Thank-you very much if you have raised this issue already with your TD, please can you write again alerting them that since you last contacted them the drug company have resubmitted and that clarification would be appreciated on what it means that any new assessment will be assessed in a timely fashion. Could the Minister find out when the HSE drugs committee will assess the new application and when will we be informed of the outcome.
If you are unsure as to who is your local TD or how to contact them you can look it up here:
If you've not yet written to your TD, here's a sample letter that you can copy and paste:
Below is a template letter which you could copy, paste and personalise and send to you local TDs. There are only 5 boys in Ireland who could be helped by this treatment - we need your help to be heard.
Dear
I am writing to draw your attention to the issue of the reimbursement of the drug Translarna. Translarna is the first drug available to treat the underlying cause of Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD).
Duchenne Muscular Dystrophy is a rare progressive condition affecting mainly boys which results in worsening muscle weakness in most of the muscles of the body. This affects the ability to walk generally resulting in the need for a wheelchair at about the age of 11 and due to the muscle wastage of breathing muscles and the heart, results in premature death generally in the early thirties. Until recently treatment has been aimed at controlling the symptoms and not treating the underlying cause.
Translarna is a new drug that treats the underlying cause of nmDMD (a lack of dystrophin, a protein, which maintains the integrity of the muscle cells). It is not a cure but has been shown to slow down the progression of this devastating disease. There are only 5 boys affected by NmDMD in Ireland and only 2 eligible for the drug (eligibility is 5 year and ambulant).
In August 2014 the European Medicines agency gave conditional market approval for Translarna and since then it has been used in 22 other European countries including the UK and Northern Ireland. In October 2015 PTC (the drug company responsible for Translarna) submitted an application to get Translarna reimbursed here in Ireland. However, in January 2017 the HSE wrote to PTC to say that they were not going to reimburse Translarna as it was not deemed to be cost effective. The company itself challenged this decision legally.
On 11th December 2017 Simon Harris met with Dr Declan O’Rourke neuromuscular consultant, representatives of Muscular Dystrophy Ireland and the HSE to discuss this issue and at that meeting the Minister instructed the HSE to meet with PTC to come up with a sensible way forward. However, after this meeting the HSE contacted PTC and advised them to resubmit. As you can imagine given the first submission process took over 2 years and as this wasn’t the outcome agreed by the 11th December meeting this caused considerable stress. Initially PTC were not going to resubmit but have now dropped their legal action and resubmitted the application at the end of July.
I am writing to you to ask you to raise this important issue with the Minister for Health. There are only 5 boys in Ireland who will be eligible for this drug. Although due to such huge time delays two of those boys are becoming ineligible due to progressive loss of muscle power slowly taking away their ability to walk. It must be so hard to know that there is a drug out there that could help slow down this devastating disorder yet be unable to have it. It is very very hard to understand how 22 European countries have it available to their boys but Ireland doesn’t. 5 Boys don’t have much of a voice so I am asking you to speak up for them. Please could you ask the minister to supply us with a timeline of when the application will be processed. When will the drugs committee assess the new submission and when will we hear a decision.
I very much appreciate you taking the time to raise this very important issue with the Minister.
Yours sincerely,