While the development of orphan drugs for rare diseases has skyrocketed in the last 30 years, Dr. Scott Gottlieb, Commissioner of the Food and Drug Administration is recently questioning the system, according to NPR. When the Orphan Drug Act was put into place back in 1983, there was a significant change in the number of successful drugs developed, going from a meek 10 orphan drugs, to now over 450 that have passed the approval process. It really is a huge accomplishment, but now the market is changing and Gottlieb thinks we need to rethink it.
The Orphan Drug Act gave orphan drug research and development companies huge financial breaks to make this push. They saved millions of dollars in fees, were given huge tax breaks, as well as market exclusivity for seven years. While these are great incentives, companies were treating only about 200,000 people — so the question now surfaces, can that be financially sustainable?
Unfortunately, that’s the issue Gottlieb has brought to the table. The price of these orphan drugs is out of sight and completely unreasonable for those affected. An average patient is paying an astronomical $140,442 for their drugs.
Financial incentives are “refreshed” whenever a new drug is FDA approved. This is great for newly developed drugs, yet Gottlieb has concerns with drugs needing “secondary approvals”, meaning drugs that existed prior and are being repurposed. Repurposed drugs are have been successful for patients, but Gottlieb believes there needs to be a new structure for those drugs, and companies shouldn’t necessarily get new incentives from those. At this point though, he does not have an answer for what they will do. Any change to the Orphan Drug act will have to go through Congress. So it’s back to the drawing board.
More recently, Congress greatly reduced the tax credit for orphan drugs, which is estimated to save the government $32.5 billion dollars over the next 9 years. Controversy continues to swirl around this decision, as well as the next steps for orphan drug research. Gottlieb says they will continue to look into other ways to achieve the appropriate goals of public health.
