Challenge:
ATMPs represent a cutting-edge class of medicines that leverage genes, tissues, and cells to restore, correct, or modify biological functions. These innovative therapies hold significant promise, often requiring only one or two administrations to achieve curative or long-lasting effects. However, their unique characteristics also present substantial challenges across the preclinical, clinical, and regulatory landscapes. The complexity of navigating regulatory pathways and ensuring compliance with ATMP-specific requirements can delay development and hinder access to patients. Many developers lack clarity regarding these regulatory processes, potentially impeding the timely and sustainable delivery of ATMPs to patients.

Project Goals:
The objective of this project is to provide ATMPs developers with a practical guide to the necessary steps and regulations that must be followed. This aims to contribute to a better understanding of the development process and its requirements, enabling stakeholders to navigate the complex field of ATMP development in an efficient and informed manner.

FAST has updated and expanded the previously published handbook by the Association of Innovative Medicines (Vereniging Innovatieve Geneesmiddelen, ‘VIG’). This revised handbook will cover essential topics such as patient involvement, the academic development of ATMPs, EMA procedures, and hospital exemptions. To ensure that the handbook is relevant and practical for ATMP developers in the Netherlands, FAST has engaged with public and private stakeholders during the revision process. This updated ATMP handbook is intended as a fundamental tool for academic and industry professionals developing ATMPs to bring these therapies to the Dutch market.

The Guidebook covers the following:

The Guidebook is designed to provide comprehensive support to developers navigating the ATMP regulatory landscape, from initial development through market access in the Netherlands. It offers key insights into legal frameworks, patient involvement, product classification, clinical development, and reimbursement options and regulations. In addition it provides practical tools, national and international support networks, and expert consultations to assist developers in navigating ATMP development.

Project Status: FAST will release the Guidebook for ATMP Development in October/November 2024

Executing Party: Vivaron Consultancy

A steering committee with representatives from CBG, DARE-NL, VIG, and HollandBio is overseeing the development of the guidebook. Experts from ZIN, IGJ, CCMO, CBG, RIVM, ZN, patient organizations, companies, academia, and other research institutions have contributed to this guidebook.

Contribution of FAST: FAST is the initiator and sponsor of this project, advises the project team, and involves parties in the implementation.