HP&M Attorney Co-Authors Recommendations and “Rules of Engagement” for Successful Collaborations Between Sponsors and Patient Groups Around Clinical TrialsAugust 7, 2017
On July 27, 2017, the Drug Information Association (DIA) journal, Therapeutic Innovation & Regulatory Science, published recommendations from the Clinical Trials Transformation Initiative (CTTI) Patient Groups & Clinical Trials (PGCT) Project team, of which HP&M Attorney James E. Valentine is a member, on the best practices for effective patient group engagement. This publication supplements the PGCT Project’s official recommendations, originally released by the team in October 2015 (see prior blog post here), and describes the team’s research methods and key takeaways from the endeavor.
CTTI is a public-private partnership to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. CTTI comprises more than 80 organizations, including representatives from government agencies (e.g., FDA, CMS, NIH), industry representatives, patient advocacy groups, professional societies, investigator groups, academic institutions, and others. More information on CTTI is available on their website, here.
CTTI’s PGCT Project was initiated to find evidence-driven, actionable solutions to questions on how patient groups and sponsors can best work together to increase the chances of successful clinical trials and therapy development. The Project team consists of engagement experts from patient advocacy organizations, industry, academia, and FDA.
The team conducted 45- to 60-minute telephone interviews of 32 senior-level leaders from patient groups, academia, and industry who could provide insights into the development of meaningful partnerships between industry and patients. The discussion questions for the interview were developed by the team in consultation with an independent qualitative research and professional moderator, and were partially informed by findings from a previously reported joint CTTI/DIA survey.
A report of the team’s findings from these interviews was presented during a January 2015 expert meeting of industry, academia, patient group, and government stakeholders. The meeting attendees provided feedback on the team’s qualitative research results, and worked in subgroups to help establish detailed recommendations for best practices for patient groups and sponsors.
Key Themes Identified
Ultimately, the team identified three key themes which characterize successful patient group engagement: (1) establishing meaningful partnerships, (2) demonstrating mutual benefits, and (3) collaborating early and often.
On the first theme, the interviewees noted that meaningful partnerships require choosing partners based on complimentary interests, capabilities, expertise, and resources. Sponsors look to the following factors when choosing patient group collaborators: the size of their constituency and reach; the effectiveness of their website; their social media savvy; the group’s track record; the extent of their assets; and their level of expertise in trial recruitment.
The second theme, demonstrating mutual benefits, was important to interviewees because these benefits can serve as bargaining chips for the patient group in exchange for meaningful involvement in the clinical trial process. Some commonly cited assets of patient groups were patient registries, tissue or blood banks, and a nuanced understanding of the target patient population.
Third, despite the historical practice of seeking patient group input late in the clinical trial process, interviewees noted that patient groups can add value to clinical trial accrual and retention if they provide input early in the planning process. This input gives research sponsors the ability to make trials more relevant, acceptable, and tolerable for patients.
Best Practices for Engagement
Using the data and input collected from interviews and the expert meeting, the PGCT team formulated recommendations for best practices for effective patient group engagement. The recommendations were grouped into three categories: recommendations for all stakeholders, recommendations for research sponsors (academic and industry), and recommendations for patient groups. Several of the recommendations are excerpted below:
- Engage the “patient voice” by establishing partnerships from the beginning of the research and development program to improve trial design and execution
- From the start, clearly define the expectations, roles, and responsibilities of all partners, including the resources being committed, data being shared, and objectives of the program
- Build the trust required for successful partnerships by being transparent and trustworthy, following through on commitments, and honoring confidentiality
- Ensure that [patient groups] are essential partners through the [research and development] process and not token voices
- Select sponsors who have a product or program with significant promise for your constituents and who are committed to engaging in a meaningful way
As suggested by the stakeholders who were interviewed and who attended the 2015 expert meeting, a significant barrier to patient engagement can be the lack of well-defined best practices and guidelines. These recommendations will hopefully address this barrier and serve as a helpful resource to research sponsors and patient groups seeking to collaborate to insert the patient voice into clinical trial design and medical product development.