ArticleJAMA network open2022
Use of Extrapolation in New Drug Approvals by the US Food and Drug Administration.
Article in JAMA network open, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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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
7 citing papers in PubMed.
- FDA Approval Summary: Blinatumomab for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia in the Consolidation Phase of Multiphase Chemotherapy.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025Article
- Advances and critical aspects in cancer treatment development using digital twins.Briefings in bioinformatics · 2025Review
- Biosimilars: A Critical Review of Development, Regulatory Landscape, and Clinical Implications.AAPS PharmSciTech · 2025Review
- Empirical data drift detection experiments on real-world medical imaging data.Nature communications · 2024Article
- Improving Food and Drug Administration-Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval.The Milbank quarterly · 2023Article
- Commercial health plans use of patient subgroup restrictions: An analysis of orphan and US Food and Drug Administration-expedited programs.Journal of managed care & specialty pharmacy · 2023Article
- Characteristics of Prior Authorization Policies for New Drugs in Medicare Part D.JAMA health forum · 2023Article
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3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Importance: The US Food and Drug Administration (FDA)-approved indications can be factors in prescribing practices and insurance coverage, yet the frequency with which the extrapolation of clinical characteristics from pivotal trial data to the final approved indication occurs is not well understood. Objectives: To evaluate the frequency of extrapolation beyond pivotal trial data into approved indications in relation to disease severity, disease subtype, and concomitant medication use. Design, Setting, and Participants: In a cross-sectional study, the characteristics of patients in pivotal trials of 105 novel drug approvals from 2015 to 2017 were identified and compared with the FDA-approved indications for the drugs. Main sources analyzed included FDA reviews, published material describing the pivotal trials, and the original drug labeling. The study was conducted from July 4, 2019, to June 1, 2021. Exposures: Clinical characteristics of pivotal trials used in FDA approval. Main Outcomes and Measures: Main outcomes included the nature and frequency of extrapolation from study populations to the final indications. Extrapolation was defined as the granting of an indication for use in a broader population than was included in the pivotal trials on the basis of disease severity, disease subtype, or concomitant medication use. Results: Among the 105 novel FDA drug approvals studied, 23 extrapolations of trial population characteristics to the approved indication were identified in 21 drugs (20%): 12 times (29%) in 2015, 3 times (15%) in 2016, and 6 times (14%) in 2017. Extrapolation of trial findings to patients with greater disease severity was most common (n = 14 drugs), followed by differences in disease subtype (n = 6) and concomitant medication use (n = 3). Conclusions and Relevance: The findings of this study suggest that extrapolation from pivotal trial data to FDA-approved indications is common. Although extrapolations may be grounded in reasonable clinical predictions, they can limit the generalizability of such indications to specific prescribing decisions; these findings suggest a greater need for close postapproval monitoring to determine whether new safety issues arise, or effectiveness differs from expectations when these medications are used in broader real-world populations.
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