Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data.

BACKGROUND: Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII product that enables less frequent administration than rFVIII, which may support improved adherence. Available data indicate low breakthrough bleed rates and potentially improved long-term joint health for patients treated with rFVIIIFc prophylaxis. OBJECTIVE: This study assessed the cost effectiveness of rFVIIIFc versus rFVIII from an Italian healthcare perspective. METHODS: A Semi-Markov model was constructed to assess the lifetime costs and benefits of rFVIII and rFVIIIFc prophylaxis. rFVIII product acquisition costs from a published Italian database were included for both prophylaxis and the resolution of breakthrough bleeding. Clinical outcomes within the model were determined based on published annualized bleeding rates and literature regarding the development of target joints (TJs) as the incidence of bleeds and TJs is associated with impaired health-related quality of life. Cost effectiveness was assessed using cost per quality-adjusted life-year (QALY) gained. RESULTS: Compared with rFVIII, rFVIIIFc was associated with a per-patient cost saving of approximately €1.3 million and QALY gains of 0.39 over a lifetime horizon. Sensitivity analyses considering alternative efficacy, dosing, and structural assumptions each showed that rFVIIIFc dominated rFVIII (i.e., provided more QALYs at a reduced cost). CONCLUSIONS: This cost-effectiveness analysis demonstrated that rFVIIIFc may offer a cost-effective treatment option for patients with SHA in Italy.

NICE, in Confidence: An Assessment of Redaction to Obscure Confidential Information in Single Technology Appraisals by the National Institute for Health and Care Excellence.

INTRODUCTION: Health technology assessment (HTA) aims to provide a transparent framework within which normative judgements can be applied for decision making. Such transparency enables the public to understand the rationale for decision making, but conflicts with companies being able to offer commercially sensitive discounts. We investigated how to balance these conflicting ideals. METHODS: National Institute for Health and Care Excellence (NICE) submissions were reviewed for products with an approved, simple Patient Access Scheme (PAS) discount. The approach to censoring was noted (e.g. total cost and clinical outcomes redacted). Submissions were then assessed for transparency (i.e. whether the decision appeared justifiable given the available information) and confidentiality (i.e. whether the PAS discount could be 'back calculated'). RESULTS: One hundred and eighteen products have an approved commercial arrangement, of which 110 have simple PAS discounts considered within the NICE Single Technology Appraisal programme. A definitive incremental cost-effectiveness ratio was presented within final NICE guidance in only 20 appraisals. Documentation for seven appraisals allowed for the straightforward 'back calculation' of PAS discounts. Furthermore, a large amount of information was censored as academic-in-confidence and remains so many years later. CONCLUSION: Appropriate redaction ensures discounts remain confidential, yet maintains the transparency of the HTA decisions made. Complete redaction does not allow for transparent, justifiable decision making. However, redacting 'enough' information to preclude direct estimation of discounts provides a means of maintaining both transparency and confidentiality. This study demonstrates a lack of consensus regarding presentation of results, and the importance of appropriate redaction.

Cost-effectiveness of Trifluridine/tipiracil for Previously Treated Metastatic Colorectal Cancer in England and Wales.

BACKGROUND: Treatment options at third-line and beyond for patients with late-line metastatic colorectal cancer (mCRC) are limited, and outcomes are poor with best supportive care (BSC). This study investigated the cost-effectiveness of trifluridine/tipiracil and regorafenib relative to BSC alone in patients with mCRC who have been previously treated with, or are not considered candidates for, standard chemotherapies. MATERIALS AND METHODS: A partitioned survival model was constructed to assess the lifetime costs and benefits accrued by patients. Clinical data were derived from the pivotal phase III (Randomized, Double-Blind, Phase 3 Study of TAS-102 plus Best Supportive Care [BSC] versus Placebo plus BSC in Patients with Metastatic Colorectal Cancer Refractory to Standard Chemotherapies [RECOURSE]) and supporting phase II (J003-10040030) randomized controlled trial of trifluridine/tipiracil + BSC versus placebo + BSC, as well as the phase III Colorectal Cancer Treated With Regorafenib or Placebo After Failure of Standard Therapy (CORRECT) randomized controlled trial of regorafenib, and were extrapolated to estimate lifetime outcomes. Costs were taken from published sources, and health effects sourced from previous mCRC studies. RESULTS: Trifluridine/tipiracil was associated with a 0.27 incremental life year versus BSC alone, which corresponds to a 0.17 quality-adjusted life year gain. The incremental cost of treatment with trifluridine/tipiracil was £8,479, resulting in an incremental cost-effectiveness ratio of £51,194 per quality-adjusted life year gained. Trifluridine/tipiracil was shown to dominate regorafenib (improve outcomes with reduced costs). Sensitivity analyses showed principal areas of uncertainty were survival estimates and patient utility. CONCLUSIONS: The results show that trifluridine/tipiracil is more clinically and cost-effective than regorafenib, with clinical outcomes greatly exceeding those for patients treated with BSC alone. Based on the results of the analysis, trifluridine/tipiracil offers an important new treatment option for patients with mCRC maintaining good performance status at the end of life.