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Europe eyes arrival of worlds priciest drug

Matildes parents managed the impossible.

Over two weeks last summer, they fund-raised €2.5 million ..



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

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Spain’s coronavirus incidence rate falls for the first time this year, but pressure on ICUs rises




The third wave of the coronavirus has pushed Spain to the breaking point. According to the Spanish Health Ministry’s latest report, released on Thursday, the country has started to flatten the curve of new infections, but it has done so at crisis levels, with pressure on hospitals, especially in intensive care units (ICUs), close to unbearable. This pressure continues to rise across almost all of Spain.

The last 10 months of the pandemic have shown that there is not one but various peaks in a wave. The first is the number of new infections. The second is the spike in hospital admissions, which tends to happen a week later, and takes a little longer to be reflected in ICU figures. The final peak, which indicates a change in trend, is the number of deaths. The Health Ministry added 515 fatalities to the official count on Thursday, a terrible toll that is likely to remain at these levels for days to come while Spain transitions from peak to peak.

The latest data indicates that Spain is starting to see the other side of the peak of new infections in the third wave. A week ago, the Health Ministry reported a record-high 44,357 new coronavirus figures. On Thursday, that figure fell to 34,899. It is also the first day this year that the national incidence rate has fallen: the 14-day cumulative number of cases per 100,000 inhabitants now stands at 890, down from 900 on Wednesday.

Hospital admissions fell for the first time this year on Wednesday, with the percentage of Covid-19 patients in hospital wards dropping to 24.10%, down slightly from 24.03% on Tuesday. This data point fell again on Thursday, although the drop was again only marginal. But pressure continues to mount in Spain’s ICUs, which are the last line of defense of the healthcare system. If they are overwhelmed, authorities may be forced to introduce tougher restrictions, such as home confinement. Making matters worse, the pressure on ICUs is rising amid the uncertainty over how the emergence of new, more contagious strains of the coronavirus, such as the B.1.1.7 variant detected in the United Kingdom, will impact the pandemic in Spain.

“According to the data we have, we can expect that it [the B.1.1.7 variant] will be the dominant one in Spain by the end of February or the first fortnight of March. This has some implications because the strain is more transmittable,” said Fernando Simón, the director of the Health Ministry’s Coordination Center for Health Alerts (CCAES), at a government press conference on Thursday. The health official previously said this would not happen until a later date.

Simón admitted that there are small areas in Spain where the new strain already accounts for 20% of cases, but said nationally this figure was “around 8%.” With respect to restrictions, the health official said that “the measures that must be taken” are the same for the new and old variants of the coronavirus. “More measures can still be implemented without modifying the state of alarm,” he said, in reference to the emergency decree that gives regional governments – which are responsible for managing the health crisis – the power to introduce measures such as perimetral lockdowns, but not home confinement.

The figures released on Thursday also do not reflect how the coronavirus situation differs between each of Spain’s 17 regions. Indeed, the fall in hospitalizations over the past two weeks is mainly due to improvements in two regions: Valencia (which, despite this, remains in a “very critical situation,” said Simón) and Catalonia, which reported 466 fewer occupied beds on Thursday than on Tuesday. In Castilla y León, Galicia and Andalusia, there continue to be more hospital admissions than discharges.

“There are 11 regions where the situation is stabilizing or on a downward trend, but this is not the same as a fall in the hospital occupancy rate. The pressure on hospitals will continue over the coming days,” said Simón.

Andalusia and Galicia, as well as Murcia and Valencia, are some of the regions that are being hardest hit by the third wave of the pandemic, with the situation even worse than what it was during the first wave.

The big problem continues to be in ICUs. On Thursday, the Health Ministry reported 97 more ICU admissions than on Wednesday, and no region has managed to clearly reverse the upward trend. In other words, the peak of ICU pressure is still to come. In more than half of Spain, Covid-19 patients occupy more than 40% of all ICU beds – a similar figure to all other diseases combined. In the Balearic Islands, Castilla y León and Extremadura, the occupancy rate is more than 40%; in Castilla-La Mancha, Catalonia, Madrid and La Rioja, it is more than 50%; and in Valencia, it is 63%, a record high not seen since April last year, during the first wave.

