Patient & Public Involvement & Engagement

A key challenge in rare disease research has been the insufficient emphasis on Patient and Public Involvement and Engagement (PPIE), particularly in ensuring that patients and their families are fully integrated into the decision-making processes. Traditionally, governance in medical research has focused on protecting vulnerable patients, often overlooking the critical role that people living with rare diseases (PLWRD) play in driving the research agenda. Historically, patient involvement has often been superficial, with researchers maintaining control, leaving out vital patient perspectives that could enhance every stage of the research process. This lack of involvement is particularly evident in clinical trials for rare diseases, where the unique knowledge and insights of PLWRD and their carers are too frequently missed.

WPA aims to transform this landscape by embedding PPIE at every level of the RD-TAP. Our approach will centre on appointing PLWRD at the highest levels of governance, ensuring they are integral to decision-making. We will work to embed PPIE into each work package, promoting active involvement of patients in shaping research priorities and strategies. Our focus will also include researching patient expectations around the acceleration of rare disease translational research across the four UK nations and undertaking public engagement events to increase awareness and understanding of the challenges faced by PLWRD.

By placing patients at the heart of the research process, we aim to empower them to influence the direction of scientific advancements, leading to more relevant, impactful, and ethical research outcomes in rare diseases. This approach will not only ensure that the voices of patients and families are heard but also improve the overall quality and inclusiveness of the research, contributing to better patient outcomes and more sustainable research practices.