Is NICE becoming nicer?

How NICE’s 5-year strategy signals a shift from adversarial to collaborative engagement and how stakeholders can benefit

For two decades, interactions between the UK’s National Institute for Health and Care Excellence (NICE) and the pharmaceutical industry (Pharma) were unusual to say the least. Picture the scene at NICE headquarters: a technology appraisal committee is meeting to discuss a single technology appraisal (STA) for Drug X manufactured by Company Y. Around a large U-shaped table sit 22 committee members, 3 evidence review group (ERG) representatives, and various others. At a separate desk sit 2 nervous-looking company representatives who may only speak when spoken to by the chair of the committee; their trepidation a product of a critical ERG report on the company submission and an inquisition in which they play no effective role. Until quite recently, when we all started working in pyjamas, this sketch succinctly described the nature of the relationship between NICE and Pharma.

This is changing for the better and with it comes the opportunity to evolve and innovate in the field of evidence synthesis and economic modelling. In 2019, NICE re-imagined the STA process to include a phase of technical engagement between companies, the ERG, and the NICE staff responsible for the appraisal. The new-era appraisals allow evolution of the Company submission, thereby preventing the Committee meetings from being hijacked by moot details, usually economic.

NICE expects to publish a new health technology assessment (HTA) manual in December which will evolve and broaden decision-making criteria, such as the introduction of disease severity modifiers that adjust quality-adjusted life-year thresholds for a range of diseases to replace end-of-life criteria that previously focused on cancer technologies. The changes will be reviewed in a future edition of HTA Quarterly.

The publication earlier this year of NICE’s promisingly titled 5-year strategy, “Dynamic, Collaborative, Excellent” sets the scene for not just evolution but maybe revolution in the HTA landscape, proposing the most significant changes to its remit and activities since its inception in 2000. If successful, these changes will reinvigorate NICE’s position as the pre-eminent global HTA authority and stimulate substantial innovation in decision science. Significantly, it continues the shift from adversarial to collaborative engagement with Pharma, from which all stakeholders can benefit.

If there is a silver lining to COVID-19’s long, dark shadow, it is government’s heightened recognition of the following imperatives in healthcare:

• Acceptance of innovation by speeding up access to new and effective treatments
• Integration of real-world data into evaluation processes
• Provision of information in dynamic formats that support shared decision making
• Seamless collaboration with others to particularly reduce health inequalities

For NICE, these laudable aims will be achieved through the execution of the following four key pillars of activity.

Pillar 1. Rapid, robust, and responsive technology evaluation
In response to rapid development of new health technologies, NICE is committing to speed up the existing evaluation pathway of new medicines by proactively identifying innovative and cost-effective health technologies, the latter of which represents a new activity. Currently, detail is lacking but this is a clear signal to manufacturers that early economic evidence will be a key criterion in prioritising future appraisals, especially those of emerging health technologies. NICE’s standing membership of the Innovative Licensing and Access Pathway gives innovative manufacturers an opportunity for very early engagement with the decision science community, essentially reducing time from discovery to reimbursement. This will be reviewed in the Spring 2022 issue of HTA Quarterly.

Of additional interest is NICE’s commitment to managing uncertainty in the evidence base of promising technology by specifying data collection requirements alongside ongoing evaluation and inclusion in managed access arrangements. There is a similar ambition for the Cancer Drugs Fund (CDF) although operational details are scarce; one reason for this is the difficulty of accessing the Systemic Anti-Cancer Therapy dataset for commercial research. The reformed CDF is now complemented by the National Health Service (NHS) England Innovative Medicines Fund announced in July to support patients with any condition to get early access to the most clinically promising treatments where further data is needed for decision making. One potential template could be the data collection agreement between NHS England, NICE, the UK Cystic Fibrosis Trust, and Vertex Pharmaceuticals relating to use of Orkambi^®, Symkevi^®, Kalydeco^®, and Kaftrio^®. If conditional reimbursement is soon to become a wider reality, manufacturers and evidence synthesis and economic modelling vendors must be routinely included as meaningful partners in such activities.

Lastly is a renewed commitment to reduce health inequalities, principally across socio-economic gradients. Manufacturers must bolster their research into the socio-economic drivers of disease epidemiology, severity, and outcomes accordingly. Currently, the NICE submission template asks whether the use of a new technology is likely to raise any equality issues. Experience has shown that the response “None expected” has typically been recorded with little or no committee discussion. Expect greater rigor in the consideration of health inequalities over the next 5 years and beyond. 

