Clinical trial · Observational
Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Cancer | Malignant Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (2)
- measure
- Progression free survival (PFS).
- timeFrame
- Assessed up to 2 years after end of inclusion
- description
- 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
- measure
- Patients questionnaire EORTC QLQ-C30
- timeFrame
- Assessed from inclusion until 2 years after end of treatment
- description
- Assessment of patients reported quality of life, as measured by EORTC QLC30
Secondary outcomes (9)
- measure
- Objective tumor response rate (ORR)
- timeFrame
- Assed through study completion, an average of 1 year
- description
- 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)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available