In December 2021, the Regulation on Health Technology Assessment (HTA), as originally proposed by the European Commission, was adopted. The Regulation introduces a permanent framework for joint European collaboration on, among other things, HTA, to improve and facilitate access to innovative health technologies in the EU. The Regulation stipulates that, from January 2025 onwards, advanced therapy medicinal products (ATMPs) will be required to undergo joint clinical assessments. The relative efficacy of the ATMP will, from then on, be assessed centrally in Europe with the intent to reduce duplication of HTA activities and improve patient access.
ATMPs, such as gene, cell or tissue-based therapies, offer ground-breaking new opportunities for the treatment of various diseases and injury.
However, over the past 1 – 2 years these products have been the subject of numerous headlines due to the associated challenges with efficient patient access. Healthcare systems are often required to undergo significant adaptation in terms of infrastructure development, adoption of new legislation and implementation of alternative reimbursement solutions before access to patients can be granted. Further to this, in Europe, the requirement for individual HTAs and pricing and reimbursement discussions across different countries can result in both a daunting task and unattractive investment opportunity for manufacturers, especially those based non-EU, due to the often limited return on investment and effort.
Consequently, manufacturers of ATMPs commonly prioritise launch in countries where the necessary infrastructure, legislation and reimbursement frameworks already exist. However, for patients residing in countries where the necessary infrastructure, legislation and reimbursement do not exist, access to ATMPs is often limited. The only way to access these products in early years following regulatory approval is for patients to change domicile, pay OOP/through crowd-funding or enrol in a clinical trial. Alternatively, non-EU companies choose to sell their European commercial rights to more established partners which in turn can further delay access to treatment.
Larger, more established corporations have the capacity to expand access to small countries soon after launch. However, there are many small-medium enterprises (SMEs) with ATMPs in development (approximately 65% of companies with an ATMP in development according to a 2018 study) [3,4] for whom it is more difficult to expand access quickly and efficiently across a wide range of markets due to smaller field forces and fewer financial reserves. In this same study, these SMEs reported that country-specific requirements for access were their major concerns regarding product launch.
With an increasing number of ATMPs expected to launch in coming years, the need to explore innovative, cross-border access opportunities to overcome country-specific challenges and allow for broad patient access to these life-changing medicines is more important than ever. A 2020 analysis of the global ATMP pipeline identified 1,220 ongoing regenerative medicine and advanced therapy trials worldwide (956 unique therapies), 5% of which are expected to launch in the next 5 years.
This article examines the potential of European cross-border access opportunities that could expedite reimbursed patient access to ATMPs across a wider range of European countries and make Europe a more attractive investment for manufacturers of ATMPs.