OBJECTIVES: It has been an eventful 12 months in the gene therapy pipeline, with excitement tempered by safety issues and concerns around durability of effect and implications for pricing and access. With the FDA predicting 10-20 cell and gene therapy approvals per year by 2025, there is a clear need to take a step back and consider how to align various stakeholders. By analysing pipeline developments over the past 12 months, we explore the key themes emerging.

METHODS: Cogentia analysed 100 gene therapies currently in clinical development, exploring developments & news flow in the past 12 months, and categorising each as either green (positive development), amber (limited/no development) or red (negative development). Qualitative mixed methods were used to draw out key themes emerging, considering manufacturer and payer perspectives.

RESULTS: Our analysis found positive developments for 32 of the 100 gene therapy assets, limited/no developments for 47 of the 100, and negative developments for 21 of the 100 gene therapies. Some of the key themes emerging include the commercial challenges of ultra-orphan development, the issue of durability of effect, the balance of benefit:risk and implications for evidence sufficiency, and considerations around positioning (target vs addressable population).

CONCLUSIONS: It will be important for manufacturers and payers to align on how best to approach key challenges, balancing out the incentives to develop innovative therapies with budget constraints at the national level. One key challenge is incentives to develop gene therapies for ultra-orphan disease. Interestingly, whilst gene therapy continues to be a hot topic, the anticipated ‘pipeline boom’ is yet to be seen, with just two CHMP positive opinions issued in the previous 12 months.