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Potential Targets, Potential Treatments, & coverage


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Potential Targets, Potential Treatments, and the Debate About What Will be Covered

August 9th, 2013 - by Dr. Jack West

I recently met with the folks from Foundation Medicine, who offer a comprehensive molecular marker testing platform for lung cancer and potentially other cancers that looks not only for commonly recognized molecular targets for which established targeted therapies exist, but also potentially identifies a huge range of mutations that may be a driver of the cancer, and for which less established treatment options might be effective. As this type of molecular interrogation of cancers becomes increasingly available, not only from Foundation Medicine but also from other private vendors and academic centers, we will increasingly encounter situations in which the identification of a possible important molecular target is accompanied by a list of suggestions of treatment options that are available based on varying amounts of evidence, sometimes quite minimal.

While more information is better than less information, it’s important that we know what to do with that information. In the case of Foundation Medicine reports, and many others, the report of results is typically accompanied by a list of potential treatments that includes clinical trials with agents for that specific target, as well as agents that may be approved for that particular cancer or are commercially available as a treatment for a different kind of cancer. For example, a patient with NSCLC that has a rare BRAF V600E mutation may have a melanoma treatment of Zelboraf (vemurafenib ) suggested.

That may well prove to be effective, but it isn’t a recommendation based on evidence that the treatment has worked for lung cancer. Historically, sometimes treatments for a molecular target established as effective for one cancer prove to be effective for another, such as the finding that Herceptin (trastuzumab), an established antibody against a target called HER2/neu that is overexpressed in about 1/4 of breast cancers, is also effective in gastric cancers that overexpress HER2, and it appears to be active in the small minority of patients with NSCLC that harbors a HER2 mutation. Unfortunately, however, there are also many examples of potentially active targeted therapies that have completely failed when tested in other cancer types, even when that marker has been present.

The question of how much evidence is needed to favor an unproven therapy is subject to a great deal of judgment, but in a world in which many of these treatments cost $5-10,000 per month, we are about to encounter a great challenge over whether insurers can or should pay for treatments that might possibly be helpful but don’t have evidence yet to demonstrate significant efficacy. If not covered, would a patient and their family choose to spend that kind of money on a possibly effective treatment? I might envision an insurer agreeing to cover only a very limited fraction or perhaps none of the cost of treatments that are recommended based on a concept but no strong clinical evidence in the same patient population.

One other point that is raised from this line of work is that it will only become more important to offer these options in clinical trials, where patients can not only gain access to these appealing treatment options but also generate some of the evidence that will be needed to clarify whether these good ideas really translate into significant benefits. We’ll need more of these trials and a more user-friendly clinical trials system to enable patients with rare molecular markers to come from faraway patients to the few places offering such trials.

Molecular oncology is revolutionizing how we deliver cancer care, but there is always instability in a revolution. I welcome your thoughts about how we might navigate this process.


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