Pfizer Employee Research Articles

P-681. Estimated Incidence of Respiratory Syncytial Virus (RSV)-related Hospitalizations for Acute Respiratory Infections (ARIs), including Community Acquired Pneumonia (CAP), in Adults in Germany

Abstract Background RSV is a leading cause of ARI, including CAP, in older adults, but available data are limited and often substantially underestimate incidence. Among adults, single nasopharyngeal (NP) swab testing misses about half of RSV infections compared to using multiple specimen types. We estimated RSV-related ARI hospitalization incidence from a prospective CAP study and adjusted for undiagnosed RSV infections due to use of NP swab testing only. Methods We conducted an active, population-based multicenter surveillance of adult CAP hospitalizations in Thuringia (Germany) between 2021–2023. Participant NP swabs were RSV-tested by multiplex nucleic acid amplification tests. To estimate unadjusted RSV-related CAP incidence, age-group specific proportions of RSV detection among tested patients were applied to all-cause CAP incidence. Adjustments for underdiagnosis due to single site sampling and exclusion of non-CAP ARI were based on previous findings from a large, prospective, multispecimen study assessing the impact of collecting multiple specimens (NP swab, saliva, paired serology, and sputum) among 3,669 adults hospitalized for ARI. Results Among 1,040 radiologically confirmed CAP adults enrolled, 38 tested positive for RSV based on NP swabs, for an overall positive proportion of 3.7% among adults aged ≥18 years. Positive proportion increased to 7.8% after adjusting for higher RSV detection with multispecimen sampling compared to NP swab sampling only. The adjusted annual RSV-related CAP hospitalization rates were 4.7 (95% CI 1.5, 11.2) and 109.1 (95% CI 89.6, 131.6) per 100,000 population in adults aged 18-59 and ≥60 years, respectively. As CAP comprises only 28% of RSV-related ARI, the adjusted annual incidence of RSV-related ARI was 18.4 (95% CI 11.0, 28.9) and 377.6 (95% CI 340.5, 417.7) per 100,000 population for adults 18-59 and ≥60 years, respectively (Table1). Incidence estimates in Period 2 were about 3 times higher than in Period 1. Conclusion Older adults in Germany face a high burden of RSV-related ARI hospitalizations, including CAP, underscoring the potential utility of RSV vaccination for this population. These results align with recent time-series incidence analysis from Germany (236–363/100,000 for adults ≥60 years). Disclosures Caihua Liang, MD, PhD, Pfizer: Stocks/Bonds (Private Company) Elizabeth Begier, MD, M.P.H., Pfizer Vaccines: Employee|Pfizer Vaccines: Stocks/Bonds (Private Company) Liz Wang, MD; MS Biostatistics, Pfizer: employee|Pfizer: Stocks/Bonds (Public Company) Claudia Schwarz, PhD, Pfizer Inc.: employee|Pfizer Inc.: Stocks/Bonds (Private Company) Lea Johanna Bayer, n/a, Pfizer Pharma: Employee Christof Von Eiff, n/a, Pfizer Pharma: I am working as Sen. Medical Direktor for Vaccines at Pfizer Pharma GmbH, Berlin, , Germany and in that position I also get stocks. Qing Liu, M.S., Pfizer Inc.: Stocks/Bonds (Public Company) Jo Southern, PhD, MSc, Pfizer: Employee|Pfizer: Stocks/Bonds (Private Company) Jeffrey T. Vietri, PhD, Pfizer Inc.: Employment|Pfizer Inc.: Stocks/Bonds (Public Company) Bradford D. Gessner, M.D., M.P.H., Pfizer: Employee|Pfizer: Stocks/Bonds (Public Company) Christian Theilacker, MD, DTM&H, Pfizer Inc: I am a Pfizer Employee|Pfizer Inc: Stocks/Bonds (Public Company)

Abstract PO4-17-04: CDK 4/6 inhibitor switching patterns in Swedish patients with metastatic breast cancer: 5-year update from the SIRI study

Abstract Background: CDK 4/6 inhibitors (CDK 4/6i) combined with endocrine therapy is a well-established treatment option in patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer (MBC). However, there is limited evidence on switching patterns within this class of drugs. The Swedish Ibrance Registries Insights (SIRI) study investigated CDK 4/6i switching patterns in real-world setting using a nationwide cohort of MBC patients. Methods: This was a retrospective study utilizing population-based Swedish Health Data Registers. The overall cohort included all breast cancer patients ≥ 18 years with ≥ 1 dispensation of palbociclib from January 2017 – June 2022. This subgroup analysis focused on patients with ≥ 1 dispensation of either ribociclib or abemaciclib in addition to palbociclib. Minimum follow-up was 3 months. CDK 4/6-I sequencing patterns in total and over time as well as time from the first CDK 4/6i to the subsequent, was investigated. No information on the reason for switching was available. Results: Out of a total 2314 patients with ≥ 1 dispensation of palbociclib, 256 patients (11%) had ≥ 1 dispensation of either ribociclib or abemaciclib, either prior to (60% of cases) or following (40 % of cases) palbociclib treatment. The share of patients with dispensation of > 1 CDK 4/6i increased over the study period from 7% of the patients initiating treatment in 2017 to 15% in 2021. Of the total 2161 patients initiating CDK 4/6i treatment on palbociclib, about 5% were subsequently prescribed another CDK 4/6i. The median age at treatment initiation in patients with > 1 dispensed CDK 4/6-i was similar to the overall study cohort (67.1 vs 68.4 years). Half of the patients identified with > 1 CDK 4/6i were initiated on ribociclib and prescribed palbociclib subsequently. The second and third most common switches were palbociclib to abemaciclib (25%) and palbociclib to ribociclib (10%), respectively. Seven patients received all three CDK 4/6i. In terms of time to subsequent CDK 4/6i, 108 patients (42%) were prescribed a subsequent CDK 4/6i within three months of treatment initiation with ribociclib-palbociclib being the most common sequence in 74 patients whereas 17%, 8%, and 4% of switches happened 4-6, 7-9, and 10-12 months after treatment initiation, respectively. Seventy-two patients (28%) had a dispensation of a subsequent CDK 4/6i more than 12 months after initiation of the first CDK 4/6i, with palbociclib-abemaciclib being the most common switch. Conclusions: A relatively high proportion of Swedish patients treated with CDK4/6i is switched from one to another CDK 4/6i soon after treatment initiation, implying that this strategy might be applied mainly, but not solely, due to adverse events. The increased trend over time might be associated with the regulatory approval of new CDK4/6i over time. Future studies should focus on the potential impact of switching CDK 4/6i on treatment effectiveness and toxicity as these issues have yet not been clarified. Declaration of Interest: This study is sponsored by Pfizer. AV has reported receiving research funding from Roche. HL has reported receiving consultant/advisory fees from Lilly, Novartis, Daichii, Pfizer, MSD, Pierre Fabre, Astra-Zeneca and research funding from Roche. MJ and DN are employees of Pfizer. RL and ML are employees of Quantify Research and were paid consultants to Pfizer for this research. Table. Table. Citation Format: Henrik Lindman, Antonis Valachis, Rosa Lauppe, Mathias Lilja, Daniel Nyqvist, Maria Jakobsson. CDK 4/6 inhibitor switching patterns in Swedish patients with metastatic breast cancer: 5-year update from the SIRI study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO4-17-04.

