E Hanna1, M Toumi2, C Dussart3, B Borissov4, O Dabbous5, K Badora6, P Auquier7. 1. Aix Marseille University, Public Health Department, 27, bd Jean Moulin, 13385 Marseille Cedex 05, France. Electronic address: Eve_hanna@hotmail.com. 2. Aix Marseille University, Public Health Department, 27, bd Jean Moulin, 13385 Marseille Cedex 05, France. Electronic address: mondher.toumi@univ-amu.fr. 3. Université Claude Bernard, Laboratoire Parcours Santé Systémique, EA 4129, Faculté de Médecine Laennec, 7-11 rue Guillaume Paradin, 69372 Lyon Cedex 08, France. Electronic address: claude.dussart@univ-lyon1.fr. 4. Prescriptia Ltd., Sofia, Hristo Botev Blvd. № 28, Bulgaria. Electronic address: Borislav.Borissov@prescriptia.com. 5. AveXis, inc, 2275 Half Day Road, Suite 200, Bannockburn, IL 60015, USA. Electronic address: omar.dabbous@gmail.com. 6. Creativ-ceutical, ul. Przemysłowa 12, 30-701 Krakow, Poland. Electronic address: kba@creativ-ceutical.com. 7. Aix Marseille University, Public Health Department, 27, bd Jean Moulin, 13385 Marseille Cedex 05, France. Electronic address: pascal.auquier@univ-amu.fr.
Abstract
BACKGROUND: Advanced therapy medicinal products (ATMPs) are innovative therapies likely associated with high prices. Payers need guidance to create a balance between ensuring patient access to breakthrough therapies and maintaining the financial sustainability of the healthcare system. OBJECTIVE: The aims of this study were to identify, define, classify and compare the approaches to funding high-cost medicines proposed in the literature, to analyze their appropriateness for ATMP funding and to suggest an optimal funding model for ATMPs. RESULTS: Forty-eight articles suggesting new funding models for innovative high-cost therapies were identified. The models were classified into 3 groups: financial agreement, health outcomes-based agreement and healthcoin. Financial agreement encompassed: discounts, rebates, price and volume caps, price-volume agreements, loans, cost-plus price, intellectual-based payment and fund-based payment. Health outcomes-based agreements were defined as agreements between manufacturers and payers based on drug performance, and were divided into performance-based payment and coverage with evidence development. Healthcoin described a new suggested tradeable currency used to assign monetary value to incremental outcomes. CONCLUSION: With a large number of ATMPs in development, it is time for stakeholders to start thinking about new pathways and funding strategies for these innovative high-cost therapies. An "ATMP-specific fund" may constitute a reasonable solution to ensure rapid patient access to innovation without threatening the sustainability of the health care system.
BACKGROUND: Advanced therapy medicinal products (ATMPs) are innovative therapies likely associated with high prices. Payers need guidance to create a balance between ensuring patient access to breakthrough therapies and maintaining the financial sustainability of the healthcare system. OBJECTIVE: The aims of this study were to identify, define, classify and compare the approaches to funding high-cost medicines proposed in the literature, to analyze their appropriateness for ATMP funding and to suggest an optimal funding model for ATMPs. RESULTS: Forty-eight articles suggesting new funding models for innovative high-cost therapies were identified. The models were classified into 3 groups: financial agreement, health outcomes-based agreement and healthcoin. Financial agreement encompassed: discounts, rebates, price and volume caps, price-volume agreements, loans, cost-plus price, intellectual-based payment and fund-based payment. Health outcomes-based agreements were defined as agreements between manufacturers and payers based on drug performance, and were divided into performance-based payment and coverage with evidence development. Healthcoin described a new suggested tradeable currency used to assign monetary value to incremental outcomes. CONCLUSION: With a large number of ATMPs in development, it is time for stakeholders to start thinking about new pathways and funding strategies for these innovative high-cost therapies. An "ATMP-specific fund" may constitute a reasonable solution to ensure rapid patient access to innovation without threatening the sustainability of the health care system.
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