Extent and causes of the collapse in the registration of innovative medications in Lebanon: A mixed-methods analysis

PLoS ONE · 2025-12-26

OBJECTIVES: Delays in innovative drug registration across countries, or the "drug approval lag", can cause inequities in treatment access, thereby worsening patient outcomes. Registration delays hinder the first step in making treatments available, and are often linked to regulatory inefficiencies, constrained healthcare financing, and fragmented decision-making. Since late 2019, Lebanon's health system has faced overlapping socioeconomic and political crises, yet their impact on innovative medication registration remains undocumented. This study aimed to measure this impact by comparing Lebanon's drug approval lag before (2014-2019) and after (2020-2024) the crisis and to develop an interpretive framework exploring the rationale behind an informal policy to delay innovative medication registration. METHODS: A mixed-methods approach was adopted. Innovative medications approved by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA) between 2014 and 2024 were included and compared to local registration timelines in Lebanon. In-depth interviews with policymakers, industry leaders, and healthcare providers informed the interpretive framework. RESULTS: Findings revealed a dramatic fall in innovative medication registrations post-crisis over these two periods. The proportion of FDA-approved innovative medications registered in Lebanon dropped from 43.6% to 0% and EMA-approved medications dropped from 59.4% to 0%. Pre-crisis, average registration time was under two years; post-crisis, delays are estimated to exceed four years. Our interpretive framework suggests the intermediate effects of delaying innovative medication registration are mainly to control costs and reduce reimbursement pressures on the Ministry of Public Health. However, key stakeholders believe the resulting negative consequences, such as reduced access to life-saving treatments and greater dependence on parallel market importation, outweigh the short-term benefits. CONCLUSION: Health systems are complex adaptive systems, where policies affecting innovative drug approvals may not only delay access, but also trigger unintended consequences. In Lebanon, the registration of innovative medications should remain independent from reimbursement decisions and grounded in evidence-informed health policies.

Economic evaluation of work productivity gains associated with ribociclib (RIB) in hormone receptor–positive/human epidermal growth factor receptor 2–negative (HR+/HER2−) early breast cancer (EBC) in the United States.

Journal of Clinical Oncology · 2025-05-28

e23117 Background: Despite treatment with standard-of-care adjuvant endocrine therapy, many patients with HR+/HER2− EBC continue to experience recurrences, most of which are distant. Recurrences can lead to extensive costs, including indirect costs associated with the loss of income due to reduction or complete loss in the ability to work. The cyclin-dependent kinase 4/6 inhibitor RIB + an aromatase inhibitor (AI) was FDA-approved in Sept 2024 to reduce risk of recurrence among patients with HR+/HER2− disease (stage II and III at high risk of recurrence). At a 44.2 mo median follow-up of NATALEE, with all patients off RIB, RIB + a nonsteroidal AI (NSAI) vs NSAI significantly improved invasive disease-free survival (iDFS; HR, 0.715 [0.609-0.840]) with a 4y landmark Δ of 4.9%. This analysis assessed long-term work productivity gains of treating patients with HR+/HER2− EBC with RIB+NSAI vs NSAI from a US societal perspective. Methods: A Markov model was developed to estimate the distribution of being in 4 health states (invasive disease-free [IDF], locoregional recurrence [LR], distant recurrence [DR], and death) for patients with HR+/HER2− EBC treated with RIB+NSAI vs NSAI over a lifetime horizon. Efficacy data were modeled using patient-level iDFS data from the NATALEE trial up to 4.5y (median follow up: 44.2 mo) using Kaplan Meier (KM) method and parametric extrapolation of the KM curves afterward. Other clinical data were obtained from literature. Data on age- and gender-adjusted employment rates, wages, and number of workdays lost in each health state were obtained from published literature and applied to the distribution of patients across each health state. Work productivity-related indirect cost across health states was estimated until the cohort reached 65y, retirement age assumed for the base-case. All cost inputs were in 2024 USD. Work productivity was estimated at individual pt and US population levels. Scenario analyses to test alternative assumptions were conducted. Results: Patients receiving RIB+NSAI on average spent more time in IDF and less time in LR and DR states vs those receiving NSAI alone. A pt receiving RIB+NSAI was estimated to have more lifetime earnings ($494,317) vs those receiving NSAI ($482,581), with the work-related productivity gain from the addition of RIB estimated at $11,736 per patient. At the US population level, the total lifetime work productivity gains for the estimated 54,257 patients receiving the addition of RIB to NSAI in 2024 were projected to be $637.7 million. Conclusions: The addition of RIB to NSAI in the adjuvant treatment of HR+/HER2− EBC not only improves iDFS but can also yield substantial work productivity gains for individual patients with HR+/HER2− EBC and for society as a whole.

