2024-03-13
Nordic ATMP stakeholders meet to discuss opportunities in collaboration
The 4th of December 2023, participation from each of the Nordic countries was invited to initiate discussions on what a Nordic ATMP network could look like. Based on the principle that each country alone is too small to have all needed onshore capabilities for manufacture, clinical trials and even health technology assessment for payment model evolution and reimbursement, the countries came together to explore synergistic opportunities. The number of potential ATMP modalities eg. mRNA, CD34+ gene modified, in vivo AAV GT, pluripotent stem cell derived, mean that no single actor or country will satisfy the needs and expertise for all ATMPs. Working together in a synergistic model was well received by all involved and recognized as necessary to all countries being part of a globally competitive ATMP ecosystem.
The program included presentations from each Nordic country, breaking up into working groups to brainstorm possible collaborative projects and finally a discussion of where to go from here. Summaries from the working groups can be found below, including the happy, smiling faces of participants in those working groups.
Where to now? Each working group has been encouraged to stay in touch and for example, the GMP group have organized to have monthly online meet ups to keep a finger on the pulse of possible collaborative opportunities. A small group of self-nominated individuals will work towards establishing a regular Nordic ATMP conference, collaborative marketing opportunities and online opportunities for information exchange. To offer your support, please feel free to get in touch!
PROGRAM 4th Dec 2023
Workgroup summaries and participants
Online, lead by Anna Pasetto, online platforms for promoting collaboration – ”The overarching goal is to establish a dynamic online space for knowledge exchange and partnership building. We are considering informal forums like chat platforms for question-based discussions. Additionally, more formal resources such as CCRM and ISCT round tables could be utilized to promote formal engagement through seminar series and case studies.” Anna Pasetto, Director of the Centre for Advanced Cell Therapy (ACT), Oslo University Hospital.
image from top left: Anna Pasetto, Carin Dahlberg, Petri Mäkinen, Hanne Haslene-Hox, Marcela Franco, Agnete Kirkeby, Heather Main. Emmet McCormack and Sébastien Wälchli no picture.
Pre-GMP, lead by Elias Uhlin. Considering the significance of proper process development strategies needed to successfully develop an ATMP, this is an area where exchange of expertise and collaboration will be essential to become a world leading region. During the course of the meeting there was great alignment between the Nordic actors towards leveraging each others strengths and competences in early process development. No single actor is likely to be the world-leading expert in all processes related to ATMP development. Therefore exchange of expertise and collaboration will be essential to become a world leading region.
From a pre-GMP perspective, there are synergies to pursue in educating operators into cleanrooms, building a directory of expertise and capabilities and listing the developmental stages of actors in early process development. For the industrial developers there is currently a focus on scaling and automatization where a proper CMC strategy is of paramount importance. The needs for a Nordic process development forum was raised. A reccurring Nordic ATMP meeting based on a similar format to the annual ATMP Sweden conferences was suggested.
image from left: Jim Lund, Salwa Suliman, Elsa Abranches, Lindsay Davies, Michael Delahaye, Sara Nilsson, Janna Saarela, Elias Uhlin, Jennifer Rosendahl, Mauro Sbroggiò
Reimbursement, lead by Björn Skölving. Relating to the access challenges for ATMPs, from the patient perspective there is often a clear need to make treatment available even if the approval is conditional or based on single arm studies. The way to address this is by increased engagement by all parties involved to establish high quality follow-up for generation of proper evidence. Hospital Exemption is there to fill unmet medical need where there are no approved therapies, not to create competition. If we were able to convert initial use under the Hospital Exemption to product development and product approval, many more patients could benefit from such a scalable solution.
The discussion also touched on the discrepancy in evaluating evidence in the regulatory process and the health economic evaluation, where Maria (TLV) raised that products supported by weak evidence may be less likely to be reimbursed and/or recommended for use. Nordic synergy in Health Technology Assessment may be warranted in certain cases. This is especially needed where available evidence could be insufficient for relative effectiveness assessment due to limited size target patient groups, single arm clinical trials and limited duration of follow-up data when there are expectations for very long effects, even lifelong. Models for funding the treatments may need to be revised, for example by funding of highly specialised care from national sources, rather than regional.
image from left: Dag Larsson, Maria Eriksson, Agneta Edberg, Björn Skölving
GMP, lead by Pontus Blomberg. To enable the Nordics to be competitive now and in the future we aim to collaborate on competence development: there are Nordic synergies to pursue in education, knowledge, manufacturing systems, quality/reg, training for GMP manufacture. Study visits would also be beneficial. This group has started to run a monthly digital get-together.
”There are lot of innovative therapies being developed and there are good possibilities to manufacture these in the Nordic countries. By collaborating and sharing experiences between the Nordic actors in GMP manufacturing, we are ready to take on the number of possible ATMP modalities and the diversity of needs in their manufacture from phase I/FIH, all the way to commercial manufacture. This will benefit the whole Nordic ecosystem by developing competences needed in our manufacturing infrastructures and maximizing the number of modalities that we can provide in high quality and scale for a global market.” Janet Hoogstraate, NorthX Biologics.
image from left: Luisa Lundin, Fredrik Wessberg, Janet Hoogstraate, Karl-Johan Malmberg, Peer Horn, Ditte Caroline Andersen, Pontus Blomberg, Thomas Carlsen, Anna Tegnesjö
Education, lead by Anita Kavlie. There are synergies to pursue in PhD programs, master programs and funding programs (eg. norforsk), to build education programs that combine the competencies of Universities and industry. There is a critical need for academic programs to partner with those who have the necessary competencies. Academic education programs in this space must take in many competencies, including but not limited to HTA, quality assurance, regulatory affairs, reimbursement/Health Economy, GMP facilities and manufacture. It is lazy to only teach what you know. We must have intersectorial, national and international collaborations. This Nordic education group has already identified synergies between a planned Novo Nordisk Foundation education and workforce gap analysis that will be combined with that of the ATMP2030 educational status analysis.
image from left: Marie Jönsson, Johan Flygare, Mattias Magnusson, Maria Kraft, Anita Kavlie
Clinical, lead by Hannah Karlsson. Since the Nordic countries, from an international perspective, are all small we need to see the Nordics as one market. Our collective population will provide a larger patient base more attractive for international clinical trials and provide us with greater possibilities to provide ATMPs for our patients. We are already collaborating and have several networks established where healthcare for children is a good example of collaboration that we could apply also to other patient groups, providing best possible care for these patients. There is also a need to show what each country can offer, in the Nordic setting, and we need to consider how this can be communicated. Showcasing out experiences, capabilities and resources at hand and communicating our Nordic collaboration model. It is also important to understand to what extent these therapies reach the patients. We know which ATMPs that have been approved and recommended in the different Nordic countries but not the extent of their use. Finding a simple solution to gather this information and share it would be relevant for all stakeholders in the Nordic countries.
image from left: Hannah Karlsson, Kristina Levan, Ewa Ellis, Hlif Steingrimsdottir, Johan Hyllner