Negotiations for potentially life-saving drug ‘long overdue’

Chris MacLeod is a lawyer originally from Saskatchewan who has long pushed for access to a drug advocates say could save cystic fibrosis patients’ lives./Facebook Photo

After years of advocacy, the alliance of federal, provincial and territorial governments responsible for negotiating access to drugs for Canada’s provinces and territories has agreed to begin negotiations on a drug highly coveted by the nation’s cystic fibrosis community.

The Pan-Canadian Pharmaceutical Alliance, or pCPA, announced Friday that it would begin negotiations with Vertex Pharmaceuticals on the purchasing price for Kalydeco and Orkambi, the first and second generation of drugs more commonly known as CFTR-modulators.

The announcement did not include Trikafta, which is the third generation of the drug and is being fast-tracked in Europe and the US for approval. Canada has not begun the approval process for Trikafta.

Cystic fibrosis, or CF, is a fatal genetic disease affecting Canadian children and young adults. It has no cure, and causes many detrimental side-effects on the body, including on the digestive system and lungs.

Typical complications include difficulty digesting fats and proteins, malnutrition and vitamin deficiencies, progressive lung damage, diabetes and sinus infections. CF Canada estimates that one in every 3,600 children born in Canada has CF.

Lung disease eventually worsens to where the person is disabled. According to the US government, the average life span for people with CF who live to adulthood is 37 years. Death is most often caused by lung complications.

Dr. John Wallenburg, chief scientific officer at Cystic Fibrosis Canada, explained that the two new drugs, Kalydeco and Orkambi, treat the defective gene as opposed to just treating CF’s symptoms.

“This is a genetic disease, it’s a multi-organ disease, but that defective gene has downstream consequences and those are the symptoms that people live through,” Wallenburg explained.

“This (medication) is going in and correcting the defect, instead of trying to treat individual symptoms like infections in the lungs, or pancreatic insufficiencies, or poor digestion. Now, you take a pill and it’s correcting the CF protein throughout your body. That’s a huge difference for people.”

The drug approval process in Canada is a complicated one.

While provinces and territories administer most health care plans, they set up the pCPA to negotiate on their behalf to use buying power and get a bulk deal for medications.

Once the pCPA negotiates a price, individual drug plans can choose to list it or not.

Until Friday, the pCPA had not chosen to negotiate a price for Orkambi, and no provinces had stepped up to ask for such negotiations to begin.

Getting health jurisdictions to agree to ask for Orkambi negotiations has been a campaign that Chris MacLeod of the Cystic Fibrosis Treatment Society has undertaken. MacLeod originally hails from Saskatoon, but now lives in Oakville, Ont.

He has been living with CF since he was just two years old.

MacLeod is set to publish a report on June 23 outlining an increase in Saskatchewan’s CF population between 2014 and 2018.

According to MacLeod, his report found that the province’s CF population grew 14.3 per cent over those years, while the overall population grew 4.6 per cent. He’s delivering the report to each of Saskatchewan’s elected MLAs, hoping to get traction ahead of this fall’s provincial election.

“We are advising all of the province’s elected representatives, as well as those seeking to win a seat in the Legislative Assembly this fall, that our organization intends to make cystic fibrosis – as well as the accessibility of medical treatments – an election issue,” MacLeod said in a press release.

When reached by phone, he characterized Friday’s announcement as an important first step. Those negotiations, though, need to have a timeline. Even if they are successful, there’s no guarantee individual provinces will list the drugs.

“The number in Saskatchewan for the CF population appears to be growing,” he said Thursday.

“The funding is not rising in a commensurate matter. The real issue is governments across the board … not sitting down to negotiate for access to the new lifesaving medication that’s available.”

MacLeod said that as children with CF get older, they get sicker and end up in the hospital more and more “until they get a lung transplant or they die.”

“When your population is increasing and there’s a game-changing, lifesaving drug that 20 other countries have negotiated access to, and you refuse to negotiate, that’s alarming.”

The decision to negotiate comes after years of pressure from advocates.

Cystic Fibrosis Canada attempted to convince the cPCA to begin negotiations in 2016 and in 2019.

In 2016, though, the Canadian Agency for Drugs and Technologies, or CADTH, recommended that Orkambi not be publicly covered. CADTH conducts health technology assessments to help inform governments about public reimbursement of drugs. The organization’s recommendations are not binding, but governments often follow their advice.

