Emanuele Ostuni, head of cell & gene therapy for Novartis Oncology in Europe, outlines how stakeholders across the biopharmaceutical value chain can surf the wave of innovation that breakthrough therapies such as CAR-T represent.
Waves and Innovation
CAR-T therapies are the frontrunners of a wave of amazing biomedical innovation
Over the summer, I watched waves and surfers, thinking about how they manage risk. It reminded me of my time in Munich where a small river through the city attracts die-hard surfers to practice. I ran into this group while working for the Sandoz Division of Novartis and their motto stuck with me: “if you can’t stop the waves, surf them”.
I joined the Oncology division of Novartis in 2017 to build the European commercial organization for our CAR-T therapy launch. I learned that waves are a good analogy for breakthrough innovation. In August 2017, this medicine was approved by the FDA in what the agency called a “historic action”, the “first gene therapy available in the US, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases”. European approval followed one year ago this month, also a first.
Even for experts the science behind this new approach to tackle B-cell malignancies seemed extraordinary. CAR-T uses the body’s own immune system to fight cancer. First, a subset of the patient’s white blood-cells, T-lymphocytes, are extracted from the blood. They are shipped to a sophisticated manufacturing site where experts alter their genome to equip them with a new receptor (the “Chimeric Antigen Receptor” or CAR), they do not possess naturally. CAR-T cells are shipped back to the patients to deliver their therapeutic effect over many months after only one infusion. Treatment occurs in highly specialized hospitals as it may cause significant side effects. The efficacy of the therapy was profound in children with leukemia and adults with diffuse large B cell lymphoma who had resisted to other treatment options. In clinical trials, a significant proportion of these terminally ill patients showed complete remissions over more than 24 months. The effect of CAR-T therapies on the medical community was comparable to a wave – it caused turbulences and excitement.
Innovation and Tradition: Waves Hitting the Shore
While the global stakeholder community celebrated the biomedical breakthrough of CAR-T therapies, it became clear that such one-time therapies with a potential long-term clinical benefit challenge traditional views, processes and systems. New solutions and approaches are required while managing the unpredictability that is created when such approaches are implemented. In spite of my personal experiences in biotech, growth markets, generics, and consulting I experienced many instances that stretched my agility and that of my team as we embarked on this journey.
In Europe, we built a new commercial organization and with it a network of qualified hospitals, operational and legal structures for the transport of the (native) cells to the manufacturing site and the transport of the (genetically modified) CAR-T cells back to the treatment centers. Although the therapy was explored in clinical trials within Europe, a new regimen needed to be established to protect the cell’s chain of identity throughout the process (if ever two bags of cells were wrongfully exchanged, two lives would be at risk) – a whole cosmos of data privacy questions arose in light of Europe’s regulations. Last but not least, appropriate pricing and reimbursement processes needed to be co-developed with all stakeholders in each country – a highly complex and demanding venture as Novartis’ CAR-T therapy became the first ever reimbursed cell and gene therapy in many markets.
The first months of that journey often felt like the moment a wave hits the shoreline, when sand and stones absorb parts of the water’s energy. The vast amount of operational, legal and societal issues we faced was astonishing and we realized, that in many respects traditional approaches of healthcare systems and policies are not prepared for the advent of cell and gene therapies.
Surfing the Waves
Today’s administrative systems are not set up for one-time interventions that unfold their therapeutic potential over many months, potentially years; however, the first steps were taken in a short amount of time. With a price of over 300,000 EUR, today’s CAR-T therapies seem very expensive on a first glance. However, in terms of QALY, their value is higher than in most other cancer therapeutics – as acknowledged by many HTA bodies.
CAR-T therapies are the frontrunners of a wave of amazing biomedical innovation. We may be entering a new phase of individualized medicine as cell and gene therapies offer hope to many patients suffering from incurable diseases. These therapies also cause concerns about the sustainability of healthcare systems. Innovation and tradition seem to collide.
In nature, something new, beautiful emerges from the energetic collision of water and shoreline: a surfer’s paradise, if well-managed. Together, innovators and regulators can create an amazing future for patients and society. We need to join forces to surf the waves and manage their risks. I look forward to sharing some thoughts and some surfing recommendations over the next months with the community of PharmaBoardroom. I welcome the ensuing dialogue to ensure cell and gene therapies are adopted sustainably.
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