Significant scientific advances have created a rich clinical research pipeline, with innovative and potentially curative cell and gene therapies already available. More of these therapies are expected to enter the market for other disease areas and larger patient populations over the next decade. Potentially curative cell and gene therapies represent a key shift in patient care and will have considerable impact across the patient journey, affecting the healthcare system as we know it and the key stakeholders involved. Their launch is also met with significant debate over the uncertainty of outcomes’ durability and safety over the longer term; and the impact of treatment prices on affordability as more treatments enter the market.
In this European Pharmaceutical Review article, we draw from characteristics of treatments in the pipeline and discuss the implications these will have on elements of the healthcare system and stakeholders that are most likely to be disrupted. Specifically, we discuss the impact on provision of care, delivery and supply chain, pricing and market access and value demonstration — given the difficulty of proving a durable, curative effect.