P126 Use of oral contraceptives in females with rheumatoid arthritis is not associated with an increased risk of venous thromboembolism: United Kingdom-population based study

Abstract Background/Aims While the use of oral contraceptives in females with immune-mediated inflammatory diseases such as rheumatoid arthritis (RA) is acknowledged as a risk factor for venous thromboembolism (VTE), the interaction between oral contraceptive use and RA is poorly understood. In a large population-based study, we aimed to estimate RA-attributable VTE risk in females using oestrogen contraceptives and/or on hormone replacement therapy (HRT). Methods Females registered with a general practice from January 1999 to December 2018 were identified from the Royal College of General Practitioners Research (RCGP) and Surveillance Centre (RSC) database. Female RA patients and VTE outcome (composite of pulmonary embolism and deep vein thrombosis) were determined using previously-validated algorithms. Female unaffected controls (UCs) were matched 4:1 with RA patients by current age (per year), calendar time, and years since practice registration using nearest neighbour matching, with replacement. Absolute VTE rates over 20 years were compared in RA patients versus matched UCs, overall and in two different subgroups defined by the use of oestrogen contraceptives or HRT. Relative VTE risk over the same period was estimated using Cox proportional hazards models adjusted for sociodemographic and clinical features and established VTE risk factors (BMI [body-mass index], smoking status, alcohol use, evidence of reduced mobility, thrombophilia, fracture of the lower limb and family history of VTE). Results A total of 82,082 female adults were included in this study, of whom 16,634 were RA patients and 65,448 were UCs. Average study follow-up was 8.3 years (standard deviation [SD]=6.2 years). Female RA patients (mean age 58.3, SD 16.5; mean BMI 27.0, SD 5.9) were similar to matched UCs in their clinical characteristics. Compared to UCs, females with RA were less likely to receive oral contraceptives (RA n = 545 [3.3%], UCs n = 2,542 [3.9], p < 0.001) but more likely to receive HRT (RA n = 752 [4.5%], UCs n = 2,460 [3.8], p < 0.001). Unadjusted VTE events rates were consistently higher in females with RA compared to UCs (430.2 per 100,000 person-years [py], 95% confidence interval [CI]: 396.1, 466.3; UC: 260.5 per 100,000 py, 95%CI: 247.1, 274.4) and in those receiving and not receiving oral contraceptives and HRT. Overall, relative risk of VTE was 52% higher in females with RA compared UCs (adjusted hazard ratio [aHR]= 1.52, 95%CI: 1.36, 1.71, p < 0.001). Compared to UCs, relative risk increases were not statistically significant for females with RA receiving oral contraceptives (aHR 1.43; 95%CI 0.60, 3.63, p = 0.46) and HRT (aHR 2.32; 95%CI 0.92, 5.85, p = 0.08). Conclusion UK data suggest that all women with RA are at a similarly increased risk of VTE irrespective of the use of oestrogen contraceptives or HRT, although larger studies are needed to confirm this finding. It is therefore advisable to conduct routine VTE risk factor assessments for all females with RA. Disclosure J. Galloway: Honoraria; JG has received honoraria from Abbvie, Biovitrum, BMS, Celgene, Chugai, Gilead, Janssen, Lilly, Novartis, Pfizer, Roche, Sanofi, Sobi and UC. V. Basey: Corporate appointments; Pfizer employee. A. Barkaway: Corporate appointments; Pfizer employee. S. de Lusignan: Grants/research support; SdeL has received funding through his universities from Eli Lilly, GSK, Astra Zeneca, MSD, Sanofi, Seqirus, Takeda and Moderna. Other; Director of the RCGP RSC. M.H. Buch: Consultancies; M.H.B. has provided expert advice and received/paid to employer consultant fees from AbbVie, Eli Lilly, EMD serono, Gilead, Pfizer and Sanofi. Grants/research support; .H.B. has received research grants paid to her employer from Gilead Ltd, Pfizer Ltd, Roche and UCB.

P052 Rheumatoid arthritis associated risk of venous thromboembolism is not dependent on disease duration: United Kingdom-based population study

Abstract Background/Aims Risk of venous thromboembolism (VTE) is higher in patients with rheumatoid arthritis (RA) compared to the general population; however, whether this risk changes with RA disease duration remains poorly understood. Acknowledging potential heterogeneity in risk by RA duration could support more tailored screening and effective preventive approaches. In a large population-based study, we aimed to characterise the RA-attributable VTE risk in patients with varying disease durations. Methods Adults registered with a general practice from January 1999 to December 2018 were identified from the Royal College of General Practitioners Research (RCGP) and Surveillance Centre (RSC) primary care database. RA patients (diagnosed prior to and during the study period), VTE outcome (as a composite of pulmonary embolism [PE] or deep vein thrombosis [DVT]) and individual PE and DVT outcomes were determined using previously validated algorithms. RA-unaffected controls (UCs) were matched 4:1 with RA patients by current age, sex, calendar time, and years since practice registration using nearest neighbour matching with replacement. Absolute VTE, PE and DVT rates over 20 years were compared in RA patients versus matched UC across four subgroups defined by disease duration (0-2, 2-5, 5-10 and ≥10 years since diagnosis). Across the same subgroups, relative risks over the same period were estimated using Cox proportional hazards models adjusted for sociodemographic and clinical features and established VTE risk factors (body-mass index [BMI], smoking status, alcohol use, evidence of reduced mobility, thrombophilia, fracture of the lower limb and family history of VTE). Results 117,050 individuals were included, of whom 93,640 were UCs and 23,410 were RA patients. Among those with RA, 63.9% were included at or within two years of diagnosis, 7.8% within two to five years of diagnosis, 9.8% within five to ten years of diagnosis, and 18.5% more than 10 years after diagnosis. Average study follow-up for the primary outcome (VTE) was 8.2 years (standard deviation [SD]=6.6 years). RA patients (mean age 59.0, SD 15.5; 28.9% male [n = 6776]; mean BMI 27.1, SD 5.6) were similar to matched UCs in clinical characteristics. Unadjusted VTE events rates were consistently higher in RA patients compared to UCs across all subgroups. Relative risk of VTE was similarly increased in RA patients compared to the general population regardless of the time elapsed since RA diagnosis (adjusted hazard ratios [aHR] range: 1.49-1.63, all p < 0.001). The results obtained when PE (aHR range 1.46-2.02, all p < 0.001) and DVT (aHR range 1.43-1.89, all p < 0.001) were analysed separately were similar to those observed in the composite outcome. Conclusion Healthcare professionals should recognize that individuals with RA face a consistently elevated VTE risk compared to the general population, irrespective of disease duration. This highlights the need for vigilant monitoring and assessment of VTE risk factors even in early-stage cases. Disclosure J. Galloway: Honoraria; JG has received honoraria from Abbvie, Biovitrum, BMS, Celgene, Chugai, Gilead, Janssen, Lilly, Novartis, Pfizer, Roche, Sanofi, Sobi and UC. V. Basey: Corporate appointments; Pfizer employee. S. Rana: Corporate appointments; Pfizer employee. S. de Lusignan: Grants/research support; SdeL has received funding through his universities from Eli Lilly, GSK, Astra Zeneca, MSD, Sanofi, Seqirus, Takeda and Moderna. Other; Director of the RCGP RSC. M.H. Buch: Consultancies; M.H.B. has provided expert advice and received/paid to employer consultant fees from AbbVie, Eli Lilly, EMD serono, Gilead, Pfizer and Sanofi. Grants/research support; M.H.B. has received research grants paid to her employer from Gilead Ltd, Pfizer Ltd, Roche and UCB.

