ReviewFrontiers in pharmacology2021
How to Value Orphan Drugs? A Review of European Value Assessment Frameworks.
Review in Frontiers in pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 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
29 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Valuation of Treatments for Rare Diseases: A Systematic Literature Review of Societal Preference Studies.Advances in therapy · 2023Pooled it
- A Delphi Study to Identify Priority Indicators for Formulary Selection of Innovative Anticancer Drugs in China.Health expectations : an international journal of public participation in health care and health policy · 2026Article
- Medicine Development and Access for Rare Diseases: Can We Do Better?Journal of inherited metabolic disease · 2026Review
- Health technology assessment and medical insurance access for rare disease drugs in China: A policy review with quantitative insights from publicly available data.Intractable & rare diseases research · 2026Review
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Reforming China's Rare Disease Security System: Risk Management Perspectives and a Dedicated Insurance Innovation.Healthcare (Basel, Switzerland) · 2025Article
- Bridging Clinical Outcomes and Cost-Effectiveness: The Role of Real-World Data in Cystic Fibrosis Therapy.Pharmacology research & perspectives · 2025Article
- How is Value Defined in Molecular Testing in Cancer? A Scoping Review.Applied health economics and health policy · 2025Article
- Understanding healthcare efficiency-an AI-supported narrative review of diverse terminologies used.BMC medical education · 2025Review
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Establishment of a value assessment framework for orphan drugs in China: an application of the discrete choice experiment in multicriteria decision analysis.Frontiers in pharmacology · 2025Article
- Considerations when applying equity weights within economic evaluation when making drug reimbursement decisions.Health economics review · 2024Article
- Uncertainties in evaluating the health-related quality of life and disease burden of people with rare diseases and their caregivers in NICE HST submissions.Orphanet journal of rare diseases · 2024Review
- Establishment of a value assessment framework for orphan medicinal products in China.Orphanet journal of rare diseases · 2024Article
- Navigating the unknown: how to best 'reflect' standard of care in indications without a dedicated treatment pathway in health technology assessment submissions.Journal of comparative effectiveness research · 2024Review
- Article
- Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.Frontiers in pharmacology · 2024Article
- The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.Journal of comparative effectiveness research · 2023Article
- Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases.Journal of market access & health policy · 2023Article
- Voluntariness or legal obligation? An ethical analysis of two instruments for fairer global access to COVID-19 vaccines.Frontiers in public health · 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
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
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.