NCHR Testimony on Olaparib for the Treatment of Germline BRCA-Mutated Pancreatic Cancer

Nina Zeldes, National Center for Health Research, December 17, 2019.

Thank you for the opportunity to speak here today. My name is Nina Zeldes and I am here as a senior fellow speaking on behalf of the National Center for Health Research. Our research center analyzes scientific and medical data and provides objective health information to patients, providers and policy makers. We do not accept funding from drug and medical device companies, so I have no conflicts of interest.

Pancreatic cancer is often deadly and new treatment options are desperately needed. However, it is equally important that new treatments have been proven to be safe and effective and have a clear benefit for patients that outweigh the risks. It’s not clear that this drug fulfills those goals.

First of all, we agree with FDA scientists’ assessment of the limitations of this study. Problems with the study include:

  • a small number of patients,
  • the possibility that the patients in the experimental group differ from the other group,
  • the use of a primary endpoint that doesn’t reflect clinical benefit, and
  • uncertainty measuring tumor size.

These limitations make the evaluation of the risks and benefits associated with this drug difficult, if not impossible.

Next, it is not clear whether the chosen primary endpoint of progression-free survival is clinically meaningful for patients, particularly since the study demonstrated only a small improvement. And unfortunately, there is no evidence for increased survival, the study’s secondary endpoint. At the same time, patients in the study experienced more adverse events, including fatigue, nausea and constipation. These results indicate that the drug does not give patients more time or improve their quality of life. In fact, the adverse events will inevitably lower patients’ quality of life.

Given the risks and the unproven clinical benefit, there is no urgency to approve this drug, especially since there are other treatments available. If this drug were to be approved, it is likely that some patients would choose this new drug over the current treatment recommendations, not realizing that the current treatments have evidence of improved survival, but this new drug does not. Patients and doctors often assume that when the FDA approves a cancer drug, it increases overall survival. And that would be logical. When there are treatments that are proven to have benefits that outweigh the risks, there is no need for the FDA to rush to approve a treatment that lacks that evidence.

As advisors to the FDA, it is essential that you speak on behalf of patient safety as you carefully consider the data available for how this drug could help or harm patients.

Thank you for your careful scrutiny of the evidence.

The Oncologic Drugs Advisory Committee voted 7 to 5 for approval. You can read more about the meeting here.