Analysis of Costs per Responder in US Adults with Paroxysmal Nocturnal Hemoglobinuria with a Suboptimal Response to Prior Eculizumab Treatment.

European Society for Blood and Marrow Transplantation (EBMT) hematologic response categories comprehensively assess complement inhibitor responses in patients with paroxysmal nocturnal hemoglobinuria (PNH). Using data from the 16-week randomized controlled period of the phase 3 PEGASUS trial (N = 80), we estimated the treatment cost per responder by the EBMT response category for pegcetacoplan and eculizumab in adults with PNH and a suboptimal response to eculizumab. Average drug costs per responder, number needed to treat, and incremental drug costs per responder were estimated using dosages administered during the trial (base case). A US payer perspective (2020 US dollars) was used. Scenario analyses were conducted for various costs, dosages, treatment durations, patient populations, and settings. In total, 30 of 41 (73%) who switched to pegcetacoplan and 2 of 39 (5%) patients who continued eculizumab had a good, major, or complete response (good-to-complete responders) at Week 16. Average weekly drug costs per good-to-complete responder were USD 15,923 with pegcetacoplan and USD 216,100 with eculizumab; average weekly drug costs per patient were USD 11,651 and USD 11,082, respectively. Average drug costs per good-to-complete responder with pegcetacoplan were similar across complement inhibitor-naïve populations and were consistently lower than with eculizumab. Switching from eculizumab to pegcetacoplan allowed more patients with a suboptimal response to attain a good-to-complete response at lower costs. These results apply to patients with a suboptimal response to prior eculizumab treatment only.

Cost-effectiveness of a 3-antigen versus single-antigen vaccine for the prevention of hepatitis B in adults in the United States.

OBJECTIVES: The first 3-antigen hepatitis B vaccine was approved by the United States (US) Food and Drug Administration in November 2021 and was recommended by the Centers for Disease Control and Prevention in 2022. We estimated the cost-effectiveness of this 3-antigen vaccine (PreHevbrio™) relative to the single-antigen vaccine, Engerix-BTM, to prevent hepatitis B virus (HBV) infection among US adults. METHODS: A cost-effectiveness model was developed using a combined decision-tree and Markov structure to follow 100,000 adults over their remaining lifetimes after vaccination with either the 3-antigen or single-antigen vaccine. Outcomes from societal and healthcare sector perspectives were calculated for adults aged 18-44, 45-64, and ≥65 years; adults with diabetes; and adults with obesity. Seroprotection rates were obtained from the phase3, head-to-head PROTECT trial (NCT03393754). Incidence, vaccine costs, vaccine adherence rates, direct and indirect costs, utilities, transition probabilities, and mortality were obtained from published sources. Health outcomes and costs (2020USD) were discounted 3% annually and reported by vaccine and population. One-way sensitivity and scenario analyses were conducted. RESULTS: In the model, the 3-antigen vaccine led to fewer HBV infections, complications, and deaths compared with the single-antigen vaccine in all modeled populations due to higher rates and faster onset of seroprotection. Compared with the single-antigen vaccine, the 3-antigen vaccine had better health outcomes, more quality-adjusted life-years (QALYs), and lower costs in adults aged 18-64 years, adults with diabetes, and adults with obesity (dominant strategy). For adults aged ≥65 years, the 3-antigen vaccine was cost-effective compared with the single-antigen vaccine ($26,237/QALY gained) below common willingness-to-pay thresholds ($50,000-$100,000/QALY gained). In sensitivity analyses, results were sensitive to vaccine cost per dose, incidence, and age at vaccination. CONCLUSION: The recently approved 3-antigen vaccine is a cost-saving or cost-effective intervention for preventing HBV infection and addressing the long-standing burden of hepatitis B among US adults.

Estimating the cost of university-based outbreaks of serogroup B meningococcal disease with different pre-matriculation vaccination policies in the United States.

Objective: We developed an Excel-based cost calculator to assess the economic burden of university-based Neisseria meningitidis serogroup B (MenB) outbreaks. Participants: Hypothetical university with 6,354 students. Methods: Total societal costs of outbreak were estimated for three MenB pre-matriculation immunization policies-vaccination required, vaccination recommended, and no vaccine policy-under three different cost assumptions (low/mid-range/high cost). Results: Mid-range cost estimates of an outbreak under "no policy" were $2.60 and $2.70 million (of which 35% were incurred by the university) if targeting all undergraduates for mass vaccination with a two-/three-dose vaccine, respectively. The "required" and "recommended" policies lowered the burden to $2.17-$2.18 million and $2.34-$2.39 million, respectively. For a larger university with 40,000 students, costs were almost $9 million for a two-dose vaccine with "no policy" in place. Conclusions: The economic burden of a university MenB outbreak is substantial, but could be mitigated by a pre-matriculation MenB vaccination requirement or recommendation.

