If I told you that over 50% of the new novel drugs approved in 2023 are used to treat rare conditions, would you believe me? Well, you should as the chart below shows that 51% (28 of 55) of the novel drugs approved by the Center for Drug Evaluation and Research (CDER) last year were for orphan drugs!
As a matter of fact, for four of the last five years, orphan drugs exceeded 50% of all novel approvals (with a high of 58% of the novel drugs approved in both 2018 and 2020). It appears that this segment of the market may be particularly attractive to the pharmaceutical industry for a few reasons. First, there are a number of firms that have built their business models on the development of these products. Secondly, these products are usually eligible for seven years of Orphan Drug Exclusivity (ODE), which provides protection against competition for seven years from the date of approval (note that the award of ODE, especially for different indications, has come under judicial review—see posts here and here). If not eligible for ODE, because they are first-time approvals of a new chemical entity, these products will likely receive five-year NCE exclusivity. In addition, because there are few patients with such rare and ultra-rare diseases, the price tags on many of these drugs is very high, which permits firms to capture their investments in the development of these drugs while assuring a reasonably high profit so they can continue their research on cures or treatments for other rare diseases. It must be noted that, according to the FDA, “novel” drugs are new drugs never before approved or marketed in the U.S. Certain drugs are classified as New Molecular Entities (NME) for purposes of FDA review.
Nonetheless, the information presented in a new post titled “9 Things to Know About CDER’s Efforts on Rare Diseases” on the CDER website (here) provides some interesting additional observations regarding drugs for rare diseases. Take a look for yourself!
