ArticleBMJ (Clinical research ed.)2023
Therapeutic value of first versus supplemental indications of drugs in US and Europe (2011-20): retrospective cohort study.
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 13 papers.
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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
13 citing papers in PubMed.
- The lack of head-to-head randomised trials and the consequences for patients and national health service: The case of non-small cell lung cancer.European journal of clinical pharmacology · 2024Trial
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- GPCR drug discovery: new agents, targets and indications.Nature reviews. Drug discovery · 2025Review
- Pricing for Multi-Indication Drugs in the Italian Regulatory Context.PharmacoEconomics - open · 2025Article
- New Serious Safety Warnings for Targeted Anticancer Agents After Their Initial FDA Approval.Cancers · 2025Article
- Updated analysis of the prescription and evaluation of protein kinase inhibitors for oncology in Germany.Naunyn-Schmiedeberg's archives of pharmacology · 2025Article
- Why are our medicines so expensive? Spoiler: Not for the reasons you are being told….The European journal of general practice · 2024Article
- Vibration of effects resulting from treatment selection in mixed-treatment comparisons: a multiverse analysis on network meta-analyses of antidepressants in major depressive disorder.BMJ evidence-based medicine · 2024Article
- Trust in the Food and Drug Administration: A National Survey Study.Clinical pharmacology and therapeutics · 2024Article
- Timely, Cheap, or Risk-Free? The Effect of Regulation on the Price and Availability of New Drugs.Pharmacy (Basel, Switzerland) · 2024Article
- Anastrozole Protects against Human Coronavirus Infection by Ameliorating the Reactive Oxygen Species-Mediated Inflammatory Response.Antioxidants (Basel, Switzerland) · 2024Article
- A Regulatory Roadmap for Repurposing: Comparing Pathways for Making Repurposed Drugs Available In The EU, UK, And US.The Journal of law, medicine & ethics : a journal of the American Society of Law, Medicine & Ethics · 2024Article
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Authors and funding
6 authors.
Funding
Abstract
objectiveTo analyze the therapeutic value of supplemental indications compared with first indications for drugs approved in the US and Europe.
designRetrospective cohort study.
settingNew and supplemental indications approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) between 2011 and 2020.
main outcome measuresProportion of first and supplemental indications rated as having high therapeutic value using ratings from the French and German national, independent health authorities.
resultsThe cohort study included 124 first and 335 supplemental indications approved by the FDA and 88 first and 215 supplemental indications approved by the EMA between 2011 and 2020; the largest subset was for cancer disorders. Therapeutic ratings were available for 107 (86%) first and 179 (53%) supplemental indications in the US and for 87 (99%) first and 184 (86%) supplemental indications in Europe. Among FDA approved indications with available ratings, 41% (44/107) had high therapeutic value ratings for first indications compared with 34% (61/179) for supplemental indications. In Europe, 47% (41/87) of first and 36% (67/184) of supplemental indications had high therapeutic value ratings. Among FDA approvals, when the sample was restricted to the first three approved indications, second indication approvals were 36% less likely to have a high value rating (relative ratio 0.64, 95% confidence interval 0.43 to 0.96) and third indication approvals were 45% less likely (0.55, 0.29 to 1.01) compared with the first indication approval. Similar findings were observed for Europe and when weighting by the inverse number of indications for each drug.
conclusionsThe proportion of supplemental indications rated as having high therapeutic value was substantially lower than for first indications. When first or supplemental indications do not offer added therapeutic value over other available treatments, that information should be clearly communicated to patients and physicians and reflected in the price of the drugs.
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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.