September 27, 2007

In the Wall Street Journal today, Richard Miller (CEO of Pharmacyclics and an adjunct professor at Stanford) argues that terminally ill patients are being denied promising treatments by the FDA:

In recent years, the FDA has made it increasingly difficult for terminally ill patients with cancer and other deadly diseases to gain access to new but promising drug treatments even after testing in large clinical trials. The FDA has rejected five promising cancer drugs this year alone. Why should the FDA block approval of new lifesaving drugs based on different standards than are currently employed by physicians when prescribing drugs for "off-label" uses?

Terminally ill patients may not have a constitutional right to choose experimental drugs, but the FDA does have a legislative mandate to grant accelerated drug approval based on limited but promising clinical data. We need the FDA to utilize the regulations promulgated as a result of the AIDS epidemic to give patients the right to choose these treatments while more lengthy definitive trials are undertaken. This in turn will spark greater private-sector investment in breakthrough drugs that will further improve the quality of health care.

Miller's underlying point is that the FDA's gatekeeper role is schizophrenic: once drugs are approved by the FDA physicians can employ them in any manner they see fit, and they do. This means that clinicians often make important new discoveries along the way, particularly in the treatment of complex illnesses like cancer. In fact, Miller estimates that up to 60% of all cancer treatments are used "off-label", i.e. for indications that weren't originally approved by the FDA.

So if doctors can use "experimental drugs" like these, perhaps the FDA should view its role not as a binary (pass/not pass) gatekeeper for drugs but as an informational resource that can help physicians find the best ways to use new medicines.

Posted by Paul Howard at September 27, 2007 11:35 AM

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