Availability of comparative real-world evidence research in Medicare patients: implications for Centers for Medicare and Medicaid Services drug price negotiations.

Aim: To evaluate the availability of published comparative real-world evidence (RWE) studies in Medicare patients for the ten drugs set to undergo Centers for Medicare and Medicaid Services (CMS) price negotiations in 2026. Materials & methods: A scoping review was completed in MEDLINE/PubMed to evaluate the availability of comparative RWE investigations conducted among Medicare-eligible patient populations in the US for the following drugs: apixaban, rivaroxaban, sitagliptin, ibrutinib, empagliflozin, etanercept, dapagliflozin, sacubitril/valsartan, ustekinumab and insulin aspart. Results: Of the 170 real-world comparative studies identified, 55 (32.4%) used Medicare real-world data (RWD) while 34 (20.0%) used commercial claims data in conjunction with either Medicare Advantage or Medicare Supplementary databases. The number of studies varied considerably by drug with apixaban and rivaroxaban studies accounting for the majority (i.e., 67.1%) of comparative RWE studies. Approximately a third or less of the comparative RWE studies were conducted in CMS RWD per drug. Conclusion: Our results demonstrate there is a considerable amount of comparative RWE for apixaban, rivaroxaban, and etanercept but limited comparative RWE for the other drugs set to undergo CMS price negotiations in 2026; additionally, our findings set up a number of next steps (e.g., risk of bias assessments) for further exploration of the available evidence base. Overall, CMS and manufacturers should consider proactively generating high-quality comparative RWE studies in the Medicare population to ensure that future price negotiations are based on robust evidence.

Comparison of adverse pregnancy and birth outcomes using archival medical records before and during the first wave of the COVID-19 pandemic in Kinshasa, Democratic Republic of Congo: a facility-based, retrospective cohort study.

BACKGROUND: Little research has been conducted on the impact of the coronavirus disease 2019 (COVID-19) pandemic on either birth outcomes or the ability of archival medical records to accurately capture these outcomes. Our study objective is thus to compare the prevalence of preterm birth, stillbirth, low birth weight (LBW), small for gestational age (SGA), congenital microcephaly, and neonatal bloodstream infection (NBSI) before and during the first wave of the COVID-19 pandemic in Kinshasa, Democratic Republic of Congo (DRC). METHODS: We conducted a facility-based retrospective cohort study in which identified cases of birth outcomes were tabulated at initial screening and subcategorized according to level of diagnostic certainty using Global Alignment of Immunization Safety Assessment in pregnancy (GAIA) definitions. Documentation of any birth complications, delivery type, and maternal vaccination history were also evaluated. The prevalence of each birth outcome was compared in the pre-COVID-19 (i.e., July 2019 to February 2020) and intra-COVID-19 (i.e., March to August 2020) periods via two-sample z-test for equality of proportions. RESULTS: In total, 14,300 birth records were abstracted. Adverse birth outcomes were identified among 22.0% and 14.3% of pregnancies in the pre-COVID-19 and intra-COVID-19 periods, respectively. For stillbirth, LBW, SGA, microcephaly, and NBSI, prevalence estimates were similar across study periods. However, the prevalence of preterm birth in the intra-COVID-19 period was significantly lower than that reported during the pre-COVID-19 period (8.6% vs. 11.5%, p < 0.0001). Furthermore, the level of diagnostic certainty declined slightly across all outcomes investigated from the pre-COVID-19 to the intra-COVID-19 period. Nonetheless, diagnostic certainty was especially low for certain outcomes (i.e., stillbirth and NBSI) regardless of period; still, other outcomes, such as preterm birth and LBW, had moderate to high levels of diagnostic certainty. Results were mostly consistent when the analysis was focused on the facilities designated for COVID-19 care. CONCLUSION: This study succeeded in providing prevalence estimates for key adverse birth outcomes using GAIA criteria during the COVID-19 pandemic in Kinshasa, DRC. Furthermore, our study adds crucial real-world data to the literature surrounding the impact of the COVID-19 pandemic on maternal and neonatal services and outcomes in Africa.

Transferability of real-world data across borders for regulatory and health technology assessment decision-making.

Recently, there has been increased consideration of real-world data (RWD) and real-world evidence (RWE) in regulatory and health technology assessment (HTA) decision-making. Due to challenges in identifying high-quality and relevant RWD sources, researchers and regulatory/HTA bodies may turn to RWD generated in locales outside of the locale of interest (referred to as "transferring RWD"). We therefore performed a review of stakeholder guidance as well as selected case studies to identify themes for researchers to consider when transferring RWD from one jurisdiction to another. Our review highlighted that there is limited consensus on defining decision-grade, transferred RWD; certain stakeholders have issued relevant guidance, but the recommendations are high-level and additional effort is needed to generate comprehensive guidance. Additionally, the case studies revealed that RWD transferability has not been a consistent concern for regulatory/HTA bodies and that more focus has been put on the evaluation of internal validity. To help develop transferability best practices (alongside internal validity best practices), we suggest that researchers address the following considerations in their justification for transferring RWD: treatment pathways, nature of the healthcare system, incidence/prevalence of indication, and patient demographics. We also recommend that RWD transferability should garner more attention as the use of imported RWD could open doors to high-quality data sources and potentially reduce methodological issues that often arise in the use of local RWD; we thus hope this review provides a foundation for further dialogue around the suitability and utility of transferred RWD in the regulatory/HTA decision-making space.

