The way evidence is used to approve new treatments for use on the NHS is set to change, under a review being carried out by the National Institute for Health and Care Excellence (NICE).
The review of methods, running throughout 2020, is an opportunity to address some of the challenges in approving treatments: for example, ensuring NICE decisions more effectively take into account changes in the burden of illness or quality of life in myeloma.
The review will enable stakeholders with an interest in drug approval to consider how NICE methods and processes will need to change as products become more complicated to evaluate as a result of innovations such as personalised medicine and cell therapy. It will also look at how NICE considers treatments with less established evidence, and how the system can deliver faster decision making.
The review has two elements: one will look at NICE processes (the steps taken throughout the assessment and when and how stakeholders are involved), and the other will examine the methods used in decision making (including key principles underpinning appraisals and how different types of evidence are used).
- Issues being looked at under the methods review include:
The types and sources of evidence used (how real-world evidence and quality of life data is used in NICE appraisals)
- Defining modifiers (how factors such as unmet need, burden and severity of illness and innovation can impact decision making)
- Exploring uncertainty (how uncertainty due to less established evidence can be quantified and reduced, including the role of data analytics and extrapolation methods)
Head of Patient Advocacy at Myeloma UK, Shelagh McKinlay has been part of a working group recommending ways to improve patient involvement in NICE appraisals.
This included proposing changes on issues ranging from changes to NICE committee culture and the way patient experts are involved in meetings, to delivering feedback to make it clear what impact stakeholder evidence has had.
Shelagh is also a member of the Methods Review Task and Finish Group looking at modifiers.
“This is an opportunity for Myeloma UK to advocate for changes at NICE which address some of the challenges in approving myeloma treatments. As a relapsing and remitting cancer with multiple lines of expensive treatments often prescribed over long periods, myeloma treatments can face an uphill struggle in proving cost-effectiveness. The review is an opportunity to make sure that the inherent complexity of myeloma is properly recognised by the system.
“However, it is important changes at NICE are not seen in isolation. Many elements go into making a case for treatments to be approved, not least the price charged by companies. We need to see changes in NICE methods mirrored by flexible and responsible commercial approaches on the part of industry.”
At the moment proposals are being developed to deliver a consultation in the summer of next year. Following this consultation, a single manual for NICE methods and processes covering all of its appraisal activity will be published toward the end of 2020.
The commitment to review NICE ways of working was made in the 2019 Voluntary Scheme for Branded Medicines Pricing and Access. The review will not look at the challenges of approving combination treatments, a key issue in myeloma as this is being taken forward by the Association of the British Pharmaceutical Industry (ABPI) in partnership with NICE. Myeloma UK is closely monitoring this work.