RSNN Special Interest Group Advanced Therapies

  • Mission and Governance
  • Detailed Information

RSNN Special Interest Group Advanced Therapies

Advanced therapy medicinal products (ATMPs) such as gene and cell-based therapies are highly innovative medicines that provide new opportunities to treat conditions with high unmet medical need. It is often described that the characteristics of advanced therapies are considerably different from more conventional medicines such as small molecules or biologicals. Differences lay in their mechanism of action, live raw materials, intended function and manufacturing. In addition, ATMPs consist mostly of live cell and tissue materials.

The number of ATMPs stranded during (clinical) development is unknown but perceived as considerable given the contrast between high clinical activity and low number of products reaching advanced (regulatory) development milestones. Research has highlighted that ATMPs experience several clinical, regulatory, and economical challenges, more so than more conventional medicinal products. It is also observed that most ATMP-developers are not companies but hospitals, academic facilities, and so-called small and medium enterprises (SMEs); higher compared to the small-molecule and biotechnology industry.

RSNN has previously brought together stakeholders that are involved in ATMP development and discussed hurdles in bringing advanced therapies to patients, emphasizing the role of the national hospital exemption (HE) procedures. However, substantial regulatory questions remain unanswered, in and outside of HE. Examples are safety and quality concerns due to loss of centralized overview of ATMP development (in part due to HE), implications of acceptance of less complete evidence packages for marketing authorization (e.g., for conditional marketing authorization) for downstream assessment, constructive interactions between stakeholders, organizational learning. These questions require additional, tailored investments in terms of research and discussions to answer them.

Mission and Governance

Mission

The RSNN SIG Advanced Therapies aims to facilitate increased knowledge development about ATMPs and their regulation. Therefore, it can draw on the trusted position, experience, and network of RSNN as an independent broker within the regulatory field. However, at the same time, it can also draw on the networks of its individual SIG members within the advanced therapies space. The aim is to identify knowledge gaps and actions to address challenges perceived by stakeholders in ATMP development and assessment. In addition to identification, actions are drafted to address important questions related to current and future ATMP development in a bottom-up fashion, thereby bringing together public and private parties. The objectives of the SIG Advanced Therapies are met if these products have become an integral part of the drug development landscape.

Overall, the added value of the RSNN SIG Advanced Therapies is that it is positioned closely to developers in early development (mostly academic parties and hospitals), more advanced developer and marketing authorization holders (mostly pharmaceutical companies), and that this model can ensure a consistent dialogue with and amongst developers, academics, and all types of regulatory bodies.

Governance

The governance of the RSNN SIG Advanced Therapies is as follows:

  • Dr. Lourens Bloem, Assistant Professor Clinical Therapeutics at the Division of Pharmacoepidemiology and Clinical Pharmacology; Utrecht Institute for Pharmaceutical Sciences; Utrecht University (Co-Chair)
  • Dr. Renske ten Ham, Assistant Professor Health Technology Assessment at the Department of Healthcare Innovation & Evaluation and Medical Humanities; Julius Center; University Medical Center Utrecht (Co-Chair)
  • More SIG Advanced Therapies-members will be published in the nearby future.

RSNN SIGs are open and inclusive initiatives and subject to continuous development. Are you interested in contributing? Please contact info@RSNN.nl.

Detailed Information

Documents & Network

Key ATMP-related reports, organizations, and initiatives in the Netherlands are listed below. Please note, this overview will be reviewed and updated periodically and does not claim to be complete. In the case of additions of questions, please contact info@RSNN.nl.

ATMP products

An overview of the ATMPs that have been submitted for Marketing Authorization Application (MAA) at the EMA are being displayed in this document. The document provides information on each ATMP regarding brand name, active substance, indication, MAA assessment, and authorisation status. The document has been composed by Eleanor van Dijk. The information has been updated until 30 June 2022.

 

View document

Private developers

  • Amarna
  • Amylyx
  • Avexis
  • Biogen
  • Celgene
  • Gilead
  • HollandBIO (branch organization)
  • Janssen
  • Kiadis Pharma
  • Kite Pharma
  • Novartis
  • Pfizer
  • Roche
  • UniQure
  • VIG (branch organization)

Academic developers

  • Amsterdam UMC
  • Erasmus MC
  • Hovon
  • LUMC
  • Maastricht UMC+
  • NKI/AVL
  • Princes Maxima Centrum Utrecht
  • RadboudUMC
  • Sanquin
  • UMCG
  • UMC Utrecht

Regulatory authorities/government bodies

 

Initiatives

  • DARE NL: Dutch platform for cancer-specific ATMP Research to ensure harmonized development, clinical testing and sustained patient access (2022 - ongoing)
  • CURE4LIFE: Development and societal impact of stem cell based genetic medicines (2022 ongoing)
  • ICAT – Utrecht Innovation Center for Advanced Therapies
  • NecstGen
  • RegMedXB
  • reNEW
  • T2EVOLVE: Accelerating development and increasing awareness and accessof patients with cancer to immunotherapy