Expert review of pharmacoeconomics and outcomes research: high impact articles from 2023

KEYWORDS:

• Outcomes research
• editorial board
• top articles
• pharmacoeconomics
• 2023

1. Introduction

Expert Review of Pharmacoeconomics & Outcomes Research (ERP) is a prestigious journal within the field of health economics and outcomes research, distinguished for its commitment to publishing high-quality, pertinent, and diverse content. The journal’s burgeoning impact within its field and output owes much to the contributions of our esteemed authors, who consistently submit relevant and exemplary research to ERP, as well as our dedicated editorial board, who provide invaluable guidance, and our peer reviewers, who offer exceptional support in enhancing the quality, rigor, and presentation of submitted papers.

As we embark on a new year, we wish to highlight selected papers from volume 23, published in 2023. To this end, editorial board members were invited to nominate their top article of the year and furnish a brief commentary elucidating its significance. No additional criteria were stipulated for the article selection process. In this foreword, we are pleased to present a compilation of five papers, each chosen by an editorial board member and by Editor-in-Chief Mickael Hiligsmann.

2. Editor-in-Chief, Mickaël Hiligsmann’s selection

Recommendations for Economic Evaluations of Cell and Gene Therapies: A Systematic Literature Review with Critical Appraisal [1].

My pick is a paper authored by Toumi M et al., entitled ‘Recommendations for Economic Evaluations of Cell and Gene Therapies: A Systematic Literature Review with Critical Appraisal.’ It comes as no surprise that this paper emerged as one of the most widely read articles in ERP of 2023. With the advent of gene therapies entering the market and an anticipation of many more in the years to come, the development and evaluation of these innovative treatments pose multifaceted challenges in clinical, economic, and reimbursement realms. Economic evaluations play a pivotal role in balancing the considerable costs associated with gene therapies against their potential for substantial clinical benefits. Toumi et al.‘s paper makes a significant contribution to the literature by addressing the challenges and offering recommendations for conducting economic evaluations of gene therapies. Drawing upon the expertise of professionals from health technology assessment agencies, academia, and industry, the authors initially delineated 21 key recommendations for economic evaluations in the context of gene therapies. Subsequently, these recommendations were scrutinized against published economic evaluations of gene therapies. The findings revealed a notable discrepancy, indicating that most researchers failed to adhere to the established standards. Consequently, there arises an imperative for future economic evaluations of gene therapies to align more closely with these standards, and the insights provided by Toumi et al. are instrumental in this regard. Nevertheless, it is essential to recognize that designing economic evaluations for gene therapies is inherently complex, necessitating further exploration and guidelines in the near future. Challenges such as validating surrogate endpoints and the prevalence of small, often single-arm clinical studies, underscore the intricate and challenging nature of these studies. In conclusion, I firmly believe that this paper furnishes a basic framework to facilitate enhanced and more rigorous economic evaluations of gene therapies in the future. It sets the stage for continued advancements in our understanding and approach toward assessing the economic value of these transformative treatments.

3. Senior editor Mitchell P DeKoven’s selection

Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program [2].

Pursuant to the landmark Inflation Reduction Act (2022), the Centers for Medicare & Medicaid Services released guidance (2023), emphasizing an approach to assessing the value of selected drugs compared to their therapeutic alternatives, as a means to guide drug price negotiation (DPN) with primary manufacturers. This narrative review focused on the challenge of measuring value for the DPN program by identifying and describing six potential approaches to assessing value: 1) equal value life-years (evLY); 2) health years in total (HYT); 3) generalized risk-adjusted cost-effectiveness (GRACE); 4) severity weighting based on absolute or proportional shortfall; 5) comparative effectiveness based on conventional clinical endpoints; and 6) comparative effectiveness based on both conventional endpoints and patient-centric value elements. Given strong arguments against the use of quality adjusted life years (QALYs), these approaches were assessed using five criteria (indicative of cost-effectiveness and comparative effectiveness) including potential to lead to discrimination, feasibility, transparency, flexibility, and ability to incorporate factors beyond traditional value elements. Four of the assessed approaches (e.g. evLY, HYT, GRACE, severity weighting) were found to leverage a methodology based upon QALYs. The study identified a recent multi-criteria decision framework-based approach combining quantitative ranking/weighting of value elements to mitigate biases in deliberations and incorporating patient-centric value elements.

4. Associate editor Carolyn Gotay’s selections

The economic value of knowing BRCA status: universal BRCA testing for breast cancer prevention [3].

The economic value of knowing BRCA status: BRCA testing for optimizing treatment in recurrent epithelial ovarian cancer [4].

Economic value of knowing BRCA status: BRCA testing for prostate cancer prevention and optimal treatment [5].

