The patient community has been taking a more active role in clinical trials and is rapidly evolving in its areas of focus, sophistication about the research process, level of expertise, reach, and desired outcomes when pursuing research agendas. Thus, patient groups (PGs), defined here as patient advocacy organizations, voluntary health agencies, public health organizations, and their affiliated members, have been increasingly recognized as equal partners in the clinical research ecosystem.

However, gaps in knowledge and understanding about how and when to best interact with PGs have the potential to delay trials or lead to less efficient trials. Complex legal, ethical, and regulatory issues and ill-defined expectations can lead to unproductive relationships and disparate or unanticipated outcomes. While key stakeholders had committed to create a more effective model for engagement between research sponsors, investigators, and PGs, leading to better clinical trials, guidelines for best practices were needed. CTTI’s diverse membership was well-positioned to generate these recommendations through its collaborative problem-solving model.

Metrics and evidence on return on investment are needed to drive adoption of best practices for PG engagement. CTTI’s literature review, survey, and interviews revealed that there are no widely accepted models for assessing the value and impact of PG engagement. Thus, CTTI developed an economic model to provide an evidence base for PG engagement methods in clinical trials.

PGCT: Best Practices for Effective Engagement with Patient Groups around Clinical Trials (2013 - 2015)

• Understand the nature of PGs and their engagement in clinical trials
• Understand current practices for how research sponsors engage PGs
• Identify best practices for engaging PGs that will lead to more efficient and successful clinical trials
• Understand the value and impact of engaging patients in clinical trials

• Inform and optimize engagement with PGs around clinical trials so that the clinical trial process will be more efficient and quality driven
• Gain a better understanding of the value proposition for engaging PGs at each stage of the development process to drive adoption of engagement practices

• Landscape and literature reviews of current practices and tools
• Survey and semi-structured interviews
• Assessment of previous engagement efforts
• Expert meeting
• Developed an economic model on the value of patient engagement

PGs foster a trusted relationship with patients, but their characteristics, capabilities, assets, and research sophistication vary widely.

Preliminary evidence from the literature review, survey, and interviews revealed that there are currently no widely accepted metrics or models for assessing the value and impact of PGs on the clinical trials ecosystem. Little empirical data was found in the literature to define or optimize key factors for successful PG relationships or to provide accepted metrics to support the value proposition of such relationships for sponsors. Survey responses indicated that approximately a third of polled sponsor organizations are currently engaged with PGs, and the same proportion of sponsor organizations had no plans of fostering such a collaboration in the future. Primary motivators for PG engagement include enhancing corporate culture or therapeutic areas in industry and gaining funding in academia. Barriers to sponsor-PG engagement included a lack of tools or unclear processes for PG engagement, a lack of understanding of the benefits of PG engagement, insufficient transparency or openness, and misaligned/unaligned priorities, objectives, or incentives. Read the publication for more details on these findings.

At an expert meeting, attendees discussed the importance of integrating patient voices in the clinical trials ecosystem, the roles of PGs in all stages of clinical trials, and best practices for PG engagement and sponsor-PG relationships. Both companies and PGs need to assess their organizations’ goals and objectives in relation to others’. Once a collaboration is initiated, PGs can influence early and late stages of drug development. Some ways in which PGs facilitate trial planning and conduct include the following:

• Contribute insight into the daily struggles with a particular disease condition
• Contribute to a more successful trial design
• Raise awareness about a trial, thereby increasing participant recruitment
• Provide or direct funding
• Assist with creating the informed consent form
• Provide feedback on meaningful clinical endpoints
• Report feedback from patients on sites, investigators, and participant experiences
• Serve on an FDA advisory committee
• Help return study results to participants
• Facilitate or ease operational issues that can improve cost, timing, technical/regulatory risk, and compliance and provide other benefits (e.g., patient/public health, sponsor’s reputation)

The official CTTI recommendations provide a background to the current landscape of PG engagement in the clinical trials ecosystem and detailed considerations and best practices for PG involvement and relationship building. Briefly, these recommendations describe the following:

For all stakeholders:

• How and why to engage the “patient voice” and establish partnerships to increase trial efficiency and quality
• How and why to define the expectations, roles, and responsibilities of all partners
• How and why to build trust and rapport and establish a culture of transparency
• How and why to gain a broader perspective
• How and why to manage conflicts of interest

For sponsors:

• How and why to integrate PG involvement in ongoing research
• How and why to match PG expertise to the specific needs of a clinical program
• How and why to ensure that PGs are full, essential partners throughout the research and development process
• How and why to establish guiding principles for communication and collaboration with PGs
• How and why to measure the impact of PG engagement
• How and why to establish ongoing relationships and a continual, open dialogue with PGs

For PGs:

• How and why to identify stakeholders with R&D interests relevant to a specific cause and integrate the patient voice
• How and why to promote a PG as an essential partner in the clinical trials ecosystem by emphasizing expertise and assets
• How and why to deliver expertise throughout the entire research and development process
• How and why to select and encourage continual engagement with sponsors who are developing a product/program that hold promise for PG constituents
• How and why to manage conflicts of interest by establishing policies to require full disclosure

Within the recommendations document, tools are embedded to assist with evaluation of PG expertise and assets, assessment of PG internal considerations, and assessment of PG external relationships.

To address the lack of metrics for assessing the financial value of patient engagement, CTTI also developed a framework to estimate the impact of patient engagement on key business drivers such as cost, risk, revenue, and time. CTTI’s published work shows how expected net present value (ENPV) can be used to demonstrate that patient engagement can contribute considerable financial value.

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.