ISPOR Annual
May 13 - 16, 2025
Montreal, Canada
Booth 821
Trinity Life Sciences looks forward to attending ISPOR US from May 13 – 16 in Montreal, Canada. Join us in booth 821 to learn more about why an integrated approach to evidence planning and generation is critical when crafting a strategic value story optimized to achieve commercialization goals. Be sure to attend Trinity’s podium presentation on “Implementation of the IRA: Evolving Perspectives from US Stakeholders” on May 16 at 10 AM or stop by one of our sixteen poster sessions to talk to our experts.
POSTER SESSIONS
Assessing the Impact of Sociodemographic Factors on Patient Perceptions of Chronic Heart Failure Management
Poster Session 3 / Poster Code PCR108
Thursday, May 15 | 10:30 AM – 1:30 PM
Authors: Brittany Smith1, Amal Gulaid1, Clive Mendonca1, Lisa Bailey1
1 Trinity Life Sciences, Waltham, MA, USA
OBJECTIVES: Chronic Health Failure (CHF) is one of the leading causes of death and morbidity in the United States, affecting over 6 million people. Disparities exist in CHF management by race and gender. The objective of this study is to evaluate patient preferences in care and the patient-HCP relationship from the perspective of patients with CHF and how they vary by sociodemographic factors (e.g., race, gender, etc.).
METHODS: A quantitative, IRB-exempted, non-interventional study was conducted in the United States from November 2024 to January 2025. The study comprised of a questionnaire for US patients with CHF. Key metrics included perceptions of their current treatment experience, relationship with their cardiologist, and new treatments.
RESULTS: A total of 75 patients with CHF participated in the study with an average age of 51. Among the patients, 36% (27) identified as Black alone, and 44% (33) identified as White alone; 43% (32) identified as men and 57% (43) identified as women. Among Black patients, 63% were men; among White patients, 67% were women. Patients were most likely managed by cardiologists of the same race (89%-Black, 88%-White) and same gender (91%-Man, 65%-Woman). Overall, 41% felt their cardiologist valued their thoughts regarding their CHF, similar by race and gender. White patients were more likely to bring up switching medications when unsatisfied (55% vs 33%) and consider participating in CHF clinical trials (52% vs 37%), similar by gender.
CONCLUSIONS: A majority of patients with CHF did not feel their cardiologist valued their thoughts. Additionally, Black patients were more hesitant to bring up treatment switching even when unsatisfied. This research suggests a lack of patient comfort in the patient-HCP relationship, further exacerbated by sociodemographic factors. These findings highlight an opportunity for improvement in patient-centered care.
Burden of Illness in US Patients With Liver Cancer and Kidney Disease: A Real-World Claims Analysis
Poster Session 5 / Poster Code RWD159
Friday, May 16 | 9:00 AM – 10:00 AM
Impact of China’s NRDL Value-Rating System on Reimbursement and Pricing of Innovative Therapies
Poster Code HTA57
Authors: Wenting Zhang1, Allen Liu1, Rya Zhang1
1 Trinity Life Sciences
OBJECTIVES: This research aims to explore the newly implemented value-rating system of China’s National Reimbursement Drug List (NRDL) to assess its impact on innovative products’ reimbursement & pricing outcomes.
METHODS: An in-depth analysis of the 2024 NRDL Negotiation results was performed to evaluate how the value ratings of participating drugs may influence their pricing outcomes. Our analysis employs a three-step approach: 1) Identify key successful drug candidates from the 2024 NRDL negotiation, focusing on oncology, rare diseases, and chronic conditions; 2) Evaluate the added clinical benefit compared to standard of care/NRDL-listed competitor(s) and estimate their likely value ratings; 3) Examine the potential relationship between value ratings and pricing outcomes for these therapies.
RESULTS: The value-rating system is designed to objectively evaluate a product’s clinical benefits during the expert review process, categorizing drugs into one of four tiers to determine their eligibility for negotiation. This framework has significantly raised the threshold for entering negotiations, with 63% of products failing to pass the expert review—likely due to low value ratings assigned. Among the 117 new products that advanced through the expert review, 88 were successfully added to the NRDL, reflecting a 75% success rate. Our analysis indicates that products with higher value ratings (e.g., “”breakthrough””) are more likely to achieve favorable pricing outcomes than those with lower ratings (e.g., “”equivalent””). As a result, the value tiering system may indirectly influence NRDL pricing outcomes.
