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battle for drug access a fight for life for people with cystic fibrosis

'this whole battle for access to medication is literally a fight for my life,' hailey laxer.

by emma jones

aug 25 2020
read time 7 minute read
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access to "life changing' cystic fibrosis medications delayed in canada
hailey laxer lives with cystic fibrosis and has been in an ontario hospital for the past two weeks due to a complication with chronic lung disease caused by cystic fibrosis. laxer / cystic fibrosis canada

cystic fibrosis canada (cfc) is calling on vertex pharmaceuticals and the government of canada to fast-track the application and approval of a precision medication for the rare disease. this comes amid concerns that changing regulations to the treatment approval process have dissuaded pharmaceutical companies from working with the federal government.

“the federal government’s attempt to oversimplify the issue and suggest that their hands are tied until the manufacturer applies is a diversion tactic and a complete abdication of their responsibility in the process,” cfc wrote in a statment.

a study released by the hospital for sick children and st. michael’s hospital in toronto looked at how canadians living with cystic fibrosis (cf) would be affected by the introduction of the drug, trikafta. researchers believe that having access to this treatment over the next decade can improve overall quality of life for those living with cf and extend life expectancy by an average of nine years.

vertex pharmaceuticals has yet to submit trikafta to health canada for approval. cystic fibrosis canada is concerned that proposed changes to how the federal government approves the cost of new treatment regimens has made companies wary of working with the government body.

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christine elliott has a secret talent: tap dancing! ontario's minister of health put on an impressive display of bobbing, weaving, sidestepping and waffling at queen's park on wednesday, all to dance around the serious subject of life-saving drugs for canadians with cystic fibrosis. the princess of prevarication could not or would not directly answer questions from the press about government funding for drugs from vertex pharmaceutical that are life-savers for people with cf. despite the fact that all four drugs from vertex are being supplied to cf patients in 18 other countries around the world — despite the presence at queen's park of worried-sick parents whose children are in declining health, and despite her firm promise to extend an official invitation to vertex to negotiate a portfolio deal — the minister has no answers for anyone. all the more interesting, then, to watch elliott equivocate on these life-saving cf medications and hide behind various bureaucracies — she must know something those 18 other countries and canada's leading cf physicians don't know.
health minister christine elliott is pictured in question period on feb. 26, 2020. (ernest doroszuk, toronto sun)

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“we understand that the patented medicine prices review board (pmprb) regulations do inject a level of uncertainty, but this drug is incredibly important,” says john wallenburg the chief scientific officer at cystic fibrosis canada. “while we recognize that business doesn’t really like uncertainty, we don’t want the company waiting for the perfect conditions before submitting the drug to health canada. we believe it’s just too important.”

trikafta, known in europe as kaftrio, received approval for marketing in the european union on friday . the national health service in england has already signed a deal with vertex pharmaceuticals to make the drug available to patients.

it was also fast tracked for approval for use in the u.s. by the fda and approved in october 2019 — a move rated as the number one best thing to happen in 2019 in washington’s post’s ranking of 19 good things to happen in 2019 . the treatment price was set at $311,503 usd per year.

a next-generation treatment for cystic fibrosis

“this whole battle for access to medication is literally a fight for my life,” hailey laxer, a patient with cystic fibrosis, told healthing over the phone from her hospital room. laxer has been in an ontario hospital for the past two weeks due to a complication with chronic lung disease caused by cystic fibrosis.

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cystic fibrosis is a genetic disorder in which cells produce a sticky and thicker variation of body’s mucus, sweat and digestive fluids, according to the mayo clinic . because of their altered state, these secretions can then build up and block various passageways in the body, damaging important organs and leading to an increase in infections.

the lungs and digestive system are the most commonly affected by cystic fibrosis. without treatment, individuals may experience persistent coughing, frequent lung infections, intestinal obstruction, and poor weight gain and growth. currently, patients can live into their 50s with treatment, however, quality of life is greatly affected.
“the vast majority of the current treatments focus on treating the various symptoms of the disease,” says wallenburg. “[patients] will have between two and four hours of therapy every day just to maintain their health, [as well as] antibiotics, enzymes to help them digest their food and nutritional supplements to compensate for poor absorption. it is a progressive disease and so the quality of life deteriorates progressively as well.”
laxer, who estimates she takes 57 pills a day to combat the various side effects of cystic fibrosis, says that access to this medication has the potential to change her life.

