The biotech investor community and many others have demonstrated profound difficulty in recent years when it comes to understanding the premises behind FDA approval of new drugs.The FDA sets out to evaluate new drugs based on their risk/benefit ratio, which inevitably varies significantly by disease state. So, for example, a new drug that provided a month’s survival benefit or even 6 months of time to progression benefit in the treatment of pancreatic cancer would be a BIG deal — even if the drug had some significant side effects. By comparison, any new drug developed for the treatment of esophageal reflux disease today should have a very clear clinical benefit over currently available medications AND should probably be able to demonstrate a superior safety profile. It’s not as though we can’t manage pretty well without a new drug in this area!
The Abigail Alliance for Better Access to Developmental Drugs, with the significant support of some at the Wall Street Journal, have been fighting for years to improve access to development-stage drugs that have potential to treat patients who have exhausted their marketed therapeutic options. The Alliance sought to establish a constitutional right to such possible life-extending therapies, but the US Supreme Court was not prepared to accept this theoretical argument.
Here’s a different theoretical opportunity. Could we seek to establish a new category of FDA approval, in which drugs that had clearly established a reasonable, relative degree of safety in a particular clincial use, but had failed to establish effectiveness, could be approved solely on the basis of their safety, with the clear understanding that there was no requirement for any insurer to cover the clinical use of such drugs? This model might, for example, have allowed the FDA to approve the use of Dendreon’s Provenge (sipuleucel-T) for treatment of advanced prostate cancer. Provenge clearly appears to be safe, but equally clearly did not demonstrate clinical efficacy in line with the predefined endpoints of its pivotal Phase III clinical trial.
Some will argue that there is no point to approving a drug that no one could afford to pay for. My position is rather different. If a drug like Provenge was given approval under such a guideline, then the onus is on the manufacturer to find a way to make it sufficiently affordable for those who may decide they need it (in concert with their physicians). It also provides the FDA with the right to advise the manufacturer that all use of any drug approved under such a guideline must be carried out within the context of a registry trial so that everyone can learn more about the risks and benefits of a product of this type. In the meantime, the company can, of course, continue to develop and execute additional trials to seek full approval based on efficacy AND safety.
It was just an idea. Don’t shoot the messenger!
Filed under: Drug approvals and regulation | Tagged: FDA, drug approval, safe, effective, Abigail Alliance, Provenge
