ENGAGE EARLY. ENGAGE OFTEN.
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. Having a clear example of how to calculate a return on investment is expected to support the broader uptake of patient engagement.
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.
Joel Beetsch, PhD, VP, Global Patient Advocacy, Celgene Corporation
“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.”
Donna Appell, RN, CEO/Founder, Hermansky-Pudlak Syndrome Network Inc.
“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!”
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.
Science Translational Medicine
"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)
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.
PGCT: Best Practices for Effective Engagement with Patient Groups around Clinical Trials (2013 - ongoing)
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:
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:
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.
|Team Member||Albert Roy||Alliance for Lupus Research|
|Team Member||Sharon Hesterlee||Asklepios BioPharmaceutical, Inc|
|Team Member||Patricia Cornet*||Bristol-Myers Squibb|
|Team Member||Joel Beetsch*||Celgene|
|Team Leader||Jaye Bea Smalley||Celgene Corporation|
|Project Manager||Zachary Hallinan||Clinical Trials Transformation Initiative|
|Social Science Lead||Amy Corneli||Clinical Trials Transformation Initiative|
|Team Leader||Linda Brennan||Cystic Fibrosis Foundation|
|Team Member||Jeffrey Sherman||Drug Information Association|
|Team Member||Eric Eisenstein||Duke University|
|Team Member||Matthew Harker||Duke University|
|Team Member||Bray Patrick-Lake||Duke University|
|Team Leader||Sophia Smith||Duke University|
|Team Member||Paulo Moreira*||EMD Serono|
|Team Leader||Richard Klein*||Food and Drug Administration|
|Team Member||Ron Bartek*||Friedrich's Ataxia Research Alliance|
|Team Member||James Valentine*||Hyman, Phelps & McNamara|
|Team Member||Michelle Goldberg||Johnson and Johnson Pharmaceutical Research and Development|
|Team Member||Bennett Levitan||Johnson and Johnson Pharmaceutical Research and Development|
|Team Leader||David Leventhal||Pfizer Inc|
|Team Member||Steve Roberds*||Tuberous Sclerosis Alliance|
|Team Member||Joseph DiMasi||Tufts University|
|Team Member||Kenneth Getz||Tufts University|
|Team Member||Scott Weir*||University of Kansas|