There is no better way to understand your customers and their challenges in this rapidly evolving space of “Advanced Therapies” than to spend time together and use it for long discussions on how to provide patients with access to these wonderful products and therapies. That’s why we took the opportunity to attend some key events in the last couple of months (the “Advanced Therapies Week Europe” in Lisbon in September, the “Cell Gene Meeting on the Mesa” in Carlsbad, and the “BSMA Annual Meeting” in Brussels in October) to meet many of the colleagues working in this field and we would like to share some thoughts and take aways from the discussions we had.
While the enthusiasm and believe in the potential of ATMPs/CGTs among the participants we talked to is unwavering, the unbound optimism has been reigned in. The main effects came from the reduced availability of capital and more challenging financing rounds as well as the frosty reception of a number of gene therapies in Europe which led to their removal from the market in the EU again. Both are serious issues and have rightfully been broadly discussed. Tim Hunt from the Alliance for Regenerative Medicine (ARM) took it up in his opening presentation of the Cell Gene Meeting on the Mesa and provided encouragement and outlined a fact-based approach to the discussion with payers about the unique value of these therapy options and why it is our job to find a way to provide patient access. Our feeling was that the industry will come together and address the concerns and align on an agreeable model for assessing the value and setting the price for new therapies to not impede patient access. Failure is not an option.
To look on the bright side, several providers could celebrate FDA approval of new therapies in 2023 while many others are moving from pre-clinical to clinical stages, raising hope for many new life-saving therapies in the future.
We have participated in a number of roundtables and discussions addressing key challenges that ATMP/CGT developers are facing in areas like regulatory affairs, supply chain planning & execution, manufacturing, digitalization projects, and patient access. In general, it was amazing to experience the openness and willingness to share lessons learned and best practice amongst the peers. For us, that is the best reason to believe that these products and therapies are here to stay, and the potential will be realized in the next couple of years. Yes, the treatment centers experience a “portal fatigue”, yes, scaling up manufacturing from 20 treatments in a year to 200 or 2000 is a real challenge and cannot be solved with the same approach you started with, yes, customs processing and import of these precious products is unnecessarily complex and can have many surprises, yes, you can start on paper, but don’t expect it to be easier to switch to a digital platform later (because if you don’t have time to do it now, are you really going to have more time later?), and we could go on and on, but these issues have been identified, are being discussed, lessons are being learned and shared and so the issues will eventually be solved, which is why we believe in the future of ATMPs/CGTs!
To give just one concrete example: Ori Biotech organized a workshop on the topic of “Moving Beyond the Status Quo: A Roadmap for Digitization” and invited a broad audience representing ARM, treatment centers, ATMP/CGT developers, CDMOs, a variety of solution providers, consulting service providers etc. The level of involvement, sharing, and joint discussion was extraordinary and if we had to pick, that workshop was the highlight of the whole event for us. This kind of collaboration and co-innovation in areas that we believe do not really provide much of a competitive advantage but will have a huge impact on patient access, should be the norm and updates on the progress deserve a prime spot on the agenda of the future events. This is a great approach and the right spirit to create the starting point for the much needed standardization of common processes within the industry.
One thing that we did not mention yet is the science: the progress is truly mind-blowing! While many challenges remain (a set of reliable and effective cell- (or at least organ)-specific delivery systems for (small) RNA anybody?), the presented developments are absolutely astonishing! The data presented by ARM in the “sector snapshot” shows that a large number of therapeutic developers is located in the APJ region, they for sure were underrepresented and we need to think about how to include them in our discussions, too!
We believe we will see real and meaningful progress on the regulatory (“accelerated approval”) as well as payer side (interesting innovative payment systems were discussed and presented) in the near future paving the way for all the therapies in development and we salute the trail blazers for that!
In general, our mission is to turn the ATMPs/CGTs products from being special and complicated to prescribe by health care providers, unfamiliar to the regulatory agencies, and difficult to access by patients, into just another category of products, but with amazing potential addressing patients’ needs that could not be addressed until now.
We owe it to the patients and their families!
Our journey doesn’t end here. We’re already looking forward to Advanced Therapies Week in Miami, where we can delve deeper into these conversations. Let's connect, share, and innovate together, your insights are invaluable!
