US and EU Laws Have Led to Rare Disease Gains

5 takeaways:

The Orphan Drug Act in the U.S. has spurred innovation for rare disease treatments. Before the 1983 law, only 10 new drugs had been approved for rare diseases. By creating incentives for pharmaceutical companies to develop “orphan drugs” – those abandoned by l companies as unprofitable – the law has led to 950 approvals for the treatment of 250 different rare diseases, said Aaron Friedman, senior regulatory counsel in the FDA’s Office of Orphan Products Development.  Approvals are accelerating: In 2020, of 53 new drugs that the U.S. Food and Drug Administration approved, 31 – or 58% – were for rare diseases. Financial incentives for drugmakers can include tax credits, exemption from fees and guaranteed market exclusivity for seven years.

A drug must be deemed “clinically superior” to existing treatments for its maker to be awarded financial incentives. That can mean that it has shown greater effectiveness than exiting treatments, that it’s safer, or that it offers a major contribution to patient care. The FDA has multiple programs to spur such developments, which Friedman breaks down here. One example: Zokinvy (generic name: lonafarnib), which is intended to reduce the risk of death due to Hutchinson-Gilford progeria syndrome, a rare genetic disease that causes premature aging and death. Drugmaker Eiger BioPharmaceuticals Inc. was given priority review and other incentives for its application.

Cancer drugs are earning orphan drug designations. The largest share of designation requests is for cancer drugs, at about 40%, followed by neurology drugs.

➃ Only about 5% of the 7,000 or more rare diseases have an approved therapy. Patients with one of the other 95% of rare diseases don’t have effective treatments or rely on so-called “off-label” – unapproved – drug uses. Doctors can prescribe these drugs but they haven’t been through the FDA’s rigorous testing for safety and effectiveness for the specific disease. “We hear that many, many rare disease patients use existing products off label,” Friedman said. “Unfortunately, they have to resort to that.”

The EU lags the U.S. in rare disease drugs but is catching up since it passed orphan drug legislation in 1993. “The [US] Orphan Drug Act has not only prompted the development of treatments for rare diseases and a large investment, but it created a mass of knowledge even when clinical trials are failing,” said Yann Le Cam, chief executive officer of EURORDIS-Rare Diseases Europe. “… It had a huge impact.” EU nations and rare diseases advocates are working to boost incentives for their companies to pursue more orphan drugs.


Speakers: 

Aaron Friedman, Senior Regulatory Counsel, Office of Orphan Products Development, U.S. Food and Drug Administration

Yann Le Cam, Chief Executive Officer, EURORDIS-Rare Diseases Europe


This program was funded by Fondation Ipsen. NPF is solely responsible for the content.

Aaron Friedman
Senior Regulatory Counsel, Office of Orphan Products Development, FDA
Yann Le Cam
Chief Executive Officer, EURORDIS-Rare Diseases Europe
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Transcripts
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Resources On Orphan Drugs and Government Incentives for Rare Disease Treatments
Aaron Friedman NPF Presentation
Yann Le Cam NPF Presentation
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