Switch to Partial Approvals for Drugs: Commentators
Two Forbes commentators say that FDA’s current “all-or-nothing” system for approving drugs hurts patients and should be replaced with a system of partial approvals. University of Chicago economist Tomas Philipson and University of Southern California pharmacy and public policy professor Darius Lakdawalla write that patients are hurt by the current system in two ways. First, they say, the approval system hurts the subpopulation that would benefit from access to unapproved drugs and are less risk averse than FDA scientists. And second, the system imposes too much uncertainty on developers of new drugs, which they say limits the flow of future products for patients.
The professors write that a system allowing drugs to be partially approved by FDA based on limited information would avoid many of the risks created by the current approval process. The agency could then expand or limit approvals as new evidence emerges later, they say. While ideally access to a drug would be granted to a random sample of patients, consistent with the labeling indication, such an approach would be practically and politically challenging, the report says. Another approach, the authors write, would be to grant innovators temporary licenses to sell a fixed quantity of their drug, where the quantity rises and falls with the quality of the pre-launch evidence provided to regulators. Innovators could apply for an expanded approval with better evidence later.
“Such a strategy would also help to bring FDA approvals more in sync with payer coverage decisions,” the authors say. “As the Duchenne’s example (the recent approval of Exondys 51) indicates, a surprise FDA approval can precede not-so-surprising coverage denials from payers. A partial approval mitigates financial risk for the payer, making coverage more palatable. Failure to align FDA and payer incentives results in hollow approval decisions that do little for society other than waste marketing and access dollars.”
The article says there are few really daunting practical obstacles to implementing partial approvals. It notes that innovators already implicitly make decisions on how to target a newly-launched product, and partial approval would simply require that they target an explicit and fixed number of patients. The authors suggest that insurers might welcome a system that limits the scope of a new drug’s launch while evidence on its real-world benefits is still being gathered. Most important, they say, patients who are more willing to accept the risks posed by experimental drugs than FDA scientists are would have a feasible and faster track to access new therapies.
“Allowing partial approvals of drugs could also improve our understanding of how drugs work in the real world,” the authors conclude. “Pharmaceutical companies might be encouraged to take more innovative approaches to data collection. There would be stronger incentives for post-launch surveillance, and for adaptive trial design…. Allowing FDA to act more cautiously when evidence is limited penalizes the drugs with the weakest evidence base while leaving open the opportunity for reward when stronger evidence is available, a preferable outcome for all involved.”