Precision oncology holds tremendous potential to provide better outcomes based on prevention, screening, and treatment options that are matched to patient characteristics. Genetic mutations are one key patient characteristic, with BRCA1/2 being among the most widely studied genes. While interventions based on BRCA status are available, these therapies are often not optimally applied, with concerns about costs paramount. A series of 2023 papers in Expert Review of Pharmocoeconomics & Outcomes Research from Oh, et al. examined the impact of BRCA information in the three cancer settings: breast cancer prevention, treatment, and screening in low risk localized prostate cancer, and treatment for patients with recurrent epithelial ovarian cancer. All three studies used data from clinical trials cohorts, registries, and the literature to model the effects of knowing BRCA mutation status on a range of short- and long-term outcomes including (as per study) cancer incidence, treatment selection, morbidity, and mortality. Costs, both from the payer and societal perspective, were assessed. The results across all studies were clear: knowledge about the BRCA mutation status was cost-saving for payers and society. The authors suggest that as testing costs decrease, the benefits of broader genetic testing are likely to become even greater. Further, the impact of detecting a BRCA mutation in one individual may lead to screening in other family members, with potential gains that are, as the authors say, ‘incalculable.’ These thoughtful papers contribute to this important field.

5. Editorial board member, Carlo Lazzaro’s selection

Review of economic modeling evidence from NICE appraisals of rare disease treatments for spinal muscular atrophy [6].

My pick for 2023 is Review of economic modeling evidence from NICE appraisals of rare disease treatments for spinal muscular atrophy [6]. Two topics captured my attention while reading this article. First, NICE UK£50,000 willingness to pay (WTP) for incremental quality-adjusted life year (QALY) gained for this disease is higher than NICE usual UK£20,000-UK£30,000 acceptability range [7]. Further research could contribute to setting local WTPs for medications targeting rare diseases to be cost-effective and investigate, especially for national health services, the opportunity cost of higher than standard WTPs [8]. Second, caregiver’s (dis)utility. Adopting the societal perspective, the numerator of the incremental cost-utility ratio (ICUR) changes (as cost of patient and their family resources, loss or working/leisure time, and informal care add on to cost of health care sector resources) [9], whereas the incremental QALYs gained totaled by patient equal those calculated following narrower viewpoints (e.g. the national health service one). Given the personal burden that rare diseases put on caregivers, their utility unavoidably decreases [1]. Therefore, everything else being equal, including caregivers’ disutility reduces the incremental QALYs gained, and increases the ICUR. This article provides relevant guidance to decision makers dealing with the reimbursement of drugs targeting rare diseases.

6. Editorial board member, Jayashri Sankaranarayanan’s selection

Differences in evidentiary requirements for oncology drug effectiveness assessments among six European health technology assessment bodies – can alignment be improved? [10].

Achieving enhanced alignment of varying evidentiary requirements among Health Technology Assessment (HTA) bodies can reduce time to coverage decision-making and improve equity in patient access. The ‘degrees of evidence acceptability per HTA body and alignment on evidentiary requirement subdomains among HTA bodies’ for oncology drug assessments may affect reimbursement decisions from full public, limited, only private, or no coverage. A recent 2024 pivotal study examined stakeholder interviews from six European HTA bodies (Italy, the Netherlands, Poland, Portugal, England and Wales, and Sweden) about differences in evidentiary requirements for oncology drugs’ relative effectiveness assessments. The HTA bodies were least aligned about the acceptability of extrapolation to other (than target) populations, class effects, progression-free survival, and (other) surrogate endpoints as outcomes, the absence of quality-of-life data, trial designs (single-arm, cross-over, short duration), and the clinical relevance of effect size. Therefore, to improve alignment, the authors proposed recommendations of joint early dialogs, intensified collaboration/exchange between countries, centralized relative effectiveness assessment processes, and the use of Access agreements. This relevant guidance to continuously align and standardize evidentiary requirements of oncology drug assessments among European HTA bodies can be applied to other novel life-saving drug assessments, as well as to decision-makers in more countries worldwide.

We hope you enjoyed reading this collection of articles and found them as interesting as we did.

We would also like to highlight that we are welcoming submissions to our upcoming special issue ‘Health economics of diagnostic tests for tumors and rare diseases,’ which aims to provide insight into the evolving paradigms in cost and reimbursement of lab tests.

We encourage our authors and audience to contact us with submission inquiries, ideas for articles and special issues, and queries about supporting the journal as an Editorial Board member or peer reviewer.

Declaration of interest

M Hiligsmann and M P DeKoven are the Editors in Chief of Expert Review of Pharmacoeconomics and Outcomes Research. C Gotay is Associate Editor of Pharmacoeconomics and Outcomes Research. C Lazzaro and J Sankaranarayanan are Editorial Board Members of Expert Review of Pharmacoeconomics and Outcomes Research. R Hajjar is the Commissioning Editor of Expert Review of Pharmacoeconomics and Outcomes Research, and an employee of Taylor & Francis.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Additional information

Funding

This foreword was not funded.