CONCLUSIONS: While no official guidelines delineate its role in NRDL pricing, the value rating system provides a practical and directional framework for assessing pricing and access outcomes for innovative medicines in China. Although budget impact and pricing thresholds remain critical to NRDL negotiations, the increasing significance of the value rating system has revealed emerging pricing trends tied to product value.
Consumer and Healthcare Provided Preferences for Adult Combination COVID-19 and Influenza Vaccines: Results from a Discrete Choice Study in France, Italy and Germany
Poster Session 3 / Poster Code PCR136
Thursday, May 15 | 10:30 AM – 1:30 PM
Authors: Mariana Servin1, Darshan Mehta1, John Crocker2, Shivam Jindal2, Omer Ismail2, Nicolas Van de Velde1
1 Moderna, Inc
2 Trinity Life Sciences
*Presenting author
OBJECTIVES: Quantify consumer and healthcare providers (HCPs) preference for an adult combination vaccine for influenza and COVID-19 compared with standalone influenza and COVID-19 vaccines; and estimate the resulting impact on influenza and COVID-19 vaccine coverage rates (VCR) for adults in France, Italy, and Germany.
METHODS: A discrete-choice experiment (DCE) with hypothetical vaccine profile was conducted in each country. Each DCE included following attributes with varying levels: influenza only, combined influenza & COVID-19, vaccine composition, influenza immunogenicity and COVID-19 immunogenicity, tolerability (). The study population, recruited via an online panel, included consumers aged ≥18 years (FR: 256, DE: 256, IT: 250) and HCPs responsible for vaccination (FR: 80, DE: 78, IT: 80). Respondent data were weighted to reflect the population by age segment and to align influenza and COVID-19 VCR for 2023 – 2024. Respondent preferences from the DCE were used to estimate the change in influenza and COVID-19 coverage rates due to availability of a combined vaccine.
RESULTS: The mean age of the consumer sample was 53.9 years including male (62%) and female (38%) respondents. Consumers in all countries expressed preference for a combination vaccine; on average, 53% of consumers strongly preferred a combination vaccine, 23% strongly preferred individual vaccines, and 24% expressed no preference. The majority of HCPs demonstrated high likelihood to recommend a combination vaccine to patients (FR: 52%, DE: 57%, IT: 65%). Modelled preference shares projected that flu and COVID-19 VCR will likely increase because of availability of combination vaccine across markets (COVID VCR increase in percentage points – FR: 6%, DE: 7%, IT: 5%: FLU VCR increase in percentage points – FR: 5%, DE: 3%, IT: 3%)).
CONCLUSIONS: Results suggests that most consumers and HCPs preferred and recommended a combination flu+COVID-19 vaccine compared with standalone vaccines. Further, availability of a combination vaccine could help increase COVID-19 and flu VCR.
FUNDING: This study is funded by Moderna, Inc
Digital Health Interventions and Environmental Sustainability: A Targeted Literature Review
Poster Session 2 / Poster Code MT17
Wednesday, May 14 | 4:00 – 7:00 PM
Authors: Zipporah R. Abraham Paiss1*, Ellie Goldman, MPH1, Liz Hamilton, MPH1, Sumudu Dehipawala, MPH1, Abigail Silber, MPH1, Matthew O’Hara, MBA1
1 Trinity Life Sciences
*Presenting Author
OBJECTIVE: Healthcare is a substantial contributor to global greenhouse gas emissions. Digital health interventions (DHIs), such as telemedicine and remote patient monitoring (RPM), offer the potential to reduce healthcare’s environmental footprint while maintaining or improving patient outcomes. This study evaluates the environmental impact of remote-care DHIs, analyzes metrics for evaluating this impact through HEOR methodologies and frameworks, and examines implications for healthcare decision-makers and systems.
METHODS: A targeted literature review was conducted through PubMed and gray literature sources from January 2019 to December 2024. Inclusion criteria focused on articles, case studies, and industry reports quantifying environmental benefits of DHIs. Data extraction captured intervention type, environmental metrics, and methodological frameworks.
RESULTS: Fifteen papers reported on metrics relevant for HEOR evaluations, including life cycle assessment-based CO2 reductions, avoided resource use, and economic valuations of environmental benefit for DHIs. Telemedicine consultations in 2020 from ~640,000 patient appointments in Spain avoided over 6,600 tons of CO2 emissions by eliminating travel and using digital health reports. Additionally, DHIs showed no evidence of adversely affecting patient outcomes; one study found no significant difference in readmission rates for heart failure patients receiving telemonitoring, while another indicated that RPM for chronic disease management reduced hospital readmissions.
