Publications by authors named "D Casso"
Article Synopsis
- - Real-world data (RWD) can enhance the understanding of treatment effectiveness in uncontrolled trials, especially with the rising use of external controls in regulatory submissions.
- - A systematic review from 2015 to 2021 analyzes how external controls were used in submissions to major regulatory and health technology assessment agencies, highlighting challenges in data quality and consistency in evaluation.
- - The study identifies key issues, such as missing data and endpoint selection, and emphasizes the need for better collaboration between stakeholders to improve the generation of real-world evidence (RWE).
Regulators and Health Technology Assessment (HTA) bodies are increasingly familiar with, and publishing guidance on, external controls derived from real-world data (RWD) to generate real-world evidence (RWE). We recently conducted a systematic literature review (SLR) evaluating publicly available information on the use of RWD-derived external controls to contextualize outcomes from uncontrolled trials submitted to the European Medicines Agency (EMA), the US Food and Drug Administration (FDA), and/or select HTA bodies. The review identified several key operational and methodological aspects for which more detailed guidance and alignment within and between regulatory agencies and HTA bodies is necessary.
View Article and Find Full Text PDFBackground: Teriparatide, a recombinant human parathyroid hormone analogue, is associated with increased bone mineral density and a decreased risk of fractures. A dose-dependent increase in the incidence of osteosarcoma was observed in toxicology studies conducted in rats. The primary objective of this study was to estimate the incidence of osteosarcoma over a 10-year period among teriparatide-treated patients versus patients unexposed to teriparatide with osteoporosis and patients in the general population using national pharmacy dispensing data linked with data from participating state cancer registries (SCRs) in the US.
View Article and Find Full Text PDFThere is limited real-world evidence on hereditary angioedema (HAE) patient characteristics and health-care resource utilization (HCRU); in addition, pediatric patients have been described in small cohorts. To describe patient characteristics, treatment patterns, and HCRU among adult and pediatric patients treated for HAE in a large U.S.
View Article and Find Full Text PDFArticle Synopsis
- Pyruvate kinase deficiency is a rare genetic disorder linked to the PKLR gene, with over 300 mutations causing its occurrence, prompting a literature review to assess its prevalence.* -
- A systematic review of 1390 references led to four high-quality studies, revealing diagnosed PK deficiency rates between 3.2 and 8.5 cases per million, with additional data suggesting a total prevalence (diagnosed and undiagnosed) of 51 per million.* -
- The findings indicate that the prevalence of clinically diagnosed PK deficiency in Western populations is relatively low but could be significantly higher when including undiagnosed cases.*