Op-Ed: FDA About to Make Drug Approvals More Difficult

This piece was originally published in RealClear Policy on April 21, 2023.

For drug producers trying to bring their lifesaving treatments to market, sky-high approval costs are a formidable, and sometimes unbeatable, foe. The cost of creating a new medication and getting it approved tops an astounding $5 billion. And now, the Food and Drug Administration (FDA) is poised to make it more difficult to get cancer medications approved. The agency is proposing to subject cancer drugs to costlier and lengthier pre-approval trials, even if there’s promising preliminary evidence that these therapeutics shrink tumors. It’s unclear if drug sponsors will even be able to recruit enough volunteers needed to run a fully randomized clinical trial, given the rarity of cancer types being treated. Policymakers and FDA officials must recognize the changing landscape of therapeutics testing.  It’s critical that patients have access to game-changing medications without red tape and regulatory delay.

In announcing its new draft guidance, the FDA bemoaned small datasets and “reliance on cross-trial comparisons to historical trials to assess whether the observed treatment effect represents an improvement over available therapy…” This status-quo is admittedly not ideal. In a perfect world, researchers would be able to recruit an army of volunteers, randomly assign them medications (vs. placebos) and assess medication impacts with a high degree of certainty. This becomes difficult to pull off when about a quarter of all cancers diagnosed in the U.S. are types that afflict fewer than 40,000 people per year. As a team of researchers at the University of Rochester point out, “[b]y necessity, clinical trials in rare disorders enroll small samples. In combination with high inter-individual variability in clinical course observed in many rare diseases, this diminishes a study’s power.” 

The full piece can be found online here.