Dr. Wallenburg joined Cystic Fibrosis Canada five years ago. In 2016, he had an opportunity to speak to CADTH about their rejection of Orkambi.

“I remember thinking at the time that it was a silly decision,” he recalled.

“I’ll explain it to them and they’ll realize the error of their ways and correct it. Of course, that’s not the way bureaucracy works. We felt the initial decision from CADTH was flawed and we’ve been pushing ever since.”

The complex journey drugs take before they can be made available in Canada is a “bureaucrat’s dream,” MacLeod said. The system, in his mind, makes it easier for different bodies to deflect criticism when it comes to what’s made available, and what’s not.

That system, though, is important for those who rely on drugs to treat rare, but fatal, genetic disorders.

“When we learned (negotiations would begin Friday), it was like, ‘oh my gosh, did we hear that right? It was a long time coming, it really was,” Dr. Wallenburg said.

“This is important news because up to now, pretty much all of the public jurisdictions have opted not to even negotiate on Orkambi. This articulates a will to consider bringing these drugs now to the public system, which is really important because that’s where the vast majority of people living with CF will get their drugs.”

The beginning of negotiations is no guarantee of success. It’s also no guarantee that the drug will become available in any one province. MacLeod, who was pleased negotiations were beginning and said he’s cautiously optimistic, worried it’s just lip service. He’d like the negotiations on Orkambi to have a deadline. Previous negotiations for the first iteration of the drug, Kalydeco, he said, took nine years. Letter of intent were signed for one mutation, with others excluded. Other mutations were later approved in 2019. He’s disheartened to see it on the list once again, this time as part of the negotiations for Orkambi.

Dr. Wallenburg also stressed that there is still a long way to go.

“There’s no guarantee,” he said.

“We don’t know the negotiations will be successful. We do know the company has been highly motivated for some time now to advance these to public access. It’s certainly in their interest. If this is an indicator from the pCPA that all jurisdictions are now interested, that’s great news.”

For now, though, patients are left with little they can do. While three provinces, including Saskatchewan, introduced special access for patients to access those drugs, Dr. Wallenburg said the criteria make that access nearly impossible.

“The special access … is under very strict conditions, to the point that in the over a year it’s now been available through that program, there is only a single individual win Canada who has been able to access the drug,” he said.

“Those criteria are unreasonably challenging.”

The exception is Quebec, where patients do have access under a special program.

“They are somewhat distinct in that sense,” he said.

In Saskatchewan, the provincial government did not provide a response to its intentions surrounding the new drugs, or whether it will even list them once they’re available, as of press time. An answer, a spokesperson said, might be available on Monday.

Now, with their years-long quest to get the first two generations of these drugs under federal review, Cystic Fibrosis Canada is focusing on getting the third generation, Trikafta, fast-tracked.

It’ll serve even more people, Dr. Wallenburg said. It could even save lives.

“Unless our governments get their processes aligned, it could take years for Trikafta to get through the regulatory, review and reimbursement process once it is submitted to Health Canada. Many people with CF don’t have time to wait,” Cystic Fibrosis Canada wrote.

“They may be hospitalized, need transplants or even pass away during this time. This is unacceptable. We need to come together as a community and advocate to both provincial and federal elected officials. We are calling on all governments … to get the drug to those who need it now.”

MacLeod has certainly heard that call. That’s why he’s releasing his report on June 23, and why he’s sending it to each of the MLAs in the province. While the process may be a bureaucratic nightmare, he’s confident elected officials can call and push for the medication to receive approval.

MacLeod wants someone to stand up and pay attention. He mentioned Premier Scott Moe’s 2019 Christmas message, which praised 11-year-old Cassidy Evans of Saskatoon, who was diagnosed with CF when she was four and has since raised thousands for CF research.

Moe called Cassidy a hero. Macleod, though, sees that as only words. If she’s a hero, she ought to have the support she needs and be treated as one.

“You’re the elected official,” he said.

“You can’t just feed it off on bureaucrats different agencies and levels of government”

He wants to see something substantial and to see more support for the province’s growing population of residents with CF.

“The alarming piece is we’re not doing anything,” MacLeod said.

“We promised we would, we could, and we just aren’t. Nobody is. (Governments say) we’ll wait and see, but nothing gets done.”

** This is an edited story. The story originally referred to the pCPA as a federal body. In fact, it is an alliance of federal, provincial and territorial governments. The Herald regrets the error.