INTRODUCTION:Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disorder characterized by fluctuating muscle weakness (ocular, bulbar, limb, and respiratory). Efgartigimod (VYVGART), a first-in-class neonatal Fc receptor antagonist, was approved for treatment of generalized MG (gMG). This study aimed to explore real-world sentiments, wherein in sentiments refer to real-world perceptions expressed by patients, caregivers, and healthcare professionals (HCPs) on VYVGART and VYVGART Hytrulo, through structured social media analysis. METHODS:This social media listening study was designed to collect posts mentioning VYVGART and VYVGART Hytrulo for gMG using predefined keywords from social media platforms across the globe. Organic English-language posts from several platforms, such as Reddit, X (formerly Twitter), Facebook, Forums, Review, and Tumblr etc., were analyzed for the timeframe August 1, 2021 to September 30, 2024. Posts were categorized into themes, namely "brand awareness," "treatment access," "treatment experience," and "treatment support." RESULTS:A total of 1046 posts (USA 9%, Europe 6%, Unknown 85%) contained relevant information; 1015 posts were related to VYVGART and 55 posts were related to VYVGART Hytrulo. Most posts focused on treatment experience (593 posts) and brand awareness (402 posts). Overall sentiments towards VYVGART and VYVGART Hytrulo were positive in 49%, 30%, and 20% of patient, caregiver, and HCP posts, respectively. Patient posts expressed optimism about initiating treatment. There were fewer caregiver and HCP discussions and these were predominantly neutral. CONCLUSION:These findings suggest positive real-world perceptions of VYVGART and VYVGART Hytrulo among patients in improving symptoms and health-related quality of life.

INTRODUCTION:Generalized myasthenia gravis (gMG) is a chronic neuromuscular disease that causes muscle weakness and fatigue, severely impairing quality of life. Efgartigimod is a novel drug that is recently approved for treatment of acetylcholine receptor antibody-positive (AChR-Ab+) gMG patients in Canada. In clinical practice, it is expected to be used in AChR-Ab+ gMG patients who continue to experience symptoms despite conventional therapy and primarily replace chronic immunoglobulins. METHODS:A Markov model was developed to estimate costs and benefits (measured as quality-adjusted life years [QALYs]) of efgartigimod and chronic immunoglobulins for AChR-Ab+ gMG patients. The analysis was conducted from the perspective of the Canadian publicly funded healthcare system over a lifetime horizon. The model comprised six health states based on Myasthenia Gravis Activities of Daily Living (MG-ADL) scores: MG-ADL < 5, MG-ADL 5-7, MG-ADL 8-9, MG-ADL ≥ 10, myasthenic crisis or death. Health state transition probabilities were estimated from the ADAPT and ADAPT+ studies, plus a network meta-analysis that compared efgartigimod against chronic immunoglobulins. The MyRealWorld MG study informed utility values. Modeled costs included treatment and administration, disease monitoring, complications from chronic corticosteroid use, exacerbation/crisis management, adverse events and end-of-life care. RESULTS:Over a lifetime horizon, efgartigimod and chronic immunoglobulins were predicted to have total discounted QALYs of 16.80 and 13.35 and total discounted costs of $1,913,294 and $2,170,315, respectively. Efgartigimod dominated chronic immunoglobulins with incremental QALYs of 3.45 and cost savings of $257,020. CONCLUSIONS:Efgartigimod provides greater benefit in terms of lower costs than chronic immunoglobulins for AChR-Ab+ gMG patients in Canada.

Introduction:Prescription drug list prices, often cited in policy discussions, do not account for rebates negotiated between manufacturers and payers. Methods:This study examines factors influencing rebates in the United States, focusing on specialty drugs-ie, high priced therapies (often biologics) for which payers issue specific coverage policies. Rebate data came from SSR Health and drug attributes from the Tufts Medical Center Specialty Drug Evidence and Coverage Database. We stratified rebate data by drug type and characteristics. Results:We identified 161 drugs found in SSR Health and SPEC as of December 2023. We found that rebates vary substantially across drugs (median of 27%, IQR 16%-53%). Biosimilar and originator drugs have the highest, most variable rebates (median of 71%, IQR 53%-79%), while rebates for cancer treatments and orphan drugs are lower (medians of 19% and 23%, respectively) and vary less (IQR of 12%-28% and 14%-29%, respectively). Drugs that face more competition from alternative options within the same therapeutic class, are self-administered, or received Food and Drug Administration approval further in the past have higher rebates. Conclusion:Our findings indicate that rebates are sizeable and vary along several dimensions, many relating to market competition, complicating policy discussions around drug pricing.

