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Rare and Orphan Diseases at Target Health Inc.

 

One of Target Health’s expertise is in the area of rare and orphan diseases. Our expertise is not just in obtaining Orphan Drug Designations in multiple areas, but also the regulatory strategy to optimize the path to the market. For one program, we worked with our client to propose to FDA to go directly from Phase 1 in normal volunteers to a pivotal trial with 30 patients with just 2 doses of the drug and no placebo arm. The drug was approved and the company saved at least 2 years by not having to do a phase 2 study. In additional, the NDA was a rolling submission, so that when the last patient visit occurred in September, the final NDA was submitted in December, just 3 months later. Of course, the planning of the submission started a year in advance which made for a smooth transition. A recent Orphan Drug Designation was approved for a rare epilepsy in children and a Fast Track Designation cleared for a metabolic disease.

 

For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website.

 

Joyce Hays, Founder and Editor in Chief of On Target

Jules Mitchel, Editor

 

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