Published July 2019
In July 2019, ARM published a report on market access to ATMPs in key European countries. Designed to provide multi-stakeholder consensus on recommendations moving forward, the report is a result of the collaboration between ARM and European policy makers and experts, developers and manufacturers of ATMPs, patient organizations and other stakeholder groups. It provides a comprehensive review of the regulatory and market access framework across key European countries and identifies potential hurdles to the uptake of ATMPs in these areas. The report was funded by ARM.
ATMPs have the potential to offer a durable, life-changing therapeutic response, possibly with a single administration, for patients who may have few or no alternative treatment options. However, the complexity and relative novelty of these therapies present challenges to ensuring they reach those in need. While regulators have worked to adopt frameworks to ensure the appropriate, rapid approval of ATMPs in Europe, payers and health technology assessment bodies have not yet established the necessary specific mechanisms to ensure timely patient access to these therapies post-approval.
Better adapt Health Technology Assessment (HTA) frameworks to ATMPs by:
The Alliance for Regenerative Medicine hopes that continued dialogue and debate, supportive policy decisions, and a willingness among all stakeholders to create a fair and equitable environment for patient access to ATMPs will help overcome existing hurdles.
An allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease.
Approved in: The European Union
Approved for: Complex perianal fistulas
Holoclar is a cell therapy based on autologous cultures of limbal stem cells. It regenerates a functional corneal epithelium allowing recovery of visual acuity.
Approved in: The European Union
Approved for: Moderate to severe limbal stem cell deficiency due to ocular burns
Imlygic is a weakened form of Herpes Simplex Virus Type 1, which is commonly called the cold sore virus, indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.
Approved in: The European Union and United States
Approved for: Unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery
Kymriah is a CAR T-cell therapy product to treat R/R follicular lymphoma.
Approved in: The United States, the European Union, Canada, Japan, and Singapore.
Approved for: B-cell acute lymphoblastic leukemia, chronic lymphoid leukemia, diffuse large B-cell lymphoma, R/R large B-cell lymphoma, and R/R follicular lymphoma
A product containing spheroids of human autologous chondrocytes for use in cartilage defects.
Approved in: The European Union
Approved for: Cartilage diseases
Strimvelis is an ex-vivo stem cell gene therapy which uses retroviral vector encoding adenosine deaminase gene transfer into hematopoietic stem/progenitor cells.
Approved in: The European Union
Approved for: Adenosine deaminase severe combined immune deficiency
Zynteglo is an ex-vivo lentiviral based gene therapy used to provide a functional copy of the the β-globin gene into a patient’s hematopoietic (blood) stem cells.
Approved in: The United States (Withdrawn in the European Union in 2021)
Approved for: ß-thalassemia who require regular red blood cell (RBC) transfusions
