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Off-label drug use in oncology: integrating molecular and genetic analysis in an academic center's real-world
E Cioli1, G Suarato1, R Napolitano1
1Medical Oncology, Department of Precision Medicine, Università degli Studi della Campania 'Luigi Vanvitelli', Naples, Italy.
Background:
Recent advancements in cancer treatment have shifted the paradigm from chemotherapy to targeted therapies and immunotherapies, proving effective even in early-stage cancers. Regulatory agencies like the Food and Drug Administration, European Medicines Agency, and Italian Agency for Drugs have approved several targeted therapies for specific genetic mutations, significantly improving outcomes for several diseases. These advancements highlight the importance of identifying molecular targets, even in historically difficult-to-treat cancers.
Materials And Methods:
A retrospective observational study was conducted at the University of Campania "Luigi Vanvitelli" from January 2015 to January 2022, involving 83 adult patients with solid tumors. Patients received off-label drugs at no cost. The study analyzed treatment and pathology data, focusing on progression-free survival (PFS) and overall survival (OS), using Kaplan-Meier estimates and the log-rank test for statistical analysis.
Results:
Patients averaged 63 years old, with a slight male majority. Off-label drugs were primarily requested for upper gastrointestinal cancers (42%) and colorectal carcinoma (21%). The most common molecular testing method was next-generation sequencing (NGS). Immunotherapy was the predominant treatment (67%), followed by targeted therapy and chemotherapy. The median PFS was 3.4 months [95% confidence interval (CI) 2.0-4.5 months], and the median OS was 10 months (95% CI 7.5-12.2 months). Early-line off-label treatment showed a trend toward longer PFS compared with later lines.
Conclusion:
Early access to off-label drugs, particularly for patients with specific genetic alterations, can improve outcomes. The study underscores the importance of molecular tumor boards and multidisciplinary collaboration in selecting off-label treatments. Despite regulatory and ethical challenges, off-label drug use offers significant therapeutic opportunities, necessitating well-designed clinical trials and registries to better understand their efficacy and safety.
Insights
Early access to off-label cancer drugs, especially those targeting specific genetic mutations, shows promise for improving patient survival. This study highlights the potential of these treatments, even with regulatory hurdles.
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Cancer treatment is evolving from traditional chemotherapy to targeted therapies and immunotherapies.
- Approved targeted therapies for specific genetic mutations have improved outcomes in various cancers.
- Identifying molecular targets is crucial, even for challenging cancer types.
Purpose of the Study:
- To evaluate the efficacy of off-label drug use in adult patients with solid tumors.
- To analyze progression-free survival (PFS) and overall survival (OS) in patients receiving off-label treatments.
- To explore the role of molecular profiling in guiding cancer therapy decisions.
Main Methods:
- Retrospective observational study of 83 adult patients with solid tumors (Jan 2015-Jan 2022).
- Analysis of treatment and pathology data for patients receiving donated off-label drugs.
- Statistical analysis using Kaplan-Meier estimates and log-rank test for PFS and OS.
Main Results:
- Median PFS was 3.4 months; median OS was 10 months.
- Immunotherapy was the most common treatment (67%), followed by targeted therapy.
- Early-line off-label treatment trended towards longer PFS than later-line treatments.
Conclusions:
- Off-label drug access, especially for specific genetic alterations, can enhance patient outcomes.
- Molecular tumor boards and collaboration are vital for selecting appropriate off-label treatments.
- Further clinical trials and registries are needed to assess the efficacy and safety of off-label drug use.
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