Trial reportPharmacoEconomics2023
Estimating the Potential Benefits of Confirmatory Trials for Drugs with Accelerated Approval: A Comprehensive Value of Information Framework.
Trial report in PharmacoEconomics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed, 2 citations in OpenAlex.
- Waiting for Trial Evidence or Using Real-World Evidence: Trade-Offs for Coverage with Evidence Development.PharmacoEconomics · 2026Review
- Moving Forward with Taking a Societal Perspective: A Themed Issue on Productivity Costs, Consumption Costs and Informal Care Costs.PharmacoEconomics · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe US Food and Drug Administration's Accelerated Approval (AA) policy provides a pathway for patients to access potentially life-saving drugs rapidly. However, the use of surrogate endpoints, single-arm designs, and small sample sizes in preliminary trials that support AAs can lead to uncertainty regarding the clinical benefits of such drugs. This study aims to develop a comprehensive value of information (VOI) framework for assessing the potential benefits of future confirmatory trials, accounting for the various uncertainties inherent in preliminary trials.
methodsI formulated an expected value of information from confirmatory trial (EVICT) metric, which evaluates the potential benefits of a confirmatory trial that would reduce those uncertainties by using a clinically meaningful endpoint, a randomized control, and increased sample size. The EVICT metric can quantify the expected benefits of a well-designed confirmatory trial or an inadequately designed one that continues to use surrogate endpoints or single-arm design. The framework was illustrated using a hypothetical AA drug for metastatic breast cancer.
resultsThe case study demonstrates that a highly uncertain preliminary trial of an AA drug was associated with a substantial EVICT. A confirmatory trial with an increased sample size for this AA drug, utilizing a clinically meaningful endpoint and randomized control, yielded a population-level EVICT of $12.6 million. Persistently using a surrogate endpoint and single-arm trial design would reduce the EVICT by 60%.
conclusionsThis framework can provide accurate VOI estimates to guide coverage policies, value-based pricing, and the design of confirmatory trials for AA drugs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.