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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