CONCLUSIONS: DHIs can improve environmental sustainability within healthcare and drive innovation in patient care. These findings highlight the opportunity for healthcare systems to implement DHIs to support sustainability targets while maintaining or enhancing patient outcomes. Life sciences companies should consider incorporating sustainability metrics into HEOR research to quantify the environmental impact of products and devices. Aligning with environmental goals address regulatory and societal pressures, positioning sustainability as a differentiator in an increasingly eco-conscious healthcare ecosystem. Future research should prioritize continued data collection on environmental outcomes to strengthen this emerging framework.
Effectiveness of Activity Criteria in Stabilizing Patient Cohorts in Open Claims Data for Healthcare Outcomes Research
Poster Session 3 / Poster Code MSR82
Thursday, May 15 | 10:30 AM – 1:30 PM
OBJECTIVES: Administrative claims are foundational in health economics and outcomes research studies, with payer-sourced, fully adjudicated “closed claims” datasets preferred given completeness of patient capture. Sometimes, however, only clearinghouse-sourced “open claims” data with selective capture of medical events and treatments is available. Activity criteria are often used in lieu of enrollment in such cases but may incur selection bias towards a more clinically active cohort. There is a gap in knowledge to quantify potential inadequacies in open claims and identification of corrective strategies for improving reliability of retrospective studies using open claims. This study evaluates several activity criteria for a similarly defined open claims population across common healthcare resource utilization (HCRU) metrics compared to a closed claims population.
METHODS: We conducted a retrospective, real-world analysis of administrative claims data across January-December 2023. Patients with two or more Type 2diabetes claims, at least 30 days apart, were identified in closed claims Merative MarketScan data and open claims Symphony Health data. A random sample of closed claim patients with 12 months of continuous enrollment in 2023 were matched against open claim patients on age and gender (1:1 ratio) using propensity score matching. Both cohorts were evaluated by the number of healthcare episodes by setting. This analysis was repeated thrice with variable minimum activity thresholds. Performance is being evaluated based on the difference in endpoints.
RESULTS: A framework to catalog and evaluate each activity criterion’s performance against closed claims is being developed. Healthcare cost analysis was excluded because reimbursement amounts in open claims are mostly unadjudicated with low capture rates.
CONCLUSIONS: Initial results indicate that open and closed claims studies yield different results, even with the application of proxy criteria for enrollment. Among type 2 diabetes patients, we observe that although some options have better performance, none directly replicate the results found using closed claims.
Foundational Insights and Costs of Care Associated With Perioperative Ticagrelor Management for Patients Undergoing Coronary Artery Bypass Graft Surgery: A Medical Chart Audit Review
Poster Session 5 / Poster Code SA75
Friday, May 16 | 9:00 – 11:30 AM
Ralph J. Riello III, PharmD14, Christina England, MPH2, Aishwarya Kulkarni, MS3, Vijay Abilash, MS3, Bhagyashree Oak, PhD3, Matthew O’Hara, MBA3*, John McKenney2, Jackie Evans-Shields, PharmD2, John Fanikos, RPh, MBA4
1 Clinical & Translational Research Accelerator, Yale University School of Medicine; New Haven, CT
2 SERB Pharmaceuticals, Conshohocken PA
3 Trinity Life Sciences, Waltham MA
4 North American Thrombosis Forum, Vasculearn Network, Brookline, MA
* Presenting author
OBJECTIVES: Characterize the patient journey, including clinical outcomes, healthcare resource utilization (HCRU), and cost of care for ticagrelor-treated patients undergoing coronary artery bypass graft (CABG) surgery after antiplatelet washout.
METHODS: We conducted an IRB-exempt, medical record review between November-December 2024. Cardiac/cardiothoracic surgeons/interventional cardiologists who annually manage ≥5 patients receiving ticagrelor undergoing CABG completed an electronic questionnaire to abstract data on patient demographics, incidence of complications, pharmacy, blood product use and HCRU. Descriptive analyses were conducted for three phases of care: pre-surgery, intraoperative, and post-procedure. Costs were aggregated across multiple sources.
RESULTS: Sixty-six physicians provided 228 patient encounters of which 195 underwent ticagrelor washout. Patients experienced bleeding pre- (17%) and post-CABG (11%). During antiplatelet washout, common complications included hospital-acquired infection (HAIs, 3%) and mechanical ventilation (3%). The average cost per patient during washout, including observation, complications, and blood products cost, was $5,757. 27% of patients were admitted to the intensive care unit (ICU) perioperatively, with an average length of stay (LOS) of 45 days in the Surgical ICU (SICU) or 17 days in the Cardiac ICU (CICU). 9% of patients had non-ICU stays, with an average LOS of 4.4 days. The average pre-CABG hospital stay cost per patient was $17,167. Post-procedure, common complications were mechanical ventilation (8%) and HAIs (3%). ICU stays were an average LOS of 7 days in SICU and 10.2 days in CICU. The average post-procedure cost per patient, including complications, blood product, and hospital stay costs, was $12,000.