Tools for Supporting the MCH Workforce in Addressing Complex Challenges: A Scoping Review of System Dynamics Modeling in Maternal and Child Health.

OBJECTIVES: System Dynamics (SD) is a promising decision support modeling approach for growing shared understanding of complex maternal and child health (MCH) trends. We sought to inventory published applications of SD to MCH topics and introduce the MCH workforce to these approaches through examples to support further iteration and use. METHODS: We conducted a systematic search (1958-2018) for applications of SD to MCH topics and characterized identified articles, following PRISMA guidelines. Pairs of experts abstracted information on SD approach and MCH relevance. RESULTS: We identified 101 articles describing applications of SD to MCH topics. APPROACH: 27 articles present qualitative diagrams, 10 introduce concept models that begin to quantify dynamics, and 67 present more fully tested/analyzed models. PURPOSE: The most common purposes described were to increase understanding (n = 55) and support strategic planning (n = 26). While the majority of studies (n = 53) did not involve stakeholders, 40 included what we considered to be a high level of stakeholder engagement - a strength of SD for MCH. TOPICS: The two Healthy People 2020 topics addressed most frequently were early and middle childhood (n = 30) and access to health services (n = 26). The most commonly addressed SDG goals were "End disease epidemics" (n = 26) and "End preventable deaths" (n = 26). CONCLUSIONS FOR PRACTICE: While several excellent examples of the application of SD in MCH were found, SD is still underutilized in MCH. Because SD is particularly well-suited to studying and addressing complex challenges with stakeholders, its expanded use by the MCH workforce could inform an understanding of contemporary MCH challenges.

Antiseizure medications and oral contraceptives: Impact of enzyme inducers on pregnancy outcomes and costs.

PURPOSE: To show the impact of drug-drug interactions (DDIs) associated with co-administration of enzyme-inducing (EI) antiseizure medications and oral contraceptives (OCs) on the annual number of unintended pregnancies, their outcomes, and their associated costs in the United States (US). METHODS: A Microsoft Excel pregnancy-outcomes model was developed to determine the impact of DDIs in women who take an OC as well as an EI antiseizure medication known to lower the effectiveness of the OC in preventing pregnancy. The model compared the number of unintended pregnancies, the expected pregnancy outcomes, and associated costs in women taking an OC and an EI medication with a matched cohort of women who took an OC and an enzyme-neutral (EN) antiseizure medication that is known not to interact with OCs. The model perspectives were patients and third-party payers in the US. Unintended pregnancy rates, pregnancy outcomes, and cost inputs for the model were taken from published studies. RESULTS: The results of the analysis showed an estimated increase in the annual number of unintended pregnancies in the US of 503 (a change from 1151 to 1654), an increase of 44.7%, for the estimated 71,922 women currently taking an OC plus an EI medication in the US when compared with a matched cohort taking an OC plus an EN medication. This resulted in an estimated annual healthcare cost increase of $3 million, which is an increase of 5.5% in the annual costs for contraception and pregnancy care. A scenario analysis showed that the annual number of unintended pregnancies could be lower (575 vs 1654) for a matched cohort of women taking EI medications and using a copper intrauterine device, a highly effective and nonhormonal form of contraception, rather than an OC. CONCLUSIONS: Physicians treating women of reproductive age for epilepsy who wish to avoid pregnancy should consider the potential for DDIs that might result in unintended pregnancies. Thus, physicians should alert women using EI medications for epilepsy control to the increased potential for unintended pregnancies if they use OCs for contraception.

Effects of Two Educational Posters on Contraceptive Knowledge and Intentions: A Randomized Controlled Trial.

OBJECTIVE: To compare the Centers for Disease Control and Prevention's (CDC) contraceptive effectiveness poster with a more patient-centered poster on factors affecting the likelihood of using effective contraceptives. METHODS: The posters were tested in a randomized controlled trial. Women were eligible if they were aged 18-44 years, could speak and read English, were not pregnant or trying to conceive, and had engaged in vaginal intercourse in the past 3 months. An online survey administered through Amazon Mechanical Turk was used to collect baseline and immediate follow-up data on three primary outcomes: contraceptive knowledge (measured using the Contraceptive Knowledge Assessment), perceived pregnancy risk, and the effectiveness of the contraceptive the woman intended to use in the next year. Subgroup analyses were conducted in women with prior pregnancy scares, low numeracy, and no current contraceptive. Within- and between-group differences were compared for the two randomized groups. RESULTS: From January 26 to February 13, 2018, 2,930 people were screened and 990 randomized. For the primary outcomes, the only significant result was that the patient-centered poster produced a greater improvement in contraceptive knowledge than the CDC poster (P<.001). Relative to baseline, both posters significantly improved contraceptive knowledge (CDC +3.6, patient-centered +6.4 percentage points, P<.001) and a constructed score measuring the effectiveness of the contraceptive that women intended to use in the next year (CDC and patient-centered +3 percentage points, P<.01). This is equivalent to 1-17 of every 100 women who viewed a poster changing their intentions in favor of a more effective contraceptive. CONCLUSION: This study suggests that both posters educate women about contraception and may reduce unplanned pregnancy risk by improving contraceptive intentions. Of the three primary outcomes, the patient-centered poster performs significantly better than the CDC poster at increasing contraceptive knowledge. CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov, NCT03372369.

