Impact of patient support programmes among patients with severe asthma treated with biological therapies: a systematic literature review and indirect treatment comparison.

INTRODUCTION: Effective treatment of severe asthma requires patient adherence to inhaled and biological medications. Previous work has shown that patient support programmes (PSP) can improve adherence in patients with chronic diseases, but the impact of PSPs in patients with severe asthma treated with biologics has not been thoroughly investigated. METHODS: We conducted a systematic literature review to understand the impact of PSPs on treatment adherence, asthma control and health-related quality of life (HRQoL) in patients with severe asthma. Embase, MEDLINE and EconLit databases were searched for studies published from 2003 (the year of the first biological approval for severe asthma) to June 2023 that described PSP participation among patients with severe asthma on biological treatment. Direct pooling of outcomes was not possible due to the heterogeneity across studies, so an indirect treatment comparison (ITC) was performed to determine the effect of PSP participation on treatment discontinuation. The ITC used patient-level data from patients treated with benralizumab either enrolled in a PSP (VOICE study, Connect 360 PSP) or not enrolled in a PSP (Benralizumab Patient Access Programme study) in the UK. FINDINGS: 25 records of 21 studies were selected. Six studies investigated the impact of PSPs on treatment adherence, asthma control or HRQoL. All six studies reported positive outcomes for patients enrolled in PSPs; the benefits of each PSP were closely linked to the services provided. The ITC showed that patients in the Connect 360 PSP group were less likely to discontinue treatment compared with the non-PSP group (OR 0.26, 95% CI 0.11 to 0.57, p<0.001). CONCLUSIONS: PSPs contribute to positive clinical outcomes in patients with severe asthma on biological treatment. Future analyses will benefit from thorough descriptions of PSP services, and study designs that allow direct comparisons of patient outcomes with and without a PSP.

Undertreatment in patients with advanced urothelial cancer: systematic literature review and meta-analysis.

Aim: To assess rates of no systemic treatment (NST), attrition across lines of therapy, and factors influencing treatment selection in patients with locally advanced or metastatic urothelial cancer (la/mUC). Methods: Systematic literature review to identify real-world studies reporting NST or attrition rates in la/mUC from 2017-2022 (including data reported since 2015). Results: Of 2439 publications screened, 29 reported NST rates, ranging from 40-74% in eight European-based studies, 14-60% in 12 US-based studies, and 9-63% in nine studies in other locations (meta-analysis estimate, 39%). Factors associated with NST or no second-line therapy included older age, female sex, poor performance status, poor renal function and distant metastases. Conclusion: A substantial proportion of patients with la/mUC do not receive guideline-recommended treatment.

People with advanced bladder cancer have a short survival. Bladder cancer is called advanced when it has spread outside of the urinary tract. Several drug treatments are available for people with advanced bladder cancer. However, sometimes people do not receive any drug treatment. We looked at published studies to see how many people with advanced bladder cancer did not receive any drug treatment and the reasons why. We also looked at how long people lived with or without drug treatment. We found that many people with advanced bladder cancer did not receive drug treatment. The number of people who received no drug treatment varied in studies from different countries. People who were older, were female, had poor health or kidney problems, or had cancer that had spread to other parts of the body were less likely to receive drug treatment. People who did not receive drug treatment lived for an average of 2 to 7 months, compared with 9 to 35 months for people who received drug treatment. More studies are needed to investigate the reasons why drug treatment is sometimes not used in people with advanced bladder cancer who could receive treatment, so that more people can benefit from available treatments.

Real-world evidence in lung and hematologic oncology health technology appraisals: a review of six assessment agencies.

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.

Comparative effectiveness and safety of pharmacological and non-pharmacological interventions for insomnia: an overview of reviews.

BACKGROUND: This review aimed to assess the existing evidence regarding the clinical effectiveness and safety of pharmacological and non-pharmacological interventions in adults with insomnia and identify where research or policy development is needed. METHODS: MEDLINE, Embase, PsycINFO, The Cochrane Library, and PubMed were searched from inception until June 14, 2017, along with relevant gray literature sites. Two reviewers independently screened titles/abstracts and full-text articles, and a single reviewer with an independent verifier completed charting, data abstraction, and quality appraisal. RESULTS: A total of 64 systematic reviews (35 with meta-analysis) were included after screening 5024 titles and abstracts and 525 full-text articles. Eight of the included reviews were rated as high quality using the Assessment of Multiple Systematic Reviews 2 (AMSTAR2) tool, and over half of the included articles (n = 40) were rated as low or critically low quality. Consistent evidence of effectiveness across multiple outcomes based on more than one high- or moderate quality review with meta-analysis was found for zolpidem, suvorexant, doxepin, melatonin, and cognitive behavioral therapy (CBT), and evidence of effectiveness across multiple outcomes based on one high-quality review with meta-analysis was found for temazepam, triazolam, zopiclone, trazodone, and behavioral interventions. These interventions were mostly evaluated in the short term (< 16 weeks), and there was very little harms data available for the pharmacological interventions making it difficult to evaluate their risk-benefit ratio. CONCLUSIONS: Assuming non-pharmacological interventions are preferable from a safety perspective CBT can be considered an effective first-line therapy for adults with insomnia followed by other behavioral interventions. Short courses of pharmacological interventions can be supplements to CBT or behavioral therapy; however, no evidence regarding the appropriate duration of pharmacological therapy is available from these reviews. SYSTEMATIC REVIEW REGISTRATION: PROSPERO CRD42017072527.