How to address the affordability challenge for advanced therapy medicinal products

During a symposium at the Belgian sick Insurance agency RIZIV-INAMI on Wednesday 6 November 2019, the highlights and recommendations of the Vlerick-Inovigate multi-stakeholder policy report ‘Innovative Funding Solutions for transformative advanced therapy medicinal products (ATMP)’ have been presented and discussed by an expert panel. The organisers and participants hope that this policy report and its recommendations will facilitate continued dialogue and debate, and support policy decisions. It supports a willingness among all stakeholders to create a fair and equitable environment for patient access to gene-therapies and to overcome existing hurdles.

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.

Download the policy report

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.    

About the multi-stakeholder symposium
During the symposium at RIZIV/INAMI on 6 November 2019, the report highlights were discussed together with a debate with stakeholder representatives on the practical implementation aspects.

PROGRAMME

Welcome & introduction 
Keynote – Problem statement by Jo De Cock , CEO National Institute for Health and Disability Insurance (RIZIV – INAMI) 
Game changing therapies - Gene therapy  by Prof. Thierry Vandendriessche, Director of Gene Therapy VUB
The EU perspective - Recommendations for Timely Access to ATMPs in Europe by Annie Hubert, Senior Director Alliance for Regenerative Medicine (ARM) 
Highlights & Recommendations from the multi stakeholder round table meetings and the policy report, by Ingrid Maes, Managing Director Inovigate and Prof. Walter Van Dyck, Director Vlerick Healthcare Management Centre 
Short-term budget & affordability challenges – the haemophilia gene therapy case, by Rudy De Cock, Health & Value Director Pfizer 
Debate on practical implementation, moderated by Ingrid Maes, Managing Director Inovigate
• Prof. Thierry Vandendriessche, Director of Gene Therapy VUB
• Annie Hubert, Senior Director Alliance for Regenerative Medicine (ARM)
• Diane Kleinermans, Advisor to the Ministry of Health and Social Affairs Belgium
• Francis Arickx, Head of the directorate Pharmaceutical Policy at RIZIV – INAMI
• Kristel De Gauquier, Medical Director at pharma.be
• Prof. Walter Van Dyck, Director Vlerick Healthcare Management Centre 
Conclusions 

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