Brothers have the same deadly disease but only 1 gets what their parents hope is life-saving treatment

Two young brothers, both with a deadly genetic disease, could face two very different futures because only one has accessto cutting-edge medication.

AndreLarocque, 8, and his youngerbrother, Joshua, 6, have cystic fibrosis.

The relatively rare disease leads to the destruction of the lungs and an early deathfor most people who haveit.

There is no cure. Once lung function is lost, there is no way to get it back.

"When I put them to bed at night, I hear Andre go to sleep peacefully and then I hear Josh laying awake in his bed coughing himself to sleep," motherSasha Haughian said during an interview from the family's home in the small Ontario town of New Tecumseth.

"I don't know how to describe the feeling of listening to that and knowing it's just gonnaget worse."

Andre is ona medication called Symdeko through a drug trial, but Joshua wasn't selected for thetrial and has no accessto two similar Health Canada-approved medications. That's becausethe Ontario government doesn't fund the drugs for the vast majority of cystic fibrosis patients and his parents can't afford to pay out of pocket.

"It's a progressive, fatal disease ... we havemedication right now that more than half the population [with cystic fibrosis] could benefit from, and we're not getting access to it," Haughian said.

About 4,200 Canadianshave cystic fibrosis. There are three gene-modifying drugs available Symdeko, Kalydeco and Orkambi that are believed to slow downlung damage caused by the disease.Other treatments only easesymptoms.

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Access to all three medications is severely limited.

Unlike many other countries, Canada does not have a special process for evaluating drugs for rare diseases. Clinical trials for such drugs are limited by their small pool of potential participants. This makes proving a drug's effectiveness more difficult which makes convincing provincial governments and private insurers to cover the cost for patients more unlikely, health advocates say.

"It's a heartbreaking situation ... These are symptoms of a larger issue, which is essentially our system was not built to evaluate and fairly assess drugs for rare diseases," saidKim Steele,director of government and community relationsforCystic Fibrosis Canada.

'Josh has nothing'

Haughian and her husband, Jamie Larocque, say Symdeko has been a life-changer for Andre since he startedtaking it in January 2018. While he can run around and play hockey with his friends, his younger brother's condition is deteriorating.

"When we first found out Andre was accepted for the trial, we were so excited," Larocque said.

"And then after a couple days, I realized, 'Oh wait, Josh has nothing.' Now that it's been over a year, we're really starting to see Andre just shoot ahead and Josh falling behind, and that's tough."

The family and their doctor believe Orkambi would change that. But the familycan't afford the annual cost of $250,000. Their private health-care plan won't pay for it either.

They are desperate for Ontario to cover the medication for their younger son. They saythe criteria forwho qualifies for coverage are too strict.

The medication is only funded for children between the ages of sixand 17 who are really sick for a long time it's called the "exceptional cases" criteria. To qualify, a patient must show that they lost20 per cent of their lung function for a six-week period over the course of six months.

That's the standardin three provinces Alberta, Saskatchewan and Ontario.The other provinces and territories don't cover the drug at all and no province funds the drug for adults.

Under the rules, less than two per centof children with cystic fibrosis qualify for Orkambi coverage in Ontario, according toCystic FibrosisCanada.

And Joshua isn't one of them.

The Ontario Health Ministrysays ithas not received any requests for coverage since the introduction of the exceptional casescriteriaonFeb.20.

The board that recommends whether medications should bepublicly funded is the Canadian Agency for Drugs and Technologies in Health (CADTH). It's made up of representatives fromthe health-care system, academiaand the public.

It decided there wasn't enough evidence of therapeutic value to recommend governments cover Orkambi, but provinces aren't bound by CADTH's decisions.

Steele says a lot of good medications are rejected for coverage because the testing criteria aredesigned for drugs that treat more common diseases and can be evaluated inlarge clinical trials.

"We can't do that in small populations. And so, often when our evidence goes to the table for government to look at, they say there's not enough evidence. And our problem is there's not enough people to give you the evidence that you want."

'Hard to put a price on life'

Then there is the cost.

"It's expensive," Steele said. "I'm not going to shy away from saying price is definitely a concern. However, it's very hard to put a price on life."

