Following NICE's announcement in July 2019 that it was undertaking a review of its methods and processes for issuing guidance, the All-Party Parliamentary Group on Access to Medicines and Medical Devices (the APPG) has published a report setting out a number of recommendations to assist NICE's upcoming methods review (the Report). The Report was prompted in particular, by the need to ensure that NICE's methods review takes into account the ever-increasing number of innovative and technological developments in the healthcare sector and the need to ensure swift patient access to these treatments and therapies.

In producing the Report, published in September 2019, the APPG gathered 53 written submissions from industry stakeholders, patient organisations and clinicians in addition to oral evidence from a cross section of interested parties. The evidence focussed on areas for methodological change and NICE's processes, particularly in relation to the need for flexibility and the analysis of cost-effectiveness. The Report set out seven recommendations for NICE to consider in its methods review relating to process, methodology, and governance. It also touched on areas for future research and work outside NICE's review, on the understanding that NICE does not operate in a vacuum. 

Methodological Change

Reflecting the concerns of industry stakeholders, the Report calls for greater flexibility in the methodology applied by NICE in the assessment of technologies in order to manage uncertainty and the changing landscape of medicines, which has brought smaller patient populations, new treatments and earlier regulatory approval.

To address this issue, the Report recommends the introduction of an "interim conditional approval mechanism" which operates in a similar way to the Cancer Drugs Fund (CDF), given the strength of stakeholder support for the CDF. The interim conditional approval mechanism would come into play where there are areas of uncertainty as to cost-effectiveness but the treatment is nevertheless "plausibly" cost-effective. Following interim approval, the pharmaceutical company would be required to continue collecting data for a set period of time prior to a re-appraisal of the treatment. The Report also recommends modifying the incremental cost-effectiveness ratio ("ICER") thresholds used in the appraisal of new treatments in situations where there is a clear unmet need in relation to a severe medical condition. The ICER is a measurement used in the analysis of new treatments to indicate the cost-effectiveness of a health care intervention. The Report recommends that NICE expand the ICER threshold for treatments that address rare and severe diseases where there exists a clear unmet need, in order to improve treatment access for those suffering from these rare and severe conditions.

The Report also recommends that NICE adopt the Treasury's recommended discount rate of 1.5% for risk to health and life values, as set out in the Green Book. NICE currently uses a default discount rate of 3.5%.

NICE's Processes

The Report recommends review of the criteria for medicines to be referred for highly specialised technology (HST) evaluations by NICE, and calls for greater engagement with patients and patient input in the decision-making process. The Report states that certain criteria for the referral process for HST evaluations are unclear or too stringent. As a result, specialised treatments, which cater for a small number of patients, are wrongly referred to single technology appraisal ("STA"), which was not set up to assess ultra-orphan conditions. One solution the Report suggests is for NICE to consider removing the criterion that a treatment be delivered through a highly specialised service, for example enzyme replacement therapy or proton beam therapy, in order to qualify for HST. The Report also suggests that the number of patients required in order for a treatment to be eligible for HST be clearly stated, and that there be a right of appeal if a treatment is rejected on the grounds of budget impact.  On patient engagement, the Report recommends that NICE explore ways to enhance engagement so that patients can play a greater part in the decision-making process relating to the guidance NICE issues. One specific recommendation is that, prior to appraisal Committee meetings for STAs relating to orphan treatments where there is a clear unmet need, a preliminary meeting should be held to provide patient groups and clinicians with an opportunity to contribute to a summary statement about the relevant condition and any possible treatments. This is a similar process to that used by the Scottish Medicines Consortium and is intended to address a concern that appraisal Committee meetings can be intimidating for some patient groups.

Governance of the NICE Review

Finally, the Report calls for NICE to publish clear timelines for the methods review as well as a calendar of the touchpoints for stakeholders. Following NICE's review, the report calls for a full public consultation on the suggested changes. To date NICE has confirmed that specific topics are being researched until January 2020, involving stakeholder input where required. The findings will then be reviewed from January to June 2020 followed by a 6 week public consultation on the proposed changes in Summer 2020. The aim is to publish the resulting programme manual in December 2020 and to implement the changes from 2021 onwards.