How to address the affordability challenge for advanced therapy medicinal products

The objective - the short-term affordability challenge

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, our current Belgian healthcare system is not attuned to these breakthrough therapies, which require short-term substantial payment for potentially lifelong patient value.

Multi-stakeholder round table meetings

Round table meetings brought together different stakeholders (academics, healthcare professionals, insurance and health technology specialists, patients and patient associations, authorities and other stakeholders) to explore innovative solutions to ensure short-term affordability, long-term sustainability and patient access. A total of four round tables were organised during 2018 and 2019. The project was coordinated and managed by Inovigate and the Healthcare Management Centre of Vlerick Business School in collaboration with RIZIV/INAMI and the Cabinet of Minister of Health and Social Affairs.

Policy report summarising input from multi-stakeholder dialogue and recommendations

The 4 round table meetings led to a policy report summarising the views of the different stakeholder groups involved and the consensus reached. This policy report provides an overview of the current challenges and proposals for future funding solutions for ATMPs and more specifically gene-therapies in Belgium. It also identifies hurdles to adoption and implementation and makes policy recommendations to address those challenges. 

The consensus report recommends a number of key measures including:

Recommendation 1:  Leverage international horizon scanning project and facilitate early dialogue

Continuing the constructive multi-stakeholder dialogue with all involved stakeholders in Belgium

• Leveraging international horizon scanning to facilitate early dialogue between authorities and innovators

This would offer developers early insight on ways to address product specific uncertainties and mitigation of them.

Recommendation 2: Favour application of new funding arrangements to new gene-therapies

New payment models are needed to ensure timely patient access to innovation while preserving sustainability of healthcare system. Without the adoption of these new models, some transformative therapies may not reach patients. An optimal solution, that meets the critical success factors and addresses the short-term budgetary challenge and uncertain long-term benefits, should be based on:

• spread payments (e.g. annuity-based)
• outcomes-based Managed Entry Agreements (MEA)
• more dynamic transversal budget reallocation model (gain sharing)

Recommendation 3: Develop initiatives to create adoption of new funding arrangements to new gene-therapies

There is unlikely to be a single route for all gene-therapies, as this is a broad, growing, and highly heterogenous class. Therefore, it is important that new funding approaches for accelerating access continue to be tested and refined, and, where possible, lessons learned are shared to support future progress.

Recommendation 4: Establish evidence collection (patient outcomes and RWE data) infrastructure and policies to facilitate electronic evidence capture and use

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, a common framework and procedures at Belgian (and also European level) to support long-term evidence generation and to enhance the quality of evidence collected specifically for gene-therapies.

Recommendation 5: Confirm compliance of spread payment-based solutions with RIZIV and EU accounting rules

Confirmation is needed that within Belgian context spread payments are in compliance with the European Accounting Rules (ESA) and the RIZIV accounting rules under below formulated conditions.

Consensus report of the Alliance for Regenerative Medicine (ARM)

During the symposium The Alliance for Regenerative Medicine (ARM), the international advocacy organisation representing the cell and gene therapy and broader advanced therapies sector, also presented their consensus report and key recommendations to ensure European patients have access to Advanced Therapy Medicinal Products (ATMPs). The ARM 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.