The National Institute for Health and Clinical Excellence's Single Technology Appraisal process - the new streamlined procedure for recommending products which should be made available through the National Health Service - takes around six months to complete compared to some 12-14 months for the standard Multiple Technology Appraisal. It also relies purely on evidence supplied by the manufacturer or sponsor and aims to provide guidance within eight weeks of the product’s approval. However, it is not a “soft option” for manufacturers, Professor David Barnett, chairman of the NICE Appraisals Committee, has warned.
While the STA represents a “sea change” in NICE’s approach to appraisals, “a quicker process and timely decision-making does not mean a less restrictive outcome,” Prof Barnett told NICE’s annual conference, held last week in Birmingham. The process - set up to examine applications for a single product in a single indication close to its launch, and also to speed up the guidance development process on life-saving drugs that have already been licensed - maintains the key principles of NICE technology assessment, ie, transparency, inclusiveness, robust assessment, independent decision-making and regular review, he stressed.
NICE technology appraisal is recognised as a global standard, but the STA procedure was introduced in October last year in response to growing demand from the NHS for an increase in the rate of guidances issued by the Institute, and for them to appear closer to the product’s point of entry onto the UK market.
For an STA, the clinical and cost-effectiveness evidence supplied by the product’s manufacturer or sponsor continues to be reviewed independently by the Institute’s Evidence Review Groups, as is the case for MTAs. But an ERG does not undertake an independent analysis of the evidence provided by the manufacturer/sponsor for an STA; it will critique the completeness, validity and acceptability of the evidence but will not provide an alternative view.
Also, the process provides little scope for the Appraisals Committee to explore issues which have not been presented by the manufacturer. However, the procedure remains open to appeal and challenge; the first STA to go to appeal, in a case brought by a Primary Care Trust, was against NICE’s recommendation of Roche’s Herceptin (trastuzumab) for the treatment of women with early-stage HER2-positive breast cancer (except where there are concerns about cardiac function). Prof Barnett commented that he was not expecting many such “yes” decisions to be appealed in future.
60 STAs in production
10 STAs have already been completed, all of which so far have been for drug products, and a further 60 are currently in production. With this new speeded-up process, NICE is also now trying to make the selection of products undergoing STA assessment more “fleet of foot,” but awaiting ministerial decisions can be very frustrating, said Dr Carole Longston, director of NICE’s Centre for Health Technology. Anyone can suggest a topic for NICE review, she reminded delegates, but also warned against the practice by some manufacturers of applying for review of products which had received their licences last year. “We’re trying to stop that,” she said.
Moreover, as some STAs track the new product’s development pipeline, there may well be some which start the process but do not make it to final approval, the conference heard.
A major issue with NICE recommending the use of such new products is the increased level of uncertainty over the evidence base which is available for them, for example in the area of long-term effectiveness and the risk/benefit ratio. When a new technology arrives its benefits, but not its “harms,” are evident, and this picture can change over time, delegates pointed out. Prof Barnett acknowledged that when the evidence is immature there will be degrees of uncertainty, but he again warned that this will not lead to a more ”lenient” appraisal.
Comparisons with Scotland “wrong”
Nor, he said, should the STA process be compared to that of the Scottish Medicines Consortium, whose fast assessment decisions have been widely praised in the media. Prof Barnett pointed out that SMC examines all new drugs, while NICE does not, and that the Consortium grants permission to list a drug but not to have it funded. The SMC and NICE processes are very different, he added.
The evidence of the last year has shown the STA process to be “fit for purpose,” he said; it is robust, timely and consistent. He also pointed out that the process gives NICE the option to issue a “minded not to recommend” decision on a product, and forecast that it may well issue more of these “minded nos” than people might like. By Lynne Taylor