Cystic Fibrosis (CF) Warrior Madi Vanstone and her mother Beth are hoping to draw more attention to their campaign to have the high cost of new life-saving medications covered by the government.
The fight has become even more urgent, after a young Halifax woman lost her life earlier this month to CF.
On Feb. 19, 23-year-old Chantelle Lindsay died in hospital after her request for a lifesaving medication, not marketed in Canada, was declined.
Her family had asked that she be prescribed the $300,000-per-year drug Trikafta on compassionate grounds, but by the time her request was processed, she no longer met the criteria, and she was turned down.
“It’s heartbreaking,” said an emotional Beth.
Trikafta is currently available to patients in the U.S., but not in Canada. It can only be made available in compassionate cases if the patient meets specific criteria. News reports indicated that the family's request was declined by drug manufacturer, Vertex, but Beth doesn’t think the manufacturer should be blamed.
“For our government to try and handoff blame to a company that is manufacturing these drugs, is really distasteful to me,” she said. “Had she (Lindsay) been given the Trikafta I have no doubt she would be here today.”
Beth reached out to local media in hopes of having the message heard: Canada needs 'orphan' drugs and needs them now, to avoid deaths like Lindsay’s.
Orphan drugs are those drugs developed, often after lengthy and costly research, to treat rare diseases that impact only a tiny percentage of the population. As a result, they tend to have extremely high price tags - beyond reach, without government assistance.
"Most people don't understand what's happening," said Beth, noting that the issue doesn't only affect the CF community, but all Canadians fighting rare diseases treatable by orphan drugs.
While Health Canada has indicated they can't comment on an individual's medical condition for privacy reasons, they acknowledged that the death of Lindsay has been difficult for all involved.
"We are very sympathetic to the circumstances that Ms. Lindsay and her family have faced, and the Department has tried to assist with access to treatment," read a response from Health Canada's Media Relations Officer, Geoffroy Legault-Thivierge.
He noted that in some circumstances, Health Canada’s Special Access Program (SAP) is a way for Canadians to access drugs not yet marketed in Canada but needed to treat serious or life-threatening conditions, when conventional therapies have failed, are unsuitable or unavailable.
These requests are initiated by a health practitioner and are considered on a case-by-case basis. Decisions are based on the information provided by the practitioner, triaged by urgency and need, and usually evaluated within 24 hours, Legault-Thivierge said.
Health Canada noted, however, that in some cases a manufacturer of a non-marketed drug may impose conditions for access that are separate from those of SAP.
As in Lindsay's case, "the timelines and criteria are at the discretion of, and are dependent on, the manufacturer... The timeframe for access may be extended."
Health Canada said that Vertex, the manufacturer of Trikafta, requires patients to receive pre-approval from the company before applying to Health Canada’s SAP.
"The SAP recognizes that this environment can be challenging, and provides assistance and information to both the requestor and manufacturer in these situations to help the request process. The manufacturer ultimately decides whether a drug would be supplied to the practitioner following an SAP approval," said Legault-Thivierge.
Eighteen-year-old Madi is currently on the medication Kalydeco to treat her CF - another orphan drug.
She was diagnosed with CF when she was just nine months old, and was in and out of elementary school in Bond Head, as she struggled with the consequences of the disease and frequent hospitalization.
Six years ago, when Kalydeco was approved in Canada as a treatment for a form of CF linked to a specific genetic mutation - the form of the disease that Madi has - she and Beth fought hard to have the government cover the cost of treatment.
The family initially fundraised extensively to cover the approximately $60,000 per year that was their share of the cost (Vertex and private insurance covered the rest of the six-figure bill) and were eventually successful in persuading government health insurance to pick up the tab. Madi was put on Kalydeco as part of a drug trial, and has been doing very well on it.
The lengthy fight earned her the title 'CF Warrior.' Now, she and her mother are doing all they can to help other CF patients, and those in need of other orphan drugs.
“We’ve got three modulators (drugs) from Vertex, and there’s very little access in Canada, because Canada is not on board to support them," claimed Beth, warning that the new Patented Medicine Prices Review Board (PMPRB) may make it more difficult for companies like Vertex to market in Canada, due to pricing.
“If they submit and do the pricing that Canada is asking - #1, they aren’t going to make a lot of money here, and #2, that then drives down the pricing around the world,” said Beth.
Both Madi and Beth have been fighting for years to have the drugs covered in Canada. The news of Lindsay's death has fuelled their fight.
“Let’s give Chantelle a legacy, and let’s say the real story why Chantelle doesn’t have access to that medication. Let’s hold Canada accountable for their part in this,” said Beth.
She acknowledged that the drugs are expensive, but pointed out that the manufacturer is open to negotiating a reduced price - and that there is only a short window of opportunity to save the life of a patient.
“Chantelle died in my opinion because Canada is not stepping up to the plate to get the medications,” she said.
Beth warned that this was “just the beginning,” and that there will be more cases like Lindsay’s unless there is action.
The Vanstones met with Ontario Health Minister Christine Elliott back in November, asking the Minister to invite Vertex to Canada to negotiate a portfolio deal for current and future drugs.
