Key Takeaways
- Cell and gene therapies require changes to established “ways of working” in health systems, which can hinder their adoption, says a senior figure at Novartis.
- Pharma must work on the administration and formulation of gene therapies with the health system in mind, a senior figure from AstraZeneca says.
- NHS England’s head of innovative treatments invites companies to discuss their product plans early, to help integrate therapies into the health system.
Commercializing cell and gene therapies is notoriously challenging, and while pricing is often seen as a key problem, companies must also consider how their product fits in logistically with health care systems, experts in the advanced therapy space said during the Advanced Therapies Congress in London, UK, on 19 March.
“A lot of great products succeed, but a lot of great products fail too commercially…cell and gene therapies often require people to change their ways of working in the health care system, and we are all creatures of habit, and the bar for asking anybody to change their habit is very high,” said Joshi Venugopal, general manager and head of Europe at Novartis Gene Therapies, during a panel discussion at the congress.
He said that the advanced therapy medicinal product (ATMP) sector was going through a “Darwinian evolution,” where there would be a “survival of the fittest” and that not every company could have the same outcome, even if their product had good proof-of-concept evidence.
Venugopal used the example of inhaled insulin, which he said is “much more simple” than an ATMP, to explain his statement on cell and gene therapies.
“When you give a choice to the diabetic patient if they want to take an inhaled insulin or they want to take an injection, the vast majority of them say we would prefer an inhaled insulin… but inhaled insulin failed. It flopped,” Venugopal said.
He explained that this was despite market research pointing towards product success. It is believed that the product failure was due to regulators asking endocrinologists to perform lung function tests, which required the endocrinologists to purchase spirometry machines.
“That’s why a great product did not succeed, because the system was not ready for it, and people had to change the habit, and they had a lot of other alternatives. They were comfortable. They were giving insulin for a long time,” Venugopal argued.
The inhaled insulin’s failure is a lesson that cell and gene therapy manufacturers can learn from, he said.
“In our case, we are talking about newborn screening [and] a pay for performance model, you know… complex administration, logistics, supply chain, some of these therapies have complications and post-administrative care… therefore the bar has to be really high for somebody to change their habits,” Venugopal explained.
For this reason, he said, ATMPs must be focused, “at least for now,” on “very severe diseases with limited or no treatment options, ideally.”
AstraZeneca’s global director of innovative value and access strategy, global market access and pricing, Katja Berg said she “agreed with everything” that Venugopal stated, adding that companies “need to be focused, and it goes back to what the health system is set up to do today.”
There are specific disease areas for which cell and gene therapies will more easily adjust to the requirements needed to deliver these products, she said.
“From a pharma perspective, we need to be working on technological advances in not just the science, but things like the formulation, the administration advantages,” Berg contended.
Companies ‘Underestimate’ Complexity Of Products For NHS
Offering the perspective of a health care system organization, Ben Doak, head of innovative treatments at National Health Service (NHS) England, said that companies must “begin with the end in mind.”
“Always think about how the person is going to receive the product... Are they going to be in the hospital? Do they currently have their treatment at home? How is that going to work? Think about the practicalities,” he said at the congress.
Doak also urged companies to consider the impact of their product on pharmacy services and aseptics units.
“NHS aseptic units are under immense pressure, so taking up half a day to prepare one specific dose of one product is a significant impact for that particular hospital. Think about these very practical elements when you’re thinking about bringing the product to market, and engage early,” he said.
Doak said his team within NHS England “exists to help bring your products into the NHS.”
“Come and talk to us. What I’ve experienced is that large companies underestimate the complexities that these products bring to the service,” he said, adding that smaller companies also “may not know what the environment is like within the UK.”
