Updated 5.45pm with Health Ministry's reply
The father of 26-year-old woman with cystic fibrosis is pleading for the government to provide his daughter with life-saving EU-approved medicine.
Mandy Vella has just been discharged from Mater Dei hospital after an infection caused her deteriorating lung capacity to fall to 25 per cent.
Her father says she urgently needs an €18,000-a-month treatment called Trikafta, which was approved by the EU’s medicine’s watchdog in August last year but is currently unavailable in Malta.
“My daughter deserves to live,” he told Times of Malta. “I am her father. I will try anything to keep my daughter alive.”
He said that while he understood the cost was high, especially during a pandemic, the medicine was the only therapy available to help regenerate her lungs. “I don’t want to have to bury my daughter,” he said.
Cystic fibrosis is a genetic disease that affects around 20 people in Malta. It causes thick mucus that builds up in the lungs, digestive tract, and other parts of the body, leading to severe respiratory and digestive problems as well as other complications such as infections and diabetes.
How does Trikafta work?
Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene.
The Trikafta treatment is a combination of three drugs that target the defective CFTR protein and helps the protein made by the CFTR gene mutation function more effectively.
Some weeks ago, Mandy resorted to Facebook to speak up about the fact that her family cannot afford the medication she needs adding that her requests for help have been ignored.
“My life is being stamped with a price, the doors of the authorities are all closed. My wish for the only medicine that can save my life and the lives of all those Maltese who suffer from cystic fibrosis are being ignored,” she wrote.
Mandy's recent hospitalisation drove her father to join in to the appeals.
In a recent Facebook post, he said: “At the moment my daughter is at Mater Dei Hospital again with a lung related problem due to cystic fibrosis. Her lungs are deteriorating and deteriorating fast… How much longer are we going to wait?”
Times of Malta has asked the health ministry for the latest update. A spokesperson previously said the Government Formulary List Advisory Committee is discussing the issue.
'Inclusion in formulary list being discussed'
On Monday, a Health Ministry spokesperson explained that although a group of novel medicines for cystic fibrosis has been licensed and authorised by the European Medicines Agency, it did not mean that they would be automatically available in EU countries.
"Licensing of these medications is fluid and the situation is changing constantly. Nonetheless, discussions were held with the consultants of these identified patients and a caring pathway has been drawn up. This includes the latest medications required for each patient and health technology assessments.
"The inclusion of these medications in the Government Formulary List Advisory Committee is currently being discussed."