflazacort (for the treatment of Duchenne muscular dystrophy), which is not a new drug and has listed its price at “$89,000, when the generic version generally cost less than $2,000 annually”, which inflamed the DMD community. Another drug Spinraza, which is prized at ~$750,000 for the first year and about $375,000 in the subsequent years, is facing reimbursement issues from the insurance companies. According to this article the drug-makers will have to provide more clarity while evaluating orphan drugs to the payers.