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Publications

Journal Article Ppublish

PubMed: 42762559 DOI: 10.1016/j.ejca.2026.117058

Access to early-phase oncology trials: Trajectories and outcomes of more than 26,000 patients referred to a high-volume phase I unit.

Eur J Cancer 247:117058

Authors: Matthieu Roulleaux Dugage, Camille Mazza, Paul Matte, Cyril Roussel-Simonin, Arnaud Bayle, Antoine Hollebecque, Kaïssa Ouali, Madona Sakkal, Anas Gazzah, Jean-Marie Michot, Rastislav Bahleda, Thomas Hueso, Anna Patrikidou, Sophie Postel-Vinay, Cédric Pobel, François-Xavier Danlos, Vincent Ribrag, Capucine Baldini, Isabelle Borget, Fabrice André, Fabrice Barlesi, Yohann Loriot, Aurélien Marabelle, Christophe Massard

Affiliations

  • Gustave Roussy, Drug Development Department (DITEP), INSERM (U1363, U981), Paris Saclay University, Villejuif, France.
  • Institut Godinot, Early Phase Trial Unit, Reims, France.
  • Gustave Roussy, Drug Development Department (DITEP), Medical Oncology Department, Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), Medical Oncolo Deparment, Interdisciplinary Department of Patient Pathway Coordination (DIOPP), Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), Hematology Department, Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), INSERM (DNA repair, chromatin remodeling and epigenetics U981), Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), INSERM (U981), Paris Saclay University, Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), INSERM (Translational Immunotherapy Lab U1015, CIC1428), Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), INSERM (U1363), Villejuif, France.
  • Gustave Roussy, Department of Epidemiology and Biostatistics, University Paris Saclay, Villejuif, France.
  • Gustave Roussy, Medical Oncology Department, Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), Department of Medical Oncology, INSERM (U981), Université Paris Saclay, Villejuif, France.
  • Gustave Roussy, Drug Development Department (DITEP), Paris Saclay University, Villejuif, France.

Abstract

BACKGROUND

Early-phase oncology trials (EPT) are a mandatory step in anticancer drug development, providing early access to innovative therapies for patients with cancer. The trajectory of patients referred for such selective trials - including treatment access and outcomes across tumour types - remain a challenge.

METHODS

This retrospective monocentric study included all patients referred to Gustave Roussy's Drug Development Department between January 1, 2007 and December 31, 2023. Patient characteristics, tumour types, referral patterns, trial inclusion and investigational treatments were collected. Time to treatment failure (TTF) was analysed among treated patients.

FINDINGS

Among 26,394 patients referred for EPT, 13,713 (52%) were evaluated in consultation and 5847 signed informed consent for an EPT (22%). Overall, 4769 unique patients received at least one dose of experimental treatment (18%), corresponding to 5438 inclusions. Patients were recruited across France, but distance from the unit did not influence TTF. The therapeutic landscape evolved markedly, with the emergence of antibody-drug conjugates and T-cell engagers and a decline in conventional monoclonal antibodies. Access to EPT varied substantially across tumour types and was not proportional to referral volume. Non-small cell lung cancer was the most referred tumour type (n = 5025, 19%). Breast, colorectal and pancreatic cancers accounted for a large share of referrals but had relatively low treatment rates, highlighting potential unmet medical needs.

INTERPRETATION

This study represents the largest real-world cohort evaluating the referral-to-treatment trajectory in EPT. While providing access to therapeutic innovation, disparities in trial access and in treatment duration persist across tumour types.

MeSH

Cells, Pharmaceutical Preparations, Neoplasms, Therapeutics