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

NCT04457713 | Recruiting

• 1 other identifier: Off Label Study protocol
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

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.

Conditions

Cancer

Keywords

Off Label Use

Outcome Measures

Primary Outcomes (2)

• 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

  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

  Assessed from inclusion until 2 years after end of treatment

Secondary Outcomes (9)

• Objective tumor response rate (ORR)

  Assed through study completion, an average of 1 year

• Duration of response (DR)

  Assed through study completion, an average of 1 year

• Time to next treatment (TTNT)

  Assed through study completion, an average of 1 year

• Overall survival (OS)

  Assessed up to 2 years after end of inclusion

• Fatigue

  From inclusion until 2 years after end of treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• None

Sponsors & Collaborators

• Oslo University Hospital: lead

Study Sites (1)

Oslo University Hospital

Oslo, 0379, Norway

RECRUITING

MeSH Terms

Conditions

Neoplasms

Study Officials

• Knut Smeland, PhD/MD

  Oslo University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Knut Smeland, PhD/MD

CONTACT

+4722934000; knusme@ous-hf.no

Tormod Guren, PhD/MD

CONTACT

+4722934000; uxtour@ous-hf.no

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Attending Physician

Study Record Dates

• First Submitted: June 25, 2020
• First Posted: July 7, 2020
• Study Start: June 1, 2020
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: August 15, 2025

Record last verified: 2025-08

Locations

NO (1)