ISPOR US 2024 Research Posters

Healthcare Resource Utilization of Patients with Short Bowel Syndrome Dependent on Parenteral Support: A Retrospective Claims Analysis

AUTHOR(S): Ali A¹, Micic D², Gallivan M¹, Kulkarni A³, Henderson J⁴, Mitchell G<sup>4 

1</sup>Trinity Life Sciences, Waltham, MA, USA, ²University of Chicago Medical Center, Gastroenterology, Hepatology, and Nutrition, Chicago, IL, USA, ³Trinity Life Sciences, Malden, MA, USA, ⁴VectivBio AG, Basel, Basel, Switzerland

OBJECTIVES: Short bowel syndrome with intestinal failure (SBS-IF) is a rare, chronic, debilitating malabsorptive condition often caused by massive resection of the small intestine, leading to lifetime dependence on parenteral support (PS). Objectives of this study were to quantify patients’ healthcare resource utilization (HCRU), frequency of disease-related symptoms, comorbidities, and PS-specific complications. 

METHODS: A retrospective, real-world analysis of insurance claims between January 2019 – March 2023 was conducted employing the Komodo Healthcare Map™ data. Patients were indexed at first observed PS insurance claim and classified SBS-IF if they had: (1) chronic and continuous PS claims for ≥6 months, (2) intestinal resection or congenital abnormality history, and (3) concurrent diagnosis of intestinal malabsorption. Quantitative analyses estimated symptom and comorbidity frequency, PS-specific complications, and HCRU. 

RESULTS: Overall, 1,587 SBS-IF patients dependent on PS were included in the analysis. Mean age was 35 years at index and 65% were females, with a median follow-up of 2.8 years. Most common comorbidities included depression/anxiety (54%), hypertension (43%), kidney stones (14%), and deep venous thrombosis (DVT) (7%). Most common symptoms included abdominal pain (67%), esophageal disorder (62%), and fever (57%). Approximately 31% of patients had claims for catheter-related bloodstream infections (CRBIs), while 7% of patients had a DVT claim. Patients with ≥1 nutrition claim/week had twice the frequency of CRBIs than patients with ≤0.5 claims/week (39% vs. 19%). Patients frequently required care from high-intensity healthcare settings; 85% of patients visited the emergency department (ED) (mean 4.5 visits) and 75% were admitted as inpatients (mean 2.6 admissions). The most common diagnosis leading to inpatient care was CRBIs (36%). 

CONCLUSIONS: Patients with SBS-IF requiring chronic PS demonstrated high rates of ED and inpatient encounters, oftentimes from PS-related complications. Future treatments should aim to minimize frequency of PS administration, which is associated with elevated risk of CRBIs and greater HCRU. 

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The Institutional Level Impact of Additional Apheresis Days for Multiple Myeloma Patients Undergoing Autologous Stem Cell Transplantation on Costs and Healthcare Resource Utilization

AUTHOR(S): Skaar J¹, Lessor J<sup>2  

1</sup>Trinity Life Sciences, Waltham, MA, ²BioLineRx USA Inc., Waltham, MA 

Topics: Suggest Economic Evaluation 

Financial Disclosure Statement: This study was funded by BioLineRx USA Inc. 

OBJECTIVES: Collection of adequate numbers of CD34+ cells for autologous stem cell transplantation of multiple myeloma patients continues to be a challenge, and multiple mobilization and apheresis attempts may be required. Uncertainty associated with mobilization may result in financial inefficiencies for facilities related to chair time for differentially reimbursed procedures. This economic model sought to understand the cost and healthcare resource utilization impacts of multiple apheresis attempts via an indirect comparison between plerixafor and motixafortide. 

METHODS: The Excel-based model assessed apheresis days associated with mobilization with filgrastim alone or in combination with either plerixafor or motixafortide. Drug costs were from Micromedex, and procedure costs were from CMS.gov. Costs for apheresis days represent a cost per hour average across apheresis procedures, to reflect opportunity loss due to rescheduling events. Apheresis days required were sourced from clinical trials or product labels.   