As experts warned, the source of the problem is that Spain entered the third wave – which started after the December 6 long weekend – before the second wave was over. This meant that the ICU occupancy rate of Covid-19 patients, which was below 15% in October when the number of new cases began to rise, was already at around 30% in some regions when the third wave hit.

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Covid-19 vaccine doses going to waste in some of Spain’s regions due to unsuitable syringes




Spanish regions such as Andalusia, Catalonia and Madrid are wasting thousands of doses of the Pfizer-BioNTech Covid-19 vaccine by not injecting patients with the sixth dose contained in the vials. Since January 8, when the European Medicines Agency granted authorization, the five initial doses contained in the vials are now officially six, increasing the number of potential recipients.

But this change has not been exploited by a number of Spain’s territories, who are in charge of their own vaccination campaigns. Speaking on Thursday in the regional parliament, Andalusia’s health chief Jesús Aguirre admitted that this was the case. “You could take out up to five doses and there was a wee drop left, and that little bit is used to administer that sixth dose,” he said. “But we are talking about 20% more vaccinations.”

The Andalusian regional government has blamed the failure to use this dose on a “deficit of 0.1-millimeter syringes,” the type that is usually used to inject insulin or in pediatrics, and has promised to solve the issue. The extraction of the sixth dose is more difficult with the 0.2-millimeter syringes also being used to administer the vaccines.

Based on the information offered by Spain’s regions, it is impossible to calculate how much of the vaccine has been wasted. Catalonia, for its part, has admitted it is only using the sixth dose from “two-thirds” of the vials. The region has administered more than 165,000 doses of the Pfizer-BioNTech vaccine so far, which would suggest wastage of around 10,000 doses.

A similar situation is playing out in Madrid, which on Thursday announced the purchase of 280,000 syringes in order to take advantage of this sixth dose. This is a low amount, however, compared to the millions of suitable syringes that have been purchased by regions with a much smaller population, such as Murcia and the Balearic and Canary Islands. The Madrid region has stated that in “the majority of cases” it is managing to use the sixth dose, but it has offered little extra information.

Aragón, which purchased 1,870,000 syringes in September, says that it has managed to use “100% of the first five doses from each vial and 81% of the sixth, which is 97% of the total.” Castilla-La Mancha, meanwhile, puts the percentage at 90%, while Asturias, Castilla y León and the Basque Country opted not to provide figures when consulted by EL PAÍS. The rest of the regions, however, stated that the use of the sixth dose is widespread across their territories.

Meanwhile, Spain’s regions are having to improvise a response to news that politicians, retired health workers and relatives of medical staff are jumping the line and receiving the Covid-19 vaccine ahead of their time. Currently, Spain is still in the first stage of the vaccination campaign, which is focussing on senior home residents, their carers, front-line medical workers and adults with serious disabilities.

The Health Ministry and the country’s regions have been working on the vaccination plan since September, but the scheme has failed to adequately set out how the order of the vaccinations should be supervised, and whether there should be some kind of sanction or punishment for those who jump the line or indeed what to do with unused doses.

The excuses offered so far by those who have got the vaccine early range from “wanting to boost confidence” in the vaccination, to claims of making use of “leftover doses.”

EL PAÍS has requested information from the country’s health departments as to the measures being taken against these irregularities. The majority have not responded. Extremadura, Madrid and Navarre claim that they are observing protocols, while Valencia, La Rioja, Andalusia and the Basque Country explain that they are cross-referencing databases: anyone who is found to have been given the vaccine despite not being on the list of phase one recipients must offer an explanation.

Health Minister Salvador Illa said this week that “proper planning” must be put in place to avoid wasting injections, adding that any sanctions for breaking protocol are the responsibility of the regions. For now, none of Spain’s regional governments have confirmed whether or not they have levied sanctions against offenders.

No second dose

One decision that has been made, however, is that Valencia is opting not to administer the second vaccine dose to anyone who has jumped the line. This includes, for example, the Socialist Party (PSOE) mayors of the municipalities of El Verger, Els Poblets and Rafelbuñol, who were found to have skipped the protocol and were given the vaccines in senior homes.

The PSOE premier of the region, Ximo Puig, has taken the decision not to administer the second dose to the 150 to 185 people thought to have jumped the line. They will instead be fully vaccinated when it is their turn, he insisted.

