Points of View Results of the evaluation of HST at NICE in the U.K. and the factors taken into account in decision making in addition to ICER
Factors taken into account in decision making in addition to ICER

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Takahiro Shiraishi, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute
Yuki Miura, Senior Researcher, Pharmaceutical Industry Policy Institute

SUMMARY

1. Introduction

The National Institute for Health and Care Excellence (NICE) in the UK has developed evaluation guidance based on a review of clinical evidence and cost effectiveness submitted by pharmaceutical companies for medical technologies that have obtained marketing approval. The National Institute for Health and Care Excellence (NICE) prepares evaluation guidance based on the review of clinical evidence and cost effectiveness submitted by pharmaceutical companies. The guidance takes into account various factors in addition to the results of clinical evidence and cost effectiveness within the framework of technology appraisal (TA), and is used to develop recommendations for use by the National Health Service (NHS) in the recommendations made by NICE. Health Service (NHS) in the recommendations made by NICE. ¹⁾

Various evaluation processes have been established for the evaluation guidance, depending on the number of indications for the targeted medical technology, etc. In 2015, HST (Highly specialized technologies; evaluation of medical technologies for ultra-rare diseases) ²⁾ was introduced as one of the TA frameworks. The criteria for ^the selection of HST targets include: (1) very few patients (prevalence of the disease in the UK is 1 in 50,000 or less), (2) very low number of patients (prevalence of the disease in the UK is 1 in 50,000 or less), (3) low number of patients (prevalence of the disease in the UK is 1 in 50,000 or less), and ( ⁴⁾ low number of patients with the disease (prevalence of the disease in the UK is 1 in 50,000 or less). (i.e., the prevalence of the disease is less than 1 in 50,000 or 1,100 in the U.K.), (ii) less than 300 patients are suitable for the technology among the indications approved in the country (less than 500 patients in total if there are multiple indications), (iii) extremely rare diseases that significantly shorten life or significantly impair quality of life, (iv) no other adequate treatment is available, and (v) the disease is not amenable to treatment, or is likely to provide a greater benefit than existing treatments, all of which must be met. These selection criteria have been changed as of April 2019 (Figure 1), and the content of these criteria has been tightened and clarified except for (1), which remains unchanged. We suspect that one of the reasons for this is that the NHS is facing budget limitations amid tight healthcare finances in the UK. On the other hand, there is also the view that the criteria have been more clearly defined and the interpretation of HST eligibility has become more standardized, leading to the elimination of the cases that in the past led to appeals over meeting the selection criteria and caused delays in patient ^access5).

In addition, if these selection criteria are met, the incremental cost-effectiveness ratio ( ^ICER6 ) (£20,000 to £30, ^000/QALY7) for TA) will be significantly increased (£100,000 to £300,000/QALY), and the TA will be subject to the same criteria as the other TAs (£20,000 to £300,000/QALY). In addition, qualitative factors (e.g., family burden and patient's potential for social participation, which were not quantitatively incorporated in ICER) may be considered more in the decision-making process than in TA and taken into account in making ^{recommendations8)} in the appraisal (comprehensive evaluation) process.

This paper examines decision factors other than ICER and their trends resulting from the determination of medical technology recommendations, with the objective of identifying the diverse values of medicines considered in decision making despite their financial impact, for the evaluation guidance posted on the NICE ^website3) as of June 6, 2024. The study was conducted by NICE.

Survey Methodology

The target of this paper is all 31 HST evaluation guidance (HST1-31) published on NICE's public ^website3), and 27 ^cases9) identified on the website were surveyed and tabulated (Table 1). In Table 1, brand names (product names) are those of the United Kingdom. The ICERs in the evaluation guidance and the weights (weights) of 1 to 3 applied according to the QALYs gained by the new technology during the patient's lifetime are indicated ^.10) The application of weights 1 to 3 adjusts the ICER threshold between 100,000 and 300,000 ^{pounds/QALY6)}. ¹¹⁾⁾; the Scheme is denoted PAS (Patient Access Scheme) and MAA (Managed AccessAgreement), the contents of which are discussed below.

As a result of the evaluation, Typeofrecommended or deprecated for use in the NHS is indicated.

In addition to QALY and Cost, which are used when calculating ICER, the factors reported in the evaluation guidance are organized into three categories: Carer (impact on the quality of life of caregivers and others), Innovation, and Equity, with five for Carer, Innovation, Factors such as QOL values that are included in the calculation of ICER were defined as quantitative evaluations, while factors that are not included in the calculation of ICER were defined as qualitative evaluations. Among the qualitative factors, those that were explicitly stated to be a factor in the decision-making process for the recommendation were considered in the decision-making process, while those that were not explicitly stated were distinguished as mentioned.

