Hardly a cure
In a comparative study in 669 patients with high-dose dexamethasone (a corticosteroid used to treat multiple myeloma), the time taken for the disease to get worse was, on average, 189 days with Velcade and 106 days with dexamethasone – hardly a cure, but an improvement nonetheless over existing therapy. In two other studies in which there was no comparison with other treatment, around a third of the 256 patients responded partially or completely to treatment with Velcade.
Side effects
The treatment is not risk-free. Side effects include shingles, low blood platelet counts, anaemia, too few of the white blood cells that fight infection, reduced appetite, numbness and tingling, headache, breathlessness, nausea, diarrhoea, vomiting, constipation, rash, muscle pain, fatigue and fever. But all the patients studied had received at least one previous treatment for their disease, and their disease was getting worse during their most recent treatment. In other words, there was little alternative.
The medicines agency's experts decided that Velcade's benefits were greater than its risks, and recommended it should be given marketing authorisation. The authorisation was granted under an ‘exceptional circumstances' provision of EU law, because it had not been possible to obtain the complete range of scientific information that is usually demanded for a new medicine. But the need was pressing, and in desperate cases some treatment – even imperfectly tested – can be better than no treatment. The company that makes Velcade will carry out further studies, looking in particular at the how side-effects may be reduced and efficacy enhanced, and the authorities will constantly review any new information that becomes available.
