October 1, 2014
Good morning. Thank you for the opportunity to speak today. My name is Dr. Laurén Doamekpor, and I am a senior fellow at the Cancer Prevention and Treatment Fund. Our research center scrutinizes scientific and medical data and provides objective health information to patients, providers and policy makers. We do not accept funding from device companies and therefore I have no conflicts of interest.
We all know that current treatments for prostate cancer have side effects that harm men’s quality of life. While most men diagnosed with localized, low-risk prostate cancer will likely die from something else, not from prostate cancer, recurrent prostate cancer frightens patients and needs to be treated. However, any new treatment should show clear evidence that the benefits outweigh the risks. We are not convinced that the HIFU device achieves this goal.
The analysis of the primary endpoint showed that 64% of the patients who were treated with this device had obtained local control of prostate cancer at 1 year after treatment. But that doesn’t tell us anything about long-term effectiveness, which is key for prostate cancer. Proof of long-term effectiveness would also require a control group or comparison group, which this application lacks.
The sponsor’s goal was a 40% success rate at 1 year, and they achieved that. However, even a 64% success rate at one year is very low compared to the long-term success rate of prostate surgery. We share the FDA’s skepticism that this device should be approved based on this low 1-year success rate.
In addition, the safety profile is not impressive. Incontinence was reported in 43% of patients, and was still 31% after one year. That is worse than most studies of prostate surgery, which has a much higher long-term success rate.
With only 100 patients in the study and no long-term data, the sponsor has not provided enough data to justify approval. The small sample includes only 13 Black patients, even though prostate cancer is more deadly for Blacks. There are only 5 Hispanic patients. The sponsor says they did subgroup analysis of effectiveness and that Blacks did well with the treatment. The Advisory Committee should demand to see the safety and effectiveness outcomes separately for Blacks and Whites.
The sponsor is already enrolling a 2nd cohort of 100 patients, and FDA should delay an approval decision until those data are submitted. The FDA should also require at least 2-3 years of data before the agency decides whether to approve this device. While we commend the sponsor for its plan to conduct a 5-year post treatment study, it would be unfair to patients to pay for an unproven device based on such a small, short-term study.
In summary, the study is too small, too short term, and has no comparison sample. It has too few Blacks and Hispanics. Evidence of long-term effectiveness is needed BEFORE approval, not as a post-approval study.
That’s why we urge you to vote NO 3 times: the data from the study do NOT support a reasonable assurance of safety OR effectiveness for this device and that therefore we can NOT conclude that the benefits outweigh the risks.
At the end of the meeting, the panel voted 10 to 0 with 1 abstention that benefits of the device do NOT outweigh the risks.