BioWorld International Correspondent

LONDON - The UK drugs watchdog is backing an unprecedented scheme in which a pharma company would give a refund if patients do not respond to its drug.

The proposal was made by Janssen-Cilag in a bid to counter concerns about the perceived cost-effectiveness of its drug Velcade (bortezomib) in treating relapsed multiple myeloma patients. Velcade, a proteasome inhibitor, has UK marketing approval, but the health economics agency, the National Institute for Health and Clinical Excellence (NICE), declared last October that prescribing it was not a cost-effective use of National Health Service (NHS) money.

There is a challenge in providing access to expensive but potentially effective treatments for life-threatening conditions, said NICE CEO Andrew Dillon. "If the drug's manufacturer accepts the proposals we are consulting on, it will mean that when the drug works well, the NHS pays, but when it doesn't, the manufacturer should bear the cost." He added, "This would mean all patients suitable for treatment will get the chance to see if the drug works well for them."

However, the rub is in the use of the term "works well." When it made the proposal to NICE, Janssen-Cilag, a subsidiary of Johnson & Johnson, proposed giving a refund for any patient who does not achieve at least a 25 percent reduction in levels of the paraprotein that is generated by the cancerous plasma cells at the heart of the disease, after four cycles of treatment.

However, NICE's recommendation calls for a refund to be given for all patients who do not achieve a 50 percent reduction in paraprotein levels. That means that patients who experience a minimal response or stable disease, as defined by the European Group for Blood and Marrow Transplantation and the International Myeloma Working Group, would not be eligible for further Velcade treatment paid for by the NHS.

At present, there is no way of assessing which patients may respond to Velcade treatment. However, in April, the drug's developer, Millennium Pharmaceuticals Inc, of Cambridge, Mass., announced it has discovered biomarkers that may make it possible to identify responders.

Velcade is priced at about £3,000 (US$5,900) per treatment cycle in the UK.

NICE is pondering the proposal and is accepting comments up to June 22, but said the final decision on whether to put the refund scheme into practice will rest with Belgium-based Janssen-Cilag and the UK Department of Health. It is expected that final guidance will be published by October.

Myeloma UK, a patient advocacy group, which campaigned against the October 2006 decision, said the recommendation was "a step in the right direction."

Velcade is "without question clinically effective," said Eric Low, Myeloma UK CEO. "We now need to carefully consider the details of the draft recommendation as we have concerns that it may exclude some patients who could benefit."

The charity Cancer Research UK supported the idea. CEO Harpal Kumar said, "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 idea of pricing drugs according to clinical benefit was floated in February by the UK's Office of Fair Trading (OFT), in a report that suggested a move from the current UK system based on profit controls, to one based around therapeutic value. That would not only result in better treatments, it also would benefit the industry, argued John Fingleton, the OFT's CEO. "It would focus innovation and investment on the areas where patients need it most, creating more valuable drugs in the future."