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Department supporting local charity to secure access to ataluren

Wednesday, 7 September 2016

The Isle of Man Department of Health and Social Care (DHSC) is supporting local charity Finley’s Tracks in its efforts to secure access to a new treatment called ataluren (also known as Translarna) for a young boy who has a condition called Duchenne muscular dystrophy. 

Finley’s Tracks wants to raise money to fund the treatment. The DHSC can help to obtain ataluren and will try to secure the same specially negotiated and confidential discount price agreed between the manufacturer, US-based PTC Therapeutics, and NHS England.

The DHSC wishes to acknowledge the tireless work and dedication of Finley’s Tracks, the family of the young boy with the condition and others in the community campaigning to raise awareness about Duchenne muscular dystrophy and their efforts to secure access to ataluren.


The DHSC has a duty to provide the people of the Isle of Man with a comprehensive health service which is largely free at the point of delivery, but must do so within a finite budget set by Tynwald.

Making decisions on which new treatments to approve and fund – or decline – is a most difficult, emotive and sensitive aspect of the work of the DHSC – an organisation made up of dedicated, caring and compassionate professionals committed to delivering good quality health and social care for the people of the Isle of Man. New treatments are often very expensive.  Ataluren, for example, which is prescribed by weight of the patient, will, based on its list price, have a total cost per year of approximately £246,448 according to its manufacturer for an average 8-year-old child weighing 26 kg (4 st 1 lb).

Duchenne muscular dystrophy causes progressive muscle weakness from childhood, usually becoming evident at around three years of age: with patients eventually losing the ability to walk and becoming wheelchair dependent. Sadly the condition’s effect on the body usually causes death at around 30 years of age.

It is hoped that ataluren may, in time, be proven to delay the loss of the ability to walk in a patient with a particular form of Duchenne muscular dystrophy. The drug has undergone limited clinical trials and initial indications offer some hope, but the drug’s long term effectiveness and safety is at present considered to be unproven.  As a result, ataluren currently has a limited licence from the UK’s Medicines and Healthcare Products Regulatory Agency. The drug has been approved for use in England and Wales for five years under a special “managed access agreement”.  Under this agreement, the drug manufacturer has further time to collect data and demonstrate the drug’s effectiveness and at the same time provides the drug at a discounted price (which is confidential). This is, in effect, a rare instance of an NHS funded study/clinical trial.

Because of the finite amount of money available to the Island’s health service, care must be prioritised and new treatments, medicines and procedures have to be carefully evaluated in terms of their effectiveness – which means taking into account the likely benefit to patients as well as the potential cost to the taxpayer.

The work to prioritise new treatments is carried out by the DHSC’s Clinical Recommendations Committee, which is made up of a group of senior clinicians.  The committee considers evidence about new treatments and makes an objective and impartial recommendation on whether a particular treatment should be approved and funded – taking into account other demands upon the DHSC’s resources. It follows a clear process and makes recommendations against an ethical framework.

The uncertainty regarding ataluren’s long-term clinical effectiveness, its high cost and other demands on the DHSC’s resources meant that the Clinical Recommendations Committee could not recommend that the drug be funded.  The committee considered all of the documentation produced by the National Institute for Health and Care Excellence (NICE) in the UK, including detailed information on views of specialists, families and carers.

The prioritisation process is vital in ensuring an affordable and effective health service for all, as the DHSC cannot fund everything that is requested of it: meaning that difficult decisions will unfortunately be required on occasion. Making an exception for a single case could lead to every future funding decision being challenged.

Whilst ataluren is available in the United Kingdom, it remains unavailable in many countries, including the United States of America and the Republic of Ireland due to the lack of evidence of its long term effectiveness.

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