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. 2023 Apr 24;13(4):e064159.
doi: 10.1136/bmjopen-2022-064159.

Understanding potential participation barriers to improve trial design and outcomes: clinical trial simulation in palmoplantar pustulosis as a case study

Christian Boisvert-Huneault  1 , David Trigos Herraez  2 , Andreas Pinter  3 , Satomi Kobayashi  4 , Stacie Bell  5 , Kimberley Kallsen  6 , Tristan Gloede  7 , Nobutaka Yagi  8 , Steven Brunette  7 , Yakov Datsenko  9 , Frank Baehner  6 , Emmanuelle Clerisme-Beaty  6 , Peter Van de Kerkhof  10
Affiliations

Understanding potential participation barriers to improve trial design and outcomes: clinical trial simulation in palmoplantar pustulosis as a case study

Christian Boisvert-Huneault et al. BMJ Open. .

Abstract

Objectives: Evaluate the experiences and perceptions of patients participating in a simulated clinical trial and identify ways to enhance future patient-centric trial designs.

Design: International, multicentre, non-interventional, virtual clinical trial visits with patient debriefs and advisory boards.

Setting: Virtual clinic visits and accompanying advisory boards.

Participants: Nine patients with palmoplantar pustulosis for simulated trial visits; 14 patients and patient representatives for advisory boards.

Main outcome measures: Qualitative responses to trial documentation, visit schedule and logistics, and trial design were collected during patient debriefs. Results were discussed at two virtual advisory board meetings.

Results: Patients identified key barriers to participation and potential difficulties encountered when attending trial visits and completing assessments. They also proposed recommendations to overcome these challenges. Patients recognised the need for comprehensive informed consent forms, but recommended use of non-technical language, brevity and additional support to aid understanding. Other trial documentations should be relevant to the disease and include known efficacy and safety of the study drug. Patients were concerned about receiving placebo, stopping existing medications and being unable to receive the study drug after trial completion; therefore, patients and physicians recommended an open-label extension following trial completion. Trial visits were too numerous (n=20) and too long (3-4 hours each); patients recommended improvements to the design to make best use of their time and reduce unnecessary waiting. They also requested financial and logistical support. Patients expressed a desire for study outcomes that matter to them, related to their ability to undertake normal daily activities and not be a burden to others.

Conclusions: Simulated trials are an innovative method for assessing trial design and acceptance from a patient-centric perspective, enabling specific improvements to be made prior to trial initiation. Incorporation of recommendations from simulated trials could enhance trial recruitment and retention, and optimise trial outcomes and data quality.

Keywords: DERMATOLOGY; Psoriasis; STATISTICS & RESEARCH METHODS.

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Conflict of interest statement

Competing interests: All authors have completed the ICMJE uniform disclosure form at www.icmje.org/coi_disclosure.pdf and declare the following: CB-H—payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AbbVie, Boehringer Ingelheim and La Roche Posay; participation in advisory boards and co-chair of the Canadian Association of Psoriasis Patients. DTH—no conflicts of interest. AP—payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AbbVie, Almirall-Hermal, Amgen, Biogen Idec, Biontec, Boehringer Ingelheim, Celgene, Celltrion, Eli Lilly, Galderma, Hexal, GlaxoSmithKline, Janssen, LEO Pharma, MC2, Medac, Merck Serono, Mitsubishi, MSD, Novartis, Pascoe, Pfizer, Regeneron, Roche, Sandoz Biopharmaceuticals, Sanofi-Genzyme, Schering-Plough, Tigercat Pharma, and UCB; participation in a Data Safety Monitoring Board or Advisory Board for AbbVie, Almirall-Hermal, Amgen, Biogen Idec, Biontec, Boehringer Ingelheim, Celgene, Celltrion, Eli Lilly, Galderma, Hexal, GlaxoSmithKline, Janssen, LEO Pharma, MC2, Medac, Merck Serono, Mitsubishi, MSD, Novartis, Pascoe, Pfizer, Regeneron, Roche, Sandoz Biopharmaceuticals, Sanofi-Genzyme, Schering-Plough,Tigercat Pharma, and UCB. SK—grants or contracts from Kyowa Kirin and honoraria from AbbVie, Eli Lilly, Janssen, Maruho Pharmaceutical, Novartis and Taiho Pharmaceutical. SBell served as the chief scientific and medical officer for the National Psoriasis Foundation during the conduct of this project. KK, TG, NY, SBrunette, YD, FB and EC-B are employees of Boehringer Ingelheim and supported provision of study materials for the present manuscript. PVdK received fees for consultancy service or lectureships from Almirall, AbbVie, Boehringer Ingelheim, Bristol Myers Squibb, Dermavant Sciences, Eli Lilly, Janssen, LEO Pharma, Novartis and UCB.

Figures

Figure 1
Figure 1
Schematic of the palmoplantar pustulosis trial design showing the visit schedule (vertical lines).
Figure 2
Figure 2
Preferences for (A) process for administration and (B) frequency of administration.
See this image and copyright information in PMC

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