Friday 3 August 2012
Future of high-cost drugs for rare diseases
Baroness Thomas of Winchester, who spoke during a House of Lords debate last month on the future of high-cost drugs for rare diseases, has put the issue in the spotlight in the Letters to the Editor section of The Times published today.
Baroness Thomas wrote to The Times:
Sir, During a recent debate in the House of Lords on the future of high-cost drugs for rare diseases, it was announced by Earl Howe, the Health Minister, that the National Institute for Health and Clinical Excellence (NICE) is to take over the work in this important area.
The work is currently undertaken by the Advisory Group for National Specialised Services (AGNSS). AGNSS assesses the feasibility of very high-cost drugs to treat people with rare conditions, such as some forms of neuromuscular disorders. It is widely recognised for its impartiality and a comprehensive review process.
NICE's current "cost per quality-adjusted life year" approach to the appraisal of new drugs would rule out highly expensive treatments for small numbers of people with rare conditions. While assurances have been made that NICE will follow a process closer to that of the AGNSS, the Muscular Dystrophy Campaign and other patient organisations representing people with rare diseases have raised concerns. NICE has a history of placing cost-effectiveness at the heart of reviews. The 100 people in England affected with the rare, treatable condition Pompe disease for example, are now faced with the uncertainty of knowing whether NICE would deny them treatment on cost grounds.
It is essential that NICE puts the needs of people with very rare conditions at the heart of these reforms.
In support of Baroness Thomas' letter, the Muscular Dystrophy Campaign is seeking to establish details about the process that NICE is developing and whether there will be a ring-fenced budget for high-cost drugs for very rare conditions like Pompe disease in the new NHS Commissioning Board set-up.
You can read Baroness Thomas' speech during the House of Lords debate on the Parliament website.
For more information about the debate and the charity's parliamentary activity, please get in touch:
020 7803 4839
campaigns@muscular-dystrophy.org
