A woman awaiting a transplant for a rare condition, atypical haemolytic uraemic syndrome (aHUS), an inherited condition that has destroyed her kidneys had her operation cancelled at the 11th hour because the Government refused to pay for the drug she needs to prevent the organ being rejected. Up to 70 patients are in the same position. They have been forced to put their lives on hold, and risk their condition deteriorating, while ministers and officials argue over whether the NHS can afford the drug, Eculizumab, which costs more than £300,000 per patient per year. The health minister, Earl Howe, rejected a recommendation from an expert committee that the drug be "routinely provided nationally".
Instead, Earl Howe referred the drug for further investigation by the National Institute for Health and Care Excellence (Nice), which took over responsibility for treatments for rare diseases. Nice has yet to explain how it is going to assess drugs for rare diseases. Nice's verdict on Eculizumab, brand name Soliris, is not expected before summer 2014. The drugs can be life-saving but are hugely expensive because they affect very small numbers of patients. They cannot therefore be assessed according to the usual cost-effectiveness threshold of £30,000 per patient per year (adjusted for quality of life) .
Experts fear the move could signal a tough new approach to the funding of expensive treatments for marginal groups. But that would undermine the role of a national health system to pool the risks for the whole population in order to fund individuals with exceptional health needs.
MP Madeleine Moon, chair of the All-Party Kidney Group, said: "It was heartbreaking to see the agony that people denied access to Eculizumab went through... The Government has made a heartless decision to allow existing trial and new patients access to the drug but not those who have been refused access on cost grounds by their hospital."
Professor Tim Goodship, chair of aHUS Action, said: "A transplant transforms people's lives. This opportunity has been dangled in front of them and then withdrawn. I feel it is just cruel."
Instead, Earl Howe referred the drug for further investigation by the National Institute for Health and Care Excellence (Nice), which took over responsibility for treatments for rare diseases. Nice has yet to explain how it is going to assess drugs for rare diseases. Nice's verdict on Eculizumab, brand name Soliris, is not expected before summer 2014. The drugs can be life-saving but are hugely expensive because they affect very small numbers of patients. They cannot therefore be assessed according to the usual cost-effectiveness threshold of £30,000 per patient per year (adjusted for quality of life) .
Experts fear the move could signal a tough new approach to the funding of expensive treatments for marginal groups. But that would undermine the role of a national health system to pool the risks for the whole population in order to fund individuals with exceptional health needs.
MP Madeleine Moon, chair of the All-Party Kidney Group, said: "It was heartbreaking to see the agony that people denied access to Eculizumab went through... The Government has made a heartless decision to allow existing trial and new patients access to the drug but not those who have been refused access on cost grounds by their hospital."
Professor Tim Goodship, chair of aHUS Action, said: "A transplant transforms people's lives. This opportunity has been dangled in front of them and then withdrawn. I feel it is just cruel."
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