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RDUK press release: Reminder: Your input needed on recommendations to develop a strategy for rare diseases

Posted on 15th October 2010 by info@pnh-alliance.org.uk

Rare Disease UK have issued a press release as a reminder of the need for recommendations on a strategy for rare diseases. Please find the press release below:

Reminder: Your input needed on recommendations to develop a strategy for rare diseases

Deadline 29th October
Thank you to all those who have responded to the Rare Disease UK consultation paper so far. We want as many people as possible to respond. The more responses we receive, the more evidence we can gather, the more informed the recommendations will be and the more weight they will carry. Even if you just respond to say you agree with the recommendations, please do!

Background:

For the past year Rare Disease UK, with the guidance of our five Working Groups, has been working on a report to develop recommendations on what should be included in a strategy for rare diseases. The final report will be launched to coincide with Rare Disease Day 2011 on February 28th.
We want your feedback on our findings so far and your evidence detailing your own valuable experience to inform the final report.
The health departments of the four UK home nations have all signalled their willingness to take our findings into consideration when developing a strategy for rare diseases. Your input could help shape this vital strategy.
You can download the consultation document outlining our findings so far here. This document offers an overview of the initial recommendations of the Working Groups and it is aimed at stimulating further input.
We would appreciate contributions from all interested parties. That includes patients and family members, clinicians, researchers, academics, industry members, and any other groups interested in or affected by rare diseases.
Please feel free to distribute the consultation document to anyone who may have an interest in responding.

Download the consultation document (pdf)

Responses are requested by Friday 29th October 2010 in order to allow time for production of a final report by Rare Disease Day 2011.

Please send your responses to Lauren Limb either by email:  lauren@raredisease.org.uk, or post:
Rare Disease UK,
Unit 4D Leroy House,
436 Essex Road,
London,
N1 3QP.
We look forward to your responses.

Best wishes,

Stephen Nutt

RDUK Secretariat

RDUK press release: Your input on recommendations to develop a strategy for rare diseases

Posted on 22nd September 2010 by info@pnh-alliance.org.uk

Please find below the latest RDUK press release regarding the consultation:

Wanted: Your input on recommendations to develop a strategy for rare diseases.

Dear Rare Disease UK member,

For the past year, Rare Disease UK, with the guidance of our five Working Groups, has been working on a report looking at developing recommendations on what should be included in a strategy for rare diseases. The final report will be launched at four parliamentary receptions to coincide with Rare Disease Day 2011 (February 28th 2011).

We want your feedback on our findings and your evidence to inform the final report.

Each of the four health departments in the UK has signalled their willingness to take our findings into consideration when developing a strategy, so your input could help inform a strategy for rare diseases.

We have produced a consultation document outlining our findings. This document offers an overview of the discussions of the Working Groups and it is aimed at stimulating further input. We would appreciate contributions from all interested parties, whether as an individual or on behalf of an organisation, from patients and patient organisations, to clinicians, researchers, academics and industry members to provide insight on the topics discussed.

Please feel free to distribute the consultation document to anyone who may have an interest in responding.

The consultation document is accessible by clicking here (pdf) or by visiting: http://www.raredisease.org.uk/documents/consultation_rduk.pdf

Responses are requested by Friday 29th October 2010 in order to allow time for production of a final report by Rare Disease Day 2011.

Please send your responses to Lauren Limb at lauren@raredisease.org.uk, or to:
Rare Disease UK,
Unit 4D Leroy House,
436 Essex Road,
London,
N1 3QP

We look forward to your responses.

Best wishes,
Stephen

Rare Disease UK Secretariat

RDUK press release: Newsnight report highlights concerns about GP NHS Commissioning for patients with rare diseases

Posted on 26th August 2010 by info@pnh-alliance.org.uk

Please find below the RDUK press release regarding the recent Newsnight report:

Newsnight report highlights concerns about GP NHS Commissioning for patients with rare diseases.

