Contrasting evidence to reimbursement reality for off-label use (OLU) of drug treatments in cancer care: rationale and design of the CEIT-OLU project.
Antineoplastic Agents
/ economics
Evidence-Based Medicine
/ economics
Female
Health Services Accessibility
/ economics
Humans
Male
Medical Oncology
/ economics
Multicenter Studies as Topic
Neoplasms
/ drug therapy
Observational Studies as Topic
Off-Label Use
/ economics
Progression-Free Survival
Reimbursement Mechanisms
/ economics
Research Design
Switzerland
/ epidemiology
cancer therapy
evidence-based healthcare
off-label use
reimbursement
Journal
ESMO open
ISSN: 2059-7029
Titre abrégé: ESMO Open
Pays: England
ID NLM: 101690685
Informations de publication
Date de publication:
2019
2019
Historique:
received:
16
09
2019
revised:
21
10
2019
accepted:
24
10
2019
entrez:
6
12
2019
pubmed:
6
12
2019
medline:
17
6
2020
Statut:
epublish
Résumé
Off-label use (OLU) of a drug reflects a perceived unmet medical need, which is common in oncology. Cancer drugs are often highly expensive and their reimbursement is a challenge for many healthcare systems. OLU is frequently regulated by reimbursement restrictions. For evidence-based healthcare, treatment ought to be reimbursed if there is sufficient clinical evidence for treatment benefit independently of patient factors not related to the treatment indication. However, little is known about the reality of OLU reimbursement and its association with the underlying clinical evidence. Here, we aim to investigate the relationship of reimbursement decisions with the underlying clinical evidence. We will extract patient characteristics and details on treatment and reimbursement of cancer drugs from over 3000 patients treated in three Swiss hospitals. We will systematically search for clinical trial evidence on benefits associated with OLU in the most common indications. We will describe the prevalence of OLU in Switzerland and its reimbursement in cancer care, and use multivariable logistic regression techniques to investigate the association of approval/rejection of a reimbursement requests to the evidence on treatment effects and to further factors, including type of drug, molecular predictive markers and the health insurer. Our study will provide a systematic overview and assessment of OLU and its reimbursement reality in Switzerland. We may provide a better understanding of the access to cancer care that is regulated by health insurers and we hope to identify factors that determine the level of evidence-based cancer care in a highly diverse western healthcare system.
Sections du résumé
Background
Off-label use (OLU) of a drug reflects a perceived unmet medical need, which is common in oncology. Cancer drugs are often highly expensive and their reimbursement is a challenge for many healthcare systems. OLU is frequently regulated by reimbursement restrictions. For evidence-based healthcare, treatment ought to be reimbursed if there is sufficient clinical evidence for treatment benefit independently of patient factors not related to the treatment indication. However, little is known about the reality of OLU reimbursement and its association with the underlying clinical evidence. Here, we aim to investigate the relationship of reimbursement decisions with the underlying clinical evidence.
Methods/ design
We will extract patient characteristics and details on treatment and reimbursement of cancer drugs from over 3000 patients treated in three Swiss hospitals. We will systematically search for clinical trial evidence on benefits associated with OLU in the most common indications. We will describe the prevalence of OLU in Switzerland and its reimbursement in cancer care, and use multivariable logistic regression techniques to investigate the association of approval/rejection of a reimbursement requests to the evidence on treatment effects and to further factors, including type of drug, molecular predictive markers and the health insurer.
Discussion
Our study will provide a systematic overview and assessment of OLU and its reimbursement reality in Switzerland. We may provide a better understanding of the access to cancer care that is regulated by health insurers and we hope to identify factors that determine the level of evidence-based cancer care in a highly diverse western healthcare system.
Identifiants
pubmed: 31803503
doi: 10.1136/esmoopen-2019-000596
pii: S2059-7029(20)30095-8
pmc: PMC6890379
doi:
Substances chimiques
Antineoplastic Agents
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Pagination
e000596Informations de copyright
© Author (s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. Published by BMJ on behalf of the European Society for Medical Oncology.
Déclaration de conflit d'intérêts
Competing interests: BK declares consultant activities for Roche and Siemens, research grants from Roche/AbbVie, travel support from Riemser, AbbVie and Amgen. UN declares consultation or advisory role for Roche, Astra, Gilead and Celgene, and honoraria (congress participations) from Amgen, Novartis, Takeda and Roche.
Références
Lancet. 2000 Mar 25;355(9209):1041-7
pubmed: 10744089
N Engl J Med. 2004 Jun 3;350(23):2335-42
pubmed: 15175435
J Clin Pharm Ther. 2017 Jun;42(3):251-258
pubmed: 28164359
Eur J Clin Pharmacol. 2014 Jun;70(6):719-25
pubmed: 24609468
Stat Med. 2008 Feb 28;27(5):625-50
pubmed: 17590884
N Engl J Med. 2008 Apr 3;358(14):1427-9
pubmed: 18385495
ACP J Club. 1995 Nov-Dec;123(3):A12-3
pubmed: 7582737
Syst Rev. 2017 Nov 28;6(1):234
pubmed: 29179733
BMJ. 2009 Jun 29;338:b2393
pubmed: 19564179
BMJ. 2009 Jul 21;339:b2535
pubmed: 19622551
Trials. 2018 Sep 19;19(1):505
pubmed: 30231912
J Clin Epidemiol. 1995 Jan;48(1):45-57; discussion 59-60
pubmed: 7853047
Ann Oncol. 2013 Oct;24 Suppl 6:vi7-23
pubmed: 23970019
J Clin Oncol. 2007 Aug 10;25(23):3456-61
pubmed: 17687149
Lancet Oncol. 2012 Dec;13(12):1225-33
pubmed: 23168362
Ann Oncol. 2014 Oct;25(10):1871-88
pubmed: 25234545