Alpha warrior: NI Grandmother 'won't see children grow up' after drugs trial cancelled over price
A Northern Ireland woman has said that a decision to end a drugs treatment trial will likely mean she never gets to see her grandchildren grow up.
Grandmother Maria Mannion suffers Alpha 1 antitrypsin deficiency (AATD). An avid campaigner to raise awareness of the illness, she likes to call herself the "Alpha 1 warrior".
Health authorities in the Republic took the decision to stop buying the drug Respreeza after they failed to reach a supply agreement with the manufacturer. The drug has been shown to slow the progression of lung disease caused by the genetic condition.
It is widely available across Europe, but the decision by the Republic's Health Service Executive means it is no longer available in the UK or Ireland on the health services.
Glengormley woman Maria was due to start treatment on the drug next month. Her lung capacity is at around 40% and small every day tasks are a struggle for her. Walking a short distance with a stick can pose a difficulty for the 61-year-old and she reverts to sign language to communicate with her husband when breathing becomes too difficult.
The retired teacher has suffered from the condition from birth and is highly susceptible to infection and has to carry antibiotics with her at all times.
She said she was disgusted when she heard of the end of the treatment programme and said if more people knew about it, there would likely be more funding for it.
"The drug can reduce the deterioration of the lungs and those that have been on it say they have noticed a real change and it has prolonged their lives.
"I was disgusted to hear the trial was ending, particularly for those that are still on it.
"Without it I believe I will struggle to see 70 and I will likely not see my three grandchildren grow up. It would be nice to see them become young adults."
Maria said that little is known of the condition and even her doctors in Belfast struggled when it was first raised. She said many people could possibly be misdiagnosed with the condition thinking it to be asthma.
"In my own family seven people have the gene but so little is known about it. When I said to a nurse about suffering from it, she asked if it was contagious.
"I wear a pedant which says 'Alpha 1 warrior' to try and raise awareness. If more people knew about it and to the extent it affects so many lives, there would no doubt be more done to help people and provide funding."
Thousands of other people are thought to be living with the condition with varying degrees of severity. It is estimated to cost about 85,000 euro per patient per year.
The Alpha One Foundation said 60 patients could benefit from the drug if the HSE could secure a price deal with its manufacturers.
Geraldine Kelly, the foundation's chief executive, appealed for health chiefs to make the necessary money available and for drug maker CSL Behring to cut its price.
"We have a therapy that works and that has been proven effective, and it is wrong of the HSE and the Department of Health not to fund it," she said.
The HSE said it was aware of the upset the decision had caused to those affected but it added that its research found Respreeza did not result in a significant improvement in quality of life.
The HSE said it has an onus to ensure that any new drug is cost effective.
It said: "The HSE has to have regard to its wider obligations to the 4.7 million population it serves and needs to maintain the full range of health services to all of the other patient groups within the finite resources at its disposal."
Manufacturers CSL Behring said it wanted a different outcome and was deeply disappointed.
"While we had hoped for a different conclusion, we remain committed to AATD research to improve the quality of life of people living with the condition," the company said.
It said it was looking at a new clinical trial across Europe, including Ireland, where severe Alpha 1 patients would be given the drug.
"We worked tirelessly to identify and agree on a solution that would work for both patients and for the HSE and give Respreeza the best chance of gaining reimbursement," CSL Behring said.