In 2018, the industry saw a record breaking 56 new drug approvals by the FDA. While 2019 is lagging behind with only 20 approvals, the Director of the FDA’s Center for Drug Evaluation and Research, Janet Woodcock, M.D., states the FDA is still committed to accelerated drug approvals. 

Fast FDA approvals have shown benefits, especially to those with serious, difficult-to-treat diseases, most recently seen with the approval for Novartis’ drug, Zolgensma, the first and only treatment for spinal muscular atrophy (SMA), a rare, progressive disease in children. However, with these potential benefits, Woodcock warns of the potential of these fast FDA approvals to backfire in more ways than one.

Woodcock notes that several possible outcomes can be seen as a result of the FDA’s recent flexibility of drug approvals:

  • Most immediate, the Center for Biologics Evaluation and Research (CBER) may lack the resources to handle the potential flood of new drug applications stemming from the recent advances in drug development such as gene therapy
  • There is also a notable “wide skill gap” in industry, especially in smaller companies, when it comes to regulatory expertise
  • Companies of all sizes often fall into the trap of developing drugs further based on “post hoc analyses” of a small subset of patients’ responses from a previous trial, which can lead to flawed conclusions
  • Lastly, even if the FDA grants approval, insurance companies may be less likely to follow suit, as Novartis has been learning first hand with Zolgensma

Woodcock continues that clinical trial design could also be changing. The gold standard of “randomized, placebo-controlled, double-blind studies” may no longer be realistic as more and more patients with these rare diseases do not want to be given placebos and the FDA’s flexibility will allow these changes.

It will be interesting to see how the number of new drug approvals rack up by year end in 2019 vs 2018, and how this further impactspatients with new medical treatment options and the industry as a whole in the context of future of drug development. Are randomized, placebo-controlled, double-blind studies on their way out and more customized studies, such as dose comparisons and delayed start trials, going to become the norm? How will this affect drug pricing and insurance approvals? The implications could prove to be significant across the industry. Will 2020 develop in a similar fashion as 2018, with a large number of approvals stemming mainly from the double-blind placebo-controlled clinical trials of the past? Or will we see a shift further away from the standard into a more varied and unique space?