A novel framework to assess the prevention value of a health intervention

Diabetes Obesity and Metabolism · 2025-11-05 · 1 citations

AIMS: Interest in capturing chronic disease prevention impacts on broader economic and societal outcomes is growing; however, this has been challenging to date. We present a comprehensive framework to support assessment of prevention value from a range of stakeholder perspectives, using obesity as an illustrative example. MATERIALS AND METHODS: We identified three domains in which to group value components: individual health/health system, societal, and government. Longlisting and shortlisting of value components was supported by a targeted literature review, quality assessment against four criteria and review by six health economic and policy experts. Worked examples illustrate the applicability of the framework using two UK-based hypothetical intervention effects: a 5% obesity incidence reduction and a 0.5-unit improvement in SF-12 scores. RESULTS: Twenty-nine value components were identified: 7 related to individual health/healthcare system, 18 to societal and 4 to government value. Of these, 17 were shortlisted into 9 categories: individual health, healthcare system, productivity, family impact, health equity and social mobility, environmental, income-related benefits, sickness benefits, and income tax. We also identified six components for future consideration, where current data and methodologies are limited. CONCLUSIONS: We propose a value framework that goes beyond traditional health benefit measures to holistically assess chronic disease prevention value. While data limitations prevented the quantification of all shortlisted components, this does not preclude a qualitative discussion of these components in evaluating a preventative intervention. The framework should now be tested with live interventions and key stakeholders to standardise methodologies and enhance future acceptability and use.

EE411 Estimating the Potential Cost-Effectiveness of Long-Acting Naltrexone versus Oral Naltrexone for Alcohol Use Disorder

Value in Health · 2025-07-01

Author response for "Metrics that matter: Identifying endpoints for capturing the broad health impacts of prevention of obesity"

2025-07-21

Cost per responder for teclistamab and elranatamab in relapsed or refractory multiple myeloma in the United States

Journal of Medical Economics · 2025-06-11 · 5 citations

AIMS: Teclistamab and elranatamab are bispecific antibodies recently approved for the treatment of triple class-exposed relapsed/refractory multiple myeloma (RRMM). This study assessed the relative efficacy and economic value of teclistamab and elranatamab through a matching-adjusted indirect comparison (MAIC) and cost per responder analysis using data from the MajesTEC-1 (NCT03145181/NCT04557098) and MagnetisMM-3 (NCT04649359) trials. MATERIALS AND METHODS: The MAIC compared overall response rate (ORR) between the therapies after weighting individual patient data from MajesTEC-1 to match key baseline characteristics in MagnetisMM-3. Matched covariates included age, refractory status, prior lines of therapy, extramedullary disease, performance status, disease stage, and cytogenetic risk profile. Cost per responder was calculated based on estimated per-patient drug acquisition and administration cost (2024 United States dollars) over 6 months divided by ORR. One-way and probabilistic sensitivity analyses were conducted to characterize uncertainty. RESULTS: = 123) (odds ratio after matching: 1.02; 95% confidence interval [CI]: 0.59, 1.77). Per-patient costs were estimated to be $231,435 for teclistamab and $285,201 for elranatamab (difference: -$53,766; 95% confidence interval [CI]: -$59,094, -$48,311), yielding costs per responder of $376,930 and $467,730, respectively (difference: --$90,800; 95% CI: -$183,680, $8,148). LIMITATIONS: Because MajesTEC-1 and MagnetisMM-3 are single-arm trials, the MAIC was unanchored and therefore susceptible to confounding from any unadjusted effect modifiers or prognostic variables. CONCLUSIONS: Teclistamab was associated with significantly lower treatment costs and numerically lower cost per responder than elranatamab in triple class-exposed RRMM.

A prospective model of the potential clinical and economic impact of cervical cancer screening supported by a mobile phone app

