“The disease modifying treatments are essentially prophylactic, and like all drugs whose aim is to prevent events (relapses) that are unpredictable and relatively uncommon (the average relapse rate in multiple sclerosis is around one a year), their efficacy is difficult to assess even in large scale trials and impossible in the real world of clinical practice. If a treated patient is relapse-free for, say, two years, is that because of the efficacy of the drug or the natural course of the disease? No one knows.

To make things even more difficult, the calculations assessing whether disease modifying treatments might be affordable in the NHS were predicated on the putative effects of preventing the accumulation of disability in multiple sclerosis, not on relapse rates. To many this seemed hazardous—firstly, because only a proportion of patients (who are unidentifiable) become progressively disabled; and secondly, because the relation between relapses and disability is more complex and more indirect than many imagined. Reducing the frequency of relapses does not necessarily reduce the progression of disability. Indeed, studies with powerful monoclonal antibodies show that progression can continue unabated even when relapses are abolished completely.

In 2002, NICE judged the cost effectiveness equation unfavourable and ruled against provision on the NHS. But the Department of Health brokered a deal with the drug companies in the same year—the risk sharing scheme—whereby disease modifying treatments would be available within the NHS under the conditions of a large study. Treated patients would be regularly assessed and disability measured and recorded. If the drugs were more effective than the NICE predictions, and so achieved cost effectiveness, then all would be well. If not, there would need to be a financial reckoning—payback from the drug industry to the Department of Health or reduced drug costs—to achieve “affordability” post hoc.

The first substantial assessment of the accumulation of disability within the 5000 patients who were recruited and treated has now been performed. It found that patients taking disease modifying treatments have acquired more disability than an untreated historical control group. Cost effectiveness and Health Technology Assessment experts now call for the “deal” to be honoured and the risk sharing scheme discontinued—presumably meaning reversion to the original negative NICE judgment and so “their withdrawal from NHS practice”—or alternatively for the drug to be provided free by the drug companies.”

The multiple sclerosis risk sharing scheme: 

British Medical Journal - Published June 3 2010

http://www.bmj.com/content/340/bmj.c2882

New Studies Show the MS Drugs Don’t Slow Progression.

Ashton Embry - July 7 2010  

http://tinyurl.com/42bukpe