The Effects of Model Misspecification in Unanchored Matching-Adjusted Indirect Comparison: Results of a Simulation Study.

Objectives: To assess the performance of unanchored matching-adjusted indirect comparison (MAIC) by matching on first moments or higher moments in a cross-study comparisons under a variety of conditions. A secondary objective was to gauge the performance of the method relative to propensity score weighting (PSW).

Methods: A simulation study was designed based on an oncology example, where MAIC was used to account for differences between a contemporary trial in which patients had more favorable characteristics and a historical control.

Evaluation of survival extrapolation in immuno-oncology using multiple pre-planned data cuts: learnings to aid in model selection.

Background: Due to limited duration of follow up in clinical trials of cancer treatments, estimates of lifetime survival benefits are typically derived using statistical extrapolation methods. To justify the method used, a range of approaches have been proposed including statistical goodness-of-fit tests and comparing estimates against a previous data cut (i.e.

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.

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.

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.

How do we avoid disaster when exiting the European Medicines Agency? Making the most of Brexit in pharmaceutical regulation.

As the UK prepares to leave the EU, it must decide what path it is to take with a large number of regulatory and technical agencies who provide collaboration at the European level. In the case of pharmaceuticals, the European Medicines Agency (EMA) provides pan-European licencing for novel pharmaceuticals. Should the UK depart from the EMA system, this article highlights the loss to patients immediately (slower access to novel treatments), and in the long term by having no access to some novel products as companies choose not to launch in the UK.

Measuring quality of life in opioid-induced constipation: mapping EQ-5D-3 L and PAC-QOL.

Background: In health economic evaluations, quality of life should be measured with preference-based utilities, such as the EuroQol 5 Dimension 3-level (EQ-5D-3 L). Non-preference-based instruments (often disease-specific questionnaires) are commonly mapped to utilities. We investigated if the relationship observed between the Patient Assessment of Constipation Quality of Life (PAC-QOL) and the EQ-5D-3 L in patients with chronic idiopathic constipation (CIC) also applies in opioid-induced constipation (OIC).