PLoS ONE · 2025-01-31 · 1 citations

INTRODUCTION: Cervical cancer is a preventable and highly curable disease when detected early and adequately treated, yet it remains the leading cause of cancer-related death in women in Kenya due to low screening coverage and treatment. Implementing World Health Organization screening guidelines for human papillomavirus (HPV) is challenging due to the complex logistics of result return and follow-up requiring multiple clinic visits. Increasing the use of mobile technology can support follow-up care in cervical cancer screening programs. METHODS: We developed a prospective clinico-economic model to assess the potential impact of a mobile phone-based application ("app") communicating laboratory results and recommendations to improve follow-up care for cervical cancer screening in Kenya. The model is structured to simulate a three-visit pathway for HPV-based screening used in a clinical trial of the app and based on epidemiological data, clinical guideline-based workflow, and patient-based behavioral pathways. Published literature, expert elicitation, and time-and-motion observations were used to estimate clinical data, care pathways, and visit-related costs. This analysis was conducted from a base-case healthcare system perspective with a scenario from a "limited" societal perspective. RESULTS: In a simulated cohort of women using the app-based intervention compared to conventional care, with 10,000 women in each arm, use of the app is projected to increase healthcare costs by $12.53 per enrolled woman during the trial period and to detect and treat an additional 247 women-229 with precancerous cervical lesions and 18 with cervical cancer. The incremental cost-effectiveness ratio of the app versus conventional care was $174 per case detected and treated. This would be cost-saving given the average lifetime cost per cervical cancer case of $1,000-$3,000. CONCLUSION: Use of a mobile phone-based app is costlier than conventional screening but by improving visit compliance, it can be a cost-effective and cost-saving strategy to enhance detection and treatment in cervical cancer screening programs.

Author response for "A novel framework to assess the prevention value of a health intervention"

2025-10-13

Metrics that matter: Identifying endpoints for capturing the broad health impacts of prevention of obesity

Diabetes Obesity and Metabolism · 2025-08-25 · 1 citations

AIMS: The link between health and economic prosperity is well established, yet quantifying the value of illness prevention remains challenging due to lack of comprehensive metrics. Obesity exemplifies this issue, having widespread societal and health care impacts but limited prevention funding. Existing metrics often fail to capture broader effects of prevention of obesity. This study aimed to identify key components for a holistic metric to assess obesity and cardiometabolic health progression, better articulating value of prevention. MATERIALS AND METHODS: We conducted a targeted literature review to identify existing individual and composite metrics for prevention in adults, agnostic of disease or risk factor. We categorized endpoints into: (i) holistic composite measures; (ii) user-centred composite measures; (iii) health outcome composite measures; (iv) single clinical endpoints. Metrics were evaluated against eight criteria including holistic outcome, cardiometabolic relevance, data feasibility, geographical generalizability, established outcome, economic modelling methodology, stakeholder relevance and preventive value; alongside semi-structured review from six clinical, health economics and policy experts. RESULTS: Cardiometabolic endpoints numbering 74 were identified: 7 holistic composites, 16 user-centred composites, 31 health-based composites and 20 single clinical endpoints. Five endpoints were shortlisted according to assessment criteria and expert input: waist-to-height ratio, low-density lipoprotein cholesterol, systolic and diastolic blood pressure, blood glucose and the 12-Item Short Form Health Survey (SF-12). These endpoints collectively reflected physiological cardiometabolic risk factors with an adverse association with obesity, while SF-12 provided health-related quality-of-life measurement. CONCLUSIONS: We developed a novel framework that shortlisted five key cardiometabolic outcome measures for assessing obesity primary prevention benefits, with potential applications in health economic modelling and public health.

Extent and causes of the collapse in the registration of innovative medications in Lebanon: A mixed-methods analysis

medRxiv · 2025-08-08

Abstract Objectives Delays in innovative drug registration across countries, or the “drug approval lag”, can cause inequities in treatment access, thereby worsening patient outcomes. Registration delays hinder the first step in making treatments available, and are often linked to regulatory inefficiencies, constrained healthcare financing, and fragmented decision-making. Since late 2019, Lebanon’s health system has faced overlapping socioeconomic and political crises, yet their impact on innovative medication registration remains undocumented. This study aimed to measure this impact by comparing Lebanon’s drug approval lag before (2014-2019) and after (2020-2024) the crisis and to develop an interpretive framework exploring the rationale behind an informal policy to delay innovative medication registration. Methods A mixed-methods approach was adopted. Innovative medications approved by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA) between 2014 and 2024 were included and compared to local registration timelines in Lebanon. In-depth interviews with policymakers, industry leaders, and healthcare providers informed the interpretive framework. Results Findings revealed a dramatic fall in innovative medication registrations post-crisis over these two periods. The proportion of FDA-approved innovative medications registered in Lebanon dropped from 43.6% to 0% and EMA-approved medications dropped from 59.4% to 0%. Pre-crisis, average registration time was under two years; post-crisis, delays are estimated to exceed four years. Our interpretive framework suggests the intermediate effects of delaying innovative medication registration are mainly to control costs and reduce reimbursement pressures on the Ministry of Public Health. However, key stakeholders believe the resulting negative consequences, such as reduced access to life-saving treatments and greater dependence on parallel market importation, outweigh the short-term benefits. Conclusion Health systems are complex adaptive systems and policies affecting the drug approval lag can delay access to innovative treatments. Evidence-informed policies, supported by health technology assessment, are essential to restore Lebanon’s standing as a regional leader.