PP11 Would A Highly Specialized Technology Be Approved In England Under The New NICE Guidance?

2018 ◽  
Vol 34 (S1) ◽  
pp. 69-70
Author(s):  
Erika Turkstra ◽  
Silvy Mardiguian ◽  
Sangeeta Budhia

Introduction:In April 2017, the National Institute for Health and Care Excellence (NICE) updated its guidance for highly specialized technology (HST) appraisals, whereby it would automatically fund technologies for very rare diseases that fall below a threshold of an incremental cost-effectiveness ratio (ICER) of GBP 100,000 (USD 133,000) per quality-adjusted life year (QALY). In addition, NICE proposed to introduce a ‘QALY modifier’, weighting QALYs gained by the size of gain, which will advantage treatments that offer greater QALY gains.Methods:We reviewed all technologies reviewed through the NICE HST process until November 2017 and assessed whether additional QALYs may be awarded, and subsequently result in ICERs below the new NICE threshold.Results:Six products (eculizumab, elosulfase alfa, ataluren, migalistat, eliglustat, and asfotase alfa) have been through HST process. Within the appraisal documents, most analyses were cost consequence analyses with no ICERs reported. The estimated cost per patient per year ranged from approximately GBP 100,000 (USD 133,000) to GBP 400,000 (USD 532,000; listed prices). Of the six technologies, three resulted in at least ten incremental QALYs (eclizumab, elosulfase alfa and asfotase alfa). From the information in the public domain, it is unclear whether this would result in ICERs below GBP 100,000 (USD 133,000) per QALY.Conclusions:It may become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 (USD 133,000) per QALY.

Immunotherapy ◽  
2021 ◽  
Author(s):  
Wei Jiang ◽  
Zhichao He ◽  
Tiantian Zhang ◽  
Chongchong Guo ◽  
Jianli Zhao ◽  
...  

Aim: To evaluate the cost–effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost–effectiveness ratio to evaluate the cost–effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost–effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.


Trauma ◽  
2017 ◽  
Vol 21 (1) ◽  
pp. 45-54 ◽  
Author(s):  
Maxwell S Renna ◽  
Cristiano van Zeller ◽  
Farah Abu-Hijleh ◽  
Cherlyn Tong ◽  
Jasmine Gambini ◽  
...  

Introduction Major trauma is a leading cause of death and disability in young adults, especially from massive non-compressible torso haemorrhage. The standard technique to control distal haemorrhage and maximise central perfusion is resuscitative thoracotomy with aortic cross-clamping (RTACC). More recently, the minimally invasive technique of resuscitative endovascular balloon occlusion of the aorta (REBOA) has been developed to similarly limit distal haemorrhage without the morbidity of thoracotomy; cost–utility studies on this intervention, however, are still lacking. The aim of this study was to perform a one-year cost–utility analysis of REBOA as an intervention for patients with major traumatic non-compressible abdominal haemorrhage, compared to RTACC within the U.K.’s National Health Service. Methods A retrospective analysis of the outcomes following REBOA and RTACC was conducted based on the published literature of survival and complication rates after intervention. Utility was obtained from studies that used the EQ-5D index and from self-conducted surveys. Costs were calculated using 2016/2017 National Health Service tariff data and supplemented from further literature. A cost–utility analysis was then conducted. Results A total of 12 studies for REBOA and 20 studies for RTACC were included. The mean injury severity scores for RTACC and REBOA were 34 and 39, and mean probability of death was 9.7 and 54%, respectively. The incremental cost-effectiveness ratio of REBOA when compared to RTACC was £44,617.44 per quality-adjusted life year. The incremental cost-effectiveness ratio, by exceeding the National Institute for Health and Clinical Effectiveness’s willingness-to-pay threshold of £30,000/quality-adjusted life year, suggests that this intervention is not cost-effective in comparison to RTACC. However, REBOA yielded a 157% improvement in utility with a comparatively small cost increase of 31.5%. Conclusion Although REBOA has not been found to be cost-effective when compared to RTACC, ultimately, clinical experience and expertise should be the main factor in driving the decision over which intervention to prioritise in the emergency context.


