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Loose Regulatory Standards Portend a New Era of Imprecision Oncology

Ali Raza Khaki1

  • 1Division of Medical Oncology, Department of Medicine, University of Washington School of Medicine, Seattle, WA, USA.

Cancer Investigation
|December 8, 2020
PubMed

Insights

Lenient U.S. Food and Drug Administration (FDA) approvals for precision oncology drugs may jeopardize this approach. Broad indications beyond studied populations risk patient harm and undermine cancer treatment advancements.

Area of Science:

  • Oncology
  • Pharmacology
  • Regulatory Science

Background:

  • Precision oncology aims to tailor cancer treatments to individual patient molecular profiles.
  • Recent advancements in cancer drug development focus on targeted therapies.
  • The United States Food and Drug Administration (FDA) plays a crucial role in drug approval processes.

Purpose of the Study:

  • To analyze recent FDA drug approvals within the precision oncology framework.
  • To evaluate whether FDA indications align with studied patient populations.
  • To discuss the potential negative consequences of overly broad drug approvals.

Main Methods:

  • Review of two specific FDA drug approvals: pembrolizumab and olaparib.
  • Comparison of FDA-granted indications with the populations studied in clinical trials.
  • Commentary on the implications of these approval decisions for precision oncology.

Main Results:

  • Identified instances where FDA drug indications exceeded the scope of clinical trial populations.
  • Pembrolizumab approved for tumor mutation burden-high solid tumors, broader than studied.
  • Olaparib approved for castration-resistant prostate cancer with specific mutations, with a broad indication.

Conclusions:

  • Broad FDA approvals can deviate from precision oncology principles.
  • Overly broad indications may lead to patient harm and inappropriate treatment selection.
  • Regulatory leniency in precision oncology risks undermining the field's potential.
Keywords:
OlaparibPrecision oncologyTMB-highhomologous recombination deficiencypembrolizumab

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