RESULTS: Under base case assumptions of apheresis days required, the planned cost for a patient ranged from $10,154 to $23,350 based on time for a single chair, days required, and drug price for filgrastim alone (4 days; $20,453-$23,350), filgrastim + plerixafor (2 days; $12,628-$21,755), and filgrastim + motixafortide (1 day; $19,016-$20,826). Based on clinical trial data, 54.2% of patients receiving filgrastim + plerixafor and 86.3% of patients receiving filgrastim + motixafortide achieve target stem cell collection on first day. A subsequent, additional day of apheresis was associated with an unplanned, increased cost of $4,913-$14,743 for filgrastim + plerixafor versus $4,413-$4,775 for filgrastim + motixafortide, showing substantial deviation from projected costs.   

CONCLUSIONS: The requirement for additional stem cell collections was associated with substantial variations in projected cost for facilities, including the opportunity cost for other procedures. At the practice-level, filgrastim + motixafortide was associated with less cost variability for additional collection days, which may facilitate efficient practice-level planning. 

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US Infusion Center Capacity to Accommodate Anti-Amyloid Treatments for Alzheimer’s Disease: A Quantitative Survey 

AUTHOR(S): Silber A¹, Athavale A¹, Kulkarni A², O’Hara M³, Mattke S⁴, Bajaj PS<sup>5 

1</sup>Trinity Life Sciences, Waltham, MA, USA, ²Trinity Life Sciences, Malden, MA, USA, ³Trinity Life Sciences, HINGHAM, MA, USA, ⁴University of Southern California, Center for Economic and Social Research, Los Angeles, CA, USA, ⁵Prothena Biosciences, Inc, San Francisco, MA, USA 

OBJECTIVES: Estimates suggest that approximately 5-7 million US adults have mild cognitive impairment due to Alzheimer’s disease (AD) and an additional 2 million have mild AD dementia, making them potentially eligible for new AD disease-modifying therapies. As these therapies are administered intravenously, infusion center (IC) capacity could become an obstacle to treatment access. This study evaluates the current and future IC capacity to deliver anti-amyloid IV treatments for AD. 

METHODS: A questionnaire was fielded in November 2023 among IC respondents in the US with at least 2 years of experience in the role of overseeing IC capacity and operations. 

RESULTS: The study included 50 US respondents representing unique standalone (50%), hospital-based (26%), and neurology office-based (24%) ICs; 44% (n=22) oversaw a single site IC and 56% (n=28) oversaw a total of 382 centers. Respondents indicated current capacity utilization is on average 83% (range 60%-100%), with 30% (n=15) currently experiencing capacity constraints at their ICs. Among respondents experiencing constraints, 93% (n=14) reported capacity limitations can lead to delayed treatment initiation, missed doses, or delayed doses. 34% (n=17) indicated they currently offer anti-amyloid infusions and can accommodate additional AD patients, and 44% (n=22) reported that their ICs currently do not offer anti-amyloid infusions. Of all respondents, 44% (n=22) prioritized infusion appointment scheduling for patients with severe/rare diseases and comorbidities. Among ICs that plan to offer anti-amyloid treatments in the next five years, 53% (n=25/47) will need to expand their center(s) to treat more patients with AD. 

CONCLUSIONS: ICs are operating at high-capacity utilization on average with current patient volume, raising concerns about the ability to accommodate additional patients now or in the future. Capacity constraints may be further exacerbated with increased availability and demand for anti-amyloid therapies. This study suggests that patients with AD may face challenges with treatment access at ICs. 

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Diversity in Clinical Trials: Life Science Initiatives and Challenges in Light of the FDA’s Latest Guidance 

AUTHOR(S): Andrews M¹, Marsh M¹, Walters D¹, King A¹, Daou S¹, Ingersoll C<sup>2 

1</sup>Trinity Life Sciences, Princeton, NJ, USA, ²Trinity Life Sciences, Waltham, MA, USA 

OBJECTIVES: To understand current initiatives undertaken by pharmaceutical and life science companies to improve clinical trial diversity in the US and recommend tactics to help companies improve their inclusion of underrepresented racial and ethnic groups within their trials. 

