Launching the Novartis cell and gene therapy business has been like building a start-up, according to Dr Emanuele Ostuni.
Ostuni is the head of cell and gene therapy for Novartis oncology in Europe - he has overseen the organisation's commercial launch of CAR-T therapy KYMRIAH (tisagenlecleucel).
He has a somewhat different background for a commercial head as a scientist with a PhD in chemistry from Harvard University.
Ostuni is still focused on the rapid construction of an organisation to oversee the country-by-country launch of KYMRIAH across Europe – a process that seems to share many of the same challenges experienced in Australia.
He told BiotechDispatch "it really is not a joke" when he says the cell and gene therapy team he now leads started with just two people.
"That was exactly two years ago, to the day, and since then we have had to build the whole infrastructure for delivering the product, sourcing it, shipping it back and forth, and then building the teams on the ground working with physicians, hospitals and our market access teams."
He said the decision was made to create an organisation focused on cell and gene therapy rather than embedding the team in an existing Novartis franchise. "We wanted to have people who live and breath these products so we can go very deeply."
"We are now starting to see real scale," said Ostuni, pointing to 65 KYMRIAH treatment centres across Europe and reimbursement in 15 countries.
According to Ostuni, Australia is not unique in having to deal with the challenge of accommodating a completely new therapeutic technology. Each market is managing the issue in its own way, said Ostuni, often underpinned by legal frameworks constructed based on medicines that "can be shipped around in a box".
"In some countries, we have had to work locally to adjust laws because you may only be able to get an approval if you have a box of product in country. We cannot make a box of KYMRIAH unless we have the patient already there."
Ostuni said the country-by-country complexity is a reality but worth it given the potential benefit to patients.
"The systems are willing to support it, based on a legal and reimbursement perspective, and this reflects an agreement on the need for a positive outcome.”
Ostuni said he could not point to a single country that has done everything well when it comes to KYMRIAH but that this reflects the complexity.
"We have had countries where qualifying and training treatment centres has been very smooth but reimbursement has been challenging. In other countries, we have had a smooth path to reimbursement but the treatment centres have been more challenging.
"The framework is different depending on the country," he said.
He said Australia’s challenge of funding across jurisdictions – with shared responsibility between the federal, state and territory governments – is also an issue in Europe.
“Europe is even more fractured. In some countries, governments have said they will just manage access out of one fund and the regions fall in line with that approach – it is expeditious and practical.
“For us, it means we avoid negotiating with every region, which can become very time-consuming.”
On the future, and in the context of KYMRIAH's extensive clinical trial program, Ostuni said he would be surprised if CAR-T therapies were not being used more extensively in the future given the number in development across the industry.
"I would be very surprised," he said. "In five years, we will probably have a better data set on what is working and not working in solid tumours. The early data shows promise but more may be required."
Ostuni said geography is an issue to the extent it "helps" to have patients within a certain distance of treatment to avoid too much travel.
"Having this therapy administered in a village hospital is not, at this point, possible," he said. “The physicians tell us patients are better off when treated in a centre that does 10, 15 or 20 per year, compared to one that does five. However, from the perspective of reaching patients, for us that is the least issue because we use a cryo-preserved product and ship anywhere.”
He continued, “We have only been in the market for a year. Some countries are just coming and some have been there for 11 or 12 months. I am really curious about how the early launch countries evolve and what we can learn from them that we can share with the new countries.
“The rate of adoption seems to be faster than expected and I am looking forward to having more data from the registries and clinical experience the physicians have with KYMRIAH to see how the product does in the real world compared to the trials – that is really exciting.”
BiotechDispatch attended the Novartis R&D global media day with the company's assistance.