Faced with an ever-increasing workload, and squeezed NHS resources, one would guess that living up to the prestigious title of National Institute for Health and Care Excellence (better known as NICE) could on occasion be described as a challenge. What better then, than to propose a scheme which would both save on resources and speed new drugs from already-established classes on the way to their patient recipients in record time, with minimal loss of scrutiny? If this sounds like it might be too good to be true, the early planning stage of the scheme may be a good stage to examine its details.
This is the brief for which NICE has designed a new streamlined pathway, the Abbreviated Technology Appraisal (ATA) scheme, to save cost and time resources for funding decisions on medicines which show similar efficacy and safety to those already recommended by NICE in the same indication. A document detailing the proposal for consultation was submitted to a NICE board meeting on 18 May 2016, and a draft guidance for public consultation was published on 2 June 2016.
Saving time and money by streamlining the economic analysis stage and using direct comparators
The aim of the process is to combine resource savings for NICE with accelerated patient access to drugs from the more established classes, by eliminating analytical steps which may be redundant. NICE themselves have suggested there may be concerns from outside parties about the possibility of insufficient evidence being available to make a fully informed decision, and the perception that the procedure may favour companies producing less innovative therapies.
To be eligible for NICE’s proposed ATA scheme, a new drug should have safety and efficacy similar to or greater than a NICE-recommended drug for the same indication, with a similar or lower cost, and should be comparable with at least one drug already recommended in published NICE guidance for that indication. NICE envisages streamlining of the approval process by eliminating the need for cost-per-quality-adjusted life year (QALY) analyses, and would require the same level of clinical evidence as a standard Single Technology Appraisal (STA). Economic analyses would be performed as cost comparisons against comparator drugs. In addition, patient access schemes may only be considered if submitted at the outset, but not during the course of the ATA process.
The process in a nutshell
The proposed ATA process would basically follow an identical process for evidence submission, review and appraisal as for a conventional STA, but with the following modifications:
- Timelines for company submissions and evidence review group (ERG) assessments would be reduced from 8 weeks to 6 weeks each.
- After the first NICE appraisal committee meeting, a final appraisal determination (FAD) would be produced if the drug was recommended. An appraisal consultation document (ACD) would only be produced if the drug was not recommended, or if use of the drug was to be limited more than stipulated in the marketing authorization or comparator drug guidelines.
- Patient access schemes would only be considered if approved by the UK Department of Health before the company submission deadline, but not if submitted during the course of an ATA.
- An additional engagement step is included, involving review of a redacted executive summary by non-company consultants and commentators to assess clinical and resource similarities of the drug in clinical practice, and comparator companies will have the opportunity to comment on any factual inaccuracies regarding their own product.
- The level of clinical evidence considered would be similar to that for an STA, including a full systematic review to identify all relevant studies and indirect comparisons.
- Appraisal of costs would be via a direct cost-comparison analysis with competitor products. Unlike the STA process, QALYs would not be required to assess benefit. The cost analysis would then be considered alongside evidence of clinical effectiveness to evaluate the best use of National Health Service (NHS) resources.
- If two or more clinically similar treatments are approved by NICE, the recommendation would normally start with the lowest-cost option.
- The new process would retain an option for appeal, with similar principles and processes as for STAs.
- The decision about whether a drug would be appraised by an ATA or STA process would rest with NICE itself, but they stressed that companies would have a say in the final decision.
The NICE board meeting discussion document detailing the ATA proposal is available (on pages 115-141).
What are likely to be the pros and cons?
Even at this preliminary discussion stage, one key advantage of this scheme could not be argued – the ATA process would provide a realistic system for dealing with an ever-increasing workload, while requiring significantly fewer resources than for an STA. NICE reported that resource savings could be as high as 50% over a standard STA, plus the review process could be shortened by 4-6 weeks.
NICE did express the concern themselves that companies may be reluctant to follow the ATA process, and that it could be perceived externally as a less intensive appraisal, favouring second- or third-in-class technologies, or small additions to options for which effective treatments are already available. However, they did not envisage a significant market access advantage for the less innovative drugs, since guidance would be published well within six months of marketing authorization, as with a regular STA, and it was stressed that the ATA decision-making process was no more or less rigorous than for an STA.
The inability to propose a patient access scheme (PAS) part way through the ATA process is an interesting point. In certain cases, this may represent a problem – it has been seen recently that late-stage PAS proposals have often been used to turn an initial rejection into an approval, for example in the final recommendation of Novartis’ Zykadia for lung cancer (UK NICE recommends Novartis’ Zykadia for non-small cell lung cancer). It will be interesting to see how companies adapt to this aspect of the new scheme, if implemented, where the more expensive drugs are involved, particularly in cancer – however, the more innovative therapies will of course still be appraised by the more conventional route, which is amenable to this approach, and PAS details can be submitted, though only at the outset.
Few would argue the fact that that the new scheme would clearly represent a much-needed major cost saving to NICE, with concomitant advantages of resource streamlining, throughput and time savings. However, it remains to be seen whether the hoped-for minimal impact on quality and rigour of analysis would be evident in practice, and whether any controversy would arise over the comparator product assessments. Much will depend on the detail in practice.
Since the proposal is still at a very early stage of planning, we are likely to see much discussion and amendments to the procedure. A draft guidance document for public review was published on 2 June 2016.