Europe eyes arrival of worlds priciest drug

Matildes parents managed the impossible.

Over two weeks last summer, they fund-raised €2.5 million to get the toddler a life-saving medicine thats not yet authorized in Europe.

The little girl was lying in serious condition in intensive care when her parents reached the €2 million threshold.

“We were really focused on giving her this, no matter what,” said her mother, Carla Martins.

Matilde Martins Sande was diagnosed with the most severe form of spinal muscular atrophy (SMA) in her second month of life. A rare genetic disease affecting fewer than one in 10,000 people in the EU, it causes muscles in the body to deteriorate, leading to breathing problems. Most children die after a couple of years.

“We are talking about the life of a child, about the future of a family. Thats obviously bringing a lot of pressure along” — Belgian Health Minister Maggie De Block

That fate was something Matildes mom couldnt accept.

She found out about Zolgensma, an innovative therapy authorized in the U.S. in May, which is the closest to a cure the world has seen so far. Listed at €1.9 million, its also the most expensive therapy ever produced. The European Medicines Agency is still assessing the drug, and its expected to recommend its approval in Europe in early 2020.

Crowdfunding campaigns like the one organized for Matilde have played out in recent months across Europe. There was Pia in Belgium, Kris in Slovenia, Panagiotis-Rafael in Greece, Zente and Levente in Hungary, Antonis in Cyprus and Annabel in Estonia.

Fueled by intense press coverage and social media, the campaigns allowed these children to access the therapy. But theyve also put pressure on governments to pay for the drug even before its approved in Europe.

A spinal muscular atrophy (SMA) patient walks with a bionic exoskeleton at a Barcelona hospital | Pau Barrena/AFP via Getty Images

“We are talking about the life of a child, about the future of a family. Thats obviously bringing a lot of pressure along,” Belgian Health Minister Maggie De Block told POLITICO. Belgium, along with Estonia, Hungary, Greece and Cyprus, has so far resisted the pressure to pay.

By contrast, Portugal, France and Germany decided to pay under provisional schemes allowing patients access to therapies for life-threatening conditions, even if theyre not approved in Europe.

But in Germany, the intense pressure has also set off alarm bells.

Health insurers, university hospital doctors and the German federal health technology assessment body GBA wrote to the countrys health minister, Jens Spahn, earlier this month to ask for a law to address this question.

Securing and financing high-quality therapies should be based on the state of the art in medicine and legal requirements, “not depend on press campaigns,” they write.

The signatories also warned that if Zolgensma is reimbursed as a result of public pressure, drug companies could use it as a blueprint to get new drugs onto the market before authorization. That could undermine the EUs drug approval system, as well as Germanys health technology assessment and reimbursement procedures.

“I hope the debate will go forward and something fundamentally changes about how these prices are set” — Ellen De Meyer, baby Pias mother

As more advanced therapies, based on genes and cells, are expected to be approved in Europe in the next two years for even larger patient groups than those needing Zolgensma, this dilemma adds to the worries of national authorities already struggling with high drug prices.

Handling the pressure

Zolgensmas arrival on the EU market next year will happen at a time when Europe is already polarized on drug prices, with pharma companies and governments at times trading blame about the lack of patients access to life-saving therapies.

This risks creating a toxic environment, warned Martin Seychell, DG SANTEs deputy director general, speaking at a pharma event in Brussels in early November.

That has already played out in Belgium, where De Block has called the attitude of drugmaker Novartis — the parent company for AveXis, which came up with Zolgensma — cynical.

“You cannot start asking [for] prices that are not in proportion to the real development cost, just because the therapy happens to be groundbreaking,” she charged.

Rather, she said, pharmaceutical companies often get a great deal of assistance from public sources, with state-funded research and facilities, or favorable tax schemes, for example.

German Health Minister Jens Spahn | Maja Hitij/Getty Images

“Its very cynical to see that some companies consider society as nothing more than a cash cow,” she said.

Baby Pia, in Belgium, finally got the therapy in Antwerp. The family is expected to pay the full bill from the money they raised from people donating through text messages.

“I hope the debate will go forward and something fundamentally changes about how these prices are set,” Ellen De Meyer, Pias mother, told POLITICO.

“Its the most expensive medicine now, but theres no stopping them,” she added, calling for action from Belgian and European politicians to rein in prices.

Novartis, however, has justified the price by comparing it with how much the existing therapy approved in Europe, Spinraza, would cost to treat children with SMA.

Spinraza has to be administered throughout a patients life, and its price is in the hundreds of thousands of euros per patient per year. “In many ways, the introduction of Zolgensma offsets those costs of treating someone with SMA,” Dave Lennon, president of AveXis, told POLITICO.

More broadly, the arrival of these new therapies means that business as usual wont work anymore, according to Nathalie Moll, director general of Brussels-based pharma lobby EFPIA.

He said the company didnt offer the therapy for compassionate use in countries with crowdfunding cases “because if we approve one child, how do we say no to the others?”

Compassionate use schemes allow people with life-threatening conditions to access therapies that arent yet legally authorized, and if there is no existing therapy available. Such treatment is usually free.

Belgium asked Novartis to use it for Pia, but the company refused because Spinraza is already reimbursed in Belgium, according to Lennon.

“We do believe that its the governments responsibility to decide on how theyre going to allocate their health care funding and how they want to cover kids, especially pre-approval,” he said. This was a decision the Belgian government needed to take, “which they abdicated, which was unfortunate,” he added.

The fear of opening the floodgates for more very expensive therapy requests was also what drove Estonia to decline financing baby Annabels treatment in the U.S. In the end, the money her family raised paid for it instead.

“Had we decided to finance the treatment of gene therapy of this particular patient, we would have to treat all the future patients in a same manner,” Erki Laidmäe, head of the pharmaceuticals and medical devices department at the Estonian Health Insurance Fund, said via email. By extension, the “regular process of assessing effectiveness and value for money … of a new therapy” wouldnt apply anymore.

Unlike some of their fellow EU countries, Portugal, France and Germany decided to pay for Zolgensma under special schemes.

Moreover, given the growing number of expensive gene therapies, “the sustainability of an insurance system that has to serve eRead More – Source