According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that the majority of these approvals were for diseases that already have approved therapies. Meanwhile, there are still thousands of rare diseases that do not have any approved treatments. These details demonstrate the priorities of the drug industry and shows just how much progress still needs to be made.
That vast majority of experimental therapies that get tested ultimately do not go on to get approved for use. The approvals from last year are actually less than in 2018; 59 novel drugs were approved in that year. In many cases, these new therapies offer incremental improvements for patients that were not responding to previously approved treatments. The new drugs offer little for patients that need more affordable treatments or for rare diseases that still lack approved therapies.
However, 23 of these drugs can be considered “structurally novel” from a chemistry perspective, and some of them offer real innovations. One example includes Mazent, which is catered specifically for patients with the secondary progressive form of multiple sclerosis. Another is the drug Rozlytrek, which is only the third cancer drug approved on the basis of a commonly found biomarker found on many types of cancer tumors as opposed to based on the location of the tumor.
Further investigation by the Chemical Abstracts Service (CAS) revealed that many of the well known, “Big Pharma” companies that have come to dominate the medicines industry actually played a less meaningful role in developing novel treatments; only about a third of the 48 approvals were developed by large companies. This suggests that these larger companies, who are in a more secure financial position, are less motivated to innovate. Many of the other novel therapies were the result of small start-ups and spin-offs from research labs or medical schools.
Unfortunately, drugs that disproportionately affect impoverished populations in the global south got scant attention from drug developers in 2019, with only four treatments being approved for such diseases.
Hopefully, 2020 will herald a shift towards greater innovation and the prioritization of rare diseases and diseases that affect the poor.
