At first glance, medicine is paying off just in time.
Europe’s population is aging, and by 2100 nearly a third of the population will be over 65 years old. The big challenge of this century is how to keep people healthy.
At the same time, the last decade has brought personalized medicine within reach. Cellular and gene therapies tailored to individual patients can cure disease, from correcting remnants to training the immune system to kill cancer cells.
But with a price tag of up to 3 million euros per treatment, the medical system is not designed to absorb this kind of blow.
The question now is whether Europe can square the yen and make the most of new treatments. Or will budget constraints keep these medical advances out of reach?
show me money
To date, the European Commission has approved 25 cell and gene therapies for use in Europe.
These treatments involve the careful manipulation and growth of human cells and are given as a one-time treatment, sometimes as a cure, for rare diseases and certain types of cancer that are debilitating and often fatal. Novartis’ Kymriah, a CAR-T therapy that sends genes into the body to instruct the immune system to attack and kill blood cancer cells, is one such example.
These medicines are often tailored to a single patient and therefore very expensive.
Novartis’ Zolgensma, a gene therapy for children with spinal muscular atrophy, was approved in May 2019 (and approved in the EU in May 2020) for a one-time dose at list price in the US. was $2.1 million. It has since lost the title of the world’s most expensive treatment to CSL Behring’s hemophilia B gene therapy Hemgenix. Hemgenix is a one-time injection that costs $3.5 million for his single dose.
Matthew Durdy, chief executive of the Cell and Gene Therapy Catapult, said the high initial cost of building the first manufacturing facilities for these types of treatments has contributed to the high prices., An independent not-for-profit organization that brings together academia, industry, health services and government to deliver these products in the UK
But those costs will come down, as will all new technology, from mobile phones to laptops, he said. For that to happen, the health system needs to start using cures. Without a market, competitors would not follow, and without competition, prices would not come down.
But that is easier said than done.
Finding prices that are affordable for health payers and profitable for businesses “It’s a big, big challenge,” said Tim Hunt, chief executive of the Alliance for Regenerative Medicine (ARM), which represents the developers of these therapies.
And that is what has forced companies to close some EU operations and encouraged investors to step up, he said.Seven out of 25 cell and gene therapies approved in Europe have already been withdrawn by pharmaceutical companies From the EU market.
Since 2018, the EU cell and gene therapy market has grown by only 11% compared to 43% in the US and 531% in China.
As a result, the EU According to Hunt, patients are missing the next generation of treatments.
One way to get things back on track is to provide data showing that a particular treatment works, justifying the high initial cost. However, obtaining this data can be difficult.
This is because, for example, it takes years to prove that a drug prevents progressive disease. I’m here.
Therefore, to approve these treatments, Lydie Meheus, managing director of the Cancer Foundation, a non-profit research organization, said Europe is steadily “lowering the bar” of clinical evidence.
This will allow more treatments to be approved, but Lack of data poses a significant problem for insurers. If resources are particularly scarce, if staff are on strike, or if services are still grappling with the backlog of the pandemic, the health system will have to take huge financial risks.
Yannis Nassis, CEO of the European Social Insurance Platform (ESIP), said that “we really need to rebalance the scale when it comes to evidence generation” for these types of products. Limited data and high prices are a “huge challenge” for payers, he said.
glass half full
One hope lies in Europe’s 2021 Health Technology Assessment (HTA) regulations.
From 2025, all cell and gene therapies will receive a single EU assessment for the value they add to patients and health systems, hopefully eliminating the need for 27 duplicate reviews. This recommendation is used by national payers in price negotiations with developers.
Companies also hold joint meetings with the European Medicines Agency and the European HTA Coordinating Group to come up with the best clinical trial designs that provide data not only on safety and efficacy, but also on added value.
And technology is advancing rapidly. While most cell and gene therapies involve taking patient samples to tailor treatments, the ultimate goal is to provide more products developed without the need for patient samples. It is intended for
“Many companies are trying to focus more on in vivo gene therapy. [where the genetic changes happen inside the body as opposed to in a lab] Stephen Majors, Head of Communications at ARM, said:
This cuts production costs, reduces the need for the intensive process of taking a patient’s cells and sending them to another location for modification, and uses a cold chain storage infrastructure, he said. .
And payers may be more willing to pay if more long-term data bear fruit on curative efficacy. .
For Durdy in the British Catapult, these types of treatments save money in the long run. He said it could save up to $12 million in medical costs.
In the future, he imagined, payers would think: But if all of these treatments come to fruition, I’m going to change the way we do healthcare.”