Geneesmiddelen voor Geavanceerde Therapie (ATMP’s) in Nederland: Veldverkenning, Knelpuntanalyse en Activiteitenkaart

Advanced therapy medicinal products (ATMPs) offer treatment options for previously poorly treatable and untreatable conditions. ATMPs are formally categorised into gene therapies, somatic cell therapies, tissue engineered products, and combined ATMPs. As part of the ZonMw programme ‘Regulatory Pandemic Preparedness,’ overviews have been created of stakeholders, legislation and guidance, routes for marketing authorisation and reimbursement, bottlenecks and solutions, and recent and current initiatives related to marketing authorisation and reimbursement of ATMP’s in the Netherlands, taking into account the European context. Therefore, the Dutch ATMP field was mapped and a bottleneck analysis was performed through a systematic literature review, a multi-stakeholder workshop, and interviews. To validate and supplement findings, a field consultation was performed. The identified and updated bottlenecks were categorised in various phases of the drug lifecycle: preclinical development, clinical development, marketing authorisation, reimbursement, and integration in clinical practice. Additionally, a ‘systemic’ category was created for bottlenecks identified for multiple phases. A graphical overview of the identified bottleneck themes is provided below. Thereafter, an activity map was created by connecting recent and current initiatives in the ATMP field to bottleneck themes. This map illustrates the themes that have been or are being addressed as well as the themes that may require new initiatives. Finally, recommendations for actions were formulated to address currently unaddressed bottlenecks. These recommendations are listed on the following page. Overall, this report offers actionable insights and recommendations for developers, assessors, policy makers, healthcare professionals, and other interested parties in the Dutch ATMP field, thereby aiming to facilitate patient access to ATMPs.