Evidence map›Paper›PMID 29473145›Full record

ReviewClinical pharmacology and therapeutics2018

Considerations for Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease.

Robert N Schuck, Janet Woodcock, Issam Zineh, Peter Stein, Jonathan Jarow, Robert Temple, Thomas Permutt, Lisa LaVange, Julia A Beaver, Rosane Charlab and 8 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Clinical pharmacology and therapeutics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed.

  1. FDA Approval Summary: Niraparib plus Abiraterone Acetate Fixed-Dose Combination for BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025
    Trial
  2. FDA Approval Summary: Ripretinib for Advanced Gastrointestinal Stromal Tumor.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023
    Trial
  3. FDA Approval Summary: Gilteritinib for Relapsed or Refractory Acute Myeloid Leukemia with aClinical cancer research : an official journal of the American Association for Cancer Research · 2021
    Trial
  4. Article
  5. Review
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2020
    Article
  12. Review
  13. Article
  14. Review
  15. Review
  16. Health Care Evolves From Reactive to Proactive.Clinical pharmacology and therapeutics · 2019
    Article
  17. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

Robert N SchuckOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Janet WoodcockCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Issam ZinehOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Peter SteinOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Jonathan JarowCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Robert TempleCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Thomas PermuttOffice of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Lisa LaVangeOffice of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Julia A BeaverOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Rosane CharlabOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Gideon M BlumenthalOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Sarah E DorffOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Christopher LeptakOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Steven LemeryOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Hobart RogersOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Badrul ChowdhuryOffice of New Drugs, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
E David LitwackOffice of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Michael PacanowskiOffice of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

Funding

Intramural FDA HHS FD999999
6 · The paper itself

Abstract

Advances in our understanding of the molecular underpinnings of disease have spurred the development of targeted therapies and the use of precision medicine approaches in patient care. While targeted therapies have improved our capability to provide effective treatments to patients, they also present additional challenges to drug development and benefit-risk assessment such as identifying the subset(s) of patients likely to respond to the drug, assessing heterogeneity in response across molecular subsets of a disease, and developing diagnostic tests to identify patients for treatment. These challenges are particularly difficult to address when targeted therapies are developed to treat diseases with multiple molecular subtypes that occur at low frequencies. To help address these challenges, the US Food and Drug Administration recently published a draft guidance entitled "Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease." Here we provide additional information on specific aspects of targeted therapy development in diseases with low-frequency molecular subsets.

Indexed as

Gene FrequencyGenetic Predisposition to DiseaseMutation RateAnimalsClinical Trials as TopicDrug Evaluation, PreclinicalEvidence-Based MedicineHumansMolecular Targeted TherapyPhenotypePrecision MedicineUnited StatesUnited States Food and Drug Administration

Identifiers

PMID29473145
PMCPMC6347014

What OpenQuestion holds

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Registered trials

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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.