ArticlePloS one2016
Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.
Article in PloS one, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
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.
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Who cites it
28 citing papers in PubMed, 59 citations in OpenAlex.
- Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.Orphanet journal of rare diseases · 2026Article
- Developing therapeutics for rare cardiovascular diseases.American heart journal · 2026Review
- Reconceiving orphan drug market exclusivity as a conditional public-law entitlement: a sustainable regulatory governance model with comparative lessons for China.Frontiers in medicine · 2026Article
- Economic burden of Sanfilippo syndrome in the United States.Archives of public health = Archives belges de sante publique · 2025Article
- Evidence available and used by the Food and Drug Administration for the approval of orphan and nonorphan drugs.Health affairs scholar · 2025Article
- Rare disease challenges and potential actions in the Middle East.International journal for equity in health · 2025Article
- Reforming the innovation system to deliver affordable medicines: a conceptual framework of pharmaceutical innovation as a complex adaptive system (forest) and theory of change.Journal of pharmaceutical policy and practice · 2025Article
- Personalized nutrition: aligning science, regulation, and marketing.Health affairs scholar · 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
- Costs of orphan medicinal products: longitudinal analysis of expenditure in Wales.Orphanet journal of rare diseases · 2023Article
- Article
- Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?Orphanet journal of rare diseases · 2022Review
- Development of a core evaluation framework of value-added medicines: report 2 on pharmaceutical policy perspectives.Cost effectiveness and resource allocation : C/E · 2021Review
- Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison.Healthcare (Basel, Switzerland) · 2021Article
- The Potential Benefit of Expedited Development and Approval Programs in Precision Medicine.Journal of personalized medicine · 2021Article
- Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab.PloS one · 2021Article
- Evaluating New Zealanders' Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions.PharmacoEconomics · 2021Article
- Registries for orphan drugs: generating evidence or marketing tools?Orphanet journal of rare diseases · 2020Article
- Experience With Pediatric Medical Device Development.Frontiers in pediatrics · 2020Article
- The case of eculizumab: litigation and purchases by the Brazilian Ministry of Health.Revista de saude publica · 2020Article
Corrections and comments
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Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundConcerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.
methodsWe conducted a retrospective, propensity score matched study of publicly-listed orphan companies. Cases were defined as holders of orphan drug market authorisation in Europe or the USA between 2000-12. Control companies were selected based on their propensity for being orphan drug market authorisation holders. We applied system General Method of Moments to test whether companies with orphan drug market authorization are valued higher, as measured by the Tobin's Q and market to book value ratios, and are more profitable based on return on assets, than non-orphan drug companies.
results86 companies with orphan drug approvals in European (4), USA (61) or both (21) markets were matched with 258 controls. Following adjustment, orphan drug market authorization holders have a 9.6% (95% confidence interval, 0.6% to 18.7%) higher return on assets than non-orphan drug companies; Tobin's Q was higher by 9.9% (1.0% to 19.7%); market to book value by 15.7% (3.1% to 30.0%) and operating profit by 516% (CI 19.8% to 1011%). For each additional orphan drug sold, return on assets increased by 11.1% (0.6% to 21.3%), Tobin's Q by 2.7% (0.2% to 5.2%), and market to book value ratio by 5.8% (0.7% to 10.9%).
conclusionsPublicly listed pharmaceutical companies that are orphan drug market authorization holders are associated with higher market value and greater profits than companies not producing treatments for rare diseases.
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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.