Story on Reuters:
Patients fared worse than expected, but scheme continues
* Experts say drug firms were main beneficiaries
By Kate Kelland
LONDON, June 4 (Reuters) - A scheme enabling multiple sclerosis patients to get expensive drugs paid for by Britain's state-funded National Health System has been a "costly failure", health experts and economists said on Friday.
In a series of commentaries in The Lancet, health economists said the scheme had wasted an estimated 50 million pounds ($74 mln) a year since its 2002 launch and should now be abandoned.
The drugs included in the scheme were Biogen Inc's (BIIB.O), Avonex, Bayer's (BAYGn.DE) Betaseron, and Merck KGaA's (MRCG.DE) Rebif, as well as Copaxone from Israel's Teva Pharmaceuticals (TEVA.TA).
"The scheme was a success for the drug companies, who sold at close to full price to the NHS," said James Raftery, a professor of health technology assessment at Southampton University. "For the NHS, however, the scheme can be judged only a costly failure."
Raftery said an assessment of the scheme in 2009 by its scientific advisory group, which included the drug firms, found that patients fared worse on the drugs than had been expected, suggesting the medicines were not cost effective. Yet the panel decided to continue with the project.
The risk sharing scheme was set up by the government in 2002 to make disease-modifying multiple sclerosis drugs available on the NHS after the country's health costs watchdog, the National Institute of Health and Clinical Excellence (NICE), ruled that they were not cost effective.
Under the terms of the scheme, the government agreed to pay for the drugs on the NHS while research was carried out to assess their long-term cost effectiveness. The agreement was that the NHS would then gradually stop paying for the drugs if patients did not appear to be benefiting.
In 2009, seven years after the scheme was set up, the first analysis of the data published in the British Medical Journal showed that patient outcomes were worse than predicted, but the scheme's scientific advisory group said it was too soon to reduce prices without further analysis.
Christopher McCabe, a professor of health economics at the University of Leeds, said in a separate analysis that the decision to delay a price review was not justified.
"It is difficult to see how they can justify such an expensive divergence from the scheme rules," he wrote.
Both experts, whose commentaries were also backed by several colleagues, questioned the independence of the advisory group, which included patients, doctors and drug firms.
McCabe added that if a proper assessment had been done after the first two years of the scheme, the NHS could have already saved around 250 million pounds ($369 million).
But Alastair Compston a professor of neurology at Cambridge University, who helped set up the scheme, argued that although it had not been run entirely properly or adequately governed, it had helped patients.
"Regardless of the scheme's outcome, it has advanced the situation for people with multiple sclerosis," he wrote. "Now that the principles of when and who to treat are better understood, more effective treatments can be developed." (Editing by David Cowell)
Full report in the BMJ---I copied a few pertinent sections below:
go to link for full report
The first report on the scheme was published in late 2009, with details of patients’ outcomes for 2005-7.3 Disease progression was not only worse than predicted by the model used by NICE,1 it was worse than that in the untreated control group. The primary outcome—the difference between actual and expected benefit as a percentage of expected benefit—was 113%, well above the 20% tolerance for price changes (any value above 0 indicates that benefit is less than expected). The report stated "the outcomes so far obtained in the pre-specified primary analysis suggest a lack of delay in disease progression."3
Other aspects of the scheme must also be questioned. Firstly, designed to confirm cost effectiveness, it seems to have been unprepared for the possibility that the drugs would be ineffective, something noted in 2003.14 The use of historical controls was an inherent part of the scheme, which was accepted and refined by the scientific advisory committee in 2002. The decision to pursue "alternative sources of data of progression in untreated patients"3 at this stage is extraordinary. Secondly, the design of the scheme around the Sheffield cost effectiveness model used by NICE has proved difficult. Several assumptions in the model did not hold in the scheme. One was that those patients whose disease progressed would discontinue taking the drugs—many did not. Another assumption, necessitated by the way the historical control data were reported, was that treatment could at best only slow the decline in patients’ disability scores. In the scheme, 38% of patients had improved scores at one year. However, the limitations of the extended disability status scales as a measure of disability were noted around the start of the scheme.14
None of these problems constitute compelling reasons for disregarding the terms of the scheme, which specified using the two year results to set the price. The fact that the prices would have had to be cut to zero (given outcomes were less than those of the controls) is hardly a reason for not proceeding. If patients are to continue to receive drugs through the scheme, big price cuts seem necessary.
The scheme was a success for the drug companies, who sold at close to full price to the NHS. It has also been a success for the companies making natalizumab (Biogen Idec and Elan International), which would not have been recommended by NICE in the absence of the scheme.
For the NHS, however, the scheme can be judged only "a costly failure"11 as suggested by the House of Commons Health Committee which has been raising concerns about the Scheme for several years. The biggest losers are the other NHS patients who would otherwise have benefited from the money spent on the scheme.