The impact of rarity in NICE’s health technology appraisals

Clarke, S Lindsey; Ellis, Michelle; Brownrigg, Jack

doi:10.1186/s13023-021-01845-x

Cited by 15 publications

(10 citation statements)

References 8 publications

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“…Using these alternative methods, based upon achievement of an ICER below standard thresholds, patients could have received access to caplacizumab approximately 5 months earlier, and the appraisals for teduglutide and pirfenidone would have resulted in a positive recommendation following appraisal consultation meeting 1 when neither of these products is available at the time of writing, over 5 years from initial submission. Our results align with other recent work, which showed that orphan medicines were subject to a significantly longer mean time in the NICE process than non-orphan medicines: 370 versus 277 days [ 9 ]. The majority of pharmaceutical companies have a global presence and need to make decisions on where to launch and when.…”

Section: Discussionsupporting

confidence: 92%

“…Out of 797 technology appraisals conducted by NICE to date [ 8 ], 21 have been HSTs. A review of NICE appraisals between 2015 and 2020 found that only a third of orphan medicines appraised had been reviewed via the HST route, with the remainder going through the standard STA process [ 9 ]. The review found that orphan medicines were subject to a significantly longer mean time in the NICE STA process than non-orphan medicines (370 [ n = 44] vs 277 [ n = 118] days; p < 0.0001) and that orphan medicines in the STA process were disadvantaged by worse outcomes with respect to positive recommendations than those of orphan medicines assessed by HST (100% of HSTs recommended in full vs 73% of orphan STAs).…”

Section: Introductionmentioning

confidence: 99%

“…In addition, all five NICE Committees (four STAs and the HST Committee) may now assess products for diseases routed to HST rather than the assessment only being conducted by the HST Committee. The STA Committees have traditionally been less lenient when faced with the limited data available in rare diseases [ 9 ]. This underscores the importance of both ensuring consistency in the approach to the assessment of rare diseases across committees and closing the gap between HST and STA processes and applicable WTP thresholds for products that narrowly miss the bar for HST (see Sect.…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products

Lee

McCarthy²,

Saeed

et al. 2022

PharmacoEconomics Open

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Background The National Institute for Health and Care Excellence (NICE) is responsible for ensuring that patients in England and Wales can access clinically and cost-effective treatments. However, NICE's processes pose significant reimbursement challenges for treatments for rare diseases. While some orphan medicines have been appraised via the highly specialised technology route, most are appraised via the single technology appraisal programme, a route that is expected to be increasingly used given new more restrictive highly specialised technology criteria. This often results in delays to access owing to differences in applicable thresholds and the single technology appraisal approach being ill-equipped to deal with the inevitable decision uncertainty. NICE recently published their updated methods and process manual, which includes a new severity-of-disease modifier and an instruction to be more flexible when considering uncertainty in rare diseases. However, as the threshold gap between the single technology appraisal and highly specialised technology programmes remains, it is unlikely that these changes alone will address the problem. Objective We explored the potential impact of quality-adjusted life-year weights in decision making. Methods We explored the impact of NICE's new severity-of-disease modifier weighting and two alternative methods (the use of alternative quality-adjusted life-year weights and the fair rate of return), using three recent single technology appraisals of orphan medicines (caplacizumab, teduglutide and pirfenidone for mild idiopathic pulmonary fibrosis). Results Our results suggest NICE's severity-of-disease modifier would not have affected the recommendations. Using alternative methods, based upon achievement of an incremental cost-effectiveness ratio below standard thresholds, patients could have received access to caplacizumab approximately 5 months earlier, and the appraisals for teduglutide and pirfenidone would have resulted in a positive recommendation following appraisal consultation meeting 1 when neither of these products was available over 5 years from the initial submission. Conclusion Ultimately, moving from a restrictive end-of-life modifier to one based on disease severity is a more equitable approach likely to benefit many therapies, including orphan products. However, NICE's single technology appraisal updates are unlikely to result in faster reimbursement of orphan medicines, nor will they address concerns around market access for orphan medicines in the UK.

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Section: Discussionsupporting

confidence: 92%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products

Lee

McCarthy²,

Saeed

et al. 2022

PharmacoEconomics Open

View full text Add to dashboard Cite

show abstract

“…A survey in 2020 found that thirteen of thirty-two investigated countries have included supplementary processes specifically targeting orphan drugs. In the UK, NICE has introduced the highly specialized technology evaluation process for ultra-orphan drugs targeting the disease with the prevalence of <1 in 50,000, where the ICER threshold increases to £100,000 (32). Some HTA agencies implement MEAs to generate additional evidence for later reappraisal to accelerate access to orphan drugs (33).…”

Section: Discussionmentioning

confidence: 99%

A systematic literature review of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment

Wang

Qiu

Nikodem³

et al. 2022

Int J Technol Assess Health Care

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Objectives This review intends to provide an overview of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment (HTA) among the different agencies. Methods A systematic literature search was performed in MEDLINE and EMBASE databases from inception to July 2020. The studies were eligible for inclusion if they conducted a quantitative analysis of HTA’s previous decisions for cancer drugs. The factors with p-values below the significance level of .05 were considered as the statistically significant factors for HTA decisions. Results A total of nine studies for six agencies in Australia, Belgium, France, South Korea, the UK, and Canada were eligible to be included. From the univariable analysis, improvements in clinical outcomes and cost-effectiveness were found as significant factors for the agencies in Belgium, South Korea, and Canada. From the multivariable analysis, cost-effectiveness was found as a positive factor for the agencies in the UK, South Korea, and Canada. Few factors related to characteristics of disease and technology were found to be significant among the included agencies. Conclusions Despite the different drug reimbursement systems and the socioeconomic situations, cost-effectiveness and/or improvement on clinical outcomes seemed to be the most important factors for recommendations of cancer drugs among the agencies.

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“…16,21,23 Drugs for the treatment of rare diseases typically have higher costs and higher data uncertainty driven by the lower number of patients; as such frameworks and thresholds established for the assessment of costs per quality-adjusted life years might fall short for rare and orphan diseases such as GPP. [49][50][51] The cost burden is primarily mitigated by accelerated and sustained therapeutic success. 5,23,52 Not specific to GPP, the mean (range) annual treatment costs across 20 non-oncological orphan medicines in Germany was €296 881 (€27 811 to €1 647 627).…”

Section: Source: Cited In Tablementioning

confidence: 99%

A narrative review of the socioeconomic burden associated with generalised pustular psoriasis

Zimmermann

Hofmann

Chiu

2023

Experimental Dermatology

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Generalised pustular psoriasis (GPP) is a rare, heterogeneous, chronic and potentially life-threatening autoinflammatory skin disease, characterised by episodes of widespread eruption of sterile, macroscopic pustules that occur with or without symptoms and signs of systemic inflammation. 1,2 The aberrant activation of the interleukin-36 pathway in patients with GPP causes an inflammatory reaction and widespread eruption of pustules. Flares, the acute phase of GPP involving widespread pustules, skin lesions and erythema, 3 typically last 2-5 weeks, 4 but may persist longer than 3 months and may be accompanied by systemic symptoms such as fever, pain, and fatigue. [5][6][7] While the most dominant cutaneous symptoms are pustules, erythema and scaling,

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The impact of rarity in NICE’s health technology appraisals

Cited by 15 publications

References 8 publications

The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products

The Challenge for Orphan Drugs Remains: Three Case Studies Demonstrating the Impact of Changes to NICE Methods and Processes and Alternative Mechanisms to Value Orphan Products

A systematic literature review of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment

A narrative review of the socioeconomic burden associated with generalised pustular psoriasis

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