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Background: Single-tablet combination therapies (STCTs) combine multiple drugs into one formulation, making drug administration more convenient for patients. STCTs were developed to address concerns with treatment adherence and persistence, but the impact of STCT use is not fully understood across indications. Objectives: We conducted a systematic literature review (SLR) to examine STCT-associated outcomes across 4 evidence domains: clinical trials, real-world evidence (RWE), health-related quality of life (HRQoL) studies, and economic evaluations. Methods: Four SLRs were conducted across the aforementioned domains. Included studies compared STCTs as well as fixed-dose combinations ([FDCs] of non-tablet formulations) with the equivalent active compounds and doses in loose-dose combinations (LDCs). Original research articles were included; case reports, case series, and non-English-language sources were excluded. Databases searched included EconLit, Embase, and Ovid MEDLINE® ALL. Two independent reviewers assessed relevant studies and extracted data. Conflicts were resolved with a third reviewer or consensus-based discussion. Results: In all, 109 studies were identified; 27 studies were identified in more than one SLR. Treatment adherence was significantly higher in patients receiving FDCs vs LDCs in 12 of 13 RWE studies and 3 of 13 clinical trials. All 18 RWE studies reported higher persistence with FDCs. In RWE studies examining clinical outcomes (n = 17), 14 reported positive findings with FDCs, including a reduced need for add-on medication, blood pressure control, and improved hemoglobin A1C. HRQoL studies generally reported numerical improvements with STCTs or similarities between STCTs and LDCs. Economic outcomes favored STCT use. All 6 cost-effectiveness or cost-utility analyses found FDCs were less expensive and more efficacious than LDCs. Four budget impact models found that STCTs were associated with cost savings. Medical costs and healthcare resource use were generally lower with FDCs than with LDCs. Discussion: Evidence from RWE and economic studies strongly favored STCT use, while clinical trials and HRQoL studies primarily reported similarity between STCTs and LDCs. This may be due to clinical trial procedures aimed at maximizing adherence and HRQoL measures that are not designed to evaluate drug administration. Conclusions: Our findings highlight the value of STCTs for improving patient adherence, persistence, and clinical outcomes while also offering economic advantages.
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Background: The complex nature of asthma has resulted in a poor understanding of its epidemiology, particularly in low-and middle-income countries (LMIC). Clinical subgroups, such as patients with severe asthma, eosinophilic asthma, allergic rhinitis, or nasal polyps, experience additional barriers to care. Methods: Prevalence estimates for asthma and key clinical subgroups were extracted from the Global Burden of Diseases, Injuries, and Risk Factors Study 2019 and from a targeted literature review conducted through PubMed in October of 2021. National estimates were calculated and the roles of potential explanatory factors were explored through qualitative analysis. Results: In total, 162 publications from 69 countries were included. Across continents, asthma prevalence values ranged from 3.44% (Asia), 3.67% (Africa), 4.90% (South America), 5.69% (Europe), 8.29% (North America), to 8.33% (Oceania). Globally, of those with asthma, 26.70% had severe asthma, 30.99% had eosinophilic asthma, 48.95% had allergic rhinitis, and 7.0% to 25.40% had nasal polyps. Countries with higher air quality, income status, and healthcare access and quality reported a higher asthma prevalence. Conclusion: Asthma prevalence values were low in LMICs, potentially indicating health system deficiencies resulting in low diagnosis and reporting. The prevalence of eosinophilic asthma and severe asthma phenotypes was high in many countries, although the prevalence estimates of all asthma subgroups were quite variable.
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Aim: To assess the use and acceptability of real-world evidence (RWE) in lung and hematologic cancer appraisals. Materials & methods: A review of appraisals published by National Institute for Health and Care Excellence (NICE) in the UK was conducted. A total of 20 case studies employing RWE were identified and compared across five additional health technology assessment agencies: Scottish Medicines Consortium (SMC) (Scotland), CADTH (Canada), INESSS (Quebec), HAS (France) and IQWiG (Germany). Results: Of 80 RWE references from 20 case studies from NICE, 67 were identified in the respective CADTH submissions, 46 in IQWiG, 37 in INESSS, 37 in HAS, and 33 in SMC. NICE had the highest RWE acceptance rate (90%), followed by HAS (88%), SMC (82%), INESSS (73%), IQWiG (68%) and CADTH (67%). Conclusion: RWE was generally accepted by respective committees, allowing improved access to innovative treatments.
Use of real-world evidence for assessing the value of cancer treatments Health technology assessment (HTA) is a process used to decide whether a drug works well enough to be worth paying for. Most drugs have data showing how well they work from special studies called clinical trials. Sometimes a manufacturer also has evidence of a drug or disease that is not from a clinical trial but from the real world. This review discusses how real-world evidence (RWE) is being used for HTAs of new lung and blood cancer therapies. We reviewed twenty HTA submissions for new therapies. All twenty were submitted to these agencies: National Institute for HealthCare and Excellence (NICE; UK), Scottish Medicines Consortium (SMC; Scotland), Canadian Agency for Drugs and Technologies in Health (CADTH; Canada), National Institute of Excellence in Health and Social Services (INESSS; Quebec), French National Authority for Health (HAS; France) and Institute for Quality and Efficiency in HealthCare (IQWiG; Germany). RWE was often used to describe the type of patient that needs the new therapy. RWE was also used to show the cost of the treatment and how well the treatment worked in relation to its cost. It was also used to show how well the new therapy works compared with other treatments. Most of the RWE was accepted by the agencies. High-quality RWE in relevant patients helped support access to new treatments.