Developments in trial design A methodological perspective Rolf H.H. - PowerPoint PPT Presentation

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Developments in trial design A methodological perspective Rolf H.H. Groenwold RSNN meeting, 17 April 2019 1 18-Jun-19 Trial design used to be easy Challenges RWE Patient subgroups Small populations Rapid

Developments in trial design A methodological perspective Rolf H.H. Groenwold RSNN meeting, 17 April 2019 1 18-Jun-19
Trial design used to be ‘easy’
Challenges ▪ RWE ▪ Patient subgroups ▪ Small populations ▪ Rapid developments ▪ Costly infrastructure
Adaptations to conventional trial design (not exhaustive) Challenge Adaptation RWE Pragmatism - ‘real - world’ populations - ‘real - world’ physicians - ‘real - world’ monitoring (wearables) - Trials within cohorts (Twics) Patient subgroups / - N-of-1 studies small populations - Single arm studies - Informed analysis - Co-enrolment Rapid Developments - Platform trial - Master protocol Costs - Learning healthcare system (LHS)
Pragmatic trials
Pragmatic trials Example: EHR-based trials - Recruitment - Outcome assessment - Safety monitoring(?) GCP vs. pragmatism Mentz e.a., Circulation 2016; Ford & Norrie, NEJM 2016; Thorpe e.a., CMAJ 2009
Van Staa e.a., Health Technol Assess 2014
BMJ 2019;364:l1092
Trials within cohorts Infrastructure for trials - Staged recruitment - Staged informed consent - Consent to be randomized - Control arm remains ignorant www.twics.global/the-design-twics
Pragmatic trials Possible advantages: - RWE Possible limitations: - RWE
N-of-1 Trials
N-of-1 Trials Stunnenberg e.a., JAMA 2017
N-of-1 Trials Possible advantages: - Small samples - Adaptive Possible limitations: - Deblinding (gastrointestinal adverse reactions) - Only for chronic/stable diseases and treatment with rapid response
Informed analysis
Informed analysis Laptook e.a., JAMA 2017
Informed analysis Laptook e.a., JAMA 2017
Informed analysis Possible advantages: - Small samples (statistical efficiency due to external information) Possible limitations: - Willingness to accept external information?
Co-enrollment Possible advantages: - Multiple studies in small populations Possible limitations: - Contamination - Burden of participation
Platform trial Woodcock e.a., NEJM 2017
Umbrella trial Woodcock e.a., NEJM 2017
Basket trial Woodcock e.a., NEJM 2017
Master protocols Possible advantages: - Trial infrastructure - Improve recruitment - Access to small populations (?) - Respond quickly to new developments Possible limitations: - Institutional review process (?) - Costs to maintain infrastructure (?)
Learning Healthcare system
r.h.h.groenwold@lumc.nl

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