For Carer, those that were quantitatively incorporated into the ICER and qualitatively mentioned in the evaluation guidance as being taken into account in the decision-making process were classified as ◎, those that were quantitatively incorporated into the ICER only were classified as ○, and those that were not quantitatively incorporated into the ICER but were qualitatively mentioned as being taken into account in the decision-making process were classified as ●. Innovation and Equity are ○ if they are mentioned in the evaluation guidance but it is unclear whether or not they were taken into account in the decision-making process, and - if they are not mentioned in the evaluation guidance. For Innovation and Equity, those that were mentioned in the evaluation guidance but it was unclear whether or not they were considered in the decision-making process were scored ○, those that were mentioned but it was unclear whether or not they were considered in the decision-making process were scored △, and those that were not mentioned were scored -.

These scales in the above were determined by the two authors within the limits of what could be read from the evaluation guidance, and if there was a divergence in interpretation between the two authors, the final decision was made through consultation between them.

Results

3-1. type of recommendation (Figure 2)

In the results of this evaluation guidance, 26 (96%) of the 27 cases included in the HST were recommended, while 1 (4%) was not recommended.

For reference, in TA (as of August 29, 2024), the framework under which medicines are generally evaluated at NICE, 589 (44%) were recommended and 162 (12%) were not recommended out of a total of 1336 cases. HST was considerably higher than TA.

One of the reasons for the one Not recommended case, HST27, is that no application for PAS was made (this case is conditionally recommended in the NHS in ^Scotland12). Even if the drug is evaluated as Not recommended by NICE, the pharmaceutical company can propose PAS, and if PAS is approved, the price is discounted to become ^{Recommended13)}. MAAs are a mechanism for NICE to recommend drugs with large uncertainties in therapeutic efficacy, such as rare diseases, within a timeframe for early access, and PASs are part of the MAA framework. ¹⁴⁾ PASs and MAAs are described in Table 1 in the Scheme. PAS and MAA are described in the Scheme in Table 1.

3-2. impact on caregivers' QOL (Figure 3)

With respect to Carer, of the 27 cases included in the tabulation, 5 (18%) were described in the evaluation guidance as being quantitatively incorporated into ICERs and also qualitatively considered in decision-making, while 8 (30%) were only quantitatively incorporated into ICERs (no qualitative decision-making consideration).

Seven (26%) were mentioned as qualitatively taken into account in the decision-making process but not quantitatively incorporated into the ICER, and four (15%) were mentioned in the evaluation guidance but it was unclear whether or not they were taken into account in the decision-making process. And 3 (11%) were not mentioned in the evaluation guidance. Further disaggregated results (Figure 4) are presented below.

3-3. results for Innovation and Equity (Figure 5)

Of the 27 HST cases, 3 (11%) were qualitatively considered in the decision-making process, 17 (63%) were mentioned in the evaluation guidance but it was unclear whether or not they were considered in the decision-making process, and 7 (26%) were not mentioned in the evaluation guidance. Equity was qualitatively taken into account in decision-making in 4 cases (15%), mentioned in the evaluation guidance but unclear whether it was taken into account in decision-making in 22 cases (81%), and not mentioned in the evaluation guidance in 1 case (4%). One case (4%) was identified.

Conclusion and Discussion

Type of recommendation (Figure 2) 4-2.

A comparison of the percentage of Recommended cases in TA and HST shows that TA is 44% while HST is 96%. The reason why HST tended to be considerably higher as a percentage than TA is that the ICER threshold is raised significantly when evaluated in HST, and also because the appraisals process In addition, qualitative factors (e.g., family burden and possibility of patient participation in society) are taken into account in the decision-making process more than in TA, and may be taken into account in making a ^{recommendation8)}.

4-2. impact on caregivers' quality of life (Figure 4)

In a high percentage, 24 (89%) of the 27 cases included in the HST tally, there was either quantitative inclusion in the ICER or qualitative decision-making consideration or mention of Carer's factors.

In 20 of those cases (74%), the incorporation into the quantitative ICER or consideration for qualitative decision-making was made, a high level.

In terms of the breakdown, 48% of the cases were quantitatively incorporated into ICERs with or without qualitative decision-making considerations, and 44% were qualitatively taken into account in decision-making with or without quantitative ICER incorporation.

