Eurodis, Rare Impact Offer List of Policies to Improve Access to Advanced Therapies

Rare Daily Staff

Eurodis-Rare Diseases Europe, together with the partners of Rare Impact initiative, offered a list of policies to improve access to gene and cell therapies for people with rare diseases in Europe in a new report.

Though cell and gene therapies offer hope for people with rare diseases for which there are limited or no therapeutic options, the complex nature of their development and delivery has hampered patient access to these advanced therapies due to both practical and technical challenges, such as delivery of treatments at specialist centers, and reimbursement for the high cost of these treatments.

Rare Impact is a consortium of manufacturers of gene and cell therapies and umbrella organizations, such as the European Federation of Pharmaceutical Industries and Associations, the European Confederation of Pharmaceutical Entrepreneurs, and the Alliance for Regenerative Medicine. EURORDIS-Rare Diseases Europe chairs the consortium with support from Dolon Ltd.

The report addresses challenges of access to cell and gene therapies across four areas: accessibility, assessment, availability, and affordability.

Among the solutions it calls for:

• Greater collaboration between EMA, HTA bodies and heads of medicines agencies on guidance on health technology assessment of advanced therapies because of new uncertainties, as the complexity of these therapies as much as the new type of uncertainties, require long term follow up.
• A coordinated approach on the development and use of registries serving multiple purposes as the limited set of data and today’s fragmented approach needs to be addressed.
• Greater cooperation and clarity on use of the cross border healthcare provisions as many advanced therapies are delivered in only a few highly specialized hospital centers across Europe and cannot be delivered in all countries.
• More informative and technical discussions to grasp the specifics on advanced therapies’ cost and value.
• Payment based on outcomes and payment over time, though innovative payment mechanisms will be pre-empted by the removal of barriers.

EURORDIS is calling on stakeholders – patients, industry representatives, payers, regulatory bodies, and policymakers in the European Union to advance solutions to improve access to cell and gene therapies.

“The fact is that science is breaking through and will increasingly deliver innovative treatments that are potentially transformative or curative for people living with a rare disease,” said Yann Le Cam, CEO, Eurodis-Rare Diseases Europe. “It is now our collective responsibility to turn these indescribable hopes into a reality.”

Photo: Yann Le Cam, CEO, Eurodis-Rare Diseases Europe