OA38 Venous thromboembolism risk is consistently increased in people with rheumatoid arthritis across different ages, sexes and obesity status: United Kingdom population-based study

Abstract Background/Aims Risk of venous thromboembolism (VTE) is increased in people with rheumatoid arthritis (RA) when compared to the general population, but the variation of this risk among patients is not well understood. Acknowledging the heterogeneity in RA-attributable VTE risk could support tailoring of treatment plans and screening protocols based on an individual’s specific risk profile. In a large population-based study, we aimed to evaluate for variation in RA-attributable VTE risk by age, sex and body-mass index (BMI). Methods Adults registered with a general practice from January 1999 to December 2018 were identified from the Royal College of General Practitioners Research (RCGP) and Surveillance Centre (RSC) primary care database. RA patients and VTE outcome (composite of pulmonary embolism and deep vein thrombosis) were determined using previously validated algorithms. RA-unaffected controls (UCs) were matched 4:1 with RA patients by current age, sex, calendar time, and years since practice registration using nearest neighbour matching with replacement. Absolute VTE rates over 20 years were compared in RA patients versus matched UCs, overall and by age category (18-49, 50-69, ≥70), sex, and BMI category (BMI <25, 25-29.9, ≥30). Across the same subgroups, relative VTE risk over the same period was estimated using Cox proportional hazards models adjusted for sociodemographic and clinical features and established VTE risk factors (BMI, smoking status, alcohol use, evidence of reduced mobility, thrombophilia, fracture of the lower limb and family history of VTE). Results 117,050 individuals were included, of whom 23,410 were RA patients and 93,640 were UCs. Average follow-up was 8.2 years (standard deviation [SD]=6.6 years). RA patients (mean age 59.0, SD 15.5; 28.9% male [n = 6776]; mean BMI 27.1, SD 5.6) were similar to matched UCs in clinical characteristics. Unadjusted VTE events rates were consistently higher in RA patients compared to UCs overall and across all subgroups (overall rates RA: 442.4 per 100,000 person-years [py], 95% confidence interval [CI]: 413.0, 473.2; UC: 262.2 per 100,000 py, 95%CI: 251.9, 273.9); subgroup rates). Similarly, overall relative VTE risk was 46% higher in people with RA compared to UCs (adjusted hazard ratio [aHR]= 1.46, 95%CI: 1.36, 1.56; p < 0.001), and was consistently higher across age-, sex- and BMI-defined subgroups (aHR range: 1.34-2.13, all p < 0.001). Although relative risk was highest in RA patients under 50 years (aHR= 2.13, 95%CI: 1.62-2.79; p < 0.001), this patient subgroup had the lowest absolute risk of VTE (approximately 2.2- and 3.4-fold lower than that observed in those aged 50-69 and ≥70, respectively). Conclusion Clinicians should be aware that risk of VTE is consistently higher in people with RA compared to the general population, regardless of age, sex and BMI. Findings suggest close monitoring and assessment of VTE risk factors is required even in younger individuals with RA. Disclosure J. Galloway: Honoraria; JG has received honoraria from Abbvie, Biovitrum, BMS, Celgene, Chugai, Gilead, Janssen, Lilly, Novartis, Pfizer, Roche, Sanofi, Sobi and UC. V. Basey: Corporate appointments; Pfizer employee. M. Mclean: Corporate appointments; Pfizer employee. S. de Lusignan: Grants/research support; SdeL has received funding through his universities from Eli Lilly, GSK, Astra Zeneca, MSD, Sanofi, Seqirus, Takeda and Moderna. Other; Director of the RCGP RSC. M.H. Buch: Consultancies; M.H.B. has provided expert advice and received/paid to employer consultant fees from AbbVie, Eli Lilly, EMD serono, Gilead, Pfizer and Sanofi. Grants/research support; M.H.B. has received research grants paid to her employer from Gilead Ltd, Pfizer Ltd, Roche and UCB.

2650. Characteristics of In-Hospital Care and Immediate Post-Discharge Care Status among Older Adults Hospitalized with Respiratory Syncytial Virus Infection, Seasonal Influenza, Acute Myocardial Infarction, and Stroke in the US

Abstract Background Despite increased recognition of the morbidity, mortality, and economic burden of respiratory syncytial virus (RSV) in adults, few studies have documented the status of post-discharge care after RSV hospitalization. This study assessed in-hospital and immediate post-discharge care status in older adults hospitalized with RSV in the US and compared it with other serious medical conditions (influenza, acute myocardial infarction (AMI), and stroke) that have long-standing disease prevention efforts. Methods This retrospective cohort study used data from Premier Healthcare Database between January 1, 2015, to December 31, 2019. Adults aged ≥ 65 years hospitalized due to primary diagnosis of RSV, influenza, AMI, or stroke were included. Discharge statuses were used to categorize the levels of immediate post-discharge care as shown in Figure 1. Descriptive statistics and Sankey diagrams were used to summarize the four cohorts. Results Overall, 2,559 RSV, 174,826 influenza, 208,448 AMI, and 274,102 stroke hospitalizations were identified with similar mean patient ages of 79.8, 76.8, 76.3, and 78.0 years, respectively. Almost all patients ( > 90%) had underlying comorbidities. The mean inpatient length of stay (LOS) was similar in the four cohorts (4.0, 4.4, 3.7, and 4.1 days, respectively), but the prevalence of mechanical ventilator use varied, i.e., 0.4%, 13.7%, 5.9%, and 1.4%, respectively. Intensive Care Unit (ICU) admissions during RSV hospitalizations were infrequent (3.0%) as compared to influenza (16.8%), AMI (24.5%), and stroke (20.5%). The mean ICU LOS for these four cohorts were 2.3, 2.8, 1.7, and 1.7 days in the same order. Immediately following discharge, moderate care was required for 23.9%, 22.9%, 14.1%, and 17.5% of RSV, influenza, AMI, and stroke hospitalizations, respectively. Elevated care was considerably more frequent for stroke hospitalizations (48.1%) than RSV (18.8%), influenza (21.4%), and AMI (19.3%) hospitalizations (Figure 2). Conclusion Older adults discharged from RSV hospitalizations require considerable care with needs comparable to influenza and AMI discharges. Given the substantial care requirements of RSV hospitalizations in older adults, focus on disease prevention efforts for RSV are needed. Disclosures Reiko Sato, PhD, Pfizer Inc: employee|Pfizer Inc: Stocks/Bonds Jennifer Judy, MS, PhD, Pfizer: Stocks/Bonds Kari Yacisin, M.D., Pfizer: Employee|Pfizer: Stocks/Bonds Elizabeth Begier, M.D., M.P.H., Pfizer: EB is an employee of Pfizer, the sponsor of this study|Pfizer: Stocks/Bonds Poorva Sardana, MSc, Pfizer Inc: Contracted Research Neha Agrawal, MA, Pfizer Inc: Contracted Research Anchita Goswami, MSc, Pfizer Inc: Contracted Research Manvi Sharma, RPh, MBA, PhD, Pfizer Inc: Contracted Research

