Cancer
Conditions
Keywords
Off Label Use
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.
Interventions
None listed
Sponsors
Oslo University Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Progression free survival (PFS). | Assessed up to 2 years after end of inclusion | 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 |
| Patients questionnaire EORTC QLQ-C30 | Assessed from inclusion until 2 years after end of treatment | Assessment of patients reported quality of life, as measured by EORTC QLC30 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of response (DR) | Assed through study completion, an average of 1 year | Duration of response among patients with an objective response, according to RECIST v1.1 |
| Time to next treatment (TTNT) | Assed through study completion, an average of 1 year | Time from inclusion to institution og next therapy |
| Overall survival (OS) | Assessed up to 2 years after end of inclusion | Time from date of inclusion until the date of death from any cause |
| Pain intensity | From inclusion until 2 years after end of treatment | Assessment of patient reported outcomes, as measured by an 11 point Numerical Rating Scale (NRS) for pain intensity |
| Depression | From inclusion until 2 years after end of treatment | Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9) |
| Adverse event | From inclusion until 2 years after end of treatment | Patients files and self-report. Classified according to CTCAE v 5.0 and MedDRA |
| Quality adjusted life years (QALYs) | From inclusion until 2 years after end of treatment | Patient self reported EQ-5D |
| Fatigue | From inclusion until 2 years after end of treatment | Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ) |
| Objective tumor response rate (ORR) | Assed through study completion, an average of 1 year | 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) |
Countries
Norway
Contacts
Primary ContactKnut Smeland, PhD/MD
Backup ContactTormod Guren, PhD/MD
Outcome results
None listed