Charity groups in the UK, and the drugmakers concerned, are jubilant over the news that the country’s National Institute for Health and Clinical Excellence has decided to review its own ruling denying patients NHS access to Velcade, a cancer treatment developed by Millennium Pharmaceuticals and sold by Johnson & Johnson unit Janssen Cilag.

Velcade (bortezomib), which treats myeloma, a form of bone marrow cancer, had been rejected by NICE on the grounds of cost but the agency would appear to have had a rethink. NICE issued a statement saying that as a result of the “unauthorised release of the appeal panel decision”, it is going to look again at Velcade following complaints by Janssen-Cilag, UK Myeloma Forum, Cancerbackup, Leukaemia CARE and a joint appeal from the British Society Haematology and Royal College of Pathologists about NICE’s draft guidance on the use of bortezomib.

NICE’s appeal panel ruled that as Velcade is the first in a new class of treatment then, "if it [NICE] should decline to recommend bortezomib for use on the NHS, [it] must explain fully its reasons for failing to recommend such treatment,” especially given that it had been accepted that the drug “would prolong, significantly, the life of patients with an incurable disease." The appeal had originally been made by Janssen and the charities who said that NICE’s initial rejection of Velcade was based mainly on the grounds of cost, rather than efficacy, and that this decision was therefore "perverse and unfair", seeing as the cost of the drug was just over the £30,000 threshold for NHS drugs.

Peter Littlejohns, NICE clinical and public health director, said it is important that the guidance NICE issues is based on “a robust evaluation and interpretation of the best available evidence of both clinical and cost effectiveness.” However, he added that “the appeal mechanism ensures that, if consultees feel our draft guidance is not appropriate, they have the opportunity to raise this in a formal setting” and until NICE issues final guidance on the use of bortezomib, “funding for it will continue to be determined at a local level, based on a local assessment of available evidence.”

Welcoming the news, Eric Low, chief executive of Myeloma UK said “we are grateful to NICE for accepting the validity of the appeal and for having responded in a positive manner. We hope that further review of the evidence will mean that myeloma patients get access to what is unquestionably a clinically effective and important treatment.” Tony Gavin, chief executive of Leukaemia CARE, added that this is welcome news for the three charities involved in the appeal “and indeed for NICE, because it shows that the appeal process does work.”

NICE’s change of heart is seen by pharmaceutical companies and patient groups as a hugely positive move and comes at a time when the UK parliament's Health Select Committee is gearing up for an inquiry into the workings of the agency which will look at why its decisions are increasingly being challenged and whether public confidence in NICE is waning, and if so why.