Orphan-drug designation application

Response 2) We disagree with the comment because some sponsors have more experience with submitting Orphan-Drug Designation applications/ requests and, therefore, may require less human resource hours to compile all... 

The information requested from respondents represents, for the most part, an accounting of information already in the possession of the applicant. It is estimated, based on frequency of requests over the past five years, that 171 persons or organizations per year will request orphan-drug designation and none will request formal recommendations on design of preclinical or clinical studies. 

The estimated 130h pertains to Sec. Sec. 316.20,316.21, and 316.26. These apply primarily to initial applications/requests seeking orphan-drug designation. Many applications/requests received in the Office of Orphan Products Development contain multiple volumes include an exact duplicate copy of the original and may include 50 or more documented references. Additional information is requested when an application/request is denied. The sponsor usually supplies the requested information in the form of an amendment. 

It is obvious that arthritis, epilepsy, depression, asthma and other similar diseases are not rare and drugs to treat them do not qualify for orphan drug designation. But would a medically plausible subset of each disease qualify as an orphan indication if there were fewer than 200 000 patients with, for example, a severe form of the disease The FDA s principle in addressing this common question is to ask the question Could (and would) patients with less severe forms of the disease use the new treatment If so, then the FDA says that the indication is not a true orphan and usually denies the application for orphan designation. 

Sponsors need to notify the EMEA of the intent to file an application for orphan designation. A presubmission meeting with COMP will be arranged, if viewed as desirable. The submission is usually validated within 10 days of receipt, and then COMP will assess the submission within a further 60-90 days. When the COMP has adopted an opinion, it is send to the European Commission, whose binding decision is issued within a further 30 days. Orphan drug designations are published in the Official Register on the EMEA web site, after applicants have had the opportunity to review a draft and redact proprietary information. 

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 FDA Orphan-Drug Review checklist has been designed in consultation with FDA reviewers, industry consultants, and regulatory professionals to serve as a summary tool indicating the evaluative criteria used by the Office of Orphan-Drug Development to critique and assess applications received. It can be used internally as a part of the Quality Assurance process, as a guideline for regulatory development of an application, and/or as a self-assessment tool to predict likely FDA response to an application. 

Food and Drug Administration assistance to orphan drug developers in protocol design for new drug approval (NDA) or product license approval (PLA) applications 28... 

With over 650 active designations and now about 275 orphan drugs approved for the market (and even one medical device), plus numerous grants awarded since 1983, the Act has clearly been a success. Let us not forget that nothing in the Act creates compulsion, and that voluntary participation, as measured by applications for designation submitted, increase year by year. 

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