Public and patient involvement in health data governance (DATAGov): protocol of a people-centred, mixed-methods study on data use and sharing for rare diseases care and research

Abstract

IntroductionInternational policy imperatives for the public and patient involvement in the governance of health data coexist with conflicting cross-border policies on data sharing. This can challenge the planning and implementation of participatory data governance in healthcare services locally. Engaging with local stakeholders and understanding how their needs, values and preferences for governing health data can be articulated with policies made at the supranational level is crucial. This paper describes a protocol for a project that aims to coproduce a people-centred model for involving patients and the public in decision-making processes about the use and sharing of health data for rare diseases care and research.Methods and analysisThis multidisciplinary project draws on an explanatory sequential mixed-methods study. A hospital-based survey with patients, informal carers, health professionals and technical staff recruited at two reference centres for rare diseases in Portugal will be conducted first. The qualitative study will follow consisting of semi-structured interviews and scenario-based workshops with a subsample of the participant groups recruited at baseline. Quantitative data will be analysed using descriptive and inferential statistics. Inductive and deductive approaches will be combined to analyse the qualitative interviews. Data from scenario-based workshops will be iteratively compared using the constant comparison method to identify cross-cutting themes and categories.Ethics and disseminationThe Ethics Committee for Health from the University Hospital Centre São João/Faculty of Medicine of University of Porto approved the study protocol (Ref. 99/19). Research findings will be disseminated at academic conferences and science promotion events, and through public meetings involving patient representatives, practitioners, policy-makers and students, a project website and peer-reviewed journal publications.

Public Views About Involvement in Decision-Making on Health Data Sharing, Access, Use and Reuse: The Importance of Trust in Science and Other Institutions

“…Assessing and attending to consent preferences and offering time and support to anyone expected to make informed decisions is essential ( 68 , 69 ). However, with the exponential growth of data sources and data uses, informal support may not be sufficient to enable informed consent ( 44 ). As argued by Fiske et al ( 65 ), it is necessary to make way for a new group of professionals—health information counselors—who can advise on the far-reaching implications of data decisions and assist in addressing arising ethical, legal and social challenges and dilemmas that often extend beyond the individual sphere (e.g., the right to choosing not to know and, thus, to decline the return of incidental research findings that may identify a predisposition for late-onset genetic diseases with implications for the offspring) ( 70 ).…”

“…This observational and cross-sectional study is part of a mixed methods project focusing on public involvement in health data governance whose protocol is described elsewhere ( 44 ). For the purposes of this paper, participants include people with rare diseases and their informal carers who are both stakeholders directly involved in decisions regarding their own data sharing, access, use and reuse for biomedical and health care research.…”

“…The questionnaire was pretested by specialists with combined experience as professionals, informal carers and researchers (social and health sciences) and subsequently piloted by a group of patients and carers. The full questionnaire is available online [see ( 44 )].…”

Benefits and Risks of Sharing Genomic Data for Research: Comparing the Views of Rare Disease Patients, Informal Carers and Healthcare Professionals

“…This cross-sectional study was carried out in the scope of a larger project addressing public and patient involvement in health data governance, whose protocol is published elsewhere [ 27 ]. For the purposes of this paper, participants include patients with rare diseases, as well as their informal carers (e.g., family members) and healthcare professionals involved in their care.…”

A Community-Led Approach as a Guide to Overcome Challenges for Therapy Research in Congenital Disorders of Glycosylation

“…Mixed methods research combines quantitative and qualitative data collection approaches that are especially suited to respond to complex questions involving multiple stakeholders. In rare diseases, mixed methods are already being used in therapeutic research to assess patients’ health needs and design rigorous patient-centric clinical and translational research [ 20 , 21 , 22 ].…”