Collaboration between patient organisations and a clinical research sponsor in a rare disease condition: learnings from a community advisory board and best practice for future collaborations

Abstract

Introduction Transparent collaborations between patient organisations (POs) and clinical research sponsors (CRS) can identify and address the unmet needs of patients and caregivers. These insights can improve clinical trial participant experience and delivery of medical innovations necessary to advance health outcomes and standards of care. We share our experiences from such a collaboration undertaken surrounding the SENSCIS® clinical trial (NCT02597933), and discuss its impact during, and legacy beyond, the trial.Summary We describe the establishment of a community advisory board (CAB): a transparent, multiyear collaboration between the scleroderma patient community and a CRS. We present shared learnings from the collaboration, which is split into three main areas: (1) the implementation and conduct of the clinical trial; (2) analysis and dissemination of the results; and (3) aspects of the collaboration not related to the trial.1. The scleroderma CAB reviewed and provided advice on trial conduct and reporting. This led to the improvement and optimisation of trial procedures; meaningful, patient-focused adaptations were made to address challenges relevant to scleroderma-associated interstitial lung disease patients.2. To ensure that results of the trial were accessible to lay audiences and patients, written lay summaries were developed by the trial sponsor with valuable input from the CAB to ensure that language and figures were understandable.3. The CAB and the CRS also collaborated to co-develop opening tools for medication blister packs and bottles. In addition, to raise disease awareness among physicians, patients and caregivers, educational materials to improve diagnosis and management of scleroderma were co-created and delivered by the CAB and CRS.Conclusions This collaboration between POs and a CRS, in a rare disease condition, led to meaningful improvements in patient safety, comfort and self-management and addressed information needs. This collaboration may serve as a template of best practice for future collaborations between POs, research sponsors and other healthcare stakeholders.

Patient involvement in rare diseases research: a scoping review of the literature and mixed method evaluation of Norwegian researchers’ experiences and perceptions

“…95% had also had undertaken research activity such as: financing, lobby A questionnaire survey was used for evaluating the impact of PI PI may improve lack of financial resources, lack of interest on rare diseases, increase the recruitment rate and registry. Therefore important that researchers must work with Patient-Organizations The study reported that PI is important and could help advance scientific knowledge and therapy, thereby alleviating the personal and societal burden of RDs Described PI in research dissemination 78% reported being involved in disseminated of research, such as doing it available at the web site RDPO websites showed that leaders had compiled information about PI in current research projects and findings PI had increased the use of more relevant research dissemination channels Interviewed leaders gave examples of how this information had led organizational members to develop ideas for further research, which they then used in discussions with researchers or as a basis for awarding research funding Roennow et al [ 36 ]. Collaboration between patient organisations and a clinical research sponsor in a rare disease condition: learnings from a community advisory board and best practice for future collaborations BMJ Open International collaboration Qualitative design with communication of experiences To share our experiences from such a collaboration undertaken surrounding the SENSCIS® clinical trial (NCT02597933), and discuss its impact during, and legacy beyond, the trial Diagnosis: scleroderma / systemic sclerosis 15 patient representatives from Scleroderma patient associations from 10 countries Describes establishment of a community advisory board (CAB): a transparent, multiyear collaboration between the scleroderma patient community and a clinical research sponsor A community advisory board was established where patient offer their expertise to clinical research sponsor to discuss overall program development.…”

“…Roennow et al [ 36 ]. Collaboration between patient organisations and a clinical research sponsor in a rare disease condition: learnings from a community advisory board and best practice for future collaborations…”

“…All 12 primary articles were all published in English, conducted between 2012 [ 31 ] and 2021 [ 35 ], and originating from USA (n = 4), Europe (n = 4), Oceania (n = 3) and Japan (articles = 1). The studies described PI in research of different types of RDs: neuromuscular diseases [ 32 , 33 ], congenital hypogonadotropic Hypogonadism /Kallmann syndrome [ 11 ], Fontan disease [ 34 ], eosinophilic gastrointestinal disorder [ 35 ], scleroderma disease [ 36 ], vasculitis disease [ 12 ], Pachyonychia Congenita [ 37 ], skeletal muscle channelopathies and hereditary angioedema [ 13 ], and rare disease in general [ 31 , 38 , 39 ]. Both patients, patient representatives and patient organization representatives were involved.…”

“…There was vide variation of design and approaches of PI in the different studies. Most articles [ 11 , 12 , 32 – 34 , 36 , 37 ] used qualitative design and three [ 13 , 35 , 39 ] had mixed-methods design, combining participatory research, surveys and online meetings. The PI-approaches were based on participatory active research methods, using work-shops groups, individual interviews, Delphi methods, participation in Consortiums, “partnership identification methods” and “shared decision making”.…”

Digital Transformation in Medical Affairs Sparked by the Pandemic: Insights and Learnings from COVID-19 Era and Beyond

“…There has been a gradual shift from a disease-centred to a patient-centred strategy and from a product-led to a patient-led development process [ 18 ]. While generally no direct-to-patient advertising is allowed (except in USA, Hong Kong and New Zealand, with some restrictions) [ 1 ], patient advocacy groups play an important intermediary partner for medical and public affairs [ 19 , 20 ]. Other endeavours, including studying and collecting patient-reported outcomes in clinical and especially real-world studies [ 21 , 22 ], leading tailored disease awareness and patient education campaigns [ 23 , 24 ], real-time diagnostic and treatment analytical support [ 25 ] have helped to not only empower patients but also generate valuable real-world data [ 26 – 29 ].…”