2021 ◽  
pp. OP.20.00783
Author(s):  
Emma S. Ryan ◽  
Laura J. Havrilesky ◽  
Julia R. Salinaro ◽  
Brittany A. Davidson

PURPOSE: Two recent clinical trials have demonstrated that direct oral anticoagulants (DOACs) are effective as venous thromboembolism (VTE) prophylaxis in patients with moderate-to-high risk ambulatory cancer initiating chemotherapy. Patients with advanced ovarian cancer receiving neoadjuvant chemotherapy are at particularly increased risk of VTE. We performed a cost-effectiveness analysis from a health system perspective to determine if DOACs are a feasible prophylactic strategy in this population. METHODS: A simple decision tree was created from a health system perspective, comparing two strategies: prophylactic DOAC taken for 18 weeks during chemotherapy versus no VTE prophylaxis. Rates of VTE (7.3% DOAC v 13.6% no treatment), major bleeding (2.6% v 1.3%), and clinically relevant nonmajor bleeding (4.6% v 3.3%) were modeled. Cost estimates were obtained from wholesale drug costs, published studies, and Medicare reimbursement data. Probabilistic, one-way, and two-way sensitivity analyses were performed. RESULTS: In the base case model, DOAC prophylaxis is more costly and more effective than no therapy (incremental cost-effectiveness ratio = $256,218 in US dollars/quality-adjusted life year). In one-way sensitivity analyses, reducing the DOAC cost by 32% or raising the baseline VTE rate above 18% renders this strategy potentially cost-effective with an incremental cost-effectiveness ratio below $150,000 in US dollars/quality-adjusted life year. CONCLUSION: Further confirmation of the true baseline VTE rate among women initiating neoadjuvant chemotherapy for ovarian cancer will determine whether prophylactic dose DOAC is a value-based strategy. Less costly VTE prophylaxis options such as generic DOACs (once available) and aspirin also warrant investigation.


2018 ◽  
Vol 8 (2) ◽  
pp. 148-152 ◽  
Author(s):  
Melanie D. Whittington ◽  
Jonathan D. Campbell ◽  
R. Brett McQueen

Section 340B of the Public Health Service Act requires drug manufacturers to enter into price agreements with the Department of Health and Human Services. These agreements result in variation in the price paid to acquire a drug by sector, which complicates the price used in cost-effectiveness analyses. We describe the transactions and sectors in a 340B agreement using a multiple sclerosis drug. Cost-effectiveness estimates were calculated for the drug using drug prices from the manufacturer and payer perspective. We found the amount paid to the manufacturer (340B price) was a good value ($118,256 per quality-adjusted life-year); however, from the payer drug cost perspective, good value ($196,683 per quality-adjusted life-year) was not achieved. Given that emerging value frameworks incorporate cost-effectiveness, these price variations may have downstream negative consequences, including inaccurate coverage and reimbursement policy recommendations. Upcoming policy changes to the 340B program should incentivize pricing schemes hinged on transparency and value.


2019 ◽  
Vol 8 (11) ◽  
pp. 865-877 ◽  
Author(s):  
Maobai Liu ◽  
Shuli Qu ◽  
Yanjun Liu ◽  
Xingxing Yao ◽  
Wei Jiang

Aim: To compare the clinical effects and cost–effectiveness of maximum androgen blockade (MAB), docetaxel to androgen deprivation therapy (Doc-ADT) and ADT alone for the treatment of patients with metastatic hormone-sensitive prostate cancer in China. Methods: A network meta-analysis and a Markov model were adopted for effectiveness and economic evaluation. Results: The hazard ratios of overall survival and progression-free survival were 0.782 and 0.628 for Doc-ADT versus ADT alone; 0.897 and 0.824 for MAB versus ADT alone. Doc-ADT was cost-effective compared with MAB and ADT alone, with an incremental cost–effectiveness ratio of CNY 96,848 and CNY 67,758 per quality-adjusted life year, respectively. MAB was cost-effective compared with ADT alone, with an incremental cost–effectiveness ratio of CNY 137,487 per quality-adjusted life year. Conclusion: Doc-ADT is likely the optimal option from the perspective of both clinical outcomes and economic considerations.


