The client was planning to launch a new CGT therapy. There is a need to identify Dutch market access challenges and provide solutions with actionable recommendations to unlock access to CGT for Dutch patients who need this transformational promise of innovative treatment.
In recent decades, cell, gene, and tissue therapies have emerged as innovative treatment options. These potentially curative treatments are associated with large health gains for patients with conditions for which there are often limited treatment options available. Despite the promise of CGT, there are several challenges to ensuring timely and optimal reimbursed access to these innovations. CGTs are complex therapies with relatively high initial costs, and the curative claim is often based on small studies with limited follow-up. The challenges surrounding CGT are widely acknowledged in the scientific literature. International reports and white papers provide recommendations on how to overcome Health Technology Assessment (HTA) challenges and how to improve access to CGT for patients. In Europe, the joint clinical assessment of CGT will start in 2025. However, the remaining challenges in assessing the comparative (cost)effectiveness, and affordability of CGT therapies will require country-specific solutions.