But Fernando Simón, the director of the Health Ministry’s Coordination Center for Health Alerts (CCAES) and the government’s most visible face during the coronavirus crisis, criticized the approach. “I understand that people who were vaccinated when they shouldn’t have been should admit their responsibility,” he said when asked by reporters about the incidents. “But from my point of view, I believe it would committing two errors: vaccinating people ahead of time and not administering the second vaccine.”

The issue is whether it is worse to waste the first dose by not giving the second one 21 to 42 days later, as stipulated by the manufacturer, or to fully immunize those who jumped the line. The Pfizer-BioNTech vaccine needs two doses for the recipient to develop 94% immunity to Covid-19, according to clinical trials. The regional government has said that it respects Simón’s opinion but that this was “a matter of institutional credibility.” The aforementioned mayors who jumped the line have been suspended from the party and their local councils will be deciding whether or not they should be fired.


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Coronavirus cases in Spain officially top two million




The number of coronavirus cases recorded in Spain since the beginning of the pandemic officially exceeded two million on Thursday, amid warnings of difficult weeks ahead. According to the Health Ministry’s latest report, all data points clearly indicate that the pandemic continues to follow an upward trend. Thursday’s report – which also included figures from Wednesday as it was a public holiday (the Health Ministry does not release data on holidays or over the weekend) – registered 42,360 new coronavirus cases. The 14-day cumulative number of cases per 100,000 inhabitants now stands at 321, up from 296 on Tuesday. The number of Covid-19 patients in hospital and intensive care units (ICUs) rose by 1,514 and 134, respectively. Thursday’s report added 245 Covid-19-related deaths to the official toll.

But the Health Ministry does not believe the rising trend will impact the return to school, which has already happened in some regions, and will begin on Monday in others. When asked about the possibility of closing schools on Thursday, health official María José Sierra replied: “We do not recommend it.”

“It [schools] is one of the areas that have worked best, where there has been the most control. There were no outbreaks,” said Sierra, from the Health Ministry’s Coordination Center for Health Alerts (CCAES), who was standing in for the center’s director, Fernando Simón. The health official highlighted the enormous social and learning impact of closing schools and said that any new measures would target sectors that are hubs for contagions.

On the question of a new home lockdown, which has been introduced in countries like the United Kingdom, Sierra replied: “There are many measures to take before a strict confinement.” The health official defended the use of partial restrictions, like perimetral lockdowns and limits on social gatherings, which “proved their effectiveness” during the second wave of the pandemic. On November 9, Spain recorded its highest incidence rate when the 14-day cumulative number of cases per 100,000 inhabitants reached 529. The incidence rate began to fall after that date until rising again at the beginning of December.

It is important to note that Thursday’s report recorded a 25-point rise in Spain’s incidence rate even though the 14-day cumulative number includes three public holidays (Christmas, New Year’s Day, King’s Day), when there is typically underreporting. What’s more, fewer PCR and antigen tests have been carried out: while 140,000 tests were being done every day in the middle of December, between December 28 and January 3 – the last week with complete data – only 110,000 tests were administered in the entire week.

With fewer tests being done – most of which are carried out on patients with symptoms –, the positivity rate i.e. the percentage of tests that come back positive out of the total, has also risen. While 20 days ago this figure stood at 8%, it rose to 13.5% last week and to 15.6% on January 4. But experts warn the rise suggests that many chains of transmission are going undetected.

“It is a trend that is worrying us,” said Sierra on Thursday, who added that the weekly positivity rate exceeded 15% in five regions and 20% in three. “Among suspected cases diagnosed in primary healthcare centers, [the positivity rate] is already close to 30%,” she added.

According to Thursday’s report, Extremadura has the highest incidence rate in Spain, recording a 14-day cumulative number of cases per 100,000 inhabitants of 717. It is followed by the Balearic Islands (529) and Madrid (452). Asturias, Andalusia and the Canary Islands are the only regions where the incidence rate is below 200.

Hospital and ICU admissions – considered one of the most objective indicators of the pandemic – also rose significantly since Tuesday. A total of 14,543 Covid-19 patients are currently admitted to hospital, occupying 11.9% of all hospital beds. The occupancy rate in ICUs is 24%, with 2,307 patients in intensive care.

Sierra confirmed that Spain has detected nearly 50 cases of the new, more contagious strain of the coronavirus that was discovered in the United Kingdom, while many more cases are being studied.


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