Development and electronic health record validation of an algorithm for identifying patients with Duchenne muscular dystrophy in US administrative claims.

BACKGROUND: Muscular dystrophies (MDs) comprise a heterogenous group of genetically inherited conditions characterized by progressive muscle weakness and increasing disability. The lack of separate diagnosis codes for Duchenne MD (DMD) and Becker MD, 2 of the most common forms of MD, has limited the conduct of DMD-specific real-world studies. OBJECTIVE: To develop and validate administrative claims-based algorithms for identifying patients with DMD and capturing their nonambulatory and ventilation-dependent status. METHODS: This was a retrospective cohort study using the statistically deidentified Optum Market Clarity Database (including patient claims linked with electronic health records [EHRs] data) to develop and validate the following algorithms: DMD diagnosis, nonambulatory status, and ventilation-dependent status. The initial study sample consisted of US patients in the database who had a diagnosis code for Duchenne/Becker MD (DBMD) between October 1, 2018, and September 30, 2020, who were male, aged 40 years or younger on their first DBMD diagnosis, and met continuous enrollment and 1-day minimal clinical activities requirement in a 12-month measurement period between October 1, 2017, and September 30, 2020. The algorithms, developed by a cross-functional team of DMD specialists (including patient advocates), were based on administrative claims data with International Classification of Diseases, Tenth Revision, Clinical Modifications coding, using information of diagnosis codes for DBMD, sex, age, treatment, and disease severity (eg, evidence of ambulation assistance/support and/or evidence of ventilation support or dependence). Patients who met each algorithm and had EHR notes available were then validated against structured fields and unstructured provider notes from their own linked EHR to confirm patients' DMD diagnoses, nonambulatory status, and ventilation-dependent status. Algorithm performance was assessed by positive predictive value with 95% CIs. RESULTS: A total of 1,300 patients were included in the initial study sample. Of these, EHR were available and reviewed for 303 patients. The mean age of the 303 patients was 14.8 years, with 61.7% being non-Hispanic White. A majority had a Charlson comorbidity index score of 0 (59.4%) or 1-2 (27.7%). Positive predictive value (95% CI) was 91.6% (85.8%-95.6%) for the DMD diagnosis algorithm, 88.4% (80.2%-94.1%) for the nonambulatory status algorithm, and 77.8% (62.9%-88.8%) for the ventilation-dependent status algorithm. CONCLUSIONS: This work provides the means to more accurately identify patients with DMD from administrative claims data without a specific diagnosis code. The algorithms validated in this study can be applied to assess treatment effectiveness and other outcomes among patients with DMD treated in clinical practice. DISCLOSURES: This study was funded by Pfizer, which contracted with Optum to perform the study and provide medical writing assistance. Ms Schrader reports being an employee of Parent Project Muscular Dystrophy. Mr Posner reports being an employee and stockholder of Pfizer and receiving support from Pfizer for attending conferences not related to this manuscript. Dr Dorling reports being an employee and stockholder of Pfizer at the time the study was conducted and is a current employee of Chiesi USA, Inc. Ms Senerchia reports being an employee of Optum and owning stock in Pfizer and UnitedHealth Group, the parent company of Optum. Dr Chen reports being an employee and stockholder of Pfizer. Ms Beaverson reports being an employee of Pfizer and owning stock in Pfizer and Amicus Therapeutics. Dr Seare reports being an employee of Optum at the time the study was conducted. Dr Garnier and Ms Merla report being employees of Pfizer. Ms Walker reports being an employee of Optum. Dr Alvir reports being an employee and stockholder of Pfizer. Dr Mahn reports being an employee and stockholder of Pfizer. Dr Zhang reports being an employee of Optum. Ms Landis reports being an employee of Optum. Ms Buikema reports being an employee of Optum and holding stock in UnitedHealth Group, the parent company of Optum.

Overview of alopecia areata for managed care and payer stakeholders in the United States.

Alopecia areata (AA) is an autoimmune disease with a complex pathophysiology resulting in nonscarring hair loss in genetically susceptible individuals. We aim to provide health care decision makers an overview of the pathophysiology of AA, its causes and diagnosis, disease burden, costs, comorbidities, and information on current and emerging treatment options to help inform payer benefit design and prior authorization decisions. Literature searches for AA were conducted using PubMed between 2016 and 2022 inclusive, using search terms covering the causes and diagnosis of AA, pathophysiology, comorbidities, disease management, costs, and impact on quality of life (QoL). AA is a polygenic autoimmune disease that significantly impacts QoL. Patients with AA face economic burden and an increased prevalence of psychiatric disease, as well as numerous systemic comorbidities. AA is predominantly treated using corticosteroids, systemic immunosuppressants, and topical immunotherapy. Currently, there are limited data to reliably inform effective treatment decisions, particularly for patients with extensive disease. However, several novel therapies that specifically target the immunopathology of AA have emerged, including Janus kinase (JAK) 1/2 inhibitors such as baricitinib and deuruxolitinib, and the JAK3/tyrosine kinase expressed in hepatocellular carcinoma (TEC) family kinase inhibitor ritlecitinib. To support disease management, a disease severity classification tool, the Alopecia Areata Severity Scale, was recently developed that evaluates patients with AA holistically (extent of hair loss and other factors). AA is an autoimmune disease often associated with comorbidities and poor QoL, which poses a significant economic burden for payers and patients. Better treatments are needed for patients, and JAK inhibitors, among other approaches, may address this tremendous unmet medical need. DISCLOSURES: Dr King reports seats on advisory boards for and/or is a consultant and/or clinical trial investigator for AbbVie, Aclaris Therapeutics Inc, AltruBio Inc, Almirall, Arena Pharmaceuticals, Bioniz Therapeutics, Bristol Meyers Squibb, Concert Pharmaceuticals Inc, Dermavant Sciences Inc, Eli Lilly and Company, Equillium, Incyte Corp, Janssen Pharmaceuticals, LEO Pharma, Otsuka/Visterra Inc, Pfizer, Regeneron, Sanofi Genzyme, TWi Biotechnology Inc, and Viela Bio; and speakers bureaus for AbbVie, Incyte, LEO Pharma, Pfizer, Regeneron, and Sanofi Genzyme. Pezalla is a paid consultant to Pfizer for market access and payer strategy concerns; Fung, Tran, Bourret, Takiya, Peeples-Lamirande, and Napatalung are employees of Pfizer and hold stock in Pfizer. This article was funded by Pfizer.

