At the end of 2021 we predicted what 2022 may behold for the pricing and reimbursement landscape of Gene therapies in Europe. We anticipated a shift in payment models, a more collaborative approach to assessments, growing use of real-world evidence and how this can be implemented in post-authorisation follow ups to aid valuation of benefits. As another year draws to a close, we reflect on the 2022 trends and establish what changes have been made, if any, to the gene therapy pricing and reimbursement landscape in Europe.
2022 has seen positive progress to support access to ATMPs through multi-stakeholder collaboration
| 2022 Trend | Progress |
| Collaborative action on gene therapy assessment procedures and requirements | Full |
| A shift to more annuity-based models for gene therapies, as seen in Germany | Partial |
| Refinement and evaluation of real-world evidence requirements | Partial |
| Incorporating demonstration of clinical value in post-authorisation follow ups in conditional MA and reimbursement mechanisms | Full |
Foundations have been laid in 2022 that provide stepping stones to be developed in upcoming years
The European Medicines Agency (EMA) launched a pilot project in September, to provide enhanced regulatory support for five selected advanced therapy medicinal products (ATMPs) developed by academic and non-profit organisations. The aim of the pilot is to assess what further support or regulatory tools are required to increase access for patients to cell and gene therapies. Initial results of this pilot are expected to be available in three to four years1. This is an example of collaborative action taking place between regulatory bodies and developers to optimise assessment procedures and requirements and increase likelihood of ATMPs meeting regulatory requirements. A shift in approach to pricing models has also seen the collaborative sharing of risk, and benefits between manufacturers and payers. Utilising annuity-based models for gene therapies rather than volume-based agreements has been perceived well as a method to alleviate the burden of the high cost and mitigate clinical uncertainties associated with ATMPs limited clinical data package. In September the French government as part of the Social Security Financing Bill (PLFSS), proposed the use of innovative financing models.
The experience of recent launches in the European landscape demonstrate the potential success of novel outcomes-based reimbursement mechanisms. Affordability of ATMPs is a major challenge, so continuing to trial innovative payment models will help to ensure patient access to promising novel therapies.
A parameter of many novel reimbursement models is payments based on real-life results. The collection of further data can help justify large upfront payments. Real-world data is becoming of increasing importance for decision-making at both regulatory and HTA level. The DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative exemplifies this, and is expected to deliver over 150 studies by 2025 to support HTA decision-making, it is hoped it can also be a valuable tool to provide post-approval data. This would be useful given managed entry agreements for cell and gene therapies are incorporating generation of further long-term data as a condition. In April, NICE’s appraisal of elosulfase alfa, for treating mucopolysaccharides type 4A (MPS 4A), new evidence collected as part of its managed access agreement was considered in NICE’s reassessment and subsequent recommendation of the therapy2.
However, as digital databases scale up, data collected must be specific to the question being asked and real-world evidence requirements refined. Project HERCULES is an example of a multinational collaboration between multiple stakeholders to establish what is of clinical value and how it can be assessed, in this case with relevance to Duchenne Muscular Dystrophy (DMD). 2022 has seen a rise in patient-led disease models that can incorporate meaningful outcomes to patients to better inform decision-making.
Conclusion
Multi-stakeholder collaboration must continue for Europe to maintain its position as a leader in gene therapies, fostering innovation and enhancing patient access to these novel treatments. 2022 has demonstrated the positive outcomes that collaboration can achieve: databases, access frameworks and patient-led disease models. In 2025, ATMPs will be among the first selected therapeutic to undergo joint clinical assessment (JCA). A joint effort must continue, the JCA for ATMPs in 2025 has great potential to utilise the learnings of the next few years in terms of refining real-word evidence requirements and usage and use of clinical data in post-launch follow-ups.
Foundations have been laid in 2022 that provide stepping stones to be developed in upcoming years
The European Medicines Agency (EMA) launched a pilot project in September, to provide enhanced regulatory support for five selected advanced therapy medicinal products (ATMPs) developed by academic and non-profit organisations. The aim of the pilot is to assess what further support or regulatory tools are required to increase access for patients to cell and gene therapies. Initial results of this pilot are expected to be available in three to four years1. This is an example of collaborative action taking place between regulatory bodies and developers to optimise assessment procedures and requirements and increase likelihood of ATMPs meeting regulatory requirements. A shift in approach to pricing models has also seen the collaborative sharing of risk, and benefits between manufacturers and payers. Utilising annuity-based models for gene therapies rather than volume-based agreements has been perceived well as a method to alleviate the burden of the high cost and mitigate clinical uncertainties associated with ATMPs limited clinical data package. In September the French government as part of the Social Security Financing Bill (PLFSS), proposed the use of innovative financing models.
The experience of recent launches in the European landscape demonstrate the potential success of novel outcomes-based reimbursement mechanisms. Affordability of ATMPs is a major challenge, so continuing to trial innovative payment models will help to ensure patient access to promising novel therapies.
A parameter of many novel reimbursement models is payments based on real-life results. The collection of further data can help justify large upfront payments. Real-world data is becoming of increasing importance for decision-making at both regulatory and HTA level. The DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative exemplifies this, and is expected to deliver over 150 studies by 2025 to support HTA decision-making, it is hoped it can also be a valuable tool to provide post-approval data. This would be useful given managed entry agreements for cell and gene therapies are incorporating generation of further long-term data as a condition. In April, NICE’s appraisal of elosulfase alfa, for treating mucopolysaccharides type 4A (MPS 4A), new evidence collected as part of its managed access agreement was considered in NICE’s reassessment and subsequent recommendation of the therapy2.
However, as digital databases scale up, data collected must be specific to the question being asked and real-world evidence requirements refined. Project HERCULES is an example of a multinational collaboration between multiple stakeholders to establish what is of clinical value and how it can be assessed, in this case with relevance to Duchenne Muscular Dystrophy (DMD). 2022 has seen a rise in patient-led disease models that can incorporate meaningful outcomes to patients to better inform decision-making.
Conclusion
Multi-stakeholder collaboration must continue for Europe to maintain its position as a leader in gene therapies, fostering innovation and enhancing patient access to these novel treatments. 2022 has demonstrated the positive outcomes that collaboration can achieve: databases, access frameworks and patient-led disease models. In 2025, ATMPs will be among the first selected therapeutic to undergo joint clinical assessment (JCA). A joint effort must continue, the JCA for ATMPs in 2025 has great potential to utilise the learnings of the next few years in terms of refining real-word evidence requirements and usage and use of clinical data in post-launch follow-ups.
Sources:
- Advanced therapy medicinal products: overview. ATMP pilot for academia and non-profit organisations. EMA. https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview Accessed: 15/11/22
- Elosulfase alfa for treating mucopolysaccharidosis type 4a. Highly specialised technologies guidance. NICE. https://www.nice.org.uk/guidance/hst19/chapter/1-Recommendations Accessed: 15/11/22