ArticleHealth affairs (Project Hope)2021
Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases.
Article in Health affairs (Project Hope), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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
11 citing papers in PubMed.
- Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025Review
- Value-Based Indication-Specific Pricing and Weighted-Average Pricing: Estimated Price and Cost Savings for Cancer Drugs.PharmacoEconomics · 2025Article
- 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
- The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success.The Milbank quarterly · 2024Article
- Cost of Exempting Sole Orphan Drugs From Medicare Negotiation.JAMA internal medicine · 2024Article
- Launch and Post-Launch Prices of Injectable Cancer Drugs in the US: Clinical Benefit, Innovation, Epidemiology, and Competition.PharmacoEconomics · 2024Article
- Article
- FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis.BMJ (Clinical research ed.) · 2023Article
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Review
- Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?Orphanet journal of rare diseases · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
The Orphan Drug Act of 1983 provides benefits to promote the development of treatments for rare diseases that have limited sales potential. Policy makers have questioned whether this purpose is furthered in the case of "partial orphan drugs" approved to treat both rare and common diseases, as many of these drugs are top sellers. In this study we used national commercial claims data to estimate the proportion of spending in the US on fifteen top-selling partial orphan drugs that was assigned to orphan indications in 2018. Of this spending, 21.4 percent was assigned to orphan indications, 70.7 percent to nonorphan indications, and 7.9 percent to neither orphan nor nonorphan indications (for example, off-label use). These findings support growing concerns regarding the costs of granting orphan drug benefits to the sponsors of top-selling partial orphan drugs.
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.