Coughing bouts that lasted 30 to 40 minutes and retirement at the age of 56. Those were painful realities for civil engineer Johnny Hannan from Mallow, Co Cork. Diagnosed with a severe form of a rare condition called Alpha-1 Antitripsin Deficiency (genetic emphysema) the future looked bleak, given that there is no cure. Irish Country Living readers may remember that Johnny did a horse ride from Mizen Head to Malin Head to highlight the condition back in 2007.
Hope came six months after diagnosis in 2006, however, in the form of clinical trials of a new drug, now called Respreeza. “It’s a wonder drug – I will always call it that,” says Johnny. Only the second person in Ireland to try it, he responded very well and saw his own condition “improve and improve” as time went on.
“It worked wonders for me. Ten years ago I couldn’t talk without coughing. I was in hospital three or four times a year for weeks at a time. It has improved me immeasurably.”
There are 21 people in Ireland on the drug at the moment, he says, 17 in the south and four in the north. For the past 18 months they have all had the worry that their medication may not be available after the end of September 2017.
To date, the drug manufacturers CSL Behring have been giving Respreeza free to those with Alpha-1. Now they want the government, via the HSE, to pay for it. They are citing a cost of €84,000 per year per person, ex VAT.
BEGGING FOR YOUR LIFE
The government stance is not to pay more than €45,000 per year* per person for any new drug. That leaves those with Alpha-1 in a very difficult position. “We are literally looking down the barrel of a gun,” Johnny says.
“You really don’t know what’s going to happen at any time. It’s a horrible way, at 68 years of age, to be begging for your life to be extended. It should not happen.”
Those affected are now living month to month. “We never hear officially that the drug is being carried on from month to month. Instead we hear from a pharmacy in the midlands, because we have to give our weight every month as the drug is administered by weight: so many millilitres per kilogramme.
“Respreeza is supposed to stop at the end of September, and if you take it that all of us are very susceptible to winter colds and flu, you’ll see that we are facing a time of great uncertainty – unless some agreement is reached. To have to live without the treatment, knowing how well the treatment has worked, is a frightening thought,” he says.
COMPROMISE NEEDED
“The ironic thing is that the government funds the Alpha-1 Foundation research in Ireland and funds the targeted detection programme that has started in Beaumont Hospital,” he continues, “but now when a drug has successfully been found to slow down the progression of Alpha 1, they walk away from it and won’t pay for it! Does that make sense to anyone?”
Those with Alpha-1 are demanding that the HSE decision not to fund Respreeza be reversed. “We’re asking the HSE to come forward a bit with their money and CSL Behring to come back a bit. The overall cost for 17 people certainly wouldn’t run the country into bankruptcy. Of course the HSE has to balance the books, but they have to take people’s lives into consideration as well.
“€45,000 to €50,000 isn’t an awful expensive life when you think of the money that is being wasted across the board. The only satisfactory outcome for us is that we continue to get the drug, whoever pays for it. Obviously individuals can’t.”
Johnny points out that the drug is reimbursed in 12 European countries, including Greece. He also takes the the National Centre for Pharmacoeconomics (NCPE) to task in relation to evaluation costs.
“It is a very expensive body, in my opinion, and evaluates drugs after they have already been licensed and approved by the European Medicines Agency. Evaluating them all again doesn’t make economic or practical sense. If we are part of the EU, there should be no need to duplicate what’s being done on our behalf in Europe.”
www.alpha1.ie
Facebook – Access to Medicines Ireland
‘They are pawns in the middle. That shouldn’t be’
Dr Kieran Harkin is a member of the organisation Access to Medicines Ireland and is concerned about the Respreeza situation. “While the term “compassionate access” for patients is used by government and pharmaceutical companies, he says, it isn’t an accurate term.
“I don’t believe there is compassion, as patients are being held to ransom. The only reason CSL Behring is talking about withdrawing Respreeza is to maximise pressure on the government. It is a negotiating ploy. With Respreeza, patients have to lobby the government to get their drugs continued and that will play into pharma hands. They are pawns in the middle. That shouldn’t be.”
