Evidence map›Paper›PMID 38112833›Full record

ArticleInternational journal of clinical oncology2024

Platform trial for off-label oncology drugs using comprehensive genomic profiling under the universal public healthcare system: the BELIEVE trial.

Sae Ishimaru, Tatsunori Shimoi, Kuniko Sunami, Miho Nakajima, Yayoi Ando, Natsuko Okita, Kenichi Nakamura, Taro Shibata, Yasuhiro Fujiwara, Noboru Yamamoto

Abstract readAdaptive Clinical Trial
In one paragraph

Article in International journal of clinical oncology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–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

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Review
  5. Review
  6. Article
  7. Article
  8. Drug-resistantSurgical neurology international · 2024
    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

10 authors.

Sae IshimaruResearch Management Division, Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.
Tatsunori ShimoiDepartment of Medical Oncology, National Cancer Center Hospital, Tokyo, Japan.
Kuniko SunamiDepartment of Laboratory Medicine, National Cancer Center Hospital, Tokyo, Japan.
Miho NakajimaDepartment of Pediatric Oncology, National Cancer Center Hospital, Tokyo, Japan.
Yayoi AndoResearch Management Division, Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.
Natsuko OkitaResearch Management Division, Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.
Kenichi NakamuraDepartment of International Clinical Development/Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.
Taro ShibataBiostatistics Division, Center for Research Administration and Support, National Cancer Center, Tokyo, Japan.
Yasuhiro FujiwaraDepartment of Medical Oncology, National Cancer Center Hospital, Tokyo, Japan.
Noboru YamamotoDepartment of Experimental Therapeutics, National Cancer Center Hospital, 5-1-1 Tsukiji, Chuo-ku, Tokyo, 104-0045, Japan. nbryamam@ncc.go.jp.ORCID http://orcid.org/0000-0002-0787-2851

Funding

Japan Agency for Medical Research and Development 20ck0106622h0001Ministry of Health, Labour and Welfare 19EA1008
6 · The paper itself

Abstract

backgroundPrecision medicine has transformed cancer treatment by focusing on personalized approaches based on genomic abnormalities. However, comprehensive genomic profiling (CGP) and access to targeted therapies are limited in Japan. This study investigates the BELIEVE trial, which aims to improve drug accessibility for patients with actionable genetic abnormalities through off-label drug administration.

methodsThe BELIEVE trial is a platform trial with a single master protocol, conducted under the Clinical Trials Act and the patient-proposed health services (PPHS) scheme. Eligible patients with solid tumors exhibiting actionable alterations were enrolled, and CGP tests covered by national health insurance were employed. Treatment selection, study drugs from collaborating pharmaceutical companies, and treatment schedules adhered to predefined protocols. Primary and secondary endpoints were evaluated, and statistical analysis was conducted based on patient response rates.

resultsThe BELIEVE trial offered treatment opportunities for patients with relapse/refractory disease who lacked standard therapies or clinical trial options. This study addresses unmet medical needs and contributes to the establishment of precision medicine systems. Similar trials like NCI-MATCH and TAPUR are being conducted globally. The BELIEVE trial provides a platform for off-label drug administration, collects essential clinical data, and contributes to drug approval applications.

conclusionThe BELIEVE trial provides hope for patients with actionable genetic abnormalities by facilitating access to targeted therapies through off-label drug administration. It establishes a regulatory framework and promotes collaboration between industry and academia by expanding organ-specific and cross-organ biomarker-based treatments.

Indexed as

NeoplasmsOff-Label UseDelivery of Health CareGenomicsHumansPharmaceutical PreparationsPharmaceutical PreparationsComprehensive genomic profilingGenomic medicineMolecular targeted therapyPatient-proposed healthcare systemUmbrella or basket-type trial

Identifiers

PMID38112833
PMCPMC10808137

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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.