Cognitive interviews to improve a patient-centered contraceptive effectiveness poster.

OBJECTIVES: To refine the Centers for Disease Control and Prevention (CDC)'s contraceptive education poster using patient-centered design. STUDY DESIGN: We conducted cognitive interviews with 26 women aged 18-44 living in North Carolina who spoke and read English and had ever had sex. We interviewed women about both a CDC and a patient-centered poster in alternating order. Participants were contraceptive users and non-users that we selected purposively to have a range of characteristics that might influence their perspective: age, race/ethnicity, previous births and pregnancies, contraceptive method(s) used in the past three months, pregnancy intentions, and numeracy. The initial response rate for participants was 55%. We used cognitive theory to code interviews for comprehension, relevance, and acceptability, as well as design and overall preference. We structured the 26 interviews into four rounds and revised the patient-centered poster after each round to improve these measures. RESULTS: By the final round, 83% of women preferred the patient-centered poster. The majority of women favored this poster's relevance (86%), and design (100%) and ease of comprehension (86%). Women raised few concerns about the acceptability of the final version of the patient-centered poster. Women identified many issues with both posters that the researchers did not anticipate, highlighting the value of patient-centered design approaches to educational materials. CONCLUSIONS: This study refined a patient-centered poster so that its language is clear and it addresses the informational needs of its target audience. IMPLICATIONS: The Centers for Disease Control and Prevention and the Office of Population Affairs recommend that clinicians educate women about contraception. This study developed a poster that could help clinicians follow this recommendation. Before widespread implementation, more research is needed to evaluate the poster's impact on contraceptive knowledge and behaviors.

Using Behavior Over Time Graphs to Spur Systems Thinking Among Public Health Practitioners.

Public health practitioners can use Behavior Over Time (BOT) graphs to spur discussion and systems thinking around complex challenges. Multiple large systems, such as health care, the economy, and education, affect chronic disease rates in the United States. System thinking tools can build public health practitioners' capacity to understand these systems and collaborate within and across sectors to improve population health. BOT graphs show a variable, or variables (y axis) over time (x axis). Although analyzing trends is not new to public health, drawing BOT graphs, annotating the events and systemic forces that are likely to influence the depicted trends, and then discussing the graphs in a diverse group provides an opportunity for public health practitioners to hear each other's perspectives and creates a more holistic understanding of the key factors that contribute to a trend. We describe how BOT graphs are used in public health, how they can be used to generate group discussion, and how this process can advance systems-level thinking. Then we describe how BOT graphs were used with groups of maternal and child health (MCH) practitioners and partners (N = 101) during a training session to advance their thinking about MCH challenges. Eighty-six percent of the 84 participants who completed an evaluation agreed or strongly agreed that they would use this BOT graph process to engage stakeholders in their home states and jurisdictions. The BOT graph process we describe can be applied to a variety of public health issues and used by practitioners, stakeholders, and researchers.

Screening for and Treating Postpartum Depression and Psychosis: A Cost-Effectiveness Analysis.

Objectives Postpartum depression impacts 6.5-12.9% of U.S. women. Postpartum depression is associated with impaired bonding and development, marital discord, suicide, and infanticide. However, the current standard of care is to not screen women for postpartum depression. This study modeled the cost-effectiveness of physicians screening for and treating postpartum depression and psychosis in partnership with a psychiatrist. Methods This study follows a hypothetical cohort of 1000 pregnant women experiencing one live birth over a 2-year time horizon. We used a decision tree model to obtain the outcomes of screening for and treating postpartum depression and psychosis using the Edinburgh Postnatal Depression Scale. We use a Medicaid payer perspective because they cover approximately 50% of births in the U.S. The cost-effectiveness of the intervention is measured in cost per remission achieved and cost per quality-adjusted life-year (QALY) gained. We conducted both deterministic and probabilistic sensitivity analyses. Results Screening for and treating postpartum depression and psychosis produced 29 more healthy women at a cost of $943 per woman. The incremental cost-effectiveness ratios of the intervention branch compared to usual care were $13,857 per QALY gained (below the commonly accepted willingness to pay threshold of $50,000/QALY gained) and $10,182 per remission achieved. These results were robust in both the deterministic and probabilistic sensitivity analyses of input parameters. Conclusions for Practice Screening for and treating postpartum depression is a cost-effective intervention and should be considered as part of usual postnatal care, which aligns with the recently proposed recommendations from the U.S. Preventive Services Task Force.