Adherence to face mask use during the COVID-19 pandemic among women seeking antenatal care in Kinshasa, Democratic Republic of Congo: a facility-based cross-sectional study.

OBJECTIVES: To describe face mask use among pregnant women seeking antenatal care (ANC) in Kinshasa, Democratic Republic of Congo and to identify factors associated with masking adherence in this population. DESIGN: Facility-based cross-sectional study nested within a prospective cohort study. SETTING: Random sample of 10 health facilities, including 5 primary health centers and 5 secondary facilities or hospitals. PARTICIPANTS: A total of 934 pregnant women aged 18 years or above with a gestational age of at least 32 weeks were consecutively surveyed from 17 August 2020 to 31 January 2021. PRIMARY AND SECONDARY OUTCOME MEASURES: We estimated the proportions of pregnant women wearing a face mask and masking correctly (ie, over the mouth and nose), and assessed their knowledge regarding the COVID-19 pandemic. Multivariable logistic regression was employed to identify factors associated with overall and correct face mask use. RESULTS: Overall, 309 (33.1%) women wore a mask during the interview after their antenatal appointments, but only 33 (10.7%) wore a mask correctly. The odds of masking and correct mask use were significantly higher among women who had their ANC visit in a facility that provided COVID-19 care. Additionally, women who experienced COVID-19-like symptoms in the past 6 months had higher odds of wearing a mask correctly compared with those reporting no recent symptoms. Although 908 (97.2%) women were aware of the COVID-19 pandemic, only 611 (67.3%) thought that COVID-19 was circulating locally in Kinshasa. CONCLUSION: Overall and correct face mask adherence levels were low among pregnant women attending ANC in Kinshasa. Our study highlights the need for improving adherence to correct face mask use in order to help control the spread of COVID-19 within Kinshasa alongside other control measures, like vaccination.

Assessing the feasibility of passive surveillance for maternal immunization safety utilizing archival medical records in Kinshasa, Democratic Republic of the Congo.

INTRODUCTION: Since the establishment of the Global Alignment of Immunization Safety Assessment in pregnancy (GAIA) case definitions in 2015, there has been an urgent need for field validation of pharmacovigilance feasibility in low- and middle-income countries. In this study, we assess the availability and quality of archival medical records at ten randomly selected high-traffic maternity wards in Kinshasa province, Democratic Republic of Congo (DRC). METHODS: A retrospective cohort of mother-child pairs was established from all recorded births taking place at study sites between July 1, 2019 to February 28, 2020 through digitization of medical records. Adverse birth outcomes and maternal vaccination status, where available and linkable, were defined according to GAIA. Basic demographic information on mothers and newborns was also tabulated; birth outcomes were assessed for both intra-site prevalence and a pooled prevalence. RESULTS: A total of 7,697 mother-newborn pair records were extracted, with 37% of infants screening positive as cases of adverse outcomes. Maternal vaccination information was linkable to 67% of those cases. In total, 51% of stillbirths, 98% of preterm births, 100% of low birthweight infants, 90% of small for gestational age infants, 100% of microcephalic infants, and 0% of neonatal bloodstream infections were classifiable according to GAIA standards following initial screening. Forty percent of case mothers had some indication of tetanus vaccination prior to delivery in their medical records, but only 26% of case mothers met some level of GAIA definition for maternal vaccination during the pregnancy of interest. CONCLUSIONS: Archival birth records from delivery centers can be feasibly utilized to screen for stillbirth and maternal tetanus vaccination, and to accurately classify preterm birth, low birthweight, small for gestational age, and congenital microcephaly. Assessment of other neonatal outcomes were limited by inconsistent postpartum infant follow-up and records keeping.

The incidence of acute myocardial infarction after elective spinal fusions or joint replacement surgery in the United States: a large-scale retrospective observational cohort study in 322,585 patients : Post-surgical myocardial infarction data.

BACKGROUND: Acute myocardial infarction (AMI) is an uncommon but fatal complication among patients undergoing elective spinal fusion surgery (SF), total hip arthroplasty (THA), and total knee arthroplasty (TKA). Our objective was to estimate the incidence of AMI among adults undergoing elective SF, THA, and TKA in different post-operative risk windows and characterize high-risk sub-populations in the United States. METHODS: A retrospective cohort study was conducted using data from a longitudinal electronic healthcare record (EHR) database from January 1, 2007 to June 30, 2018. ICD codes were used to identify SF, THA, TKA, AMI, and selected clinical characteristics. Incidence proportions (IPs) and 95% confidence intervals were estimated in the following risk windows: index hospitalization, ≤ 30, ≤ 90, ≤ 180, and ≤ 365 days post-operation. RESULTS: A total of 67,533 SF patients, 87,572 THA patients, and 167,480 TKA patients were eligible for the study. The IP of AMI after SF, THA, and TKA ranged from 0.36, 0.28, and 0.25% during index hospitalization to 1.05, 0.93, and 0.85% ≤ 365 days post-operation, respectively. The IP of AMI was higher among patients who were older, male, with longer hospital stays, had a history of AMI, and had a history of diabetes. CONCLUSION: The IP of post-operative AMI was generally highest among the SF cohort compared to the THA and TKA cohorts. Additionally, potential high-risk populations were identified. Future studies in this area are warranted to confirm these findings via improved confounder control and to identify effect measure modifiers.

