On the face of it, medical science has delivered at just the right time.
Europes population is growing older: by 2100 nearly a third of people will be aged 65 or over. A major challenge for this century is how to keep people healthy.
At the same time, the last decade has seen personalized medicines come within reach. Cell and gene therapies tailored to individual patients can cure diseases, from fixing misfiring biology to training the immune system to kill cancer cells.
But with price tags of up to 3 million per treatment,healthsystems simply arent designed to absorb these kinds of hits.
The question now is whether Europe will be able to square the circle and take full advantage of the new therapies. Or will budget constraints keep these medical advances out of reach?
To date, the European Commission has authorized 25 cell and gene therapies for use in Europe.
These therapies involve careful manipulation and growth of human cells to be given often as one-off treatments and in some cases cures for debilitating and often deadly rare diseases and certain types of cancer. Novartis Kymriah, a CAR-T therapy that delivers genes into the body to tell the immune system to attack and kill blood cancer cells, is one such example.
These medicines are often tailor-made for a single patient, making them extremely expensive.
Novartis Zolgensma, a gene therapy for children with spinal muscular atrophy, had a U.S. list price of $2.1 million for a one-off dose when it was approved in May 2019 (it was approved in the EU in May 2020). Since then, it has lost the title of the worlds most expensive therapy to CSL Behrings hemophilia B gene therapy Hemgenix, a one-off infusion that costs $3.5 million a dose.
Big upfront costs in building the first manufacturing facilities for these types of therapies play a role in high prices, said Matthew Durdy, chief executive of the Cell and Gene Therapy Catapult, an independent nonprofit that brings together academia, industry, health services and government to deliver these products in the U.K.
But these costs will come down, he said, just as they do with every new technology, from mobile phones to laptops. For that to happen, health systems must start using the therapies. Without a market, competitors will not follow, and without competition, prices wont drop.
But thats easier said than done.
Finding a price that is both affordable to health care payers and profitable for companies is a major and significant challenge, said Tim Hunt, chief executive of the Alliance for Regenerative Medicine (ARM), which represents developers of these therapies.
And its one which has pushed companies to shutter some EU operations and investors to hold back, he said. Seven of the 25 cell and gene therapies approved in Europe have already been withdrawn by drugmakers from EU markets.
Since 2018, the EU cell and gene therapy market has grown just 11 percent, compared with 43 percent in the U.S. and 531 percent in China.
Consequently, EU patients are losing out on the next generation of treatments, Hunt said.
One way to bring things back on track is delivering data that shows a particular therapy works and justifies those initial high costs. But this data can be hard to come by.
Thats because it takes years to prove that a drug prevents a progressive disease, for example. And in rare conditions, there are only a handful of patients to test out new therapies, limiting the evidence.
Therefore, to approve these therapies, Europe has been steadily lowering the bar for clinical evidence, said Lydie Meheus, managing director at the not-for-profit research organization the Anticancer Fund.
While this authorizes more therapies, lack of data creates significant problems for payers asking health systems to take big financial risks when resources are especially stretched, staff are on strike and services are still grappling with pandemic backlogs.
We really need to rebalance the scale when it comes to the evidence generation of these types of products, said Yannis Natsis, chief executive of the European Social Insurance Platform (ESIP). Limited data and high prices are a huge challenge for payers, he said.
One hope lies in Europes 2021 Health Technology Assessment (HTA) regulation.
From 2025, all cell and gene therapies will undergo a single EU assessment of the value they add to patients and health systems, hopefully ending the need for 27 duplicate reviews. This recommendation will be used by payers in each country in their pricing talks with developers.
Companies will also meet jointly with the European Medicines Agency and Europes HTA coordinating group to thrash out the best clinical trial designs that deliver data, not only on safety and efficacy but also on added value.
And the technology is rapidly progressing. While the majority of cell and gene therapies involve taking a patient sample and tailor-making a treatment, in time the aim is to deliver more products that are developed without the need of a patient sample.
A lot of companies are trying to focus more on in vivo gene therapies [where the genetic changes happen inside the body as opposed to in a lab] and off-the-shelf cell therapies that use donor materials, said Stephen Majors, head of communications at ARM.
This will lessen the production costs, curtailing the need for the intensive process of removing a patients cells, sending them elsewhere to be modified, and using cold chain storage infrastructure, he said.
And if the longer-term data bears fruit on curative benefits, payers may be more willing to pay. Some are already adapting payment models to spread the costs over several years.
To Durdy, at the U.K. catapult, these types of therapies will save money in the long run. Spending for example $2 million to $3 million on curing a hemophilia patient will prevent up to $12 million in lifetime care costs for that patient, he said.
He imagined that, in the future, payers would think along the following lines: Its going to blow my budget this year. But if all of these therapeutics come through, Im going to transform the way I do health care.
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Can Europe afford the next generation of medicines? - POLITICO Europe
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