The Alliance for Regenerative Medicine to host European ATMP Access Meeting in Brussels

Meeting on 12^th December brings together stakeholders from across Europe

WASHINGTON, D.C. and BRUSSELS, BELGIUM – December 6, 2019

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, is hosting a multi-stakeholder meeting in Brussels on 12 December, focused on European market access to Advanced Therapy Medicinial Products (ATMPs). The event will be webcast live, with full details available here.

Representatives from patient associations, industry, alongside government and regulatory agencies, including the EMA, the European Commission and payers from Germany, the UK and Italy, will share insights through panels and presentations at the event, entitled Getting ready: Act now for access to ATMPs in Europe.

Speakers will discuss their current experience with access to ATMPs in Europe as well as exploring topics such as evidence requirements, registries and innovative payment schemes. In addition, ARM’s CEO Janet Lambert will give an overview of the ATMP landscape in Europe and ARM’s recently published consensus report on the subject. The full schedule of the day is below.

ARM’s European consensus report brings together together the views of European policymakers and experts, developers and manufacturers of ATMPs, patient organizations, and other stakeholder groups. It acts as a comprehensive review of the regulatory and market access framework across major European countries and identifies potential hurdles to adoption. While regulatory frameworks have been implemented to ensure appropriate, rapid approval of ATMPs, the report highlights that 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 full list of recommendations can be found below.

Janet Lambert, CEO of ARM, commented: “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, this new approach to treatment has created challenges on how to most effectively get these therapies to patients in a sustainable manner. At ARM, we are committed to the advancement of these life changing therapies and we therefore produced our recommendations, alongside key stakeholders, to help accelerate this in a European consensus report. Organizing a European stakeholder meeting was the natural next step and our hope is that the discussions held on 12th December will facilitate helpful pan-European dialogue and improved access to ATMPs for patients.”

Journalists interested in attending in person should contact arm@consilium-comms.com for further details.

Brussels Event Schedule

Recommendations from European ATMP consensus report

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.

1. 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.

1. 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:

1. Pay-for-performance
2. Annuity payments
3. 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.

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 350+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centers, 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.