METHODS: 60-minute qualitative research interviews with 15 pharma/life sciences company executives involved in executing and spearheading diversity initiatives for clinical trials in the US for their organization. 

RESULTS: Efforts to enhance diversity and inclusion in US clinical trials are pervasive and expanding. Life science and pharmaceutical companies acknowledge the importance of underrepresented populations for robust and generalizable research outcomes in their trials, however recruiting these patients is often challenging. Our research found that companies have enacted a wide array of strategies including creating dedicated teams to address challenges in recruiting diverse populations, broadening eligibility criteria, expanding trial locations, and on-the-ground initiatives targeting specific demographic groups. These initiatives have yielded inconsistent success and efforts are often still limited by human and financial resources. 

CONCLUSIONS: Despite progress, challenges persist in recruitment and retention. With the FDA’s draft guidance getting closer to becoming final, companies are focused more on the clinical trial diversity strategies more now than ever before. This research provides directives on initiatives life sciences companies can consider to support their diversity enrollment goals. 

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Disease- and Glucocorticoid-Related Comorbidities in Classic Congenital Adrenal Hyperplasia: A Claims-Based Retrospective Cohort Analysis

AUTHOR(S): Cheng H¹, Sen GP¹, Kim H¹, Luo MX¹, Cicero S¹, Leinwand B², Li M², Jeha GS<sup>1 

1</sup>Neurocrine Biosciences, Inc., San Diego, CA, USA, ²Trinity Life Sciences, Waltham, MA, USA 

OBJECTIVES: Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD), a genetic disorder characterized by cortisol deficiency and excess adrenal androgens, typically requires management with supraphysiological glucocorticoid (GC) doses. Patients with classic CAH often face multiple complications related to excess androgen and/or supraphysiological GC doses. Here, we characterize those complications among US patients with classic CAH. 

METHODS: Merative’s MarketScan database retrospective insurance claims from 2020-2022 were analyzed. The classic CAH cohort was defined as patients with: (1) 2+ diagnoses of E25.0 30 days apart anytime from 2020-22; (2) a proprietary market definition specific to treatments and tests; and (3) 12 months of continuous 2022 enrollment. The frequency of comorbidities related to excess androgens and/or supraphysiological GC were captured using ICD-10 codes and compared to a general population cohort matched by age, sex, payer type, region, and enrollment duration. 

RESULTS: The classic CAH cohort consisted of 687 patients (57% female) with a mean age of 20 years. Compared to control, classic CAH patients had significantly greater rates of multiple chronic conditions related to both excess androgens and supraphysiological GC doses, including cardiometabolic diseases (cardiovascular disease, hypertension, and diabetes (10.6% vs. 6.6%, 13.2% vs. 8.2%, 5.1 vs. 3.3%, respectively), short statures (7.0% vs. 0.9%), and obesity (17.9% vs. 8.7%). Rates of conditions often related to excess androgens, such as hirsutism and precocious puberty were higher in classic CAH patients (11.0% vs. 1.0%, 9.5% vs. 0.7%, respectively). Rates of comorbidities generally related to supraphysiological GC such as bone fracture were also higher in classic CAH patients (8.9% vs. 6.5). All p-values <0.05. 

CONCLUSIONS: Patients with classic CAH have higher rates of comorbidities related to excess androgens and/or supraphysiological GC compared to the matched general population. 

