ArticleClinical pharmacology and therapeutics2019
Weighing of Evidence by Health Technology Assessment Bodies: Retrospective Study of Reimbursement Recommendations for Conditionally Approved Drugs.
Article in Clinical pharmacology and therapeutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
23 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
- HTA Barriers for Conditional Approval Drugs.PharmacoEconomics · 2023Pooled it
- Efficacy gap between phase II and subsequent phase III studies in oncology.British journal of clinical pharmacology · 2020Trial
- Article
- Challenges and reforms in Spain's health technology assessment system: analysis of criteria influencing medicines' reimbursement decisions between 2019 and 2022 in Spain.The European journal of health economics : HEPAC : health economics in prevention and care · 2025Article
- Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.ESMO open · 2025Article
- Article
- Ethical challenges for Health Technology Assessment (HTA) in the evolving evidence landscape.International journal of technology assessment in health care · 2024Review
- How can health technology assessment be improved to optimise access to medicines? Results from a Delphi study in Europe : Better access to medicines through HTA.The European journal of health economics : HEPAC : health economics in prevention and care · 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
- 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
- Article
- The Beneluxa Initiative domain task force health technology assessment: a comparison of member countries' past health technology assessments.International journal of technology assessment in health care · 2023Article
- Synergy between health technology assessments and clinical guidelines for multiple sclerosis.Clinical and translational science · 2023Article
- The Role of Regulator-Imposed Post-Approval Studies in Health Technology Assessments for Conditionally Approved Drugs.International journal of health policy and management · 2022Article
- Real World Data in Health Technology Assessment of Complex Health Technologies.Frontiers in pharmacology · 2022Article
- Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.Clinical and translational science · 2021Article
- The Application and Implications of Novel Deterministic Sensitivity Analysis Methods.PharmacoEconomics · 2021Article
- Decision Making Under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the United States and Europe.Clinical pharmacology and therapeutics · 2020Article
- HTA for pharmaceuticals in Europe: will the mountain deliver a mouse?The European journal of health economics : HEPAC : health economics in prevention and care · 2020Article
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This study assessed whether five Health Technology Assessment (HTA) bodies in Europe were more negative about drugs with a Conditional Marketing Authorization (CMA) that are approved without controlled studies compared to CMA drugs that are approved based on controlled studies. The HTA recommendations were categorized into positive, restricted, and negative. A total of 92 HTA recommendations were available for 27 drugs. Thirty of 62 (48%) and 17 of 30 (57%) of the recommendations were negative for drugs with and without controlled studies, respectively. Overall, only 12 (13%) recommendations were positive. In all jurisdictions, recommendations between drugs with and drugs without controlled data were comparable, which suggests that the presence of controlled data is not decisive in HTA evaluations. The small proportion of unrestricted positive recommendations highlights difficulties with recommending the drugs in this cohort, which may be caused by scientific uncertainty or other factors. Earlier collaboration between stakeholders is advised in order to improve patient access.
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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.