Process for Preparing for an FDA Advisory Committee Meeting

1. Process for Preparing for an FDA Advisory Committee Meeting Vann Parker, PhD 24 April 2013

2. Agenda Advisory Committee Background Preparation

3. Advisory Committee Background Charter and Membership

4. Advisory Committee Charter The Federal Advisory Committee Act became law in 1972 Established framework for seeking expert advice A series of Guidance Documents were issued in 2008 Preparation and Public Availability of Information Given to Advisory Committee Members • Briefing materials will be made available to the public as well as the committee Guidance for the Public and FDA Staff on Convening Advisory Committee Meetings • Still a “draft” guidance • Provides factors for deciding when to convene a meeting: significant public interest; controversial; specific expertise to evaluate • First-of-a-Kind, First-in-Class Medicinal Products must be referred to an Advisory Committee or provide a justification in the action letter for why it was not referred

5. Advisory Committees The committees are established to provide functions which support the FDA's mission of protecting and promoting the public health, while meeting the requirements set forth in the Federal Advisory Committee Act. • Provide independent, expert advice to the agency on a range of complex scientific, technical, and policy issues Committees are either mandated by statute or established at the discretion of the Department of Health and Human Services. Each committee is subject to renewal at two-year intervals unless the committee charter states otherwise. Composed of voting and nonvoting members (academics, clinicians, consumer and industry reps)

6. Standing Committees in 21 CFR§14.100 (1) Risk Communication (2009) Office of the Commissioner Board of Tea Experts (1897) Science Board to the FDA (1992) Pediatric (2004) Tobacco Products Scientific (2009) • Center for Biologics Evaluation and Research (CBER) Cellular, Tissue and Gene Therapies (1988) Vaccines and Related Biological Products (1979) Allergenic Products (1984) Blood Products: (1980) Transmissible Spongiform Encephalopathies (1995)

7. Standing Committees in 21 CFR§14.100 (2) Center for Drug Evaluation and Research (CDER) Medical Imaging (1967) Peripheral and Central Nervous System (1974) Nonprescription Drugs (1991) Reproductive Health Drugs (1978) Cardiovascular and Renal Drugs (1970) Anesthetic and Life Support Drugs (1978) Dermatologic and Ophathalmic Drugs (1980) Pharmacy Compounding (1998) Arthritis (1974) Gastrointestinal Drugs (1978) Anti-Infective Drugs (1980) Psychopharmacologic Drugs (1974) Pharmaceutical Science and Clinical Pharmacology (1990) Oncologic Drug (1978) Pulmonary-Allergy Drugs (1972) Antiviral Drugs: February 15, 1989 Drug Safety and Risk Management (1978) Endocrinologic and Metabolic Drugs (1970)

8. Standing Committees in 21 CFR§14.100 (3) Center for Devices and Radiological Health Technical Electronic Product Radiation Safety Standards (1968) National Mammography QA (1993) Medical Devices (1990) Device GMP (1987) • National Center for Toxicological Research – Science Advisory Board (1973) • Center for Veterinary Medicine – Veterinary Medicine Advisory Committee (1984) • Center for Food Safety and Applied Nutrition – Food Advisory Committee (1991)

9. Advisory Committee Membership Types Physician-Scientists Statisticians Epidemiologists Pharmacologists Nutritionist Nurses Experts in animal (preclinical) studies Committee membership includes ethnic, gender, and geographic diversity

10. Advisory Committee Meetings – General Information Advisory committee meeting often involve marketing applications/submissions such as: • New drug applications and application supplements; • New animal drug applications and application supplements; • Biologics license applications and application supplements; • Premarket approval applications for medical devices and their supplements; • Premarket notifications for medical devices; and • Medical device classifications and reclassifications. General matters; guidance documents, issues pertaining to trial design, post-approval monitoring, citizen’s petitions, and policy issues related to FDA-regulated products

11. Roles and Responsibilities

12. Preparation for Advisory Committee Meetings

13. Prepare, Prepare, Prepare! Know your drug Pick your team • Familiarity with program, issue identification • Potential speakers • Important review functions including Medical, Statistical, Regulatory • Outside experts, KOLs, Study PI • Support staff, Medical Writing, slide generation • Set aside time for INTENSIVE preparation • Practice • Have a consistent storyline for presentation • Provide a briefing book that presents a compelling position • Company presentation at meeting should highlight strengths of program, in as balanced a way as possible • Assume the FDA or committee members will slice and dice your data • What issues have been raised in FDA interactions such as pre-NDA meeting • Generate a list of questions you might be asked • Generate answers to ALL of the potential questions • Practice presentations and responses Know their team • Who is on the Advisory Committee • Who are the FDA review team • What was the outcome and discussion at previous meetings of this group

