Patient Group Engagement

overview icon Overview

Realizing the Value of Effective Patient Group Engagement

Patient groups have increasingly been recognized as equal partners in the clinical trials enterprise; however, there is uncertainty about how patients and research sponsors can best work together to improve the medical product development process. CTTI’s recommendations highlight important roles for patient groups throughout all stages of the process. These solutions also provide best practices that sponsors, patient groups, and other stakeholders can use to ensure the relationship is mutually beneficial.

To further support beneficial patient engagement, CTTI published a financial model that can be used to estimate the value of patient engagement on key business drivers such as cost, risk, revenue, and time—demonstrating that patient engagement can have a considerable impact on the bottom line. We also issued findings on the factors that sponsors and patient groups should consider when evaluating potential engagement activities. Having a clear example of how to calculate a return on investment is expected to support the broader uptake of patient engagement.

CTTI has also developed an online prioritization tool that patient groups and sponsors can use to identify high-value opportunities to work together. The tool lets users select relevant engagement opportunities for their project, rate the benefits and investments of each (low, moderate, high), visualize and adjust the analysis, and compare the output with their partner.

Through this project, CTTI continues to explore ways to support effective engagement of patient groups, leading to higher quality, patient-focused, and efficient clinical trials.

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Discover Additional Deliverables Generated From This Project


Tool 3. Assessment of PG External Relationships: Other PGs


Effective Engagement Between Sponsors & Patient Groups: A Structured Process from the Clinical Trials Transformation Initiative (CTTI)


The Rules of Engagement: CTTI Recommendations for Successful Collaborations between Sponsors and Patient Groups around Clinical Trials


Framework for Aligning Research Sponsors and Patient Groups on Methods for Engagement


Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey. PLoS ONE
*CLICK HERE to download the SPSS dataset for this publication.


Presenting CTTI Recommendations: Effective Engagement with Patient Groups Around Clinical Trials

Executive Summary

Summary of CTTI's Recommendations on Patient Groups & Clinical Trials


Patient Engagement in Neurological Clinical Trials Design: A Conference Summary published in the Clinical and Translational Science Journal

Expert Meeting Materials

Summary, presentations, and more from the Patient Groups and Clinical Trials Expert Meeting hosted on January 21- 22, 2015


Development and Application of a Patient Group Engagement Prioritization Tool for Use in Medical Product Development

“The CTTI PG&CT recommendations represent a true breakthrough in establishing and maintaining productive research partnerships between clinical trials sponsors and patient groups. These important and very practical recommendations serve to provide the framework for patient groups and sponsors to initiate contact as well as provide ways in which established partnerships can enhance their quality, productivity, and measured success.”

Joel Beetsch, PhD, VP, Global Patient Advocacy, Celgene Corporation

“I LOVE the PG&CT Recommendations!!! I have highlighted, dog-eared, written in margins and made slides for my Board of Directors meeting from them!!! It is like opening a cookbook and reading a recipe. They are tangible and somehow make me feel validated and encouraged. Thank you so much!”

Donna Appell, RN, CEO/Founder, Hermansky-Pudlak Syndrome Network Inc.

In the report, Key Considerations for Developing and Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study, by Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD), CTTI's Patient Groups & Clinical Trials work is cited and used as a case study.

"Some of the first formal efforts to outline the science of patient input borrow, from software development, the use of frameworks to provide a logical structure for organizing information, identifying sources of the information, and suggesting ways it might be used and viewed by distinct parties… The Clinical Trials Transformation Initiative (CTTI) created perhaps the most recognizable tool, and its work has become a guidepost.” (Source: On the path to a science of patient input)

Science Translational Medicine

in detail icon In Detail

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 enterprise.

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.

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

  • 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.
  1. Inform and optimize engagement with PGs around clinical trials so that the clinical trial process will be more efficient and quality driven.
  2. Gain a better understanding of the value proposition and 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
  • Test 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 trial enterprise. 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. CLICK HERE to read the publication of these findings.

At an expert meeting, attendees discussed the importance of integrating patient voices in the clinical trial enterprise, 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:

  • Contributing insight of 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 (eg, patient/public health, sponsor’s reputation)

The official CTTI recommendations provide a background to the current landscape of PG engagement in the clinical trial enterprise 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 trial enterprise by emphasizing expertise and assets
  • How and why to deliver PGs’ 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 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. Having a way to measure return on investment is expected to support the broader uptake of patient engagement strategies.


Project Team Members

Rolesort ascending Name Affiliation
Team Member Michelle Goldberg Johnson and Johnson Pharmaceutical Research and Development
Team Member Matthew Harker Duke University
Team Member Sharon Hesterlee Asklepios BioPharmaceutical, Inc
Team Member Bennett Levitan Johnson and Johnson Pharmaceutical Research and Development
Team Member Paulo Moreira* EMD Serono
Team Member Bray Patrick-Lake Duke University
Team Member Steve Roberds* Tuberous Sclerosis Alliance
Team Member Albert Roy Alliance for Lupus Research
Team Member Jeffrey Sherman Drug Information Association
Team Member James Valentine* Hyman, Phelps & McNamara
Team Member Scott Weir* University of Kansas
Team Member Ron Bartek* Friedrich's Ataxia Research Alliance
Team Member Joel Beetsch* Celgene
Team Member Patricia Cornet* Bristol-Myers Squibb
Team Member Joseph DiMasi Tufts University
Team Member Kenneth Getz Tufts University
Team Member Eric Eisenstein Duke University
Team Leader Linda Brennan Cystic Fibrosis Foundation
Team Leader Richard Klein* Food and Drug Administration
Team Leader David Leventhal Pfizer Inc
Team Leader Jaye Bea Smalley Celgene Corporation
Team Leader Sophia Smith Duke University
Social Science Lead Amy Corneli Clinical Trials Transformation Initiative
Project Manager Zachary Hallinan Clinical Trials Transformation Initiative