The U.S. Food and Drug Administration (FDA) approved 50 new drugs last year, which is at par with the average of about 50 new drug approvals each year that the FDA has maintained over the last decade. There are some trends that have continued this year and could help developers strategize for the years to come.
Cancer drugs dominate, as in previous years, with 15 (30%) novel approvals, followed by dermatology and non-malignant hematology with 6 (12%) approvals apiece, and cardiology agents with 5 (10%). Neurology and infectious diseases posted lower numbers than in recent years. The FDA approved 32 (64%) small molecules, and 16 (32%) proteins, including 10 new monoclonal antibodies, 3 bispecific antibodies, and 2 oligonucleotides. The proteins and oligos were approved by the Center for Drugs (CDER) as BLAs regulated by CDER. In addition, the Center for Biologics (CBER) approved 10 new products including 3 new gene therapies, 2 new vaccines, 2 cell therapies, 1 new CAR-T, 1 stem cell, and 1 human protein product.
These are not just numbers; they could indicate the FDA’s preferred methods for new product approvals. Almost half of the new drugs approved (48%) were first-in-class and 52% were orphan drugs. Whether it reflects the FDA’s prioritization of first-in-class drugs for rare diseases or an industry trend of submitting more applications for such indications to the FDA is hard to point out. The FDA rejected new drugs for diabetes and PTSD but about half of the new drugs approved were for non-orphan indications. It seems that the orphan versus non-orphan drugs have similar chances of approval overall, although most cancer drugs were orphan-designated as well. Breakthrough therapy drugs accounted for a third of the approvals (36%) while Fast Track drugs were slightly ahead at 44%. Accelerated drug approval accounted only for 14% of the overall approvals. This should show that the Fast Track designation is as good as the Breakthrough Therapy designation. With the new restrictive requirements for drugs approved via the accelerated approval pathway, this pathway may lose its charm for the developers.
The FDA touted 74% first-cycle approval and 94% on-time approvals but that number is skewed as it does not account for the applications that were withdrawn and resubmitted at a later time by the sponsors at the FDA’s request (read “strong suggestion”), and delays in accepting an application by repeated refuse-to-file (RTF) notifications. It would have been more transparent if the data included the total number of all applications submitted, the number of applications with RTF with time between each subsequent submission, and the number of applications withdrawn by applicants based on the FDA’s request. However, there would be challenges with those timelines since they do not depend on the FDA alone but the sponsors as well who may take different time to respond to RTF and FDA’s request for application withdrawal. A truly FDA-independent assessment would be needed for that.
The FDA celebrates approving new drugs before its peers by presenting that 68% of the drugs it approved were first in the US. However, it should be noted that not many countries, including those in the European Union, are as focused on new drugs for rare diseases as the US. The number of first-in-the-US drugs drops significantly for non-orphan drugs.
The FDA report and its independent analysis provide some useful perspectives for strategists to consider.
