PhD Scientific Days 2026

Pharmaceutical Sciences and Health Technologies 3.

Bridging the gap between off-label practice and on-label authorization: Unmet medical need and the Avastin Case

Name of the presenter

Parapatics, Tamás

Institute/workplace of the presenter

Nemzeti Népegészségügyi és Gyógyszerészeti Központ / National Center for Public Health and Pharmacy

Authors

Tamás Parapatics Dr.¹
1: Nemzeti Népegészségügyi és Gyógyszerészeti Központ / National Center for Public Health and Pharmacy

Text of the abstract

Introduction
Off-label use of medicines is common in clinical practice, especially where no authorised therapeutic alternative adequately meets the needs of certain patient groups. In such cases the concept of unmet medical need may justify exceptional use. However, routine and long-term use of unapproved therapies raises legal, regulatory, and economic concerns, particularly when widely applied indications remain outside the authorised label. The Avastin (bevacizumab) case illustrates the tension between patient access, financing, regulatory certainty and market incentives.

Objectives
This research examines the need to convert off-label drug uses into on-label status through a combined assessment of regulatory incentives, market rationality, and marketing authorisation frameworks. The presentation specifically analyses the role of unmet medical need in off-label approvals and its economic relevance and justification, with particular attention to the Avastin case.

Methods
The research applies comparative legal analysis, supported by qualitative stakeholder interviews and quantitative data analysis. It reviews the legal framework governing off-label use and the modification or extension of marketing authorisations at national, EU, and selected third-country levels, with particular focus on Hungarian practice. It also considers the economic behaviour of marketing authorisation holders, including incentives and disincentives affecting applications for new indications. The Avastin case serves as a case study.

Results
Preliminary findings show that unmet medical need alone does not sufficiently motivate marketing authorisation holders to pursue formal expansion of indications. Where expected returns are low, legal and administrative pathways remain underused even when off-label use is a routine. The Avastin case suggests that cost-effectiveness and equitable access may support broader use, but without a tailored incentive system such practices remain legally uncertain and regulatorily fragmented.

Conclusion
A sustainable solution requires a targeted incentive system integrating patient interests, regulatory certainty, and market motivation. Such a framework could support the formal authorisation of routinely used off-label indications and improve both patient safety and access.

Funding
This research received no external funding.