Tom Hughes

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. 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." 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. These findings suggest positive real-world perceptions of VYVGART and VYVGART Hytrulo among patients in improving symptoms and health-related quality of life.

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 US, focusing on specialty drugs – i.e., 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 (FDA) 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.

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. 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. 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. 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.