英文_EFPIA_患者W.A.I.T.指标2025_69页_1mb

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

Core Content and Purpose

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

The W.A.I.T. Indicator is designed to compare European access to innovative therapies by measuring availability, restrictions to availability, and time to availability. It includes both public and private access routes, with particular emphasis on individual patient schemes and formulary restrictions. The report is updated annually to reflect evolving access landscapes and includes new indicators for greater granularity.

Main Viewpoints and Key Information

1. Availability Metrics

• EU average rate of full availability remained at 46% (up from 43% in 2023), but the increase may be due to a smaller cohort of orphan products.
• EU average rate of limited availability was 17%, a significant proportion of all products.
• EU average time to availability increased to 578 days, over 1 month longer than the 2023 study (531 days).
• Access disparity remains high, with more than 80% difference between the highest and lowest performing countries.

2. Oncology Medicines

• EU average rate of availability for oncology products was 50%, down from 52% in 2023.
• EU average time to availability for oncology products was 586 days, up by 33 days from last year and 66 days from the 2022 cohort.
• Four countries (Germany, Switzerland, Austria, and Italy) had a rate of availability above 80%, significantly higher than the rest.
• The time to availability for oncology products ranged from 4 to 34 months, with some countries experiencing delays of over 2.5 years.

3. Orphan Medicines

• EU average rate of availability for orphan medicines was 42%, lower than non-orphan products.
• EU average time to availability for orphan medicines was 611 days, longer than non-orphan products.
• The trend of limited availability has become more prominent since the 2019 study, with 35% of orphan products available in 2023.

4. Non-Oncology Orphan Medicines

• EU average rate of availability was 39%, down from 32% in 2023.
• EU average time to availability was 607 days, slightly higher than the 2023 study (530 days).

5. Combination Therapies

• EU average rate of availability was 55%, down from 54% in 2023.
• EU average time to availability was 553 days, up from 433 days in 2023.

6. Methodology and Limitations

• The study is based on data provided by local pharmaceutical industry associations and includes definitions in the appendix for transparency.
• Availability is defined as the inclusion of a centrally-approved medicine on public reimbursement lists.
• Time to availability measures the period between marketing authorisation and reimbursement.
• The report does not capture all nuances of patient access, and country-specific definitions should be consulted for accurate interpretation.
• Malta is excluded from EU27 averages due to limited data.

Additional Context and Updates

• The 2024 report retains core availability metrics but adds context around access landscape, including restrictions and individual patient schemes.
• The cohort composition has changed, with orphan products being a smaller proportion, affecting results.
• Historic comparisons and extended periods are included to highlight trends and changes over time.
• Box and whisker plots are used for time to availability, though they can be complex to interpret.
• Data not available is noted for certain countries, and the report is supplemented by other studies for a more complete view.

Summary of Key Observations

Key Insights

Rate of Availability

• Europe's rate of availability for all products increased slightly to 46%, but the trend for oncology products showed a deterioration.
• Orphan medicines continue to face significant access challenges, with a lower rate of availability compared to non-orphan products.

Time to Availability

• The average time to availability for oncology products has continued to rise, with a 33-day increase from the previous year.
• The time to availability for oncology products varied significantly across countries, with delays reaching up to 34 months.
• The median time to availability for oncology products was 540 days, with some countries still experiencing long delays.

Overall Trends

• Access disparity remains a critical issue, with a significant difference in availability and time to availability between countries.
• The report highlights the importance of local regulatory procedures in affecting access times.
• Alternative access routes (e.g., individual patient schemes, managed entry agreements) have become more common, reducing the relevance of full availability as the primary access metric.