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.
Converting traditionally anecdotal patient input into rigorous, credible evidence for use by a broad range of stakeholders could better align medical product research and development (R&D) and regulation with patient experience of and preferences for disease management and treatment. Accordingly, many efforts have been launched to advance a science of patient input—the development and use of systematic approaches and tools to collect, analyze, and apply patient input to the medical product R&D lifecycle and regulatory decision-making processes. Despite significant progress made in highlighting the value and impact of patient input throughout these processes, and momentum towards the development of a science therein, there is a critical need to discuss gaps in the knowledge base and other barriers that are hindering advancement of a science of patient input and explore a research agenda for addressing them.
The Advancing the Science of Patient Input action collaborative (the Collaborative) is an ad hoc activity associated with 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.
This Collaborative will foster development of a strategic research agenda to advance the science of patient input by hosting discussions to:
- 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 supported a focus on 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.