Decisions regarding National Institute for Health and Care Excellence (NICE) appraisals are not made in the boardroom alone. Input provided by patients, caregivers and clinicians is increasingly shaping outcomes.
Many companies focus mainly on clinical and economic data, underestimating the strategic weight of stakeholder insights in health technology assessment (HTA) submissions. During HST/STA evaluations, where uncertainty is common due to small populations or limited data, qualitative input from patients, caregivers and clinicians can tip the balance.
This article explores how integrating these insights early can enhance submission strength, credibility and appeal – and how Remap can help make it happen.
Why Stakeholder Input Matters in HST/STA Submissions
Stakeholder input plays a critical role in NICE’s Highly specialised technology (HST)/single technology appraisal (STA) submissions, as NICE explicitly values the perspectives of patients, carers, and clinicians to ensure its guidance reflects real-world needs and experiences.1 These insights help contextualise evidence gaps, highlight areas of unmet need, and validate outcomes such as health-related quality of life (HRQoL) that may not be fully captured in clinical trials. In some cases, stakeholder input has influenced decisions. For example, in the nusinersen appraisal for spinal muscular atrophy, input from patients, caregivers and clinicians contributed to a positive recommendation for use under a managed access agreement after an initial negative assessment.2,3
Who to Involve and When
Key stakeholders in NICE’s HST/STA processes include patient groups, clinical experts, and carers. NICE involves these stakeholder at defined points, such as scoping and committee hearings.4 Engaging with stakeholders early (before scoping or submission) helps ensure that outcomes, comparators, and assumptions reflect real-world clinical practice.5,6
Advisory boards with clinicians and patients can clarify where the new therapy fits in the current treatment pathways. Additionally, insights can be used to inform the economic model by confirming that assumptions such as treatment pathways, resource use, and selected overall survival curves are clinically plausible. This helps strengthen the submission and reduce uncertainty during appraisal.7
The patient voice provides first-hand evidence of the day-to-day burdens, limitations, strains and spill-over costs that are often not captured in clinical trials. Early engagement (through qualitative interviews, validated patient-reported outcome measures or carer-burden surveys) can quantify lost productivity, out-of-pocket expenses, and persisting unmet need, supplying concrete inputs for the cost-utility model (for example, disutility values and incremental resource use). Additionally, narrative testimonies from patients and carers help the NICE committee judge disease severity and equity considerations. Capturing and articulating these insights therefore strengthens the value case and aligns with NICE’s obligation to embed stakeholder involvement.
Strategic Approaches to Stakeholder Engagement
Effective stakeholder engagement in NICE HST/STA submissions ensures patient, caregivers and clinician input aligns with the key value messages of the technology, such as, unmet need, treatment benefits, or HRQoL improvements. NICE committees are more receptive when this input reinforces, rather than contradicts, the rest of the evidence base.8 Holding advisory boards early can help align messages, focus the discussions, and confirm that assumptions in the economic model (like treatment pathways or survival curves) are realistic.7
Structured input also helps. Research shows that the patient voice is more useful when supported by data from surveys or organised feedback, not just personal opinions.5 This makes it easier for committees to understand and use what stakeholders are saying. It is also important to be clear about who was involved, present their views honestly, and focus on points that are relevant to NICE decisions, such as real-world impact, disease burden, unmet need, HRQoL, and costs.1
Pitfalls to avoid
While NICE values stakeholder engagement and sees it as an important part of the appraisal process, poorly planned or inconsistent input can reduce its impact, or even undermine the submission. Common pitfalls include tokenism, where patients or clinicians are involved too late or in a limited way. NICE surveys show some stakeholders felt unsure whether their input made a difference, especially when no feedback was given.1 Early engagement (before scoping) and ensuring they have the information, time, and opportunity to contribute to key aspects of the submission, such as defining outcomes, validating assumptions, and shaping the value narrative, helps ensure their input is relevant, informed, and taken into account.7
Misalignment between stakeholder input and the submission narrative can also reduce credibility. For example, if the patient voice does not align with clinical evidence, committees may discount both.8 Holding advisory boards early helps align perspectives and clarify expectations.
Finally, inconsistencies between written and oral input can confuse committees.5 These issues can be avoided through preparation and coordination: reviewing key messages in advance and giving stakeholders the context they need.1,7
How Remap Consulting Supports Stakeholder Integration
Remap Consulting adopts a structured, patient-centric approach to stakeholder engagement, ensuring that insights from patients, carers, and clinicians are both meaningful and aligned with the requirements of HTAs. We assist clients in identifying relevant stakeholders early, to ensure that their input shapes the value narrative and informs the selection of outcomes and comparators. This includes facilitating advisory boards, structured interviews, or patient surveys to capture real-world evidence on treatment pathways, unmet needs, and quality of life. Our approach is informed by recognised best practices and aligned with guidance on effective stakeholder involvement.
Conclusion
NICE HST/STA success is not just about data; it is about demonstrating the value of the therapy through multiple lenses, including those of the people most affected.
If you are planning a UK launch, speak to Remap Consulting about how to meaningfully integrate stakeholder perspectives into your HTA strategy.
Get in touch with our team to explore how we can help strengthen your next NICE submission.
References
- National Institute for Health and Care Excellence. Patient and public involvement policy. 2024.
- National Institute for Health and Care Excellence. TA588- Nusinersen for treating spinal muscular atrophy. 2019.
- National Institute for Health and Care Excellence. Nusinersen for treating spinal muscular atrophy – 3rd appraisal committee meeting. 2019.
- National Institute for Health and Care Excellence. NICE health technology evaluations: the manual: Involvement and participation. 2022.
- Livingstone H, Verdiel V, Crosbie H, Upadhyaya S, Harris K, Thomas L. Evaluation of the impact of patient input in health technology assessments at NICE. Int J Technol Assess Health Care. Jan 29 2021;37:e33. doi:10.1017/S0266462320002214
- Remap Consulting, Atim JV, Rahul. Unlocking Access: The Role of Patient Engagement in Rare Disease HTA Accessed 10/07/2025, https://remapconsulting.com/hta/unlocking-access-the-role-of-patient-engagement-in-rare-disease-hta
- National Institute for Health and Care Excellence. NICE health technology evaluations: the manual. 2022.
- Hashem F, Calnan MW, Brown PR. Decision making in NICE single technological appraisals: How does NICE incorporate patient perspectives? Health Expect. Feb 2018;21(1):128-137. doi:10.1111/hex.12594