Following the news that the National Institute for Health and Clinical Excellence has accepted a proposal for a new type of scheme to provide patients with access to Millennium Pharmaceutical and Janssen Cilag’s previously rejected cancer drug Velcade, industry observers have been analysing its implications.

Earlier this year, Velcade (bortezomib) was rejected by NICE for use in patients with myeloma on grounds of cost, but a subsequent appeal by cancer charities Myeloma UK, Cancerbackup Leukaemia CARE agreed that the appraisal committee should take another look at its use on the National Health Service.

Now, under the proposed risk-sharing scheme, the NHS will fund treatment with Velcade as long as patients show a “full or partial response” to the drug but, if there is no response, the cost will be refunded by the product’s manufacturer.

A step forward

Commenting on the proposal, a spokeswoman for Myeloma UK told PharmaTimes that the group is delighted patients are closer to getting access to Velcade. “We are still looking at the finer details of the agreement, including the fact that patients could be taken off the drug after just four treatment cycles…but the overriding sense is that it is a positive step forward, no matter what the caveats are.”

And 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 says that the Health Technology Assessment should be more flexible especially with regard to funding expensive cancer medicines targeting small patient populations.

NICE has come under increasing scrutiny in recent times - culminating in a current investigation by the Office of Fair Trading into the way the Institute operates - and its decision on which medicines to reject or include on the NHS treatment menu are often fiercely criticised. On the one hand, there are those that question the use of increasingly expensive medicines on a cash-strapped NHS, but others argue that patients have a right to access the latest advances available under the health system.

This latest move by NICE will likely spark some heated debate and, if successful, may even signal a seismic shift in the way medicines are funded in the UK.

According to Ernst & Young analyst Andrew Jones: “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.”

He added that “the news should come as a welcome development to the biopharmaceutical industry which is being hit hard by pricing and reimbursement pressure across Europe. NICE’s endorsement of the scheme signals willingness for flexibility, and if agreed and successfully implemented, the scheme could help guide future change to the UK system and support market access for other new therapeutics”.

Addressing affordability

But he also warned that risk-sharing schemes “do little to address the issue of affordability – the NHS will still have to find the budget to meet the cost of new and effective drugs…the issue of affordability raises the question of how the healthcare systems such as the NHS will be able to support the introduction of new and important drugs in the future without compromising in other areas.”

And management consultancy Hay Group has also voiced its concern. Stephen Welch, Head of Pharma Sector at the company, says that, at £25,000 per patient, Velcade is expensive, regardless of its clinical efficacy. “Could the money-back guarantee distort doctors’ views on which drugs to prescribe for their patients? And what of patient pressure?” Furthermore, he says, “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.”

He also points out that the time invested by PCT managers in calculating what refund, if any, they might be due, will only increase if more companies follow suit in offering such deals. “All in all, the principle of partnership will likely make for better, more cost-effective drugs, while increasing accessibility of those on the borderline. However, until the organisational and process implications have been thought through in detail, the most likely clinical outcome is confusion,” Mr Welch concludes. By Selina McKee