Wonder drugs for cystic fibrosis, deemed too expensive for the NHS, have been cleared by watchdogs for toddlers.
Last week, The Telegraph revealed draft recommendations from the National Institute for Health and Care Excellence (Nice), which said three drugs for the disease are not cost-effective and should not be funded by the NHS for future patients.
Nice said any patient who is already taking the drug before a final decision will remain so.
But until now, one of the Kaftrio drugs was only approved for people aged six and over, leaving parents desperate that their young children would never benefit from it and lives could be shortened.
The Medicines and Healthcare products Regulatory Agency (MHRA) has authorized these medicines for much younger children, from the age of two.
Wednesday’s change will likely see a flood of parents come forward in a desperate attempt to get their children on medication before Nice’s final decision.
The safety of the drug is under surveillance
For the first time, patients aged two to five years old with a certain type of cystic fibrosis were able to access the drugs Kaftrio and Kalydeco, taken together.
These drugs were already authorized for the treatment of cystic fibrosis with a common genetic mutation in patients aged six and older, and Wednesday’s announcement extends that authorization.
Julian Beach, Acting Executive Director for Healthcare Quality and Access at the MHRA, said: “We have prioritized the evaluation of Kaftrio and Kalydeco for this age group, taking into account the needs dissatisfied children with cystic fibrosis.
As with all products, we will closely monitor the safety of Kaftrio and Kalydeco in this age group.
Cystic fibrosis is an inherited disease that causes sticky mucus to build up in the lungs and digestive system.
This causes lung infections and problems digesting food. Symptoms usually begin in early childhood and vary from child to child, but the disease slowly worsens over time as the lungs and digestive system become increasingly damaged.
Miracle treatment
The NHS authorized Kaftrio for use in children aged six and over in 2020, with health officials later describing it as a miracle treatment.
The UK has the second highest prevalence rate of cystic fibrosis in the world, with around 10,000 people affected.
Only half will live beyond the age of 40, according to the NHS.
Research suggests the drugs can give patients decades, with one study finding Kaftrio can extend life by an average of 33 years.
The draft Nice guidelines published earlier this month are now open for consultation which runs until November 24.
A second commission is expected to meet in December to discuss its findings and deliver a final verdict.
Kaftrio and Kalydeco are taken by patients as long-term treatment to manage their cystic fibrosis symptoms.
The MHRA said extending authorization to younger age groups was supported by research into toddlers and young children.
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