CONCLUSIONS: Perioperative ticagrelor management for patients undergoing CABG is expensive, with high HCRU and corresponding costs, particularly prior to surgery. This study highlights the need for a rapid ticagrelor reversal agent to reduce the clinical and economic burden of antiplatelet washout among patients undergoing CABG surgery.
Health equity and HTA: Is Equity Data Shaping Recommendations
Poster Code HTA28
Authors: Derek Xu1, Mary Fletcher-Louis1, Andreia Ribeiro1
1 Trinity Life Sciences, London, UK
OBJECTIVES: The purpose of health technology assessment (HTA) agencies globally is to make evidence-based evaluations of the value of new health interventions. Increasingly, HTA agencies ask manufacturers to include reference to health equity in their submissions. Following an earlier pilot study, this study aims to understand how frequently HTA agencies (United Kingdom, Canada and Australia) ask manufacturers to provide evidence relating to health equity, and how frequently they refer to that evidence in their assessment reports.
METHODS: A comparison of the most recent HTA agency guidance (including submission templates) and assessment reports was undertaken to assess the nature of health equity-related guidance. Evaluation was performed to assess the presence and role of health-equity related drivers and detractors of value in HTA assessments by NICE, CDA and PBS in 2024. All 57 pharmaceuticals assessed in single technology appraisals (STAs) by NICE in 2024/5 served as the index list for review of assessments by all three agencies. We considered elements of value in two broad categories; therapy area-related value (e.g. investment in indications that disproportionately impact vulnerable or underserved populations) and intervention-related value (including clinical trial diversity, patient support initiatives, and intrinsic features of the intervention that may impact equitable access).
RESULTS: Our analysis found evidence for health equity considerations impacting HTA decision making. The frequency with which equity is referenced within assessment reports varies between countries; of NICE, CDA, and PBAC, NICE most consistently states how health equity-related deliberations have impacted the assessment outcome.
CONCLUSIONS: Health equity considerations may impact the overall recommendation, or the size of the population recommended for reimbursement. As equity-related economic modeling methodologies mature, it may be that agency requirements around equity data submitted become more prescriptive.
Implementation of the IRA: Evolving Perspectives from US Stakeholders
Friday, May 16 | 10:00 – 11:00 AM
Authors: Alexis Bryan1, Katharine Wu1, Jessica Counihan, PhD1, Ismail Ismailoglu, PhD1
1 Trinity Life Sciences
OBJECTIVES: In August 2022, the Inflation Reduction Act (IRA) was signed into law with provisions to improve Medicare through cost containment measures. These measures include mandatory negotiations for top selling Medicare drugs, Medicare beneficiary annual out of pocket cap over $2,000, and manufacturer penalty rebates if therapy prices are increased at a steeper rate than inflation.
On August 15, 2024, CMS released the results of the first round of negotiated prices for the IRA involving 10 Medicare Part D branded drugs which raised questions around future negotiations and how they may impact drug pricing strategies moving forward.
METHODS: This study presents the results of primary research from N=8 US payer stakeholders that provide current perceptions of the IRA and its potential impact on the pharmaceutical landscape. By exploring stakeholder concerns and expectations, this research identifies emerging trends and raises key questions around the evolving pharmaceutical landscape resulting from the CMS negotiations.
RESULTS: The first batch of CMS negotiations have yielded modest drug price cuts, primarily due to existing substantial rebates on the selected products. While expected, these outcomes highlight uncertainties surrounding future negotiation rounds. Payers emphasize the economic burden of IRA changes as a key concern (e.g., plan contribution for OOPs above the $2K cap). Uncertainties cited include which therapeutic areas will be impacted, whether manufacturers are expected to adapt life cycle management strategies (e.g., reformulating products to delay negotiations), and how payer dynamics may shift in response to these changes.
CONCLUSIONS: The broader impact of the IRA on Medicare and CMS negotiations is becoming clearer, though there remains ambiguity around how the pharmaceutical industry will respond. The impact of the IRA on the commercial sector remains uncertain, with stakeholders debating whether Medicare-focused policies will drive broader systemic changes or further exacerbate pricing challenges seen in healthcare.
