Daily Rules, Proposed Rules, and Notices of the Federal Government
FDA is requesting emergency processing of this proposed collection of information under section 3507(j) of the PRA (44 U.S.C. 3507(j) and 5 CFR 1320.13) to enable the agency to jointly announce with EMEA the adoption of the Common EMEA/FDA Application Form for Orphan Medicinal Product Designation at the European Union (EU)-wide Administrative Simplification Workshop on November 28, 2007. The information is essential to the agency's mission of protecting and promoting the public health. The use of the normal clearance procedures would likely result in the prevention or disruption of this collection of information.
With respect to the collection of information, FDA invites comments on these topics: (1) The clarity and ease of use of this proposed common application form; (2) whether the proposed collection of information is necessary for the proper performance of FDA's functions, including whether the information will have practical utility; (3) the accuracy of FDA's estimate of the burden of the proposed collection of information, including the validity of the methodology and assumptions used; (4) ways to enhance the quality, utility, and clarity of the information to be collected; and (5) ways to minimize the burden of the collection of information on respondents, including the use of automated collection techniques, when appropriate, and other forms of information technology.
This common application form is intended to benefit sponsors who desire to seek orphan designation of drugs intended for rare diseases or conditions from both the European Commission and FDA by reducing the burden of preparing separate applications to meet the regulatory requirements in each jurisdiction. It highlights the regulatory cooperation between the United States and EU mandated by the Transatlantic Economic Council (TEC). The TEC mandate involves: Removal of barriers to transatlantic commerce; rationalizing, reforming, and, where appropriate, reducing regulations to empower the private sector; achieving more effective, systematic, and transparent regulatory cooperation to reduce costs associated with regulation to consumers and producers; removing unnecessary differences between jurisdictional regulations in order to foster economic integration; reinforcing the existing transatlantic dialogue structures in regulatory cooperation, both by intensifying our sector-by-sector United States-EU regulatory cooperation and our dialogue between OMB and the European Commission services on methodological issues.
At present, when seeking orphan designation of the same drug for the diagnosis, treatment, or prevention of the same rare disease or condition in the United States and in the European Community, a sponsor must submit a designation request to FDA (in accordance with section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb)) and a separate designation application to EMEA (in accordance with Regulation (EC) No. 141/2000 of December 16, 1999, and Commission Regulation (EC) No. 847/2000). In most cases, the two documents are formatted differently to meet regulatory demands, but the required core information elements are similar with the exception of some unique regulatory requirements exclusive to each jurisdiction. Therefore, FDA and EMEA believe that a common application form will help reduce the sponsor's regulatory burden and costs to produce and submit a differently-formatted request/application. In addition, a common application form may also streamline the administrative and substantive regulatory review processes, and aid in information exchange between the agencies. In accordance with the Confidentiality Arrangements concluded on September 12, 2003, between the European Commission, EMEA, and FDA,
Any sponsor seeking orphan designation of the same drug for the same disease or condition from both FDA and EMEA may use this common application form for regulatory filing purposes. A sponsor may also use this common application form when seeking designation only from FDA. This
To use the common application form, the sponsor must provide the required information in each applicable section as instructed in the explanatory notes. Certain information elements are identified in the form as required exclusively by either FDA or EMEA regulations, and as such they must be included only in the application to that jurisdiction. Where additional explanations and/or supportive documents are necessary, the sponsor should sequentially append them at the end of the common application form in the order they appear in the form. The sponsor must also complete the declaration and signature page. For FDA, the completed common application form and required appended documents must be submitted to the Office of Orphan Products Development (HF-35), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857. For EMEA, the completed documents must be submitted to European Medicines Agency, 7 Westferry Circus, Canary Wharf, London E14 4HB, United Kingdom.
FDA estimates the reporting burden of this common application form as follows. Between January 2000 and May 2006, FDA and EMEA received 226 comparable orphan designation requests/applications of the same drugs for the same diseases or conditions, or an average of 35 per year. With the ease of a common application form, FDA anticipates the number of such requests/applications may increase over time. Therefore, generally there is one request/application per respondent and as, at the extreme, all respondent are U.S.-based, FDA believes up to 40 such respondents may use the common application form each year. The respondents will be primarily pharmaceutical companies or other for-profit organizations. The collection of information for sponsors requesting orphan drug designation from FDA is currently covered by the Orphan Drug Regulations (21 CFR Part 316) and approved under OMB No. 0910-0167 (expires August 31, 2010). For applications submitted exclusively to FDA, we do not believe the new form will result in any increased burden on the respondents and therefore we estimate no additional burden for those respondents. FDA believes the information required for the EMEA submission, for the most part, is very similar to that in the FDA submission, which is already in the respondents' possession. The respondents, however, may have to search existing data sources or gather additional needed data, such as on the prevalence or the availability of alternative methods of diagnosis, prevention, and treatment of the rare disease or condition of interest in the European Community, to complete the EMEA submission. FDA estimates that it will take an additional 32 hours (16 hours of professional time and 16 hours of support time) to compile information required for the EMEA submission. Hence, the estimated total annual human resource hours, at most, would be 1,280 hours.