Alpha-1 Foundation Ireland today (November 15th) welcomes the decision of the HSE/Department of Health and CSL Behring to approve the continued supply and administration of the life-changing therapy, Respreeza, for a small group of patients with Alpha-1.  The development is a huge relief to 19 of 21 patients who have been receiving the therapy for over 10 years on a “compassionate-use basis”, from CSL Behring, the makers of the therapy.  Regrettably two of the original 21 patients died while off the treatment in 2017 as the supply and administration of the therapy was stopped for a period of 6 weeks. 

Respreeza was approved by the European Medicines Agency in 2015 and is the only therapy proven in a clinical study to slow the progression of emphysema caused by Alpha-1.  An Alpha-1 Foundation Ireland patient survey has also shown a decrease in the frequency and severity of chest infections and associated hospital admissions. Respreeza is already available in 12 European countries.

Geraldine Kelly, Chief Executive, Alpha-1 Foundation Ireland, comments:
“Today’s decision brings to an end a hugely stressful and difficult time for these 19 patients and for the families of the late Anna Cassidy and Marion Kelly who campaigned for over a year for the approval of Respreeza.  The Alpha-1 Foundation Ireland is pleased that a mutually-acceptable arrangement has been agreed between the HSE and the manufacturer of the therapy CSL Behring. 

“Today is a good day for a very small number of Alpha-1 patients, however we cannot forget that there are a further 40 or so patients who were not part of the “compassionate use programme” and who would benefit from this treatment.  For these patients the wait continues until a re-submission by the makers of Respreeza, CSL Behring is made to the National Centre for Pharmacoeconomics (NCPE) for full reimbursement approval.  For patients with severe Alpha-1, Respreeza is the only approved drug which has been shown to slow down the progression of emphysema which is one of the most severe effects of Alpha-1.  The Alpha-1 patient group supported by Alpha-1 Foundation Ireland will continue to campaign on their behalf.

“Having been through the drugs approval process, we are firmly of the view that the system of approving new therapies in this country needs to work better for patients.  With rapid developments in the area of personalised medicine, the methodologies used in assessing new therapies needs to be overhauled.  There needs to be transparency, a clear process, clear timelines, greater involvement of patients and clearer communication with all parties. Alpha-1 Foundation Ireland is very happy to contribute our insights into developing a more effective national system for the reimbursement of new therapies.

To watch a short film about Alpha-1 launched as part of the campaign for access to Respreeza click below.