The incidence of stroke among selected patients undergoing elective posterior lumbar fusion: a retrospective cohort study.

BACKGROUND: Although stroke is a rare complication among spinal surgery patients, the recognition of this adverse event is critical given the aging population undergoing surgical procedures. The objective of this study was to estimate the incidence of stroke among selected adults undergoing elective posterior lumbar fusion (PLF) during various post-operative risk windows and among different subgroups. METHODS: A retrospective cohort study using a longitudinal electronic healthcare record (EHR) database was conducted from January 1, 2007 to June 30, 2018. Elective PLF, stroke, and select clinical characteristics were defined based on International Classification of Disease codes. Patients aged 18 to 85 years with ≥183 days of enrollment in the database prior to undergoing elective PLF were followed from the index date until the occurrence of stroke, death, loss to follow-up, or end of study period, whichever occurred first. The incidence of stroke was estimated in the following risk windows: index hospitalization, ≤ 30 days, ≤ 90 days, ≤ 180 days, and ≤ 365 days post-operation. RESULTS: A total of 43,063 patients were eligible for the study. The incidence of stroke following elective PLF was 0.29% (95% confidence interval [CI]: 0.25, 0.35%) during index hospitalization, 0.44% (95% CI: 0.38, 0.50%) ≤ 30 days, 0.59% (95% CI: 0.52, 0.67%) ≤ 90 days, 0.76% (95% CI: 0.68, 0.85%) ≤ 180 days, and 1.12% (95% CI: 1.03, 1.23%) ≤ 365 days post-operation. Stratified analyses revealed that older patients had a higher incidence of stroke. Additionally, black patients had higher stroke incidences. Post-operative stroke incidence was higher among patients with a history of type 2 diabetes than among patients without such history; similarly, stroke incidence was higher among patients with a history of stroke compared to patients without such history. CONCLUSIONS: The incidence of stroke following elective PLF using an EHR database in this study is slightly higher than that reported in the literature. Our results suggest that stroke risk modification prior to PLF may be important for patients who are older, black, type 2 diabetic, and/or have a history of stroke.

Incidence rates of health outcomes of interest among Chinese children exposed to selected vaccines in Yinzhou Electronic Health Records: A population-based retrospective cohort study.

INTRODUCTION: Oral poliovirus vaccine (OPV) and diphtheria-tetanus-acellular pertussis vaccine (DTaP) are widely used in China while Haemophilus influenzae type b vaccines (Hib) and a DTaP, inactivated poliovirus (IPV) andHib polysaccharide conjugated to tetanus protein (PRP ~ T) combined vaccine (DTaP-IPV//PRP ~ T) have lower coverage. There are limited safety data on these vaccines in Chinese pediatric populations. METHODS: To estimate incidence rates (IRs) of health outcomes of interest (HOIs) among children exposed to OPV, DTaP, Hib, and DTaP-IPV//PRP ~ T, we conducted a retrospective cohort study using a population-based electronic health record (EHR) database in Yinzhou district, Ningbo City. Children 0-2 years of age receiving at least one dose of these vaccines between January 1, 2012 and March 31, 2017 were included in the study. Yinzhou EHR database consisted of immunization records and healthcare data of children from hospitals and community health centers in the district. Eight HOIs (i.e., anaphylaxis, febrile seizures, all seizures, asthma, apnea, Kawasaki disease [KD], urticaria/angioedema, Guillain-Barré syndrome [GBS]) were identified using ICD-10 codes. RESULTS: A total of 220,422 eligible children was identified. No cases of apnea, KD, and GBS were observed within 7 days post-vaccination. During 0-7 days post-vaccination for OPV, DTaP, Hib, and DTaP-IPV//PRP ~ T, the IRs of anaphylaxis, febrile seizures, all seizures, urticaria/angioedema and asthma ranged from 0.0 to 50.0, 0.0 to 99.9, 29.1 to 249.8, 297.8 to 949.1, and 992.7 to 2298.2 per 100,000 person-years, respectively, and 0.0 to 0.9, 0.0 to 1.9, 0.6 to 4.6, 5.6 to 17.5, and 18.7 to 42.3 per 100,000 doses, respectively. CONCLUSION: IRs of some HOIs in our study were comparable with those in the literature while IRs of other HOIs were not due to differences in study design, post-vaccination risk periods assessed, and vaccine types. Future studies should consider medical chart review for validating HOIs obtained in the EHR.