Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Aug;12(16):17112-17125.
doi: 10.1002/cam4.6360. Epub 2023 Jul 26.

Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution

Sandra Fontanals  1 , Anna Esteve  2 , Andrea González  3 , Cristina Ibáñez  4 , Javier Martínez  5 , Ricard Mesía  3 , Ana Clopés  6
Affiliations

Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution

Sandra Fontanals et al. Cancer Med. 2023 Aug.

Abstract

Purpose: Medicines in special situations (MSS) refer to off-label or to unlicensed drugs under investigation (compassionate use). Our objectives were to evaluate characteristics and to estimate overall survival (OS), event-free survival (EFS), and the duration of treatment (DT) of MSS used for cancer treatment at a multicentre comprehensive cancer institution.

Methods: Retrospective cohort study on adult cancer patients for whom an MSS treatment was requested (January 2011-December 2020). A descriptive analysis was performed and median OS and EFS and 95% confidence intervals (CIs) were estimated. Survival curves were stratified by type of tumor, ECOG (Eastern Cooperative Oncology Group) performance status (PS), age, sex, treatment stage and type of drug (mechanism of action and target).

Results: Treatment was initiated in 2092 episodes (1930 patients) out of 2377 MSS episodes (2189 patients) requested, 33% for hematological treatment and 87% for advanced stage cancer. Median OS (months) was 21.1 (95% CI 19.4-22.7), median EFS was 5.6 (95% CI 5.1-6.0) months, and median DT was 4.5 [0.0; 115.3] months. OS and EFS statistically significantly favored female patients, ECOG PS ≥2 episodes showed worse OS and EFS outcomes (p < 0.0001). Statistically significant differences in survival were found within solid and hematological cancer, disease stage, drug mechanism of action, and type of cancer (p < 0.001) but not for age. Survival outcomes by tumor subtype and drug are presented both globally and separately based on disease stage.

Conclusion: MSS uses are practiced across almost all cancer types, mostly for advanced disease. ECOG PS ≥2, along with advanced disease, was related to worse survival. Information about real-world outcomes is valuable and contributes to better decision-making regarding MSS and our experience in this field could be of interest for other colleagues.

Keywords: cancer; compassionate use; off-label use; survival; treatment outcomes.

PubMed Disclaimer

Conflict of interest statement

The author(s) declare(s) that there are no conflicts of interest regarding the publication of this article.

Figures

FIGURE 1
FIGURE 1
Flow chart.
FIGURE 2
FIGURE 2
Overall survival (OS) (A) and event‐free survival (EFS) (B) according to cancer type.
See this image and copyright information in PMC

References

    1. 2021 FDA approvals of drugs for cancer treatment—the ASCO post . 2021. https://ascopost.com/issues/december‐25‐2021/2021‐fda‐approvals‐of‐drugs...
    1. Casali PG. The off‐label use of drugs in oncology: a position paper by the European Society for Medical Oncology (ESMO). Ann Oncol. 2007;18(12):1923‐1925. doi:10.1093/annonc/mdm517 - DOI - PubMed
    1. Weda M, Hoebert J, Moltó Puigmarti C, et al. Study on off‐label use of medicinal products in the European Union. Eur Union. 2017;1‐185. https://www.nivel.nl/sites/default/files/bestanden/Report_OFF_LABEL_Nive...
    1. Trotta F, Leufkens HGM, Schellens JHM, Laing R, Tafuri G. Evaluation of oncology drugs at the European Medicines Agency and US Food and Drug Administration: when differences have an impact on clinical practice. J Clin Oncol. 2011;29(16):2266‐2272. doi:10.1200/JCO.2010.34.1248 - DOI - PubMed
    1. Lee VC. Off‐label drug information: regulation, distribution, evaluation, and related controversies. P&T. 2009;34(8):428‐40. https://pubmed.ncbi.nlm.nih.gov/20140107/ - PMC - PubMed

Publication types