BACKGROUND AND OBJECTIVES:Myasthenia gravis (MG) is a rare neuromuscular disorder where IgG antibodies damage the communication between nerves and muscles, leading to muscle weakness that can be severe and have a significant impact on patients' lives. MG exacerbations include myasthenic crisis with respiratory failure, the most serious manifestation of MG. Recent studies have found MG prevalence increasing, especially in older patients. This study examined trends in hospital admissions and in-hospital mortality for adult patients with MG and readmissions and postdischarge mortality in older (65 years or older) adults with MG.METHODS:Data from the Nationwide Inpatient Sample (NIS), an all-payer national database of hospital discharges, were used to characterize trends in hospitalizations and in-hospital mortality related to MG exacerbations and MG crisis among adult patients aged 18 years or older. The Medicare Limited Data Set, a deidentified, longitudinal research database with demographic, enrollment, and claims data was used to assess hospitalizations, length of stay (LOS), readmissions, and 30-day postdischarge mortality among fee-for-service Medicare beneficiaries aged 65 years or older. The study period was 2010-2019. Multinomial logit models and Poisson regression were used to test for significance of trends.RESULTS:Hospitalization rates for 19,715 unique adult patients and 56,822 admissions increased from 2010 to 2019 at an average annualized rate of 4.9% (MG noncrisis: 4.4%; MG crisis: 6.8%; all p < 0.001). Readmission rates were approximately 20% in each study year for both crisis and noncrisis hospitalizations; the in-hospital mortality rate averaged 1.8%. Among patients aged 65 years or older, annualized increases in hospitalizations were estimated at 5.2%, 4.2%, and 7.7% for all, noncrisis, and crisis hospitalizations, respectively (all p < 0.001). The average LOS was stable over the study period, ranging from 11.3 to 13.1 days, but was consistently longer for MG crisis admissions. Mortality among patients aged 65 years or older was higher compared with that in all patients, averaging 5.0% across each of the study years.DISCUSSION:Increasing hospitalization rates suggest a growing burden associated with MG, especially among older adults. While readmission and mortality rates have remained stable, the increasing hospitalization rates indicate that the raw numbers of readmissions-and deaths-are also increasing. Mortality rates are considerably higher in older patients hospitalized with MG.

Objective: To better understand the needs of patients living with generalized Myasthenia Gravis (gMG) and impacted by Social Determinants of Health (SDOH) challenges in terms of Patient Support Program (PSP) services to improve patient outcomes. Background: SDOH challenges are factors of the environment or patients' lives that affect health, functioning, and quality of life outcomes. Design/Methods: Phase One – Qualitative research was conducted with 11 patients and 2 Patient Advocacy Groups (PAGs) to understand PSP awareness, experience, and unmet needs. Phase Two – Quantitative research was conducted with 38 patients, the majority were people of color, non-urban residents and low-income women. We identified SDOH challenges, assessed current utilization of services and unmet needs for PSP services to be addressed through new offerings. Phase three – Qualitative research was conducted with 11 Health Care Professionals (HCPs) to understand how they address SDOH challenges in treating patients with gMG. Results: In Phase Two, four prioritized PSP solutions were identified: 1) Expand current PSP services, including a medication information hotline, symptom tracking app, and updated educational resources; 2) Develop a personalized "human touch", e.g., 1:1 patient mentoring program for people initiating treatment or experiencing medical complications; 3) Create an innovative giving strategy and establish a long-term financial commitment to support patients living with gMG, 4) Increase PAGs awareness of Nurse Case Manager services. In Phase Three, we found most HCPs are not adequately trained to identify patients with SDOH challenges, have limited training on educating patients on gMG, and often depend on social workers and nurses for these tasks. Conclusions: For patients living with gMG encountering SDOH challenges, there are opportunities to bolster PSP offerings to improve patient outcomes. HCPs receive limited training on educating patients about potential solutions to SDOH challenges. Disclosure: Dr. Hughes has received personal compensation for serving as an employee of Argenx . Dr. Hughes has stock in argenx. Cecilia Zvosec has nothing to disclose. Dr. Anderson has nothing to disclose. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for argenx. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from RA pharmaceuticals. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron. Mrs. Suchotliff has received personal compensation for serving as an employee of ZS. Kathy Perez has received personal compensation for serving as an employee of argenx. Kathy Perez has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for argenx. Kathy Perez has stock in argenx. Kathy Perez has received personal compensation in the range of $100,000-$499,999 for serving as a VP Global Patient Advocacy with argenx. Cathleen Bergin has stock in argenx. Dr. Phillips has received personal compensation for serving as an employee of argenx. Dr. Phillips has stock in argenx.

