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ARTICLES  &  COMMENTARY
FDA's Caution Hurting Patients
 
While the FDA is trying to save patients from the harmful effects of new medicines,manymore patients will die waiting for the good medicines than from using bad ones.
 

Cancer patients today continue to live longer and face better odds. New medicines that are the result of recent innovations in biotechnology are more targeted to the tumors they aim to treat, prolonging lives with fewer of the side effects that came with traditional cancer drugs.

This was the clear conclusion at the recent annual meeting of the world’s cancer specialists, the American Society of Clinical Oncology. Cancer still causes too much death and suffering, but investments in research are paying off.

Contributing to these gains are relaxed regulatory standards at the Food and Drug Administration for drugs that treated unmet medical needs like advanced cancer. Lower regulatory hurdles allowed poorly funded biotech firms with good ideas to get new drugs to market sooner, and made it easier for doctors to make quick use of the best new medicines.

But in recent months, the FDA has abruptly raised the bar when it comes to rapidly approving new cancer drugs, at the very moment when cancer seems more beatable.

While more rigid standards might make sense for common medicines like blood pressure pills, the same is not true of cancer. Despite recent success, many cancers still have few effective treatments. Even diseases like breast cancer and colon cancer, which have benefited from recent innovations, still claim too many victims, especially when these diseases reach advanced stages.

One big change is in the way FDA evaluates new cancer medicines for what is called “accelerated approval,” where the agency rapidly approves promising drugs for advanced diseases that are poorly treated with existing medicines. The FDA’s cancer division is no longer considering drugs for accelerated approval based solely on findings such as its ability to shrink tumors or stall their growth.

The cancer division is composed of well intentioned cancer specialists, many of whom once practiced medicine but have since left patient care behind. Over time, having lost touch with the realities of everyday practice, they become too absorbed by the statistical work of drug review rather than the practical need to get new options to sick patients as soon as possible.

For example, FDA medical reviewers focus only on a new treatment’s effectiveness relative to existing drugs. But they usually ignore the fact that some new treatments have fewer side effects than existing medicines, even though the newer medicine may not be as effective at shrinking tumor.

In the real world, the patients I see on my medical rounds make tradeoffs like these every day, opting for slightly less effective medical regimens if it means they will suffer fewer side effects from their treatments.

FDA cancer specialists, in some recent public meetings, have also openly bemoaned what they call a “race to the bottom,” where biotech companies opt for the shortest possible clinical trials in order to get their drugs to market as soon as possible. The FDA wants cancer companies to spend more time to generate more data about the ultimate effectiveness of their new drugs.

Delaying new treatments for the sake of generating more rigorous and complete medical evidence helps patients, to a point. But in the field of cancer, where practicing oncologists already do a good job of developing their own medical evidence, and prescribing new medicines based closely on the results of these scientific studies, the FDA’s strict posture is probably overkill.

The FDA is trying to save patients from the harmful effects of new medicines that haven’t fully proved their mettle. In the process, many more patients will die waiting for the good medicines than from using bad ones.

Scott Gottlieb is a physician and a resident fellow at AEI.