The National Institute for Health and Clinical Excellence has published final guidance for the National Health Service in England and Wales on the use Millennium Pharmaceuticals and Janssen Cilag’s Velcade for the treatment of the bone cancer multiple myeloma.

The Institute has given its seal of approval to the landmark money-back scheme proposed by the company, under which the NHS will fund the treatment of patients showing a full or partial response to Velcade (bortezomib), while the costs incurred for those not responding will be refunded by the drugmaker.

“Providing expensive new cancer drugs is a challenge for the NHS, particularly when it is not possible to identify those patients who will benefit most from treatment,” commented the Institute’s chief executive Andrew Dillon. “The scheme…will ensure that patients at first relapse, who have had one prior therapy and who have had or are unsuitable for a bone marrow transplantation, will get the chance to see if the drug works well for them. This is a win-win situation for patients and the NHS,” he explained.

NICE initially turned down the use of Velcade on the NHS on grounds that it was not a cost-effective use of resources, prompting Janssen-Cilag to come up with a scheme that ensures treatment access for patients who would benefit from it, as well as provide the group with UK sales for the drug.

The drugmaker had originally suggested that the NHS pay for therapy with Velcade in those patients exhibiting a minimal response, as measured by the serum-m protein test, but NICE deemed this too expensive and narrowed the group down to those showing a partial and complete response to treatment, a spokesman for the Institute explained to PharmaTimes.

Some believe that the move could set a precedent for similar deals in the future but, as the spokesperson pointed out, Velcade particularly lends itself to this type of scheme because there is a very clear test of response, which is often not the case with other therapies.

Mixed feeling

Reaction to the risk-sharing scheme has been mixed. Upon there first announcement back in June, a spokesman for the Association of the British Pharmaceutical Industry told PharmaTimes that the agency “supports the selective use of such agreements when there is no other way to get patients access to medicines.”

But, “given that the drugs bill is a stable part of the NHS budget and only one pence in every £1 spent is invested in cancer therapies,” he also stressed that the Health Technology Assessment should be more flexible especially with regard to funding expensive cancer medicines targeting small patient populations.

At the time, Ernst & Young analyst Andrew Jones said the agreement “offers the prospect of a win-win situation for patients, the NHS and industry: patients get access to a new medicine; the NHS ensures efficiency of expenditure and the manufacturer secures an opportunity to get a return on its investment.”

But he also warned that such schemes “do little to address the issue of affordability”, a point echoed by Stephen Welch, Head of Pharma Sector at management consultancy Hay Group, who said: “at £25,000 per patient, Velcade is expensive, regardless of its clinical efficacy”, and added: “In offering these sorts of deals the pharmaceutical companies are effectively putting greater pressure on NICE and prescribers by undermining the health-economic argument against using high-cost drugs for lower-volume conditions with limited efficacy.”

The Department of Health and Janssen-Cilag will be responsible for setting up the scheme, which is to remain in place until NICE’s next review of Velcade, scheduled for consideration in September 2010.