OBJECTIVES: Cell and gene therapies (CGTs) is a rapidly growing therapeutic area holding great promise for treating rare diseases with high unmet medical needs, with nearly 60% of the 2,400 ongoing CGT trials at the end of 2021 targeting prevalent diseases. Our paper aims to highlight, among the markets in scope, where recent policy developments suggest the most promising or challenging outlook.

METHODS: An extensive screening of 2021 news and reports was performed for 20 markets globally spanning 5 regions, consulting official sources and relevant media in local languages. Selected developments covered healthcare policy enacted/proposed reforms, discussions around pricing and reimbursement (P&R) adaptation, trade associations and patient organizations advocacy, alongside regulatory approvals, P&R decisions and HTA opinions on 9 key CGTs. Implications and outlook were analyzed and classified into positive, neutral/mixed or negative from the perspective of multinational manufacturers.

RESULTS: A total of 169 significant developments were selected and analyzed, with almost 54% relating to the 9 key therapies in scope. 89 developments had positive implications, with the UK and US leading the efforts. The UK provides a variety of supporting tools, while the US prioritizes CGTs’ expedited access and increased use of value-based purchasing arrangements. 40 negative developments were identified, and another 40 entailed neutral/mixed implications. The highest number of negative events were registered in Germany, adding pressure on high-cost medicines threatening its early-launch market attractiveness, followed by China, presenting important challenges for imported overseas products by favoring domestic production.

CONCLUSIONS: Although CGTs still face important budget constraints and general unpreparedness to higher uncertainties, several markets show increasing endeavors to improve market access conditions. While our study suggests the UK and US are at the forefront, and Germany and China present higher forthcoming challenges, evolving P&R policies in the CGT space require constant monitoring to drive market access success.