• 5/31/2005
  • New York, NY
  • Scott Gottlieb, M.D.
  • Forbes.com

Cancer survival rates are climbing. Earlier detection is one reason. Another is the new medicines that created through recent innovations in biotechnology. Drugs today are more targeted to tumors, so patients are seeing their lives prolonged with fewer of the side effects that came with traditional cancer drugs.

The hope for the future is even better. More than 400 new cancer drugs are in development. One of the clearest beneficiaries of recent innovations has been the field of breast cancer, where 15-year survival rates for early stage breast cancer patients have gone from one in ten to one in five in just the past decade

With a series of new breast cancer drugs such as Bristol-Myers Squibb’s Taxol, the aromatase inhibitors, Genentech’s Herceptin and now Avastin (developed for colon cancer, it was recently shown to almost double the length of time women in late stages of breast cancer survive after chemotherapy), doctors are finally piecing together all of the new drugs they have into cocktails that are saving many more lives.

Some experts now say that recent product launches for colon cancer, including ImClone’s drug Erbitux, a similar drug made by Abgenix, another from Amgen and Genentech’s Avastin, will mean similar gains for that cancer.

The bottom line is clear: There’s still far too much death and suffering from cancer, but investments in research are paying off.

Relaxed regulatory standards at the Food and Drug Administration when it came to drugs that treated unmet medical needs (like advanced cancer) have also contributed to these gains. 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 now the FDA is raising the bar when it comes to approving new cancer drugs at the very moment when cancer seems more beatable. In recent months, the FDA’s cancer division has issued a number of new policies that, taken together, will make it harder for new cancer drugs to reach patients. As I noted in my recent issue of the Forbes/Gottlieb Biotech Investor, this higher bar is already weighing on the approval of some drugs in development right now.

While more rigid standards might make sense for routine medicines like blood pressure pills and antihistamines, since medical options already exist for these conditions, the same is not true of cancer. Despite recent success, many cancers still have few effective treatments, and even 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 the 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.

Previously, if no other drugs were approved for a particular kind of cancer, then a new medicine would be considered for this accelerated approval process. Now the FDA will consider all of the off-label medicines that doctors might be experimenting with before it decides if a new cancer drug should be eligible for the rapid approval process. Since information on off-label uses of drugs is usually preliminary and sometimes scant, comparing new drugs to off-label medicines is going to give biotech companies a very hard and hazy hurdle to beat.

The FDA’s cancer division also said it is no longer going to consider drugs for accelerated approval based solely on findings such as a new drug’s ability to shrink cancer tumors or stall their growth.

The FDA’s cancer division is comprised of well-intentioned cancer specialists, many of whom used to practice medicine but have since left patient care behind. Having lost touch with the realities of everyday medical practice over time, it has become too easy for these former physicians to be 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, the FDA’s 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 tumors. In the real world, the patients I see on hospital rounds make tradeoffs like these every day, opting for slightly less effective medical regimens if they come with fewer side effects.

The FDA’s 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 quickly as possible. The FDA wants cancer companies to spend more time on trials and 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 very good job of developing their own medical evidence and prescribe new medicines based on the results of these scientific studies, the FDA’s strict posture is probably overkill.

Delays make drug development more expensive bye closing the market to small biotech firms with good ideas and delaying new drugs from getting to dying patients. The FDA is trying to save patients from the harmful effects of new medicines that have not fully proved their mettle, but in the process, many more patients will die from the extended wait for the good medicines, than from using bad ones.