2017 ◽  
Vol 42 (3) ◽  
pp. 318-327 ◽  
Author(s):  
Laurent Alain Frossard ◽  
Gregory Merlo ◽  
Brendan Burkett ◽  
Tanya Quincey ◽  
Debra Berg

Background: In principle, lower limb bone-anchored prostheses could alleviate expenditure associated with typical socket manufacturing and residuum treatments due to socket-suspended prostheses. Objective: This study reports (a) the incremental costs and (b) heath gain as well as (c) cost-effectiveness of bone-anchored prostheses compared to socket-suspended prostheses. Study design: Retrospective individual case-controlled observations and systematic review. Methods: Actual costs were extracted from financial records and completed by typical costs when needed over 6-year time horizon for a cohort of 16 individuals. Health gains corresponding to quality-adjusted life-year were calculated using health-related quality-of-life data presented in the literature. Results: The provision of bone-anchored prostheses costed 21% ± 41% more but increased quality-adjusted life-years by 17% ± 5% compared to socket-suspended prostheses. The incremental cost-effectiveness ratio ranged between –$25,700 per quality-adjusted life-year and $53,500 per quality-adjusted life-year with indicative incremental cost-effectiveness ratio of approximately $17,000 per quality-adjusted life-year. Bone-anchored prosthesis was cost-saving and cost-effective for 19% and 88% of the participants, respectively. Conclusion: This study indicated that bone-anchored prostheses might be an acceptable alternative to socket-suspended prostheses at least from a prosthetic care perspective in Australian context. Altogether, this initial evidence-based economic evaluation provided a working approach for decision makers responsible for policies around care of individuals with lower limb amputation worldwide. Clinical relevance For the first time, this study provided evidence-based health economic benefits of lower limb bone-anchored prostheses compared to typical socket-suspended prostheses from a prosthetic care perspective that is essential to clinicians and decision makers responsible for policies.


2020 ◽  
Vol 24 (30) ◽  
pp. 1-116
Author(s):  
Juliet Hounsome ◽  
Gerlinde Pilkington ◽  
James Mahon ◽  
Angela Boland ◽  
Sophie Beale ◽  
...  

Background Impacted third molars are third molars that are blocked, by soft tissue or bone, from fully erupting through the gum. This can cause pain and disease. The treatment options for people with impacted third molars are removal or retention with standard care. If there are pathological changes, the current National Institute for Health and Care Excellence guidance states that the impacted third molar should be removed. Objective The objective of this study was to appraise the clinical effectiveness and cost-effectiveness of the prophylactic removal of impacted mandibular third molars compared with retention of, and standard care for, impacted third molars. Methods Five electronic databases were searched (1999 to 29 April 2016) to identify relevant evidence [The Cochrane Library (searched 4 April 2016 and 29 April 2016), MEDLINE (searched 4 April 2016 and 29 April 2016), EMBASE (searched 4 April 2016 and 29 April 2016), EconLit (searched 4 April 2016 and 29 April 2016) and NHS Economic Evaluation Database (searched 4 April 2016)]. Studies that compared the prophylactic removal of impacted mandibular third molars with retention and standard care or studies that assessed the outcomes from either approach were included. The clinical outcomes considered were pathology associated with retention, post-operative complications following extraction and adverse effects of treatment. Cost-effectiveness outcomes included UK costs and health-related quality-of-life measures. In addition, the assessment group constructed a de novo economic model to compare the cost-effectiveness of a prophylactic removal strategy with that of retention and standard care. Results The clinical review identified four cohort studies and nine systematic reviews. In the two studies that reported on surgical complications, no serious complications were reported. Pathological changes due to retention of asymptomatic impacted mandibular third molars were reported by three studies. In these studies, the extraction rate for retained impacted mandibular third molars varied from 5.5% to 31.4%; this variation can be explained by the differing follow-up periods (i.e. 1 and 5 years). The findings from this review are consistent with the findings from previous systematic reviews. Two published cost-effectiveness studies were identified. The authors of both studies concluded that, to their knowledge, there is currently no economic evidence to support the prophylactic removal of impacted mandibular third molars. The results generated by the assessment group’s lifetime economic model indicated that the incremental cost-effectiveness ratio per quality-adjusted life-year gained for the comparison of a prophylactic removal strategy with a retention and standard care strategy is £11,741 for people aged 20 years with asymptomatic impacted mandibular third molars. The incremental cost per person associated with prophylactic extraction is £55.71, with an incremental quality-adjusted life-year gain of 0.005 per person. The base-case incremental cost-effectiveness ratio per quality-adjusted life-year gained was found to be robust when a range of sensitivity and scenario analyses were carried out. Limitations Limitations of the study included that no head-to-head trials comparing the effectiveness of prophylactic removal of impacted mandibular third molars with retention and standard care were identified with the assessment group model that was built on observational data. Utility data on impacted mandibular third molars and their symptoms are lacking. Conclusions The evidence comparing the prophylactic removal of impacted mandibular third molars with retention and standard care is very limited. However, the results from an exploratory assessment group model, which uses available evidence on symptom development and extraction rates of retained impacted mandibular third molars, suggest that prophylactic removal may be the more cost-effective strategy. Future work Effectiveness evidence is lacking. Head-to-head trials comparing the prophylactic removal of trouble-free impacted mandibular third molars with retention and watchful waiting are required. If this is not possible, routine clinical data, using common definitions and outcome reporting methods, should be collected. Study registration This study is registered as PROSPERO CRD42016037776. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 30. See the NIHR Journals Library website for further project information.


