The news that the National Institute for Health and Clinical Excellence has had a rethink and will permit the use (under a special refund scheme) on the National Health Service of the cancer drug Velcade, developed by Millennium Pharmaceuticals and sold by Johnson & Johnson unit Janssen Cilag, has received a warm welcome.

The agreement is a landmark move, in that Velcade (bortezomib), which treats myeloma, a form of bone marrow cancer, had been rejected by NICE on the grounds of cost but the agency has now accepted in principle a refund scheme offered by the drug’s manufacturer. Specifically, NICE’s independent advisory committee has recommended that “all suitable patients” should be offered treatment and all those showing “a full or partial response to Velcade should be kept on it and funded by the NHS. However, patients showing a minimal or no response should be taken off the drug, and the costs refunded by the drug’s manufacturer.

Andrew Dillon, NICE’s chief executive, 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.”

The proposal has received the support of charities and patient groups and

Cancer Research UK's chief executive Harpal Kumar said: "We commend NICE for recommending this innovative approach and will respond positively to the consultation.” He added that it is essential “that patients get access to treatments that will benefit them” but “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.

Mr Kumar stated that "we have recommended conditional approval of new drugs in our responses to consultations on the subjects of NICE and pharmaceutical pricing” and “the proposed refund scheme for Velcade fits well with this idea. He concluded by saying that “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."

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 "perverse and unfair", particularly as the cost of the drug was just over the £30,000 threshold for NHS drugs. The guidance is subject to a three-week consultation, with final approval scheduled for October.

Velcade shines at ASCO

Meantime, Millennium has noted the promising results presented at the American Society of Clinical Oncology meeting in Chicago on Total Therapy 3 (TT3), a treatment for newly diagnosed multiple myeloma patients, which includes a Velcade-based therapy prior to and as maintenance following stem cell transplantation. The results, presented by the University of Arkansas for Medical Sciences and the Center for Cancer Research and Biostatistics, showed a significant improvement over Total Therapy 2 (TT2), a current standard of care which did not include Velcade.

The study included 303 patients who received two cycles of a combination called VTD-PACE (Velcade, thalidomide, dexamethasone, cisplatin, adriamycin, cyclophosphamide and etoposide) followed by VTD (Velcade, thalidomide and dexamethasone) once monthly for one year and TD (thalidomide and dexamethasone) once a month for two additional years.

Data from the Phase II study at two-year follow-up showed a complete response and near complete response rate of 84% with TT3, a statistically significant improvement over TT2 at 68%, and an event-free survival of 83% over 75% for its predecessor. Fewer patients on TT3, compared to TT2, experienced grade 3 and 4 side effects including tremor, constipation, syncope and thromboembolic events.