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 groups, and regulatory decision-makers—could better align medical product development and regulation with patient perspectives on disease experience, burden, management, and treatment. The science of patient input has continued to advance thanks to numerous ongoing efforts. At the same time, integration of the science of patient input across the R&D life cycle has been hampered by gaps in research, siloing of ongoing efforts, and a lack of metrics to demonstrate impact.

Objectives

The Advancing the Science of Patient Input in Medical R&D Action Collaborative (the collaborative), an ad hoc activity associated with the National Academies Forum on Drug Discovery, Development, and Translation, will lay the groundwork for a research agenda to advance the science of patient input by hosting discussions to:

1. Catalogue current efforts to advance the science of patient input;
2. Identify critical gaps in the knowledge base and other barriers that impede progress;
3. Prioritize research questions that should be addressed; and
4. Lay out next steps to help advance the field.

Project Scope

Input from collaborative participants and external stakeholders will focus on pre-market medical product R&D and cover the following topics of interest:

• Understanding the patient experience with a disease or medical condition;
• Capturing patient perspectives and preferences on benefit-risk;
• Incorporating patient input in clinical trial development and continuous improvement.

Workplan

With the participation of stakeholders involved in clinical trials, this collaborative aims to:

• Survey experts to inform scoping decisions about priority areas to address in workshop discussions and proposed research agenda. [complete]
• The Forum on Drug Discovery, Development, and Translation hosts a workshop, Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda. [occurred May 2018]
• Conduct follow-up strategic discussions to set priorities for a research agenda to advance the science of patient input. [ongoing]

Potential Contributions 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 useful across the spectrum of medical product R&D and regulatory decision making.
• Lay the foundation for 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 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.