The value of co-creating a clinical outcome assessment strategy for clinical trial research: process and lessons learnt

Abstract

Abstract Background In support of UCB pharmaceutical research programs, the aim of this research was to implement a novel process for patient involvement in a multidisciplinary research group to co-create a clinical outcome assessment strategy to accurately reflect the experience of people living with early-stage Parkinson’s. Patient experts were an integral part of the decision-making process for patient-reported outcome (PRO) research and instrument development. Methods In partnership with two patient organizations (Parkinson’s UK and the Parkinson’s Foundation), 6 patient experts were recruited into a multidisciplinary research group alongside clinical, patient engagement and involvement, regulatory science, and outcome measurement experts. The group was involved across two phases of research; the first phase identified what symptoms are cardinal to the experience of living with early-stage Parkinson’s and the second phase involved the development of PRO instruments to better assess the symptoms that are important to people living with early-stage Parkinson’s. Patient experts were important in performing a variety of roles, in particular, qualitative study protocol design, conceptual model development, and subsequent co-creation of two PRO instruments. Results Involving people with Parkinson’s in PRO research ensured that the expertise of these representatives from the Parkinson’s community shaped and drove the research; as such, PRO instruments were being developed with the patient at the forefront. Working with patient experts required considerable resource and time allocation for planning, communication, document development, and organizing meetings; however, their input enriched the development of PRO instruments and was vital in developing PRO instruments that are more meaningful for people with Parkinson’s and clinicians. Conclusions Conducting PRO research, in the context of clinical development involving pharmaceutical companies, requires balancing regulatory and scientific rigor with tight time constraints. Incorporating a multi-stakeholder perspective, which included patient experts as joint investigators, had a strong positive impact on our research, despite the logistical complexities of their involvement. Due to the input of patient experts, the innovative clinical outcome assessment strategy and the co-created novel PRO instruments were more relevant and holistic to the patient experience of early-stage Parkinson’s. Graphical abstract