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. 2023 Oct 24;9(1):98.
doi: 10.1186/s40900-023-00505-7.

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

Thomas Morel  1 , Karlin Schroeder  2 , Sophie Cleanthous  3 , John Andrejack  2 , Geraldine Blavat  2 , William Brooks  2 , Lesley Gosden  4 , Carroll Siu  4 , Natasha Ratcliffe  4 , Ashley F Slagle  5
Affiliations

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

Thomas Morel et al. Res Involv Engagem. .

Erratum in

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.

Keywords: Clinical outcome assessment; Co-creation; Early-stage Parkinson’s; Outcome measure; PEIRS-22; Parkinson’s disease; Patient and public involvement; Patient engagement; Patient expert; Patient-focused drug development; Patient-reported outcomes.

Plain language summary

Patient-reported outcome (PRO) instruments allow people living with a disease and participating in a clinical study to describe the symptoms and experiences that they consider meaningful. PRO instruments use tools such as questionnaires and scales to capture patient perspectives on a treatment that might not be captured by a clinical measurement. It is recommended that the patient community and patient experts are included in the development of PRO instruments to accurately capture information that is important to them. Building on the experience of a recent PRO research project in support of UCB pharmaceutical programs, this article provides recommendations on how pharmaceutical companies can partner with patient organizations and involve patient experts as joint investigators in the co creation of PRO instruments. Despite the additional resource and time required, involving patient experts and patient organizations into the research collaboration had a strong positive impact and ensured that the PROs were meaningful to patients (in this instance, people living with early-stage Parkinson’s). Patient organizations facilitated patient engagement and recruitment in research activities, maintained communication with the pharmaceutical company’s research team, and built trust between collaborators by implementing patient engagement tools and best practices. Patient experts contributed to several parts of the PRO instrument development process: study design, identifying key symptoms and experiences, and developing individual PRO questions. Co-creation between the pharmaceutical company, patient experts, and patient organizations resulted in considerable improvements to typical PRO instrument development for use in clinical trials and is thus recommended.

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Conflict of interest statement

TM is an employee and shareholder of UCB. JA reports that they are a patient advocate in training, Parkinson’s Foundation (volunteer work, unpaid). NR and KS report receiving support from UCB for travel, lodging, and meals to attend one in-person, research team scientific meeting for the purpose of PRO development for this research. Since the completion of the research, NR has changed her affiliation from Parkinson’s UK, London, UK to COUCH Health, Manchester, UK and KS has changed her affiliation from Parkinson's Foundation, New York, NY, USA to Novartis, New Jersey, NJ, USA. LG has participated in a Glial Cell Line-Derived Neurotrophic Factor Advisory Board (working voluntarily with Parkinson’s UK as a patient expert). AFS reports receiving consulting fees from UCB for advisory services related to outcome assessment development and patient engagement in early-stage Parkinson’s; receiving payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from UCB for a clinical outcomes assessment regulatory workshop presentation that was provided to the patient experts who were involved across this research; receiving travel reimbursement from UCB for attending face-to-face meetings that were relevant to this manuscript. All authors report receiving support from UCB for funding the study and third-party medical writing assistance.

Figures

Fig. 1
Fig. 1
The Patient engagement model. This Patient engagement model, adapted from de Wit et al. [20] describes the levels of patient involvement throughout the development of a PRO strategy for early-stage Parkinson’s, ensuring that patient participation was meaningful at every stage of this process. COA clinical outcomes assessment, PRO patient-reported outcome
Fig. 2
Fig. 2
Patient roles for study design, identifying concepts of interest, and evaluating legacy clinical outcomes assessments. The figure displays the extent of patient contribution [–28], which includes PRO research design, development of the conceptual model of the early-stage Parkinson’s experience, and appraisal of existing COAs in this context of use and identification of cardinal concepts. In addition to these patient roles, interviewees in phase I acted as ‘patient discussants’: 50 people living with early-stage Parkinson’s and 9 caregivers were interviewed to elicit concepts of interest and to appraise pre-existing PRO instruments, namely the MDS-UPDRS parts Ib and II and the PDQ-39. COA clinical outcomes assessment, DMT disease-modifying therapy, PDQ-39 Parkinson’s Disease Questionnaire, PIP Patient Involvement Plan, PRO patient-reported outcome, MDS-UPDRS Movement Disorder Society-Unified Parkinson’s Disease Rating Scale
Fig. 3
Fig. 3
Patient roles in patient-reported outcomes development and scientific dissemination. The figure displays the levels of patient contribution to PRO instrument development iterations and scientific communications. In addition to these patient roles, interviewees in phase II acted as ‘patient discussants’: 60 people living with early-stage Parkinson’s were interviewed for cognitive debriefing activities on the preliminary PRO instrument item set and other PRO instruments. F2F face-to-face, PRO patient-reported outcome
Fig. 4
Fig. 4
Patient Engagement In Research Scale (PEIRS) scores across phases I and II. PEIRS scores measure the degree of meaningful patient engagement across both stages of this COA research, from a patient perspective. The PEIRS-22 scoring manual [32] distributes the 22 items of the PEIRS-22 across three levels of meaningful engagement in research. Advanced engagement (gold level) is the most difficult to achieve as positive experiences of those important elements of meaningful engagement are least often reported by patient experts. Intermediate engagement (silver) and foundational engagement (bronze) reflect the aspects that are moderately and least difficult to achieve, respectively. COA clinical outcomes assessment, PEIRS Patient Engagement In Research Scale
Fig. 5
Fig. 5
Lessons for the future. F2F face-to-face, PO patient organizations, PEIRS Patient Engagement In Research Scale
See this image and copyright information in PMC

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