The three cystic fibrosis drugs are priced at about $250,000 a year in Canada. But Chris MacLeod, chair of the Canadian Cystic Fibrosis Treatment Society,which advocates that patients with the disease should have access to any medicationtheir doctor prescribes,says that doesn't reflect what the real cost would be if Canada did a better job negotiating with drug companies. He points toothercountries where the drugs are available for less than half that price.

The problem, he says, is Canada does not negotiate prices with drug companies until the end of the CADTH review, so many drugs end up being rejected for coverage because of cost.

In some cases, provinces won't negotiate at all, or they go it alone instead of negotiating on a national scale and buying in bulk to lower the cost.

"Canada is an outlier. We're the only developed country that either doesn't have a deal, or isn't negotiating a deal," MacLeod said ofOrkambi.

"Quite frankly, I think it's bureaucrats that have become so baked in the system that they've lost perspective."

Meanwhile, Canadians like Joshua Larocquego without the medications they need, he said.

"It's shameful. Absolutely shameful."

MacLeod is also a lawyer and is heading up a class action lawsuit against the B.C. government, federal government,CADTH and others over the lack of funding for Orkambi. The suit claims governments are"denying access to lifesaving drugs in a very non-transparent, non-evidence-based manner."

None of the allegations has been proven in court.

In an email to Go Public, B.C.'s Ministry of Health saidCADTH recommended Orkambi not be funded due to a lack of evidence on two separate occasions.

"These recommendations are based on clinical and cost factors," a spokesperson said. "And with Vertex setting the cost of Orkambi at $250,000 a year per patient, its cost is unsustainable for public and private drug plans."

In an emailto Go Public, a CADTH spokesperson said that over the past five years, the agency has taken steps to clarify and enhance processes to address the unique challenges of evaluating drugs for rare diseases.

Ontario health minister ignores family's calls for help

Andre and Joshua's parents have repeatedly reached out to OntarioHealth MinisterChristine Elliott.

In October, the family asked for a meeting with Elliott. They said theywere told by her office it would happen. They're still waiting to hear when, and their followup messages have gone unanswered.

As opposition health critic, Elliottcriticized the government at the time for not funding Kalydeco, one of the three gene-modifying cystic fibrosis medications.The drug was later added to the coverage list inOntario, but few patients qualify.

It's asimilar story with Orkambi. In February, Ontario agreed to fund the drug on a case-by-case basis under what's called the Exceptional Access Program.So far, not one cystic fibrosis patient hasqualifiedfor provincial coverage under theprogram's criteria, said MacLeod.

Watch: Elliott challenged the Liberalgovernment to cover Kalydecoseveral years agowhen she was Opposition health critic.

The minister turned down a request for an interview with Go Publicand didn't respond to specific questions we sent about Joshua and Andre's situationand about access to the cystic fibrosis medications,providing insteada general statement from a spokesperson.

"Young Ontarians with rare diseases deserve every opportunity to enjoy healthy and happy childhoods,"spokesperson Hayley Chazan wrote in an email to Go Public.

"Orkambi, which helps treat pediatric cystic fibrosis, is currently considered for funding in Ontario in exceptional cases consistent with clinical evidence."

Joshua and Andre's parents are stillhopeful the federal government will deliver on a 2019 federal budget promise to implement a rare disease strategy that could help people like Joshua.

The family believes Andre will continue to get access to Symdeko indefinitely. The trial is to test the medication on younger children and track it as they grow. It's already approved by Health Canada for children ages 12 and older.

Vertex Pharmaceuticals makes both Orkambiand Symdeko. It tells Go Public it has no plans to ask CADTH to reviewSymdeko for provincial funding, saying the present system for assessing drug coverage in Canada "has challenges."

When asked why the cost of Orkambi is so high, Vertex didn't answer, but Cystic Fibrosis Canada says developing medications for rare disease is expensive because the market forthem is so small.

"That leaves Symdeko and the other drugs coming down the pipeline completely inaccessible,"Haughian said."Without a proper rare disease strategy for evaluating drugs in place, there is not much incentive for drug companies to even try to develop drugs for rare diseases."

She and her husband worry their younger son will never get the medication he needs.

"I fear Joshua will spend his childhood getting sicker, living in and out of the hospital, needing more and more treatments, and ultimately dying young," Haughian told Go Public in an email after the interview, because she didn't want her sons to hear about her fears.

"While Andre is out playing hockey and hanging with friends and as he gets older and understands more and more, the 'survivor's guilt' he could potentially feel is a dangerous thing."