The Next Steps Matter? New Cancer Medicines and Their Indication Extensions in Europe 2010-2020
Author(s)
Anna-Maria Ruuskanen, MSc (Health Economics), Terhi Kurko, PhD, Kati Sarnola, Assoc. Prof. (Docent), Katariina Klintrup, M.D., PhD, Hanna Koskinen, PhD.
The Social Insurance Institution of Finland, Helsinki, Finland.
The Social Insurance Institution of Finland, Helsinki, Finland.
OBJECTIVES: Extensions of therapeutic indications are a common method to extend the lifecycle of medicines in the post-authorisation phase. The aim of this study was to explore the frequency and the level of evidence for extensions of indication in the European cancer medicine approvals, and compare three groups: initial indications for multi-indication medicines, extensions and medicines without extensions.
METHODS: A document analysis was conducted on all new outpatient cancer medicines approved in Europe between 2010 and 2020, including their indication extensions. Study design characteristics were compared using data from European public assessment reports, evaluated with the Joanna Briggs Institute critical appraisal tool, and clinical added value using assessments by the French Haute Autorité de Santé.
RESULTS: Of the 55 new cancer medicines identified, 31 had one or more extensions, while 24 had none. In total, 57 extensions were observed, most commonly involving a new line of treatment (35%) or a new cancer type (30%). On average, the first extension was granted 2 years and 7 months after the first marketing authorisation. Compared to the initial indications, the studies supporting the extensions generally demonstrated higher quality in terms of study design. Furthermore, the proportion of medicines offering clinical added value was greater among extensions than among initial indications and medicines without extensions.
CONCLUSIONS: Our analysis shows that extensions of indication are a prevalent and significant component in the lifecycle management of cancer medicines in Europe as for instance many extensions are meant for use in earlier treatment lines compared to initial indications. Moreover, the results suggest that the clinical value of these medicines tends to increase with the addition of new indications. Thus, the role of extensions should be given more attention in the evaluation of medicines.
METHODS: A document analysis was conducted on all new outpatient cancer medicines approved in Europe between 2010 and 2020, including their indication extensions. Study design characteristics were compared using data from European public assessment reports, evaluated with the Joanna Briggs Institute critical appraisal tool, and clinical added value using assessments by the French Haute Autorité de Santé.
RESULTS: Of the 55 new cancer medicines identified, 31 had one or more extensions, while 24 had none. In total, 57 extensions were observed, most commonly involving a new line of treatment (35%) or a new cancer type (30%). On average, the first extension was granted 2 years and 7 months after the first marketing authorisation. Compared to the initial indications, the studies supporting the extensions generally demonstrated higher quality in terms of study design. Furthermore, the proportion of medicines offering clinical added value was greater among extensions than among initial indications and medicines without extensions.
CONCLUSIONS: Our analysis shows that extensions of indication are a prevalent and significant component in the lifecycle management of cancer medicines in Europe as for instance many extensions are meant for use in earlier treatment lines compared to initial indications. Moreover, the results suggest that the clinical value of these medicines tends to increase with the addition of new indications. Thus, the role of extensions should be given more attention in the evaluation of medicines.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR211
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Approval & Labeling, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology