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Canadian children with a rare and often fatal neuromuscular disorder are about to gain public funding for one of the world’s most expensive medications.

The group that negotiates confidential drug discounts on behalf of the provinces and territories has reached a deal for Spinraza, the first drug to treat the underlying genetic cause of spinal muscular atrophy (SMA), a disease that in the worst cases kills children before their second birthdays.

Pediatric neurologists and the families of children with SMA have been watching closely to see how the Canadian health-care system would handle Spinraza, a case that highlights the trade-offs governments face when they’re asked to pay steep prices to improve – and even save – the lives of children.

“We’re very relieved,” said Nicole Lamont, the mother of a two-year-old girl who has been receiving injections of the medication. “It’s a very positive thing for the whole SMA community, not just our family. It’s something these kids really need.”

Nicole and Lilah Lamont on Oct. 3, 2018, in Essex, Ont.Dennis Pajot/Globe and Mail

The new agreement is the culmination of a process that is supposed to take the politics out of sensitive drug-funding decisions. The process includes an expert committee’s review of the cost-effectiveness of all new drugs, including Spinraza.

The drug is considered a significant breakthrough for a disease that is believed to affect several hundred Canadians. Researchers stopped two randomized-controlled trials of the drug early because Spinraza worked so well they could not in good conscience keep injecting half the participants with a placebo.

The pan-Canadian Pharmaceutical Alliance (pCPA) cut a deal with Spinraza’s maker, Biogen, under which provincial and territorial drug plans will pay an undisclosed lower price for the drug and cover only a narrow subset of patients with the severest incarnation of SMA. The pCPA always keeps confidential the discounted prices it negotiates with brand-name drug makers.

Biogen has agreed – for now – to foot the Spinraza bill for some other SMA patients while the expert committee re-evaluates the drug for patients with less-severe forms of the disease. The drug comes with a sticker price of $708,000 for the first year of treatment and $354,000 for subsequent years.

That expert committee is overseen by the Canadian Agency for Drugs and Technologies in Health (CADTH,) an independent, government-funded organization that reviews the evidence and evaluates the cost-effectiveness of new drugs, then makes recommendations about whether those drugs should garner public funding.

SMA is an inherited, progressive condition in which a genetic mutation renders the body incapable of producing enough of a protein critical to the development of motor neurons in the spine.

In the worst cases, called Type 1, babies have muscle weakness from the get-go, can’t sit up and usually die before they turn 2.

Patients with types 2 and 3 – whose symptoms can vary widely – are often diagnosed when they are older, and can wind up in a wheelchair for life.

Spinraza, which is injected into the spinal fluid, is a manufactured bit of genetic material that tricks a partnering gene into producing more of the insufficient protein, stopping or even reversing the decline of some patients' motor function. Health Canada approved the drug in 2017.

Ms. Lamont’s two-year-old daughter Lilah has Type 1 SMA. She spent most of her first year in the intensive care unit of a London, Ont. hospital, incapable of movement save for the wiggling of her fingers. Doctors predicted she wouldn’t live to see her first birthday.

Eager to have Lilah serve as a flower-girl in her wedding, Ms. Lamont, now 26, and Lilah’s father married in the chapel at London Health Sciences Centre. The nurses and other hospital staff who had come to know and love the family decorated Lilah’s wheelchair and carried her medical equipment down the aisle.

Less than a month after her parents' wedding in October, 2016, Lilah received her first injection of Spinraza, paid for by Biogen. About an hour later, “We saw a twitch in her leg,” Ms. Lamont said. “We freaked out.”

“After each injection, more and more things kept happening. Now she can fully move her legs. She’s just starting to lift her heels off the bed.” She can hold her head up for as long as a minute, and is now able to breathe on her own for an hour and 10 minutes every day, Ms. Lamont said.

For patients who have Type 2 or 3, Biogen has set up a steering committee of Canadian pediatric neurologists who will decide on a case-by-case basis who should qualify for free Spinraza from Biogen until the CADTH releases a new recommendation late this year or early next.

Marina Vasiliou, the vice-president and general manager of Biogen Canada, would not say what the company would do if the CADTH again recommends against funding Spinraza for children with SMA types 2 and 3. “We do not believe that’s a possibility,” she said, emphasizing the strength of new evidence in the drug’s favour.

However, Trevor Richter, the director of the CADTH’s Common Drug Review, which oversees the expert committee that makes drug-funding recommendations, sounded a note of caution. When the committee made its first recommendation last year, it concluded "there was too little evidence, or there were issues with the evidence, or there was no evidence at all,” for Spinraza’s effectiveness in children with less-severe cases.

“The coverage for Type 1," he said, "reflects the fact that that’s where the best evidence is for the drug.”