ARM Press Release

The Alliance for Regenerative Medicine Publishes Recommendations for Timely Access to Advanced Therapy Medicinal Products in Europe

New approaches to assessment and payment needed to ensure patients can benefit from latest advancements in cell and gene therapies

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, today published a consensus report and key recommendations to ensure European patients have access to Advanced Therapy Medicinal Products (ATMPs), including potentially transformative cell and gene therapies.

The report, which brings together the views of a number of European policy makers and experts, developers and manufacturers of ATMPs, patient organizations, and other stakeholder groups, provides a comprehensive review of the regulatory and market access framework across major European countries and identifies potential hurdles to adoption. The report was funded by ARM.

ATMPs have the potential to offer a durable, life-changing therapeutic effect, possibly with a single administration, for patients who may have few or no alternative treatment options. However, the therapies’ complexity and relative novelty present challenges to ensuring these products reach those in need. While regulatory frameworks have been implemented to ensure appropriate, rapid approval of ATMPs, the report highlights payers and health technology assessment bodies have not yet established all the necessary specific mechanisms to capture the full benefits of these therapies, creating potential barriers to timely patient access post-approval.

The consensus report recommends a number of key measures. These include:

• A quick adoption of new payment models such as conditional reimbursement, pay-for-performance, and annuity-based payments
• ATMP-dedicated funds, allowing health systems to invest in ATMPs that offer the potential for long-term benefits
• Better-adapted health technology assessment (HTA) methods, including greater use of real-world evidence (RWE) and development of infrastructure required to collect and use high-quality RWE
• Expansion of opportunities for early dialogue between ATMP developers and payers, supported by increased EU funding
• Development of pan-European initiatives to ensure timely and effective access to cross-border healthcare for patients

Janet Lambert, CEO of ARM, commented: “Advanced therapy medicinal products have extraordinary potential to alleviate human suffering. Clinical studies indicate that many cell and gene therapies will provide a long-lasting, even curative effect for many patients with debilitating or fatal disorders. These treatments represent a growing share of the biopharma development pipeline, and an exciting wave of transformative therapies will be coming to market soon. As a society, it is imperative that we promote innovation in this area and tear down barriers to patient access.

“This report gathers together leading stakeholders in the gene and cell therapy sector, including prominent pharmaceutical and biotech companies, health technology assessment representatives, patient groups, and payers. Working collaboratively, we have outlined key steps to ensuring these life-changing therapies are widely available to patients in Europe.”

A copy of the full report can be accessed here.

Recommendations in full

Recommendation 1: Better adapt Health Technology Assessment (HTA) frameworks to ATMPs:

1. Enhancing acceptability of validated surrogate endpoints to estimate long-term outcomes
2. Conducting further research to improve methodology of indirect comparisons
3. Supporting development, validation and use of pan-European natural history datasets
4. Leveraging scientific, clinical, and HTA expertise from centers of excellence
5. Adopting changes in economic modelling such as improving methods for extrapolation

Recommendation 2: Favor wider application of conditional reimbursement schemes:

Conditional reimbursement schemes have the potential to mitigate uncertainty on duration of effect based on data available at time of regulatory approval. This approach is in use in several countries and a wider application in Europe for ATMPs is recommended.

Recommendation 3: Develop pan-European initiatives to create:

1. Real-World-Evidence infrastructure: Real-World Evidence (RWE) development is instrumental in addressing uncertainties on long-term effect, safety, health-related quality of life and use of healthcare resources. There is a need to develop RWE infrastructure at European level to support long-term evidence generation and procedures to enhance quality of evidence collected specifically for ATMPs.
2. New early-dialogue opportunities: There is a need for more opportunities for early-dialogue activities through additional EU and National funding. This would offer developers (and in particular SMEs) early insight on ways to address product specific uncertainties and how to mitigate them.
3. Timely and effective access to cross-border healthcare for all EU patients: Despite existing legislation to facilitate cross-border healthcare in Europe, there are still barriers limiting access to ATMPs as they are most often delivered through centers of excellence which are not always present in all countries, and within countries in all regions. In particular, there is urgent need for measures to coordinate and fund access to cross-border ATMP treatment at European level.

Recommendation 4: Favor wider application of innovative access and funding arrangements such as:

• Pay-for-performance
• Annuity payments
• Special funds for transformative treatments

New payment models are needed to ensure timely patient access to innovation while preserving sustainability of healthcare systems. Without the adoption of these new models, some transformative therapies may not reach patients in some or all European countries and some may be at risk of withdrawal from the market.

Press enquiries

For more information about the report or media requests, please contact Lyndsey Scull from the Alliance for Regenerative Medicine at lscull@alliancerm.org or Consilium Strategic Communications at arm@consilium-comms.com.

###

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. Founded in 2009, ARM works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its 330+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centres, investors, and patient groups that comprise the broader regenerative medicine community and is the prominent international advocacy organization in this field.

ARM has 70+ members across 15 countries in Europe. ARM aims to work closely with European stakeholders, leveraging its membership to create a supportive commercial and regulatory environment to create better conditions for the development and commercialization of ATMPs in Europe; develop strong stakeholder support around proposed solutions to improve patient access to ATMPs; promote clear, predictable and efficient regulatory framework across Europe; and promote international convergence of key regulations and guidances. For more information, visit alliancerm.org.