CHARGE WHAT THEY LIKE
If Respreeza really cost CSL Behring that much, that would be a different story, he adds. “The company is asking for €103,000 (€84,000 plus VAT), but the reality is that it’s probably costing them about €1,000 to manufacture – but no figures will be given.
The fundamental problem is that a company that develops a new drug can charge whatever price they like for it.
“That price has very little to do with the cost of the drug or the cost of bringing it to market. Like anything in short supply, they’re going to squeeze people for as much as they can.
“Some people will inevitably lose out because they can’t afford it. The manufacturing process may not be that expensive but you’re paying for the intellectual property, which we would argue is overpriced.”
New drugs coming on the market can cost anything from €1,000 to €1m per person, per year. New cancer drugs average around €150,000 per year, for example.
“This is clearly something that can’t go on,” Dr Harkin says.
TRIAL PATIENTS HAVE MORAL ENTITLEMENT
He makes the point that those with Alpha-1 took part in clinical trials and put themselves at risk.
“They didn’t know whether they were going to benefit or lose out by being part of the trial.
“I would say that these patients therefore have a moral entitlement to treatment and the company has a moral responsibility to continue with their access to this medication.”
He points to the fact that deals have been done behind closed doors before, when it comes to drug costs being covered. “We don’t actually know how much Orkambi, the CF drug cost, for example, because pharmaceutical companies insist on secrecy.”
GLOBAL PROBLEM
The issue of access to high-tech, expensive medicines is a global problem, he says. He acknowledges that it is a difficult situation for the government to address.
“The Irish government is a relatively small player in the international pharmaceutical field, but it is in a position to take a lead and is doing so, but action needs to be EU action and the pharma companies need to be stood up to.”
He acknowledges that limited budgets can force difficult decisions for government. “The money has to come out of somewhere, unfortunately. You have to stand back and say we have X amount of money and how do we spend this?”
COMPULSORY LICENCE solution
One solution for governments could be to issue a compulsory licence, he adds. “The patent could be overridden, and governments could say: ‘We want to import this drug and give it to a generic company and authorise them to manufacture the drug.’
“The downside would be that the USA would see this as a threat to the pharmaceutical industry and sanctions might be threatened. “It used to be just a problem in the developing world but now it’s a problem in wealthier countries, like Ireland, too.
“Other governments have done it with Hep C and HIV drugs in Brazil and Malaysia, to make them accessible to patients, but it’s a mechanism that takes political courage.”
He acknowledges that it is very difficult for patients caught in the middle of this situation.
“Patients shouldn’t be put in that position. Legislative change, political will and transparency is needed. If the public is expected to spend huge amounts of money, at the very least they should be entitled to know how the costs have been arrived at.
“Otherwise it’s difficult to negotiate. It’s like a fire brigade coming along saying how much are you going to pay me to put out your house fire? That’s no way to run a service.” CL
>> Calculating drug costs
How the drug is evaluated is very complicated for the lay person to understand. On the face of it, the calculation, according to the NCPE’s Respreeza decision document, looks like this:
• Vials of Respreeza cost €312 each.• Average number needed per patient per week = 5.2 • 312 x 5.2 = €84,364.80 (ex VAT) of €103,768.70 (inc VAT)But it is more complicated than that. The figures above are only the annual acquisition cost of Respreeza. The cost-effectiveness of the drug is also taken into account.
Putting a value on life
This is calculated in terms of the quality-adjusted life year (QALY): a measure of the value and benefit of health outcomes. Health is defined as a function of two components:
• Length of life (mortality). • Quality of life (morbidity).The QALY tries to combine these two components into a single figure/number. QALY gives an idea of how many extra months or years of reasonable quality of life a person might gain as a result of treatment. “The threshold the HSE has set is €45,000/QALY. This is not the same as €45,000 per person/per year,” says Dr Harkin.
The NCPE says: “The cost-effectiveness of Respreeza has not been demonstrated, at a threshold of €45,000/QALY and is therefore not recommended for reimbursement’. Respreeza, the document states, “is associated with an incremental cost of €712,563 and an incremental QALY gain of 1.2, giving a calculated base-case incremental cost effectiveness ratio (ICER) of €581,322”.(This is over 12 times the HSE’s €45,000/QALY limit).
