DIA-PCORI

View Model & Webinar

Project Summary

Benefit-risk assessments of medical products, the weighing of benefits against the risks for harm from using a product for treatment, are the foundation for making decisions about the product throughout its life cycle. For a medical product to truly meet the needs of the patient for whom it is intended, its benefits, risks, and uncertainties must be balanced in the context of the patient’s perspective, requiring both technical assessments of the evidence base and stakeholders’ value judgments about relative importance. Because patients are the beneficiaries of effective treatments and also bear the risks associated with those treatments, their perspectives and judgments about value and relative importance are at the heart of this process.

In many cases, patients or patient partners (patients, family, caregivers, advocates, and patient organizations) are not engaged effectively or at all in the benefit-risk assessment process, especially in the early development stages of medical products. This will change only with widespread awareness of the importance of patient engagement and with collaboration among all research stakeholders, including patients, to develop and adopt more effective engagement tools and processes.

The conference “Patient Engagement in Benefit-Risk Assessment Throughout the Life Cycle of Medical Products” was targeted to patient partners, industry and academic medical researchers, and regulators, and addressed the important challenge of how and when to best engage patient partners in benefit-risk assessment. The overall goals of the conference were to:

1. Raise awareness of these stakeholders of the importance of patient engagement in benefit-risk assessment throughout the life cycle of the medical product
2. Involve the stakeholders in sharing of existing approaches, identifying implementation challenges and gaps or needs for new information, and best practices
3. Identify recommended next steps for addressing the identified gaps to inform stakeholder actions, including current legislative and regulatory processes

The outputs from this project include the conference summary and briefing materials, including a visual model, that can be disseminated for education and awareness building, practice assessment, problem-solving, and system improvement, and used to inform the development of new knowledge, best practices, and guidelines to address gaps in this area. A manuscript on cultural transitions necessary to effect change among all stakeholders has been published.

Project collaborators include Program Committee representatives from Amgen; AstraZeneca; Duke Clinical Research Institute; Eli Lilly and Company; FasterCures; GlaxoSmithKline; Health Canada; Janssen Research & Development; JDRF; Johns Hopkins Bloomberg School of Public Health; Merck Research Laboratories; Parent Project Muscular Dystrophy (PPMD); T1D Exchange; and the University of Colorado/Colorado School of Public Health. Graphic facilitation was provided by Visual Ink, LLC.

More on This Project

Project Deliverables:

• Visual Model of Patient Engagement in Benefit-Risk Assessment
• Model Webinar
• Manuscript: Patient Engagement at a Tipping Point— The Need for Cultural Change Across Patient, Sponsor, and Regulator Stakeholders: Insights From the DIA Conference, ‘‘Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle of Medical Products" Therapeutic Innovation & Regulatory Science September 2016 vol. 50 no. 5 546-553.

Project Resources:

• Conference Program
• Conference Short Summary
• Dissemination Plan