Ensuring timely access to advanced therapies in Europe

Recommendations from the Alliance for Regenerative Medicine aim to improve cross-border healthcare to ensure patients in Europe can access advanced therapies.

The Alliance for Regenerative Medicine (ARM) is an international community of small and large companies, non-profit research institutions, patient organisations, and other sector stakeholders dedicated to realising the promise of regenerative medicine for patients around the world.

ARM represents the significantly growing stem cell and gene therapy and broader advanced therapies sector.

A new ARM position paper focuses, and further elaborates, on the recommendations of ARM’s July 2019 report on ensuring timely access to alternative medical treatment in Europe. It represents the views of the ARM members and aims to stimulate debate and reach consensus among key stakeholders, including marketing authorisation holders, payers and treatment centres, on solutions to ensure all European patients can secure access to these new solutions, irrespective of their country or region of origin.

Not all approved procedures are expected to be made available to all countries in Europe or to all regions of a given country.

The legal frameworks that grant the right to cross-border healthcare for patients in Europe are not optimal for these treatments.

The lack of pricing assessment in the patient’s country of origin and regional budgets or multiple payers/insurers in some countries can constitute barriers to cross-border or cross-region treatment.

ARM’s key recommendations:

• Establish a ‘one-stop shop’ Advanced Therapy Medicinal Products (ATMP) coordination body at EU/EEA level to act as a broker between the different stakeholders and facilitate cross-border patient treatment and funding.
• Create one-stop shop ATMP coordination bodies in countries with regional funding or with multiple payers/insurers to ensure authorities in the regions of treatment are compensated for the costs of treating patients from other regions.
• Encourage more effective coordination of activities to ensure greater alignment within Europe on product value assessment measures.
• Improved opportunities for cross-country collaboration, removing duplicative processes at national level, and adopting policy principles to enhance cross-country collaboration.

Janet Lambert of ARM, has commented: “Europe has always been a leader in innovation, however, to ensure that patients have access to these transformative treatments, there are several challenges that need to be overcome at EU, national and regional levels.”

ATMP medicine includes gene therapies, cell therapies, and tissue-engineered products intended to augment, repair, replace or regenerate organs, tissues, cells, genes, and metabolic processes in the body.

Regenerative medicine aims to alter the current practice of medicine by treating the root causes of disease and disorders. The regenerative medicine and advanced therapies sector is creating transformative, durable treatments and potential cures for some of humankind’s most devastating diseases (many currently untreatable via conventional treatments) through the use of ground-breaking scientific discoveries and technologies.

ARM has a database of the regenerative medicine sector globally. It tracks 900 companies around the world in this sector, and 1,000 active clinical trials.

In terms of the conditions and diseases that these medicines are addressing, much of the early work has been in rare diseases, monogenetic diseases, as well as in oncology. Within five years there are likely to be many approved gene therapies and tissue engineered products on the market in the USA and Europe.

The biggest challenge is trying to fit a new kind of medicine into an old kind of reimbursement system. Most medicines are taken over a long period of time. Many of the most exciting new medicines in this field are likely to be one-time treatments. These products can be expensive so public and private payers are trying to figure out how they can incorporate these kind of medicines into their health system.