FDA official talks up new review approach
The FDA's Director of the Office of Oncology Drug Products Richard Pazdur is talking up a new way of approving cancer drugs. Scientific American reports that the “oncology czar” advocated approving drugs based on the pathways they attack, as opposed to the tumors they can affect. Scientific American notes that the advantage would be that the approval process could then bypass the need for location-specific disease analysis, a benefit in oncology where breast cancer and lung cancer have commonalities but are currently contemplated separately because of where the tumors occur in the body.
The alternative approval process was discussed unofficially at a Washington, DC, conference earlier this month.
The proposal is a bit of an image shift for Pazdur, whom Forbes noted in June has been derided as a “compassionless bureaucrat who denies cancer patients lifesaving medicines using red tape and fine print." This is despite his also being the person who has said that better drugs have made the FDA approval process easier, and who recommends drug companies call his office if they want to apply for breakthrough designation status before undertaking all the paperwork.
Pazdur's approach is just one of several ways the FDA is talking about change. The regulator has said it may do away with outcomes studies for experimental cholesterol-lowering drugs known as PCSK9s based on their ability to lower LDL levels. The agency has also talked about decoupling the global and cognitive impact requirements for Alzheimer's medications, meaning a drug could be approved if patients show progress on one, not just both, measurements.
A new oncology approach could mean a streamlined approval process, but Scientific American notes that drugs can behave differently, even if they interact with the same pathway, and could have an unintended coattail effect that could hide adverse events. Their example: ponatinib for chronic myeloid leukemia, which was pulled 11 months after it hit the market because it was associated with blood clots and heart issues.
Several other drugs, with the same target, had been approved beforehand, and SA says “several cancer doctors worry that the deleterious impacts may not have been picked up as quickly” if pathways were the basis of approvals, instead of the current method of clinical trials that requires patient monitoring and data collection. “Once the FDA greenlights a drug and it is ushered into the hands (and IVs) of patients, there is no real incentive for a company to continue to collect information about side effects in a systematic way,” noted Scientific American's Dina Fine Maron.