Broader Implications of Vemurafenib Approval

Maurie Markman, MD


September 07, 2011

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Hello. I'm Dr. Maurie Markman from Cancer Treatment Centers of America. I wanted to briefly discuss a very important event in the oncology world and specifically for patients diagnosed now or in the future with metastatic or unresectable malignant melanoma -- the approval by the US Food and Drug Administration (FDA) of a new targeted therapy for the treatment of metastatic melanoma in individuals who have a documented BRAF V600E mutation.

It should be noted that approximately half of the patients with malignant melanoma have a BRAF mutation. So clearly this approval of vemurafenib is a very important advance for a substantial percentage of patients with this very difficult malignancy. The results of a phase 3 randomized trial recently reported[1] showed a really impressive improvement in response rates, survival, and quality of life for the individuals who were treated with the experimental drug compared with the control arm, a drug used for several decades with very little activity, that is, DTIC.

I think the results of this trial and the approval by the FDA clearly demonstrate once again how we are entering a brand new era, and you can even call it a revolutionary era, where treatment will be based in oncology increasingly on demonstrated molecular abnormalities present within the tumors of patients. We are in the era of genomic medicine as it relates to cancer treatments.

The data that led to the development of this trial and now the approval of the drug really do, however, make us ask some important questions. First, and particularly with regard to this drug, one must ask: Was it necessary to do a phase 3 randomized trial with survival as an endpoint when the phase 1 data[2] demonstrated that the drug was substantially better than anything that we had seen before in the treatment of malignant melanoma? It's a question to ponder. It's certainly a question for our society, our medical community, and the FDA to consider.

One also obviously needs to ask the question of what happens next. There are a number of tumor types, including lung cancer, where BRAF mutations are noted. Is it reasonable to speculate? Is it reasonable to treat patients with these malignancies in the documented presence of a BRAF mutation, specifically considering the incredible potency and the effectiveness of this new drug in this particular setting?

Finally, of course, the question comes up of the cost of these agents. There's nothing about this drug that makes it more expensive than other novel targeted therapies that have recently been approved. But, of course, this once again raises the question of: can we afford as a society these very expensive drugs? The answer, of course, is not simply provided by a yes or no. But it is a discussion that our society must continue to consider and ponder and hopefully come up with solutions, because these important new drugs that are substantially affecting the quality of life and survival of our patients are not inexpensive. The cost of medical care is clearly something that is on the minds of all members of our society today.

I thank you for your attention. This is a very important day with a very important announcement, in particular for patients with metastatic melanoma. The approval of this drug may help a very large percentage of patients in their struggle, in their fight with cancer. Thank you for your attention.


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