Health Economic Studies of Surfactant Replacement Therapy in Neonates with Respiratory Distress Syndrome: A Systematic Literature Review.

BACKGROUND: Neonatal respiratory distress syndrome (RDS) is one of the most common problems for preterm infants, and symptoms include tachypnoea, grunting, retractions and cyanosis, which occur immediately after birth. Treatment with surfactants has reduced morbidity and mortality rates associated with neonatal RDS. OBJECTIVE: The objective of this review is to describe the treatment costs, healthcare resource utilization (HCRU) and economic evaluations of surfactant use in the treatment of neonates with RDS. METHODS: A systematic literature review (SLR) was performed to identify available economic evaluations and costs associated with neonatal RDS. Electronic searches were conducted in Embase, MEDLINE, MEDLINE In-Process, NHS EED, DARE and HTAD to identify studies published between 2011 and 2021. Supplementary searches of reference lists, conference proceedings, websites of global health technology assessment bodies and other relevant sources were conducted. Publications were screened by two independent reviewers for inclusion and followed the population, interventions, comparators and outcomes framework eligibility criteria. Quality assessment of the identified studies was performed. RESULTS: Eight publications included in this SLR met all eligibility criteria: three conference abstracts and five peer-reviewed original research articles. Four of these publications evaluated costs/HCRU, and five (three abstracts and two peer-reviewed articles) investigated economic evaluations (two from Russia, and one each from Italy, Spain and England). The main cost drivers and causes of increased HCRU were invasive ventilation, duration of hospitalization and RDS-associated complications. There were no significant differences in neonatal intensive care unit (NICU) length of stay or NICU total costs between infants treated with beractant (Survanta®), calfactant (Infasurf®) or poractant alfa (Curosurf®). However, treatment with poractant alfa was associated with reduced total costs compared with no treatment, continuous positive airway pressure (CPAP) alone or calsurf (Kelisu®), due to shorter duration of hospitalization and fewer complications. Early use of the surfactant after birth was more clinically effective and cost-effective than late intervention in infants with RDS. Poractant alfa was found to be cost-effective and cost-saving compared to beractant for the treatment of neonatal RDS in two Russian studies. CONCLUSION: There were no significant differences in NICU length of stay or NICU total costs between surfactants evaluated for treating neonates with RDS. However, early use of surfactant was found to be more clinically effective and cost-effective than late treatment. Treatment with poractant alfa was found to be cost-effective versus beractant and cost-saving compared with CPAP alone or beractant or CPAP in combination with calsurf. Limitations included the small number of studies, the geographic scope of the studies and the retrospective study design of the cost-effectiveness studies.

A systematic literature review of the epidemiology, quality of life, and economic burden, including disease pathways and treatment patterns of relapsed/refractory classical Hodgkin lymphoma.

INTRODUCTION: A systematic literature review was conducted to understand disease burden in patients with relapsed/refractory classical Hodgkin lymphoma (R/R cHL). AREAS COVERED: Embase®, PubMed®, and Cochrane were searched for records from 2001 to 2020 in accordance with PRISMA guidelines. A total of 13,257 abstracts and 1731 papers were screened; 144 studies were identified. cHL accounted for 0.5% of all cancers, with 4‒66.7% of cases progressing to R/R disease (studies with >500 patients); this range varied across countries. Quality of life (QoL) was assessed via EORTC-QLQ-C30 (n = 7), EQ-5D (n = 5), SF-36 (n = 3), FACIT-F (n = 1), and MFI (n = 1) questionnaires. In general, pembrolizumab and other programmed cell death protein-1 inhibitors improved QoL scores. Brentuximab vedotin showed mixed outcomes, and high-dose therapy (HDT) and autologous stem-cell rescue (ASCR) showed worsening functionality/symptoms. Economic burden studies (n = 21) reported increased costs and health care resource in R/R cHL. Across clinical guidelines (n = 13) and treatment pattern studies (n = 46), HDT followed by ASCR was recommended as initial R/R cHL treatment. Pembrolizumab and nivolumab were frequently recommended for patients relapsing following HDT/ASCR. EXPERT OPINION: Despite recent treatment advances, patients with R/R cHL continue to report reduced quality of life. Unmet medical needs remain, particularly with respect to slowing disease progression and identifying the best treatment approaches for improving longer-term survival and quality of life. This systematic literature review provides an extensive overview of the current landscape in patients with R/R cHL, focusing on four key areas: epidemiology, QoL, economic burden, and disease management. These findings will be useful to those with an interest in managing patients with R/R cHL or in designing future studies.

Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews.

Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disorder characterised by reduced levels of circulating alpha-1 antitrypsin and an increased risk of lung and liver disease. Recent reviews of AATD have focused on diagnosis, epidemiology and clinical management; comprehensive reviews examining disease burden are lacking. Therefore, we conducted literature reviews to investigate the AATD disease burden for patients, caregivers and healthcare systems. Embase, PubMed and Cochrane libraries were searched for AATD publications from database inception to June 2021, in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Most published AATD studies were small and short in duration, with variations in populations, designs, measures and outcomes, complicating cross-study comparisons. AATD was associated with significant pulmonary and hepatic morbidity. COPD, emphysema and bronchiectasis were common lung morbidities, where smoking was a key risk factor. Fibrosis and steatosis were the most common liver complications reported in patients with a PiZ allele. Health status analyses suggested a poorer quality of life for AATD patients diagnosed with COPD versus those with non-AATD-associated COPD. The burden for caregivers included loss of personal time due to caring responsibilities, stress and anxiety. AATD was also associated with high direct medical costs and healthcare resource utilisation.

Health-related quality of life and economic burden of chronic lymphocytic leukemia in the era of novel targeted agents.

OBJECTIVE: To quantify the health-related quality of life (HRQoL) and economic burden of chronic lymphocytic leukemia (CLL). METHODS: Studies were searched through Embase, MEDLINE, PubMed, and Cochrane Library, as well as conference abstracts (1 January 2000-2 June 2019). RESULTS: Overall, 12 and 17 primary studies were included in the HRQoL and economic burden reviews, respectively. Patients with CLL reported impairment in various quality of life domains when compared with healthy controls, including fatigue, anxiety, physical functioning, social functioning, depression, sleep disturbance, and pain interference. Key factors associated with a negative impact on the HRQoL burden of CLL included female gender, increased disease severity, and the initiation of multiple lines of therapy. Economic burden was assessed for patients with CLL based on disease status and the treatment regimen received. The main cost drivers related to CLL were outpatient and hospitalization-related costs, primarily incurred as a result of chemo/chemoimmunotherapy, adverse events (AEs), and disease progression. Treatment with targeted agents, i.e. ibrutinib and venetoclax, was associated with lower medical costs than chemoimmunotherapy, although ibrutinib was associated with some increased AE costs related to cardiac toxicities. Cost studies of targeted agents were limited by short follow-up times that did not capture the full scope of treatment costs. CONCLUSIONS: CLL imposes a significant HRQoL and economic burden. Our systematic review shows that an unmet need persists in CLL for treatments that delay progression while minimizing AEs. Studies suggest targeted therapies may reduce the economic burden of CLL, but longer follow-up data are needed.

Development of a fast and economical genotyping protocol for bovine leukocyte adhesion deficiency (BLAD) in cattle.

Fast and economical means of assaying SNP's are important in diagnostic assays, especially when a large number of animals have to be screened for a genetic disease. This study was aimed at the development of a fast and economical screening assay for bovine leukocyte adhesion deficiency (BLAD) which is an important genetic disease of cattle industry. Four primers were designed where the outer primers amplify a 354 bp amplicon of CD18 gene carrying the polymorphism responsible for BLAD. The specifically designed inner primers in conjunction with the modified reaction mixture and cyclic conditions ensured amplification of either of wild or mutated alleles. Together with outer primers, the inner primers generated typical banding pattern in agarose gel which discriminated the normal animal against the carrier. We successfully used this protocol in 200 bulls for genotyping the BLAD allele which confirmed by sequencing, showing a cent percentage concordance. With the developed assay the need for restriction digestion or use of costly equipment viz. real time PCR was eliminated. This genotyping assay ensured fast and economical genotyping and could be adopted in every laboratory with a minimum equipment requirement of thermocycler and gel documentation system.

Midwifery 2030: a woman's pathway to health. What does this mean?

