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Record W3036900991 · doi:10.1186/s13063-020-04466-0

Stakeholder perspectives on adaptive clinical trials: a scoping review

2020· review· en· W3036900991 on OpenAlexafffund
Tina Madani Kia, John C. Marshall, Srinivas Murthy

Bibliographic record

VenueTrials · 2020
Typereview
Languageen
FieldMathematics
TopicStatistical Methods in Clinical Trials
Canadian institutionsUniversity of TorontoBC Children's Hospital
FundersCanadian Institutes of Health Research
KeywordsSample size determinationStakeholderClinical study designResearch designClinical trialMedicineInclusion (mineral)PopulationExternal validityComputer sciencePsychologySocial psychologyPublic relationsStatistics

Abstract

fetched live from OpenAlex

BACKGROUND: Adaptive clinical trials (ACTs) represent an emerging approach to trial design where accumulating data are used to make decisions about future conduct. Adaptations can include comparisons of multiple dose tiers, response-adaptive randomization, sample size re-estimation, and efficacy/futility stopping rules. The objective of this scoping review is to assess stakeholder attitudes, perspectives, and understanding of adaptive trials. METHODS: We conducted a review of articles examining stakeholders encompassing the broad medical trial community's perspectives of adaptive designs (ADs). A computerized search was conducted of four electronic databases with relevant search terms. Following screening of articles, the primary findings of each included article were coded for study design, population studied, purpose, and primary implications. RESULTS: Our team retrieved 167 peer-reviewed titles in total from the database search and 5 additional titles through searching web-based search engines for gray literature. Of those 172 titles, 152 were non-duplicate citations. Of these, 119 were not given full-text reviews, as their titles and abstracts indicated that they did not meet the inclusion criteria. Thirty-three articles were carefully examined for relevance, and of those, 18 were chosen to be part of the analysis; the other 15 were excluded, as they were not relevant upon closer inspection. Perceived advantages to ADs included limiting ineffective treatments and efficiency in answering the research question; -perceived barriers included insufficient sample size for secondary outcomes, challenges of consent, potential for bias, risk of type 1 error, cost and time to adaptively design trials, unclear rationales for using Ads, and, most importantly, a lack of education regarding ADs among stakeholders within the clinical trial community. Perceptions among different types of stakeholders varied from sector to sector, with patient perspectives being noticeably absent from the literature. CONCLUSION: There are diverse perceptions regarding ADs among stakeholders. Further training, guidelines, and toolkits on the proper use of ADs are needed at all levels to overcome many of these perceived barriers. While education for principal investigators is important, it is also crucial to educate other groups in the community, such as patients, as well as clinicians and staff involved in their daily implementation.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.302
metaresearch head score (Gemma)0.967
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch, Meta-epidemiology (narrow), Meta-epidemiology (broad), Research integrity, Insufficient payload (model declined to judge)
Consensus categoriesMetaresearch, Meta-epidemiology (narrow), Meta-epidemiology (broad), Research integrity, Insufficient payload (model declined to judge)
DomainCandidate signal: Methods · Consensus signal: none
Study designCandidate signal: Other design · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.814
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.3020.967
Meta-epidemiology (narrow)0.0020.001
Meta-epidemiology (broad)0.0540.010
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0020.003
Insufficient payload (model declined to judge)0.0040.001

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.987
GPT teacher head0.784
Teacher spread0.202 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; both teacher heads agree on what is shown here.

Study designOther design
DomainMethods
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations21
Published2020
Admission routes2
Has abstractyes

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