Clinical characteristics and health care resource use of patients at risk for wild-type transthyretin amyloid cardiomyopathy identified by machine learning model

BACKGROUND: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening systemic disorder that is an underrecognized cause of heart failure (HF). When the diagnosis of wild-type ATTR-CM (ATTRwt-CM) is delayed, patients often undergo additional assessments, deferring appropriate management as symptoms potentially worsen. Prompt recognition of patients at risk for ATTRwt-CM is essential to facilitate earlier diagnosis and disease-modifying treatment. A previously developed machine learning model performed well in identifying ATTRwt-CM in patients with HF vs controls with nonamyloid HF using medical claims/electronic health records, providing a systematic framework to raise disease suspicion. OBJECTIVE: To further evaluate this model's performance in identifying ATTRwt-CM using a large claims database of older adults with HF and confirmed ATTRwt-CM or nonamyloid HF; and to explore the characteristics and health care resource utilization (HCRU) of patients with confirmed and suspected ATTRwt-CM. METHODS: In this retrospective study, the prior model was applied using Humana administrative claims for patients diagnosed with ATTRwt-CM (cases) and nonamyloid HF (controls [1:1]). Patients were aged 65-89 years, had at least 2 claims for HF diagnosis (2015-2020), and were continuously enrolled in a Medicare Advantage prescription drug plan for at least 12 months before and at least 6 months after HF diagnosis. For the assessment of characteristics and HCRU, the suspected risk level was categorized based on the predicted probability (PP) from model output (high, moderate, and low risk: PP≥0.70; ≥0.50 and < 0.70; and < 0.50, respectively). RESULTS: Of 267,025 eligible patients, 119 (0.04%) had confirmed ATTRwt-CM; of 266,906 patients with nonamyloid HF, 10,997 (4.1%), 68,174 (25.5%), and 187,735 (70.3%) were categorized as high, moderate, and low risk for ATTRwt-CM, respectively. The model demonstrated sensitivity/specificity/accuracy/receiver operating characteristic area under the concentration-time curve of 88%/65%/77%/0.89, respectively, in differentiating ATTRwt-CM from nonamyloid HF. In patients with confirmed ATTRwt-CM, the mean (SD) time between HF and ATTRwt-CM diagnoses was 751 (528) days; 65% and 48% were hospitalized before and after ATTRwt-CM diagnosis, respectively. Atrial fibrillation was more common in patients with confirmed ATTRwt-CM and high risk (39% and 55%) vs low risk (27%). Hospitalization and emergency department visits after HF diagnosis were reported in 57% and 46% of patients with high ATTRwt-CM risk, respectively. CONCLUSIONS: The ATTRwt-CM predictive model performed well in identifying disease risk in the Humana Research Database. Patients at high risk for ATTRwt-CM had high HCRU and may benefit from the earlier suspicion of ATTRwt-CM. The model may be used as a tool to identify patients with a suspected high risk for the disease to facilitate earlier detection and treatment. DISCLOSURES: This study was sponsored by Pfizer. Medical writing support was provided by Donna McGuire of Engage Scientific Solutions and funded by Pfizer. Drs Bruno and Schepart and Mr Casey are currently employees of Pfizer and equity holders in this publicly traded company. Dr Reed was an employee of Pfizer at the time that this analysis was planned and conducted. Mr Sheer and Dr Simmons are currently employees of Humana, which received research funding from Pfizer. Dr Nair was an employee of Humana at the time that this analysis was planned and conducted.

In-lab Assessment of a Sternum-Worn Accelerometer to Measure Gait Speed in Healthy Individuals (P3-11.010)

<h3>Objective:</h3> To assess the validity of gait speed derived using a sternum-worn accelerometer, thus supporting the deployment of a single device to measure multiple biosignals, e.g., gait and ECG. <h3>Background:</h3> Gait assessment is essential to understand, diagnose, and monitor numerous neurological disorders. In contrast to conventional clinical evaluations, wearable sensors used in free-living environments grant more objective measurements. While wearing the widespread lumbar-worn accelerometers may be burdensome over the long-term, sternum-worn devices, already validated for at-home vitals monitoring, may be more comfortable. <h3>Design/Methods:</h3> During two in-lab visits, twenty healthy individuals (13 females, mean±sd age: 33.9±9.1 years, BMI: 24.4±4.67kg/m²) were instrumented with a 6-sensor set (APDM), including lumbar-worn and sternum-worn accelerometers. Participants underwent two assessments: A1) walking three 20-ft laps on an instrumented mat (GAITRite), at normal, slow, and fast speeds; A2) freely performing in-lab 20-min activities with frequent walking bouts. Gait speed was extracted by sternum-worn and lumbar-worn accelerometers (test devices) using the in-house built GaitPy algorithm, and compared against the values collected by GAITRite and APDM (reference devices for A1 and A2, respectively). Agreement was evaluated via Bland-Altman analysis, Pearson’s correlation (R), and intraclass correlation coefficient (ICC). <h3>Results:</h3> In A1, sternum-worn device underestimated gait speed compared to GAITRite, but the values were highly correlated, and the ICC showed moderate-to-good agreement (R: 0.91, 0.81, 0.66; bias (m/s): −0.137, −0.162, −0.209; ICC: 0.59, 0.50, 0.43, for slow, normal, and fast speeds, respectively). In A2, bias=0.055, R=0.82, and ICC= 0.78 demonstrated excellent agreement between gait speed derived from APDM and sternum-worn device. Sternum-worn and lumbar-worn accelerometers showed comparable performances. <h3>Conclusions:</h3> Sternum-worn devices can reliably measure gait speed both in scripted walking tasks and naturalistic walks. They can be deployed to simultaneously capture gait parameters and vital signs, thus opening new opportunities for more in-depth, free-living studies of patients with neurological disorders. <b>Disclosure:</b> Dr. Camerlingo has received personal compensation for serving as an employee of Pfizer. Mr. Welbourn has received personal compensation for serving as an employee of Mindlance, Inc. (employee servicing Pfizer). Mr. Psaltos has received personal compensation for serving as an employee of Pfizer. Mr. Messere has nothing to disclose. Dr. Zhang has received personal compensation for serving as an employee of Pfizer. Dr. Demanuele has received personal compensation for serving as an employee of Pfizer. Dr. Demanuele has stock in Pfizer. Dr. Santamaria Serra has nothing to disclose. Mr. Adamowicz has received personal compensation for serving as an employee of Pfizer. Mr. Adamowicz has stock in Pfizer. Dr. Karahanoglu has received personal compensation for serving as an employee of Pfizer. Dr. Cai has received personal compensation for serving as an employee of Pfizer. Dr. Cai has stock in Pfizer.

