Funding breakthrough therapies: A systematic review and recommendation

Area of Science:

• Health Economics
• Pharmaceutical Policy
• Innovative Therapies

Background:

• Advanced therapy medicinal products (ATMPs) represent innovative treatments with significant associated costs.
• Payers require strategic guidance to balance patient access to novel therapies with healthcare system financial viability.

Purpose of the Study:

• To identify, define, classify, and compare literature-based funding models for high-cost medicines.
• To assess the suitability of these models for ATMPs.
• To propose an optimal funding framework for ATMPs.

Main Methods:

• Systematic literature review of 48 articles on funding models for innovative, high-cost therapies.
• Classification of identified models into financial agreements, health outcomes-based agreements, and healthcoin.
• Analysis of model appropriateness for ATMP funding.

Main Results:

• Funding models were categorized into financial agreements (discounts, rebates, price caps, etc.), health outcomes-based agreements (performance-based payment, coverage with evidence development), and healthcoin.
• Each category presents distinct mechanisms for managing the cost of innovative medicines.

Conclusions:

• The development pipeline for ATMPs necessitates proactive consideration of novel funding pathways.
• An "ATMP-specific fund" is proposed as a viable strategy to ensure timely patient access to innovation while safeguarding healthcare system sustainability.