ArticleJournal of general internal medicine2020
Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs.
Article in Journal of general internal medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
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The trial behind it
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Who cites it
25 citing papers in PubMed, 50 citations in OpenAlex.
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest.Clinical and translational science · 2025Article
- A structured approach for identifying disease analogs for pulmonary arterial hypertension.Journal of managed care & specialty pharmacy · 2025Article
- Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.Healthcare (Basel, Switzerland) · 2025Review
- An analysis of US net cancer drug launch prices and clinical efficacy and certainty of evidence from 2008 to 2022.Health affairs scholar · 2025Article
- Bridging Gaps in Oncology: Comparative Analysis of Development and Approval Pathways for Anticancer Drugs and Companion Diagnostic in the United States and Japan.Clinical and translational science · 2025Article
- The impact of rare diseases on the quality of life in paediatric patients: current status.Frontiers in public health · 2025Review
- Caregiver-Reported Economic Impacts of Pediatric Rare Diseases-A Scoping Review.Healthcare (Basel, Switzerland) · 2024Article
- Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Review
- Impact of mental health in persons living with rare disease: Findings from the AMCP Market Insights Program.Journal of managed care & specialty pharmacy · 2024Article
- Launch and Post-Launch Prices of Injectable Cancer Drugs in the US: Clinical Benefit, Innovation, Epidemiology, and Competition.PharmacoEconomics · 2024Article
- The Innovative Medicines Fund: a universal model for faster and fairer access to new promising medicines or a Trojan horse for low-value creep?Journal of the Royal Society of Medicine · 2023Article
- Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges.International journal of environmental research and public health · 2023Review
- The economic impact of living with a rare disease for children and their families: a scoping review protocol.HRB open research · 2023Article
- Economic Evaluation of a Tumour-Agnostic Therapy: Dutch Economic Value of Larotrectinib in TRK Fusion-Positive Cancers.Applied health economics and health policy · 2022Article
- Is an Orphan Drug's Cost-Effectiveness Associated with US Health Plan Coverage Restrictiveness?PharmacoEconomics · 2022Article
- How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.Frontiers in pharmacology · 2022Article
- Trends in orphan drug use and spending among children and adolescents during 2010-2020 in Korea.Frontiers in pharmacology · 2022Article
- Compendium of causative genes and their encoded proteins for common monogenic disorders.Protein science : a publication of the Protein Society · 2022Article
- Priority setting at the clinical level: the case of nusinersen and the Norwegian national expert group.BMC medical ethics · 2021Article
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundOrphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan drugs, leading to questions about their cost-effectiveness.
objectiveTo compare the value of orphan and non-orphan drugs approved by the FDA from 1999 through 2015.
designWe searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions).
resultsWe identified estimates for 49 orphan drug-indication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009).
conclusionsOur study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending.
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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.