One of the reasons for the high percentage of Carer factors being included in quantitative ICERs or considered qualitatively in decision-making is that the NICE manual states that "health-related quality of life that is important to patients and their caregivers may be included in cost-effectiveness analyses" (NICE Manual, p. 11). ¹¹⁾. In addition, as stated in the selection criteria for HST, the fact that many illnesses are associated with severe disabilities and that pediatric illnesses frequently involve parental care is also a factor that is more likely to be apparent. In addition, in many cases, the opinions of patient ^{experts15) and} clinical experts are fully considered, and factors with social benefits are taken into account in decision making. For example, in the case of HST17, comments on the time burden associated with caregiving (reduced work hours and need for long periods of time off for caregiving), financial burden, and frequency of hospital visits were clearly stated, and it was reported that the possibility of reducing these burdens through drug treatment was recognized and considered in the decision-making process ²⁾.

4-3. innovation, equity (Figure 5)

The Innovation factor evaluates the magnitude of benefits to patients, not only from a technical ^{perspective16)}. For example, innovations in dosage forms that improve patient adherence, the establishment of treatments for diseases for which there is a high therapeutic need, and the emergence of drugs with new mechanisms of action may increase treatment These are very important factors to increase treatment options and treatment satisfaction. In the present results, Innovation was qualitatively considered in decision-making in 3 cases (11%), but in 20 cases (74%), including those cases where it was mentioned but it was unclear whether it was considered in decision-making. Specifically, HST25 stated that qualitative decision-making considerations were taken into account in the decision-making process, "We evaluated and took into account the fact that treatment with this drug can prevent disease progression, reduce the number of kidney stone procedures, and reduce the need for dialysis or transplantation ^.) The benefits to the patient, such as avoidance of invasive treatments such as procedures and surgeries, were accurately taken into account in the decision-making process as a value of medical technology, apart from ICER, as an element of Innovation.

The Equity component is "the right to a certain level of medical care should be guaranteed regardless of ^wealth18)," and a recent report by the "International Society for Pharmacoeconomics and Outcomes Research (ISPOR)2024 (ISPOR), a recent report on the issue of economic disparity leading to health problems, etc. has been ^presented19). In the United Kingdom, where the results of cost-effectiveness analysis are used as one of the factors in determining reimbursement, it is assumed that cost-effectiveness analysis is becoming one of the most important factors to be prioritized in order to ensure early access to superior drugs for ultra-rare ^diseases20). In the present study, Equity was qualitatively taken into account in decision-making in 4 cases (15%), but in 26 cases (96%), including those where it was mentioned but it was unclear whether it was taken into account in decision-making or not. For example, HST31 concluded that "for this disease, people from Black, Asian, and other minority ethnic backgrounds have increased cardiometabolic health risks at lower BMI thresholds than people from White ethnic backgrounds, but the drug is equally applicable regardless of ethnicity. Innovation factor, and Equality factor, ^and the drug is equally applicable regardless of ethnicity.

Although the percentage of both Innovation and Equity factors is smaller than that of Carer's factor, it is important to note that a certain number of factors are taken into account in qualitative decision-making in addition to the quantitative evaluation in ICER.

In addition, although not listed in Table 1 of this report, the evaluation guidance also includes other factors, such as HST11, which states that "there are considerable uncaptured benefits related to vision maintenance in children, which were considered qualitatively in the decision-making process. Although these examples are still very few, it is possible to read that NICE tends to consider them as important decision-making factors, which is a characteristic of NICE's appraisals that consider qualitative factors to complement the quantitative evaluations in ICER. This can be inferred to be a manifestation of the characteristics of NICE's appraisals, which take into account qualitative factors to supplement the quantitative assessment in ICER.

Conclusion

This paper examines the decision-making factors other than ICER and their trends resulting from the determination of medical technology recommendations, covering the HST evaluation guidance posted on the NICE ^website3) as of June 6, 2024.

The percentage of HST evaluation guidance in which the Carer factor was quantitatively or qualitatively considered in the decision-making process as one other than medical value in the ICER was high at 74%. This may be due to the fact that HST is more likely to be manifested in very serious diseases.

In addition, as other factors that cannot be quantitatively evaluated or supplemented by ICER, Innovation and Equity were confirmed to have a certain number of reports at 11% and 15%, respectively, suggesting the importance of considering factors other than ICER in order to appropriately evaluate the value of medical technology, as some of the evaluation guidance cases mentioned above The results of this survey suggest that the UK's HST is a good example of the importance of considering factors other than ICER to properly assess the value of medical technology.

The results of this survey confirmed that, although the HST in the U.K. is for diseases with extremely high unmet medical needs that meet the selection criteria of HST, the various values of pharmaceuticals, including efficacy, safety, and medical economy obtained from clinical trials, as well as factors that bring benefits to society, tend to be considered in decision making and evaluated. The results of the survey confirmed the trend that the various values of pharmaceuticals are taken into account in decision-making and evaluated. In order to understand the actual situation of appropriate value evaluation of innovative medical technology in HST even in the midst of tight healthcare finances, it is necessary to watch the trend of factors considered in decision making in addition to ICER.

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