Mapping the hurdles for the clinical applications of Advanced Therapy Medicinal Products (ATMPs)

New pioneering treatments called Advanced Therapy Medicinal Products (ATMPs), including cell and gene therapies, have the potential to bring new cures to patients affected by diseases with limited or no available treatments. However, several hurdles impede or slow down the access of ATMPs to patients in the EU and Associated Countries. These include e.g. regulatory challenges, underlying scientific uncertainties, differences in assessing the values of ATMPs by the various Health Technology Agencies (HTA), difficulties to perform randomised-controlled clinical trials or to obtain long-term safety and effectiveness data, the lack of harmonised approaches to the reimbursement of the high upfront costs by health systems, manufacturing processes, etc.

The proposals should address all of the following activities:

• Map the regulatory, safety and efficacy assessment, manufacturing, organisational and infrastructural needs to improve the translation of ATMPs from preclinical development to clinical use.
• Address the gaps and uncertainties in regulatory and policy aspects pertinent to complex innovative ATMPs.
• Address predictivity of preclinical data for safety and efficacy testing of ATMPs. Improved novel models could be proposed.
• Tackle decision-making processes relating to ATMPs, such as for instance the assessment of their values, the demonstration of the long-term safety and effectiveness, or new pricing and reimbursement frameworks.
• Propose opportunities for an improved knowledge of the regulatory processes among academic ATMP developers.
• Involve regulatory authorities, Health Technology Agencies (HTA), clinicians, ethics committees, and patients, with the aim to ensure higher clinical use of ATMPs. The findings of the project will be available to competent authorities, ATMP developers and manufacturers as well as to national/regional funding agencies.