Industry and patient groups have broadly welcomed the proposals to speed up and broaden the availability of innovative medicines which are outlined by Professor Lord Ara Darzi in his report on the National Health Service (NHS), published this week.

Lord Darzi says that the National Institute for Health and Clinical Excellence (NICE)’s topic selection and appraisal process will be improved to enable the Institute to produce “consistently fast guidance on significant new drugs” and issue the majority of its appraisal guidance within a few months of a new drug’s launch. Despite NICE’s “worldwide reputation,” it has “sometimes taken too long” for its guidance to be made available on newly-licensed drugs, says the report.

Moreover, it adds that while all Primary Care Trusts (PCTs) have a legal duty to fund drugs recommended by NICE, “unexplained variation” remains in the way local decisions are made on the funding of new drugs before appraisal takes place or where no guidance is issued. Steps to end this “postcode lottery” will include making explicit, through the NHS Constitution, the right of patients everywhere to receive NICE-approved treatments, where their doctor judges these to be of benefit, and to expect rational local decisions on funding of new drugs and treatments. “Open and honest explanation will be due if the local NHS decides not to fund a drug or treatment that patient and clinician feel would be appropriate,” says the report.

“Patients want the most effective treatments, and staff want to be able to provide them. As the NHS becomes more personal, patients and the public want to be assured that the most clinically and cost effective treatments are available everywhere. During this Review, patients and the public were very clear that they had zero tolerance for variations in access to the most effective treatments,” said Lord Darzi.

Patient groups welcomed his recommendations, with caution. While these developments can only be positive, changes must also ensure that that the wider benefits of treatments to society, particularly carers, are considered, said Neil Hunt, chief executive of the Alzheimer’s Society, while Ian Beaumont of Bowel Cancer UK suggested that by appraising treatments earlier, NICE is likely to claim that the evidence for them is not sufficiently robust.

“If the government is genuinely committed to increasing access to new treatments, then NICE needs to move away from its outdated and simplistic Quality Adjusted Life Years (QALY) system and tailor its appraisal process to match the criteria used in drug trials, said Mr Beaumont.

NICE chief executive Andrew Dillon was reported by the Financial Times as estimating that the enhanced responsibilities for the Institute will necessitate a tripling of its budget, to £90 million over the next four years, while ahead of the Darzi report NICE chairman Professor Sir Michael Rawlins told the BBC that it had urged the government to invest much more heavily in its guidelines programme, “so that we can not just keep the existing ones up to date but do a whole lot of further guidelines.”

Closer working with industry

In his report, Lord Darzi also calls for strengthening the horizon-scanning process for new medicines in development. The industry will be involved “systematically” to support better forward planning and develop ways of measuring the uptake of clinically and cost- effective medicines, once they introduced, and a single evaluation pathway will be created for new clinical technologies to simplify their passage from development into wider use, while ways to benchmark and monitor their successful uptake will be developed, he says.

The Association of the British Pharmaceutical Industry (ABPI) called his recommendations “a huge leap forward in driving the use of best available treatment” and said “the task now is to deliver on the promise.”

The measures are “good news for patients, who can now look forward to receiving the same standard of care wherever they live,” said ABPI director-general Richard Barker. “Britain’s innovative pharmaceutical industry will also gain, by serving a more innovative customer, one that will also be a more attractive location for future clinical trials – a further benefit for UK patients,” he added.

ABPI president Chris Brinsmead said the measures “offer the opportunity to effect a seismic change in patients’ right to access the best and most modern medicines, based on clinical need.”