Will the NICE Review of Methods and Processes pave the way to HTA reform? As we wait for the consultation, Nicola Boyd argues that we should be realistic about what the Review will deliver

Pressure is building in the regulatory arena as advancing biomedical innovation surpasses the methods and processes used in evaluation. That pressure has now come to a head with the latest NICE methods review, where the industry is hoping for answers that will deliver a system built for innovation and personalisation. But what can this review reasonably achieve?

Since its first appraisal was published in 2000, 82% of NICE recommendations have been positive. This could prompt the question: Is the system as broken as people claim? Rather than the percentage of approvals, it seems the speed of appraisal and the type of recommendations made are what are falling short of optimal. In attempts to mitigate this, NICE routinely examines its methods and processes, cycling reviews around every three years. This has seen some welcome changes, most notably the introduction of end-of-life criteria and the formation of the Cancer Drugs Fund (CDF) that relaunched under NICE in 2016. But in general, there is an inherent scepticism within the sector about the appetite within NICE for wholesale reform.

Bargaining chip or breakthrough?

What sets this review apart from its predecessors is its genesis from a wider deal struck between industry and the government. Following contentious negotiations, the Voluntary Pricing and Access Scheme (VPAS) came into effect in January 2019, replacing the Pharmaceutical Price Regulation Scheme (PPRS). Whilst

the shift in the budget and repayment rates may have fallen unfavourably for industry under the new scheme, VPAS contains promising initiatives to improve the speed of access to new medicines. Most notably, it committed to a review of the NICE methods for both technology appraisals and highly specialised technologies (HST). This was presented by industry as a major concession and positive outcome when VPAS was unveiled.

The contention hasn’t stopped there. This review’s announcement in July 2019 attracted substantial interest from external stakeholders surrounding the implications for NICE’s work, impact on patients, affordability in the NHS and the success of the life sciences sector.

Interest groups have been mobilising to set out their key demands from the review. Calls have been made for the processes to have clearer routing criteria for HST and improved representation of the patient view. An inquiry by the All-Party Parliamentary Group (APPG) on Access to Medicines highlighted demand for wider value thresholds and modifiers, better management of uncertainty and improvements in the use of data and real-world evidence be brought into the methods. Fortunately, these broadly correspond to the scope set out by NICE. Yet, disappointingly for industry, NICE will not be reviewing one of the prime decision tools – the QALY, this time around. What’s more, the Institute has indicated that changing its methods will only occur if there is a compelling case, which is likely to see much of the expectation fall short of reality.

So what can industry expect? VPAS encourages industry to feed its views into the Review, and considers industry as an ‘active participant’. MHP understands that industry representatives are calling for more holistic value assessments that reach beyond cost- effectiveness, include health and non-health related benefits and for consideration of the societal and patient value. This relies on a system built with flexibility and capable of incorporating real- world evidence into decisions. Additionally, as we move towards specialised and personalised medicine, patient populations are becoming smaller and more targeted resulting in higher levels of uncertainty.

Preparing for personalisation

A real measure of success in this review will be how NICE manages uncertainty whilst allowing for the value and benefit-risk assessments to be made irrespectively of it. It is important to state that increased uncertainty does not equal increased risk. Failing to effectively address this nuance will see new treatments trapped by gatekeepers that are stalling access whilst manufacturers provide more evidence.

Critically, the evidence that is provided may not substantiate a recommendation for a treatment’s full indication. Of the 82% of positive recommendations, 29% were optimised, meaning they were not recommended for their full licence, and 5% were only recommended for the CDF. It is also important to consider what is currently happening to the 18% of technologies that are not receiving approval. NICE states that a treatment will not be recommended when there is a lack of evidence for the clinical effectiveness of the technology, or it’s not considered to be cost-effective. Allowing for more flexibility in the appraisal process and incorporating better value measures could greatly improve the percentage of technologies being accepted, and for their full indication.

Playing catch-up

Going back to the problems that got us here, VPAS also promises more and faster NICE appraisals for all new active substances and speeding up of appraisals for non-cancer medicines to be in line with cancer medicine timelines.

Despite NICE reducing the lag time between EMA authorisation and NICE commencing its appraisal, what happens after this hasn’t improved. The Institute of Cancer Research found the average time taken for an evaluation between 2009 and 2016 was 16.0 months, which made little impact in comparison to the 16.7 months it took between 2000 and 2008. It’s difficult to gauge at this stage the impact that reform will make on the speed of appraisals. Making steps towards managing uncertainty and gathering real-world evidence have potential for faster access, as it is likely to allow for early and managed access around surrogate endpoints, as real-word evidence feeds into decisions. However, NICE hasn’t yet made commitments to how they will improve speed, which could see new medicines continuing to get trapped at the gates.

Increasing the speed of access is most likely to be realised by the delivery of the NHS Commercial Framework for Medicines, another commitment made by VPAS. This Framework should enable more open and regular dialogue between industry, NICE and the NHS, streamlining discussions about value, affordability and transactability and driving earlier and more purposeful engagement. This ultimately has the potential to prevent unnecessary delays whilst negotiations take place.

Alongside delivery of the Commercial Framework, significant responsibilities will be placed on external stakeholders if the Review delivers on the expectations. The call for more impactful modifiers to be used in assessment suddenly flips expectations onto the patient advocacy community to produce the evidence. Having called and campaigned for a greater role for decades, would all be ready to mobilise their HTA skills and provide quantitative evidence that can be reasonably fed into the decision-making process? From MHP’s experience working with charities to upskill them on HTA, we understand that there is disparity of resource within the patient group community to address these issues. The increased responsibility placed on patient groups could risk furthering the inequality and lead to a misrepresentation of some therapy areas. There may be some way to go before this increased demand and responsibility can feasibly and fairly be placed on this community.

NICE has made exceptional progress in allowing access to safe and efficacious medicines over the past 20 years, but it has now reached a fork in the road. Continuing on its well-trodden path risks compromising the UK’s position as a leader in life sciences as it will fail to keep pace with innovation – a path that could fail to grasp the opportunities of life outside of the EU and EMA remit.

In the advent of personalised medicine, there is an opportunity now for the system to build in the flexibility to prepare for a future of rapid advancement. The outcomes of the Review and subsequent reform will be a decider on the potential for NICE to deliver another exceptional 20 years of health technology assessment. The extent of reform to the HTA process that the Review will bring is uncertain but likely to be limited by its scope. Regardless of its outcome, the systemic issues that have led to this Review must continue to be addressed beyond its time frame if NICE is to pave the way to personalisation.

Nicola Boyd is an account executive in the Health team at ENGINE MHP, which works with many of the UK’s leading pharmaceutical companies, healthcare providers and charities.