Characteristics and survival of patients with cancer with intended off-label use-a cohort study.

Schmitt, Andreas Michael; Walter, Martin; Herbrand, Amanda Katherina; Jörger, Markus; Moffa, Giusi; Novak, Urban; Hemkens, Lars; Kasenda, Benjamin (2022). Characteristics and survival of patients with cancer with intended off-label use-a cohort study. BMJ open, 12(5), e060453. BMJ Publishing Group 10.1136/bmjopen-2021-060453

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OBJECTIVE

To describe the characteristics and the survival of patients with cancer with intended off-label use (OLU) cancer treatment and reimbursement request.

DESIGN

Cohort study using medical record data.

SETTING

Three major cancer centres in Switzerland.

PARTICIPANTS

519 patients with cancer and a reimbursement request for OLU between January 2015 and July 2018.

MAIN OUTCOMES

Characteristics of patients with cancer with and without access to intended OLU. Characteristics included the Glasgow prognostic score (GPS) which includes C reactive protein and albumin and discriminates prognostic groups.

RESULTS

OLU was intended for 519 (17%) of 3046 patients with cancer, as first-line treatment in 51% (n=264) and second-line in 31% (n=162). Of the 519 patients, 63% (n=328) were male, 63% (n=329) had solid cancer and 21% (n=111) had a haematological malignancy. Their median overall survival was 23.6 months (95% CI: 19.0 to 32.5). Access to OLU had 389 (75%) patients who were compared with patients without access on average 4.9 years younger (mean; 95% CI: 1.9 to 7.9 years), had a better overall prognosis according to the GPS (51% with GPS of 0 vs 39%; OR: 1.62 (95% CI: 1.01 to 2.59)), had less frequently solid cancer (62% vs 71%; OR: 0.66 (95% CI: 0.41 to 1.05)) and advanced stage cancer (53% vs 70%; OR: 0.48 (95% CI: 0.30 to 0.75)), were more frequently treatment-naive (53% vs 43%; OR: 1.55 (95% CI 1.01 to 2.39)) and were more frequently in an adjuvant/neoadjuvant treatment setting (14% vs 5%; OR: 3.39 (95% CI: 1.45 to 9.93)). Patients with access to OLU had a median OS of 31.1 months versus 8.7 months for patients without access (unadjusted HR: 0.54; (95% CI: 0.41 to 0.70)).

CONCLUSION

Contrary to the common assumption, OLU in oncology is typically not primarily intended for patients with exhausted treatment options. Patient characteristics largely differ between patients with and without access to intended OLU. More systematic evaluations of the benefits and harms of OLU in cancer care and the regulation of its access is warranted.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Medical Oncology

UniBE Contributor:

Novak, Urban

Subjects:

600 Technology > 610 Medicine & health

ISSN:

2044-6055

Publisher:

BMJ Publishing Group

Language:

English

Submitter:

Pubmed Import

Date Deposited:

27 May 2022 08:25

Last Modified:

05 Dec 2022 16:20

Publisher DOI:

10.1136/bmjopen-2021-060453

PubMed ID:

35613810

Uncontrolled Keywords:

epidemiology haematology health policy oncology

BORIS DOI:

10.48350/170268

URI:

https://boris.unibe.ch/id/eprint/170268

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