Most pharmaceutical products hoping to launch in the UK will be assessed by NICE as a Single Technology Appraisal (STA) or a Highly Specialised Technology (HST). These assessments are critical for market access, with a positive recommendation securing national reimbursement and providing commercial access to the UK’s publicly funded healthcare system.
NICE’s published recommendations also have the power to positively influence the perception of a new product within clinical pathways, influence prescriber behaviour, and, beyond the UK, even influence global payers. Conversely, a negative NICE opinion can be a significant roadblock to patient access and commercial success in the UK.
In short, the NICE assessment is far from a tick-box exercise and is absolutely critical to a product’s UK launch strategy. So, what are the most common reasons that submissions fall short? And how can future submissions learn from past mistakes?
In this article, we will explore the top 5 reasons that NICE STA/HST submissions fail, how these can be anticipated, and more importantly, avoided!
Reason 1: Misalignment to NICE’s evidence expectations
The problem: In submissions, there are often misalignments between the data submitted and what NICE actually wants to see. NICE has preferred methodology, including a reference case which is a standardised framework for how cost-effectiveness analyses should be conducted. If a submission deviates from this reference case without convincing justification, NICE are likely to reject the modelling assumptions or request major revisions.
The example: A company has submitted a cost-effectiveness model to compare a new treatment to no intervention (as there are no targeted treatments in their disease area). NICE highlights that patients do, in fact, in practice receive some form of standard care, meaning that the comparator expectations have not been met. This results in a rejection of the model and a request for rework.
The solution: NICE offers the opportunity for companies to seek early scientific advice (ESA), where companies can test their proposed comparators, modelling assumptions, and evidence plans against NICE’s expectations. Engaging with NICE early, ideally before finalising pivotal trials or economic model structures, can help ensure the submission is aligned with NICE’s expectations from the outset, avoiding costly missteps later on.
Top tip: NICE’s ESA is non-binding, meaning that the advice they give does not have to be followed. In addition, teams are kept separate, so the ESA process will have no influence on the actual submission whatsoever. We therefore recommend that companies ask the hard questions when seeking ESA from NICE!
Reason 2: Inadequate definition of the target population
The problem: When submitting for reimbursement, it can be tempting to define a wide population that maximises reach or reflects an entire trial cohort. However, vague, overly broad, or clinically unrealistic definitions can create serious problems in NICE assessments. NICE wants to understand exactly who treatments are for, and whether the evidence is sufficient to support use in those patients. If the proposed population does not align with real-world practice, NICE will struggle to identify the right patients; if they cannot do that, they cannot (and will not!) make a positive recommendation.
The example: A company submits an appraisal for a respiratory therapy and defines the population as “all adults with chronic respiratory symptoms”. However, NHS treatment guidelines segment patients based on disease severity. Without clearly targeting one of these patient groups, NICE struggles to assess cost-effectiveness and the submission stalls.
The solution: Companies should carefully consider their target population, taking into account the trial inclusion criteria and real-world UK practice. If a treatment may benefit different subgroups in different ways, this should also be acknowledged, and subgroup analyses should be provided. The end goal here is to convince NICE that your evidence robustly supports treatment use in the right patients, in the right setting.
Reason 3: Inadequate economic modelling
The problem: With the UK being a cost-effectiveness market, the economic model is at the heart of a NICE submission. When assessing cost-effectiveness, NICE will not simply look at the ICER headline; instead, they will pick apart the model’s structure, assumptions, and assess if the model truly reflects real-world practice. If NICE find that the model is overly complex, unjustified, or poorly designed, they will not trust its outputs.
The example: A company submits a model projecting long-term treatment benefits, but the rationale for how outcomes are extrapolated beyond the trial period are unclear. NICE tries to follow the logic, but assumptions span multiple tabs, and key inputs are lacking source references. As a result, NICE cannot verify the ICER, and the submission is stalled until the model is resubmitted.
The solution: Economic models do not need to overly sophisticated or complex. Instead, companies should focus on building a model that can be clearly understood and accurately captures the cost-effectiveness of a new treatment. Submissions should clearly outline the model structure, justify inputs (with links to evidence/expert consensus), and include sensitivity analyses to assess uncertainty openly. The end goal is for NICE to be able to follow the logic, test the assumptions, and reach the same conclusions as the company when reviewing a model.
Reason 4: Excluding key stakeholders
The problem: NICE highly values input from key stakeholders, especially from clinicians and patient groups. Insights from these key stakeholders provide an understanding of how treatments will actually impact the UK, beyond hypotheticals and lists of data. In submissions, companies often overlook stakeholder engagement, which can lead to a disconnect between the submission and its impact on reality (and your value proposition being viewed as purely theoretical!).
The example: A company submits and appraisal for a chronic condition but includes limited input from patients and clinicians. In the committee meeting, NICE raises concerns around how the treatment would be used in real clinical practice and is unsure if the treatment is meeting the most important outcomes for patients. Without the patient voice, NICE is unable to verify this and is uncertain of the treatment’s real-world relevance.
The solution: Engage with relevant stakeholders early, meaningfully, and often. Advisory boards with clinicians and interviews with patients/carers can be effective methods of collecting insights, which can then be used to shape a treatment’s value narrative. These sorts of inputs can communicate benefits that numbers and data alone cannot, particularly for rare diseases, chronic conditions, or where quality of life is greatly impacted.
Reason 5: Overcomplicating it!
The problem: Dense text, overly complex language, and hundreds of pages have the potential to drown out even the best data and value messages. NICE reviewers and committee members may miss the key information if submissions fall into this pitfall.
The example: A company submits a lengthy STA for a novel therapy, supported by promising data. However, the submission is very long-winded, filled with specialist jargon, and lacks a clear line of argument. NICE struggles to understand the core value proposition so begins to lose confidence in the value of the treatment.
The solution: Submissions do not need to be overcomplicated and should instead communicate the key messages as simply as possible in plain English. Logical structures and visual aids can be simple, but very valuable tools in a NICE submission.
Top tip: When preparing a submission, the writers can get lost in the details and lose sight of the big picture. Conducting an internal mock review can test whether a story is easy to follow ahead of the real thing
Conclusion
Getting a positive NICE recommendation is not about overengineering or overpromising; it is about presenting convincing evidence for the right patients, in a way that is clear, credible, and aligned with NICE’s expectations. As we have seen, many submissions fall short not because the product lacks value, but because that value is not effectively demonstrated or communicated.
With the right support, these pitfalls are entirely avoidable. By leveraging ESA, stakeholder engagement, payer insights, and robust submission strategies, companies can build compelling, evidence-based value stories that resonate with the NICE committee.
At Remap, we support clients through every stage of the NICE process, from early strategic planning to final submission. To learn more about how we can help, visit our website or get in touch.