Coughing bouts that lasted 30 to 40 minutes and retirement at the age of 56. Those were painful realities for civil engineer Johnny Hannan from Mallow, Co Cork. Diagnosed with a severe form of a rare condition called Alpha-1 Antitripsin Deficiency (genetic emphysema) the future looked bleak, given that there is no cure. Irish Country Living readers may remember that Johnny did a horse ride from Mizen Head to Malin Head to highlight the condition back in 2007.
Hope came six months after diagnosis in 2006, however, in the form of clinical trials of a new drug, now called Respreeza. “It’s a wonder drug – I will always call it that,” says Johnny. Only the second person in Ireland to try it, he responded very well and saw his own condition “improve and improve” as time went on.
“It worked wonders for me. Ten years ago I couldn’t talk without coughing. I was in hospital three or four times a year for weeks at a time. It has improved me immeasurably.”
There are 21 people in Ireland on the drug at the moment, he says, 17 in the south and four in the north. For the past 18 months they have all had the worry that their medication may not be available after the end of September 2017.
To date, the drug manufacturers CSL Behring have been giving Respreeza free to those with Alpha-1. Now they want the government, via the HSE, to pay for it. They are citing a cost of €84,000 per year per person, ex VAT.
BEGGING FOR YOUR LIFE
The government stance is not to pay more than €45,000 per year* per person for any new drug. That leaves those with Alpha-1 in a very difficult position. “We are literally looking down the barrel of a gun,” Johnny says.
“You really don’t know what’s going to happen at any time. It’s a horrible way, at 68 years of age, to be begging for your life to be extended. It should not happen.”
Those affected are now living month to month. “We never hear officially that the drug is being carried on from month to month. Instead we hear from a pharmacy in the midlands, because we have to give our weight every month as the drug is administered by weight: so many millilitres per kilogramme.
“Respreeza is supposed to stop at the end of September, and if you take it that all of us are very susceptible to winter colds and flu, you’ll see that we are facing a time of great uncertainty – unless some agreement is reached. To have to live without the treatment, knowing how well the treatment has worked, is a frightening thought,” he says.
COMPROMISE NEEDED
“The ironic thing is that the government funds the Alpha-1 Foundation research in Ireland and funds the targeted detection programme that has started in Beaumont Hospital,” he continues, “but now when a drug has successfully been found to slow down the progression of Alpha 1, they walk away from it and won’t pay for it! Does that make sense to anyone?”
Those with Alpha-1 are demanding that the HSE decision not to fund Respreeza be reversed. “We’re asking the HSE to come forward a bit with their money and CSL Behring to come back a bit. The overall cost for 17 people certainly wouldn’t run the country into bankruptcy. Of course the HSE has to balance the books, but they have to take people’s lives into consideration as well.
“€45,000 to €50,000 isn’t an awful expensive life when you think of the money that is being wasted across the board. The only satisfactory outcome for us is that we continue to get the drug, whoever pays for it. Obviously individuals can’t.”
Johnny points out that the drug is reimbursed in 12 European countries, including Greece. He also takes the the National Centre for Pharmacoeconomics (NCPE) to task in relation to evaluation costs.
“It is a very expensive body, in my opinion, and evaluates drugs after they have already been licensed and approved by the European Medicines Agency. Evaluating them all again doesn’t make economic or practical sense. If we are part of the EU, there should be no need to duplicate what’s being done on our behalf in Europe.”
www.alpha1.ie
Facebook – Access to Medicines Ireland
‘They are pawns in the middle. That shouldn’t be’
Dr Kieran Harkin is a member of the organisation Access to Medicines Ireland and is concerned about the Respreeza situation. “While the term “compassionate access” for patients is used by government and pharmaceutical companies, he says, it isn’t an accurate term.
“I don’t believe there is compassion, as patients are being held to ransom. The only reason CSL Behring is talking about withdrawing Respreeza is to maximise pressure on the government. It is a negotiating ploy. With Respreeza, patients have to lobby the government to get their drugs continued and that will play into pharma hands. They are pawns in the middle. That shouldn’t be.”
