Poland to Reimburse Cost of Spinraza to Treat SMA, Ending Lengthy Struggle

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by Larry Luxner |

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Spinraza paid for in Poland

Dorota Raczek and her daughter, Emilka, who has SMA type 3, at the 2018 SMA Conference in Warsaw, Poland. (Photo by Larry Luxner)

Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless of age or disease severity — ending a year-long struggle by the country’s nonprofit Fundacja SMA to gain access to one of the world’s most costly drugs.

The surprise announcement came during a Dec. 17 Polish-language radio program with Health Minister Łukasz Szumowski. It followed an intense media campaign by the Warsaw-based patient advocacy group, as well as an online petition started by Małgorzata Rybarczyk-Bończak — the mother of a teenager with SMA type 1 — signed by 67,000 people.

SMA patients and Poland

Yndhira Ramírez and Lukasz Szczepara, with their daughters Abril, 11, and Gabriela, 6. (Photo by Larry Luxner)

“This was the best Christmas gift we could hope for,” said Yndhira Ramírez, whose 6-year-old daughter, Gabriela, has SMA type 1. She and her husband, Łukasz Szczepara, have been making the difficult, expensive trip every four months by ambulance from their home in Zawiercie, Poland, to Belgium so their daughter could get her Spinraza injections.

Each year in Poland, some 50 babies are born with SMA — half of those type 1. Poland, whose population of 38 million makes it the largest EU member nation in Eastern Europe, is home to an estimated 700 to 800 people with SMA.

The treatment — which on the U.S. market retails for $750,000 the first year and $375,000 each year thereafter — will be available in Poland starting Jan. 1, when it will officially appear on the Health Ministry’s website as a reimbursable drug.

Patryk Marczuk, the scientific information coordinator at Fundacja SMA, is the father of 6-year-old identical twins — Gabriela and Patrycja — both of whom have SMA type 3.

“We’ve been celebrating,” he said by phone from Warsaw. “The Polish SMA community is really happy that the Ministry of Health and Biogen finally came to an agreement, and that they put the patients above money. But this wouldn’t have been possible if not for the involvement of the whole SMA community in Poland.”

Secret talks led to agreement

The final cost of the agreement to Polish taxpayers may never be known, because negotiations between the Polish Health Ministry and Biogen — which manufactures the injectable medicine — took place behind closed doors.

AVXS-101 and SMA

Hundreds of SMA patients and their caregivers gather in Warsaw in 2018. (Photo by Larry Luxner)

It now brings to 24 the number of EU member nations that have agreed to pay for Spinraza in one form or another. Germany and Italy, for example, offer the therapy for all types of SMA. Four EU member states do not fund Spinraza: Estonia, Ireland, Latvia and the United Kingdom.

Several months ago, British SMA patients and their families organized protests in London and Manchester to voice anger over a draft recommendation by the independent, government-funded National Institute for Health and Care Excellence (NICE) not to provide state funds to reimburse for Spinraza on the grounds that the drug wasn’t cost-effective.

Polish regulators came to a different conclusion.

“For the last couple of years, we’ve been fighting for this, and finally it’s coming to an end,” Marczuk said, estimating that between 10 and 20 families have already left Poland for other EU member states because they couldn’t get Spinraza at home. “I can only imagine they’ll be coming back, unless they’re happy wherever they’ve settled. But from what I’ve heard, most of the families that emigrated are not really happy.”

‘This will change our lives’

Ramírez, whose daughter had been receiving Spinraza in Belgium through Biogen’s Extended Access Program — a temporary “compassionate use” arrangement — now takes Gabriela to the Polish city of Gdansk for the treatment.


Dorota Raczek and her daughter, Emilka, who has SMA type 3. (Photo by Larry Luxner)

“I still don’t believe it,” she said. “Belgium was so far away, and even though we didn’t have to pay for the Spinraza, we did have to pay about €2,500 [roughly $2,900] for the hospitalization, the ambulance there and back, and hotels. Now she’ll get Spinraza through social security, and we won’t have to pay anything at all.

Dorota Raczek has an 11-year-old daughter, Emilka, with SMA type 3. For now, the girl is getting only physiotherapy — no medications of any kind. But her prognosis without Spinraza is poor; girls with SMA often lose the ability to walk by age 12 or 13 because of hormones linked to puberty. For this reason, Raczek and her husband were seriously thinking of moving to Germany so her daughter could access the medication. Now that won’t be necessary.

“It’s very good news. This will change our lives,” she said. “This will allow us to stay in Poland. Maybe my daughter will get Spinraza in two or three months, and with this medicine, I hope she will never stop walking.”