An overview of the drugs approved by the US Food and Drug Administration (FDA) in 2019 reveals the diseases that are getting priority when it comes to bringing drugs to market. It also shows just how few of the thousands of conditions that plague humanity are getting new treatments, and the tiny proportion of molecules deemed “promising” that actually come to market.
The media is full of reports of potential medications that show promise in mice, and the stories that get coverage are a small sample of the media releases that flood science journalists' inboxes. If all of these lived up to the hopes, thousands of new pharmaceuticals would hit the market every year, medical science would have solved almost every medical condition and we'd all be living to 200.
Instead, the FDA approved just 48 novel drugs last year, less than one a week and down from 59 in 2018. What's more, most are for conditions that already have approved medications. The new drug may help a portion of those who don't respond well to the existing one, but it's unlikely to be transformative. Meanwhile, there is nothing new available for a vast range of rare but very damaging conditions, or common ones that primarily affect people with little capacity to pay.
An additional 10 “biosimilars”, drugs resembling those already on the market but whose minor modifications may make them more suitable for some users, were also approved
It's not all bad news, however. The Chemical Abstracts Service (CAS) conducted a review of the approvals and noted that 23 are “structurally novel” from a chemistry perspective. The CAS's Managing Director Todd Wills highlighted some to IFLScience that he considers particularly exciting. Top of the list is Rozlytrek, which Wills said marks only the “third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated.”
Another approval carrying great hopes is Mazent, which Wills explains is “The first oral drug specifically for patients with active secondary progressive multiple sclerosis.”
Pretomanid, designed to tackle treatment-resistant tuberculosis, is important not only for addressing a major killer threatening a come-back, but as only the second drug approved through a pathway designed to spur antibacterial and antifungal treatments.
The CAS dug into the approvals and found that where once the companies considered part of “Big Pharma” dominated the discovery of new drugs, only one-third of the structurally novel drugs approved this year came from that source. The rest originated with start-up companies, often spin-offs from universities or other research facilities.
“The shift has been increasing over the last decade as the discovery sophistication continues to advance, aided by technology which is helping level the playing field,” Wills told IFLScience.
In recent years greater attention has been drawn to tropical diseases and other conditions that primarily affect the world's poorest nations. Along with efforts to make existing drugs and vaccines more widely available to address the shocking rates of infant mortality and other early deaths in developing nations, there is also a lot of work to find treatments for diseases that barely exist in the rich world.
Judging by 2019's approvals, however, there's a long way to go – only four conditions concentrated in poorer nations got new treatments. There was, however, the approval of the use of two already available drugs for HIV in combination, which the FDA found sometimes makes them more effective than either on its own. Similarly, approval was granted for several other existing drugs to be prescribed in new ways.