Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for...

Area of Science:

• Health economics and outcomes research
• Pharmaceutical policy and regulation
• Market access strategies

Background:

• Performance-based managed entry agreements (PB-MEAs) offer potential patient access to novel therapeutics.
• Competent authorities for pricing and reimbursement (CAPRs) face practical challenges, leading to reluctance in implementing PB-MEAs.
• Existing regulatory postauthorization requirements may not fully align with PB-MEA data generation needs.

Purpose of the Study:

• To assess if aligning regulatory postauthorization requirements with PB-MEA data generation can improve feasibility.
• To explore the role of active collaboration and data sharing among CAPRs in enhancing PB-MEA implementation.
• To identify conditions under which PB-MEAs may effectively reduce uncertainty for pricing and reimbursement decisions.

Main Methods:

• Review of regulatory postauthorization requirements from the European Medicines Agency (EMA) for 15 recently authorized products.
• Structured assessments by seven CAPR reviewers on the helpfulness of regulatory data for PB-MEA implementation across key domains.
• Analysis of reviewer comments on PB-MEAs, inter-CAPR cooperation, and information sufficiency for PB-MEA development.

Main Results:

• Regulatory postauthorization requirements were deemed at least partly helpful for most products and domains, except for comparators.
• Publicly available EMA information was insufficient for PB-MEA implementation in one quarter of the assessments.
• While few PB-MEAs were in place, potential for implementation and cross-CAPR collaboration was viewed favorably.

Conclusions:

• PB-MEAs are not a primary choice for CAPRs due to implementation complexities.
• Conditions exist where PB-MEAs could be valuable for reducing uncertainty in drug access and reimbursement.
• Enhanced transparency in postauthorization studies and inter-agency collaboration are crucial for overcoming PB-MEA implementation barriers.