Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study

Study Overview
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Brief Summary
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Off-label drug use, where a marketed drug is used outside its approved indication, may allow early access to new and promising treatments. However, its use can be a source of controversy, due to limited evidence for clinical benefit and lack of cost/QALY-estimates, leading to challenging prioritization issues. The number of drugs suitable for off-label use is expected to further increase in the coming years, owing to the rapid progress in the field of oncology, in particular with the current era of precision medicine and targeted therapies. This also challenges the traditional method of running clinical trials, with eligible patient populations commonly being small, underpinning the importance of gaining supplementary real-world evidence from well performed observational studies. This prospective observational study will therefore assess real-world outcomes of patients treated with off-label anti-cancer drugs, including efficacy in terms of response rates, time to progression/relapse measures and survival; patient-reported outcome measures (PROMS) and self-reported side-effects/toxicity; as well as collecting blood samples for a biobank for further translational research. Further, the study will give a descriptive analysis of the current practice of off-label use of anti-cancer drugs in Norway, including prevalence estimation and health care related cost analyses.

Official Title
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Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study

Conditions
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Cancer

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Verified cancer diagnosis (based on radiological, histological/cytological or operative evidence). Treatment with off-label anti-cancer drug. Age ≥ 18 years Able to provide written informed consent. Exclusion Criteria: None

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression free survival (PFS). | Time from date of inclusion until the date of first documented progression or date of death from any cause, whichever come first, according to RECIST v1.1 | Assessed up to 2 years after end of inclusion | 
| Patients questionnaire EORTC QLQ-C30 | Assessment of patients reported quality of life, as measured by EORTC QLC30 | Assessed from inclusion until 2 years after end of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Objective tumor response rate (ORR) | Defined as the proportion of patients with an objective tumor response (either partial response [PR] or complete response [CR] using RECIST v1.1) response (DR), time to next treatment and overall survival (OS) | Assed through study completion, an average of 1 year | 
| Duration of response (DR) | Duration of response among patients with an objective response, according to RECIST v1.1 | Assed through study completion, an average of 1 year | 
| Time to next treatment (TTNT) | Time from inclusion to institution og next therapy | Assed through study completion, an average of 1 year | 
| Overall survival (OS) | Time from date of inclusion until the date of death from any cause | Assessed up to 2 years after end of inclusion | 
| Fatigue | Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ) | From inclusion until 2 years after end of treatment | 
| Depression | Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9) | From inclusion until 2 years after end of treatment | 
| Pain intensity | Assessment of patient reported outcomes, as measured by an 11 point Numerical Rating Scale (NRS) for pain intensity | From inclusion until 2 years after end of treatment | 
| Adverse event | Patients files and self-report. Classified according to CTCAE v 5.0 and MedDRA | From inclusion until 2 years after end of treatment | 
| Quality adjusted life years (QALYs) | Patient self reported EQ-5D | From inclusion until 2 years after end of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Off Label Use