Patterns and Predictors of Multiple Sclerosis Phenotype Transitions: A Longitudinal Analysis Using the CLIMB Study (P5-3.018)

Patterns and Predictors of Multiple Sclerosis Phenotype Transitions: A Longitudinal Analysis Using the CLIMB Study (P5-3.018)

Low Dose Carbon Monoxide Is Neuroprotective in Models of Parkinson’s Disease (S42.008)

<h3>Objective:</h3> To evaluate the neuroprotective potential of low dose CO treatment in Parkinson disease (PD) models. <h3>Background:</h3> Despite the many risks of smoking tobacco, the risk of PD is markedly reduced among smokers and may derive from biological activity of specific tobacco smoke constituent(s). On the basis of accumulating evidence supporting the therapeutic potential of CO in other disease contexts, this study set out to determine whether low dose CO is neuroprotective in PD models. <h3>Design/Methods:</h3> In an AAV-alpha-synuclein (aSyn) model, rats underwent right nigral injection of AAV1/2-asynA53T and left injection of empty AAV, followed by treatment with oral CO drug product (HBI-002 10ml/kg, daily by gavage) or vehicle. In a short-term MPTP model (40mg/kg, i.p.), mice were treated with inhaled CO (iCO) (250ppm) or air. In an in vitro rotenone model, SH-SY5Y cells exposed to rotenone were treated with CO (200 ppm) or air. All analyses, including immunohistochemistry for nigral aSyn and tyrosine hydroxylase, HPLC measurement of striatal dopamine, stereological cell counting, and biochemical analyses were conducted blinded to treatment condition. <h3>Results:</h3> Each HBI-002 treatment increased carboxy-hemoglobin to 6%. In the aSyn model, treatment with HBI-002 reduced ipsilateral loss of both striatal dopamine and TH-positive neurons in the substantia nigra pars compacta, and HBI-002 reduced aSyn aggregates and aSyn S129 phosphorylation. In the MPTP model, treatment with low dose iCO also reduced loss of striatal dopamine and loss of TH+ neurons. In saline-treated mice, iCO had no effect on striatal dopamine or TH+ cell counts. HBI-002 upregulated heme oxygenase-1, HIF-1a, and cathepsin D. In the rotenone model, treatment with low dose CO reduced cell death. <h3>Conclusions:</h3> These results demonstrating reduced cell death and aSyn pathology in animal and cell models support the therapeutic potential of low dose CO for PD. <b>Disclosure:</b> Dr. Gomperts has received personal compensation in the range of $0-$499 for serving as a Consultant for EIP. Dr. Gomperts has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Jannsen. The institution of Dr. Gomperts has received research support from NIH. The institution of Dr. Gomperts has received research support from LBDA. The institution of Dr. Gomperts has received research support from DOD/CDMRP. The institution of Dr. Gomperts has received research support from FFFPRI. The institution of Dr. Gomperts has received research support from NIH. The institution of Dr. Gomperts has received research support from MJFF. The institution of Dr. Gomperts has received research support from NIH. The institution of Dr. Gomperts has received research support from NIH. The institution of Dr. Gomperts has received research support from MJFF. The institution of Dr. Gomperts has received research support from NIH. The institution of Dr. Gomperts has received research support from NIH. Dr. Rose has nothing to disclose. Dr. Zorlu has nothing to disclose. Dr. Xue has nothing to disclose. Dr. Cai has received personal compensation for serving as an employee of Pfizer . Ms. Lin has nothing to disclose. Mr. Lee has nothing to disclose. Dr. Gomperts has stock in Hillhurst Biopharmaceuticals inc. The institution of Dr. Gomperts has received research support from Hillhurst Biopharmaceuticals inc. Dr. Gomperts has received intellectual property interests from a discovery or technology relating to health care. The institution of Dr. Schwarzschild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bial Biotech (indirectly, as a service of the Parkinson Study Group service). The institution of Dr. Schwarzschild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen (indirectly, as a service of the Parkinson Study Group service). The institution of Dr. Schwarzschild has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB (indirectly, as a service of the Parkinson Study Group service). Dr. Schwarzschild has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Eli Lilly. The institution of Dr. Schwarzschild has received research support from NIH. The institution of Dr. Schwarzschild has received research support from Parkinson’s Foundation. The institution of Dr. Schwarzschild has received research support from Michael J Fox Foundation. The institution of Dr. Schwarzschild has received research support from Farmer Family Foundation. Dr. Schwarzschild has a non-compensated relationship as a Chair, Executive Committee with the Parkinson Study Group that is relevant to AAN interests or activities. Dr. Chen has nothing to disclose.

Longitudinal Clinical and MRI Outcomes in Relapsing Multiple Sclerosis (RMS) Patients After Short-term Ponesimod Treatment Interruption and Re-initiation (P6-3.007)

Longitudinal Clinical and MRI Outcomes in Relapsing Multiple Sclerosis (RMS) Patients After Short-term Ponesimod Treatment Interruption and Re-initiation (P6-3.007)

The Completion of a PGY-1 Neurology Bootcamp Leads to Increased Trainee Knowledge and Confidence in Neurologic Emergencies Management and Procedural Skills (P2-2.001)

<h3>Objective:</h3> To evaluate the impact of an end-of-the-year, post-graduate year 1 (PGY-1) simulation-based neurology bootcamp on trainee knowledge, self-confidence, and skill independence regarding acute neurologic emergencies management and bedside procedural skills. <h3>Background:</h3> The post-graduate year 2 (PGY-2) neurology resident is often the first line of triage for evaluation of acute neurologic deterioration on the inpatient neurology service, requiring autonomy and responsibility. We implemented and evaluated a bootcamp prior to this critical training period. <h3>Design/Methods:</h3> In this prospective cohort study, 14 rising PGY-2 neurology residents attended a two-day bootcamp with a simulation-based curriculum led by neurology faculty practicing cerebrovascular, critical care, and inpatient neurology medicine. Pre- and postbootcamp, trainees completed multiple-choice knowledge assessments and via Likert scales, self-reported confidence in managing neurologic emergencies. Faculty observed and rated trainee ability to independently perform five neurologic procedures (lumbar puncture, external ventricular drain [EVD] management, botulinum toxin injection for migraine, digital fundoscopy, and pupillometry), with residents additionally reporting their own perceived degree of independence. <h3>Results:</h3> Proportion of trainees demonstrating independence per faculty rating significantly increased for all five procedural skills post-bootcamp (for all, p &lt; .05). Trainee self-reported independence of EVD management, pupillometry, and fundoscopy skills also increased postbootcamp (for all, p &lt; .05), with &lt; 25% of trainees reporting total independence pre-bootcamp to 50%, 86%, and 79% post-bootcamp, respectively. Knowledge assessment scores increased from a pre-bootcamp mean of 54% of questions correct to mean 70% correct (p = 0.002), driven by increases in vascular neurologic emergency and procedural knowledge. On a 5-point Likert scale, trainee self-reported confidence in emergency management increased from median 2.5 (a little confident) to median 4 (fairly confident) (p &lt; .001) <h3>Conclusions:</h3> A simulation-based, two-day bootcamp with focus on acute cerebrovascular neurologic emergencies and neurologic procedural skills showed significant improvements in trainee knowledge along with faculty-observed and self-reported confidence and independence. <b>Disclosure:</b> An immediate family member of Dr. Swetlik has received personal compensation for serving as an employee of Pfizer. Dr. Buletko has nothing to disclose. Dr. Marquardt has nothing to disclose. Dr. Higgins has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Organon. Ms. Hassett has nothing to disclose.

EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012)

EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012)

Association of Disease Characteristics with Utilization of Behavioral Medicine in Patients with NMOSD and MOGAD (P13-5.016)

<h3>Objective:</h3> To evaluate utilization of behavioral medicine in patients with neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein-IgG-associated disorder (MOGAD). <h3>Background:</h3> The relationship between disease characteristics, utilization of behavioral medicine, and other healthcare services has not been well-characterized in patients with NMOSD/MOGAD. <h3>Design/Methods:</h3> In this retrospective cohort study, patients with NMOSD/MOGAD confirmed by a neuroimmunology clinic with available clinical data between June 2015 and April 2022 were identified. Relevant variables were collected, including number of behavioral medicine appointments, diagnosis, number of other healthcare visits (admissions, emergency room visits, neurology appointments, outpatient physical and occupational therapy [PT/OT] visits), and number and type of relapses (brainstem, transverse myelitis, and optic neuritis). Nonparametric t-tests were performed to compare characteristics of patients who did and did not use behavioral medicine. Pearson correlations were used to explore relationships between disease characteristics and number of behavioral medicine appointments. <h3>Results:</h3> 196 patients (70.9% NMOSD [92.8% aquaporin-4 antibody positive], 29.1% MOGAD) were identified (median duration of symptoms 8.4 years, IQR [4.6, 16.4 years]). Of these, 21 patients (90.5% NMOSD, 9.5% MOGAD) attended at least one outpatient behavioral medicine appointment. These patients had more emergency room visits (median 3, IQR [1, 8]) than non-attendees (median 1, IQR [0, 2]) (p = .04). No significant difference was detected regarding number or type of relapse or utilization of other healthcare services (for all, p &gt; .05). Brainstem flares and number of PT/OT visits correlated with number of behavioral medicine appointments (r = .72 and r = .56, respectively, p &lt; .05). <h3>Conclusions:</h3> Patients with NMOSD/MOGAD who received behavioral medicine services had greater emergency room utilization than those who did not. In our cohort, nearly 1 in 8 patients attended behavioral medicine appointments. Screening for mental health disorders in neurology clinics remains critical to appropriately identify and treat NMOSD/MOGAD patients with behavioral comorbidities. <b>Disclosure:</b> An immediate family member of Dr. Swetlik has received personal compensation for serving as an employee of Pfizer. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Pharmaceuticals. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Banner Life Sciences. Dr. Conway has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Miss Widmar has nothing to disclose. Dr. Kunchok has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Kunchok has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon therapeutics . Dr. Kunchok has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology. Dr. Tworek has nothing to disclose. Dr. Sullivan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bristol-Myers Squibb. Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Dynamed. Rachel Galioto has nothing to disclose. Dr. Aboseif has a non-compensated relationship as a AAN Autoimmune Neurology Section -- Education Workgroup with AAN that is relevant to AAN interests or activities. Dr. Abbatemarco has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD Serono. Dr. Abbatemarco has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon. Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech . The institution of Dr. Abbatemarco has received research support from Horizon.

Baseline Characteristics and Interim Safety in RESPOND: A Phase 4 Study in Children with Spinal Muscular Atrophy (SMA) Treated With Nusinersen After Onasemnogene Abeparvovec (P7-9.003)

Baseline Characteristics and Interim Safety in RESPOND: A Phase 4 Study in Children with Spinal Muscular Atrophy (SMA) Treated With Nusinersen After Onasemnogene Abeparvovec (P7-9.003)

Baseline characteristics and secondary medication adherence among Medicare patients diagnosed with transthyretin amyloid cardiomyopathy and/or receiving tafamidis prescriptions: A retrospective analysis of a Medicare cohort.

BACKGROUND: Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed, life-threatening condition that mostly affects older persons. In May 2019, regulatory approval of tafamidis provided the first pharmacologic treatment of ATTR-CM. In the pivotal phase 3 Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT), 97.2% of patients were classified as adherent (defined as taking ≥ 80% of scheduled doses). Given its recent approval, there is limited real-world evidence examining patient adherence to tafamidis. OBJECTIVE: To evaluate adherence patterns, demographics, and clinical characteristics of patients in the United States receiving tafamidis prescriptions through Medicare. Secondarily, we aimed to evaluate concomitant medications filled by this patient population. METHODS: We conducted a retrospective cohort study of US Medicare claims data, limited by the Health Insurance Portability and Accountability Act of 1996, in adult patients with an adjudicated pharmacy claim for tafamidis (tafamidis free acid 61-mg capsule once daily or tafamidis meglumine four 20-mg capsules once daily) between May 1, 2019, and June 30, 2021. Gaps in therapy were measured using day gaps between prescription refills and continuous measure of medication gaps. Implementation adherence was assessed through modified medication possession ratio (MPRm), medication refill adherence (MRA), and proportion of days covered (PDC). Patients were grouped based on Medicare coverage. Patients were analyzed by subgroups based on age and at the zip code level, via distressed communities index quartiles and rural-urban tiers. RESULTS: A total of 3,558 patients who received a prescription fill of a tafamidis formulation were identified using Medicare Fee-for-Service (FFS) and Medicare Advantage (MA) claims data from May 1, 2019, to June 30, 2021. The characteristics of this patient population were consistent with published literature, as 98.6% were older than 65 years, 53.4% were between 75 years and 84 years, and 81.5% were male. In the patient population receiving tafamidis refills, adherence was high across all 3 measures, with mean MPRm greater than 90% and mean MRA greater than 80%, across all age groups. Mean PDC adherence rates were 79% or more across all age groups. Concomitant medications were generally indicated for heart failure and thrombosis. Among monotherapy groups with similar demographic makeup, adherence was significantly higher among users of tafamidis free acid vs tafamidis meglumine (P < 0.0001 across all mean adherence measures). CONCLUSIONS: Our results demonstrate that real-world adherence to tafamidis in the Medicare population is high, regardless of age, zip code-level socioeconomic quartile, or geography. Adherence was higher among patients receiving tafamidis free acid, suggesting that the enhanced convenience of a single capsule once daily may positively contribute to adherence among patients with ATTR-CM. DISCLOSURES: Darrin Benjumea is an employee of Genesis Research who has been contracted by Pfizer, Inc., for involvement in this study. Andrew Peterson is an employee of University of the Sciences who has been contracted by Pfizer, Inc., for involvement in this study. Zach Bredl is an employee of Care Journey who has been contracted by Pfizer, Inc., for involvement in this study. Anuja Roy, Nick Marchant, Jose Alvir, Rahul Bhambri, Jason Kemner, and Bhash Parasuraman are employees of Pfizer, Inc., and own stock and/or stock options. This study was supported by Pfizer, Inc.

