艾昆纬-2025年患者W.A.I.T.指标_欧洲创新药物的可及性（英）-2025_69页_1mb

EFPIA Patients W.A.I.T. Indicator 2024 Survey Summary

Core Content and Purpose

The Patients W.A.I.T. (Waiting to Access Innovative Therapies) Indicator 2024 is a comprehensive report that assesses the availability, restrictions to availability, and time to availability of innovative medicines across 36 European healthcare systems. The study focuses on 173 centrally-approved medicines approved between 2020 and 2023, with data accurate as of January 5th, 2025.

The report aims to provide stakeholders with insights into the access landscape in Europe, highlighting the challenges and variations in patient access to novel therapies. It includes both core metrics and additional context to reflect the evolving access environment.

Main Points and Key Insights

Availability Metrics

• Total availability by approval year: The number of medicines available in European countries by the year they received marketing authorization.
• Rate of availability: The percentage of medicines available in the EU as of January 5th, 2025. The EU average is 46%, slightly up from 43% in 2023.
• Breakdown of availability: Shows the proportion of medicines with full availability, limited availability, or available only via individual patient schemes.
• Breakdown of total availability: Provides a detailed view of the availability status across all countries, categorized by overall availability and full availability.

Time to Availability Metrics

• Time from central approval to availability: The days between the central EU approval and the date the medicine becomes available to patients. The EU average is 578 days, up from 531 days in 2023.
• Time to availability: The days between marketing authorization and availability. The EU average is 578 days, with significant variation between countries.
• Median time to availability: A simpler metric for international comparison. The EU average is 518 days, with notable differences across countries.

Key Observations

• Access disparity: There remains a significant disparity in access, with access disparity over 80% between the highest and lowest performing countries.
• Oncology products: Show a slight deterioration in availability compared to the previous year, with 50% available on average (down from 52% in 2023).
• Orphan medicines: Continue to face greater challenges in access, with an average availability rate of 42% (down from 35% in 2023).
• Non-oncology orphan medicines: Average availability is 39%, also down from 32% in 2023.
• Combination therapies: Show a slightly better availability rate (55%) compared to the previous year (54%).

Notable Trends and Updates

• Evolution of access landscape: Full availability is less common, with limited availability and individual patient access routes becoming more prevalent.
• Methodology updates: The report now includes greater granularity on access restrictions and provides additional context for understanding the broader access landscape.
• Country-specific nuances: Local pharmaceutical industry associations provide the data, and their definitions are detailed in the appendix for transparency.

Limitations and Considerations

• Data completeness: Some countries did not complete a full dataset, which may affect the representativeness of the results.
• Price negotiation impact: In France, the inclusion of products under the Accès précoce system increases the average time to availability. If these products are considered as directly available, the average decreases to 570 days.
• Early access schemes: The UK's MHRA Early Access to Medicines Scheme provides access prior to marketing authorization but is not included in the analysis, potentially reducing the overall time for a small subset of medicines.
• EU27 average exclusions: Malta is excluded from the EU27 average due to limited data.

Summary of Metrics (EU27 Averages)

Conclusion

The W.A.I.T. 2024 report underscores the continued challenges in accessing innovative medicines across Europe. While the rate of availability has slightly improved, the time to availability has increased, highlighting the need for more efficient reimbursement processes. The report remains a valuable tool for stakeholders to understand and compare access dynamics across different countries and therapeutic areas.