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“i’ve given up so much this last year. i had a full time job before; i’ve been on sick leave for six months now,” she says. “it’s heartbreaking knowing that there’s a drug out there [that i can’t get].”
cystic fibrosis is typically caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (cftr) gene. trikafta is a combination of three precision medications that target the most common mutation, known as f508del. it is estimated that 90 per cent of cystic fibrosis cases are caused, at least in some part, by this mutation.
“there is a method for us to get some patients who are in severe need of the drug compassionate access,” says anne stephenson, a pulmonologist and clinician scientist at st. michael’s hospital who works with cystic fibrosis patients. “i’ve seen unbelievable improvements in patients who you wouldn’t necessarily think would be able to be improved because they have such severe disease.

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“they have improved their lung function dramatically. they have been removed from the transplant list because they have clinically responded and improved so much, they’ve come off oxygen… just being able to breathe, being able to do something that is as simple as smelling things.”
on top of the increase in life expectancy, the study found that if every cystic fibrosis patient in canada eligible for this drug received it and had the improvements that were reported in the clinical trial, there would be 60 per cent fewer instances of severe lung disease, 15 per cent fewer deaths, and 18 per cent fewer hospitalizations or home intravenous needs for chest infections.
“when you tell someone [they’re getting special access] they burst into tears. they’re completely overwhelmed and so thankful to be able to have access to it, but they have mixed feeling because…they know their friends and other people who need it, haven’t gotten it,” continues stephenson.
“patients are in a situation where they say, ‘you know what, i kind of want my lung function to go down so that i can qualify for this compassionate program because i know [the drug] is not coming to canada anytime soon.'”

patients caught in the middle

the canadian government is working to change the way drugs are approved and priced in canada, which they claim will allow them to lay the foundation for a national pharmacare program.

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according to the federal government, canadians rank third in highest patented drug prices — after only the u.s. and switzerland. amendments already made to the patented medicines regulations are estimated to save canadians $13.2 billion over 10 years on drug costs.

cystic fibrosis canada says that the uncertainty imposed by this overhaul has caused pharmaceutical corporations to hold back on submitting new treatments for approval in canada.
“vertex has not submitted its investigational triple combination medicine to health canada because of the ongoing uncertainty created by the patented medicine prices review board pricing reforms,” said a spokesperson for vertex pharmaceuticals. “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. for our part, we are actively engaging with multiple arms of the canadian government to create an environment where access to rare disease medicines for eligible patients is achievable.”

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the pmprb, however, maintains that any business decisions made by pharmaceutical companies due to the proposed changes would be premature.
“it should be noted that manufacturers do not need the pmprb’s prior approval of their price in order to launch drugs in canada,” a spokesperson for pmprb said. “the price review is conducted after the date of first sale or market authorization is received. in fact, the reforms are expected to reduce the gap between the prices that consumers find fair and that manufacturers are seeking.
“a closer alignment of these prices has the potential to improve the outcome and timeliness of a negotiation by public and private payers.”
the amended patented medicines regulations are expected to be published within the next few weeks, and take into account consultations from stakeholders and patient advocacy groups. the changes will come into force on january 1, 2021.
for laxer and other patients caught in the push and pull between government and corporation, four months for clarity is four months too many.
“we’re for decreasing drug prices in canada, obviously to make things more accessible and more sustainable…but not at the expense of rare disease [treatments] and precision medications,” says laxer. “changes to the pmprb need to [consider rare diseases] and need to have a different set of criteria for these medications.”

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“i need [access to these medications] for me and i need it for my friends living with cf so that we can all live to our full potentials and have a better quality of life. i’m not done.”
why are people with cystic fibrosis waiting for a medication that adds years to their lives? add your voice to the fight for this life-changing drug.
 

emjones@postmedia.com |  @jonesyjourn

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