The patient's view on rare disease trial design - a qualitative study.

Autor: Gaasterland CMW; Pediatric clinical Research Office, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105, AZ, Amsterdam, The Netherlands. c.m.gaasterland@amc.uva.nl., van der Weide MCJ; Pediatric clinical Research Office, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105, AZ, Amsterdam, The Netherlands., du Prie-Olthof MJ; Pediatric clinical Research Office, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105, AZ, Amsterdam, The Netherlands., Donk M; Department of Cognitive Psychology, Vrije Universiteit Amsterdam, Amsterdam, the Netherlands., Kaatee MM; PSC Patients Europe, Bennebroek, the Netherlands., Kaczmarek R; Laboratory of Glycoconjugate Immunochemistry, Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wroclaw, Poland.; Polish Hemophilia Society, Warsaw, Poland., Lavery C; MPS Society, Amersham, UK., Leeson-Beevers K; Alström Syndrome UK, Torquay, Devon, UK., O'Neill N; EUPATI Fellow, Bennebroek, the Netherlands., Timmis O; AKU Society, Cambridge, UK., van Nederveen V; Stichting Patiëntenstem.nu, Den Haag, The Netherlands., Vroom E; Duchenne Parent Project, Veenendaal, The Netherlands., van der Lee JH; Pediatric clinical Research Office, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105, AZ, Amsterdam, The Netherlands.
Jazyk: angličtina
Zdroj: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2019 Feb 07; Vol. 14 (1), pp. 31. Date of Electronic Publication: 2019 Feb 07.
DOI: 10.1186/s13023-019-1002-z
Abstrakt: Background: Clinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a 'classical' Randomized Controlled Trial. Therefore, novel designs are developed by statisticians. However, it is important to be aware of possible design aspects that may jeopardize the feasibility of trial conduct. If the burden of participation is considered out of proportion by patients or parents, recruitment may fail or participants may drop out before trial completion. In order to maximize the chance of success of trials in small populations, it is important to know which aspects of trial design are considered important by patients.
Results: We have interviewed all ten members of the Patient Think Tank (PTT) of the ASTERIX project, a European research consortium on methodology for clinical trials in small populations. The PTT members are rare disease patient representatives who have completed extensive training in clinical trial methodology. We have analyzed the interviews qualitatively according to Grounded Theory using a thematic analysis, and we structured the topics in four chronologically ordered themes: 1. Involvement in trial design; 2. Opinions on trial design; 3. Trial participation; 4. Phase after the trial. Our main findings are that the PTT-members recommend that patients are involved in trial design from an early stage on, and have influence on the outcomes and measurement instruments that are chosen in the trial, the length of the study, the choice of participants, and the information that is sent to potential participants. Also, according to the PTT-members, patient groups should consider setting up disease registries, placebo groups should be minimized, and more education on clinical trials is advised.
Conclusions: Rare disease patient representatives who have been educated about clinical trial methodology think it is important to involve patient representatives in research at an early stage. They can be of advice in trial design in such a way that the ratio of potential benefit and burden of trial participation as well as the chosen outcome measures and in- and exclusion criteria are optimized.
Databáze: MEDLINE
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