The Glaucoma Quality of Life-15 Questionnaire correlated well with objective measures of visual function and discriminated between quality of life in patients with glaucoma and subjects without glaucoma.

Patient-reported outcomes (PROs) associated with insulin therapy are potentially important determinants of adherence to diabetes management programs. This article reviews published evidence of PROs over the past 3 decades in patients with type 1 diabetes (T1D) and/or type 2 diabetes (T2D) who used vial and syringe for insulin delivery compared to those who used insulin pens. Based on predetermined selection criteria, articles were identified through a search of primary sources published from January 1980 to February 2008. Two independent reviewers determined study eligibility and performed a detailed evaluation of the articles that met the selection criteria. Of the 124 articles screened, 41 met selection criteria. Approximately 75% of the selected articles were published between 1990 and 2008, and a majority (78%) of the research studies was conducted outside the United States. Most (>75%) of the studies evaluated male and female patients with T1D and/or T2D and mean ages around 45 years. Studies used varied comparative study designs with follow-up periods ranging from 2 weeks to 5 years. The PROs assessed in these articles included preference, acceptability, treatment satisfaction, ease of use, convenience, injection pain, handling, and dosing. Most articles (n = 36) showed more favorable PROs for insulin pen users compared to vial and syringe users. These findings have potential clinical and policy implications for patients, diabetes care providers, and/or payers to make evidence-based decisions regarding ways to facilitate initiation and management of insulin therapy.

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Aim: To provide an assessment of published literature on the demographic representation in Phase I trials of biopharmaceutical oncology agents. Materials & methods: We conducted a rapid evidence assessment to identify demographic representation reported in Phase I clinical trials for biopharmaceutical oncology agents published in 2019. Results: Globally, the population was predominantly White/Caucasian (62.2%). In the USA, the distribution was heavily skewed toward White/Caucasian (84.2%), with minimal representation of Blacks/African–Americans (7.3%), Asians (3.4%), Hispanics/Latinos (2.8%) or other race/ethnicity groups. Conclusion: Our data highlight that Phase I oncology trials do not reflect the population at large, which may perpetuate health disparities. Further research is needed to understand and address barriers to participation, particularly among under-represented groups

Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.