ArticleClinical pharmacology and therapeutics2020
Decision Making Under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the United States and Europe.
Article in Clinical pharmacology and therapeutics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers, 2 of them syntheses 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
43 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.Orphanet journal of rare diseases · 2025Pooled it
- Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis.International journal of environmental research and public health · 2020Pooled it
- External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management.Clinical pharmacology and therapeutics · 2026Article
- A Cross-Sectional Survey of Evidence Needs for Medicinal Products in Europe With a Focus on Real-World Evidence.Pharmacoepidemiology and drug safety · 2026Article
- A multi-stakeholder survey on communicating cost-effectiveness uncertainties to stakeholders: a case study of ICER.Health affairs scholar · 2026Article
- Innovating HTA: a call for capacity building and standardization.International journal of technology assessment in health care · 2026Review
- Evaluating use of disease-modifying therapies for multiple sclerosis using claims data.Frontiers in health services · 2026Article
- APPRAISE: A Tool for Appraising Potential for Bias in Real-World Evidence Studies on Medication Effectiveness or Safety.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2025Article
- Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands.International journal of technology assessment in health care · 2025Article
- Article
- Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis.Clinical pharmacology and therapeutics · 2025Article
- Mapping methods gaps between EU joint clinical assessments and local health technology assessment decision-making: an environmental scan of guidance in select EU markets and harmonization challenges.Journal of comparative effectiveness research · 2025Review
- Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.PharmacoEconomics · 2025Article
- Uncertainties about the benefit-risk balance of oncology medicines assessed by the European Medicines Agency.ESMO open · 2024Article
- Evidence of clinical benefit of cancer medicines considered for funding in Australia.International journal of technology assessment in health care · 2024Article
- Utilising Health Technology Assessment to Develop Managed Access Protocols to Facilitate Drug Reimbursement in Ireland.Applied health economics and health policy · 2024Article
- The potential role of real-world evidence in Centers for Medicare & Medicaid Services' future price negotiations: Recommendations for a robust framework.Journal of managed care & specialty pharmacy · 2024Article
- An operationalization framework for lifecycle health technology assessment: a Health Technology Assessment International Global Policy Forum Task Force report.International journal of technology assessment in health care · 2024Review
- The Critical Intersect of Regulations, Health Technology Assessment, and Drug Safety Assessments.Drug safety · 2024Article
- Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study.BMJ (Clinical research ed.) · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Assessments of clinical evidence vary between regulators and health technology assessment bodies, but precise differences remain unclear. To compare uncertainties raised on the clinical evidence of approved drugs, we analyzed assessments of regulators and health technology assessment (HTA) bodies in the United States and Europe. We found that US and European regulators report uncertainties related to safety for almost all drugs (85-94%), whereas HTA bodies reported these less (53-59%). By contrast, HTA bodies raised uncertainties related to effects against relevant comparators for almost all drugs (88-100%), whereas this was infrequently addressed by regulators (12-32%). Regulators as well as HTA bodies reported uncertainties related to the patient population for 60-95% of drugs. The patterns of regulator-HTA misalignment were comparable between the United States and Europe. Our results indicate that increased coordination between these complementary organizations is necessary to facilitate the collection of necessary evidence in an efficient and timely manner.
Indexed as
Identifiers
What Socratic 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 Socratic graph.