Looking back in time: conducting a cohort study of the long-term effects of treatment of adolescent tall girls with synthetic hormones

Abstract

ObjectivePublic health research is an endeavour that often involves multiple relationships, far-reaching collaborations, divergent expectations and various outcomes. Using the Tall Girls Study as a case study, this paper will present and discuss a number of methodological, ethical and legal challenges that have implications for other public health research.ApproachThe Tall Girls Study was the first study to examine the long-term health and psychosocial effects of oestrogen treatment for tall stature.ResultsIn undertaking this study the research team overcame many hurdles: in maintaining collaboration with treating clinicians and with the women they had treated as girls - groups with opposing points of view and different expectations; using private practice medical records to trace women who had been patients up to forty years earlier; and exploring potential legal issues arising from the collection of data related to treatment.ConclusionWhile faced with complex challenges, the Tall Girls Study demonstrated that forward planning, ongoing dialogue between all stakeholders, transparency of processes, and the strict adherence to group-developed protocols were keys to maintaining rigour while undertaking pragmatic research.ImplicationsPublic health research often occurs within political and social contexts that need to be considered in the planning and conduct of studies. The quality and acceptability of research findings is enhanced when stakeholders are engaged in all aspects of the research process.

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

“…Eleven studies used qualitative or survey methods to obtain input from patients, 4,7,[18][19][20][21][22][23][24][25][26] and 17 studies were narrative reports describing one group's experiences with engagement. [27][28][29][30][31][32][33][34][35][36][37][38][39][40][41][42][43] The remaining seven studies were descriptions of specific initiatives reported on websites [44][45][46][47][48][49][50] ( Table 1 and Table 2). The studies were mostly conducted in North America and Europe, and most focused on a specific rare disease rather than rare diseases in general.…”

“…cians. 20,38,39,47,49 Seven studies were classified as sufficiently descriptive 7,18,19,[23][24][25][26] The purposes of engagement included identifying patient-centered research topics or agendas, 19,23,29,36,39 identifying outcomes important to patients or developing measures relevant to patients' needs, 7,19,20,23,25,26 increasing recruitment or enrollment through development of patient-centered study designs, 4,18,21,24,27,30,31,41 and incorporating the patient perspective into study design 19,25,35,39,40 (Table 2, Table 3). Patients and other stakeholders were most commonly engaged in the preparatory stage (n= 19 for agenda setting) 4, 7, 19, 20, 23, 25, 28, 29, 32, 35, 36, 39- (Table 4 [online]).…”

“…19 Thirteen studies described more collaborative involvement in at least one stage of the research process; for example, including patients on governing or advisory committees, 32,33,38,42,48 in developing study interview guides, study materials and websites; 21,35,52 in collecting data (conducting interviews), 4,21 in reviewing research findings, 21,28 and in disseminating findings. 4,33,37,[40][41][42] Stakeholder-driven approaches were reported; included here are examples of patients or advocacy organizations initiating new studies 4,29,37,41,42,46 and controlling the research process by setting the research agenda, having responsibility for data collection, and disseminating findings (e.g., Patient-Powered Registries 43 ). Conversely, in four studies, patient organizations were engaged only for providing access to their members.…”

“…Some perceived benefits relative to other studies that did not use engagement were practical, such as improved access to patients or improved recruitment methods, 4,19,29,30,35,42,43 improved study retention, 19 more in-depth responses in interviews (when using patients to conduct the interviews), 21 less time to complete the project, 4 and incorporation of a broader range of outcome measures to inform future risk-benefit assessments for investigational treatments. 49 Other perceived benefits were less tangible, such as increased relevance of the project, 4,29,35 broader dissemination of findings, 35 improved acceptance of research findings among end-users, 29 greater mutual respect between scientists and patients, 32 and increased transparency of and public confidence in the research. 32,33 Only one study articulated the benefits of engagement for the engaged partners, including increased awareness of the disease studied and greater access to health information.…”

“…Some studies reported that patient organizations initiated clinical trials or other studies of rare diseases. 28,29,41 Several studies also highlighted broader support provided by rare disease organizations. For example, these groups initiated the organization of collaborative research networks, 22,28,41 provided financial support for research infrastructure such as registries, 22,30,43,46 supported training activities, 22,34,44 and developed policy statements regarding engaging patients in research.…”