Dear Rare Disease UK Members,

Newsnight featured a report yesterday, 24th August, outlining concerns about the impact of the government’s plans to introduce GP Commissioning on patients with rare diseases.

The story was as a result of research by the Muscular Dystrophy Campaign highlighting that GPs have poor levels of understanding of muscular dystrophy resulting in cases of misdiagnosis and inappropriate referrals, care and advice.

The report also featured a case study of a mother whose 12-year-old son with duchenne muscular dystrophy was misdiagnosed twice, and the views of spokespeople from the British Medical Association’s GP Committee. Unfortunately the Government did not send a Minister to comment on the issue.

To watch the programme follow the link to BBC’s iPlayer.

To read the Newsnight article about the report click on the link to Newsnight’s website.

Rare Disease UK will be responding to the Government’s White Paper, Equity and Excellence: Liberating the NHS outlining our concerns and a draft response will be circulated in due course.

The European Union council recommendation

Posted on 5th June 2009 by info@pnh-alliance.org.uk

The European Union makes a council recommendation on action in the field of rare diseases:

• Ensure that rare diseases are adequately coded and classified
• Enhance research in the field of rare diseases
• Identify Centres of Expertise by the end of 2013 and foster their participation into European Reference Networks
• Support the pooling of expertise at European level
• Share assessments on the clinical added value of orphan drugs
• Foster patient empowerment by involving patients and their representatives at all stages of the decisionmaking process
• Ensure the sustainability of infrastructures developed for rare diseases

The full Council paper can be found at the following link:
http://ec.europa.eu/health/ph_threats/non_com/docs/rare_rec2_en.pdf

See the activities of Rare Disease UK on the development of a National Plan for Rare Diseases:
http://www.raredisease.org.uk

All Wales Medicines Strategy Group

Posted on 29th April 2009 by info@pnh-alliance.org.uk

The PNH alliance welcomes the announcement from the All Wales Medicines Strategy Group on funding for PNH services:

Recommendation of AWMSG

Eculizumab (Soliris®) is recommended for restricted use within NHS Wales according to agreed guidelines for the treatment of paroxysmal nocturnal haemoglobinuria.

AWMSG is of the opinion that eculizumab (Soliris®) is not suitable for shared care within NHS Wales.

AWMSG recommends that eculizumab (Soliris®) should only be used on an individual patient basis according to agreed guidelines.

• AWMSG considers that eculizumab (Soliris®) satisfies the AWMSG criteria for ultra orphan drug status.
• AWMSG will review the evidence on the use of this medicine within a period of three years.

Parliamentary announcement regarding funding

Posted on 19th October 2008 by info@pnh-alliance.org.uk

Below is the parliamentary announcement confirming the positive funding decisions for PNH patients in England (Extract from Hansard 8/10/2008, column 16WS – 17WS)

Paroxysmal Nocturnal Haemoglobinuria

The Minister of State, Department of Health (Dawn Primarolo): Over the summer, Ministers received advice reflecting the deliberations of the National Commissioning Group (NCG), the National Specialised Commissioning Group (NSCG) and strategic health authority chief executives about taking a national commissioning approach to the paroxysmal nocturnal haemoglobinuria (PNH) service, including the issue of Eculizumab’s availability on the NHS. We have given these complex issues very careful consideration.

The Government can now confirm that Ministers have decided that the NHS should nationally commission the PNH service for clinically eligible patients from 1 April 2009, through the National Commissioning Group. In taking this view, Ministers have taken account of the following key points:

• research has provided good evidence that Eculizumab is a highly effective treatment for this condition for a number of patients;
• treatment is long-term and life-long for most patients;
• the service to treat this condition meets the criteria for national commissioning; and
• the ‘national’ risk pool approach offers significant benefits to PCT commissioners who are not then vulnerable to multiple high cost patients.

The National Specialised Commissioning Team will now make arrangements with NHS stakeholders to ensure that arrangements for the national commissioning of PNH services are in place for 1 April 2009. They will also be considering with the drug’s manufacturer and with the local NHS any transitional issues that need to be managed over the next six months.