HSE U-turn 'bitter-sweet' for family of brave campaigner who died after trial drug withdrawn
The family of a woman who died last year after a trial drug that gave her a "new lease of life" was withdrawn have welcomed the HSE's decision to fund it for a number of other patients.
But they said it was a bitter-sweet moment.
Marion Kelly (53), of Nenagh, Co Tipperary, who suffered from a rare genetic disorder Alpha-1 antitrypsin deficiency said the drug Respreeza had turned her life around.
But she was devastated after the drug trial ended and she led a campaign to get the HSE to fund it.
One year after her death, the HSE said that it will make the treatment available to 19 other patients with the disease, but around 40 others are not included.
"This is a campaign that Marion started and was very passionate about while she was alive and would have wanted us to fight until a positive resolution was found for her fellow Alpha-1 patients. Thankfully, today that fight ends and Marion can rest in peace," her family said yesterday.
"For nearly three years, the Alpha-1 patients have had to live with a lot of uncertainty around the supply of Respreeza, the drug that has given them such a good quality of life for 11 years.
"They had to endure a period of time without the drug and during this time Marion and Anna Cassidy tragically passed away.
"At a time when the patients should have been focusing on their health, they and their families had to fight for the right to not have this drug and effectively their lives taken away from them. There is still a massive question mark as to why that happened and that's a question mark that rests on Marion's grave."
Geraldine Kelly, chief executive of Alpha-1 Foundation Ireland, said the decision brings to an end a stressful and difficult time for 19 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 progress of emphysema, one of the most severe effects.
"The Alpha-1 patient group, supported by the Alpha-1 Foundation, will continue to campaign on their behalf."
She said the system for approving new therapies needs to work better.