Bone cancer sufferers hailed a decision today by the Government's health watchdog to reverse a ruling which prevented them from obtaining a life-extending drug from the NHS.
The National Institute for Health and Clinical Excellence (Nice) has recommended a scheme to fund bortezomib (commonly known as Velcade) for multiple myeloma sufferers in England and Wales who respond well to it.
Under the scheme - the first of its kind considered by Nice - the drug's manufacturer would refund the NHS when the treatment does not work.
The turnaround follows an appeal by the cancer charities Myeloma UK, Cancerbackup and Leukaemia CARE, who argued Nice's initial rejection of Velcade six months ago was based mainly on the grounds of cost, rather than effectiveness.
They said the decision was "perverse and unfair", particularly as the cost of the drug was just over the £30,000 threshold for NHS drugs.
Under the draft guidance issued today, Nice recommends that "all suitable patients" have access to the treatment, with the NHS continuing to fund the drug for those who show a full or partial response.
Patients who do not benefit should be taken off the treatment, with the cost refunded by the manufacturer, Janssen-Cilag, Nice said.
It said the new guidance followed an evaluation of a refund scheme put forward by Janssen-Cilag.
The guidance is subject to a three-week consultation, with final approval tabled for October.
Nice chief executive Andrew Dillon said: "We are aware of the challenge that the NHS faces in ensuring that patients can access expensive, but potentially effective, treatments for life-threatening conditions such as cancer.
"If the drug's manufacturer accepts the proposals we are consulting on today, it will mean that when the drug works well the NHS pays but when it doesn't the manufacturer should bear the cost.
"All patients suitable for treatment will get the chance to see if the drug works well for them."
Nearly 4,000 people in the UK are diagnosed with myeloma each year and there are 20,000 myeloma patients in the UK at any one time.
The life expectancy of a newly-diagnosed myeloma patient is three to five years.
Myeloma patient Jacky Pickles, 45, one of the "Velcade Three" group of women who have been campaigning for Nice approval of the drug, said: "We are absolutely delighted with this positive decision.
"But we want to make sure that there is no discrimination and that all sufferers get the treatment if they need it."
She also praised Janssen-Cilag, saying: "They obviously feel strongly that their product is effective. They are prepared to put their money where their mouth is and foot the bill when patients don't respond."
Myeloma UK, Cancerbackup and Leukaemia Care welcomed the "creative" scheme, but said they wanted to be sure it was the best deal for all patients.
"We very much welcome the fact that Nice has agreed that this important drug should be made available to myeloma patients, as it is without question clinically effective," said Eric Low, chief executive of Myeloma UK.
"However, we now need to carefully consider the details of the draft recommendation as we have concerns that it may exclude some patients who could benefit.
"We will be seeking clarification on the proposed guidance throughout the open consultation process and hope it will ensure that all patients who could benefit from the treatment can get access to it. We strongly believe that no patient should die without having had appropriate access to Velcade."
Cancer Research UK chief executive Harpal Kumar said: "We commend Nice for recommending this innovative approach and will respond positively to the consultation.
"It is essential that patients get access to treatments that will benefit them. Sadly, issues of cost are accentuated where drugs only help some patients, especially if there is no way of knowing which patients are likely to benefit.
"We have recommended conditional approval of new drugs in our responses to consultations on the subjects of Nice and pharmaceutical pricing. The proposed refund scheme for Velcade fits well with this idea.
"Pharmaceutical companies with confidence in the new treatments they are developing should not be frightened of schemes aimed at pricing treatments according to their benefit.
"Enabling the NHS to get value out of the money it spends on drugs will improve its ability to afford new treatments for cancer patients."