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.”
Notes to editors:
1. NICE is a Non Departmental Public Body tasked with producing guidance on good clinical practice and the cost-effective use of NHS resources in England. NICE publishes a number of different types of guidance. On 1 July 2006, the Department of Health established formal links with NICE whereby all Clinical Guidelines, Interventional Procedures Guidelines and Technology Appraisals published by the Institute from that date would be locally reviewed for their applicability and, where appropriate, endorsed here.
2. Highly Specialised Technology (HST) evaluations are recommendations on the use of new and existing highly specialised medicines and treatments in the NHS. The HST programme only considers drugs for very rare conditions. Sometimes these medicines are referred to as “ultra orphan drugs”. An orphan drug is a term used to describe a treatment for a disease which affects fewer than 1 in 2000 people. The concept of an “ultra orphan” drug has been proposed for those medicines that are used to treat conditions that affect fewer than 1 in 50000 people. NICE took on responsibility for the evaluation of HSTs in April 2013.
3. HSTs tend to be priced much higher than other NICE-approved drugs. This reflects the rarity of the condition and the smaller window of opportunity for manufacturers to make a return on costs. That said there are mechanisms whereby discounts may be applied to the list price.
4. NICE published its first HST in January 2015 - HST1: eculizumab for treating atypical Haemolytic Uraemic Syndrome (aHUS). aHUS is a life-threatening disease causes inflammation of blood vessels and the formation of blood clots throughout the body. People with aHUS are at constant risk of sudden and progressive damage to, and failure of vital organs, particularly the kidneys.
5. NICE published its second HST in December 2015 – HST2: elosulfase alfa for treating mucopolysaccharidosis type IVa (MPS IVa). MPS IVa is an extremely rare, life-limiting, inherited lysosomal storage disease with a wide range of symptoms that typically appear in early childhood and worsen over time. These include joint and skeletal abnormalities, hearing and vision loss, heart valve disease, pain, fatigue, and progressive loss of endurance leading to increasing dependence on wheelchairs. MPS IVa leads to reduced life expectancy - the average life expectancy in people with this condition is about 25 years - primarily because of respiratory failure and heart problems (63% and 15% of deaths respectively). Until this drug became available, there were no medications available to treat the underlying disease.
6. NICE has today issued guidance recommending the use of ataluren (the generic name for Translarna) for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. DMD is a severe, progressive X-linked recessive disorder which predominantly, though not exclusively, affects males. Duchenne muscular dystrophy with a nonsense mutation is caused by a single base variation in a person's DNA which leads to incomplete dystrophin production in muscle. Symptoms of DMD appear by the age of 3 years. The main symptom is motor dysfunction, but as the disease progresses, major vital organs such as the gastrointestinal tract and heart are affected. People with DMD experience a decline in physical functioning with subsequent respiratory and cardiac failure which leads to death, usually before the age of 30.
7. Research has predicted that ataluren may delay the loss of walking for up to seven years. A Managed Access Agreement has been negotiated with the manufacturer to reduce the cost of the drug to the NHS for the next five years.
8. Prior to today’s decision to fund HSTs, patients would have relied on receiving these drugs either via an alternative arrangement with the manufacturer or through an application for an individual funding request.
9. Media queries to DoH Information Office on 028 9052 0579, or out of office hours contact the Duty Press Officer via pager number 076 9971 5440 and your call will be immediately returned. Follow us on Twitter @healthserviceni
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