Regulatory Focus™ > News Articles > 30 Years Later, FDA Looks Back on Passage of Orphan Drug Act

30 Years Later, FDA Looks Back on Passage of Orphan Drug Act

Posted 04 January 2013 | By

It's been a momentous few months for the US Food and Drug Administration (FDA)-a time to look back and reminisce about where the agency has come from and what it has endured along the way. In October 2012, the agency celebrated the milestone of 50 years since the passage of the Kefauver-Harris Amendments to the Federal Food, Drug and Cosmetic Act, which endowed the agency with the authority to assess the efficacy of drugs and the processes by which they were manufactured. But January 2013 marks another milestone perhaps just as important to many patients: 30 years since the passage of  the Orphan Drug Act.

The Act's passage provided key incentives for manufacturers to develop therapies to meet the needs of patients suffering from rare-sometimes referred to as "orphan"-diseases affecting fewer than 200,000 patients in the US. At the time, manufacturers had little incentive to develop the therapies. A difficult approval pathway, small market, and the uncertain approval of the product meant that limited venture or corporate capital was more likely to go toward developing therapies with larger pools of patients.

The Act allowed FDA to give the products approved to treat the orphan conditions special marketing protections (7 years of market exclusivity), tax credits to offset some of the costs of development, faster regulatory reviews and additional assistance from FDA reviewers during the development and review process.

The success of the program has been dramatic, leading to the approval of hundreds of new therapies for hundreds of rare and orphan diseases-even so-called "ultra-orphans," which affect fewer than one in every 2,000 patients. To contrast, FDA noted that fewer than 10 products to treat rare diseases had been approved in the decade prior to 1983.

The program has been so successful, in fact, that many of the 39 pharmaceutical products approved in 2012 by FDA were for orphan indications, including Kalydeco (cystic fibrosis), Elelyso (Gaucher disease), Bosulif (CML) and Signifor (Cushing's disease). The act has been so successful, in fact, that many companies (and in particular biotechnology companies) are now using the orphan drug pathway as their main source of profit. A recent Reuters analysis piece by Ben Hirschler indicates that the high costs of the products, which could soon break the $1 million a year barrier, are a strong incentive for many companies to invest in orphan drug treatments.

The anniversary of the program comes just days after the death of Jack Klugman, an actor on the once-popular TV series "Quincy, M.E.," which many credit as being one of the driving forces behind the passage of the Orphan Drug Act. Several episodes of the series focused on the difficulties of getting treatment for rare diseases, marshaling public support to tackle the then little-known problem. The actor's role in the legislation's passage seemed to be acknowledged by FDA officials in a meeting notice posted to its website. A meeting to be held on 7 January 2013 commemorating the passage of the Orphan Drug Act is set to conclude with a viewing of episodes of "Quincy, M.E."


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