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PATIENTS WITH A chronic lung condition have called on the HSE and Department of Health to strike a deal with the pharmaceutical company that makes a “life-changing” drug.
CSL Behring has indicated it will withdraw its Respreeza therapy from 21 Alpha-1 patients in Ireland at the end of the month. They are receiving the treatment through a compassionate use programme.
The Alpha One Foundation said Respreeza is “the only therapy shown to slow the progression of Alpha-1, resulting in a decrease in the frequency and severity of chest infections and associated hospital admissions”.
Patients with the condition, also known as genetic emphysema, have a deficiency of the Alpha-1 antitrypsin protein, which can lead to severe lung, liver and skin problems. Most people with the condition develop emphysema or chronic obstructive pulmonary disease (COPD) at some stage in their lives.
Kitty O’Connor, Chief Executive of the Alpha One Foundation, said: “The vast majority of these patients generously participated in a clinical trial here which was a vital part of its initial clinical research. The company argues that it has continued to provide this therapy to people on compassionate use grounds.
“However, we believe that it is unfair that patients who have served the company so well should continue to live under a cloud of deadline after deadline, with all the stress and worry that involves, and on top of having to manage a very challenging condition.”
O’Connor said, aside from the 21 patients currently receiving Respreeza, an estimated 40 others could also benefit from it.
“It is available in eight European countries – Italy, France, Germany, the Czech Republic, the Netherlands, Spain, Greece, and Slovakia. It’s time to add the Republic of Ireland to that list. It’s time to put patient’s lives first,” she said.
About 350 people in Ireland have been diagnosed with the most severe form of Alpha-1. It’s thought about 3,000 people have this form of the condition but are yet to be diagnosed, while thousands more have a moderate deficiency.
‘Living proof it works’
Johnny Hannan, 67, from Mallow in Cork, is one of the patients who took part in the clinical trial.
I am on the drug Respreeza for over 10 years and I am living proof that it works. Some time ago I was told that I might not live to see my 60th birthday. I am now 67 and am a very healthy man again thanks to Respreeza.
“I am very anxious and worried at the lack of progress by the Department of Health, the HSE and CSL Behring in resolving this horrible impasse.
“As a tax-paying Irish citizen, I deserve a reasonable quality of life and I would urge all parties to re-engage and agree a practical price to allow me and my fellow patients to have access to Respreeza into the future.”
Gillian Acheson, 52, from County Monaghan also has the condition.
“Alpha-1 is a very frightening disease. You can get out of breath very quickly.
I have a young child who is always wanting me to play with her and I feel terrible because I just can’t do it.
“If I had this therapy it could make such a difference to my life. Even if it could slow down the illness and let you live a little longer. I would like to see my daughter’s wedding day,” she said.
Not deemed cost-effective
A spokesperson for the Department of Health said the issue Is a matter for the HSE and CSL Behring.
In June 2016, the HSE asked the National Centre for Pharmacoeconomics (NCPE) to carry out a health technology assessment on the cost effectiveness of Respreeza, which costs over €100,000 per patient per year and is delivered by a weekly injection.
On 9 December 2016, the NCPE did not recommend the drug for reimbursement as it determined the manufacturer failed to demonstrate its cost-effectiveness.
A spokesperson for the HSE told TheJournal.ie the organisation is considering representations made to it by CSL Behring.
“The HSE has also met with the company to fully understand its representations. Under the 2013 Health Act, a final decision can only be made after the representations received have been carefully considered by the HSE.
The operation of compassionate schemes is at the discretion of manufacturers. However, any attempt by a manufacturer to link continued access for patients already being treated with a new drug with decisions under the statutory reimbursement process would be inappropriate and unethical.
“There should be no link between compassionate use schemes and reimbursement decisions and manufacturers should be up-front with patients and clinicians from the outset,” the spokesperson said.
In a statement to TheJournal.ie, CSL Behring said it has been working with clinicians and patients in Ireland for over a decade and provided Respreeza through its compassionate use programme “in hopes of a ‘yes’ decision by the HSE on funding”.
Additionally, we have been in dialogue with clinicians and the Health Service Executive since early 2016 and remain committed to exploring all possibilities to get this important treatment reimbursed for patients with [Alpha-1] in the Republic of Ireland for whom there is no alternative treatment available.
“CSL Behring has appealed the initial negative reimbursement decision and met with HSE in April to review new data. We are awaiting the response from HSE and hope to have another meeting with them in due course to review progress.
“The price CSL Behring has requested for Respreeza is in line with the price already approved in several EU countries and is substantially lower than the price reported by the media in Ireland recently.”
More information on the condition can be read on the Alpha One Foundation’s website.
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I believe way more people have this than is currently thought. A family member recently tested positive for this and it explains why as a family of people who have never smoked we have a high rate of COPD and lung diseases. I’m pretty tired of reading on here about people who can’t get access to drugs that will extend and improve their lives.
@Catherine Sims: Repreeza costs an estimated €84,000 per patient per year. It’s still under assessment in Ireland but but year it was turned down in Scotland because the trials showed no improvement in quality of life compared with placebo and ro reduction in chest infections, although lung density was better preserved. The ptoblem is that whilst things that get a lot of media attention but are novel, very expensive get funded in Ireland, its difficult to get funding for proven basic therapies that are much more cost effective. Orkambi for CF was approved last month but the HSE hasn’t been given a budget to pay for it yet. Orkambi is terribly over priced and, at the moment, some other more cost effective therapy or novel treatment will not be paid for as a result.
@Joe Harbison: Id like to see more than one countries experience with this drug. Clinical decisions around improvements may not be all the objective. I’ve worked with a research foundation and understand how studies work and understand that things aren’t always as clear cut as they may appear.
@Catherine Sims: Very true Catherine.Sometimes,and this is more often than one would expect,drugs that are seen to assist a patient ,while not having a huge impact on the conditions they suffer from can be released on to the open market for the sole reason of recuperating research and development costs,which I believe is wrong on many levels.
I remember the introduction of interferon way back about thirty years ago or so ,and it was announced to the world as the cure for cancer.It was vastly over priced and the lead researcher for the company admitted that it was released purely for financial reasons (recoup research costs) after a senate committee investigated its poor performance.
I can understand to a point how some may benefit from certain drugs and others do not.I also understand that companies will always give the positive side of their research projects..it is unfortunately basic sales 1.2..3… The industry is there to make money..the patient is secondary as history has shown us on so many occasions …
You would have to have a heart of stone not to feel for people who have this illness and who are desperate to have some quality of live and to live. But we have to be guided by expert scientific assessments and not emotionalism.
Again and again in recent times we have seen the issue of access to specialist drugs emerge as a problem and arguments in the media based on pure emotion. The health budget is finite. There are many underfunded areas affecting larger numbers of people ranging from primary care to prevention. More and more expensive drugs like this will emerge in the future for health conditions affecting small numbers of people. Choices will have to be made including the reality that perhaps by funding expensive drugs you are killing larger numbers of people elsewhere.
In 2001 my husband died from this aged 42 he was diagnosed only 2 years previous. At the time he struggled to breath most of the time. I don’t think there were any drugs available to help him so he would have been facing a lung and heart transplant. No one seems to know how many are affected but it appears to be genetic and any one with asthma should be automatically tested according to WHO.
Very sad but there’s no way this will get the media the Orkambi drug got.
No pictures of cute kids and teenagers to put out and manipulate the public with.
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