Author(s):  
Jonathan Siverskog ◽  
Martin Henriksson

Abstract In the past few years, empirical estimates of the marginal cost at which health care produces a quality-adjusted life year (QALY, k) have begun to emerge. In theory, these estimates could be used as cost-effectiveness thresholds by health-maximizing decision makers, but prioritization decisions in practice often include other considerations than just efficiency. Pharmaceutical reimbursement in Sweden is one such example, where the reimbursement authority (TLV) uses a threshold range to give priority to disease severity and rarity. In this paper, we argue that estimates of k should not be used to inform threshold ranges. Instead, they are better used directly in health technology assessment (HTA) to quantify how much health is forgone when a new technology is funded in place of other healthcare services. Using a recent decision made by TLV as a case, we show that an estimate of k for Sweden implies that reimbursement meant forgoing 8.6 QALYs for every QALY that was gained. Reporting cost-effectiveness evidence as QALYs forgone per QALY gained has several advantages: (i) it frames the decision as assigning an equity weight to QALYs gained, which is more transparent about the trade-off between equity and efficiency than determining a monetary cost per QALY threshold, (ii) it makes it less likely that decision makers neglect taking the opportunity cost of reimbursement into account by making it explicit, and (iii) it helps communicate the reason for sometimes denying reimbursement in a way that might be less objectionable to the public than current practice.


2021 ◽  
Vol 9 ◽  
Author(s):  
Mengran Zhang ◽  
Yu Ren ◽  
Luying Wang ◽  
Jianhao Jia ◽  
Lei Tian

Background: Atrial fibrillation (AF) is one of the most common arrhythmias in clinical practice, which brings great economic burden to patients. This study evaluated the economics of the new antiarrhythmic drug dronedarone and provides suggestions for allocation of health resources.Methods: Amiodarone was selected as the control group, and the Markov model of AF was established using nine states. The total cost and quality-adjusted life year (QALY) of dronedarone and amiodarone groups were calculated and compared. The incremental cost effectiveness ratio (ICER) value was calculated and compared with the willingness to pay (WTP) and the sensitivity analyses was conducted.Results: For China's healthcare system, the ICER of the dronedarone group compared with the amiodarone group was RMB 81,741 Yuan/QALY, which is lower than the current recommended WTP (3 times GDP per capita). Sensitivity analyses showed that the model was robust, and the drug price of dronedarone significantly impacted the results.Conclusions: Compared with amiodarone, dronedarone is more economical in the Chinese healthcare system. However, due to the lack of data on the Chinese population for some parameters, the model needs further improvement and discussion. Real-world studies on the effects of dronedarone on Chinese patients with AF would be beneficial.


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