Ken Getz, director of sponsored research programs and research associate professor, Tufts Center for the Study of Drug Development, Tufts University, offered his insights on patient engagement at CTTI’s 10-year Anniversary Symposium on Tues., Feb. 6. He shares those thoughts here in a special guest blog.

As a veteran observer and analyst of the clinical research enterprise, I am very proud of and amazed by the extent to which the patient engagement movement has touched the entire continuum of new medical interventions development.

New services have been created to improve the participant experience, including electronic informed consent forms and direct-to-patient clinical trials. Regulatory agencies have established mechanisms to gather input into clinical outcomes most relevant to patient communities. Sponsors are supporting more meaningful end-of-trial experiences, such as the return of plain language trial results summaries and e-clinical data to study volunteers. The movement has mobilized stakeholders and stimulated more collaboration than we have ever seen before.

However, while we see a proliferation of patient engagement initiatives and guidelines, there are few signs of standard and consistent commitment and practice. Almost daily, new entrants offer technology solutions that can create more fragmentation and market confusion. We also see wide variability in receptivity to, and implementation of, patient engagement initiatives. Nearly all organizations have vocalized concerns about the financial return, long-term value, and perceived risks of patient engagement.

CTTI is committed to helping organizations work through these barriers and obstacles. I have had the privilege of participating in several CTTI projects, including a recent study that quantified the high expected net present value of patient engagement practices for a typical oncology drug development program.

We must do everything we can to give the patient engagement movement the opportunity to take hold and realize its compelling promise. Only then will clinical research and clinical care best serve the needs of public and patient health.

Findings and recommendations from CTTI’s Patient Groups & Clinical Trials work were presented during the inaugural meeting of the FDA’s Patient Engagement Advisory Committee (PEAC) on Oct. 11-12, 2017.

At the meeting, Bray Patrick-Lake, director of stakeholder engagement, Duke Clinical Research Institute, discussed patient engagement efforts in the clinical trial enterprise and presented work conducted by CTTI to establish best practices for effective engagement with patient groups around clinical trials, which include:

• Engage the “patient voice” by establishing partnerships from the beginning of the research and development program to improve trial design and execution
• Clearly define the expectations, roles, and responsibilities of all partners, including the resources being committed, data being shared, and objectives of the program
• Manage real or perceived conflicts of interest by establishing policies that require full disclosure, transparency, and accountability

The PEAC is composed of patients and caregivers who provide perspective to the FDA on complex issues relating to medical devices, the regulation of devices, and their use by patients. This first meeting brought together patients, patient organizations, FDA, industry, and other medical and scientific experts for discussion on incorporating patient perspectives in the design, conduct, and reporting of medical device clinical trials.

Meeting materials, including slide presentations, are available on the FDA website.

As documented in a recent JAMA viewpoint article, the US FDA is making moves to integrate patient perspectives throughout the continuum of medical product development. While the FDA and other key stakeholders have declared a commitment to create a more effective model for engagement between research sponsors, investigators, and patient groups, no evidence-based guidelines for best practices have existed, until now.

On October 7, CTTI released recommendations that identify evidence-based best practices for engaging with patient groups, as well as provide case examples and tools. These recommendations are the result of CTTI’s Patient Groups & Clinical Trials Project.

How and when should patient groups be engaged in the research and development continuum? How do patient groups and sponsors of research best assess each other’s interests, expertise and assets to increase the chances of successful clinical trials and therapy development? We are proud to offer actionable solutions to these questions around patient engagement and encourage you to share them with colleagues involved in the clinical trials enterprise.

“The FDA is committed to integrating patient perspectives into its regulatory evaluations and decision making and also encourages medical product innovators and clinical researchers to collect information on patient perspectives to inform development programs.” (Source: JAMA)

On October 14, 2015, PLoS ONE published Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey. Written by members of CTTI’s Patient Groups & Clinical Trials Project, the objective of this publication was, “to gain a better understanding of attitudes and practices for engaging patient groups so that actionable recommendations may be developed.” After analyzing data from 179 survey respondents, significant differences were detected between “stakeholder groups in perceptions of the value of patient group engagement with academia and industry around clinical trials- finding that may represent a significant barrier to engagement that was not identified by the individual stakeholder groups independently.”

In addition to this publication, this CTTI project also developed Official Recommendations on Effective Engagement with Patient Groups around Clinical Trials.

We are pleased to share these free resources, and encourage you to share them with friends and colleagues active in the clinical trials enterprise.

If you are active on Twitter, you can participate in this ongoing conversation with #PGCT.