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Inclusion of progression criteria in external randomised pilot trials: a cross-sectional study of funding applications submitted to the NIHR Research for Patient Benefit Programme

Abstract:
Background
External randomised pilot trials aim to assess whether a future definitive randomised controlled trial (RCT) is feasible. Pre-specified progression criteria help guide the interpretation of pilot trial findings to decide whether, and how, a definitive trial should be conducted. We aimed to examine how researchers report and plan to assess progression criteria in external pilot trial funding applications submitted to the NIHR Research for Patient Benefit Programme.
Methods
We conducted a cross-sectional study of progression criteria inclusion in Stage 1 (outline) and corresponding Stage 2 (full) funding applications for external randomised external pilot trials submitted to NIHR RfPB between July 2017 and July 2019.
Results
Of the 100 Stage 1 outline applications assessed, 95 were eligible for inclusion (of these, 52 were invited to Stage 2 full application; 43 were rejected) and 49/52 were eligible for inclusion at Stage 2 full application (of these, 35 were awarded funding; 14 were rejected). Over half of applications assessed at Stage 1 (48/95, 51%), and 73% of those assessed at Stage 2 (36/49) included progression criteria in their research plans. Progression criteria were most often reported in a stop-go format, often with additional specified factors that should be considered when determining feasibility (Stage 1 33/48, 69%; Stage 2 21/36, 58%). Recruitment and retention were the most frequent indicators of feasibility to inform progression criteria. One-third of applications provided some justification or rationale for their targets (Stage 1 16/48, 33%; Stage 2 12/36, 33%). Funding committee feedback mentioned progression criteria in over 20% of applications (Stage 1 22/95, 23%; Stage 2 11/49, 22%) to either request the addition of progression criteria or provide justification for the criteria stipulated.
Conclusions
Our findings indicate that researchers do not always include progression criteria in external randomised pilot trial applications submitted to research funders. This can result in a lack of transparency in the assessment of randomised pilot trial feasibility.
Trial registration
Open Science Framework osf.io/89ap7, registered 29th June 2021.
Publication status:
Published
Peer review status:
Peer reviewed

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Files:
Publisher copy:
10.1186/s13063-022-06868-8

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
NDORMS
Oxford college:
Somerville College
Role:
Author
ORCID:
0000-0002-4054-5975


Publisher:
BioMed Central
Journal:
Trials More from this journal
Volume:
23
Issue:
1
Article number:
931
Place of publication:
England
Publication date:
2022-11-08
Acceptance date:
2022-10-25
DOI:
EISSN:
1745-6215
Pmid:
36348460


Language:
English
Keywords:
Pubs id:
1301172
Local pid:
pubs:1301172
Deposit date:
2022-11-12

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