Integrating Metrics of Environmental Sustainability into Economic Evaluations: A Targeted Literature Review
Poster Session 4 / Poster Code EE422
Thursday, May 15 | 4:00 – 7:00 PM
Authors: Zipporah R. Abraham Paiss1*, Ellie Goldman, MPH1, Liz Hamilton, MPH1, Sumudu Dehipawala, MPH1, Abigail Silber, MPH1, Matthew O’Hara, MBA1
1Trinity Life Sciences
*Presenting Author
OBJECTIVES: This study examines how environmental sustainability (ES) metrics have been incorporated into economic evaluations and proposes new innovative methodologies for advancing the integration of ES into these frameworks.
METHODS: A targeted literature review was conducted to identify existing approaches to incorporating ES metrics into economic evaluations. Peer-reviewed articles, conference materials, and HTAs were analyzed to synthesize key methodologies and identify gaps in current approaches, focusing on practical implementation and alignment with decision-making priorities.
RESULTS: Eighteen relevant papers were identified from January 2017 to December 2024. Existing approaches that HTAs have taken to integrate ES metrics into economic evaluations include hybrid cost models that adjust willingness-to-pay thresholds to account for environmental externalities (e.g., carbon emissions), incremental carbon footprint ratios that quantify trade-offs between environmental and economic outcomes, and life cycle analyses that leverage scenario-based modeling to evaluate long-term environmental impacts (e.g., waste reduction), among other approaches.
CONCLUSIONS: HTAs have sought to incorporate ES into evaluation metrics, highlighting the growing influence this component may have in future assessments. This research highlights that methods for ES analysis are still crystallizing. Creating more codified analytical approaches and tools could enhance the incorporation of sustainability in HTA evaluation frameworks, making them more accessible to non-experts, and aiding discussions with various stakeholders, including policymakers and patients.
While these methods highlight the applicability of established HEOR modeling techniques in ES, additional novel methodologies could be considered as the focus on environmental sustainability becomes more prevalent in healthcare. Based on these findings, the authors of this research propose the use of “gamified environmental metrics” to address critical gaps by using interactive dashboards that visually simulate trade-offs between clinical, economic, and environmental outcomes in a visual, intuitive way. Future research should focus on operationalizing these methods to optimize clinical, economic, humanistic, and environmental priorities.
Medical Record Review to Characterize the Foundational Insights and Costs Associated with Intracerebral Hemorrhage (ICH) Management Among Ticagrelor-Treated Patients
Poster Session 4 / Poster Code SA59
Thursday, May 15 | 4:00 – 7:00 PM
John Fanikos, RPh, MBA1, Christina England, MPH2, Aishwarya Kulkarni, MS3, Vijay Abilash, MS3, Bhagyashree Oak, PhD3, Matthew O’Hara, MBA3*, John McKenney2, Jackie Evans-Shields, PharmD2, Ralph J. Riello III, PharmD4
1 North American Thrombosis Forum, Vasculearn Network, Brookline, MA
2 SERB Pharmaceuticals
3 Trinity Life Sciences
4 Clinical & Translational Research Accelerator, Yale University School of Medicine; New Haven, CT
*Presenting author
OBJECTIVES: Characterize key aspects of the patient journey, including clinical outcomes, healthcare resource utilization (HCRU), and cost of care among ticagrelor-treated patients hospitalized for management of ICH.
METHODS: We conducted an IRB-exempt, retrospective medical record review between November-December 2024. Neurologists/neurointensivists/neurosurgeons/trauma surgeons who annually manage ≥5 patients on ticagrelor undergoing ICH treatment completed an electronic questionnaire to abstract data on patient demographics, incidence of complications, pharmacy, blood product use, and HCRU. Descriptive analyses of survey results are reported for the phases of care: during and post-treatment. Costs were aggregated across multiple sources.
RESULTS: Nineteen physicians who responded to the online survey identified 69 ICH patients with 38% experiencing hematoma expansion and some with complications like seizures or brain swelling (35%) and mechanical ventilation (42%) during care. The average cost per patient associated with treatment was $24,793. Post treatment, common complications experienced were hemorrhagic complications (26%) and mechanical ventilation (33%). The average per patient cost associated with all complications post-procedure was calculated to be $23,849. 42% of patients were admitted to the intensive care unit (ICU) with an average length of stay (LOS) of 21.7 days in the Neuro-ICU or 11.1 days in the surgical ICU; 15% had non-ICU admissions, with an average LOS of 14.7 days. The average cost per patient associated with post-treatment hospital stay was calculated to be $18,208. Other expenditures associated with ICH treatment among patients receiving ticagrelor include costs for blood product and pharmacy utilization of $647 and $418 per patient, respectively.
