Regulating Advanced Therapies

DOI: https://doi.org/10.58248/PN567 

Advanced Therapy Medicinal Products (ATMPs) are biological therapies which promise to transform many disease treatment, including a number of currently incurable ones.

The field has progressed rapidly over the last two decades with therapies now beginning to reach the market and patients. Examples of ATMPs include artificial tissues manufactured using stem cells, gene therapies targeting faulty genes responsible for incurable inherited diseases such as cystic fibrosis. and immunotherapies in which immune cells are reprogrammed to recognise and attack cancers.

As complex therapies, ATMPs present a number of challenges to developers, regulators, and healthcare providers. They require a high level of expertise to develop and produce, which makes them expensive to market. Regulators have found it difficult to integrate their biological variability into pre-existing guidelines. Healthcare providers also face issues based on their complexity and expense. However, some national healthcare agencies have found creative solutions in funding them.

ATMPs are defined and regulated centrally at the EU-level by the European Medicines Agency, as well as addressed in a number of other regulations such as the EU Cell and Tissue Directives. Since the Regulations came into force, only 9 ATMPs have reached the market. Whilst the regulatory framework has been perceived as excessively stringent by some ATMP developers, these regulations have not been the principle reason that so few therapies have been developed. Instead it has been the difficulties in developing them and in enabling national healthcare agencies to fund patient use, due to a lack of evidence for cost-effectiveness.

There are further concerns over how the UK will regulate ATMPs after Brexit and how attractive to developers the UK will be after Brexit. This will likely affect patient access as well as funding for the field in the UK.

Key Points:

• ATMPs  are biological therapies which have the potential to transform the treatment of many diseases, particularly rare and incurable diseases.
• They are centrally licensed by the European Medicines Agency and regulated by EU law, in which the UK’s regulatory bodies have had a strong voice. 
• A great deal of expertise to develop and manufacture them is required, but the UK has a strong ATMP sector, concentrated primarily in academia and smaller biotechnology firms.
• The therapies are expensive but several countries use flexible reimbursement mechanisms to fund patient use.
• Retaining harmonised ATMP regulation with the EU after Brexit is seen as key for patient access, clinical research, and for the UK to retain its leading role in  the field.