We invite you to join us in shaping the future of ATMPs/CGTs. Come find us in Miami — let's transform challenges into opportunities, together.
State of the Union
While the enthusiasm and believe in the potential of ATMPs/CGTs among the participants we talked to is unwavering, the unbound optimism has been reigned in. The main effects came from the reduced availability of capital and more challenging financing rounds as well as the frosty reception of a number of gene therapies in Europe which led to their removal from the market in the EU again. Both are serious issues and have rightfully been broadly discussed. Tim Hunt from the Alliance for Regenerative Medicine (ARM) took it up in his opening presentation of the Cell Gene Meeting on the Mesa and provided encouragement and outlined a fact-based approach to the discussion with payers about the unique value of these therapy options and why it is our job to find a way to provide patient access. Our feeling was that the industry will come together and address the concerns and align on an agreeable model for assessing the value and setting the price for new therapies to not impede patient access. Failure is not an option.
To look on the bright side, several providers could celebrate FDA approval of new therapies in 2023 while many others are moving from pre-clinical to clinical stages, raising hope for many new life-saving therapies in the future.
Challenges and opportunities
We have participated in a number of roundtables and discussions addressing key challenges that ATMP/CGT developers are facing in areas like regulatory affairs, supply chain planning & execution, manufacturing, digitalization projects, and patient access. In general, it was amazing to experience the openness and willingness to share lessons learned and best practice amongst the peers. For us, that is the best reason to believe that these products and therapies are here to stay, and the potential will be realized in the next couple of years. Yes, the treatment centers experience a “portal fatigue”, yes, scaling up manufacturing from 20 treatments in a year to 200 or 2000 is a real challenge and cannot be solved with the same approach you started with, yes, customs processing and import of these precious products is unnecessarily complex and can have many surprises, yes, you can start on paper, but don’t expect it to be easier to switch to a digital platform later (because if you don’t have time to do it now, are you really going to have more time later?), and we could go on and on, but these issues have been identified, are being discussed, lessons are being learned and shared and so the issues will eventually be solved, which is why we believe in the future of ATMPs/CGTs!
To give just one concrete example: Ori Biotech organized a workshop on the topic of “Moving Beyond the Status Quo: A Roadmap for Digitization” and invited a broad audience representing ARM, treatment centers, ATMP/CGT developers, CDMOs, a variety of solution providers, consulting service providers etc. The level of involvement, sharing, and joint discussion was extraordinary and if we had to pick, that workshop was the highlight of the whole event for us. This kind of collaboration and co-innovation in areas that we believe do not really provide much of a competitive advantage but will have a huge impact on patient access, should be the norm and updates on the progress deserve a prime spot on the agenda of the future events. This is a great approach and the right spirit to create the starting point for the much needed standardization of common processes within the industry.
Future outlook
One thing that we did not mention yet is the science: the progress is truly mind-blowing! While many challenges remain (a set of reliable and effective cell- (or at least organ)-specific delivery systems for (small) RNA anybody?), the presented developments are absolutely astonishing! The data presented by ARM in the “sector snapshot” shows that a large number of therapeutic developers is located in the APJ region, they for sure were underrepresented and we need to think about how to include them in our discussions, too!
We believe we will see real and meaningful progress on the regulatory (“accelerated approval”) as well as payer side (interesting innovative payment systems were discussed and presented) in the near future paving the way for all the therapies in development and we salute the trail blazers for that!
In general, our mission is to turn the ATMPs/CGTs products from being special and complicated to prescribe by health care providers, unfamiliar to the regulatory agencies, and difficult to access by patients, into just another category of products, but with amazing potential addressing patients’ needs that could not be addressed until now.
We owe it to the patients and their families!
Join the Dialogue at upcoming Advanced Therapies Week
Our journey doesn’t end here. We’re already looking forward to Advanced Therapies Week in Miami, where we can delve deeper into these conversations. Let's connect, share, and innovate together, your insights are invaluable!
We invite you to join us in shaping the future of ATMPs/CGTs. Come find us in Miami — let's transform challenges into opportunities, together.
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