Health care resource utilization and costs associated with advanced or metastatic nonsmall cell lung cancer in the United States.

BACKGROUND: The treatment landscape for advanced nonsmall cell lung cancer (NSCLC) has evolved from 2015 onward, since the introduction of immune checkpoint inhibitors (ICIs). Considering this shift, there have been limited prior analyses that assess the economic burden of NSCLC within the current treatment landscape. OBJECTIVE: To present an analysis of health care resource utilization (HCRU) and costs associated with the treatment of patients with advanced or metastatic NSCLC in the United States between 2010 and 2019. METHODS: Patients with locally advanced or metastatic NSCLC who initiated first-line (1L) systemic treatment between January 1, 2010, and June 30, 2019, were included from the HealthCore Integrated Research Database using a previously developed claims-based predictive model algorithm. Mean total HCRU and costs and mean per-person-per-year (PPPY) HCRU and costs were estimated for 2 follow-up periods: the time during the entire follow-up period and the time during the 1L treatment period. Distribution of treatment classes (defined as chemotherapy, ICIs, targeted therapies, and others) were also analyzed by index year. RESULTS: 27,257 patients met the eligibility criteria and were included in the analysis. The mean duration of follow-up for all patients was 16.6 months (median 10.6 months), and the median time to discontinuation of 1L treatment was 2.8 months. The number of outpatient visits accounted for the majority of HCRU across the entire study follow-up (mean 97.7 in total and 147.1 PPPY) and for the 1L treatment period (mean 46.3 in total and 167.5 PPPY). The total mean cost across the entire study follow-up was $158,908 ($250,942 PPPY). For the 1L treatment period, the total mean cost was $72,760 ($271,590 PPPY). Total mean outpatient costs for systemic anticancer treatment were $61,797 for the entire study follow-up ($85,609 PPPY) and $27,138 during the 1L treatment period ($92,412 PPPY). Total costs increased over the study duration, which were mainly due to increasing outpatient costs for systemic therapy. In both follow-up periods, inpatient costs, other outpatient costs (nonsystemic therapy-related costs), and pharmacy costs remained relatively stable but still accounted for more than 60% of the total costs. Analysis of treatment classes over time showed that chemotherapy was the most frequently used treatment, regardless of line of therapy. A trend was observed for increased ICI use from 2015 onward. CONCLUSIONS: Despite the improvement in treatment options, a high economic burden associated with the treatment of NSCLC still exists. The total costs have been increasing, mainly driven by outpatient costs for systemic therapy, which might reflect the greater use of ICIs for advanced NSCLC. Costs for inpatient services, other outpatient services, and pharmacy services remained stable but still accounted for the majority of the economic burden. Further studies are required to assess the impact of innovative treatments on the disease management costs of advanced NSCLC. DISCLOSURES: This study was funded by the healthcare business of Merck KGaA, Darmstadt, Germany (CrossRef Funder ID: 10.13039/100009945) as part of an alliance between the healthcare business of Merck KGaA, Darmstadt, Germany, and Pfizer. Zhang, Liu, and Yang are employees of EMD Serono. Beachler, Dinh, and Jamal-Allial are employees of HealthCore Inc., which received funding from the healthcare business of Merck KGaA, Darmstadt, Germany, and Pfizer for the implementation of this study. Masters and Kolitsopoulos are employees of Pfizer. Lamy was an employee of the healthcare business of Merck KGaA, Darmstadt, Germany, at the time this study was conducted.

A harm reduction model to assess the impact of new treatments for pain over standard of care among patients with osteoarthritis.

BACKGROUND: Osteoarthritis (OA) affects millions of adults in the United States and can result in substantial pain, functional impairment, and significant clinical and economic burden. To manage chronic pain associated with OA, treatment guidelines recommend a variety of pharmacologic treatments, including traditional oral nonsteroidal anti-inflammatory drugs (NSAIDs), cyclooxygenase-2 inhibitors (COX-2s), and opioids. While these drug treatments can be effective at pain management, they are also associated with significant clinical and economic burden. New treatments for chronic pain among patients with OA of the hip and/or knee have the potential to reduce the occurrence of such negative clinical outcomes, including cardiovascular events, renal events, and opioid use disorder (OUD), thereby reducing health care resource use (HRU) and medical costs. OBJECTIVE: To develop a harm reduction model (HRM) to assess potential reductions of negative clinical outcomes, HRU, and medical costs associated with the use of new treatments in place of oral NSAIDs, tramadol, and non-tramadol opioids among patients with OA of the hip and/or knee in the United States. METHODS: The HRM model integrated findings from the literature and inputs from a variety of sources, along with assumptions regarding potential ability of new treatments to replace existing treatments and market penetration into a unified framework to estimate outcomes and costs. The model outputs included estimated per-patient and population-level reductions in negative clinical outcomes associated with prescribing new treatments in place of oral NSAIDs or opioids along with number needed to treat (NNT) associated with new treatments. The model assumed that new treatments will primarily be used in place of non-tramadol opioids, but more modest adoption in place of oral NSAIDs and tramadol. RESULTS: Among patients with OA of the hip and/or knee who were prescribed oral NSAIDs, tramadol, or non-tramadol opioids for chronic use (≥ 90 days), the HRM estimated total cost savings of $3.8 billion, $5.1 billion, and $29.9 billion, respectively, from prescribing new treatments for OA pain over a 36-month period. The reduced economic burden was driven by significant reductions in the incidence of negative clinical outcomes. Estimates of the NNT to avoid a negative clinical event related to NSAID and opioid treatment initiation were low for most outcomes. Estimates of NNT associated with NSAID use ranged from 4 to 17 patients, depending on outcome, and estimates of NNT associated with opioid use was 35 non-tramadol and 134 tramadol patients for OUD and ranged from 6 to 21 patients for the other clinical outcomes, depending on treatment and outcome. CONCLUSIONS: Results from the HRM suggest that prescribing new treatments in place of oral NSAIDs and/or opioids for OA pain results in a potentially substantial reduction in patients experiencing negative clinical outcomes and reductions in all-cause HRU and costs. DISCLOSURES: This study was sponsored by Pfizer and Eli Lilly and Company. Silverman was a paid consultant to Pfizer and Eli Lilly and Company in connection with this study. Beck and Schepman are employees of Pfizer with stock and/or stock options. Robinson is an employee and minor stockholder of Eli Lilly and Company. Rice, White, and Fernan are employees of the Analysis Group, who were paid consultants to Pfizer and Eli Lilly and Company for this study and development of the manuscript.