Innovative treatments, such as gene- or immunotherapy, provide promising solutions warranting accelerated approval. Yet, in general accelerated approvals may pose safety challenges. Among the processes for access and affordability of such treatments information on safety may be less than we are used to in non-accelerated procedures. Here, we investigate to what extent existing procedures satisfy the needs of safety assessment in the healthcare system and how potential existing safety gaps can be addressed.

A pragmatic review was carried out to identify relevant literature, using PubMed as a search platform to explore MEDLINE and snowballing as an additional method for further search.

There is a trend that solutions for accelerated access for innovative treatments are implemented in models of regulatory approvals, yet with limited data. Besides efficacy data, providing adequate safety data is key to transferring conditional- to final marketing authorization. However, this remains a challenge because of the restricted availability and transferability of such data. An example to overcome such issues is the International Coalition of Medicines Regulatory Authorities (ICMRA) who provide equitable access to global clinical trials data regarding COVID-19. Similarly, HTA bodies and manufacturers are overcoming clinical data issues in the Sharing European Early Dialogs (SEED). Many countries re-invent multi-criteria decision analysis (MCDA) as alternative to focus on (cost-) effectiveness only, yet eligibility, clear criteria, choosing the right model, and outcomes interpretation remain challenges. With managed-entry agreements (MEAs), access barriers between manufacturers and health authorities can be are overcome in conditional access situations. Although a growing trend of outcomes-based MEAs is noticed, financial-oriented MEAs remain dominant, and those MEAs that are outcomes-based focus on effectiveness rather than safety.

Safety may be an aspect undervalued in accelerated access procedures. We notice and suggest increasing use of MCDA and MEAs also focussing on safety in real-life conditional access situations.