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

Oslo University Hospital200 enrolled

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.

Study Type

OBSERVATIONAL

Enrollment

200

Conditions

Cancer

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: Assessed from inclusion until 2 years after end of treatment

Secondary Outcomes

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)

Time frame: 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

Time frame: Assed through study completion, an average of 1 year

Time to next treatment (TTNT)

Time from inclusion to institution og next therapy

Time frame: 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

Time frame: Assessed up to 2 years after end of inclusion

Fatigue

Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)