CONCLUSIONS: Ticagrelor-treated patients hospitalized for ICH management require substantial HCRU due to frequent complications, prolonged hospital stays, and costly pharmacy and/or blood product use with uncertain clinical benefits. This study highlights the unmet need for more targeted treatment strategies to reduce the clinical and economic burden associated with ICH management related to ticagrelor use.
Navigating Scarcity: Leveraging AI to Address the Challenges of Identifying Rare Disease Key Opinion Leaders
Poster Session 3 / Poster Code MSR85
Thursday, May 15 | 10:30 AM – 1:30 PM
Authors: Zipporah A. Paiss1, Mason L. Yeh, PhD.1*, Matthew O’Hara, MBA1
1 Trinity Life Sciences
*Presenting Author
OBJECTIVES: Over 7,000 known rare diseases affect ~300 million patients worldwide. However, qualified Key Opinion Leaders (KOLs) with expertise in rare conditions are often difficult to identify using traditional methods, presenting challenges in identification and limiting collaborative evidence generation opportunities. This study evaluates trends in rare disease KOL identification to benchmark traditional methods against AI-driven approaches, highlighting opportunities to improve research collaboration.
METHODS: Primary research was conducted with industry leaders to identify current tactics, tools, and gaps in KOL identification methods. Building on these insights, we aim to evaluate various AI-enabled tools and unconventional resources (e.g., patient advocacy networks) for their effectiveness, scalability, and potential to address existing gaps.
RESULTS:
- Challenges Identified: A limited pool of rare disease KOLs leads to over-reliance on a few prominent experts, creating biases in research priorities and decision-making.
- Trends in KOL Identification: Increasing use of AI and data analytics enable broader and deeper insights into KOL landscapes.
- Benchmarking Outcomes: AI-enhanced tools demonstrate advantages in identifying underrecognized experts, uncovering networks, and decrease duration of workstreams by ~50%.
FORWARD-LOOKING OPPORTUNITIES:
- AI Integration: Employ machine learning (ML) and natural language processing (NLP) to aggregate and analyze data from diverse sources (e.g., gray literature, clinical trial databases, social media).
- Unconventional Data Mining: Expand searches to include patient advocacy groups, community networks, and non-traditional publication platforms.
- Collaborative Databases: Develop centralized, AI-enhanced registries of rare disease experts, fostering equitable access for researchers, policymakers, and industry stakeholders.
CONCLUSIONS: Partnering with KOLs adds rigor and credibility to evidence generation workstreams, including HEOR and Medial Affairs; leveraging AI tools offers a new opportunity to identify KOLs. Future research should focus on validating AI-driven methodologies in real-world settings, assessing their impact on rare disease research and policy outcomes, and addressing ethical considerations to ensure transparency and inclusivity.
Predictive Modeling Transition to PsA in High-Risk PsO Patients to Enable Early Intervention
Poster Session 2 / Poster Code MSR58
Wednesday, May 14 | 4:00 – 7:00 PM
OBJECTIVES: This study leveraged advanced analytical methodologies to identify patients at high risk of transitioning from Psoriasis (PsO) to Psoriatic Arthritis (PsA), with the aim of improving early detection and intervention and to enable tailored treatment strategies.
METHODS: Merative MarketScan payer sourced claims data from 2019-2022 was leveraged and patients with advanced PsO treatments were identified. Survival analysis using Kaplan-Meier curves and predictive modeling via Cox Regression and Random Survival Forest (RSF) were conducted. Chi-Square tests of independence identified key predictors, while RSF provided risk scores for patients likely to progress to PsA. Predictors encompassed demographic factors, treatment pathways (e.g., Interleukin inhibitors), comorbidities, and ICD code groupings. Model validation assessed proportional hazard assumptions and predictive accuracy.
RESULTS: Findings revealed that most PsA cases emerge within the first year of advanced PsO treatment initiation, with significant predictors including Anti-TNFand Interleukin therapies. Brand switching peaked in the initial two years, underscoring critical early intervention opportunities. RSF outperformed other models, accurately stratifying patients by progression risk. Additionally, insights from survival analysis highlighted the role of comorbidities and procedural codes in accelerating PsA development.
CONCLUSIONS: This study demonstrates the value of predictive modeling in identifying patients at high-risk of transitioning from PsO to PsA. Such models can drive targeted drug development, optimize clinical trial design, and facilitate precision medicine approaches. Future work should integrate broader datasets to validate findings and explore cost-benefit analyses for early intervention strategies.
