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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.

SUBMITTER: Roennow A

PROVIDER: S-EPMC7745690 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature

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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.

Roennow Annelise A Sauvé Maureen M Welling Joep J Riggs Robert J RJ Kennedy Ann Tyrrell AT Galetti Ilaria I Brown Edith E Leite Catarina C Gonzalez Alex A Portales Guiraud Alexandra Paula AP Houÿez François F Camp Rob R Gilbert Annie A Gahlemann Martina M Moros Lizette L Luna Flores Jose Luis JL Schmidt Friedrich F Sauter Wiebke W Finnern Henrik H

BMJ open 20201216 12

<b>Introduction</b> 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<sup>®</sup> clinical trial (NCT02597933), and discuss its impact ...[more]

PMID: 33328257

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Project description:AimsThe aim of this paper is to present a novel case for the formation, operation and evaluation of a community advisory aboard comprised of Muslims residing in the San Francisco Bay Area, California that utilised a community based participatory approach to address local Muslim mental health needs. The CAB was recruited in partnership with the Muslim Community Association (MCA), one of the largest Islamic centres in the San Franscisco Bay Area. In addition to describing the development of the CAB, the authors present the findings of the evaluation and synthesis of best processes based on CAB members' feedback.MethodsTo evaluate the perceived community advisory board members' perceptions of their roles and elicit feedback on how to enhance the relationship between the university team and the CAB, an evaluation was conducted by an independent team who was not part of the research process. Data was collected using anonymous individual surveys and small group open discussions that were conducted over three evaluation meetings. The evaluation utilised mixed method data collection strategies using questions from Schulz et al. (, Evaluation and Program Planning 26, 249-262), an instrument for evaluating dimensions of group dynamics within CBPR partnerships.ResultsResults of the evaluation within the sphere of CAB operation indicated that CAB members found the greatest satisfaction from their contributions through direct participation in the research activities that were conducted by the university-CAB team. The collective responses indicated that most CAB members were satisfied with trust built between the university-CAB team and the diversity represented in the members of the board. However, given that the Bay Area is home to a very diverse Muslim community, challenges in recruiting representatives that account for all possible self-identifying groups was reported by the CAB with recommendations to recruit religious leaders. Recommendations also included eliciting funds for potential financial compensation for CAB members.ConclusionsThe Stanford-San Francisco Bay Area CAB demonstrated that empowering community members through direct participation, creating channels and safe spaces for feedback help create community rooted research that carry the true voices of marginalised communities and reflects their evolving needs.

Project description:INTRODUCTION:Cyclopia (alobar holoprosencephaly) (OMIM% 236100) is a rare and lethal complex human malformation, resulting from incomplete cleavage of prosencephalon into right and left hemispheres occurring between the 18th and the 28th day of gestation. Holoprosencephaly occurs in 1/16,000 live births, and 1/250 during embryogenesis. Approximately 1.05 in 100,000 births are identified as infants with cyclopia, including stillbirths. Cyclopia typically presents with a median single eye or a partially divided eye in a single orbit, absent nose, and a proboscis above the eye. Extracranial malformations described in stillbirths with cyclopia include polydactyl, renal dysplasia, and an omphalocele. The etiology of this rare syndrome, which is incompatible with life, is still largely unknown. Most cases are sporadic. Heterogeneous risk factors have been implicated as possible causes. CASE PRESENTATION:A live full-term baby with birth weight of 2900 g, product of cesarean section because of severe fetal bradycardia, was born at Prince Hashem Military Hospital - Zarqa city/Jordan. This newborn was the first baby to a non-consanguineous family, and a healthy 18-year-old mother, with no history of drug ingestion or febrile illnesses during pregnancy. Antenatal history revealed severe hydrocephalus diagnosed early by intrauterine ultrasound but the pregnancy was not terminated because of the lack of medical legitimization in the country. On examination, the newborn was found to have a dysmorphic face, with a median single eye, absence of nose, micrognathia, and a proboscis above the eye, all of which made cyclopia the possible initial diagnosis. Multiple unusual abdominal defects were present that include a huge omphalocele containing whole liver and spleen, urinary bladder extrophy, and undefined abnormal external genitalia, which called for urgent confirmation. Brain MRI was done and revealed findings consistent with alobar holoprosencephaly (cyclopia). CONCLUSION:Presentation of cyclopia is not fully exposed and new cyclopian syndromes still can appear. The prenatal diagnosis of cyclopia can be made early by ultrasound, and the awareness of the spectrum of sonographic findings of cyclopia can improve the accuracy of prenatal diagnosis. The legitimization of pregnancy termination for indexed cases in many countries around the world should be revised.

Project description:The introduction of Integrated Care Systems (ICS) in England aimed to increase joint planning and delivery of health and social care, and other services, to better meet the needs of local communities. There is an associated duty to undertake collaborative research across ICS partners to inform this new integrated approach, which might be challenging given that organisations span health, local authority, voluntary and community sector, and research. This study aimed to explore the appetite for collaborative Research and Innovation (R&I) across ICSs, potential barriers and solutions. This qualitative study involved semi-structured interviews with 24 stakeholders who held senior positions within organisations across two ICS areas (Staffordshire and Stoke-on-Trent; Shropshire, Telford and Wrekin). Interview transcripts were analysed using inductive and deductive analysis, first mapping to the Theoretical Domains Framework (TDF), then considering key influences on organisational behaviour in terms of Capability, Opportunity and Motivation from the COM-B Behaviour Change Wheel. There were fundamental limitations on organisational opportunities for collaborative R&I: a historical culture of competition (rather than collaboration), a lack of research culture and prioritisation, compounded by a challenging adverse economic environment. However, organisations were motivated to undertake collaborative R&I. They recognised the potential benefits (e.g., skill-sharing, staff development, attracting large studies and funding), the need for collaborative research that mirrors integrated care, and subsequent benefits for care recipients. Related barriers included negative experiences of collaboration, fear of failing and low confidence. Capability varied across organisations in terms of research skills and confidence, which reflected the range of partners (from local authorities to NHS Trusts, primary care, and academic institutions). These findings indicate a need to shift from a culture of competition to collaboration, and to help organisations across ICS to prioritise research, and share resources and skills to mitigate the limiting effects of a constrained economic environment. This could be further explored using a systems change approach, to develop the collaborative research efforts alongside the overarching move towards integrated care.

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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.

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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.

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