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”

Abstract

Benefit-risk assessment is the foundation for decision making throughout the life cycle of medical products. Because patients are the beneficiaries of the efficacy of medical treatments and also bear their possible risks, their perspectives and judgments about value and the relative importance of benefits and risks should be at the heart of the medical decision-making process. Patient engagement is now at a tipping point; there have been a growing number of patient engagement initiatives over the past several years, but there remains the need for a common language, alignment on engagement approaches and best practices, and a shared vision regarding a desired future state. This article discusses insights gleaned from the DIA conference, ''Patient Engagement in Benefit-Risk Assessment throughout the Life Cycle of Medical Products'' (September 2015). It highlights the changes that will need to occur within the patient, medical-product sponsor, and regulatory cultures in order for patient engagement to become integrated into the medical-product development process and life cycle maintenance. Furthermore, it emphasizes that reaching the desired future state will require a conscious commitment from all stakeholders to work collaboratively to develop shared solutions and to map a common path forward.

What do stakeholders expect from patient engagement: Are these expectations being met?

“…PE in the research and development setting especially has received much focus with the development of frameworks or guidance . There are also guidance or frameworks at other milestones such as in health technology appraisal, benefit‐risk assessment and in value determination . The issue of definition and terminology of PE and patient centricity has also highlighted the need for a common understanding to facilitate multistakeholder teamwork .…”

Facing the Ethical Challenges: Consumer Involvement in COVID-19 Pandemic Research

“…Consumer involvement provides a direct opportunity in this context to help researchers, clinicians, and multi-stakeholder groups resolve uncertainties about the design of pandemic research and the appropriateness of the risk-benefit balance proposed (Smith et al, 2016 ). For example, a common contentious issue is the use of health data, yet the need for data to understand and address the pandemic is unprecedented.…”

Mapping the Landscape of Patient-centric Activities Within Clinical Research

“…eConsent was mentioned as the top planned initiative (11 companies). Other planned initiatives mentioned by less than half of the group (10 companies) were adaptive trial designs and adaptive licensing; patient communities during and after clinical trials were also reported to be a planned initiative (11). Medicine co-development partnerships were reported by many companies as not being considered.…”

“…There are a number of challenges with the use of mobile health applications, including data security and privacy, data qualification, and validation and regulatory acceptance. 10 Smith et al 11 discussed the need for a common language, alignment, and best practices around patient engagement. The Patient-Centered Outcomes Research Institute (Washington, DC) has developed general guidance through their Engagement Rubric, which describes how input from patients and other stakeholders can be gathered throughout the research cycle.…”