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

Abstract

Objective:To identify the elements necessary for successful collaboration between patient groups and academic and industry sponsors of clinical trials, in order to develop recommendations for best practices for effective patient group engagement.Methods:In-depth interviews, informed by a previously reported survey, were conducted to identify the fundamentals of successful patient group engagement. Thirty-two respondents from 3 sectors participated: patient groups, academic researchers, and industry. The findings were presented to a multistakeholder group of experts in January 2015. The expert group came to consensus on a set of actionable recommendations for best practices for patient groups and research sponsors.Results:Interview respondents acknowledged that not all patient groups are created equal in terms of what they can contribute to a clinical trial. The most important elements for effective patient group engagement include establishing meaningful partnerships, demonstrating mutual benefits, and collaborating as partners from the planning stage forward. Although there is a growing appreciation by sponsors about the benefits of patient group engagement, there remains some resistance and some uncertainty about how best to engage. Barriers include mismatched expectations and a perception that patient groups lack scientific sophistication and that “wishful thinking” may cloud their recommendations.Conclusions:Patient groups are developing diverse skillsets and acquiring assets to leverage in order to become collaborators with industry and academia on clinical trials. Growing numbers of research sponsors across the clinical trials enterprise are recognizing the benefits of continuous and meaningful patient group engagement, but there are still mindsets to change, and stakeholders need further guidance on operationalizing a new model of clinical trial conduct.

Reviewing the role of healthy volunteer studies in drug development

“…In 2018, the Clinical Trials Transformation Initiative published information about the potential advantages of patient engagement. Patient engagement efforts can result in enhanced clinically relevant hypotheses, assist in identifying relevant measurements for patient outcomes, limit time and emotional burden for research participation, and lead to improvements in recruitment and perhaps more importantly retention in clinical studies [ 79 ]. Although many aspects of patient engagement with researchers are motivated by specific interests in their own disease or that of a family member, many of the learnings from recent patient engagement efforts are directly applicable to HV studies.…”

Factors and Situations Affecting the Value of Patient Preference Studies: Semi-Structured Interviews in Europe and the US

“…PPI can be used in every phase of the MPLC (Medical Device Innovation Consortium (MDIC), 2015; Marsh, 2016). Some examples of applications include: identifying unmet medical needs (Medical Device Innovation Consortium (MDIC), 2015; FDA, 2016; Selig, 2016), informing the selection of endpoints, and informing benefit-risk assessments (Medical Device Innovation Consortium (MDIC), 2015; FDA, 2016; Stamuli et al, 2017; Bloom et al, 2018) and in HTA (Kievit et al, 2017; Muhlbacher and Sadler, 2017; Marsh et al, 2018). PPI can give insights into the trade-offs that patients make between benefits and risks and show the relative importance of outcomes for patients (Puhan et al, 2015; Minion et al, 2016; Marsh et al, 2017).…”

Giving Voice to Clinical Study Participants: Development and Deployment of Sequential Patient Experience Surveys for Global Clinical Studies

“…There is increasing recognition that structured and systematic patient engagement may help improve clinical studies [9][10][11][12][13]. Initiatives from the Food and Drug Administration (Patient Focused Drug Development) [11], Clinical Trials Transformation Initiative [9,10], European Patients' Academy on Therapeutic Innovation (EUPATI) [14], Trans-Celerate Biopharma Inc. (Patient Experience Initiative) [15], Tammy S. Ice and Daphnee S. Pushparajah Employed by UCB Pharma at the time this research was undertaken.…”

“…There is increasing recognition that structured and systematic patient engagement may help improve clinical studies [ 9 – 13 ]. Initiatives from the Food and Drug Administration (Patient Focused Drug Development) [ 11 ], Clinical Trials Transformation Initiative [ 9 , 10 ], European Patients’ Academy on Therapeutic Innovation (EUPATI) [ 14 ], TransCelerate Biopharma Inc. (Patient Experience Initiative) [ 15 ], Innovative Medicines Initiative (Patients Active in Research and Dialogues for an Improved Generation of Medicines—PARADIGM) [ 12 ], and Patient Focused Medicines Development [ 16 ], among others, aim to build practical frameworks to guide patient engagement.…”