NICE methods and process review aims to create fertile ground for innovation

The way NICE reviews new medicines is about to go through its biggest change in years – but what do the proposals mean for the future of drug development in the UK? Amanda Barrell spoke to Dr Paul Catchpole, director of value and access policy at the ABPI, to find out.

From COVID-19 and Brexit, to the emergence of patient-centric drug development and personalised medicine, the life sciences sector has experienced a complete transformation in recent years.

The NICE methods and process review, then, represents an opportunity to update, renew, and create a modern, world-class appraisals process that fosters innovation and shows the global life sciences sector that UK plc is open for business, says Dr Catchpole.

“It’s so important to continue to make the UK an attractive place to research and launch new medicines,” he says, adding that things had been “somewhat up in the air” for the industry in the run-up to and throughout the Brexit transition period.

“Part of this is about making sure the processes we use to evaluate new medicines are fit for purpose. It is important because it will be looked at globally as a beacon of how receptive the UK is to innovative medicines.”

Evolving landscape

NICE is already regarded an international leader in producing clear, transparent guidance, and these twin reviews will help cement that reputation for the future.

The key aim is to ensure the right treatment gets to the right patients at the right time, by helping pharma and biotech companies to understand how best to engage with the body, says Dr Catchpole.

“A severity modifier would be much more sensitive to a range of different disease states rather than just recognising end of life”

He says: “The kinds of medicines the pharma industry produces has changed. There are now so many more medicines for rare diseases and other smaller patient populations.

“We have gone from making drugs for breast cancer to making drugs that are targeted to just specific types of breast cancer that express certain genetic markers. We are even at a point where some cancer medicines are created for each individual patient using their own immune system. With this increased targeting has come huge increases in effectiveness.”

If the UK is to remain one of the best places in the world to invest in and launch new medical products, then, NICE’s approach must evolve.

Addressing uncertainty

An area that receives significant attention is the creation of authorisation processes that reflect the reality of today’s research and development landscape.

“Everyone wants to get medicines to patients faster and faster, and in the UK we have done a lot to achieve this. But the reality is that the earlier you go, the less mature the evidence base is.

“Combine that with the fact that the more precision medicines are being made for ever increasingly smaller patient populations means there can be greater uncertainty associated with making a decision.”

NICE needs “more sophisticated approaches for how it characterises and manages that uncertainty in the evidence base”, he adds.

“We really hope there will be detailed changes made to the way committees will review and handle uncertainty.”

These changes go hand in hand with wider policies, such as the introduction of an Innovative Medicines Fund, he went on.

Based on a similar model as the revised Cancer Drugs Fund, which was launched in 2016 to provide early access to oncology treatments, the Innovative Medicines Fund will allow NICE to make conditional recommendations while more evidence is collected.

“It is likely to particularly benefit rare disease medicines. By definition they have smaller populations and, therefore, smaller evidence basis. We need to deal with the inevitable uncertainty and make sure people can still access these often life-changing new medicines,” Dr Catchpole says.

Modifying the modifiers

Another promising proposed change in the methods review is a move to replace the end-of-life modifier, introduced in 2009 as a way to provide greater access to life-lengthening drugs, with a severity modifier.

Dr Catchpole says: “If a medicine offers valuable life extensions to people who are otherwise nearing the end of their life, it can currently be evaluated against a higher cost effectiveness threshold of up to £50,000 per quality adjusted life year (QALY).”

It has been very helpful in ensuring some of the latest medicines have been available to NHS patients, he went on.

“But newer medicines are used much earlier on in the patient pathway. For example, they can be really or even more effective before a patient has progressed into the advanced stages of cancer.”

A severity modifier, therefore, would be much more sensitive to a range of different disease states rather than just recognising end of life, Dr Catchpole says.

Modernising processes

The process review, which aims to create more agile, flexible processes that “work better for all stakeholders”, is currently open to a first public consultation until 15 April.

It includes proposals to align processes across NICE’s different technology evaluation programmes, as well as to improve stakeholder engagement through more virtual meetings and requiring companies to provide lay summaries of their submissions.

“It’s really important that patients are able to make meaningful contributions throughout the NICE appraisal process, and that as much support as possible is provided to do that. At the end of the day, medicines are developed for patients, so understanding what they want and what they need remains absolutely crucial,” says Dr Catchpole.

Ultimately, the reviews will help NICE’s methods and processes keep pace with modern drug development, create fertile ground for innovation, and ensure rapid access to lifesaving and life enhancing medications, he concluded.

  • A full public consultation will take place between August and September with the final updated methods and processes being ready later in the year. A public consultation on the processes review is open until April 15.

About the author

Amanda Barrell

Amanda Barrell is a freelance health and medical education journalist, editor and copywriter. She has worked on projects for pharma, charities and agencies, and has written extensively for patients, healthcare professionals and the general public.