Elliott was also asked to remove restrictive criteria, so that treating doctors could prescribe as they see fit, rather than go through a time-delaying application process. “Why is a little bureaucratic body involved in prescribing medications to sick patients? It doesn’t make sense,” Beth said.
Vanstone said Elliott told her it wouldn’t be possible to approve all drugs, but in regards to Trikafta, it was a “no brainer.”
“She said I could count on her and we would have a follow-up call,” said Beth.
She did receive a call from someone from the Ministry, who told her that the situation was being handled by the Pan Canadian Alliance, which represents the provinces in negotiating bulk deals on drugs. Unfortunately, not all provinces have agreed and are on board for the costly drugs, but Ontario can negotiate their own terms, she was told.
The Vanstones say they have heard nothing further from the Health Minister on the progress of those negotiations.
Beth has a letter Elliott had written to Cystic Fibrosis Canada in 2014 when she was Deputy Leader, and Ontario PC Party Critic for Health and Long Term Care, outlining the PC Government’s priority in getting life-saving orphan medications into Canada, and urging the CFC to continue their dialogue and idea-sharing for the funding of the drugs in Ontario.
Beth is wondering where that passion and drive is today, now that the PC government is in power.
“It’s hypocritical, in my opinion, to be a health critic, and think it’s important, and then to be in power to fulfil promises that were made and then go MIA. That’s not okay,” said Beth.
VillageMedia contacted Elliott's office, which noted that the Minister could not comment on individual cases. A response from the Ontario Ministry of Health assured that "we are continuously reviewing evidence that supports the funding of new medications."
"In her new role as co-chair of the Conference of Health Ministers, Minister Elliott will be working with her provincial and territorial counterparts as well as the federal government to focus shared efforts on identifying solutions to the most pressing pharmaceutical issues, including those related to expensive drugs for rare diseases and other high-cost pharmaceuticals," read the response.
On March 25, Beth and Madi will be at Queen’s Park, rallying to address the need for the drugs. Simcoe-Grey MP Jim Wilson, who has been advocating for the Vanstones since the beginning of their fight, held a press conference at Queen's Park with Dr. Elizabeth Tullis, Director of the Toronto Adult Cystic Fibrosis Clinic at St. Michael’s Hospital, and Chris MacLeod, Chair of the Canadian Cystic Fibrosis Treatment Society on Feb. 26.
Wilson also raised the matter in question period, referring to two brothers – Andre and Joshua Larocque – from Tottenham, one of whom is on a drug trial and the other is not.
“In my 30 years as an MPP and as a former Health Minister, every rare disease drug that is mentioned in the legislature ultimately ends up receiving public coverage," said Wilson at the press conference. "The government knows it will end up paying for these medications, therefore they should be proactive in their funding so people like the Larocque boys in my riding and other Ontarians don’t continue to suffer.”
The Vanstones as well as Sasha Haughian and Jamie Larocque, joined Wilson at Queen's Park on Feb. 26.
"He is going to champion for our community. He's fighting tooth and nail to get these drugs covered and hold the government accountable," said Beth. “It’s not just like 'this could happen' - this is happening, and it’s preventable. And there’s no excuse. we’re not a poor country, Canada can afford this.”
Effective July 2020, the Patented Medicine Prices Review Board (PMPRB) will put in place new rules and regulations that will lower the cost of medications for patients across Canada, but will make it harder to obtain and afford orphan drugs.
"We are losing this battle, and the most vulnerable in Canada will be paying the price with their lives due to the new regulations," said Beth. "People don't know that this block is coming to all Canadians."
The public only hears about the 'savings' part of the deal, she said, and not the ultimate cost: people's lives. She added, “This can all be changed if drugs were covered and paid for” by the government.
Health Canada said they have been in contact with Vertex, which at this time has not provided a submission to market Trikafta in Canada.
Vertex also could not comment on Lindsay's case due to privacy reasons, but said they were saddened to hear of the news. The company noted it is restricted by law in how and to whom it can supply unauthorized medicines in a compassionate use program.
"We endeavour to make these complex decisions about compassionate use as ethically, fairly and quickly as possible, in line with the various medically-driven clinical criteria, the available information about the potential medicine, as well as the patient’s specific overall medical circumstances," read a response from Vertex.
The pharmaceutical company was not able to provide comment on drugs that haven't received market authorization in Canada yet, but said, "Vertex has strong concerns that new Canadian medicine pricing reforms have the potential to limit access to treatments for Canadians living with a rare disease."
The company said it was working closely with relevant stakeholders "to re-envision an environment where access to rare disease medicines for all eligible patients is achievable, and remain committed to achieving access for all eligible patients to current and future CFTR modulators."
To date, SAP has authorized access to Trikafta for 49 Canadians, all of whom have received pre-approval from the pharmaceutical company.
"While Health Canada would welcome a submission from Vertex for Trikafta, it is up to a manufacturer to decide whether it chooses to seek market authorization for its product," noted Health Canada.