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Lung Cancer Health Technology Assessments Trends in Argentina, Brazil, Colombia, and Mexico

AUTHOR(S): Wurcel V¹, Criniti JM¹, Salgado Riveros B², Leonart Garmatter L², Rosim M³, Almeida A³, Urrego-Reyes J⁴, Marrugo Arnedo CA⁵, Acevedo R⁶, Junqueira M³, Amaya-Gutierrez MJ³, Bustamante M⁷, Boers Trilles V⁷, Murasawa W⁷, Tôrres L⁷, Villavicencio D⁷

¹MSD Argentina, Munro, Argentina, ²MSD Brazil, Curitiba, Parana, Brazil, ³MSD Brazil, Sao Paulo, Sao Paulo, Brazil, ⁴MSD Colombia, Bogota, CUN, Colombia, ⁵MSD Colombia, Bogota DC, CUN, Colombia, ⁶MSD Mexico, Ciudad de Mexico, DF, Mexico, ⁷Trinity Life Sciences, San Francisco, CA, USA

OBJECTIVES: Non-small cell lung cancer (NSCLC) is the main cause of cancer-related deaths in Latin America (LatAm). We aimed to identify trends in health technology assessment (HTA) for this disease in Argentina (ARG), Brazil (BRA), Colombia (COL), and Mexico (MEX).

METHODS: Pragmatic literature review to identify HTA reports published from 2016-2022 assessing NSCLC technologies in Biblioteca Virtual en Salud, PubMed, Lilacs, EMBASE, supplemented by manual search in government websites for HTA, regulatory and reimbursement data.

RESULTS: 23 reports were retrieved evaluating 16 technologies including pembrolizumab (3), osimertinib (3), nivolumab (2),nintedanib (2), crizotinib (2) among others. Reports produced and outcomes: ARG (12): public body CONETEC 3 (negative or conditions to be met for coverage); IECS 9 (3 negative, 6 no recommendation); BRA (10): public body CONITEC 2 (1 positive leading to reimbursement, 1 negative), ANS 8 (4 positive leading to reimbursement, 4 negative); Many technologies were reimbursed in COL and MEX, but public reports were scarce (COL’ IETS 1, MEX’s CSG published reimbursed therapies and methodological guidelines only). Average time from marketing authorization to HTA publication was 4 years (2-5). Technologies (5) assessed multiple times had inconsistent outcomes. Positive drivers were unmet need, therapeutic benefit, quality of evidence, and cost-effectiveness. High budget impact and risk of bias of clinical data negatively impacted HTA outcomes. Although stakeholders (including patients) were involved in several HTA´s in BRA and ARG, their impact in recommendations was difficult to determine from the reports.

CONCLUSIONS: 78% of HTAs were negative or no recommendation was given for NSCLC technologies in ARG and BRA. MEX and COL did not publish their assessments. Information sharing between HTA agencies, collaborative involvement of stakeholders and documents produced publicly available are needed to improve transparency, align data expectations, and feasible submissions.

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Assessment of Unmet Clinical Needs and Healthcare Resource Use Among Statin-Treated Patients with or at Risk of Developing ASCVD

AUTHOR(S): Hsieh A¹, Rambo R², Leinwand B³, Ali A⁴, Katariya D⁴, Shepherd R<sup>1 

1</sup>NewAmsterdam Pharma B.V., Naarden, n/a, Netherlands, ²NewAmsterdam Pharma B.V., Collegeville, PA, USA, ³Trinity Life Sciences, Chapel Hill, NC, USA, ⁴Trinity Life Sciences, Waltham, MA, USA 

OBJECTIVES: Atherosclerotic cardiovascular disease (ASCVD) leads to considerable clinical and economic burdens. Primary prevention and secondary prevention of cardiac events seek to control low-density lipoprotein cholesterol (LDL-C) to established goals, which vary based on prevention setting and patient factors. Statins aim to lower LDL-C and are first-line treatment. Many patients struggle controlling LDL-C, despite use of statins, leading to poor health outcomes. The study objective was to estimate US unmet clinical needs, healthcare utilization and costs in primary and secondary prevention settings, based on LDL-C goal attainment. 

METHODS: This retrospective cohort study leveraged 2020-2021 MarketScan administrative claims linked to laboratory data, to assess the clinical and economic burden of statin-treated patients not at LDL-C goals. Patients taking statins were segmented into 6 groups based on the prevention setting, LDL-C goal attainment, and risk level (secondary prevention). The number of statin treated patients in each segment was estimated and inflated to national estimates, along with annualized healthcare resource utilization and all-cause healthcare costs. 

