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Off-label Medication Use In Oncology

Off-label Medication Use In Oncology

Cancer Research and Medicinal Drugs

Cancer research and innovation are highly active, resulting in the introduction of new medications into the therapeutic arsenal. However, incorporating these new drugs into clinical practice is a challenging and time-consuming process due to regulatory requirements. Before a drug can be marketed and used, it must obtain marketing authorization from a regulatory body like the European Medicines Agency (EMA) in the European Union or the Food and Drug Administration (FDA) in the United States. Additionally, each member state in the EU and the USA decides on its own conditions for reimbursement within its National Health Service. This entire process can take more than 18 months, causing a potential mismatch between the patient’s need for the drug and the regulatory timeline.

The usage of off-label drugs, including the compassionate use of investigational medications in cancer care, is widespread, but there is no global consensus on its definition and regulations. Spain has specific legislation governing the use of medicinal products in special situations, encompassing off-label use, compassionate use, and the use of foreign drugs. However, data on the health outcomes of such practices are limited and diverse, making it difficult to draw conclusions. The Catalan Institute of Oncology (ICO) conducted a study to assess the real-world outcomes of patients treated with medicinal products in special situations. They found that overall survival was influenced by disease stage, performance status, and drug mechanism of action, while age did not show significant effects. This information can be beneficial for decision-making and may interest other medical professionals working in the field of cancer treatment.

Study Methods 

The researchers conducted a retrospective multicenter cohort study involving adult cancer patients in Catalonia, Spain, who received off-label medications called Medicinal Products in Special Situations (MSS) from January 2011 to December 2020. Patients aged 16 years or older, treated at three different ICO centers, were included in the study. Data on patient characteristics, cancer treatment details, and outcomes were collected from institutional clinical records and databases. The study analyzed overall survival (OS), event-free survival (EFS), and duration of MSS treatment (DT) based on various factors such as age, gender, performance status, treatment context, and mechanism of action of MSS drugs. Survival analyses were also performed based on tumor type and molecular target for frequently requested drugs. The study aimed to provide valuable information on the real-world outcomes of MSS use in cancer treatment. 

The study conducted a descriptive analysis using demographic, treatment, and pathology data from patients’ medical records. Continuous variables were described using medians and interquartile ranges, while categorical variables were presented as frequencies and percentages. Median overall survival (OS) and median event-free survival (EFS) with 95% confidence intervals were estimated using the Kaplan-Meier method. Survival curves were stratified by tumor type, ECOG PS, age, sex, treatment stage, and type of drug (mechanism of action and target) and compared using the log-rank test. The statistical analysis was performed using R v. 4.1.2 software.


Between January 2011 and December 2020, a total of 46,749 adult cancer patients received treatment across three centers in Catalonia, Spain. Among them, 2,189 patients met the study’s inclusion criteria, and 5% of these patients were eligible for off-label Medicinal Products in Special Situations (MSS) use. One-third of MSS requests were for hematological malignancies, and two-thirds were for solid neoplasms. The majority of MSS requests were approved, but 6% were denied. The study analyzed 2,092 MSS episodes for 1,930 patients. Most requests were for advanced/metastatic solid tumors or refractory/relapsed hematological malignancies. The most frequently administered drugs were monoclonal antibodies and oral targeted therapies. The study aimed to provide insights into the real-world outcomes of off-label medication use in cancer treatment. 

The study included 1930 patients with 268 different drug schemes administered for 51 oncological and hematological diseases. The majority of MSS requests were for refractory/relapsed hematological malignancies (83%) or advanced/metastatic solid tumors (86%). Two-thirds of treatments were administered as monotherapy, with 40% being exclusively oral. 74% of the MSS requests involved drugs supported by strong evidence and approved by regulatory agencies. The most frequently administered drugs were monoclonal antibodies (mAbs) and oral targeted therapies. The study provides detailed information about the drugs used for different tumor types in the oncology and hematology populations.

The study analyzed the outcomes of 2,092 episodes of Medicinal Products in Special Situations (MSS) used for cancer treatment. The median overall survival (OS) for the entire group was 21.1 months, while the median event-free survival (EFS) was 5.6 months. MSS treatment lasted over 3 months in most cases. Discontinuation of treatment was mainly due to disease progression (65.6%), followed by treatment completion (19%) and toxicity (13.7%). Certain factors, such as performance status, sex, and type of drug, had an impact on survival outcomes. Hematological malignancies and solid tumors showed different survival patterns. Some treatments had longer-lasting responses, particularly with oral targeted therapies and monoclonal antibodies. Overall, the study provided comprehensive information on MSS use and its outcomes for cancer patients. 


This study is the first of its kind, providing real-world outcomes of patients treated with Medicinal Products in Special Situations (MSS) for cancer in a comprehensive cancer institution. The study analyzed overall survival (OS) and event-free survival (EFS) and identified factors associated with survival outcomes. 

The proportion of patients receiving MSS at the institution was 5%, which is lower than previously reported, likely due to the exclusion of supportive care MSS and the hospital’s procedures for evaluating treatment requests. Clinicians had to justify their patients’ treatment requests, including alternative treatments, supported by evidence review reports from hospital pharmacists. Previous literature lacks sufficient data on MSS survival outcomes in oncology, often having small sample sizes or focusing on specific drugs or pathologies.

The information obtained from the present study provides valuable insights and contributes to better decision-making regarding MSS use. The findings allow for discussions within the P&T Committee about the clinical criteria that should be met before requesting an MSS, leading to improved treatment benefits. The results obtained from the cohort study enhance knowledge about real-world treatment outcomes and enable benchmarking with clinical trial results for common pathologies and drugs. This experience and the procedures related to OLU may prove useful and applicable to other colleagues and healthcare systems in different countries.

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