Orphan Drugs controversias en cost-effectiveness

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The Orphan Drug Act (ODA) was enacted on January 4, 1983. It was designed to encourage the development of drugs for rare diseases. The law was amended the following year to define rare diseases as ones that affect fewer than 200,000 people in the U.S. But it also included drugs that affect more than 200,000 people the costs of developing and marketing the drug in the U.S. would exceed revenue from U.S. sales.

In terms of increasing the development of drugs for rare diseases, it’s clear the program has been successful. In a report published recently in the Orphanet Journal of Rare Diseases, it noted that almost 60 percent of new drug approvals in 2018 were for orphan drugs.

But the ODA has its controversies. A study published in late 2018 by Trinity Partners, based in Waltham, Mass., ranked dozens of new drugs approved in 2015 by the U.S. Food and Drug Administration (FDA) based on therapeutic benefits, R&D costs, and commercial sales. 

The study found that orphan disease drugs generally haven’t done as well at launch and often continued to stumble after three years of sales.(Más)