Clinical pharmacology as a cornerstone of orphan drug development

Bashaw, Edward D.; Huang, Shiew‐Mei; Coté, Timothy R.; Pariser, Anne; Garnett, Christine; Burckart, Gilbert J.; Zhang, Lei; Men, Angela; Le, Christine D.; Charlab, Rosane; Gobburu, Joga; Lesko, Lawrence J.

doi:10.1038/nrd3595

Cited by 25 publications

(27 citation statements)

References 5 publications

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“…This proposal implies that much of the understanding of PKPD, including the characterization of resistance development, can indeed be gained from experimental systems. In addition, more efficient use of clinical pharmacology data can limit the number of trial subjects, as advocated in orphan drug development (Schmidt et al, 2008;Bashaw et al, 2011;Bassetti et al, 2011).…”

Section: F Drug Development Of Antibioticsmentioning

confidence: 99%

“…It has been suggested that antibiotic drug development should be based on development strategies similar to those for orphan drugs (Bashaw et al, 2011) where quantitative approaches have a key role in decision making during all phases of clinical development. Furthermore, with model-based analysis, the number of patients needed can be reduced substantially while preserving the statistical power of the study (Vong et al, 2012).…”

Section: Future Perspectivesmentioning

confidence: 99%

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Pharmacokinetic-Pharmacodynamic Modeling of Antibacterial Drugs

2013

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Section: F Drug Development Of Antibioticsmentioning

confidence: 99%

Section: Future Perspectivesmentioning

confidence: 99%

Pharmacokinetic-Pharmacodynamic Modeling of Antibacterial Drugs

2013

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“…Considering the regulatory flexibility exercised by the FDA (and by similar agencies outside of the US) in regard to marketing approvals where orphan drugs are concerned (Bashaw et al 2011), approval of plant-derived GCD seems a low-hanging fruit. In fact, a similar breakthrough came very recently with the emergency approval of a plant-derived monoclonal antibody cocktail to save the lives of two gravely ill physicians who contracted Ebola virus while treating patients in Africa (Bishop 2015; McCarthy 2014; Qiu et al 2014; Rybicki 2014).…”

mentioning

confidence: 99%

Molecular pharming’s foot in the FDA’s door: Protalix’s trailblazing story

Mor

2015

Biotechnol Lett

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Objectives This short commentary examines the factors that led to Food and Drug Administration’s approval of the first plant-derived biologic. Results In 2012, the first plant-derived protein pharmaceutical (biologic) was approved for commercial use in humans. The product, a recombinant form of human β-glucocerebrosidase marketed as ELELYSO, was developed by Protalix Biotherapeutics (Carmiel, Israel). The foresight to select this particular therapeutic product for development, flawless production pipeline, and serendipity seem to provide the key in explaining how ELELYSO became the first plant-derived biologic to achieve approval by Food and Drug Administration. Conclusions While the circumstances that enabled Protalix and its scientists to become the first to arrive at this historic milestone are perhaps unique, it is anticipated that more biologics will follow suit in winning regulatory endorsement.

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“…Before resources are dedicated to rare diseases, well‐defined means to overcome the obstacles of drug development should be delineated. Some of these strategies applicable to orphan drug development have been proposed in recent publications . These include cross‐subsidies, risk sharing, multiple indications in development, quantitative and Bayesian adaptive analyses, flexible and evidence‐based designs, surrogate endpoint verification, formation of rare disease frameworks, up‐to‐date formulation technologies, and potentially the pay for performance, conditional approvals, and the use of consortium/multiarm studies involving several companies.…”

mentioning

confidence: 99%