Alpha One Foundation calls on Minister to reconsider decision for patients who have no other therapy option -
Drug company, CSL Behring, urged not to stop therapy for 21 patients already receiving drug on "compassionate-use" basis -

 On behalf of patients with Alpha-1 (genetic emphysema), the Alpha One Foundation has expressed extreme regret at the decision by the HSE  ̶  which has today been formally communicated to it  ̶  that it has decided not to approve the reimbursement of the life-changing therapy, Respreeza.

The Foundation is calling on the Minister for Health to reconsider the decision which is a devastating blow to the estimated 60 patients who could benefit from the drug and whose health now hangs in the balance. Respreeza is the only therapy approved to treat the emphysema caused by severe Alpha-1 and is already available and reimbursed in 12 European countries.

Of the 60 patients, 21 have been fortunate to have been able to access a "compassionate-use" scheme operated by the drug manufacturer, CSL Behring, due mainly to their participation in a clinical trial. However, the future is also uncertain for these patients, as the company has previously stated that supplying Respreeza on a compassionate basis indefinitely is not an option. The Foundation is calling on CSL Behring to reconsider withdrawing the therapy and to show generosity of spirit in putting patients first.

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Prof Gerry McElvaney, the Consultant Respiratory Physician involved in the clinical trial of Respreeza in Ireland, and Chair of the Alpha One Foundation, comments:
"The drug approvals process is not working. The drug Respreeza has been shown to be effective in slowing progression of emphysema. The only question is one of cost. It is absolutely clear that those adjudicating on this matter are not in a position to comment or to make a decision on the efficacy of the drug. Respreeza definitely slows the progress of emphysema. All of the 21 Irish patients have experienced an improvement in the quality of their lives. No Irish patient died during the study and its extension, a time period of over 12 years. If the European Medicines Agency and 12 countries in Europe agree the drug works and is effective why is the HSE saying it isn’t?

"The longer patients are not receiving this drug the more lung damage they will suffer. This lung damage cannot be reversed. For those who have been fortuitous to have been able to access the therapy as part of CSL Behring's compassionate-use scheme, they are incredibly worried and anxious. There are no 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. The lack of proper engagement and communication with patients throughout this time has been appalling and goes back to a broken process. If the HSE really prides itself on its stated values of care, compassion and trust, these have been sorely absent."

Geraldine Kelly, Chief Executive,  Alpha One Foundation, comments:
"Today's decision is a huge blow for patients with severe Alpha-1 who have been campaigning for months for the approval of Respreeza.

"For these patients, Respreeza is the only approved drug which has been shown to slow down the progress of emphysema which is one of the severe effects of Alpha-1. They have met with public representatives, have presented their case before the Oireachtas Joint Committee on Health, and have marched outside Dáil Éireann, all the while expending a huge amount of effort and energy  ̶  often at considerable cost to their own health.

"We know Respreeza works in slowing the progression of emphysema which can have a hugely debilitating impact on people's lives. It is a much-needed lifeline for patients who have no alternative. In the absence of a positive decision, patients who have never been able to access the therapy will inevitably see their health continue to decline.
"It is not too late for the Minister for Health to reconsider this decision in the best interests of patients. This therapy is the only one available that has been shown to work in patients with emphysema caused by severe Alpha-1.

"We again urge that the Minister and the HSE look at the evidence. We ask them to consider the benefits to people's quality of life. We ask them to take into account the cost savings through reductions in hospitalisations and spending on other less effective medicines.

"Equally, we urge CSL Behring to put forward a cost that is realistic. We ask the company not to withdraw the therapy from the 21 people currently receiving it. These patients have been selfless in taking part in the clinical trial here, which has been of huge benefit to the company in demonstrating the efficacy of this therapy and their service should be recognised. Such a gesture would no doubt be noted by clinicians and future prospective patients when considering whether to support clinical trials.

"We urge the Minister, the HSE, and CSL Behring to pull back from the brink. We ask all parties involved to please put patients first."

Respreeza was approved by the European Medicines Agency in 2015 and is the only therapy documented in a clinical study to slow the progression of lung disease caused by Alpha-1. Clinical trial results published in The Lancet in 2015, and in The Lancet Respiratory Medicine in 2016, conclusively demonstrated a slowing down in the progression of emphysema by 34% in patients with severe Alpha-1. An Alpha One Foundation patient survey has also shown a decrease in the frequency and severity of chest infections and associated hospital admissions.

