Sustainability of Advanced Therapy Medicinal Products (ATMPs): overcoming the valleys of death in the translational research and post marketing-authorization
Project The literature highlights the distance between basic and clinical research (translational valley of
death) in Advanced Therapy Medicinal Products (ATMPs), the underfunding of clinical research and
the risk of economical unsustainability of authorized ATMPs with a consequent withdrawal and
patients’ use (market valley of death).
Starting from our preliminary data and from a direct experience in ATMPs failure, a multidisciplinary
team (with biomedical, industrial, quantitative, financial and legal skills) aims to:
-specify and measure the clinical, operational and financial needs of the clinical research in ATMPs;
-identify new industrial and research models able to improve the economic sustainability of
developing and approved ATMPs;
-identify new financial schemes able to improve the financial system’s allocative efficiency and to
enlarge its funding capacity;
-identify areas of (non-economic) intervention where authorities could focus their attention to improve
ATMPs’ sustainability.
The research path follows the Capital budgeting method, that is separately determining the project’s
financial flows, its risks and the sustainable financing methods, in order to assess the financial
sustainability (NPV) and social sustainability (NPSV) of different clusters of ATMPs.
This approach also enables identification of the clinical, operational, regulatory factors where action
can be suggested and taken to achieve more sustainability and make research of therapies feasible.
We use a sample of 1.042 European ATMPs clinical trials (344 of which involving Italian researchers)
identified through the ClinicalTrials.gov database (integrated by PubMed). Preliminary analyses have
outlined that currently the financial system is only indirectly funding ATMPs clinical research through
investments in 65 ATMP-engaged SMEs. These companies will be examined in depth through
financial annual reports public information and a licenced database (Crunchbase). The same
analysis will also be done on all the companies involved in management and administration of
authorized ATMPs (or in their withdrawal).
Once the operational strengths and weaknesses are identified together with financial system's
failures, the study of new operating models and financial schemes continues along two strands:
1.the management and optimisation (i.e. reduction) of operational and financial needs through the
valorisation of intermediate outputs (organoids, data, intermediate platforms, technological knowhow,
etc.), new operating models and the use of consortium structures both in clinical research and
ATMPs’ production and administration;
2.financial innovation and creation of new combinations of contracts both able to effectively catalyse
resources already present on the market towards investments in ATMP research and capable to
involve the more retail component of the market and ESG institutional funds (by managing the
minimum viability and sustainability thresholds of investments, exploring criteria for portfolio selection
and methods of risk sharing and by improving the risk/return trade-off).