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:
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:
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:
Recommendation 4: Favor wider application of innovative access and funding arrangements such as:
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.
For more information about the report or media requests, please contact Lyndsey Scull from the Alliance for Regenerative Medicine at firstname.lastname@example.org or Consilium Strategic Communications at email@example.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.