Orphan-Drug Applications

But if pharmaceutical companies focused exclusively on these major diseases, millions of people with very serious but less widespread conditions would be left without help or hope. The solution, of course, is for the government to provide incentives that supplement the potential sales value of an important treatment for a relatively obscure disease, thus encouraging development in the absence of market blockbuster potential. In the United States, the mechanism for that encouragement is orphan-drug designation. 

A drug qualifies for orphan status if credible documentation, with authoritative references, is submitted to prove that 

Guidebook for Drug Regulatory Submissions, by Sandy Weinberg Copyright 2009 John Wiley Sons, Inc. 

Designation as an orphan drug carries a number of incentives, designed to encourage development, specifically the following  

Orphan designation carries a seven-year market exclusivity. 

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An orphan-drug application and an IND both receive responses in about 30 days. Try to schedule submissions so that the response date will not fall within planned vacations or critical international travel. And both submissions (and many others) require an annual update report it is best to stagger report due dates to avoid complications in the future. 

For meeting requests, orphan-drug applications, INDs, NDAs, 505b(2) NDAs, Abbreviated New Drug Applications (ANDAs), Orphan Annual Reports, and Annual Reports, the tools, checklists, and FDA guidelines are provided. With this tool kit, your organization can regain control of a rational, predictable submissions process. 

Orphan-drug applications for prescription drug products are not subject to a fee, unless the human drug application includes an indication other than the rare disease. In addition, Orphan designation permits application (but does not guarantee) for exemption from FDA annual product and establishment fees under a section 736(d)(1) of the Federal Food, Drug, and Cosmetic Act waiver. 

Review of an orphan-drug application is generally completed within 30 days of submission. The process begins with an administrative review to assure the application is complete, conforms to the required format, and clearly designates the proposed drug and disease or condition. 

This checklist is intended for use in the preparation and submission of orphan-drug applications. It is recommended that, prior to transmission to the FDA, a second internal review should be conducted by an individual or department not involved in the preparation of the submission (presumably Quality Assurance). 

The checklist was developed through discussions with consultants and Quality Assurance directors and has been field-tested in final form with five successful Orphan-Drug Application submissions. 

Requests and Related Submissions outlines the evolving requirements for electronic submission of orphan-drug applications using the (still pending) ESG and physical media (CD-ROM). 

Comment 2) The comment suggested that our estimate of 130 h to prepare and submit an orphan-drug application is too high. 

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