FDA Advisory Committee Meeting on TOPAS Treatment for Fecal Incontinence
Thank you very much for the opportunity to speak today. My name is Dr. Jay Ronquillo and I am speaking on behalf of the National Center for Health Research. I am a physician who trained at Massachusetts General Hospital, have two engineering degrees from Cornell, a Master of Public Health from Harvard, and a master’s in biomedical informatics from Harvard Medical School. These are the perspectives I bring with me today. 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 the drug or medical device industry and I have no conflicts of interest.
Fecal incontinence is an important clinical condition affecting the quality of life for many patients and for which few effective treatments exist. In order to determine if the product presented today addresses that unmet clinical need, we should consider the risk-benefit profile of this device and put it in the proper context of related products currently being marketed to patients.
The study provided a detailed description of adverse events, some of which appeared to resolve. However, the remaining unresolved adverse events represent important concerns for patients, including chronic pelvic pain and pelvic organ prolapse, which can have serious long-term consequences on quality of life. Given the recent public safety concerns for surgical mesh products used for other indications, better data are needed so patients can understand the full impact of these potential complications.
The TRANSFORM study suggested a reduction in the number of fecal incontinence episodes and incontinence days, along with score improvements in some questionnaires assessing quality of life. However, the single-arm, unblinded study design is inadequate to provide conclusive results. It is not possible to assess the true effectiveness of this device without a control group to provide context for these findings. Previous studies have shown that control groups, even those with sham surgery, often improve significantly. Would patients in some kind of control group show similar improvements without the device? How would patients respond to newer conservative therapies that are now on the market? These are important questions that patients, physicians, and the FDA will need answered in order to evaluate the device’s effectiveness.
The proposed indication for the device is for treating women suffering from fecal incontinence who have not responded to more conservative therapies. However, the study targeted a much smaller subpopulation of patients than the proposed indication suggests. For example, some key groups were excluded from the study, such as women who are pregnant or have a history of pelvic organ prolapse, stress urinary incontinence, or relevant rectal or pelvic surgery. In addition, other important subgroups (such as younger women) made up a relatively small fraction of the study. As a result, the proposed indication, as currently described, includes patients for which there is little or no data supporting safety and effectiveness, such as young and/or pregnant women.
In summary, the lack of a comparison group or data for substantial groups of women most likely to suffer from fecal incontinence makes it impossible to determine if this device would benefit them. A control group is needed to determine if the device is effective for anyone, and if it is, it should be approved only for the types of women studied, not for the types of women who were intentionally excluded.
We encourage you to recommend that these studies be conducted before a decision is made about whether or not to approve this device.
Thank you again for the opportunity to speak today and for consideration of our views.