After Brexit, the UK is looking for ways to get ahead of the EU, which could be good news for people with rare diseases.
People with devastating rare diseases like cystic fibrosis or Duchenne muscular dystrophy might be able to live better lives with new cell and gene therapies. They can fix genetic mistakes that are passed down and cause disease or reprogram a patient’s own cells to treat a long-term illness.
It’s a field that has turned medicine on its head, moving away from drugs designed to treat large groups of people and toward one-time therapies that are tailored to each person’s body. Some of these therapies even offer the hope of a cure. This paradigm shift has caused a lot of almost impossible problems for health care systems, like how to pay for the most expensive therapies in the world.
All countries face these problems, but the U.K. hopes that Brexit will make it easier to deal with them.
“After Brexit, the U.K. is trying to compete with the U.S. and Europe by saying, “Let’s speed up everything and get as much clinical trial research as we can.” “It’s all about competition,” said Jennifer Lee, who is in charge of market access at the drug company Janssen.
One way to speed up the process of getting new cell and gene therapies to patients is to get all the authorities who will be involved in evaluating the drug together as soon as possible.
The process, which is called the Innovative Licensing and Access Pathway (ILAP), is meant to help show that cell and gene therapies are safe and work. That is, figuring out what to measure and how to measure it from what is likely to be the only clinical trial of the drug’s effectiveness in a small number of people.
Because the diseases that these therapies are meant to treat are rare, trials are done on a much smaller number of people than usual for a new medicine. The information from that one small trial will be used to decide how to go about getting the drug licensed. So, it has to convince regulators of medicines, show that these therapies add a lot of value to patients, and give a basis for price and reimbursement talks.
All the groups that evaluate the drugs need to agree on this information if a gene therapy is going to get on the market and stay there. And the market for these very new therapies is failing in Europe right now.
Since 2009, when the first therapy of this kind was approved for use, 21 products have been approved for use in Europe. Six of these have since been taken off the market, not because of safety or effectiveness concerns, but because of problems with pricing and insurance coverage.
Simone Boselli, who is in charge of public affairs for Eurordis, said that the group is looking at practices in the U.K. that “could have a big impact.” In addition to ILAP, the U.K. has set up a new fund for innovative medicines and is working to make sure that the National Health Service has access to drugs for rare diseases.
Europe is falling behind other parts of the world. From January 2014 to June 2019, the number of clinical trials for cell-, gene-, and tissue-engineered therapies grew by only 2% per year. In the United States, that number is 36%, and in Asia, it is 28%.
Paolo Morgese, director of EU market access at the Alliance for Regenerative Medicines (ARM), said there are many reasons for this. These include Europe’s strict rules on genetically modified organisms, problems setting up trials, problems with getting paid, and a slowdown in investment compared to Asia and the U.S.
Also, he said, the U.K. has “more flexible rules” for clinical trials and GMOs, which has “created a more thriving clinical trials environment.”
But things aren’t all bad for the EU.
Andrzej Rys, director for health systems, medical products, and innovation at the European Commission’s department for health and food safety, DG SANTE, said that Brussels is currently working on a change to its GMO rules, which apply to some cell and gene therapies.
In January, there were also new rules about clinical trials. These are meant to make it easier for researchers in the bloc to do clinical research. Rys said that it’s too soon to tell if it’s having a big effect on applications, but that the signs are good.
There are also other places where Europe could take the lead. The bloc set up a group of 24 specialist rare disease networks five years ago. These networks have experts in areas like bone disorders, childhood cancer, and immune system problems. They share the latest information across more than 1,600 centers and work together to learn more about and find better ways to treat these terrible diseases.
Maurizio Scarpa, director of the regional coordinating center for rare diseases at UDINE University Hospital in Italy, said that there are high hopes that these networks can be expanded to help research in the field and connect universities with startups.
He said that since most cell and gene therapy studies are done at these facilities, they can help make sure that all the authorities are happy in the future.
“Now the networks are ready… to really take part in these talks,” he said. Scarpa, who is also the coordinator of the inherited metabolic diseases network, said, “We have all the tools, but now we need directions.”
And finally, more and more people in the European Parliament want to use one of the most important changes in health care caused by the pandemic: Europe’s buying power, which it used to get COVID-19 vaccines and treatments.
With therapies costing up to €2 million per dose, this is a move that has wide support, and even the industry seems to be coming around to it, at least for ultra-rare diseases that only affect a few people each year in Europe.
But it’s still not clear if the EU will push this idea forward or not.
“To be honest, I can’t think of a way this could work right now,” said Rys at DG SANTE. But for the COVID antiviral remdesivir, the Commission “did buy medicines for the first time… directly from our budget.” Rys said, “So, if you say it’s impossible, it was possible.”