2024-04751. Advisory Committee; Genetic Metabolic Diseases Advisory Committee; Addition to List of Standing Committees

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Final rule.

SUMMARY:

The Food and Drug Administration (FDA or Agency) is amending the standing advisory committee regulations to add the establishment of the Genetic Metabolic Diseases Advisory Committee (GeMDAC or the Committee) to the list of standing committees.

DATES:

This rule is effective March 6, 2024.

FOR FURTHER INFORMATION CONTACT:

Moon Choi, Center for Drugs Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 31, Rm. 2417, Silver Spring, MD 20993, 301–796–2894, [e-mail].

SUPPLEMENTARY INFORMATION:

The Committee was established on December 12, 2023, and notice of establishment was published in the Federal Register on December 13, 2023 (88 FR 86344).

The Committee reviews and evaluates data on the safety and effectiveness of marketed and investigational human drug and biologic products for use in the treatment of genetic metabolic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs (the Commissioner).

The Committee shall consist of a core of nine voting members, including the Chair. Members and the Chair are selected by the Commissioner or designee from among authorities knowledgeable in the fields of medical genetics, manifestations of inborn errors of metabolism, small population trial design, translational science, pediatrics, epidemiology, or statistics and related specialties. Members will be invited to serve for overlapping terms of up to 4 years. Non-Federal members of this Committee will serve either as special government employees or non-voting representatives. Federal members will serve as regular government employees or ex 1652fficious. The core of voting members may include one technically qualified member, selected by the Commissioner or designee, who is identified with consumer interests and is recommended by either a consortium of consumer-oriented organizations or other interested persons. In addition to the voting members, the Committee may include one non-voting representative member who is identified with industry interests. There may also be an alternate industry representative.

The Committee name and function have been established with the establishment of the Committee charter. The change became effective December 12, 2023. Therefore, the Agency is amending § 14.100 (21 CFR 14.100) to add the Committee name and function to its current list as set forth in the regulatory text of this document.

Under 5 U.S.C. 553(b)(4)(B) and (d) and 21 CFR 10.40(d) and ©, the Agency finds good cause to dispense with notice and public comment procedures and to proceed to an immediate effective date on this rule.

Notice and public comment and a delayed effective date are unnecessary and are not in the public interest as this final rule is merely codifying the addition of the name and function of the GeMDAC to the list of standing FDA advisory committees. The establishment of the Committee is already effective, and the name and function that will be added to § 14.100 reflect the Committee charter. The Agency is amending § 14.100(c)(18) as set forth in the regulatory text of this document.

List of Subjects in 21 CFR Part 14

• Administrative practice and procedure
• Advisory committees
• Color additives
• Drugs
• Radiation protection

Therefore, under the Federal Food, Drug, and Cosmetic Act and under authority delegated to the Commissioner of Food and Drugs, 21 CFR part 14 is amended as follows:

PART 14—PUBLIC HEARING BEFORE A PUBLIC ADVISORY COMMITTEE

1. The authority citation for part 14 continues to read as follows:

Authority: 5 U.S.C. 1001 et seq.;15 U.S.C. 1451–1461; 21 U.S.C. 41–50, 141–149, 321–394, 467f, 679, 821, 1034; 28 U.S.C. 2112; 42 U.S.C. 201, 262, 263b, 264, 284m, 284m–1; Pub. L. 107–109, 115 Stat. 1419.

2. Section 14.100 is amended by adding paragraph (c)(18) to read as follows:

§ 14.100

List of standing advisory committees.

* * * * *

(c) * * *

(18) Genetic Metabolic Diseases Advisory Committee.

(i) Date Established: December 12, 2023.

(ii) Function: Reviews and evaluates data on the safety and effectiveness of marketed and investigational human drug and biologic products for use in the treatment of genetic metabolic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs.

* * * * *

Dated: March 1, 2024.

Lauren K. Roth,

Associate Commissioner for Policy.