As HSE, Government, and Respreeza drugmaker bicker, one man’s life hangs in the balance

John Hannan has been a ‘guinea pig’ for Respreeza for 11 years. Now, due to the reported €84k-a-year cost, he faces losing access to a drug that’s proven a lifesaver. He talks to Health Correspondent Catherine Shanahan

John Hannan gets his last infusion of Respreeza, for treating genetic emphysema. Picture: Dan Linehan

John Hannan sits in a chair as a drug, diluted in a bag of fluid, drips slowly into his veins. There are three witnesses to this event: Myself, a photographer and the nurse who set up his infusion.

It’s not too far a stretch to say the image is slightly suggestive of a prison execution, because, in a way, John feels a bit like an inmate on Death Row. He sits in a chair at Mallow Primary Healthcare Centre as four-and-a-bit vials of human alpha1-proteinase inhibitor, brand name Respreeza, works its way into his bloodstream for what looks like the final time.

After 11 years of weekly infusions, John is losing access to what he regards as a life-changing drug, because he’s simply too expensive to maintain: The Government and the drug manufacturer, CSL Behring, could not reach agreement on what represented a fair price for the only approved therapy known to slow the progression of genetic emphysema.

In its coldly analytical way, the National Centre for Pharmacoeconomics recommended, in the run-up to last Christmas, that the State not reimburse the cost of Respreeza — reportedly €84,364.80 per patient per annum (excluding Vat) — because its cost-effectiveness “has not been demonstrated”.

The previous year, the results of a clinical trial, published in The Lancet, found conclusively that patients treated with Respreeza saw a significant slowing down, by 34%, of the progression of emphysema, a lung disease. On foot of the findings, the therapy was licensed by the European Medicines Agency and is now available in about eight European countries as well as the US.

A second study published in December 2016, around the time the National Centre for Pharmacoeconomics recommended no State reimbursement, confirmed the results of the first study.

John’s own experience with the drug tallies with the research findings. Symptoms of his lung disorder — the result of severe deficiency in Alpha-1 antitrypsin, a protein whose main job is to protect the lungs from infection — began to manifest in his late 40s. Up to that point he’d been extremely active; “lots of tennis, horse-riding, I never carried an ounce of weight”.

At the time, he was an engineer with Pettit Engineering and used to drive from Mallow to Cork City.

“I would be stopped in traffic and I’d find myself slow to react. I could lose concentration. I could find myself dozing off in the office.” In retrospect, this was due to reduced oxygen levels caused by his disease.

He was sick all the time, in and out of hospital with recurring chest infections and eventually diagnosed in 2003 at age 54.

After an extended spell of sick-leave, he was ultimately advised by his doctors in Beaumont Hospital to take time out. “I ended up having to retire at age 56,” he says.

It hasn’t been all doom and gloom. This extortionately expensive therapy, somewhat bluntly described as “maintenance treatment for adults with emphysema”, has, up to now, improved the quality of John’s life enough to allow him occasionally ride out with friends, to run errands, to give a few private horse-riding lessons to kids who attend Glen Aire Stables, the family equestrian centre in Lavally, Mallow, Co Cork.

It has not restored him to the keen sportsman he once was, but it has cut him enough slack to still wring enjoyment from life. His four-year-old grandchild is one of his greatest blessings but he fears that without the drug, he won’t be around for many of his milestones.

Respreeza is only approved therapy known to slow progress of genetic emphysema

“When I was first diagnosed, I was told I wouldn’t see my 60th birthday. Now I’m 68,” he says. His lung function in 2006 was “down between 30%-40%, but it’s up around 60% now”.

John is feeling teary on the occasion of his final infusion, but anyone in his position would weep. He travelled to Beaumont for the first six years of infusions and, at first, it knocked the stuffing out of him, and he’d have to overnight to recover. “But once I got in the swing of it, I’d get the train up and down.”

For a while, he travelled to a lab in Tallaght, but he’s been attending Mallow Primary Healthcare Centre for the past three years. Órla, a nurse with healthcare company Point of Care, administers the infusion and she is crushed for John that the treatment is ending.

“I feel sad for John that this is his last infusion and that there are no definite answers as to where this will go next. It’s not just John. It’s all the other patients in the group. I feel the Government has completely left them down,” says Órla.

Twenty-one patients on the island of Ireland got access to Respreeza through participation in the Rapid trial, a multicentre international clinical trial, with Beaumont Hospital in Dublin as one of the trial sites.

Another 40 could potentially benefit from the drug. When the trial, which started in 2006, ended, Respreeza’s manufacturers, CSL Behring, continued to supply it to the 21 patients under a “compassionate use programme” intended, it said, “to bridge the gap” until the HSE made a decision on whether to reimburse the drug.

There have been suggestions, which the company rejects, that the compassionate use programme was part of a long-term plan to leverage reimbursement.

CSL Behring’s general manager in Britain and Ireland, Eddie Owens, wrote in a letter to Kerry Fianna Fáil TD John Brassil, who has lobbied on behalf of Alpha-1 patients, that when the compassionate use programme was set up many years ago, “reimbursement was more or less assumed once the European Medicines Agency had granted a product licence on the basis of efficacy and safety (not cost)”.

