RSNN Special Interest Groups
RSNN Special Interest Group Advanced Therapies
Advanced therapy medicinal products (ATMPs) such as cell and gene therapies are highly innovative medicines that provide new opportunities to treat conditions with often high unmet medical needs. Research has highlighted that ATMPs experience several clinical, regulatory, policy and economical challenges that are specific to or more pronounced for ATMPs as compared to small molecules and other biologicals. These challenges require tailored investments in terms of research and constructive multi-stakeholder discussions to address them and contribute to a collaborative ATMP ecosystem. To facilitate these tailored investments, RSNN established the Special Interest Group (SIG) Advanced Therapies in 2022.
Mission and Governance
Mission
The RSNN SIG Advanced Therapies aims to facilitate knowledge development and knowledge sharing about ATMPs and their regulation.
To achieve this aim, the SIG draws on the trusted position, experience, and network of RSNN as an independent broker within the Dutch regulatory and policy field. In addition, the ATMP-specific focus allows the SIG to not only identify knowledge gaps but also formulate and facilitate actions to address perceived challenges in a unique multistakeholder setting. Actions are drafted in a bottom-up fashion by bringing together public and private parties, including patients, healthcare professionals, small and large(r) developers (public and private), regulators (in the broadest sense), payers, governmental bodies, policy makers, and academics. This approach ensures a consistent dialogue between all parties involved in development and assessment of ATMPs in the Netherlands and beyond.
To inform interested parties about the ATMP field, the SIG maintains overviews of ATMPs that have been assessed by the European Medicines Agency, ATMPs in late development, important stakeholders, and key reports (see ATMP Field below).
In principle, the objectives of the RSNN SIG Advanced Therapies are met when ATMPs have become ‘business as usual’ within the drug development landscape.
Governance
The governance of the RSNN SIG Advanced Therapies is as follows:
- Lourens Bloem – Utrecht University (Co-Chair)
- Renske ten Ham – University Medical Center Utrecht (Co-Chair)
Core Members
- Mariette Driessens – Patient Alliance for Rare and Genetic Diseases (VSOP)
- Babs Fabriek – Dutch Medicines Evaluation Board (MEB)
- Francisco Hernandez – AstraZeneca
- Sophie Huiskes-Berends – RSNN/Lygature
- Pauline Meij – Leiden University Medical Center
- Connie van Oers – uniQure
- Lonneke Timmers – National Health Care Institute (Zorginstituut Nederland)
SIG Activities
SIG Network and Approach
The RSNN SIG Advanced Therapies aims to organize an annual meeting open to all interested parties. The annual meeting facilitates networking and interaction between stakeholders and allows the SIG to gather input from the ATMP field. In addition, one or more expert meetings are organized yearly to address a specific theme more in-depth. The form and structure of these thematic expert meetings may vary and are subject to its purpose.
We welcome input for annual and expert meetings. Please reach out via info@rsnn.nl.
Past Activities
-
2024
On Tuesday 11 June 2024, the RSNN SIG Advanced Therapies together with the Centre for Future Affordable Sustainable Therapy development (FAST) organised a workshop & networking event.
The topic of the event was the potential need for a national platform that could facilitate interaction, knowledge exchange and cooperation in the Dutch ATMP field. In addition to the main topic, Heather Main shared learnings from the Swedish platform ‘ATMP Sweden’ and Arjan Lankester from the AGORA network that aims to facilitate sustainable access to effective gene therapies for treatment of patients with ultra-rare diseases in Europe.
The highly multidisciplinary audience clearly expressed a need for a national platform and highlighted that it should mainly focus on continuous mapping of stakeholders and initiatives to facilitate interaction and collaboration, as well as function as a single point of contact, both from a national and international perspective.
-
2023
In June, the SIG’s annual workshop & networking event was held at Vila Jongerius in Utrecht. During a plenary session, the multistakeholder audience was invited to provide input concerning perceived challenges in ATMP development, assessment, and patient access, and rank these from highest to lowest priority. Next, in small break-out groups, prioritised challenges were assessed in more detail, including how to address them and by who. A plenary discussion brought the findings together and allowed for further input from all participants.
