January 20, 2005
Cancer Drug Approval Process Getting Tougher At FDA

Scott Gottlieb M.D., recently departed US Food and Drug Administration (FDA) Director of Medical Policy Development, a practicing physician and fellow at the American Enterprise Institute, argues that the FDA is making cancer drug approval harder and more costly.

Even more concerning, there is also a whiff of caution coming from the agency's cancer division--the Oncology Drug Advisory Committee (ODAC)--where delays on the approval of new drugs can have a dramatic impact on the lives of patients who are suffering from terminal disease.

At a meeting last month, outside advisers to ODAC as well as rank-and-file FDA medical reviewers expressed criticism of the applications they are seeing and a desire to clamp down on the number of cancer drugs qualifying for accelerated approval. Accelerated approval regulations allow the FDA to approve products based on preliminary test results, with the proviso that the company continues with clinical trials after the drug is marketed.

The chair of the FDA's cancer drug advisory committee rejected the idea that cancer drugs should be allowed onto the market if they are reasonably safe and have some degree of effectiveness (known as "efficacy") with the understanding that oncologists will determine their value through routine use of the drug.

Higher costs per drug developed translate into fewer drugs being developed at any one time and fewer new drugs available in the future.

The FDA is moving toward making it harder for a new drug to be approved if it can not be demonstrated to work better than the off-label use of an existing drug.

The other point of contention at the cancer advisory meeting was whether it is appropriate for the FDA to require drugs up for accelerated approval for treatment of a specific kind of terminal cancer to prove that they were superior to other drugs being used off-label to treat the same cancer.

The debate turned on the FDA's consideration of Inex Pharmaceutical's application for accelerated approval of Marqibo vincristine sulfate liposome injection to treat relapsed, aggressive non-Hodgkin's lymphoma (NHL). The FDA noted that no products have been approved for the indication, and suggested that the committee consider whether a number of products that are used off-label for the indication should be considered "available therapy." By suggesting that ODAC consider off-label uses as available therapies, the FDA dramatically raised the bar for approval of Marqibo.

Imagine you have relapsed NHL. Suppose there is an existing drug that works against it for some though not all people. Would you want the FDA to hold another drug off the market that also works for some but not all people? Or do you think you should have the right to choose between the two drugs or even to take both of them?

I believe that once someone has been given a diagnosis of a fatal disease that they should be given the equivalent of a "get out of the FDA drug approval jail free card". In other words, once your days are numbered due to a specific diagnosed disease you should be free to take any experimental therapy and drug and biotech companies should be free to sell you any experimental therapy without the FDA being able to stop them.

A smaller step in the right direction would be to replace the existing membership of the above mentioned Oncology Drug Advisory Committee (ODAC) with a new membership made up of people with diagnosed cancer. There are plenty of scientists, epidemiologists, and medical doctors walking around today with diagnosed cancers. So the committee would not have to lack for expertise. But what it would cease to lack is a great sense of urgency and a sense that patients should be given more choices.

If you are going down the elevator for the final check-out from the big hotel of life why should the government have any power to prevent your trying any therapy imaginable on your way down? Someone close to me is dying from metastatic cancer and I do not understand why the government should have any power at all over what experimental treatments someone such as this person might try. The government can not protect people from death. The government can not regulate death out of existence. But the government can and does impose such costs and obstacles on the drug development process that the rate of development of new drugs is greatly slowed and the date at which various diseases become curable gets pushed much farther into the future than it needs to be.

Share |      Randall Parker, 2005 January 20 01:01 PM  Policy Medical


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