Advancing the Science of Patient Input
The science of patient input encompasses the development and use of systematic approaches, tools, and methodologies to collect, analyze, and apply robust and meaningful patient input towards medical product research and development (R&D) processes and regulatory decision making.
There is growing momentum to incorporate patient input into medical product R&D and regulatory decision-making processes. Converting traditionally anecdotal patient input to rigorous, credible evidence for use by a broad range of stakeholders—including academic and clinical researchers, medical product developers, patient/disease advocacy groups, and regulatory decision-makers—could better align medical product development and regulation with patient perspectives on disease experience, burden, management, and treatment. Many efforts have been launched to advance a science of patient input. However, there is a critical need to identify gaps in the knowledge base and other barriers that are impeding progress and develop a commonly held research agenda for addressing them.
This Collaborative project will foster development of a strategic research agenda to advance the science of patient input by hosting discussions that will:
- Catalogue current efforts and progress in the science of patient input;
- Identify critical gaps in the knowledge base and other barriers that are impeding progress; and
- Prioritize among identified gaps and barriers to develop a commonly held research agenda.
Input from experts and Collaborative participants during Phase 1 of the project supported a focus on and medical product R&D (see figure below). A research agenda for advancing the science of patient input during these stages would include consideration of downstream regulatory and post-market decision making. Covered elements could include:
- understanding of disease (e.g., natural history, disease burden, treatment burden, benefit–risk considerations, and target product profiles);
- clinical trial design (e.g., selection of endpoints, informed consent, and benefit–risk assessments); and
- data collection methodologies (e.g., crowd-sourcing, remote sensors, and digital and mHealth).
Contribution to the Field
- Catalog the current state of the science of patient input, including successes and limitations of current efforts for its application in medical product R&D.
- Highlight the types of patient input, as well as the tools and methodologies for collecting and analyzing this information, needed to provide evidence that is objective, quantitative, and standardized for use in medical product R&D and regulatory decision making.
- Develop a research agenda for addressing knowledge gaps and other barriers impeding progress in the science of patient input.
- Better align medical product discovery, development, and regulation with patient preferences for disease management and treatment.
The Collaborative is an ad hoc activity convened under the auspices of the Forum on Drug Discovery, Development, and Translation (the Forum) at the National Academies of Sciences, Engineering, and Medicine (the National Academies). The work of the Collaborative does not necessarily represent the views of any one organization, the Forum, or the National Academies and is not subjected to the review procedures of, nor is it a report or product of, the National Academies.