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Clinical value of guideline recommended molecular targets and genome targeted cancer therapies: cross sectional study

Ariadna Tibau1,2, Thomas J Hwang3,4,5, Jerry Avorn3

  • 1Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA atibaumartorell@bwh.harvard.edu.

BMJ (Clinical Research Ed.)
|August 20, 2024
PubMed
Abstract

Insights

Most genome-targeted cancer drugs recommended by the National Comprehensive Cancer Network (NCCN) lack high clinical benefit. Further evaluation is needed to align NCCN recommendations with evidence-based genomic cancer treatments.

Area of Science:

  • Oncology
  • Genomics
  • Clinical Pharmacology

Background:

  • The National Comprehensive Cancer Network (NCCN) provides guidelines for cancer treatments.
  • Genome-targeted cancer drugs represent a growing area of precision oncology.
  • Assessing the clinical utility of these targeted therapies is crucial for evidence-based practice.

Purpose of the Study:

  • To evaluate the clinical benefit and actionability of molecular targets for NCCN-recommended genome-targeted cancer drugs.
  • To determine the alignment of NCCN recommendations with established frameworks for clinical benefit and target actionability.
  • To inform clinical decision-making in genomic-guided cancer therapy.

Main Methods:

  • A cross-sectional study analyzed NCCN-recommended genome-targeted drugs for advanced cancers.
  • Molecular target actionability was assessed using the European Society for Medical Oncology (ESMO) Scale for Clinical Actionability of Molecular Targets (ESCAT).
  • Clinical benefit was evaluated using the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS), categorizing benefits as 'high' or 'promising but unproven'.

Main Results:

  • Of 411 recommendations for 74 genome-targeted drugs, 84% were linked to clinical trials, but many were early-phase or single-arm studies.
  • Only 12% of scorable trials demonstrated substantial clinical benefit (ESMO-MCBS grades 4-5), while 45% showed a grade of 3 (promising but unproven benefit).
  • Combining ESCAT and ESMO-MCBS, 12% of interventions had high benefit, and 33% had promising but unproven benefit; 53% of NCCN-preferred interventions fell into these categories.

Conclusions:

  • A significant proportion of genome-targeted cancer treatments recommended by NCCN show limited or unproven clinical benefit according to ESCAT and ESMO-MCBS.
  • Only about one-eighth of these treatments offer high clinical benefit, with roughly one-third offering promising but unproven benefit.
  • Aligning NCCN recommendations with robust clinical benefit data is essential for informed, evidence-based genomic-guided cancer treatment decisions.

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