IntroductionSocial determinants of health (SDOH) are important contributors to health outcomes, and better understanding their impact on individuals diagnosed with rare, chronic diseases with high burden and unmet need is critical. Characterizing SDOH burden can help improve the design of patient support programs (PSPs), using targeted approaches to remove barriers to access.MethodsThis study used a mixed-methods strategy employing a quantitative survey, which was designed based on qualitative interviews, to understand the unmet needs and awareness/utilization of PSPs among individuals living with generalized myasthenia gravis (gMG) and experiencing SDOH barriers. The survey was completed by 38 individuals living with gMG, of which the majority were non-White/Caucasian, unemployed, low income, and enrolled in public insurance. Common SDOH challenges, awareness/utilization of available PSPs, and unmet needs were identified.ResultsFinancial and mental health concerns were the most common among individuals living with gMG and experiencing SDOH barriers throughout diagnosis, accessing treatment, initiating treatment, and continuing treatment. Awareness and utilization of existing support services were low, especially when accessing treatment. Educational, financial, and personalized support with high “human touch” were commonly perceived as the most valuable resources.ImplicationsTo better serve the needs of individuals with gMG experiencing SDOH barriers, PSPs should use a targeted approach to offer services tailored to harder-to-reach populations. Further, providers, advocacy groups, manufacturers, and public organizations in the gMG ecosystem should strengthen collaborations with PSPs to enable individuals living with gMG to access the services they need to improve their health outcomes.

Myasthenia gravis (MG) is a rare neuromuscular disorder where IgG antibodies damage the communication between nerves and muscles, leading to muscle weakness that can be severe and have a significant impact on patients' lives. MG exacerbations include myasthenic crisis with respiratory failure, the most serious manifestation of MG. Recent studies have found MG prevalence increasing, especially in older patients. This study examined trends in hospital admissions and in-hospital mortality for adult patients with MG and readmissions and postdischarge mortality in older (65 years or older) adults with MG.

Objective:Better understanding the impact of social determinants of health (SDOH) barriers from the patient perspective is crucial to improve holistic patient support in generalized myasthenia gravis (gMG), a rare autoimmune disorder with high disease and treatment burden. The objective of this study was to identify economic challenges experienced by individuals living with gMG and SDOH barriers to better address current unmet needs. Methods:Adults (18-75 years) living with gMG and experiencing SDOH barriers in the United States were recruited to a mixed-methods study including qualitative interviews and a web-based quantitative survey. Quotas were implemented to include a balanced spread of baseline demographic categories including insurance type, living environment, and employment status among the study sample. Direct and indirect economic challenges were identified by degree of concern. Results:The survey was completed by 38 individuals living with gMG, the majority of whom were enrolled in public insurance and not employed. The most commonly reported major economic concerns were managing funds for emergency care (66%), loss of income (61%), and non-medical expenses (58%), highlighting the diversity of economic challenges. Individuals who were using public insurance plans, living in non-urban environments, and unemployed experienced pronounced challenges around managing non-medical costs and accessing government assistance. Conclusion:Both direct and indirect costs were emphasized as major concerns among individuals living with gMG and SDOH barriers. Increasing access to relevant, personalized, and holistic resources, including care management, should be prioritized to improve disease management and outcomes for individuals living with gMG.

