Health Minister Michelle O’Neill today announced that eligible patients in the north of Ireland will receive innovative new drugs for the treatment of very rare conditions.
The drugs, evaluated under the Highly Specialised Technology Programme of the National Institute for Health and Care Excellence (NICE), will be made available to patients with very rare conditions including atypical Haemolytic Uraemic Syndrome, mucopolysaccharidosis type IVa and Duchenne muscular dystrophy caused by a nonsense mutation.
In confirming her decision, the Minister said: “I am delighted to announce the availability of these drugs to allow patients with very rare conditions to receive pioneering new treatments. Whilst there are only a handful of people affected by these conditions, the new drugs have the potential to make a real impact on their quality of life.
“For the very first time, there are drugs available that could keep children with one form of muscular dystrophy out of a wheelchair for up to seven years; that could avoid end stage renal failure for people with atypical Haemolytic Uraemic Syndrome; or could relieve some of the symptoms of mucopolysaccharidosis type IVa.”
Drugs considered under NICE’s programme of Highly Specialised Technologies tend to be much more expensive that other medications. NICE is in the process of evaluating a range of technologies for the treatment of other very rare conditions including Fabry disease, Gaucher disease (type 1), paediatric-onset Hypophosphatasia and Lysosomal acid lipase deficiency.
Michelle O’Neill added: “These drugs are very expensive and whilst it is right that patients in the north of Ireland should have them, we must make sure that going forward our health and social care system works in the most efficient and effective ways possible to ensure that all patients and clients have access to evidence based services and medicines that will improve outcomes for them.
“That means difficult conversations and decisions ahead. It means supporting patients to make the right choices in looking after their own health and wellbeing. It means that clinicians and other professionals must be certain that a drug, treatment or a service is the best and most effective option for their patient or client on the basis of all the information and evidence available to them. It means that manufacturers of drugs and other medical technologies must work with the system to ensure that prices are as accessible as possible to ensure better outcomes for more patients. And it means that those who will redesign our system give proper thought and attention to the best configuration of services to make sure patients and service users in the north of Ireland have a world-class health and social care system that works for them.”
The Minister went on to say: “My Department is working with the HSC on a range of initiatives to bring more efficient and effective processes right across the system in prescribing, workforce planning and service delivery. I will continue to make decisions based on clinical advice and will work with my colleagues in the Executive to make sure that health and wellbeing remains a priority for all.
“There are many challenges facing us in health and social care. From today, access to the most innovative medicines for the rarest conditions is not a challenge for patients in the north of Ireland.”