Assessing Pharmaceutical Innovation in Europe: Therapeutic Advances vs. Unmet Public Health Need
Author(s)
Paula Costa, PhD student1, Ana Margarida Advinha, Professor2, Sofia Oliveira Martins, Professor1.
1Faculty of Pharmacy, University of Lisbon, Lisbon, Portugal, 2School of Health and Human Development, University of Evora, Evora, Portugal.
1Faculty of Pharmacy, University of Lisbon, Lisbon, Portugal, 2School of Health and Human Development, University of Evora, Evora, Portugal.
OBJECTIVES: This study presents a comprehensive characterization of novel medicines authorized by the European Commission (EC) between 2014 and 2024, with a focus on evaluating their innovative potential and therapeutic relevance. Beyond descriptive profiling, the study assesses the potential of innovation introduced by these novel drugs and address the alignment between therapeutic indications and unmet medical needs, as determined by the clinical burden of disease, highlighting gaps and opportunities.
METHODS: A descriptive statistical analysis was conducted using publicly available data from EC and EMA databases and FDA annual reports. Medicines were categorized by molecule type, orphan status, therapeutic area, and innovation-related attributes (first-in-class, breakthrough and conditional approval). Disease burden was assessed using DALYs and mortality data from the GBD Compare tool (IHME), expressed as a percentage of total values for Europe (2021).
RESULTS: A total of 481 novel drugs were approved by the EC, with 33% having orphan status. The results reveal emerging trends, including a growing number of targeted therapies, increasingly complex molecular structures, and the rise of novel technologies. Although these medicines span various therapeutic areas, there has been a marked focus on oncology (28% of the cohort) and rare diseases (17%), with comparatively few approvals in areas of high public health need such as new antibiotics and dementia treatments. This pattern highlights a potential misalignment between societal health priorities and the industry's innovation pipeline. Furthermore, analysis of innovation proxies indicates that only a minority of novel medicines meet these criteria.
CONCLUSIONS: New medicines play a key role in advancing public health; however, gaps between innovation and health needs remain, particularly in areas with high disease burden and limited therapeutic options. Despite continuous flow of novel treatments, innovation does not inherently guarantee better outcomes and know the clinical value is paramount when financing choices must be made.
METHODS: A descriptive statistical analysis was conducted using publicly available data from EC and EMA databases and FDA annual reports. Medicines were categorized by molecule type, orphan status, therapeutic area, and innovation-related attributes (first-in-class, breakthrough and conditional approval). Disease burden was assessed using DALYs and mortality data from the GBD Compare tool (IHME), expressed as a percentage of total values for Europe (2021).
RESULTS: A total of 481 novel drugs were approved by the EC, with 33% having orphan status. The results reveal emerging trends, including a growing number of targeted therapies, increasingly complex molecular structures, and the rise of novel technologies. Although these medicines span various therapeutic areas, there has been a marked focus on oncology (28% of the cohort) and rare diseases (17%), with comparatively few approvals in areas of high public health need such as new antibiotics and dementia treatments. This pattern highlights a potential misalignment between societal health priorities and the industry's innovation pipeline. Furthermore, analysis of innovation proxies indicates that only a minority of novel medicines meet these criteria.
CONCLUSIONS: New medicines play a key role in advancing public health; however, gaps between innovation and health needs remain, particularly in areas with high disease burden and limited therapeutic options. Despite continuous flow of novel treatments, innovation does not inherently guarantee better outcomes and know the clinical value is paramount when financing choices must be made.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EPH20
Topic
Epidemiology & Public Health
Topic Subcategory
Public Health
Disease
No Additional Disease & Conditions/Specialized Treatment Areas