Background The aims of this research were to provide a better understanding of the specific
evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies …

Regenerative medicine, poised to transform 21st century healthcare, has aspired to enrich
care options by bringing cures to patients in need. Science-driven responsible and …

Organoids hold great promises for numerous applications in biomedicine and
biotechnology. Despite its potential in science, organoid technology poses complex ethical …

Gene therapy orphan medicinal products constitute a unique group of new drugs which in
case of hereditary diseases are usually administered only once at an early age, in the hope …

Advanced therapy medicinal products (ATMPs) are a fast-growing field of medicine with
wide potential application. Nevertheless, so far, only 19 have obtained European Union …

The emergence of gene therapies challenge health economists to evaluate interventions
that are often provided to a small patient population with a specific gene mutation in a single …

Objectives Advanced therapy medicinal products (ATMPs) are highly innovative therapies.
Their costs and uncertain value claims have raised concerns among health technology …

Objective No consensus exists on the ideal methodology to evaluate the economic impact
and value of new, potentially curative gene therapies. We aimed to identify and describe …

Gene therapies (GTs) are considered to be a paradigm-shifting class of treatments with the
potential to treat previously incurable diseases or those with significant unmet treatment …

Objective: This systematic review aimed to assess the current use and acceptance of real-
world data (RWD) and real-world evidence (RWE) in health technology assessment (HTA) …