Good afternoon. I appreciate the opportunity to address this distinguished panel and our FDA participants. I’m Jack Mitchell, director of government relations for the National Center for Health Research (NCHR). We conduct research, use research data to inform public policy, and advocate for safe and effective medical products. NCHR accepts no pharmaceutical or medical device industry money, and therefore I have no conflicts to report.
We strongly support FDA efforts to strengthen the role of patients and we urge the agency to define patients as those who use medical products, whether or not they are seriously ill. The patient-focused meetings with FDA and other patient initiatives mentioned by Dr. Mullins in her presentation are welcome and necessary. I’d like to speak to broadening those patient perspectives.
We know it is a challenge for FDA to attract patients who are truly independent, since so many patient organizations are funded by industry, and many patients are trained and recruited to participate in FDA meetings by industry. Of course, all patients deserve to be heard, but industry-supported perspectives should be augmented by independent patient voices.
For example, a recent study by Harvard researchers found that almost all patients who spoke at FDA public meeting had ties to companies that could benefit from their remarks. Another study, of an FDA advisory committee meeting on a drug for Duchenne’s muscular dystrophy, reported that of the fifty-one public speakers, all but one had financial ties to the company that makes the drug. That one public speaker was from our research center.
There are patient organizations that are not funded by industry and can offer a more independent voice. We suggest that FDA needs to do more to reach out to them and include them. For example, the USA Patient Network is a new national organization consisting of patients who have received training to help them understand clinical trial research design and analysis, so they can serve as confident, well-informed patient representatives on FDA and NIH advisory committees.
In addition, the Patient, Consumer and Public Health Coalition is an informal coalition of about two dozen non-profit patient, consumer, physician, and public health organizations. They work together to prepare public comments for the FDA and other health agencies, and to educate Congress about important health policy issues.
Patients from these organizations have made presentations before FDA advisory panels and public forums. They usually pay their own way to FDA meetings or to provide written comments. Not surprisingly, they are dramatically outnumbered by patients whose travel, meals and other expenses are reimbursed by industry.
We respectfully ask the Science Board to ensure that FDA enhances efforts aimed at including these independent patient voices, not only for new drug development, but also on the wide range of public health initiatives in which the agency is engaged.
Your focus today is on innovation initiatives mandated by the 21st Century Cures Act. We are concerned that the new law does not guarantee sufficient resources to implement all its FDA-related provisions. For instance, it encourages the FDA to reply more often on preliminary data such as biomarkers, and allow third party review to replace FDA’s premarket scrutiny.
The law has already resulted in the FDA deregulating more than one thousand moderate risk devices that will no longer be required to submit 510-(k) applications. To better ensure safety, FDA needs to expend more resources to improve post-market surveillance, especially of medical devices.
Unfortunately, neither 21st Century Cures nor the user fees that FDA has negotiated provides sufficient resources for effective post-market surveillance, particularly for medical devices. Patients from the USA Network and other independent patient organizations have provided documented evidence to FDA of serious, irreversible harm caused by fast-tracked device approvals and inadequate post-market surveillance. They tell us that FDA often is not sufficiently responsive to their requests to strengthen patient safeguards.
We respectfully urge the Board to carefully address these patient recruitment and safety issues as you advise FDA about implementing the 21st Century Cures Act. Engagement perspectives should include patients who have been harmed by medical products that were not as safe as the research indicated, or had risks about which the patients were not adequately warned. While FDA is appropriately and routinely hearing from patients desperate for new treatments, those are not the only patients who have important perspectives from which the agency can learn. Thank you.