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Platform trial for off-label oncology drugs using comprehensive genomic profiling under the universal public

Sae Ishimaru1,2, Tatsunori Shimoi3, Kuniko Sunami4

  • 1Research Management Division, Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.

International Journal of Clinical Oncology
|December 19, 2023
PubMed
Abstract

Insights

The BELIEVE trial expands access to targeted cancer therapies for Japanese patients with actionable genetic abnormalities through off-label drug use. This precision medicine initiative addresses unmet needs and facilitates drug approvals.

Area of Science:

  • Oncology
  • Genomics
  • Clinical Trials

Background:

  • Precision medicine utilizes genomic abnormalities for personalized cancer treatment.
  • Access to comprehensive genomic profiling (CGP) and targeted therapies is limited in Japan.
  • The BELIEVE trial aims to improve drug accessibility for patients with actionable genetic abnormalities via off-label administration.

Purpose of the Study:

  • To investigate the BELIEVE trial's effectiveness in enhancing drug accessibility for patients with actionable genetic abnormalities.
  • To establish a framework for off-label drug administration in precision oncology within Japan.

Main Methods:

  • A platform trial with a single master protocol under the Clinical Trials Act and PPHS scheme.
  • Enrollment of eligible patients with solid tumors and actionable alterations.
  • Utilization of CGP tests covered by national health insurance for treatment selection.

Main Results:

  • Provided treatment opportunities for patients with relapsed/refractory disease lacking standard options.
  • Addressed unmet medical needs and contributed to precision medicine system establishment.
  • Collected essential clinical data to support drug approval applications.

Conclusions:

  • The BELIEVE trial offers hope by facilitating targeted therapy access for patients with actionable genetic abnormalities.
  • Established a regulatory framework promoting industry-academia collaboration for biomarker-based treatments.
  • Expanded organ-specific and cross-organ treatments through off-label drug administration.
Keywords:
Comprehensive genomic profilingGenomic medicineMolecular targeted therapyPatient-proposed healthcare systemUmbrella or basket-type trial

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