Challenges of Incorporating Life Cycle Drug Pricing in Cost-Effectiveness Models: A Review of Methods and Modeling Suggestions.

Objectives: This study aimed to conduct a review of existing methods used to incorporate life cycle drug pricing (LCDP) in cost-effectiveness analyses (CEAs), identify common methodological challenges, and suggest modeling approaches for prospectively implementing LCDP in CEA.

Methods: Two complementary searches were conducted in PubMed, combined with hand searching and reference mining, to identify English language full-text articles that explored (1) how drug prices change over time and (2) methods used to apply dynamic pricing in cost-effectiveness models (CEMs). Relevant articles were reviewed, and authors discussed the common methodological practices used in the literature and their associated challenges on prospectively implementing LCDP in CEMs.

Surrogacy and the Valuation of ATMPs: Taking Our Place in the Evidence Generation/Assessment Continuum.

Medical technology is advancing rapidly, but established methods for health technology assessment are struggling to keep up. This challenge is particularly stark for the assessment of advanced therapy medicinal products-therapies often launched on the basis of single-arm studies powered to a surrogate primary endpoint. The most robust surrogacy methods investigate trial-level correlations between the treatment effect on the surrogate and the outcome of ultimate interest.

R and Shiny for Cost-Effectiveness Analyses: Why and When? A Hypothetical Case Study.

Introduction: Health economics models are typically built in Microsoft Excel owing to its wide familiarity, accessibility and perceived transparency. However, given the increasingly rapid and analytically complex decision-making needs of both the pharmaceutical industry and the field of health economics and outcomes research (HEOR), the demands of cost-effectiveness analyses may be better met by the programming language R.

Objective: This case study provides an explicit comparison between Excel and R for contemporary cost-effectiveness analysis.

"Stick or Twist?" Negotiating Price and Data in an Era of Conditional Approval.

Background: Changes in the regulatory context enable faster approval of transformative medicines. They also lead to health technology assessment (HTA) agencies having to make decisions with less evidence. In response, HTA agencies have also initiated forms of conditional approval.

Cost effectiveness of renal denervation therapy for the treatment of resistant hypertension in the UK.

Background: Patients with resistant hypertension are at a high risk for developing serious cardiovascular events and renal complications. Catheter-based renal denervation (RDN) is a procedure with the potential to normalize systolic blood pressure (SBP).

Objective: The overall objective of the study was to estimate the cost effectiveness of RDN in the UK for patients with diagnosed resistant hypertension, expressed as a standard cost per quality-adjusted life-year (QALY) ratio.

A comparison of the cost-effectiveness of treatment of prolonged acute convulsive epileptic seizures in children across Europe.

In the majority of children and adolescents with epilepsy, optimal drug therapy adequately controls their condition. However, among the remaining patients who are still uncontrolled despite mono-, bi- or tri-therapy with chronic anti-epileptic treatment, a rescue medication is required. In Western Europe, the licensed medications available for first-line treatment of prolonged acute convulsive seizures (PACS) vary widely, and so comparators for clinical and economic evaluation are not consistent.

A cost-utility analysis of degarelix in the treatment of advanced hormone-dependent prostate cancer in the United Kingdom.

Objective: To determine the cost-effectiveness of the treatment of advanced hormone-dependent prostate cancer with degarelix compared to luteinizing hormone-releasing hormone (LHRH) agonists in the UK using the latest available evidence and the model submitted to AWMSG.

Methods: A cost-effectiveness model was developed from the perspective of the UK National Health Service evaluating monthly injection of degarelix against 3-monthly leuprorelin therapy plus anti-androgen flare cover for the first-line treatment of patients with advanced (locally advanced or metastatic) hormone-dependent prostate cancer. A Markov process model was constructed using the patient population characteristics and efficacy information from the CS21 Phase III clinical trial and associated extension study (CS21A).

Estimating the critical and sensitive periods of investment in early childhood: a methodological note.

The identification of critical periods in early human development requires statistical analyses beyond simple cross tabulation of correlations of observed variables. This paper provides an overview of different quantitative methods available for the statistical analysis of longitudinal data regarding child development, and in particular the identification of critical and sensitive periods for later abilities. It draws heavily on the work on human skill formation developed by the economist James Heckman, which treats ability as a latent variable and explains its formation through the simultaneous estimation of structural equations of investments and achieved abilities across time.

The cost effectiveness of licensed oromucosal midazolam (Buccolam(®)) for the treatment of children experiencing acute epileptic seizures: an approach when trial evidence is limited.

Background: In the UK, two treatment options are used for acute epileptic seizures in the community-rectal diazepam and unlicensed buccal midazolam. In practice, the former is rarely used, with unlicensed buccal midazolam being widely recommended and prescribed by physicians. In September 2011, Buccolam(®) (licensed midazolam oromucosal solution) became the first medicine to receive a Paediatric-Use Marketing Authorization (PUMA) and it is indicated for the treatment of prolonged, acute, convulsive seizures by caregivers in the community for children (aged 6 months to <18 years) diagnosed with epilepsy.