Why New Standards Are Needed for Faster Cancer Drug Approvals

March 6, 2013
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Several years ago I attended an FDA Oncology Drug Advisory Committee (ODAC) hearing. What the medical experts and the FDA regulators wanted to know most was did a proposed new cancer medicine help people live longer? Not better, just longer.

Several years ago I attended an FDA Oncology Drug Advisory Committee (ODAC) hearing. What the medical experts and the FDA regulators wanted to know most was did a proposed new cancer medicine help people live longer? Not better, just longer. The CLL (chronic lymphocytic leukemia) drug in question that day was not recommended for approval because they never could show a “survival advantage.” I think the small biotech that made the drug ended up being closed down. Fortunately, the FDA has approved some other cancer drugs since then where they’ve helped people live better. I take one, Jakafi for myelofibrosis, which greatly reduces symptoms but there’s no clear data yet that people live longer because of it. So thanks to the FDA for becoming more enlightened. But now there’s a need to move still further ahead.

In some cancer conditions people are living for many years with cancer before they eventually succumb. Biochemically recurrent prostate cancer can be like that. There’s evidence the cancer has spread but it’s not obvious where. Another case is in CLL where people with less aggressive subtypes may have had treatment but then be in remission for several years. So here’s the regulatory problem: if people are going to live several or many years with a cancer, how can you early on a measure the effectiveness of a proposed new drug that might help them live longer? In prostate cancer and CLL, to name two examples, that’s the dilemma. There may be a “survival advantage,” for example for one of these promising new “small molecule” pills now in trials, or even a new drug that’s been approved, as in prostate cancer. But you need a test the FDA and other regulatory bodies will accept as a “surrogate” for how long someone lives.

In an interview with Dr. Celestia Higano, a prostate cancer expert, she tells me about just that. And in another interview, with Dr. Peter Hillmen, a CLL expert, he said he is going before the FDA to ask that sensitive tests for MRD (minimal residual disease) be acceptable measures. The idea is new tests, or ones that should be developed, can be an acceptable measure of whether a potential new drug will extend life.

My view is the FDA, and other agencies around the world, should work collaboratively with industry and scientists to expeditiously assess if a potential new drug is worth approval. If new tests can speed things up then let’s agree on them and use them.

As some cancers are now becoming more like chronic conditions where you live with them rather than dying fairly quickly from them, we need a new system to measure how lasting “chronic” is.

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