RESULTS: Almost 125,000 statin treated patients did not meet LDL-C goals. The US national estimates suggest that 7.8 and 9.7 million patients in primary prevention were above and far above goal, respectively. In secondary prevention, 5 million and 8 million patients were above and far above goals. High healthcare utilization emerged in multiple settings of care (e.g., outpatient, inpatient, and emergency department) across prevention settings and risk levels. Mean annual costs for primary prevention above goal were approximately $9,000, while annualized costs for not high risk and very high-risk secondary prevention above goal were approximately $14,000 and $27,000, respectively. 

CONCLUSIONS: Millions of patients on statins are not achieving their LDL-C goals, indicating a significant clinical burden among patients with, or at risk for ASCVD. This unmet treatment need results in substantial healthcare resource use and costs each year. 

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Going Beyond Claims: Unleashing the Power of Diverse Healthcare Datasets in Clinical and HEOR Assessments 

AUTHOR(S): S SS¹, Patel N², Li O², Lovink A², Skaar J<sup>2 

1</sup>Trinity Life Sciences, Burlington, MA, USA, ²Trinity Life Sciences, Waltham, MA, USA 

OBJECTIVES: Retrospective medical claims data are a cornerstone for health economics and outcomes research (HEOR) studies, but they lack clinical specificity and offer limited level of detail in specific care settings. This study aimed to identify constraints that hinder answering specific research questions within retrospective real-world studies reliant solely on claims data and systematically compare these limitations against alternative real-world evidence (RWE) sources, such as hospital service-level data, electronic health records (EHR), laboratory data, and physician affiliations data. The research sought to delineate potential inadequacies of claims data and propose strategies for leveraging diverse real-world sources to address these limitations and enhance the depth and reliability of retrospective real-world studies. 

METHODS: A systematic assessment of different RWE sources, including claims, hospital service-level data, EHR, and laboratory data, was conducted to assess factors such as data granularity, data completeness, and capture of clinical and economic metrics for specific HEOR use cases. The assessment framework was paired with a literature review of published material on data sources, product catalogs, and data dictionaries based in the United States. 

RESULTS: For each HEOR use case, 6-10 data products were evaluated. Over half lacked at least two or more critical components, such as clinical specificity, cost information, longitudinal capture, linkability, demographic or geographic representativeness or level of reporting detail. In use cases requiring visibility into inpatient hospital or intensive care settings, claims datasets provided little to no insight into treatments, physician encounters, or service details. Hospital billing or electronic health records (EHR) datasets provided treatment specificity but low representativeness. Linked sources (e.g., claims + EHR) often excluded critical metrics (e.g., mortality) to comply with HIPAA regulations. 

CONCLUSIONS: Novel analytical design techniques were documented that allowed for capture of additional clinical specificity in several use-cases through the combination of two or more unlinked data sources while enabling population-level insight generation. 

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Measuring Healthcare’s Environmental Impact: Data Availability across a Spectrum of Interventions and Therapeutic Areas 

AUTHOR(S): Silber A¹, Goswami L¹, LaGreca E¹, Hamilton L², O’Hara M<sup>3 

1</sup>Trinity Life Sciences, Waltham, MA, USA, ²Trinity Life Sciences, Washington DC, DC, USA, ³Trinity Life Sciences, HINGHAM, MA, USA 

OBJECTIVES: The importance of incorporating sustainability into health technology assessments and economic evaluations of new therapies and medical devices has grown in recent years. Validated environmental sustainability (ES) metrics that can be mapped directly to healthcare resource utilization (HCRU) will be crucial to an integrated environmental and economic perspective in these assessments. Thus, we conducted a targeted literature review to determine the availability of existing ES metrics which could serve as inputs in future health economic models. 

METHODS: A literature search was conducted using PubMed and Google Scholar to identify papers published in the last 5 years that evaluated ES metrics in health interventions. Search terms included, but were not limited to, “carbon footprint, “greenhouse gas”, “environmental impact,” coupled with HCRU and intervention-specific terms such as “diagnostic,” “health facilities”, “therapeutics, and “healthcare treatment.” Google searches were also used to assess the availability of existing datasets offering ES and/or HCRU metrics. 

