ArticleFrontiers in pharmacology2023
Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement.
Article in Frontiers in pharmacology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 2 of them syntheses that pooled it.
What it found
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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
11 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
- Drug pricing models, no 'one-size-fits-all' approach: a systematic review and critical evaluation of pricing models in an evolving pharmaceutical landscape.The European journal of health economics : HEPAC : health economics in prevention and care · 2025Pooled it
- Affordability of new drugs in the Brazilian public health system: a 13-year monitoring analysis of reimbursement decisions based on health technology assessments (2012-2024).Lancet regional health. Americas · 2026Article
- Rebalancing innovation, affordability, and access for orphan drugs in the European Union.The Lancet regional health. Europe · 2026Review
- Real-world treatment patterns and unmet needs in spinal muscular atrophy: a caregiver-centric survey study from China.BMC neurology · 2026Article
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- New opportunities for accessing promising non-oncological orphan drugs.The Lancet regional health. Europe · 2025Article
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Towards ethical drug pricing: the European Orphan Genomic Therapies Fund.Gene therapy · 2024Review
- A novel approach to boost drug development in paediatric oncology.Nature reviews. Drug discovery · 2023Article
- Addressing unmet needs in rare diseases: A patient-centred vision through INFORM-RD.Therapeutic advances in rare diseaseArticle
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
Legislations incentivising orphan drug development and scientific advances have made orphan drugs pharma's high-end favourite for the past two decades. Currently, around 50% of new marketing authorizations are for orphan drugs. For third-party healthcare payers ("payers") the rise of orphan drugs presents new challenges, including a high degree of uncertainty around clinical benefits and harms, a moderate effect size (for many orphan drugs), and a high price tag. The association of high clinical uncertainty and moderate effect sizes is not surprising in small target populations but in combination with high prices creates the risk of allocative and technical inefficiencies for payers. We here discuss and illustrate these risks. A combination of policies is needed for mitigation of allocative inefficiency: while there may be a rationale for higher prices for orphan than non-orphan drugs, a focus of pricing and reimbursement negotiations should include considerations of product profitability and of the consequences of orphan drug costs on the distribution inequality of medication costs for individual insured persons, coupled to knowledge generation from reimbursement contracts covering high-price orphan drugs that would benefit the wider patient community. Performance-based managed entry agreements could help to de-risk the economic consequences of clinical uncertainty and to mitigate technical inefficiency.
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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.