Skip to Main Content

Could this be a coincidence?

After months of anticipation, the US Food and Drug Administration released final guidelines on Thursday for allowing drug makers to provide experimental treatments to terminally ill patients. And Wall Street wags see the move as a way for the agency to avoid approving a drug for treating Duchenne muscular dystrophy and sidestep intense political pressure, while still making it possible for patients to obtain the drug.


As a result, stock in Sarepta Therapeutics, which makes the drug and has regularly battled with the FDA in hopes of winning marketing approval, is down more than 26 percent today.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

  • No drug company in its right mind would continue to manufacture a product simply to satisfy the demands of a compassionate use program without assurance that there would still be enough patients left to enroll in an adequate and well controlled trial. No parent in their right mind would enroll their child in a clinical trial when they can get the active drug under compassionate use, money notwithstanding. Having run a compassionate use program it becomes a three part self fulfilling prophecy:

    1) the company will issue the Duchenne drug under a compassionate use protocol; 2) the company will be unable to recruit sufficient number of patients for a RCT based on #1; and 3) at some point the company will make a decision to cease manufacturing the drug, notify the parties, take the heat and become a footnote to the annals of drug development.

  • An excellent and relatively complete explanation of the potential strategy behind FDA’s release of its new single-patient treatment use form (a form to request FDA approval for treating a single-patient with a single drug), and the new (but actually old) related guidance documents. For complete accuracy, it is necessary to also explain that these programs, including a company’s ability to charge limited costs, have been in place for decades and have been almost entirely non-functional for reasons that FDA has been unwilling to acknowledge or address over those decades. The programs don’t work, and despite the FDA’s carefully crafted PR campaign aimed at trying to blame everyone but itself for the near-complete failure of the programs, FDA knows they don’t work, and they are fine with that. Thus, if FDA points to the form and guidance documents (which explain decades old programs that haven’t worked) as cover for failing to approve a drug they should approve under the current law governing agency decisions, then Congress should take note and act immediately to strengthen its already clear direction to a FDA that a drug like eteplirsen should receive accelerated approval based on exactly the kind of evidence FDA already has. It is also important to note that FDA and its Commissioner Robert Califf report to the Secretary of HHS (Sylvia Burwell) and both report to the President. Both Secretary Burwell and President Obama should be taking note here. The credibility of the FDA with millions of Americans affected by serious and terminal rare diseases is at stake, and FDA will lose that credibility if they pull something as crass as releasing a meaningless form and guidance documents as cover for making a lethal mistake for thousands of kids with DMD.

    • I suppose the reason the compassionate use programs don’t work is money – recovering manufacturing cost is not sufficient for companies to participate. Therefore, I see three possible solutions,
      1) The federal government (HHS) subsidizes compassionate use programs.
      2) FDA allows companies to charge using a cost plus model that provides sufficient incentive for companies to participate.
      3) FDA institute a temporary approval category, whereby companies can charge market prices, but permanent approval will depend upon completion of all clinical trials to allow the accumulation of sufficient data to satisfy FDA’s full approval standards.

Comments are closed.