Widening patient engagement for rare disease drug trials: The perspectives of patients with Idiopathic Pulmonary Fibrosis on participating in clinical drug trials and drug trial design

Abstract

Background: Research about patient engagement for people with rare diseases has identified how the experiences of some members of the public are overlooked in relation to clinical trial design and trial participation. As part of a knowledge transfer partnership (KTP), the authors were granted access to patient insight reports about the needs of people with idiopathic pulmonary fibrosis (IPF), to inform clinical trial design and marketing strategy. These were contrasted with data from qualitative interviews, informed by, and collected from, people with IPF and the clinical staff who recruit them to trials. Objective: To identify patient and professional perspectives for IPF drug trials, to create opportunities for innovation in patient engagement. Design: Ethnography. Qualitative researcher embedded in a pharmaceutical organisation. Setting and participants: International patient insight reports to inform a clinical trial protocol (n=1) and marketing strategy (n=6), including the experiences of over 100 patients with IPF. In the UK, interviews with patients with IPF (n=32) and the staff who support them clinically and recruit them to trials of new medicines (n=19) at one specialist Interstitial Lung Disease (ILD) Centre. Results: Methodological practices inherent in inpatient insight reports ensured the perspectives of some people with IPF were overlooked. Interviews with a more marginalised population of people with IPF, and the staff who support them, identified that some found trial information confusing, trial practices frustrating, and the opportunities to engage in trial design absent. Discussion: Current pharmaceutical practices of working with contract research organisations and patient organisations exclude the perspectives of patients with IPF who do not engage with either. Trial recruitment information needs to be tailored to the needs of individuals and trial processes need to enable a wider group of patients to participate. Conclusions: People with IPF want the opportunity to participate in drug trials and trial science. However, methodological rigour and deliberative practices are required to enable a wider group of patients to have a stake in the design and conduct of drug trials for rare diseases. The challenge now is for regulators to mandate for such inclusive practices, and for pharmaceutical organisations to adopt them.