14. Meeting flow Introduction of Committee and Agenda Opening Remarks from FDA Sponsor Presentation FDA Presentation Open Public Hearing Clarifying Questions to Sponsor Panel Discussion and Voting on Questions from FDA Adjournment

15. Keys to Successful Outcome Begins long before meeting through productive FDA interaction (pre-IND through pre-NDA meetings and responses to review requests) Committee briefing book that tells a complete story in a well organized, concise fashion • Note the briefing book is a public document that should not contain confidential information Consistent message in presentation and Q&A session Meaningful presentations to various audiences day of meeting Appreciation for patient concerns Addressing questions posed by FDA to the Committee Support from KOLs viewed as independent Answering meeting day questions directly without being confrontational Being well organized and prepared • Allow enough time for meeting preparation • Have answers ready for questions and know who will respond • Expect to have several Mock Advisory Committee prep sessions

16. Structure of the Briefing Document The briefing book should complement the oral presentation and prepare the reader to fully understand the issues. Must be easy to read, strategic, and persuasive, consistent with all data available. Target Audience • Include essential information regarding the drug's safety and effectiveness • Misleading or promotional materials • Statement or language that are defamatory, irrelevant, or intemperate

17. Advisory Committee Meeting Presentation Target the Audience Use Information Package as Basis for Preparing Presentation Address FDA’s Position / Issues and Present Scientifically-Based Responses Be Clear; Keep to the Point • The Panel Has Reviewed the Information Package in Advance

18. Open Public Hearing Codified in 21 CFR§14.29 Meeting and Deadline for Request in Federal Register A minimum of 1 hour per meeting Chair can designate longer session Interested Persons General member of public Industry professional as individual or spokesperson (other than Sponsor) Consumer Advocate Professional Organization, Society or Association Usually 5-10 minutes per speaker http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM236144.pdf

19. Timeline for Open FDA Advisory Committee Meeting: Fully Releasable

20. Timeline for Open FDA Advisory Committee Meeting: Not Fully Releasable (1)

21. Timeline for Open FDA Advisory Committee Meeting: Not Fully Releasable (2)

22. Post both the sponsor’s and the agency’s publicly available Briefing Documents will be posted on on Website. Detailed minutes of all advisory committee meetings will be available to public after the meeting. What Gets Published to Public? Publish Prior to the Meeting Publish after the Meeting http://www.fda.gov/AdvisoryCommittees/WhatsNew/default.htm

23. Records of Meetings before Advisory Committee Agency employee prepares detailed minutes of all advisory committee meetings Minutes of open portion includes: • Time and Place • Members, committee staff, agency employees present, public participants • a copy of all written information made available for at the proceedings • A complete and accurate description of matters discussed and conclusions reached. • A copy of all reports received, issued or approved by the committee. • The extent to which the meeting was open to the public • The extent of public participation.

24. Safety and Effectiveness of Drug in 21 CFR§14.160 Technical advisory committees established to advise the Commissioner: Labeling and advertising, and regulatory control of the human prescription drugs falling within the pharmacologic class covered by the advisory committee and on the scientific standards appropriate for a determination of safety and effectiveness in that class of drugs. Specifically on any particular matter involving a human prescription drug pending before FDA, including whether the available information is adequate to support a determination that-- • A particular IND study may properly be conducted; • A particular drug meets the statutory standard for proof of safety and effectiveness necessary for approval or continued approval for marketing; or • A particular drug is properly classified as a new drug, an old drug, or a banned drug

25. Biologics in 21 CFR§14.29 The Commissioner of Food and Drugs shall appoint advisory review panels to evaluate the safety and effectiveness of biological products for which a license has been issued pursuant to section 351 of the Public Health Service Act, to review the labeling of such biological products, and to advise the biological products under review are safe, effective, and not misbranded. An advisory review panel shall be established for each designated category of biological product.

26. Questions? Thank you