A case for change
NICE have today announced their Methods Review – A Case For Change consultation which is open for six weeks.
First announced in July 2019, the methods review has of course been delayed in 2020, and in this time NICE’s approach has changed a bit too. This is in fact the first of (at least) two consultations on the methods review. It is called ‘the case for change’, but it does a bit more than confirm the premise, it also sets out proposals for change in some detail (the document is 62 pages long). The full timeline is available here, the second stage of the consultation process will be a presentation of a new methods process containing the changes agreed following this consultation.
The document lists many reasons for the review, including aligning with the changing regulatory system, equity and learning from the Cancer Drugs Fund and other managed access agreements, adapting to technology and innovation, and sustaining NICE’s reputation as a global leader in HTA. These motives are welcome additions to the focus of the original announcement which was on delivering commitments made in the latest pricing agreement between the Pharmaceutical industry and the UK Government – the Voluntary Pricing and Access Agreement for Branded Medicines (VPAS, 2018). This agreement specifies that cost effectiveness thresholds will not change, although modifiers – such as the means by which medicines for end of life care can have a higher cost effectiveness threshold – could change.
Scope of the review
The scope of the review is on the methods for four key decision making processes that NICE manages:
The highly specialised technology (HST) evaluation process
This is the process specifically designed for rare conditions launched in launched in 2013. Since then the programme has perhaps been underused producing guidance on just 13 medicines for rare conditions.
The single technology appraisal (STA) process
This is NICE’s general medicine decision-making pathway which covers all other medicines, including all other rare disease medicines that NICE selects.
The diagnostic assessment programme
This is the process NICE uses to evaluate innovative diagnostic technologies (though this is not directly connected to genomic diagnostic programmes in the NHSs).
The medical technologies evaluation programme
This is the process NICE uses to evaluate medical devices and other technology.
Genetic Alliance UK, alongside other members of the Charity Medicines Access Coalition and further patient organisations have been involved in the review from the beginning. Jayne Spink PhD, Chief Executive of Genetic Alliance UK, is a member of the methods working group, and colleagues have been involved in task and finish groups for the programme.
Proposals for change
The proposals for change are categorised as follows:
Valuing the benefits of health technologies
– This section includes proposals to change some of the modifiers used that give certain medicines higher cost-effectiveness thresholds. There is a proposal to stop using the end of life modifier (which favour treatments for certain conditions) and to start using a modifier that accounts for severity of a condition. Consideration is also given to a modifier to account for inequality in health.
– Uncertainty is also discussed with NICE indicating that there may be more acceptance of uncertainty, which would be positive for rare conditions if handled well.
– How long term benefits are assessed and priced – ‘discounting’ is also up for discussion.
Understanding and improving the evidence base
– This section proposes changes to how NICE processes and presents evidence. This includes how to present and interrogate uncertainty in evidence.
– There is a proposal for a hierarchy of preferred quality of life measures, which could be positive for our community as the standard models are notoriously poor at capturing the quality of life impact of rare conditions.
Structure decision making
– This section discusses proposals to improve how cost-effectiveness measures are presented to NICE decision-makers, and potential flexibility in how they apply their methods to subgroups.
Challenging technologies, conditions and evaluations
– This section examines innovative technologies and the challenges they place on NICE’s methods, this includes cell and gene therapy.
– There is also a discussion of the challenges in decision-making in rare conditions – where most innovative technologies are targeted.
Aligning methods across programmes
– This will be more of a focus for the second stage of the consultation process.
What is not in the consultation?
This is only about NICE Methods – how they do the bits of work that they do. NICE Processes are different and not included – this includes Topic Selection which is how NICE decides whether to assess a medicine and with which tool. There is another consultation on this open now, which we talk about here. Other elements of how NICE do their work, such as when they apply their decision-making processes in the timeline of a medicine’s life, are also out of the scope of this consultation.
It is also (obviously) only about NICE. As we said in Action for Access, there are bigger issues with the whole system. By way of example, however well NICE’s systems work would not solve the current access problem for families living with phenylketonuria who cannot access sapropterin. NHS England are the decision-maker for access to this treatment now, and the fact that a medicine licensed since 2008, and available across Europe, is not available here is a symptom of bigger problems with the systems in the UK that cannot be fixed by the methods review alone.
This is a great opportunity
This is certainly a landmark opportunity to improve medicines decision-making, and we need to grab it. Genetic Alliance UK will be analysing the proposals in detail over the next week.
We are arranging a series of meetings for patient groups to gather and learn about the consultation, and we will be sharing more detail on these very soon. In the meantime, please send your questions, comments, plans and ideas to [email protected].