To assess the number needed to treat (NNT) and costs required to achieve improvements in symptoms and functional activities with targeted therapies for myasthenia gravis (MG). Relative to conventional therapy (CT), NNTs and annual costs for achieving one point improvement in Quantitative Myasthenia Gravis score (QMG), one additional patient with minimal clinically important difference (MCID) in QMG (≥ 3 point improvement), and one additional patient achieving minimal symptom expression (MSE; Myasthenia Gravis-Activities of Daily Living score of 0 or 1) were estimated for efgartigimod (EFG), intravenous immunoglobulin (IVIg), and eculizumab (ECU). All treatments were used in conjunction with CT. Costs per improved outcome (CPR) were compared between EFG, IVIg, and ECU. Efficacy was evaluated at week 4 of respective phase 3 randomized trials (ADAPT [NCT03669588], NCT02473952, REGAIN [NCT01997229]). Annual drug acquisition and administration costs (2021 USD) were considered. Compared with CT, mean NNTs to achieve one point improvement and MCID in QMG were 0.19 and 2.31 for EFG, 0.52 and 7.14 for IVIg, and 0.56 and 6.25 for ECU. NNTs to achieve an additional patient with MSE was 3.46 for EFG and 8.13 for ECU. Compared to EFG, the mean annual CPR to achieve one point improvement and MCID in QMG were higher for IVIg (Difference [95% confidence interval] = $36,130 [$14,024, $58,237] per point improvement in QMG; $583,669 [$0, $1,473,027] per one patient with MCID in QMG) and ECU ($340,659 [$158,038, $523,280]; $3,760,825 [$1,391,543, $6,130,107]). Cost to achieve one additional patient with MSE was $4,761,649 ($2,859,671, $6,663,626) higher for ECU compared with EFG. NNT and cost per improved efficacy help inform comparative clinical efficacy and cost-effectiveness across MG treatments. Evidence indicates more favorable treatment benefit and economic value for EFG with fewer NNT and lower cost required to achieve improved outcomes compared to other treatments.

BACKGROUND/OBJECTIVES:While the clinical manifestations of myasthenia gravis (MG) are well understood, its humanistic impact is not. The objective of this systematic literature review (SLR) was to provide a comprehensive understanding of the humanistic burden of MG with regards to psychological symptoms and health-related quality of life (HRQoL) according to patients and caregivers.METHODS:A systematic search was conducted on December 27, 2019, in MEDLINE and Embase to identify English-language studies that were published from January 1, 2009-December 27, 2019 and presented relevant information on the humanistic burden among adults with MG and their caregivers. Title/abstract and full-text screening was performed by two investigators, with any discrepancies resolved by a third investigator.RESULTS:Sixty-seven publications were included in the SLR. Compared with the general population, patients with MG experienced worse HRQoL. Studies reporting on psychological symptoms of MG, including depression, anxiety, fatigue, and sleep, were heterogeneous in terms of the scales and instruments used to assess patients, as well as the patient populations themselves. However, in general those with more severe symptoms and hospitalization days had worse depression and anxiety, and fatigue and sleep improved with disease remission and/or improvement. Scores were worse for females compared with males and where evaluated, HRQoL scores generally improved following treatment.CONCLUSION:While the literature demonstrates that symptoms associated with MG get better with disease improvement and remission, additional options in efficacious therapy that adequately address the disease-related symptoms and also improve HRQoL may contribute to beneficial outcomes in a greater number of patients with MG.

Objective: To evaluate real-world utilization patterns of intravenous immunoglobulin (IVIg) among patients with generalized myasthenia gravis (gMG) over 3 years post-IVIg initiation.Methods: Patients with gMG who initiated IVIg treatment were identified from a United States claims database (Symphony Health's Integrated Dataverse [IDV](R), January 1, 2014 -December 31, 2019). The frequency of subsequent IVIg treatment and associated cost during the year post-IVIg initiation were analyzed. Usage patterns of IVIg and concomitant gMG treatments during the year preceding and 3 years post-IVIg initiation were compared.Results: Among 1225 patients with gMG who initiated IVIg treatment, 706 patients (57.6%) received 1 to 5 IVIg treatment courses (intermittent IVIg users), and 519 patients (42.4%) received >= 6 IVIg treatment courses (chronic IVIg users) within the subsequent year. Mean annual medical cost per patient was nearly 2.5-fold higher for chronic vs. intermittent IVIg users ($161,478 vs. $64,888, p < 0.001). The proportion of patients using corticosteroids and nonsteroidal immunosuppressive treatments (NSISTs) was not reduced over the 3-year follow-up period following IVIg initiation, even for patients who continued annual chronic IVIg for 3 consecu-tive years post-initiation.Conclusions: Nearly half of patients with gMG received chronic and multiple IVIg treatment courses within the first year once initiating IVIg treatment, indicating higher usage than expected. For all IVIg initiators, the pro-portion of patients using corticosteroids and NSISTs did not decrease over 3 years despite IVIg initiation.