Aim: Regenerative medicines (RMs) are expected to transform the treatment paradigm of
rare, life-threatening diseases, while substantial challenges impede its market access. This …

This review discusses the methodological challenges to the evaluation of advanced therapy
medicinal products (ATMPs) by the UK National Institute for Health and Care Excellence …

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 …

Some advanced therapy medicinal products (ATMPs) hold great promises for life-
threatening diseases with high unmet needs. However, ATMPs are also associated with …

Abstract The Oslo Medicines Initiative (OMI) is a platform through which stakeholders can
collaborate to develop ways of achieving better, more affordable access to novel, high …

Background: The patient voice is an important consideration in the availability and choice of
pharmaceuticals–however, how to capture this complex area and apply it formally within …

A comprehensive review of the challenges that exist in patient accessibility to regenerative
medicines (RMs), presenting clinical trials, marketing authorization, HTA, pricing …

Background Enhanced horizon scanning for emerging treatments has identified that both
haemophilia A and haemophilia B pathways will be enriched with a range of new medicines …

With the increase in drug development studies for rare diseases, gene therapies have
recently come to the fore more frequently. In addition to orphan drugs used in the treatment …