Delivering the precision oncology paradigm: reduced R&D costs and greater return on investment through a companion diagnostic informed precision oncology medicines approach.

BACKGROUND: Precision oncology medicines represent a paradigm shift compared to non-precision oncology medicines in cancer therapy, in some situations delivering more clinical benefit, and potentially lowering healthcare costs. We determined whether employing a companion diagnostic (CDx) approach during oncology medicines development delivers effective therapies that are within the cost constraints of current health systems. R&D costs of developing a medicine are subject to debate, with average estimates ranging from $765 million (m) to $4.6 billion (b). Our aim was to determine whether precision oncology medicines are cheaper to bring from R&D to market; a secondary goal was to determine whether precision oncology medicines have a greater return on investment (ROI). METHOD: Data on oncology medicines approved between 1997 and 2020 by the US Food and Drug Administration (FDA) were analysed from the Securities and Exchange Commission (SEC) filings. Data were compiled from 10-K, 10-Q, and 20-F financial performance filings on medicines' development costs through their R&D lifetime. Clinical trial data were split into clinical trial phases 1-3 and probability of success (POS) of trials was calculated, along with preclinical costs. Cost-of-capital (CoC) approach was applied and, if appropriate, a tax rebate was subtracted from the total. RESULTS: Data on 42 precision and 29 non-precision oncology medicines from 56 companies listed by the National Cancer Institute which had complete data available were analysed. Estimated mean cost to deliver a new oncology medicine was $4.4b (95% CI, $3.6-5.2b). Costs to bring a precision oncology medicine to market were $1.1b less ($3.5b; 95% CI, $2.7-4.5b) compared to non-precision oncology medicines ($4.6b; 95% CI, $3.5-6.1b). The key driver of costs was POS of clinical trials, accounting for a difference of $591.3 m. Additional data analysis illustrated that there was a 27% increase in return on investment (ROI) of precision oncology medicines over non-precision oncology medicines. CONCLUSION: Our results provide an accurate estimate of the R&D spend required to bring an oncology medicine to market. Deployment of a CDx at the earliest stage substantially lowers the cost associated with oncology medicines development, potentially making them available to more patients, while staying within the cost constraints of cancer health systems.

Study of patients with iron deficiency and HF in Ireland: prevalence and treatment budget impact.

This study aims to present the screening, prevalence and treatment of heart failure (HF) patients with iron deficiency in an Irish hospital and use an economic model to estimate the budget impact of treating eligible patients with intravenous ferric carboxymaltose (IV FCM). Retrospective data were collected on 151 HF patients over a one-year period from all newly referred HF patients to a secondary care hospital. This included 36 patients with preserved ejection fraction (HFpEF) and 115 with reduced ejection fraction (HPrEF). An existing budget impact model was adapted to incorporate Irish unit cost and resource use data to estimate the annual budget impact of treating patients with IV FCM. The total number of HFrEF patients who met criteria for iron replacement was 44 (38% of total HFrEF patients); of this, only nine (20%) were treated. The budget impact model estimates that treating all eligible patients with IV FCM in this single centre would save 40 bed-days and over €7,600/year. To improve the quality of life and reduce hospitalisation, further identification and treatment of iron deficient patients should be implemented. Expanding the use of IV iron nationally would be cost and bed saving.

A, B, or C? A Quasi-experimental Multi-site Study Investigating Three Option Multiple Choice Questions.

There is nearly a century of educational research that has demonstrated that three option multiple-choice questions (MCQs) are as valid and reliable as four or five option, yet this format continues to be underutilized in educational institutions. This replication study was a quasi-experimental between groups research design conducted at three Canadian schools of nursing to examine the psychometric properties of three option MCQs when compared to the more traditional four option questions. Data analysis revealed that there were no statistically significant differences in the item discrimination, difficulty or mean examination scores when MCQs were administered with three versus four option answer choices.

To HTA or Not to HTA: Identifying the Factors Influencing the Rapid Review Outcome in Ireland.

OBJECTIVES: Reimbursement systems are evolving and endeavor to balance access and affordability. One such evolution in Ireland is the compulsory rapid review (RR) process, the outcome from which is a recommendation for a health technology assessment (HTA) or no HTA. For drugs that avoid an HTA, evaluation times are shorter, lengthy price negotiations are avoided, and access is faster. In the absence of formal decision-making criteria around the requirement of an HTA, this study examines the factors influencing the outcome of the RR process in Ireland. METHODS: A database was developed combining data from publicly available sources for drug evaluations conducted by the National Centre for PharmacoEconomics (NCPE) (January 2010-June 2017, n = 296). Because Irish cost data were not publicly available for all drugs, cost data from the Scottish Medicines Consortium were employed as a proxy. Employing logistic regressions, the factors influencing the RR outcome are revealed. RESULTS: After an RR, an HTA was recommended for 55% of drugs. The regression results revealed therapeutic area (endocrine, musculoskeletal, and neoplasm), first-in-class and orphan disease increased the probability of an HTA. Furthermore, when proxy costs were included, results revealed that every €1000 increase in annual drug costs per patient increased the probability of an HTA being required by 1% and that an HTA was more likely than no HTA when annual drug costs exceeded €15 000. CONCLUSION: Given the current focus on access and affordability, this study identifies the factors influencing the requirement of an HTA in Ireland.

High time for a change: psychometric analysis of multiple-choice questions in nursing.

Nurse educators teach students to develop an informed nursing practice but can educators claim the same grounding in the available evidence when formulating multiple-choice assessment tools to evaluate student learning? Multiple-choice questions are a popular assessment format within nursing education. While widely accepted as a credible format to assess student knowledge across disciplines, debate exists among educators regarding the number of options necessary to adequately test cognitive reasoning and optimal discrimination between student abilities. The purpose of this quasi-experimental between groups study was to examine the psychometric properties of three option multiple-choice questions when compared to the more traditional four option questions. Data analysis revealed that there were no statistically significant differences in the item discrimination, difficulty or the mean examination scores when multiple-choice test questions were administered with three versus four option answer choices. This study provides additional guidance for nurse educators to assist in improving multiple-choice question writing and test design.