Experience and Prospects for the Use of Off-Label Drugs in Oncology

Abstract

According to the World Health Organization, half of all drugs available on the global pharmaceutical market are sometimes used for indications that are not included in the instruction for use. This method of therapy has the term “off-label use” which means the use “out of instruction”. Today, off-label drugs are also prescribed for cancer treatment. For example, a drug developed to treat one type of tumor can sometimes be used to treat other types of cancer. The treatment of certain types of pain with tricyclic antidepressants is also an example of the off-label drugs used in oncology. An example of an off-label prescription is anxiolytic medicine lorazepam, which can be used off-label as an antiemetic in cancer patients. Low doses of naltrexone are applied to treat cancer and autoimmune diseases. A retrospective analysis of modern oncotherapy indicates that oncologists often use off-label drugs in combination therapy, especially in the treatment of patients with concomitant diseases, in case of progressive development of the tumor, or to reduce the toxicity and cost of treatment components. American oncologists are of the opinion that if all the drugs prescribed by the International Recommendations failed in the treatment of cancer, doctors can prescribe off-label medications, but only if their effectiveness and safety are clearly established. The problem of the off-label use of drugs in oncology has not yet been studied in detail, however, this direction has certain promising prospects.

Real‐world treatment outcomes of immune checkpoint inhibitors used off‐label in oncology: A comprehensive cancer institution experience

“…Off‐label use (OLU) refers to the utilization of drugs outside their approved indications by regulatory agencies 1 . Cancer treatment often involves OLU due to the complexity of the disease, limited treatment options, and lengthy clinical trial timelines 1–4 . To bring a drug to the market, it must obtain approval from a regulatory authority such as the European Medicines Agency (EMA) in the European Union (EU).…”

“…1 Cancer treatment often involves OLU due to the complexity of the disease, limited treatment options, and lengthy clinical trial timelines. 1 , 2 , 3 , 4 To bring a drug to the market, it must obtain approval from a regulatory authority such as the European Medicines Agency (EMA) in the European Union (EU). After receiving marketing authorization, pharmaceutical companies are required to apply for marketing evaluation in each member state.…”

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

“…3,4,6,9,[12][13][14][15][16][17] Moreover, few studies have evaluated the clinical outcomes of OLU of medicines in cancer treatment in terms of effectiveness, mostly based instead on cost and reimbursement issues. 6,10,12,[18][19][20][21][22][23][24][25] The Catalan Institute of Oncology (ICO) is a comprehensive multicentre cancer institution providing clinical oncology-hematology care for approximately 45% of adult cancer patients in Catalonia. In 2022, ICO provided tertiary level cancer coverage for an adult population of 3,203,000 inhabitants.…”

“…However, data on OLU (or on MSS use as a wider concept) health outcomes in real‐word practice in oncology are scarce and heterogenic, coming from small studies with wide variability in their design, sample size and pathologies involved, being extremely difficult to compare and extrapolate their results 3,4,6,9,12–17 . Moreover, few studies have evaluated the clinical outcomes of OLU of medicines in cancer treatment in terms of effectiveness, mostly based instead on cost and reimbursement issues 6,10,12,18–25 …”

“…According to the literature, oncology/hematology, psychiatry, and rheumatology are clinical areas of interest regarding OLU 3,9–12 . Based on an EMA report, 13%–71% of adult patients with cancer will receive at least one chemotherapy as OLU during their course of treatment 3,13 .…”