The input from this meeting was used to set the agenda for future activities of the SIG and its partners. Moreover, the workshop output was included in the report: “Geneesmiddelen voor Geavanceerde Therapie (ATMP’s) in Nederland: Veldverkenning, Knelpuntanalyse en Activiteitenkaart”. Available via https://doi.org/10.33540/1874/433501 (in Dutch).
-
2022
Launch RSNN SIG Advanced Therapies at the RSNN Annual workshop; core member group established.
The ATMP Field
The SIG maintains several overviews to inform interested parties about the ATMP field. These overviews will be reviewed and updated periodically but do not claim to be complete. In case of additions or questions, please contact info@rsnn.nl.
-
ATMPs assessed by the European Medicines Agency and in late development
Overviews of the ATMPs that have been assessed by the European Medicines Agency (EMA) for marketing authorization, as well as those in late development (Horizonscan). In addition to the information from horizonscangeneesmiddelen.nl, the relevant patient organisations have been added to the Horizonscan thanks to the ‘Patiëntenplatform Gen- en Celtherapie’, Patiëntenfederatie Nederland (PFN) and the Nederlandse Federatie van Kankerpatiëntenorganisaties (NFK).
-
Reports
- EC & EMA European Commission DG Health and Food Safety and European Medicines Agency Action Plan on ATMPs (2017)
- EMA – CAT Reflection paper on stem cellbased medicinal products (2011)
- EMA Report from the CAT expert meeting on scientific and regulatory considerations for adeno-associated viral vector (AAV)-based gene therapy (2018)
- EMA – Outcomes of the multistakeholder advanced therapy medicinal products (ATMPs) expert meeting: exploring solutions to foster ATMPs’ development and patient access in Europe (2016)
- EMA - EMA warning against using unproven cell-based therapies (2020)
- KWF – Cel en gentherapie naar de oncologische klinische praktijk: Kansen en knelpunten voor innovatie vanuit de academie (2021)
- LERU - Advanced Therapy Medicinal Products (2019)
- LUMC – Advanced Therapy Medicinal Products (ATMPs) naar de reguliere klinische zorg: Knelpunten en Mogelijkheden (2016)
- RIVM – De toekomstverwachtingen over ATMPs (2018)
- RSNN - RSNN Expert Meeting Report - The route for ATMPs from bench to bedside (2021)
- VWS – Kamerbrief met stand van zaken geavanceerde therapieën (2022)
-
Patient organizations
- Hematon
- Nederlandse Federatie van Kankerpatiënten organsiaties (NFK)
- Stichting Melanoom
- Patient alliance for rare and genetic diseases (VSOP)
- Nederlandse Vereniging van Hemofilie-Patiënten
- Volwassenen Kinderen en Stofwisselingsziekten (VKS)
- Vereniging Spierziekten Nederland
- Vereniging Kinderkanker Nederland
-
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
- Central Committee on Research Involving Human Subjects (Centrale Commissie Mensgebonden Onderzoek)
- Loket Gentherapie
- Health and Youth Care Inspectorate (Inspectie Gezondheidszorg en Jeugd)
- Medicines Evaluation Board (College ter Beoordeling van Geneesmiddelen)
- Ministry of Health, Welfare and Sport (Ministerie van Volksgezondheid, Welzijn en Sport)
- National Health Care Institute (Zorginstituut Nederland)
- Committee for Advanced Therapies (CAT), The European Medicines Agency (EMA): The EMA provides information on the marketing-authorisation procedures for advanced-therapy medicinal products and offers support for advanced-therapy developers.
-
Initiatives
- DARE NL: Dutch platform for cancer-specific ATMP Research to ensure harmonized development, clinical testing and sustained patient access (2022 - ongoing)
- ATMP Course Directory with ATMP educational resources and events
- 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
- Alliance for Regenerative Medicine (ARM)
- National Health Care Institute (Zorginstituut Nederland) Horizonscan Medicines
- DARE NL: Dutch platform for cancer-specific ATMP Research to ensure harmonized development, clinical testing and sustained patient access (2022 - ongoing)