CHARGE WHAT THEY LIKE
If Respreeza really cost CSL Behring that much, that would be a different story, he adds. “The company is asking for €103,000 (€84,000 plus VAT), but the reality is that it’s probably costing them about €1,000 to manufacture – but no figures will be given.
The fundamental problem is that a company that develops a new drug can charge whatever price they like for it.
“That price has very little to do with the cost of the drug or the cost of bringing it to market. Like anything in short supply, they’re going to squeeze people for as much as they can.
“Some people will inevitably lose out because they can’t afford it. The manufacturing process may not be that expensive but you’re paying for the intellectual property, which we would argue is overpriced.”
New drugs coming on the market can cost anything from €1,000 to €1m per person, per year. New cancer drugs average around €150,000 per year, for example.
“This is clearly something that can’t go on,” Dr Harkin says.
TRIAL PATIENTS HAVE MORAL ENTITLEMENT
He makes the point that those with Alpha-1 took part in clinical trials and put themselves at risk.
“They didn’t know whether they were going to benefit or lose out by being part of the trial.
“I would say that these patients therefore have a moral entitlement to treatment and the company has a moral responsibility to continue with their access to this medication.”
He points to the fact that deals have been done behind closed doors before, when it comes to drug costs being covered. “We don’t actually know how much Orkambi, the CF drug cost, for example, because pharmaceutical companies insist on secrecy.”
GLOBAL PROBLEM
The issue of access to high-tech, expensive medicines is a global problem, he says. He acknowledges that it is a difficult situation for the government to address.
“The Irish government is a relatively small player in the international pharmaceutical field, but it is in a position to take a lead and is doing so, but action needs to be EU action and the pharma companies need to be stood up to.”
He acknowledges that limited budgets can force difficult decisions for government. “The money has to come out of somewhere, unfortunately. You have to stand back and say we have X amount of money and how do we spend this?”
COMPULSORY LICENCE solution
One solution for governments could be to issue a compulsory licence, he adds. “The patent could be overridden, and governments could say: ‘We want to import this drug and give it to a generic company and authorise them to manufacture the drug.’
“The downside would be that the USA would see this as a threat to the pharmaceutical industry and sanctions might be threatened. “It used to be just a problem in the developing world but now it’s a problem in wealthier countries, like Ireland, too.
“Other governments have done it with Hep C and HIV drugs in Brazil and Malaysia, to make them accessible to patients, but it’s a mechanism that takes political courage.”
He acknowledges that it is very difficult for patients caught in the middle of this situation.
“Patients shouldn’t be put in that position. Legislative change, political will and transparency is needed. If the public is expected to spend huge amounts of money, at the very least they should be entitled to know how the costs have been arrived at.
“Otherwise it’s difficult to negotiate. It’s like a fire brigade coming along saying how much are you going to pay me to put out your house fire? That’s no way to run a service.” CL
>> Calculating drug costs
How the drug is evaluated is very complicated for the lay person to understand. On the face of it, the calculation, according to the NCPE’s Respreeza decision document, looks like this:
• Vials of Respreeza cost €312 each.• Average number needed per patient per week = 5.2 • 312 x 5.2 = €84,364.80 (ex VAT) of €103,768.70 (inc VAT)But it is more complicated than that. The figures above are only the annual acquisition cost of Respreeza. The cost-effectiveness of the drug is also taken into account.
Putting a value on life
This is calculated in terms of the quality-adjusted life year (QALY): a measure of the value and benefit of health outcomes. Health is defined as a function of two components:
• Length of life (mortality). • Quality of life (morbidity).The QALY tries to combine these two components into a single figure/number. QALY gives an idea of how many extra months or years of reasonable quality of life a person might gain as a result of treatment. “The threshold the HSE has set is €45,000/QALY. This is not the same as €45,000 per person/per year,” says Dr Harkin.
The NCPE says: “The cost-effectiveness of Respreeza has not been demonstrated, at a threshold of €45,000/QALY and is therefore not recommended for reimbursement’. Respreeza, the document states, “is associated with an incremental cost of €712,563 and an incremental QALY gain of 1.2, giving a calculated base-case incremental cost effectiveness ratio (ICER) of €581,322”.(This is over 12 times the HSE’s €45,000/QALY limit).
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