“Patient Engagement” means different things to different people and, from our perspective, is often poorly executed resulting in little more than “check the box” token efforts.
But as the science of patient engagement evolves, it is incumbent upon patient advocacy organizations, like Heart Valve Voice US, to help define and implement these concepts into to the design of new medical products. In that spirit, in early November we hosted a webinar with the FDA’s Mimi Nguyen, who is spearheading the agency’s guidance on patient engagement for medical device manufacturers with the Center for Devices and Radiological Health. You can watch the full webinar here.
As background, the FDA defines patient engagement as “intentional, meaningful interactions with patients that provide opportunities for mutual learning, and effective collaborations.” We agree. The agency also, importantly, differentiates between Patient Advisors, who serve in a consultative capacity for the design and conduct of clinical trials, and Study/Research Participants, who are the actual recipients of the drug or device being tested.
The FDA enumerates some of the benefits of hearing the patient voice:
- Inform device design or clinical trials;
- Bring to light new considerations to inform CDRH’s thinking on current issues;
- Raise or confirm problems that may exist with specific devices;
- Communicate treatment preferences; and
- Identify specific populations approach to benefit-risk for a given treatment.
With its guidance for industry, the FDA is attempting to:
- Help sponsors understand how they can use patient engagement to elicit experience, perspectives, and other relevant information from patient advisors to improve the design and conduct of medical device clinical investigations;
- Highlight the benefits of engaging with patient advisors early in the medical device development process;
- Illustrate which patient engagement activities are generally not considered by FDA to constitute research or an activity subject to FDA’s regulations, including regulations regarding institutional review boards (IRBs); and
- Address common questions and misconceptions about collecting and submitting to FDA patient engagement information regarding the design and conduct of a medical device clinical investigation.
Heart Valve Voice US very much appreciates the agency’s forward-thinking approach to patient engagement and is largely aligned with its views. The key, of course, is adoption and implementation by industry. We found three major areas where the FDA’s draft guidance could be strengthened: identification of patient advisors; defining the patient advisor role with IRBs; and dissemination of results back to research participants.
Our comments are summarized below.
Identifying and Engaging Patient Advisors
In addressing the challenges of finding patient advisors, Heart Valve Voice US recommends the creation of a comprehensive database of trained, willing patient advisors that manufacturers, researchers and others could access to facilitate engagement.
Heart Valve Voice US is in the process of implementing a training program for patient advisors, funded through a contract with the Patient Centered Outcomes Research Institute, to create a pool of individuals equipped to fill this role. But while those familiar with Heart Valve Voice US will be aware of this resource, others who may be interested in input from heart valve patients and advocates may be unaware. A centralized resource across all disease areas, drugs and devices, would provide ease of access and promote more active engagement.
Recognizing that the construction and maintenance of such a database may not be a core function of the FDA, we may look to other institutions involved in the research process. One solution could be the Reagan-Udall Foundation for the FDA following the model of its Expanded Access Navigator. Another possibility could be the National Library of Medicine, which is charged with maintaining the clinicaltrials.gov database. Alternatively, a private non-profit organization committed to patient engagement, such as FasterCures, could potentially house such a patient advisor database.
Defining Patient Advisor Role with IRBs
While the draft guidance specifies that, generally, the work of patient advisors with sponsors would not require review of an Institutional Review Board, the agency misses an opportunity to encourage IRBs to also engage patient advisors in more meaningful ways.
We see a tremendous opportunity for IRBs to engage patient advisors to contribute to discussions on issues such as informed consent, privacy and risk/benefit. Clearly a patient’s perspective would add value to these deliberations. While non-scientists and members of the community may be members of IRBs, there is no specific direction or guidance for IRBs to take direct patient input into account.
While true that the IRB’s role is to protect research participants, we see many instances where the priorities of patients do not necessarily match the presumptions of those charged with protecting them.
We reference the research from Kraft, Stephanie Alessi, et al., A Comparison of Institutional Review Board Professionals’ and Patients’ Views on Consent for Research on Medical Practices. Clinical Trials: J Society for Clinical Trials. 2016 September. 13(5): DOI 10.1177/1740774516648907.
This research found, for instance, that patients were more likely to prefer having consent discussions with their physicians (72.6%) whereas IRB professionals ranked clinicians the least preferred person to obtain patient consent (54.6%). Similarly, patients were generally more willing to forgo consent for randomization or medical record review if the consent would make research too difficult to conduct.
The authors conclude: “Our findings highlight a need to identify appropriate ways to integrate patient preferences into prevailing regulatory interpretations as IRBs increasingly apply research regulations in the context of research on medical practices. Dialogue between IRBs and research participants will be an important part of this process and should inform future regulatory guidance.”
Clearly, these represent cases where the input of patient advisors into the IRB process could produce processes preferable to patients. Further, research could be more productive and efficient if patient preferences are considered.
Heart Valve Voice US appreciates that this draft guidance is intentionally confined in scope to address only patient engagement in the design and conduct of medical device clinical investigations. Addressing more complicated issues such as participant reimbursement or compensation, or promotion of investigational devices, would add layers of complexity that would potentially delay the release of this valuable guidance.
We disagree with the agency, however, on one aspect of dissemination. Dissemination of clinical trial results to participants is of key importance to build patient trust and advance engagement in the research process. There is a fundamental disconnect to engage patients into the design and conduct of trials but to then abandon the desires of the patients when dealing with communication of the study results.
We strongly urge FDA to include in this guidance language that encourages sponsors to solicit input from patient advisors as to how to best share results with participants. We are not addressing at this time broader dissemination; rather focusing on “closing the loop” with the participants. This would serve as validation that the patient advisor input was, in fact, “meaningful.”
We look forward to working with our patient community, regulators, industry, researchers and other stakeholders to ensure the promise of patient engagement is realized.