Healthy Living

‘Screaming for Help' Over Orkambi Drug for Cystic Fibrosis

‘Screaming for help' Over Orkambi Drug for Cystic Fibrosis

When a person has a debilitating disease, dealing with that reality is enough to bear.

But, when there is a drug out there to limit the debilitation of that disease, and governmental decision-makers don’t provide patients with it, the fight about injustice becomes overbearing.

The denial for your access to that drug makes you more than annoyed: you feel exasperated and furious. As you keep fighting to get your hands on this medication, the scale of your emotion soon tips out at enraged. (We are human, after all.)

This scenario is bad enough when you are the patient, but when it’s your child, your battle for access to what will help your kid have a better life, becomes your impassioned mission.

Such is the case for Ayrin Armstrong, a Northern Ireland mother fighting for access to Orkambi, the ‘miracle drug’ to help lessen the life-limiting symptoms of cystic fibrosis (CF). Ayrin seeks that medication for her 10-year-old daughter, Zona. (BBC News/BBC)

About Zona

BBC informs us that Zona was diagnosed with CF as a baby, has experienced lung infections as a direct result of her CF, and has spent long periods of time in the hospital.

BBC tells us that Zona spends an hour each morning wiith physiotherapy and nebulizer treatments to loosen and expel the accumulated lung mucus. She also takes ten pills a day as treatment for her condition.

Zona wants the drug so she can live longer.

Ayrin’s struggle to get Orkambi for Zona

Ayrin tells us she is "screaming and shouting for help" to access Orkambi, the life-changing drug for those with CF, but "no-one is listening”. (BBC)

According to BBC, Ayrin is frantic to get this drug for her daughter. Zona’s health status has worsened over the past year. Her lung function tests have decreased into the 80-percentile range. (As CF progresses, this decreased lung function gets worse). Ayrin says the family has talked about moving south if her lung function tests fall below 60%, so they can gain access to Orkambi.

CF has caused Zona to become very short of breath; she wants the Orkambi so she’ll be able to run again. (BBC)

More about Orkambi

Orkambi is a ‘combo-drug’ designed to work at the cellular level to ‘degrade’ the CF-caused mucus plugs in the lungs. It has proved to be highly efficient with increasing lung function, and in reducing lung infections and hospitalizations for those with CF. The medication also helps slow the progression of the genetic condition that damages lungs and shortens lives. (Cystic Fibrosis Foundation /CFF)

Orkambi is a combination drug, consisting of Lumacaftor and Ivacaftor. In tandem, they attack and resolve the main symptom of CF: the over-production of thick, sticky mucus. This ‘gunk’ compromises many organs, especially the lungs. (CFF)
In the paragraphs below, we learn from CFF exactly how Orkambi “works its magic” for those with CF:

Essentially, Orkambi is a blend of the drugs Lumacaftor and Ivacaftor. As a combo-drug, Orkambi reverses the CF mucus problems with a two-step approach.

First, the Lumacaftor component helps move the genetically defective CFTR protein to its proper place at the cell surface, where its function can be restored.

Ivacaftor then takes over, increasing the activity of that now-restored protein, and raises the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs of people with CF. (Reduced cellular flow of salt and fluids is at the heart of this genetic disease.)

FDA Approval of Orkambi

In the US, the FDA approved Orkambi in 2015, for use with those age 12 and older. It’s developed for the age-required people with CF, who have two copies of the mutated gene, F508del. In the U.S., that genetic factor is the cause of more than 50% of Americans with Cystic Fibrosis. (CFF)

In 2016, the FDA then approved a lower dosage of this medication for children aged 6-years through 11-years of age. The higher dosage previously approved for those aged 12-years and older remains the same. (Vertex)

Hurdles to Jump

According to BBC, there seems to be two great hurdles to jump so that Zona can be started on Orkambi: A waiver to approve the medication plus another one to approve its use by a ten-year-old child. Or, to convince the state to supply the lesser-dose version meant for those under 12-years of age. (The 12-year age limitation remains in effect in southern Ireland.)

The current hurdle to arrange for Zona to get started on Orkambi is one of geography.

We are told that in the southern portion of Ireland, referred to as the Republic of Ireland, Orkambi is an approved medication for those with CF, at no cost. (BBC)

However, as BBC says, Zona lives in Northern Ireland, roughly 30 minutes from the border of the Republic. In Northern Ireland, the drug is not approved for treatment of cystic fibrosis.

You can understand how frantic her Mom has been, in trying to navigate the Irish medical system. This system has been spoken of by columnist John Crowne in “The Daily Edge.” As Crowne wrote: “Everyone accepts that there are grave failings in the Irish healthcare system; the only thing that divides us is how to solve them.”

Comparisons of Irish Healthcare

Residents of Northern Ireland have free ‘health and social care benefits,’ paid for by taxation. The medications to be covered are decided upon by the Health Service Executive’s office. (HSE)

The HSE is accountable for declining the coverage for Orkambi in Northern Ireland, where Zona and her family live.

According to the BBC, Orkambi is too expensive for coverage by the HSE and is, therefore, not offered for the treatment of CF. However, the BBC does note that waivers for “exceptional clinical circumstances” related to Orkambi treatment can be submitted. (You will see below the cost of this drug).

‘Down south,’ in the Republic of Ireland, free health and social benefits are also available, but just to certain people. Their healthcare system is a two-tiered approach: Public and private.

Those in the Republic of Ireland don’t have to be taxpayers to be eligible for public healthcare, but they must be ‘confirmed residents’. They also must pass a means test to determine if they are financially eligible for public insurance. Anyone over the age of 16 is eligible to apply for a medical card. (livinginireland)

Those who aren’t eligible to receive a ‘medical card’ for free medical services, including medications, must pay for the total cost or established portion of their medical expenses either via cash or by their private health insurance company.

Regarding medications, there is a somewhat limited formulary of ‘available’ drugs and, while those medications are free for those with a medical card, those with the card are charged a processing fee. (Wikipedia)

So, considering all of this, how is it that people with CF who live in the southern part of Ireland, are allowed no-cost Orkambi and Northern Ireland does not offer the drug at all?

Why is Orkambi not available to all Irish people with CF?

To tell this story, introductions are in order. Thanks to BBC, we are fortunate to become acquainted with 41-year-old Jillian Mc Nutley. Jillian lives about 50 miles south of Zona and her family.

As BBC explains, when Jillian became aware of the new ‘wonder drug’, it was not on the list of meds available to those in the Republic of Ireland.

Jillian was doing poorly with her CF, and readily became an instrumental voice and presence “in the Republic of Ireland’s Orkambi campaign.” (BBC)

Per BBC, Jillian noted the annual cost of Orkambi is about 100,000 in British pounds, which translates into $131,716 in US dollars. But, she goes on to say, “When you take into account the cost of hospitalization that people with cystic fibrosis regularly have for up to four months of the year, it all adds up.”

"That (hospitalization) costs a lot more than Orkambi does,” she concludes.

In explaining the fight for access to Orkambi, Jillian said her group was able to garner sizeable backing from politicians. She continued, "Momentum built up - the opposition put so much pressure on the government they were basically shamed into giving us Orkambi." (BBC)

Zona’s Mom told BBC no functioning Northern Ireland Assembly or executive at Stormont were there to listen when the family joined with others across England, Scotland, and Wales in bringing their campaigns to Westminster and the devolved assemblies in June.

"We have nobody there fighting our corner, but they have it in them to help Zona and others here," Ayrin said.