OBJECTIVES: While Gene Therapies (GTs) represent transformative therapies for patients, they require a complex manufacturing and delivery process. This, coupled with small target populations, is associated with high price expectations. However, benefits of GTs are frequently only realized in the long-term, posing affordability challenges for payers. To manage this, payers have entered into risk-sharing agreements and innovative payment models with GT developers. The aim of this study was to review managed entry agreements (MEAs) and innovative payment models that have been put in place for GTs in the G7 countries and Canada to identify trends.

METHODS: A review of approved GTs in 8 countries was performed via official government and payer websites to identify pricing and reimbursement decisions for the GTs identified. A grey literature search was also conducted to supplement the website data.

RESULTS: MEA experience with GTs varies significantly by country. Italy has been at the forefront of MEAs. Despite recent reluctance from AIFA, 62.5% of all GTs appraised had some form of MEA. To manage affordability, AIFA introduced the “payment at results” contract, to spread the cost of expensive therapies. Since 2019, Spain has also been at the forefront of MEAs, with 66.7% of all GTs appraised having an MEA, 100% performance-based. MEAs have also been established in Germany, France, US and Canada, but have been limited for GTs. No MEAs were established in Japan.

CONCLUSIONS: MEAs have been used in most countries to manage the cost of GTs. However, there are still logistical challenges in implementing MEAs consistently and successfully across key stakeholder groups. Considering the GT market is forecast to grow from $5.33Bn in 2022 to $19.88Bn by 2027, it is critical for government and payer institutions to tackle these challenges, heavily supported by GT developers, to ensure patients have timely access to transformative therapies.