RESULTS: Across the 313 papers identified, 32 articles included ES metrics related to HCRU. Health interventions included surgeries (n=19 studies), dialysis (n=11), anesthesia (n=9), respiratory treatments (n=10), endoscopy (n=7), emergency care (n=7), laparoscopy (n=4), and catheters (n=5). Common ES indicators were carbon footprint (n=28 of studies included CO2 equivalent), waste generation (plastics, drugs, tools; n=7), energy consumption (n=5), water usage (n=4), and certain chemicals (nitrous oxide, isoflurane, propofol; n=3). Additional topics included transport impact, biodiversity, and mineral resource depletion. Studies of health interventions often evaluated multiple ES indicators. 

CONCLUSIONS: There is limited published research quantifying the direct environmental impact of health interventions, which may present a barrier to generating comprehensive assessments of ES in healthcare. These findings reinforce the need to develop a consolidated and validated set of ES metrics that can be mapped directly to HCRU and used in future modeling efforts to quantify the sustainability of therapies and medical devices. 

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Cost-Effectiveness of Obicetrapib As Adjunctive Therapy Compared to Maximally Tolerated Statins for Lowering LDL-c

AUTHOR(S): Davidson D¹, Oak B², De A³, Rambo R⁴, Budd D², Hadker N², Hsieh A<sup>5 

1</sup>NorthShore University Health System, Bannockburn, IL, USA, ²Trinity Life Sciences, Waltham, MA, USA, ³Trinity Life Sciences, Long Island City, NY, USA, ⁴NewAmsterdam Pharma B.V., Collegeville, PA, USA, ⁵NewAmsterdam Pharma B.V., Naarden, n/a, Netherlands 

OBJECTIVES: Assess the cost-effectiveness of obicetrapib, a novel cholesteryl ester transfer protein (CETP) inhibitor as adjunctive therapy to maximally tolerated statins to reduce low-density lipoprotein cholesterol (LDL-c) and reduce the incidence of associated Major Adverse Cardiac Events (MACE). 

METHODS: A published Markov model was adapted to simulate outcomes for a 10,000-patient cohort on maximally tolerated stable dose statin, over a lifetime horizon. The model contained three states – Above Goal, At Goal, and Death. Patients entered in “Above Goal” state, defined by LDL-c above 100 mg/dL in patients without a history of ACSVD (“Primary Prevention”). Outcomes were derived from the Phase 2 ROSE study. MACE (stroke, myocardial infarction (MI), revascularization, cardiovascular-related death) were estimated using published risk rates. Age-adjusted mortality was estimated using United States census tables. Life years gained (LYG), quality-adjusted life years (QALYs) gained, MACE and other adverse events were calculated. Direct medical costs were considered, including pharmaceutical and associated medical costs. One-way and probabilistic sensitivity analyses were conducted. 

RESULTS: Adjunctive obicetrapib was cost-effective at $150,000 per QALY compared to maximally tolerated statin monotherapy in both the Primary and Secondary prevention cohorts. Over the lifetime horizon, obicetrapib polypharmacy reduced all MACE in the base case. Patients treated with obicetrapib had 800 fewer MI, 200 fewer strokes, and 700 fewer revascularizations. Overall, obicetrapib plus maximally tolerated statins resulted in 0.49 more QALYs in the Primary Prevention arm. Fewer events in the obicetrapib cohort yielded significant medical cost savings, offsetting associated polypharmacy costs. 

CONCLUSIONS: Millions of patients treated with maximally tolerated statins are unable to reach and maintain LDL-c goals as defined by the AHA/ACC. A regimen of obicetrapib plus maximally tolerated statins is a cost-effective way to drive goal attainment, reduce serious cardiovascular events, and gain quality-adjusted life years. 

