Based on the current political developments in Europe and the formulation of a National Strategy for Rare Diseases there is a need to provide suitable therapeutic options to underserved populations such as paediatric population and people living with rare diseases. Advanced Therapy Medicinal Products (ATMPs) can help address this unmet need.
Today, Giovanni Migliaccio, member of our board of directors and a Steering Group member of the TRANSFORM Alliance, will talk about ATMPs and their role in the new Pharmaceutical Strategy for Europe.
Giovanni, what are the advantages of the use of ATMPs as a therapeutic option?
ATMPs are medicines for human use that are based on genes, tissues or cells. Gene therapy medicines can have a therapeutic, prophylactic or diagnostic effect, somatic-cell therapy medicines contain cells or tissues that have been manipulated to change their biological characteristics and can be used to cure, diagnose or prevent diseases, while tissue-engineered medicines contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue. Combined ATMPs, containing one or more medical devices as an integral part of the medicine, are also available. They offer groundbreaking new opportunities for the treatment of disease and injury and as they can be personalised to a specific disease type, they have the potential to be used as therapeutic options for rare diseases, which might have a low number of patients and the paediatric population.
What are the challenges ATMPs face?
To assure maximum quality of these medicinal products and patient safety, policy changes across the lifecycle of ATMPs, supporting translational research, clinical trials, regulatory review, marketing authorisation, access and post-marketing monitoring and pharmacovigilance are needed. More investment and multi-stakeholder collaborative approaches, combined with targeted incentives, are needed in basic and translational research and development. Moreover, there is a need for improved access models to enable better patient access. The European Alliance for Transformative Therapies (TRANSFORM) has published a position paper about the Pharmaceutical Package, highlighting the need for improvements on several areas such as research, clinical development, regulatory approval and oversight, patient access, and the need to use real-world data.
What are the possible solutions to advance the use and access of ATMPs?
One possibility to advance research on ATMPs is the use of regulatory sandboxes, which may enable the testing of new ATMPs in a real-life environment and explore their opportunities and risks. It is important to stress that regulatory sandboxes do not aim to deregulate or reduce safety and protection standards. Ultimately, the primary purpose of using regulatory sandboxes as facilities for testing innovation and regulation is to gain clear regulatory knowledge.
To allow better access between borders, particularly regarding rare disease patients, cooperation between different European Reference Networks (ERN) should be promoted and increased. Furthermore, the strengthening of cross-border healthcare rights for patients would allow access to ATMP Centres of Excellence for all EU patients regardless of where they live, as well as cross-border payment models.