NICE 在英国的下一步工作–2023 年威斯敏斯特卫生论坛
In February this year, the Westminster Health Policy Forum held a conference discussing next steps for the National Institute for Health and Care Excellence (NICE) in England, in which Isabelle Newell, a Consultant in our Rare Diseases Division, was part of a panel of experts discussing the use of real-world evidence (RWE) in supporting reimbursement, bringing her perspective on the successes, challenges and learnings to be drawn from the use of RWE in the field of rare diseases.
The policy conference was an opportunity for NICE to reflect on recent changes to its processes, outline their priorities over the year ahead and beyond, and for stakeholders in NICE processes to ask questions on these topics. Discussions through this conference highlighted the potential for increased use of RWE to further deliver on the aspirations of more timely and targeted delivery of guidelines which are adaptable in light of new evidence, and expediting the development of guidance for innovative therapies. However, with these developments comes a need for caution by NICE and industry, in ensuring that the move towards faster and more flexible access to market for new technologies, and greater acceptance of RWE to do so, does not come at the cost of high-quality, evidence-based decision-making.
Maximising the potential of RWE to deliver improved patient outcomes
The increasing use of RWE to inform NICE guidance was a central theme throughout the discussion, with Dr Sam Roberts, NICE’s chief executive, noting in her opening remarks that this is a key area of focus for NICE, who want high-quality RWE to play a larger role in their decision-making and guidance.
NICE has an important role to play in harnessing the huge potential promised by RWE to improve patient outcomes, in collaboration with the NHS. However, concerns remain over the quality of data available, the linking of data across regional data hubs, and patient privacy. Whilst NICE’s RWE framework has shown NICE to be alert to its role in enabling the use of RWE for the purposes of reimbursement, industry representatives still noted a lack of clear guidance on the most appropriate use of real-world data (RWD), which we have discussed further here. This is reflected in our own experience with NICE, where RWE is still considered with caution and has yet to gain wide acceptance. A number of speakers highlighted challenges that still remain in accessing and utilising RWE to support reimbursement activities. Promisingly, Dr Claire Bloomfield, deputy director for NHS England’s Centre for Improving Data Collaboration, outlined practical steps being undertaken by the NHS to improve data quality and accessibility. This focused on the creation of a national “data museum”, aiming to integrate regional datasets into larger national ones, and allowing stakeholders access to data via Secure Data Environments (SDEs) behind firewalls. SDEs were endorsed by industry representatives as an important development for researchers using RWE, and should allow for fast and safe access to large datasets, currently a major hurdle to RWE research – as we’ve found, accessing these datasets can take up to 18 months. SDEs remain in their infancy however, and the extent to which these can be scaled to allow access to nationwide datasets and deliver on their promise remains to be seen.
With the increasing use of RWD to inform reimbursement decisions, the issue of patients’ own involvement in the data-gathering process was cited as an important consideration by a number of speakers. The benefits of patients’ contributions should be made clear to them, and reassurances as to exactly how their data will be used should be of central importance to RWE research. In turn, this could lead to greater patient participation in the development of high-quality RWE.
Through her contributions, Isabelle provided insights on the opportunities RWE can provide for the reimbursement of treatments for rare diseases. RWD offers an end-to-end solution to gain understanding of the natural history of a disease and to optimise an evidence base to address areas of uncertainty. The burden of data collection is often greater on patients with rare diseases, due to their small numbers. Isabelle therefore emphasised the potential for greater centralisation of rare disease registries to accelerate access to treatments for patients with rare diseases, many of whom have no treatments available to them. To these points, Isabelle noted the recent increase in the number of collaborative, centralised registries being set up across the rare diseases space such as the European Reference Networks (ERNs), and in line with the recent call from the Medical Research Council (MRC) and National Institute for Health and Care Research (NIHR) for an integrated UK rare disease research platform.
Strategic priorities for NICE, including the proportionate approach
Pragmatic approaches to delivering patient access to treatments are high on NICE’s agenda. Dr Roberts explained that NICE’s recently published proportionate approach to HTA aims to deliver guidance to healthcare professionals and patients earlier, and aims to increase NICE capacity through more streamlined technology appraisal processes for low-risk treatments. Industry has welcomed the efficiencies promised by the new proportionate approach, but greater guidance and transparency from NICE would be welcome, as the threshold for a submission to be eligible for the proportionate approach remains unclear, and is currently discussed on a case-by-case basis.
In the MedTech sector, the early value assessment (EVA) scheme, launched in 2022 and piloted up to March 2023, is intended to allow fast-tracked assessment of medical technologies, with the aim of bringing promising technologies to patients sooner. Under this programme, greater uncertainty in clinical- and cost-effectiveness is accepted by NICE, on the condition that further evidence is collected and the assessment reviewed after three years. The EVA pilot is targeted towards technologies that can address areas of great unmet need within the NHS, and therefore has the potential to bring benefits to a large number of patients. The risks are tempered by a clear focus on technologies with a low-risk profile.
However, the tangible impact of this pilot remains to be seen. Recommendations need to translate to adoption, and a clear barrier to this remains the lack of a funding mandate for MedTech following recommendation. Without suitable adoption support, the true benefits of accelerating technologies through a NICE appraisal cannot be realised. Nonetheless, the introduction of this pilot demonstrates an understanding within NICE that MedTech HTA pathways need a degree of flexibility to ensure uptake of innovative technologies as fast as possible to bring the greatest benefit to patients.
NICE’s shift towards more streamlined approaches presents opportunities for faster patient access to technologies, as well as lower costs for both companies and NICE. This has so far taken the form of paired appraisals for similar medicines, combining appraisals within a single disease pathway, and updates to shorten post-submission processes, particularly for cost-comparison appraisals. Our experience supporting submissions that have undergone the proportionate approach as part of NICE’s pilot is that the time between submission and final decision is considerably reduced compared with standard appraisals. NICE presented the learnings from the pilot at a recent webinar which corroborates this trend. However, any move towards faster decision-making comes with fewer opportunities for stakeholder engagement and the potential for reduced transparency in decision-making. Careful consideration should be taken by NICE when deciding to streamline appraisals, with greater uncertainty in decision-making weighed against the level of risk associated with recommending certain technologies.
Jacques and Isabelle are employees at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners. If you would like any further information on the themes presented above, please do not hesitate to contact Jacques or Isabelle.