Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Cancer. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Norway
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Primary outcome measures
- Progression free survival (PFS). [Time frame: Assessed up to 2 years after end of inclusion]
- Patients questionnaire EORTC QLQ-C30 [Time frame: Assessed from inclusion until 2 years after end of treatment]
Secondary outcome measures (9)
- Objective tumor response rate (ORR) [Time frame: Assed through study completion, an average of 1 year]
- Duration of response (DR) [Time frame: Assed through study completion, an average of 1 year]
- Time to next treatment (TTNT) [Time frame: Assed through study completion, an average of 1 year]
- Overall survival (OS) [Time frame: Assessed up to 2 years after end of inclusion]
- Fatigue [Time frame: From inclusion until 2 years after end of treatment]
- Depression [Time frame: From inclusion until 2 years after end of treatment]
- Pain intensity [Time frame: From inclusion until 2 years after end of treatment]
- Adverse event [Time frame: From inclusion until 2 years after end of treatment]
- Quality adjusted life years (QALYs) [Time frame: From inclusion until 2 years after end of treatment]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Norway · 1 center
- Oslo University Hospital — Oslo
Identifiers
NCT: NCT04457713 · Off Label Study protocol