Framework for Patient Input in Clinical Trial Design

The vision of the Medical Device Innovation Consortium (MDIC) Science of Patient Input (SPI) Steering Committee is to provide a venue for continued collaboration to advance the art and science of patient engagement in regulatory science, including advances in methodologies and tactical considerations for integrating the patient’s perspective and preferences in the design, clinical development and regulatory review of innovative medical technologies.

The MDIC SPI Committee proposes the development of a Framework for Patient Input in Clinical Trial Design with three complementary projects to integrate patient perspectives into the design and execution of medical device clinical trials

  • Methodologies to systematically identify outcomes that matter most to patients and establish these outcomes as primary or secondary endpoints for clinical studies
  • Guidelines to integrate patient preferences into the statistical design of clinical trials
  • Methodologies to maximize patient participation in clinical trials

The MDIC SPI Steering Committee has launched two new working groups to develop the Framework for Patient Input in Clinical Trial Design.

The Statistics and Outcomes working group is working on the development of:

  • White paper: Identifying outcomes that matter to patients. The goal of this project would be to identify methodologies and tools intended to systematically identify and prioritize outcome measures that matter to patients and incorporate them as primary or secondary endpoints in clinical studies of medical devices.
  • MDIC Workshop and Report on Patient-Centered Clinical Trial Design. The development of a proposed framework and guidance for the second project will build directly on ongoing work by MDIC and partners on the incorporation of patient preferences into the statistical design of clinical trials. A stakeholder conference will be held in Washington, DC on May 18, 2018 to learn from clinical trialists, biostatisticians, clinicians and patients regarding the development of these guidelines, report on the success of the MDIC PCOR project, discuss what needs to be done to encourage the wider adoption of the practice, and establish the next steps in advancing this methodology and the guidelines for this innovative methodology.

The Participation in Clinical Trials working group is working on the development of:

  • Guideline: “How my device trial impacts the lives of the patients involved – what to consider?” From protocol design and informed consent information to visit schedules and data collection, define elements of a device clinical trial that should be considered from the patient’s perspective when designing and planning the study.
  • Methodology: “Gaining relevant and actionable patient insights” Examples of how device companies CAN talk to patients. (e.g. use of patient agreements, market research approach etc.) and a summary of core approaches (e.g. survey, focus group, in-depth interview, social listening, co-creation/co-design) and patient access channels (e.g. patient advocacy groups, social media, patient communities) for gathering patient insights/preferences that can deliver relevant and actionable insights.
  • Guideline: “Novel / emerging solutions for reducing patient burden in clinical trials,” a summary of newer services / tools that have been emerging over the last few years to reduce patient burden of traditional trials (e.g. in-home study visits, telehealth, ePRO, e-consents, use of wearables / sensors, direct-to-patient contact centers).

The MDIC SPI Steering Committee has also launched a communication working group to review the literature and develop a white paper describing the challenges and best practices for communicating information regarding benefits, risks, and uncertainty to patients and providers.  This white paper, a complement to the MDIC Framework for Patient Preferences in Benefit/Risk Assessments, is intended as a resource for MDIC members and others who want to communicate such information to patients and providers effectively.

For more details, or to get involved, e-mail