How are patient inputs considered in HTA? A thematic document analysis of NICE ultra-rare disease appraisals

Abstract

Patient organisations are increasingly involved in HTA. Given this, it is important to understand what these organisations contribute and how their voices are accounted for in the decision-making process. This study characterises inputs from patient organisations and/or their nominated patient experts in technology appraisals for ultra-rare diseases in England and Wales and seeks to understand how these are considered in NICE final recommendations. We thematically analysed all HST appraisals completed between January 2022 and August 2024 (N = 15). We appraised inputs from patient organisations' and experts' written submissions, the novelty of patient inputs, as well as financial ties between contributing organisations and the manufacturer of the technology being appraised. We compared themes identified with those found in the Final Evaluation Determination documents to understand how and to what extent patients' inputs were considered in NICE final recommendations. We found that patient submissions mainly focused on disease aspects (54%). Patients raised concerns on access challenges, caregiver burden, and mental health impacts. Most patient themes overlapped with manufacturers' submissions (82%) and doctors' testimonies (45%), with most novel insights focusing on access issues and mental health. Patient organisations reported receiving funding from the technology manufacturer in most appraisals, with amounts ranging from £5,000 to £74,113. Approximately half of patient inputs were explicitly mentioned in NICE final decision documents, with some considerations being neglected despite being raised by patients. While NICE incorporates many issues of importance to patients, there is room for improvement to ensure all aspects patients deem important are captured. Further research could pinpoint optimal areas for patient contributions and assess their impact.

Keywords: HTA; NICE; Patient organisations; Rare diseases.

Fig. 1

3I’s framework applied to the reimbursement decision-making process. Abbreviations HTA, Health Technology Assessment. Notes Adapted from Smith et al. (2014) [30]. Only key stakeholders are included and additional layers of complexity, including regulators and pricing dynamics, which ultimately impact access, have intentionally been omitted from this figure for simplicity. When considering interests, the HTA body, such as NICE, is expected to prioritise public health needs and allocative efficiency, while the manufacturer return on investments and market access. On the other hand, patients – either individually of as part of patient organisations – are the designated users of the technology being appraised, and, as such, have a vested interest in it being reimbursed as a vehicle to improve their own health. Similarly, clinicians are guided by the intrinsic altruistic interest of seeing their patients have access to potentially effective medicines. However, profit motives guiding doctors cannot be ruled out in the case of conflicts of interests. Turning to ideas, some HTA agencies, such as NICE, might be guided by a pragmatic cost-effectiveness utilitarian approach, while others might focus on different priorities, such as clinical benefit [32]. Nevertheless, they are broadly guided by a prescriptive approach positing that they act in the interest of the public by addressing information asymmetries between innovators and users of medicines. Manufacturers, on the other hand, advocate for a more flexible regulatory environment supporting innovation and access, which, for example, translates into less stringent evidence requirements to demonstrate the efficacy of their products, such as a wider use of surrogate endpoints and of phase II trials [33]. Patient organisations and experts may push for broader inclusion of patient perspectives and qualitative evidence in HTA, while clinicians are expected to uphold evidence-based approvals. Finally, an example of the institutions domain is the gatekeeping role of NICE to the English and Welsh healthcare market, with approximately 60 million potential consumers, which confers notable power to the body [34]. In fact, NICE recommendations are binding, and the National Health Service (NHS) is legally obliged to fund and resource medicines and treatments NICE recommends [35]. Similarly, stakeholder engagement platforms and mechanisms, such as public consultations and expert committees, provide avenues for actors to influence HTA processes within established institutional frameworks