# Agency Information Collection Activities; Submission for OMB Review; Comment Request; Orphan Drug Products
**AGENCY:**
Food and Drug Administration, HHS.
**ACTION:**
Notice.
**SUMMARY:**
The Food and Drug Administration (FDA) is announcing that the proposed collection of information listed below has been submitted to the Office of Management and Budget (OMB) for review and clearance under the Paperwork Reduction Act of 1995.
**DATES:**
Submit written comments on the collection of information by May 31, 2001.
**ADDRESSES:**
Submit written comments on the collection of information to the Office of Information and Regulatory Affairs, OMB, New Executive Office Bldg., 725 17th St. NW., rm. 10235, Washington, DC 20503, Attn: Wendy Taylor, Desk Officer for FDA.
**FOR FURTHER INFORMATION CONTACT:**
JonnaLynn P. Capezzuto, Office of Information Resources Management (HFA-250), Food and Drug Administration, 5600 Fishers Lane, Rockville, MD 20857, 301-827-4659.
**SUPPLEMENTARY INFORMATION:**
In compliance with 44 U.S.C. 3507, FDA has submitted the following proposed collection of information to OMB for review and clearance.
**Orphan Drug Products—21 CFR Part 316 (OMB Number 0910-0167)—Extension**
Sections 525 through 528 of the Federal Food, Drug, and Cosmetic Act (the act) (21 U.S.C. 360aa through 360dd) give FDA statutory authority to: (1) Provide recommendations on investigations required for approval of marketing applications for orphan drugs, (2) designate eligible drugs as orphan drugs, (3) set forth conditions under which a sponsor of an approved orphan drug obtains exclusive approval, and (4) encourage sponsors to make orphan drugs available for treatment on an “open protocol” basis before the drug has been approved for general marketing. The implementing regulations for these statutory requirements have been codified under part 316 (21 CFR part 316) and specifies the content and format of a request for written recommendations concerning the nonclinical laboratory studies and clinical investigations necessary for approval of marketing applications. Section 316.12 provides that, before providing such recommendations, FDA may require results of studies to be submitted for review. Section 316.14 contains provisions permitting FDA to refuse to provide written recommendations under certain circumstances. Within 90 days of any refusal, a sponsor may submit additional information specified by FDA. Section 316.20 specifies the content and format of an orphan drug application which includes requirements that an applicant document that the disease is rare (affects fewer than 200,000 persons in the United States annually) or that the sponsor of the drug has no reasonable expectation of recovering costs of research and development of the drug. Section 316.26 allows an applicant to amend the application under certain circumstances. Section 316.30 requires submission of annual reports, including progress reports on studies, a description of the investigational plan, and a discussion of changes that may affect orphan status. The information requested will provide the basis for an FDA determination that the drug is for a rare disease or condition and satisfies the requirements for obtaining orphan drug status. Secondly, the information will describe the medical and regulatory history of the drug. The respondents to this collection of information are biotechnology firms, drug companies, and academic clinical researchers.
In the *Federal Register* of September 19, 2000 (65 FR 56586), the agency requested comments on the proposed collections of information. No significant comments were received.
FDA estimates the burden of this collection of information as follows:
| 21 CFR Section | No. of Respondents | Annual Frequency perResponse | Total Annual Responses | Hours per Response | Total Hours |
| --- | --- | --- | --- | --- | --- |
| 316.10, 316.12, and 316.14 | 0 | 0 | 0 | 0 | 0 |
| 316.20, 316.21, and 316.26 | 90 | 1.78 | 160.20 | 125 | 20,025 |
| 316.22 | 5 | 1 | 5 | 2 | 10 |
| 316.27 | 5 | 1 | 5 | 4 | 20 |
| 316.30 | 450 | 1 | 450 | 2 | 900 |
| 316.36 | .2 | 3 | .6 | 15 | 9 |
| Total Burden Hours | | | | | 20,964 |
The information requested from respondents represents, for the most part, an accounting of information already in possession of the applicant. It is estimated, based on the frequency of requests over the past 10 years, that 90 persons or organizations per year will request orphan drug designation and that no requests for recommendations on design of preclinical or clinical studies will be received. Based upon FDA experience over the last decade, FDA estimates that the effort required to prepare applications to receive consideration for sections 525 and 526 of the act (§§ 316.10, 316.12, 316.20, and 316.21) is generally similar and is estimated to require an average of 95 hours of professional staff time and 30 hours of support staff time per application. Estimates of annual activity and burden for foreign sponsor nomination of a resident, agent, change in ownership or designation, and inadequate supplies of drug in exclusivity, are based on total experience by FDA with such requests since 1983.
Dated: April 24, 2001.
William K. Hubbard,
Senior Associate Commissioner for Policy, Planning, and Legislation.