ArticleBMJ (Clinical research ed.)2023
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.
Article in BMJ (Clinical research ed.), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 3 of them syntheses that pooled it.
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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.
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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
24 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Evolution of clinical benefit in cancer trials: comparing pivotal approval data with updated efficacy.Frontiers in public health · 2026Pooled it
- Patient, Physician, and Assessor Blinding in Phase III Randomized Trials in Oncology: A Meta-Epidemiological Analysis.Cancer medicine · 2025Pooled it
- Breast cancer drugs: FDA approval, development time, efficacy, clinical benefits, innovation, trials, endpoints, quality of life, value, and price.Breast cancer (Tokyo, Japan) · 2024Pooled it
- Complement inhibitor therapy should be available to patients with paroxysmal nocturnal hemoglobinuria throughout the world.International journal of hematology · 2026Review
- Article
- Progression-Free Survival Effect Sizes in Randomized Controlled Trials of Targeted Cancer Therapies with Positive Outcomes.Acta haematologica · 2026Article
- Patient Perspectives on Approval Speed vs Evidentiary Certainty in US Cancer Drug Approvals.JAMA network open · 2026Article
- Can We Trust PAICs in Rare Diseases? Methodological Challenges and Limitations.Journal of market access & health policy · 2026Article
- Cannabinoid Therapies in Less-Common Disorders: Clinical Evidence and Formulation Strategies.Diseases (Basel, Switzerland) · 2026Review
- The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog.Frontiers in pharmacology · 2026Article
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Value-Based Indication-Specific Pricing and Weighted-Average Pricing: Estimated Price and Cost Savings for Cancer Drugs.PharmacoEconomics · 2025Article
- Evidence available and used by the Food and Drug Administration for the approval of orphan and nonorphan drugs.Health affairs scholar · 2025Article
- Article
- Determinants of orphan drug health technology assessment in South Korea: an empirical analysis.Frontiers in pharmacology · 2025Article
- Obstacles to Early Diagnosis of Gaucher Disease.Therapeutics and clinical risk management · 2025Review
- Prevalence, diagnostic features, and medical outcomes of females with Turner syndrome with a trisomy X cell line (45,X/47,XXX): Results from the InsighTS Registry.American journal of medical genetics. Part A · 2024Article
- An 'Adaptive Treatment Strategy' for Oral Vancomycin in Patients with the Orphan Disease Primary Sclerosing Cholangitis.Digestive diseases and sciences · 2024Review
- Impact of competition on reimbursement decisions for cancer drugs in China: an observational study.The Lancet regional health. Western Pacific · 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
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Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo analyze the US Food and Drug Administration (FDA) approval, trials, unmet needs, benefit, and pricing of ultra-rare (<6600 affected US citizens), rare (6600-200 000 citizens), and common (>200 000 citizens) orphan cancer drug indications and non-orphan cancer drug indications.
designCross sectional analysis.
settingData from Drugs@FDA, FDA labels, Global Burden of Disease study, and Medicare and Medicaid. POPULATION: 170 FDA approved drugs across 455 cancer indications between 2000 and 2022.
main outcome measuresComparison of non-orphan and ultra-rare, rare, and common orphan indications regarding regulatory approval, trials, epidemiology, and price. Hazard ratios for overall survival and progression-free survival were meta-analyzed.
results161 non-orphan and 294 orphan cancer drug indications were identified, of which 25 were approved for ultra-rare diseases, 205 for rare diseases, and 64 for common diseases. Drugs for ultra-rare orphan indications were more frequently first in class (76%
conclusionsThe Orphan Drug Act of 1983 incentivizes development of drugs not only for rare diseases but also for ultra-rare diseases and subsets of common diseases. These orphan indications fill significant unmet needs, yet their approval is based on small, non-robust trials that could overestimate efficacy outcomes. A distinct ultra-orphan designation with greater financial incentives could encourage and expedite drug development for ultra-rare diseases.
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