About ITP
Childhood ITP
Teenage ITP
Adult ITP
Medical Advisors
Research into ITP
Events
Convention
Seminar
Send for:-
Publications
American Perspective
Downloads
Leaflets and more....
Nplate (Romiplostim) receives NICE approval!
The ITP Support Association welcomes the decision by NICE to recommend approval of Romiplostim (Nplate) for the treatment of adults with chronic symptomatic ITP. The NICE appraisal committee recognised that this was an important addition in the treatment pathway for people who had failed standard treatments and who were at risk of bleeding.
NICE document entitled "Understanding NICE guidance, Romiplostim
for chronic immune (idiopathic) thrombocytopenic purpura",
is
available here.
Shirley Watson, who represented The ITP Support Association at the NICE meetings said " It was heartening that the committee at last understood that ITP is certainly not a trivial disease as described previously in the Appraisal Consultation Document. For patients who have severe symptoms and have run out of treatment options this new drug, which works by increasing platelet production rather than depressing or confusing the immune system, will give them fresh hope and the possibility of living life without the constant fear of bleeding. We are grateful to clinicians and patients who made their views known to NICE, with special thanks to our nominated clinical experts Dr Jennie Wimperis and Dr Nicola Cooper, and to patient expert Derek Elston for their determined efforts to get this drug approved."
Eltrombopag
The ITP Support Association was extremely disappointed that in October 2010 Eltrombopag received a negative Final Appraisal Determination from NICE (National Institute of Clinical Excellence) for its use in England and Wales. GSK have indicated that they wish Eltrombopag to be available throughout the UK, but they did not appeal within the time limit. We are maintaining a dialogue with GSK and await developments with interest.
Click here for the final appraisal determination of Eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura.
The Scottish Medicines Consortium (SMC) has completed its assessment of Eltrombopag, 25mg and 50mg film-coated tablets (Revolade®). Click here for the report.
The Appraisal Process
The ITP Support Association has been involved in two NICE Health
Technology Appraisals (romiplostin and eltrombopag) and here we outline
who is involved in the approval of our medicinal products. First stop
is the European Medicines Agency, known as the EMA.
The EMA is a remote part of the European Union with its headquarters in London, which has the responsibility to protect and promote public and animal health. It does this by evaluating and supervising medicines for human and veterinary use. It provides the European-wide licence (marketing licence) to sell products and it monitors the products once they are in use.
Under a centralised procedure, companies submit a single marketing authorisation application to the EMA. Once granted by the European Commission, a centralised (or 'Community') marketing authorisation is valid in all European Union (EU) and EEA-EFTA states (Iceland, Liechtenstein and Norway). For more information see www.ema.europa.org.
Then, as we all know, in the UK comes the Health Technology Appraisal (HTA). The National Institute for Health and Clinical Excellence (part of the NHS in the UK, www.nice.org.uk) performs this appraisal. The HTA, because it adds in the dimension of costs versus benefits and risks to the product evaluations, can result in a negative opinion. This causes a conflict with the positive marketing licence situation from the EMA. In reality, this means that people may be able to access a drug in one part of the European Union but not in another. So, what happens next?
The EMA has just produced its Road Map (strategic plan) for the next five years (to 2015). We have been commenting on parts of the document as we feel that it is important that the main processes involved in bringing treatments to patients should work together.
The EMA plan to focus on rare diseases where treatments are often limited and they plan to work more closely with the agencies performing Health Technology Appraisal. So, all this looks good until we read that "The Agency, therefore, envisages to make progress in this field, albeit in a stepwise manner, while continuing to ensure that cost/benefit assessment remains distinct and separate to the licensing process".