Pillar 2. Dynamic, living guideline recommendations
NICE will move away from producing full static guidelines to a more modular, living approach to recommendations, enabling rapid updates by more efficient processes. This is particularly welcome news for Pharma, as offering manufacturers a responsive critique to emerging evidence contrasts with the current “we’ll look again in 5 years” approach. Future review cycle length is not stated, but one imagines a utopia where meaningful new evidence would immediately trigger a guideline update that would be rapidly disseminated to practitioners. This radical change to NICE’s portfolio will evolve the scientific basis of post-launch evidence synthesis and data generation. The opportunity for ongoing incorporation of robust evidence (including that from the “real world”) into living guidelines will invigorate the scientific discipline of market access, too often subject to the whim of brand and marketing bias. The prospect of dynamic economic models that support living life cycle management is an exciting one that will unify the many strands of health economics and outcomes research that often never meet. 

Pillar 3. Effective guidance uptake to maximise impact
An increased focus on adoption of guidance across the NHS will include identification of new funding approaches to remove barriers to uptake, an extension to NICE’s authority. Acknowledgment of their role in funding new technology inevitably creates opportunities for some companies and threats for others. How NICE will discharge this new role is not yet clear but presumably could include either expanding the payer horizon where appropriate (eg, where local authority-funded care is usual) or proactive disinvestment guidance for less cost-effective interventions. Each of these activities creates additional opportunities for value demonstration of new and established health technologies.

Pharma must make the case for inclusion in NICE’s partner network to build systems to routinely monitor the uptake and impact of its guidance and to measure the value of its work.

Pillar 4. Leadership in data, research, and science
NICE aims to provide thought leadership on the innovative use of real-world data and analytics. The life sciences industry is cited as a collaborating partner in driving not only the research agendas but also funding priorities. The opportunity for companies here is tangible, especially if coupled with data innovations such as NHS Digital’s General Practice Data for Planning and Research service, giving access to much larger datasets of health record data.

Novel engagement with patient and public opinion to inform the evidence base for guidance development will be explored. The involvement of well-informed patient advocacy bodies as trusted third parties in data sharing agreements (see above) is a welcome approach that enhances patient and public engagement in decision making. More flexible approaches to guidance development that respond to idiosyncrasies of certain diseases would be welcome, for example those for which the burden of illness is poorly reflected by the societal preference measures.

Lastly, NICE states that inclusion of environmental impact data in future guidance to reduce the carbon footprint of health and care services will become the norm. Although the weight this would carry in any reimbursement decision remains unclear, it is imperative that such assessments become a routine feature of HTA. A proactive positive response from Pharma in this regard would do much for public relations.

Making it happen

Key among a series of internal transformations is the establishment of a business development function to identify and secure new sources of income, and to respond to new opportunities to enhance NICE’s impact. This is the clearest practical sign of NICE’s increased willingness for industrial partnership and collaboration. It is no accident that proactive technical engagement into the STA process was introduced in parallel with the decision to begin charging applicants for the privilege; negative decisions are not only bad for patients but also bad for business.

Implications of a nicer NICE beyond the UK

Arguably, the UK punches above its weight in the field of decision science and the NICE 5-year strategy signals an intent to strengthen its international thought leadership position, likely through:

• Development of frameworks for living economic models to support dynamic guidance
• Maturation of real-world evidence (RWE) methods, especially for comparative effectiveness
• Operationalisation of “true” value-based pricing for conditional reimbursement
• A more collegiate relationship with Pharma

Ironically, the UK’s departure from the European Union (and with it the Medicines and Healthcare Products Regulatory Agency from the European Medicines Agency) may well have unlocked the path to a more seamless process from market approval to market access, with stronger engagement with manufacturers throughout. Whilst this has been discussed on the European level, the idea of parallel advice between regulatory and reimbursement bodies is more realistically achieved on a national rather than a continental level, when only 2 organizations have to agree on the approach, as opposed to 30. 

Summary

As the pandemic (and perhaps Brexit) smoke clears, NICE’s bold near-term future vision is compelling. Living guidelines, aligned to the intelligent use of RWE, and environmental impact assessment highlight an ambitious programme with myriad opportunities for Pharma to demonstrate value, IF the industry proactively embraces them. Can it afford not to?

The article should be referenced as follows:
Poole C, Tolley K. Is NICE becoming nicer? HTA Quarterly. Winter 2021. https://www.xcenda.com/insights/htaq-winter-2021-nice-becoming-nicer/

Sources

• https://digital.nhs.uk/data-and-information/data-collections-and-data-sets/data-collections/general-practice-data-for-planning-and-research
• https://www.england.nhs.uk/2021/07/nhs-england-announces-new-innovative-medicines-fund-to-fast-track-promising-new-drugs/
• https://www.gov.uk/guidance/innovative-licensing-and-access-pathway#partners
• https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/chte-methods-and-processes-consultation
• https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data-collection-agreement
• https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/charging
• https://www.nice.org.uk/about/who-we-are/corporate-publications/the-nice-strategy-2021-to-2026
• https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/company-evidence-submission-template-apr-17.docx