Information via Rare Disease UK

In this update:

  1. RDUK AGM - registration now open
  2. Rare Disease Day - save the date
  3. Take action and support our open letter
  4. London Marathon
  5. How to identify rare disease patients - workshop
  6. Patients Involved in NICE (PIN)
  7. Department of Health review of NICE
  8. British Society for Gene and Cell Therapy

RDUK AGM - Registration now open

Our AGM will take place on the 19th of January 2015, at the Royal College of Paediatric and Child Health, 5-11 Theobalds Road, London, WC1X 8SH. A map to the venue is available here.

Guest speakers include: Sarah Stevens from Public Health England (PHE), who will update attendees on PHE's progress towards a rare diseases register and Professor Katie Bushby who will inform members about patient registries.

Tea and coffee will be available from 10:30am, with official business starting at 11:00am.
Lunch will be available from 1:00pm and the meeting will close at 2:00pm.

If would like to attend, please register here.

Rare Disease Day - Save the date

RDUK will be hosting our annual events to mark Rare Disease Day across Britain; registration for these events will be open in early January.

The events have now been confirmed so please save the following dates in your diary:

  • In England, our reception will be held in the Houses of Parliament on Wednesday 25 February 2015, 4:00pm - 6:00pm.
  • In Scotland, our reception will be held in Holyrood (Scottish Parliament) on Tuesday 24 February 2015, 6:00pm - 8:00pm.
  • In Wales, our reception will be held in the Senedd (Welsh Assembly) on Tuesday 3 March 2015, 6:00pm - 8.00pm.

We will be in touch to let all members know when registration opens.

Don't forget to write to your MP!

Please write to your local MP and ask them to support our open letter to the leaders of the UK's main political parties. The letter has been sent to David Cameron, Ed Miliband and Nick Clegg asking them to reaffirm their commitment to the UK Strategy for Rare Diseases. You can read the letter here.

It's been a year since the publication of the UK Strategy for Rare Diseases, which aims to ensure that people living with a rare disease have access to the best care and treatment that health and social services can provide. There's been much progress - but there is still a risk that all 51 commitments in the UK Strategy for Rare Diseases will not be implemented. We must act now to ensure that patients affected by rare conditions and their families do not lose out. To keep up the momentum we need as many MPs as possible to support the letter and ask their party leaders to commit to it in their manifestos.

You can find out who your local MP is here and a template letter to send them is here.

News from our members

London Marathon

The Tuberous Sclerosis Association (TSA) is hiring the Abbey Centre in Westminster for the London Marathon this year. The venue is open from 8.30am to 4.30pm and has showers, somewhere for runners to store belongings and meet with friends and family after the run. This year there is space for at least two other charities to share the venue. The TSA has calculated that split three ways it would cost about £150 per charity. This includes some money for providing food and bubbly for the runners and supporters.

If you represent a small rare disease charity and would like to join in with the TSA on the day get in touch with Emma Damian-Grint. The TSA are happy to do all the organising!

Findacure's 'how to identify rare disease patients' workshop

Findacure’s next workshop in their series of training sessions for patient groups will take place on Friday 30 January 2015, on the topic of identifying people living with rare diseases.

The workshop will be a half-day event taking place between 1:30pm to 5:30pm in Central London (near Bank station). The day will explore different methods for identifying patients in order to build communities and recruit for clinical research. Throughout the day, there will be short group discussion sessions where delegates will be able to share their own experiences and work together to apply the advice from the day to their own organisations. A schedule for the workshop is available here.

For more information or if you would like to attend, please contact Flóra Raffai by Friday 16 January 2015.

Findacure are also organising a scientific conference on drug repositioning for rare diseases. You can find out more about it here.

Other news

Patients Involved in NICE (PIN)

Patients Involved in NICE (PIN), is a coalition of over 80 patient organisations and is committed to enabling patient groups to engage productively with NICE. Membership is free and open to patient organisations. If you are interested in joining PIN, please contact RDUK's Executive Officer, Farhana Ali, who is a member of PIN's Executive Group.

PIN is independent from NICE and the pharmaceutical industry and acts as a critical friend to NICE. It is a respected and equal partner in developing and shaping aspects of NICE’s work. PIN provides a forum for enabling patient groups to engage with NICE, by working alongside NICE’s patient and public involvement team.

Department of Health review of NICE

Consultation closes 2 January 2015

The Department of Health reviews its arm’s length bodies once every 3 years. The review for NICE is now open and will look at what NICE does.

The Department of Health wants to know what you think of NICE, including:
· whether NICE needs to continue in its current form
· how NICE is performing
· whether NICE is providing good value

You can also share your views in person at a workshop being held in London. You can register online for a place at the workshop which takes place on the 18 December at 11:00 am 1:00pm here.

British Society for Gene and Cell Therapy

The British Society for Gene and Cell Therapy are in the process of developing a section on their website that will signpost patients to relevant patient support groups who would be able to provide more information and support patients with accessing treatments and services to meet their needs. This “Patient Information” section will also have basic information on a variety of genetic disorders along with current gene and cell therapy clinical trials.

If you would like to add your patient group to this page, please provide a short paragraph describing the work of your group, contact details and a web-link where patients could find more information to Mustafa Munye.