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The Net Environmental Impact of Preventative Healthcare – Vaccination’s Potential in the Fight Against Climate Change

AUTHOR(S): Hamilton L¹, O’Hara M², Kataria D<sup>3 

1</sup>Trinity Life Sciences, Washington DC, DC, USA, ²Trinity Life Sciences, HINGHAM, MA, USA, ³Trinity Life Sciences, Gurgaon, Haryana, India 

OBJECTIVES: The negative impact of climate change on human health and the pharmaceutical industry’s impact on the environment are well-documented. However, not all healthcare is created equal from an environmental perspective. Negative impacts of R&D, manufacturing, distribution, use, and disposal can potentially be offset by preventing future illness and resource-intensive healthcare. Prophylaxis is cost-effective, but is it environmentally favorable? This literature review explores the holistic environmental impact of vaccination. 

METHODS: A targeted literature search of PubMed, ISPOR archives, and Google covering academic and gray literature sources from 2019-2023 was conducted. The search utilized two primary search strings with vaccination and environmental impact terms. 

RESULTS: 1,692 sources were accessed, reduced to 36 abstracts based on keyword refinement and title review; data were extracted from 26. 20 were peer-reviewed, of which 14 examined the negative environmental impact from the production, use, and disposal of vaccines, predominantly emissions. No sources converted environmental into economic impact. 4 publications, all industry-sponsored whitepapers, examined potential reductions in healthcare resource utilization based on vaccination averting negative environmental impact. One by GSK demonstrated 18% carbon savings from hospitalizations avoided due to herpes zoster vaccination. Another by AZ showed that per-case influenza treatment costs ~15x the carbon of each vaccination. No consistent methodology was used for the quantification of either negative environmental impacts or their potential offsets. 

CONCLUSIONS: Our research finds that peer-reviewed publications have focused on the negative upstream effects of vaccines, and that gaps to understand the holistic net impact are being filled by manufacturers. The hypothesis that vaccination may be net positive environmentally cannot be confirmed/refuted based on current evidence. Continued research is needed to develop standardized methodologies and prevent greenwashing in this nascent field. Converting impact to net cost and the effect of alternative options (e.g., orals and microneedle patches) should also be explored. 

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USA Vs. Global Pharmaceutical Launches: What Are the Potential Reasons That Can Result in a Pharmaceutical Not Being Commercialized Outside the USA? 

AUTHOR(S): Taxak N¹, Ismailoglu I², Hunt M<sup>3 

1</sup>Trinity Life Sciences, Gurugram, HR, India, ²Trinity Life Sciences, Minneapolis, MN, USA, ³Trinity Life Sciences, New York, NY, USA 

OBJECTIVES: To understand the frequency of cases where products are not launched in international markets following FDA approval and identify potential rationale driving the decisions. 

METHODS: We evaluated specialty medicines approved by the FDA between 2019 and July 2023. The list was matched against regional / local regulatory and access authority decisions in a set of to identify any medicines that are not launched in some regions. 

RESULTS: For 191 unique US FDA approved branded specialty medicines, following observations were made: 

1. 102 products have been approved by the EMA post FDA approval out of which 12 have not been filed for reimbursement in FRA and DEU 
   1. Manufacturers did not seek reimbursement for 21 products in the GBR
   2. Out of 86 EMA approved products which sought reimbursement, 11 withdrew from DEU (such as Rybrevant, Tabrecta) mostly due to challenges in price negotiations
2. Remaining products are either in the process of being evaluated by the EMA (N=15), had been approved by the EMA before FDA (N=20), are in the trial stage (N=25), have been abandoned (N=8) or have not yet sought EMA submission / approval (N=20) 
3. 58 products have been approved in AUS out of which 19 have not sought reimbursement 
4. 69 products have been approved in CAN out of which 8 have not sought reimbursement 

Our analysis identified potential drivers for no-launches in the international markets as concerns with the evidence package and challenges in pricing negotiations. 

CONCLUSIONS: This assessment identifies the key clinical and economic reasons behind the gaps in commercialized medicines in international markets vs. the US. Alignment between global payer communities and pharmaceutical manufacturers on evidentiary expectations and potential novel methods of generating evidence can accelerate access to innovative medicines globally.