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Medicine & Markets

FDA Unravels The Cancer Miracle

Gottlieb, M.D., 05.31.05, 2:45 PM ET

NEW YORK - 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 (nyse: BMY - news - people ) Taxol, the aromatase inhibitors,

Genentech's (nyse: DNA - news - people ) 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 (nasdaq: IMCL - news - people ) drug Erbitux, a

similar drug made by Abgenix (nasdaq: ABGX - news - people ), another

from Amgen (nasdaq: AMGN - news - people ) 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.

Gottlieb is a physician and editor of the Forbes/Gottlieb

Biotech Investor. He is a fellow at the American Enterprise Institute

and is a former senior official at the Food and Drug Administration

and Medicare program. Dr. Gottlieb may own stock in the companies

mentioned in this article or consult with firms that advise them. His

FDA policy blog can be found at www.fdainsider.com.

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