Good News on access to drugs for rare conditions: Thank you, Minister!

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 Minister has confirmed that 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.

The Minister said: “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.”

This is very welcome news, which will transform lives.
We will continue to work closely with the Department, and with the HSC to ensure that those living with rare diseases are not disadvantaged simply because of the rarity of their condition; and to support changes to make the system more efficient and effective

Stronger Together

Over a thousand people, holding hands across the River Foyle. Radio, local press, YouTube video. Families, volunteers, the mayor and a jazz band. A fun day with activities. There was even sunshine.

What was it all about?

To raise awareness of the charity Shine, and to fundraise and support people living with the conditions Spina Bifida and/or hydrocephalus.

Those of us living and working with rare disease know that we can’t do everything ourselves; we need support, help, and even just a listening ear.

That’s why NIRDP exists. Join us or contact us. Help us to support each other.

We are stronger together.

 

What do you want the UK rare disease plan to include?

Those of us who live or work with rare disease in NI have an important opportunity just now. It’s the chance to make our voices heard- to get politicians and policy makers to hear what’s important to us, what will improve the care and quality of life for those affected by rare disease.

The four UK health departments have together produced a consultation document to form part of the basis for a UK plan on rare disease. The rest of the basis for that plan will come from the responses to this document: this is our chance to influence strategy.

NIRDP will be responding to the consultation as a partnership, and are also encouraging everyone with an interest in rare disease to respond individually. The greater the number of responses, the greater the impact. The government need our help to get this right. We have a chance now to get our stories heard, and the government have to respond.

At the NIRDP/RDUK event on 8 May, there was some valuable information, tips and advice on responding to the consultation document from Nick Meade, Policy Analyst.

  • Show your experience, establish why your opinion should be considered.
  • Don’t be constrained by the questions asked.
  • Only cover the topics you want to.
  • Identify any gaps- what have they missed out?
  • Give evidence from your experience to back up your point.
  • What is good about the plan? Let them know.
  • When being critical, give suggestions for improvements.
  • Be reasonable.
  • Praise, criticise, praise.

There will probably be one indidvidual tasked with reading all the responses, so make it easy for him/ her to grasp your points.

  • Be clear and concise.
  • Consider the layout.
  • Have an introduction and conclusion, use bullet points.
  • Ask someone with no interest in rare disease to read it first to make sure it makes sense to a lay person.

 

email your response to rarediseasesconsultationresponses@dh.gsi.gov.uk

post to Sarah Bramley-Harker, Department of Health, Screening and Specialised Services Team, Room 5W35, Quarry House, Quarry Hill, Leeds, LS2 7UE

contact your politicians– give them real examples of what you need to see in your area. What will make a difference to their constituents? Remind them that, collectively, rare diseases are not rare and that, since services are patchy and poorly integrated, patients and families can struggle to get the help and support they need.

contact NIRDP– if there are particuar points you want to be sure the NIRDP response also includes, contact us: info@nirdp.org.uk, phone 0800 917 0222 and talk to Sarah McCandless, or post to 6 Ava Crescent, Belfast, BT7 3DU.