[Docket No. FDA-2016-D-2153]

The National Women’s Health Network is a nonprofit advocacy organization that works to improve the health of all women. We are supported by our members and do not accept financial support from insurance companies, drug companies or medical device manufacturers. Since the NWHN’s founding 40 years ago, we have brought the voices of women to the FDA, advocating for medical products that meet women’s real life needs and a drug and medical device development process that reflects women's lived experiences.

We want to convey our appreciation for the FDA’s continued commitment to monitoring device safety and efficacy, and for acknowledging the potential for improvement in the medical device review and approval process. We respectfully submit the following comments on the draft guidance for “Use of Real-World Evidence to Support Regulatory Decision Making for Medical Devices,” which addresses the important role that real world data—and real world evidence—could have in the approval and post-market surveying process for medical devices. We look forward to working with the FDA to ensure that the draft guidance is implemented in ways that streamline and improve device safety and effectiveness, as well as incorporate insight from women’s real experiences.

I. Importance of Real World Data & Evidence

There are many ways to collect data prior to approval as well as throughout the post-market surveillance process. Real world data, such as case reports, electronic health records, and registries, can help us better understand a more complete safety and effectiveness profile for both drugs and devices than manufacturer sponsored clinical trials alone. Preclinical and clinical trials typically have extensive exclusion criteria and controls, which make use in the research setting different from average use. Dismissing data collected in the “real world” means ignoring the voices and lived experience of women who were not part of the sponsor’s studies, those that do not fit the pharmaceutical industry’s intended narrative. These women’s stories and their voices deserve to be heard and respected.

II. Need for Real World Evidence in light of the Essure Device

Using real world data, and thus real world evidence, to impact the regulatory process is an important and pressing issue, and one which we are actively working on. In 2002, we testified at the FDA in support of the approval of the Essure device for permanent sterilization. As we stated back then, expanding the number of safe and effective contraceptive methods available would be a significant advance for women’s health—contingent on adequate post-market surveillance studies and a national registry to follow women who had received the implant. In 2015, after a high number of serious adverse events were reported to the FDA, we testified again regarding the Essure device and the continued need for appropriate postmarket surveillance. We recommended, among several things, that women’s personal accounts of safety and efficacy of the device be taken seriously and made available to other women prior to receiving the implants. We value the insight that individual women’s lived experiences provide and the FDA should use all resources in their power to make knowledge sharing of women’s experiences possible and real.

As we stated previously, women’s personal testimony of their experiences should not be dismissed simply because it did not result from a clinical trial. The physical and emotional effects women may or have experienced are real—whether they are apparent during the pre-approval clinical studies or from subsequent “real world” use. We recognize that randomized clinical trials are the gold standard for evidence-based research. We also recognize that it is not always realistic to ask women to be randomized to contraceptive methods and procedures. We also know that it is common medical practice for drugs and medical devices to be used in populations for which they were not specifically approved. Because these populations were not part of the pre-approval studies, however, there may be limited data on safety and efficacy. In these examples, real world data can “provide information on real-world device use and performance from a wider patient population than a more traditional clinical trial.” Expanding the sources of data informing the regulatory decision making process means researchers, physicians, and manufacturers have a broader data set on which to make important regulatory decisions.

The evidence available right now for Essure, and other medical devices, may only be after-the-fact studies, with all of their limitations, and the lived experiences of women, as reported by women themselves. Women’s reports of pain from the Essure implant, as well as the other frequently reported adverse effects such as autoimmune and mood disorders, should be taken very seriously.

Our hope is for a future where women considering Essure, and all medical devices, will have access to the full range of information necessary to make an informed decision. We fully support using real world data as a tool to better and more fully understand the health impact of medical devices; however, it should not replace rigorous evidentiary approval and surveillance standards. It is imperative that the FDA continue to hold medical devices and medication to the highest possible standard. So while we support using real world evidence to “contribute to a fuller understanding of the benefits and risks” of a particular medical device, it should not be used as a substitute for, or an equivalent of, evidence resulting from a randomized controlled trial. We support and appreciate the FDA’s initiative on this issue and implore the agency to consider the potential impact of real world data and evidence on the health of patients across the country.

III. Conclusion

In closing, we appreciate the FDA’s work in developing the draft guidance on using real world evidence to support regulatory decision making for medical devices. We, too, believe that real world data can “constitute valid scientific evidence” and can help inform researchers, clinicians, advocates, and patient decision-making. We hope you will take our testimony into account as you finalize the guidance to further improve the safety, efficacy, and patient and provider understanding of these devices.