Real-World Characteristics and Treatment Patterns With Nintedanib in Patients With Idiopathic Pulmonary Fibrosis: A Retrospective US Claims Study
Poster Session 4 / Poster Code RWD114
Thursday, May 15 | 4:00 – 7:00 PM
OBJECTIVES: Idiopathic pulmonary fibrosis (IPF) is a rare and life-threatening condition leading to a progressive decline in lung function. This study aimed to describe the characteristics of four treatment sub-cohorts of the IPF population.
METHODS: A retrospective, descriptive claims analysis was conducted using Komodo Healthcare Map™ cost of care data between 1st
January 2019-31st December 2023. Patients were included in the study at the start of specific dosage of nintedanib or IPF diagnosis in the study period with 12-months of continuous enrollment pre-/post-index date. Descriptive analyses were conducted in the post index period across four comparator cohorts: nintedanib 100mg BID, nintedanib 150mg BID, nintedanib discontinued for 12-months, and untreated with any IPF Therapy.
RESULTS: We identified 10,877 patients with IPF meeting study criteria: 10% were treated with nintedanib 150mg, 5% were treated with nintedanib 100mg, 8% discontinued nintedanib (either dose) for 12 months and a vast majority (79%) were untreated with any IPF therapy. Overall mean age ranged between 68-72 years, with those on nintedanib 150mg being younger (mean=68 years) compared to the other three cohorts. Patients receiving nintedanib 150mg skewed male (72%) compared to the other cohorts (52-58%). Comorbidity rates were high across cohorts with a high prevalence of cardiovascular conditions (~70%-80%) and GERD (49%-58%). Untreated and discontinued patients had higher Charlson Comorbidity Index (CCI) scores (~2.8), compared to treated patients (~2.15). Patients were comparable in other characteristics, such as region and payer mix across cohorts, except nintedanib 150mg patients who had a higher rate of Commercial insurance (36%) compared to other cohorts (20%-23%).
CONCLUSIONS: Despite known morbidity and mortality of IPF and availability of therapy for nearly a decade, there remains a very large untreated population. Understanding patient characteristics across treatment groups in a real-world setting can potentially impact treatment decisions in patients with IPF.
Systematic Literature Review of Unmet Needs and Burden of Disease in Generalized Anxiety Disorder (GAD)
Poster Session 1 / Poster Code SA5
Wednesday, May 14 | 10:30 AM – 1:30 PM
Authors: Erin Ferries, PhD, MPH1*, Susan Suponcic, PhD2, Sophia Chung3, Sayak Bhattacharya3, Aishwarya Kulkarni, MS3, Mason Yeh, PhD3, Abigail Silber, MPH3, Matt O’Hara, MBA3, Phong Duong, PharmD1
1Mindmed Inc.
2 Value and Access Advisors, LLC
3 Trinity Life Sciences
*Presenting author
OBJECTIVES: The prevailing consensus suggests patients with General Anxiety Disorder (GAD) experience substantial burden of illness and impact on quality of life (QoL). However, gaps remain in understanding the holistic impact of GAD. Our aim is to conduct a systematic literature review (SLR) to assess clinical, humanistic and economic burden of GAD.
METHODS: An ongoing SLR using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) checklist will identify peer-reviewed manuscripts and conference abstracts published in English from November 2004 to December 2024 in PubMed, MEDLINE, and Embase. An Artificial Intelligence (AI)-enhanced platform is used to facilitate the review process. The validated AI screening platform was trained on the first 50 title/abstract screenings and further validated during the course of ongoing screening. Title/abstract screening with one human and one AI screener provides reviewer-level decisions with adjudication by another independent human. Full-text screening will be conducted by three independent, human reviewers.
RESULTS: A total of 27,173 articles were identified in PubMed (N=13,989) and Embase (N=18,807). Deduplication of records in EndNote resulted in 18,759 articles. Full-text screening is ongoing and has identified, to date, a wide evidence base supporting the holistic burden of GAD. The count of papers (non-mutually exclusive) identified by key topics of interest include, but are not limited to: GAD clinical trials (N=516); observational studies (N=202); epidemiological studies (N=101); QoL studies (N=553); patient reported outcomes (PROs) (N=221); economic impact (N=37); comorbidities (N=732); treatment response (N=177) and impact on functioning / ADLs (N=44). The full complement of data will be available for presentation in May 2025.
