ArticleTargeted oncology2023
FDA Approvals of Oncology Drugs for Tissue-Agnostic Indications.
Article in Targeted oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.
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
5 citing papers in PubMed, 1 synthesis or guideline pooled it, 5 citations in OpenAlex.
- BRAF-targeted therapy in non-melanomatous BRAF-mutant tumours: a systematic review of broad but histology-modulated efficacy.International journal of clinical oncology · 2026Pooled it
- Tumor morphology on CT radiomics is largely driven by the local anatomical environment, not the primary tumor type.European radiology experimental · 2026Article
- The Utility of Tissue-Agnostic Drugs in Lung Cancer Treatment.Pharmaceutical medicine · 2025Review
- High tumor mutational burden assessed through next-generation sequencing predicts favorable survival in microsatellite stable metastatic colon cancer patients.Journal of translational medicine · 2024Article
- Companion Tests and Personalized Cancer Therapy: Reaching a Glass Ceiling.International journal of molecular sciences · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe development of molecularly targeted oncology drugs for tissue-agnostic indications represents a new paradigm, but marketing authorization may carry additional risks due to uncertainties about extrapolating drug safety and efficacy, and biomarker test accuracy, to unstudied tumor types.
objectiveTo determine tumor types represented and method of mutation identification in trials supporting the US Food and Drug Administration (FDA) approvals of tissue-agnostic drugs, and to describe post-marketing requirements and commitments (PMRs/PMCs) issued for studies to evaluate additional tumor types and to validate companion diagnostic devices.
methodsFor each tissue-agnostic drug approval identified via the FDA's Hematology/Oncology Approvals and Safety Notifications website, prescribing information, approval packages, and letters were retrieved from the Drugs@FDA website. Characteristics of approvals, details of supporting trials, and PMRs/PMCs for clinical trials and diagnostic tests were extracted.
resultsSix drugs were approved for seven tissue-agnostic indications between 2017 and 2022, with 9-15 different tumor types represented in trials supporting approvals. Only one approval prospectively utilized a commercial assay to identify the molecular alteration of interest in tumor samples. All seven approvals were issued PMRs for trials with additional tumor types, and six of seven were issued PMCs for studies to support labeling for companion diagnostic devices.
conclusionThe number of patients and cancer subtypes in trials supporting tissue-agnostic oncology drug approvals varied by mutation. Most drug approvals did not have concurrent approval of a diagnostic test. Post-marketing studies play a critical role in confirming clinical benefit and ensuring companion diagnostic device performance across a broader range of tumor types.
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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.