Development, market authorization and market access of gene and cell-based therapies

Abstract

Gene and cell-based therapies (GCTs) are highly innovative therapies and hold great promise in the treatment and potential cure of high burden and chronic diseases. However, it is reported that these products experience translational challenges in successfully surpassing development milestones towards market authorisation and market access.

The aim of this thesis is to assess gene and cell-based therapy development challenges and how these challenges play a role in marketing authorization and market access, as well as develop tools and methods to mitigate market access challenges for developers.

This thesis demonstrated that new product characteristics of GCTs do not fit particularly well within established development, regulatory and HTA-frameworks for conventional medicinal products. Novel GCT characteristics ask for redesign of established medicinal product handling, manufacturing and quality assurance which is accompanied by increased development cost and higher risk. The novelties also require capability building in interaction between developers demonstrating and authorities assessing product quality, safety and efficacy. The novel characteristics seemed to affect the health technology assessment (HTA) to a lesser extent. However, the intended curative effects, originating from new mechanisms of action, and higher target prices continue to raise concerns amongst payers and HTA-bodies on how to address budget impact and affordability as more GCTs reach late clinical development.

The research provides a cross section of (early) development challenges of GCTs and how they affect down-stream development spanning manufacturing to reimbursement. This thesis describes empirical findings from a systems and developer perspective. It also provides cost and cost-effectiveness tools and methods to classify challenges and considerations in HTA to accommodate timely, safe and sustainable patient access. This research, therefore, contributes to better understanding of the fit between these transformative therapies and existing development, regulatory and HTA-frameworks, which is not only relevant for GCTs but also for future biomedical innovations.