Clinical trial NCT04457713: Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
Official title: Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cancer
Keywords: Off Label Use
Lead sponsor: Oslo University Hospital
Sponsor class: OTHER
First posted: 2020-07-07
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Any patient accepted by the leadership in the Department of Oncology at OUH to receive anti-cancer treatment off-label from the date of start of the study is eligible for inclusion. Of note, this does not include off-label use of drugs used for supportive care.

Brief summary: 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.


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

Primary outcomes:
measure: Progression free survival (PFS).
timeFrame: Assessed up to 2 years after end of inclusion
measure: Patients questionnaire EORTC QLQ-C30
timeFrame: Assessed from inclusion until 2 years after end of treatment
Secondary outcomes:
measure: Objective tumor response rate (ORR)
timeFrame: Assed through study completion, an average of 1 year
measure: Duration of response (DR)
timeFrame: Assed through study completion, an average of 1 year
measure: Time to next treatment (TTNT)
timeFrame: Assed through study completion, an average of 1 year
measure: Overall survival (OS)
timeFrame: Assessed up to 2 years after end of inclusion
measure: Fatigue
timeFrame: From inclusion until 2 years after end of treatment
measure: Depression
timeFrame: From inclusion until 2 years after end of treatment
measure: Pain intensity
timeFrame: From inclusion until 2 years after end of treatment
measure: Adverse event
timeFrame: From inclusion until 2 years after end of treatment
measure: Quality adjusted life years (QALYs)
timeFrame: From inclusion until 2 years after end of treatment
Other outcomes:
Locations:
city: Oslo
country: Norway
facility: Oslo University Hospital
Study officials:
affiliation: Oslo University Hospital
name: Knut Smeland, PhD/MD
role: PRINCIPAL_INVESTIGATOR
References: