ReviewPharmaceutical statistics2022
Informed decision-making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments.
Review in Pharmaceutical statistics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- Methodologies for generating and evaluating clinical and performance evidence for high-risk and innovative medical devices andFrontiers in medical technology · 2026Pooled it
- Evaluating the basis for use of advanced stage as a surrogate endpoint for cancer mortality in screening trials: a simulation study of meta-correlation and an alternative framework.EClinicalMedicine · 2026Article
- Radiographic progression-free survival as a surrogate endpoint for overall survival in first-line ARPi naïve metastatic castration-resistant prostate cancer.The oncologist · 2026Article
- A surrogate endpoint-based provisional approval causal roadmap, illustrated by vaccine development.Biostatistics (Oxford, England) · 2025Article
- Use of surrogate endpoints in health technology assessment and reimbursement of treatments for the management of chronic kidney disease.EClinicalMedicine · 2025Review
- A call for transparency, improved reporting, and interpretation of trials using surrogate end points in cardiac electrophysiology.Heart rhythm · 2025Review
- A Causal Inference Approach to Mediation Analysis in Vitreomacular Traction: How Much Does Traction Resolution Mediate Functional Outcomes?Journal of market access & health policy · 2024Article
- Review
- Reporting of surrogate endpoints in randomised controlled trial reports (CONSORT-Surrogate): extension checklist with explanation and elaboration.BMJ (Clinical research ed.) · 2024Article
- Reporting of surrogate endpoints in randomised controlled trial protocols (SPIRIT-Surrogate): extension checklist with explanation and elaboration.BMJ (Clinical research ed.) · 2024Article
- The effectiveness of problem-based learning and case-based learning teaching methods in clinical practical teaching in TACE treatment for hepatocellular carcinoma in China: a bayesian network meta-analysis.BMC medical education · 2024Article
- The role of early-phase trials and real-world evidence in drug development.Nature cardiovascular research · 2024Review
- Novel Clinical Trial Designs with Dose Optimization to Improve Long-term Outcomes.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023Review
- Interpreting Randomized Controlled Trials.Cancers · 2023Review
- Less is More? First Impressions From COSMIC-313.Cancer investigation · 2023Article
- [Progress and Application of Bayesian Approach in the Early Research and Development of New Anticancer Drugs].Zhongguo fei ai za zhi = Chinese journal of lung cancer · 2022Article
- Informed decision-making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments.Pharmaceutical statistics · 2022Review
- The Impact of Model Misspecification on the Individual Causal Association in Surrogate Endpoint Evaluation.Pharmaceutical statisticsArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
The desire, by patients and society, for faster access to therapies has driven a long tradition of the use of surrogate endpoints in the evaluation of pharmaceuticals and, more recently, biologics and other innovative medical technologies. The consequent need for statistical validation of potential surrogate outcome measures is a prime example on the theme of statistical support for decision-making in health technology assessment (HTA). Following the pioneering methodology based on hypothesis testing that Prentice presented in 1989, a host of further methods, both frequentist and Bayesian, have been developed to enable the value of a putative surrogate outcome to be determined. This rich methodological seam has generated practical methods for surrogate evaluation, the most recent of which are based on the principles of information theory and bring together ideas from the causal effects and causal association paradigms. Following our synopsis of statistical methods, we then consider how regulatory authorities (on licensing) and payer and HTA agencies (on reimbursement) use clinical trial evidence based on surrogate outcomes. We review existing HTA surrogate outcome evaluative frameworks. We conclude with recommendations for further steps: (1) prioritisation by regulators and payers of the application of formal surrogate outcome evaluative frameworks, (2) application of formal Bayesian decision-analytic methods to support reimbursement decisions, and (3) greater utilization of conditional surrogate-based licensing and reimbursement approvals, with subsequent reassessment of treatments in confirmatory trials based on final patient-relevant outcomes.
Indexed as
Identifiers
What OpenQuestion holds
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.