Maurie Markman, MD
Historically, FDA approvals of oncologic agents have occurred as a result of positive phase III trial results. However, Maurie Markman, MD, proposes that in this era of precision medicine, phase III trials with the endpoint of overall survival (OS) should not be the be-all and end-all in respect to drug approvals—particularly in ovarian cancer.
, Markman discussed the need to change the paradigm of clinical trials and FDA approvals for ovarian cancer.
OncLive: Can you discuss your presentation on the treatment paradigm of ovarian cancer?
My talk at this meeting was to basically challenge the existing paradigm in clinical trials in the United States and worldwide in ovarian cancer. There has been so much progress with new drugs and new strategies, and as a result, phase III randomized trials looking at OS as a primary endpoint is simply no longer rational.
We need to come up with a novel approach. That could be looking at high objective response rates (ORRs), or time to disease progression compared with a well-categorized historical control. It could potentially be having patients’ own natural history of disease characterize their time to progression. For example, [they could go] on this novel therapy and the time to progression was 50% to 100%. There are a variety of ways we could look at this, but the point is that we need to come up with strategies that are acceptable to the regulatory agencies, insurers, and ultimately, the patients.
Are there any steps being taken toward this?
There is no question that what I am describing and advocating for is happening. The extraordinary poster child for that is the approval of a checkpoint inhibitor [pembrolizumab (Keytruda)] based on robust phase II trial data with microsatellite instability-high (MSI-H) tumors, and I applaud the FDA for this. What they have done is extraordinary important for patients.
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