Wednesday, May 19, 2010

Country Specific Orphan Drug Development and Clinical Submission

Country specific regulatory requirements have been enacted to encourage the development and clinical submission of orphan drugs for off-label and compassionate use regarding patients with life threatening disease. Countries involved in the enactment are Australia, Canada, Germany, Great Britain, France, Netherlands, Switzerland and the US.

Each country participates in ICH. ICH guided, CTD and eCTD clinical submission components, data and documentation presentations, statistical analyses and format are for the most part generic. However, country specific CTD and eCTD clinical submission differences do exist. I strongly recommend early communication with each regulatory agency to ensure country specific CTD and eCTD clinical submission compliance and to agree on a definitive, comprehensive TOC for your orphan drug filing.

A question often asked by pharma pertains to country incentives. Due to the high cost of orphan drug development, yes, there are country specific incentives to encourage the development of orphan drugs. Canada provides incentives for orphan drug development to pharmaceutical companies, clinical investigators, IRBs, interested groups and otherwise. Europe and the US offer the greatest incentives for orphan drug development whereas, Australia and Switzerland provide cooperative incentives with other countries.

In all countries, as always, patient safety is foremost. Orphan drug safety and efficacy filings are reviewed with regulatory rigor by all agencies. Data is assessed for quality, accuracy and integrity irrespective of country. Documentation is reviewed for consistency. High quality data and documentation is standard and expected by FDA and regulatory agencies worldwide.

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