 

 

The Oireachtas Joint Committee on Health met on July 12th to discuss the process for the review and approval of Orphan Drugs.  The Alpha One Foundation represented approximately 60 Alpha-1 patients, at the meeting, 21 of whom are currently receiving a drug called Respreeza on a “Compassionate Use” basis and approximately 40 more who would benefit greatly from having access to the drug for the first time.    

The Alpha One Foundation outlined to committee members the case for the life-changing therapy, Respreeza, to be made available to these 60 patients in Ireland who are affected by the preliminary decision of the NCPE and the HSE not to approve reimbursement of the drug Respreeza. Members of the Alpha-1 Patient Action Group listened to the discussion from the gallery.

To view the committee meeting in full click here.  To read the transcript of the full proceedings click here.  

Respreeza was approved by the European Medicines Agency in 2015 and is now reimbursed in 12 European countries. During what was a very informative meeting the HSE committed to communicating a decision on the reimbursement of Respreeza within the next 3-4 weeks. Over the last few days the deadline for the continued distribution of the drug to the 21 patients currently receiving it on a "Compassionate Use" basis has been extended from 31st July to 31st August.  For more information on Alpha-1, visit www.alpha1.ie

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Pictured outside Dáil Eireann, Dublin 2, following a meeting of the Joint Committee on Health on the funding of drugs for rare conditions were from Left – Right: Brendan Gallagher, Gerard Finnerty, Ann O’Rourke (Alpha-1 Patient Group), Deputy John Curran (Fianna Fáil), Orla Keane (Alpha-1 Patient Group), Deputy Mary Butler (Fianna Fáil), Professor Gerry McElvaney (Alpha One Foundation), Senator Colm Burke (Fine Gael), Frank Jennings (Alpha-1 Patient Group), Geraldine Kelly (Alpha One Foundation), Josephine McGuirk, John Hannan (Alpha-1 Patient Group).

Want to get EDUCATED? Want to get INVOLVED? The IPPOSI Patient Education Programme is now open for applications. This is a 6-month blended-learning education programme, the first of its kind in Ireland, and is being piloted from September 2017 to March 2018 with a number of topics related to healthcare and the development of new treatments.

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The pilot programme is being led by IPPOSI in partnership with a number of education partners and will cover the following topics:

  • Understanding Clinical Trials – delivered by the University College Dublin Clinical Research Centre
  • Regulatory Affairs, Medicinal Product Safety, Pharmacovigilance and Pharmacoepidemiology – delivered by the Health Products Regulatory Authority (HPRA)
  • Health Technology Assessment principles and practices – delivered by the Dept. of Medicine & Therapeutics, Trinity College Dublin, in association with the National Centre for Pharmacoeconomics (NCPE)

All of the information you need to complete the application - including a Guide for Applicants, and a link to the online application form - are available on the IPPOSI website here. The closing date for applications is 5pm on Friday, June 30th 2017.

There is a webinar for those interested in applying taking place this coming Monday, 26th June, from 2 - 3 pm. This will cover an introduction to the course and a Q&A.  Register here

For more information about what IPPOSI does visit IPPOSI.ie

What is Alpha-1 Antitrypsin?
Alpha-1 antitrypsin is a vital protein produced by the liver to protect the lungs. It provides protection from the harmful effects of infections and inhaled irritants, particularly tobacco smoke. It can be easily measured by a simple blood test. 
What is Alpha-1 Antitrypsin Deficiency?
Alpha-1 antitrypsin deficiency (Alpha-1) is a genetic condition which, after cystic fibrosis, is the commonest genetic disorder in Ireland. It severely affects more than 15,000 people, with another 250,000 carriers also at risk of lung and liver disease on the island of Ireland. It is a proven genetic risk factor for chronic obstructive pulmonary disease (COPD).
How Do I Get Tested?
The Alpha One Foundation provides free testing for Alpha-1 as part of a national screening programme which is funded by the HSE. For more details ring 01-8093871 or email alpha1@rcsi.ie.