CONCLUSIONS: To the authors’ knowledge, this is the first SLR, spanning 20 years of published literature, to elucidate and enumerate gaps in epidemiology, diagnosis criteria, burden of disease, treatment outcomes and unmet needs that are specific to GAD.
FUNDING: This study is funded by MindMed.
The Economic Burden of Illness for Patients with Idiopathic Pulmonary Fibrosis Utilizing Treatment Cohorts: A US Retrospective Claims Study
Poster Session 3 / Poster Code RWD81
Thursday, May 15 | 10:30 AM – 1:30 PM
OBJECTIVES: Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive disease associated with high morbidity and mortality. This study aimed to understand burden of illness (BOI) and impact of treatment dosage and adherence on healthcare resource utilization (HCRU).
METHODS: A retrospective, descriptive claims analysis was conducted using Komodo Healthcare Map™ cost of care data between 1st
January 2019-31st December 2023. Patients were included in the study at start of specific dosage of nintedanib or IPF diagnosis in the study period with 12-months of continuous enrollment pre-/post-index date. Descriptive analyses were conducted in the post index period across four comparator cohorts: nintedanib 100mg BID, nintedanib 150mg BID, nintedanib discontinued for 12-months, and untreated with any IPF therapy.
RESULTS: We identified 10,877 patients with IPF meeting study criteria, 10% were treated with nintedanib 150mg, 5% with nintedanib 100mg, 8% were nintedanib discontinued (either dose) for 12 months, and a vast majority (79%) were untreated with any IPF therapy. A higher proportion of the 12-month discontinued/untreated patients were hospitalized compared to those who are actively treated with nintedanib 100/150mg (35% & 31% vs 14% &16%). A higher proportion of 12-month treatment discontinued patients underwent lung biopsies compared to nintedanib 150/100mg (38%, 26%, 25% respectively) and received higher rates of oxygen support (69%, 60%, 59% respectively) suggesting that patients who discontinued nintedanib required ongoing supportive care.
CONCLUSIONS: In this real-world cohort, a large proportion of patients diagnosed with IPF remained untreated despite known efficacy of IPF therapy in reducing the rate of decline in lung function. We further observed a high rate of oxygen therapy and hospitalization among patients discontinuing treatment, highlighting a gap in the care paradigm. Further research is needed to understand this treatment gap, despite high BOI and an increasing need for medical support in patients with IPF.
The Impact of Glucocorticoid (GC) Dose on Clinical Outcomes in Congenital Adrenal Hyperplasia (CAH): A Systematic Literature Review (SLR)
Poster Session 1 / Poster Code CO16
Wednesday, May 14 | 10:30 AM – 1:30 PM
Using Machine Learning to Estimate Perceptions and Future Prescribing Intentions of Health Care Providers: A Novel Application of Integrated Primary and Secondary Data in Understanding HCP Decision-Making
Poster Session 3 / Poster Code MSR70
Thursday, May 15 | 10:30 AM – 1:30 PM
OBJECTIVES: This study is designed to demonstrate a novel application of machine learning to estimate healthcare provider (HCP) perceptions and future prescribing intentions, enriching administrative claims data with survey data.
METHODS: A representative sample of HCPs from a large US claims database was surveyed on their perceptions (e.g., preference for treatment attributes, impact of practice setting on decisions, etc.) and intended prescribing behaviors towards pipeline therapies within a specific therapeutic area. Survey responses were linked to longitudinal prescribing records in the claims data using NPIs. Machine learning models (Random Forest, AdaBoost, GBM, XGBoost, LightGBM, CatBoost) were trained on this linked dataset to classify HCPs based on associations between survey responses and historical claims data; survey metrics were estimated using claims data metrics. Model performance was assessed using a hold-out test set, evaluating accuracy, precision, recall, and F1-score. The final models for each survey metric to be estimated were applied to the full claims database to predict perceptions and intentions for non-surveyed HCPs.
RESULTS: This is an ongoing study, and results are expected in advance of the conference. Model performance metrics and descriptive statistics for predicted survey metrics will be reported. Associations between predicted perceptions/intentions and observed prescribing patterns in the full claims dataset will be assessed to evaluate the validity of the predictions. Further analyses will explore the distribution of predicted perceptions and intentions across HCP subgroups defined by practice setting and past prescribing behavior.
CONCLUSIONS: Administrative claims databases are a valuable source of real-world data for health outcomes research, but they lack information on HCP perceptions and prescribing intentions that are often critical to understand and characterize observed behaviors. This approach helps enrich claims data with estimated HCP perceptions and future intentions. This has the potential to elucidate rationale behind HCP decision-making and help improve effectiveness of medical education.