Report calls for European funding reform for gene therapies

New payment models, dedicated investment, and better health technology assessment are among a raft of changes needed to bring regenerative medicines to patients in Europe, according to a new report.

The Alliance for Regenerative Medicine (ARM), an international advocacy organisation representing the cell and gene therapy and broader advanced therapies sector, published a consensus report containing recommendations to ensure European patients have access.

While regulators have systems in place to ensure rapid approval of Advanced Therapy Medicinal Products (ATMPs), payers and health technology assessment bodies do not always have mechanisms to capture the full benefits of these novel products.

The Alliance for Regenerative Medicine (ARM) report, Getting Ready for Advanced Gene Therapies in Europe, brings together views from European policy makers, experts, developers, and manufacturers of ATMPs.

It recommends a number of key measures, including quick adoption of payment models such as conditional reimbursement, pay-for-performance and annuity-based payments.

ATMP-dedicated funds could allow health systems to invest in therapies offering potential long-term benefits.

Early dialogue between ATMP developers and payers, supported by increased EU funding, could also help things along, as well as pan-European initiatives encouraging access to cross-border healthcare for patients.

These pan-European initiatives include creating a real world evidence infrastructure addressing uncertainties on long-term effects, safety, health-related quality of life and use of healthcare resources.

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.”

There have been some notable successes bringing gene therapies to patients however – in the UK NICE recommended funding for Orchard Therapeutics’ Strimvelis for severe combined immunodeficiency due to adenosine deaminase deficiency.

Otherwise known as “bubble boy syndrome” the one-off therapy costs around £505,000 but the cost-effectiveness body deemed it to be cost effective as it is better than standard stem cell transplant treatment.

Strimvelis used to be owned by GSK, but the big pharma spun it out into the small biotech Orchard.