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eved that industry perceptions from this study can support EMA with improved regulatory decision making to benefit public health.For topically applied over-the-counter (OTC) products, the association of unwanted systemic exposure and adverse events may be difficult to ascertain without a recognition or determination of in vivo absorption. Evaluation of skin permeability using a validated in vitro permeation methodology can provide important information for both initial formulation selection and reformulation during the product life cycle. Additionally, a comparison of permeation rates between formulations using a validated methodology could reduce the number of nonclinical studies needed as part of reformulation. However, many in vitro permeation tests (IVPTs) have produced results with high variability and low reproducibility between study sites. It is unclear if this is due to a lack of a standardized protocol, or lack of control of multiple key experimental factors including skin source, preparation, receptor fluid, and study design. This review presents the authors perspective on the potential regulatory utility of IVPT and proposes steps to improve the accuracy and reproducibility of IVPT. The focus of this review is on topical dermatologic drugs with an initial emphasis on the OTC marketplace where reformulations are more common. Because of the increasing demand for drugs addressing life-threatening and rare diseases, regulatory agencies have developed a variety of accelerated regulatory pathways. These programs are aimed at prioritizing the most promising drug candidates for diseases lacking satisfactory treatments. The most prominent accelerated programs introduced have been Breakthrough-Therapy Designation (BTD) in the United States, Priority Medicine (PRIME) in the European Union and Sakigake in Japan. This article reviews these designations and looks at differences in how they are granted across the 3 jurisdictions focusing on neuroscience and oncology. Our objective was to analyze BTD, PRIME, and Sakigake approvals between 2012 and 2019 with a focus on numerical disparities of designations granted between the 2 therapeutic areas. A search of public sources pertaining to topics of BTD, PRIME, and Sakigake was undertaken. This analysis revealed that 48% of BTD were granted in oncology, while neuroscience received 8% of these designations, for PRIME designations were 27% received by oncology and 15% by neuroscience and in Japan, 50% of Sakigake were granted to oncology and 22% to neuroscience products. Given the global nature of drug development and relative similarity of these regulatory mechanisms, there is an apparent disparity between the US granting special status at 61 (oncology neuroscience) and both the EU and Japan granting at 21. This disproportionate ratio is likely impacted by multifactorial issues; however, this difference is worth further investigation.Given the global nature of drug development and relative similarity of these regulatory mechanisms, there is an apparent disparity between the US granting special status at 61 (oncology neuroscience) and both the EU and Japan granting at 21. This disproportionate ratio is likely impacted by multifactorial issues; however, this difference is worth further investigation. Typically, regulatory requirements include 2 confirmatory studies, each at a 1-sided .025 significance level, for a medicine to be approved for a specific indication. When the same medicine has been approved in related indications, 1 confirmatory study at a 1-sided .025 significance level could constitute adequate evidence of efficacy for a new indication. This article does not contain any studies with human or animal subjects performed by any of the authors. For multiple related indications developed simultaneously to constitute sufficient evidence of clinical efficacy, the combined-studies significance level can be set at the same level as if those indications are developed sequentially. This article establishes possible strategies to develop a few related indications at the same time for marketing registration approval, maintaining a desired combined-studies significance level; for example, 1-sided .0000156 for 2 indications, with 1 option having each indication assessed with 1 confirmatory study at .00395 significance level. It is possible to develop a few indications at the same time for marketing registration approval, where the combinedstudies significance level is less stringent than that of the usual paradigm with 2 confirmatory studies each at 1-sided .025 significance level for every indication.It is possible to develop a few indications at the same time for marketing registration approval, where the combinedstudies significance level is less stringent than that of the usual paradigm with 2 confirmatory studies each at 1-sided .025 significance level for every indication. Despite increasing interest and focus on patient-centric approaches to drug development, there might still be divergent views between key stakeholders in how to perceive patient involvement and how possibly divergent views influence the role of patients in the drug development process. The objective of this study is to explore how the perception of patient organizations, pharmaceutical companies, and regulatory agencies influence the role of patients in drug development. A qualitative interview study based on 12 semi-structured interviews with representatives from the 3 stakeholders. Interviews were transcribed, and data were analyzed using a social constructivist approach in the form of systematic text condensation. Three main perceptions of patient involvement were identified "a way to improve quality of life," "a way to avoid business failure," and "a way to foster a faster drug approval process." Transparency, trust, and clarification of expectations and roles were factors perceived as prerequisites development. Thus, the pharmaceutical industry appears to be the largest influencer with regard to patients' role in drug development.Clinical trials are evolving to innovative designs and capabilities. Clinical outcomes from pivotal trials are the backbone of good marketing practices. Novel study designs included in product labels and the uptick of innovative technology foreshadow a crescendo of patient empowerment not only in clinical trials, but also in the real-world setting. https://www.selleckchem.com/products/elexacaftor.html The following review will initially explore the dynamic relationship between clinical development and commercial teams. How clinical development is evolving to increase patient-focused drug development and regulatory review will then be reviewed via recent legislation; the 21st Century Cures Act is one glimpse at innovative inclusion of the patient perspective via patient experience data, related information, and real-world evidence. Trends in direct-to-consumer technology such as digital health will be also appraised. Predictions are made on how the aforementioned advances may create challenges and opportunities to promotional practices, including crosscollaboration of marketing teams and planning for the next level of patient empowerment.
