Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion

BACKGROUND: Drug development practices for rare diseases promote a promissory notion that the drug pipeline will succeed (with the next new drug/drug trial) and an imaginary of widening patient engagement (often desired but yet to be enacted). Contemporary industry practices of patient engagement are shaped by this promissory, and engagement often includes limited patient perspectives, typically relying on patients from established Patient Organisations or identified by Contract Research Organisations. OBJECTIVE: We sought to explore whether more deliberative methods might enable wider, more diverse patient engagement for orphan drug trials. DESIGN: Citizen's Jury co-designed with patient advisors. SETTING AND PARTICIPANTS: Results of an earlier ethnography of a biotech company's patient engagement practices, along with findings from clinical trials for patients with idiopathic pulmonary fibrosis, clinical practice and patient testimony, were presented to the Citizens' Jury made up of patients and carers. RESULTS: Jurors discussed and offered a 'verdict' on trial materials and processes that would optimise the engagement of more diverse patients for drug trials for rare diseases, suggesting that more could be done at an organisational level to ensure that potential trial participants were able to 'engage'. They asserted that the industry could do more to understand the unmet needs and wishes of a wider group of patients and should seek input from more diverse groups. DISCUSSION: The Citizens' Jury called for practices to enable wider engagement-for both drug trials and drug trial design-and more transparency about the risks associated with engagement, for individual patients and currently marginalised groups. CONCLUSIONS: Current drug development practices reify expert patient perspectives and often ignore the views of the wider group of patients who may participate in trials and/or use new medicines developed. More deliberative methods of engagement have the potential to democratise drug development and ensure that new medicines and trials meet the needs of a broader patient demographic. PATIENT OR PUBLIC CONTRIBUTION: A patient advisory group (PAG) comprising six people with IPF gave input into all aspects of the research design and conduct, including co-design of the Citizens' Jury. Two patients from international patient organisations served as a steering group (SG). Members of both groups provided their interpretations of the study findings and gave insight into their experiences in clinical design and participation.

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