He rejected the suggestion that the programme was “part of a long-term plan to leverage reimbursement”. CSL Behring claims to be “deeply disappointed that Respreeza will not be reimbursed and become available for treatment of patients... for whom there is no alternative available”.

The Government and HSE were lobbied extensively. The Alpha One Foundation, representing the patients, repeatedly pointed out that not only was Respreeza the only approved therapy known to slow the progression of genetic emphysema, but that no one had the faintest idea what the effects might be once patients came off the drug.

Prof Gerry McElvaney, a Beaumont-based consultant respiratory physician involved in the Respreeza clinical trial, and chair of the foundation, rightly pointed to the absence of data “on what happens when people stop this medication after being on it for a long period of time, a scenario into which these particular patients are now facing”.

Ironically, but predictably, CSL Behring is in discussion with health authorities in the US and Europe, including evaluating clinical sites in Ireland, precisely for the purpose of determining the long-term safety and efficacy of Respreeza.

However, that’s of little consolation to patients like John. He’s grateful the pharma giant gave him the drug for free for so long, but at the end of the day, he was a guinea pig, he says.

“They gained a lot of information from our bodies while we were on the Rapid trial and I think it’s immoral now that they can just walk away,” he says.

It’s a view echoed by former foundation chief executive Kitty O’Connor, who said the company “should bear in mind that it has gained great benefit from the involvement of patients here in its research”.

John Hannan. Picture: Denis Minihane

The foundation conducted its own survey of patients on Respreeza and more than four in five reported an improvement in general symptoms. Chest infections per annum dropped by 68% while hospitalisations as a result of chest infection decreased by 69%. People also reported improvements in their ability to work and to lead an active and fulfilled family and social life.

At the time of the National Centre for Pharmacoeconomics decision, CSL Behring said it would extend its compassionate access programme until the end of January 2017. The deadline was pushed out month by month as the company awaited a final reimbursement decision from the HSE national drugs committee.

It too rejected reimbursement. When the final decision was announced mid-August, CSL Behring extended the compassionate use programme to the end of September 2017 “so that patients have time to consult with their medical teams”.

A care plan for patients has been put in place at Beaumont Hospital. It involves monitoring visits and tests such as blood pressure and heart rate every few months According to Prof McElvaney, the outcomes of these tests “will identify any possible change or deterioration” in the patient’s medical condition.

It will also, he says in a letter to John, “provide valuable information on the effects of the withdrawal from Respreeza”.

It’s the next line that jars with John: “Any significant findings will be escalated to the HSE, the Dáil and CSL Behring, thus supporting our campaign for the reimbursement of Respreeza.”

John has no intention of signing the consent form that would allow his information to be passed on, especially not to CSL Behring “as part of another study”. It got enough information from his body, he says, and now it has “walked away”.

John finds it ironic that the Government funds a detection programme for Alpha-1, but refuses to fund a proven treatment. He dreads what lies in store without his weekly infusions. He suffers from severe restless leg syndrome, but the drug has kept it at bay.

He’s fearful of the approach of winter and the threat of chest infections. He can’t go to places as basic as shopping centres because of the risk of infection. He can’t go on long-haul flights. “There’s nothing nice about waking up in the middle of the night and not being able to breathe.”

Some days he can’t walk and talk simultaneously.

John should be enjoying retirement but instead he finds himself giving endless newspaper and radio interviews “begging for my life”. His wife has spent endless hours ringing politicians, trying to meet them, coming to protests, “but it’s a brick wall” he says.

Alpha One Foundation protesters outside the gates of the Dáil urging action to make Respreeza available to patients. Picture: Mac Innes Photography

He’s a reluctant spokesman. They are a private family he says, but privacy and dignity slide by the wayside when you are forced to beg for your life.

“Now my life is out there for everyone to see. Everywhere my sons go, they are asked ‘How’s your Dad?’”

John’s disillusionment and anger is spread evenly between the Government, the HSE, and CSL Behring. He says letters to politicians such as Health Minister Simon Harris and Foreign Affairs Minister Simon Coveney went unanswered. Mr Harris did give a written response to a question from Cavan-Monaghan Fianna Fáil TD Brendan Smith on October 3, asking if persons currently using Respreeza “will not be denied access to this medication”.

In response, Mr Harris said he considered CSL Behring’s withdrawal of the compassionate access scheme “unethical” and that there should “be no link between compassionate use schemes and reimbursement decisions”.

However, it was the “responsibility of both the company and the investigator (clinician) to ensure that they have considered and made arrangements for the patients, including in circumstances where the product is not reimbursed by the HSE”. What Mr Harris means by “arrangements for the patients” who are losing access to a life-changing drug is not clear.

Mr Harris adds that on his request, the HSE “sought assurances from Beaumont hospital that appropriate care arrangements are in place for patients” — which essentially amounts to a programme monitoring patients for deterioration.

He said the HSE “has also advised that it has decided to continue with the provision of this treatment to this patient cohort only, for an initial period of two weeks”.

The HSE confirmed it had asked CSL Behring to continue providing Respreeza to the 21 patients for another fortnight “to enable further deliberations on the matter”.

Yesterday, John said patients had heard “nothing official” in relation to this proposed extension.

“We can only live in hope that something will come of it this time and that they don’t leave us all in the lurch.”

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