The 2014 State of the World's Midwifery report included a new framework for the provision of woman-centred sexual, reproductive, maternal, newborn and adolescent health care, known as the Midwifery2030 Pathway. The Pathway was designed to apply in all settings (high-, middle- and low-income countries, and in any type of health system). In this paper, we describe the process of developing the Midwifery2030 Pathway and explain the meaning of its different components, with a view to assisting countries with its implementation. The Pathway was developed by a process of consultation with an international group of midwifery experts. It considers four stages of a woman's reproductive life: (1) pre-pregnancy, (2) pregnancy, (3) labour and birth, and (4) postnatal, and describes the care that women and adolescents need at each stage. Underpinning these four stages are ten foundations, which describe the systems, services, workforce and information that need to be in place in order to turn the Pathway from a vision into a reality. These foundations include: the policy and working environment in which the midwifery workforce operates, the effective coverage of sexual, reproductive, maternal, newborn and adolescent services (i.e. going beyond availability and ensuring accessibility, acceptability and high quality), financing mechanisms, collaboration between different sectors and different levels of the health system, a focus on primary care nested within a functional referral system when needed, pre- and in-service education for the workforce, effective regulation of midwifery and strengthened leadership from professional associations. Strengthening of all of these foundations will enable countries to turn the Pathway from a vision into reality.

Systematic review of humanistic and economic burden of symptomatic chronic obstructive pulmonary disease.

BACKGROUND: An understanding of the humanistic and economic burden of individuals with symptomatic chronic obstructive pulmonary disease (COPD) is required to inform payers and healthcare professionals about the disease burden. OBJECTIVES: The aim of this systematic review was to identify and present humanistic [health-related quality of life (HRQoL)] and economic burdens of symptomatic COPD. METHODS: A comprehensive search of online databases (reimbursement or claims databases/other databases), abstracts from conference proceedings, published literature, clinical trials, medical records, health ministries, financial reports, registries, and other sources was conducted. Adult patients of any race or gender with symptomatic COPD were included. Humanistic and economic burdens included studies evaluating HRQoL and cost and resource use, respectively, associated with symptomatic COPD. RESULTS: Thirty-two studies reporting humanistic burden and 74 economic studies were identified. Symptomatic COPD led to impairment in the health state of patients, as assessed by HRQoL instruments. It was also associated with high economic burden across all countries. The overall, direct, and indirect costs per patient increased with an increase in symptoms, dyspnoea severity, and duration of disease. Across countries, the annual societal costs associated with symptomatic COPD were higher among patients with comorbidities. CONCLUSIONS: Symptomatic COPD is associated with a substantial economic burden. The HRQoL of patients with symptomatic COPD is, in general, low and influenced by dyspnoea.

Infection control in delivery care units, Gujarat state, India: a needs assessment.

BACKGROUND: Increasingly, women in India attend health facilities for childbirth, partly due to incentives paid under government programs. Increased use of health facilities can alleviate the risks of infections contracted in unhygienic home deliveries, but poor infection control practices in labour and delivery units also cause puerperal sepsis and other infections of childbirth. A needs assessment was conducted to provide information on procedures and practices related to infection control in labour and delivery units in Gujarat state, India. METHODS: Twenty health care facilities, including private and public primary health centres and referral hospitals, were sampled from two districts in Gujarat state, India. Three pre-tested tools for interviewing and for observation were used. Data collection was based on existing infection control guidelines for clean practices, clean equipment, clean environment and availability of diagnostics and treatment. The study was carried out from April to May 2009. RESULTS: Seventy percent of respondents said that standard infection control procedures were followed, but a written procedure was only available in 5% of facilities. Alcohol rubs were not used for hand cleaning and surgical gloves were reused in over 70% of facilities, especially for vaginal examinations in the labour room. Most types of equipment and supplies were available but a third of facilities did not have wash basins with "hands-free" taps. Only 15% of facilities reported that wiping of surfaces was done immediately after each delivery in labour rooms. Blood culture services were available in 25% of facilities and antibiotics are widely given to women after normal delivery. A few facilities had data on infections and reported rates of 3% to 5%. CONCLUSIONS: This study of current infection control procedures and practices during labour and delivery in health facilities in Gujarat revealed a need for improved information systems, protocols and procedures, and for training and research. Simply incentivizing the behaviour of women to use health facilities for childbirth via government schemes may not guarantee safe delivery.