Duchenne Therapy Reviews on the Horizon The FDA Advisory Committee Meetings September 2, 2015 Host: Pat Furlong, PPMD President & Founder Guest presenters: FDA Office of Health & Constituent Affairs, and Division of Advisory Committee and Consultant Management ParentProjectMD.org
Everything you ever wanted to know about Advisory Committee Meetings Cicely Reese Richard Klein Steve Morin Office of Health and Constituent Affairs and the Division Of Advisory Committee And Consultant Management
Duchenne Therapy Reviews on the Horizon Pat Furlong PPMD, President & Founder ParentProjectMD.org 3
Everything you ever wanted to know about Advisory Committee Meetings Food and Drug Administration Office of Health and Constituent Affairs and the Division Of Advisory Committee And Consultant Management
What is an Advisory Committee? The Committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs 5
Why does FDA use advisory committees? Part of the FDA mission is to evaluate new therapies and determine which are safe and effective for their intended use. This is complex job often involves many areas of expertise, and sometimes FDA turns to outside experts for counsel Valuable resource contributing to the agency s decision- making processes Advisory in nature, providing recommendations to the agency FDA makes the final decisions FDA has 48 technical and scientific advisory committees and panels Potential treatments for DMD are considered by the Peripheral and Central Nervous System Drugs Advisory Committee 6
When does FDA use advisory committees? Meetings can occur during any stage of a product's review process, or if appropriate, once a product is marketed Typically, a committee is convened to assist with interpretation when questions or difficulties related to trial data arise The decision to involve an advisory committee is usually at the discretion of the director of the reviewing division 7
How and when are meetings announced? Meetings are officially announced through the Federal Register At least 15 days in advance of the meeting Includes date(s), times, location, topic of discussion, web links for further information, and instructions & contacts for written or in- person comments from the public Posted on the FDA Patient Network http://www.fda.gov/forpatients (Calendar of Public Meetings) 8
Who makes up the committee? Peripheral and Central Nervous System Drugs Advisory Committee The committee consists of a core of 9 fixed voting members including the Chairperson Members and the Chairperson are selected for their knowledge and expertise in the fields of neurology, neuropharmacology, neuropathology, otolaryngology, epidemiology, statistics, pharmacology, and related specialties, a patient representative and a member with consumer interests Temporary voting members may include a patient, parent or caregiver having experience with a specific disease/condition, and scientists or clinicians with specialized knowledge/experience In addition to the voting members, the committee may include non- voting consultants with specialized knowledge, and one non- voting member who is identified with industry interests 9
What is the role of: The Chair? The Designated Federal Official? 10
FDA Patient Representative Patient representative should have:. experience with disease as patient or caregiver ties to patient advocacy organizations the ability to analyze technical data an understanding of research design the ability to discuss benefits and risks the ability to evaluate the safety and efficacy of the medical products under review
FDA Consumer Representative The consumer representative: represent the consumer perspective on issues serves as a liaison between the committee and interested consumers, associations, coalitions, and consumer organizations facilitate dialogue with the advisory committees on scientific issues that affect consumers
Distinctions between FDA patient reps and consumer reps Patient representatives can address specific needs and preferences of patients for whom the treatment is intended. Consumer representatives offer broader knowledge of consumer rights and needs. Both have access to proprietary background information and materials not available to the public
How will the day unfold? Introduction Conflict of Interest Presentation by sponsor Presentation by FDA Open Public Hearing FDA s questions for the committee Committee discussion and deliberation Vote and discussion 14
How does the public participate? Audience Public Comment In person Written submissions 15
How can the public participate? Submit written comments to designated official in meeting announcement by deadline date by mail, FAX, or e- mail To speak during the open public hearing, interested persons need to contact FDA by deadline in the announcement with a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation 16
How can the public participate? At the meeting, speakers should identify themselves to FDA staff members and provide the staff with any handouts or other materials for distribution to committee members, and be included in the meeting s permanent record Special seating is reserved for registered speakers from the public Those who speak are encouraged to disclose any financial relationships they may have with the topic of the meeting or with the sponsors or competitors of the products under discussion 17
How can the public participate? FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our Web site at Public Public Conduct During FDA Advisory Committee Meetings for procedures on public conduct during advisory committee meetings http://www.fda.gov/advisorycommittees/aboutadviso rycommittees/ucm111462.htm 18
How can the public participate? orderly conduct of FDA advisory committee meetings Register with FDA before the meeting Those who have not registered before the meeting will only be invited to speak at the discretion of the Chair, and should submit their request to FDA officials at the registration desk on the day of the meeting Open Public Hearing participants who are designated to speak may be questioned only by the Chair or other members of the Committee 19
How can the public participate? orderly conduct of FDA advisory committee meetings Audience members may not present comments or questions to the Committee unless recognized by the Chair Attendees may be subject to security screening, such as presenting identification, passing through metal detectors, and inspection of briefcases, packages, etc. 20
How does the public participate? orderly conduct of FDA advisory committee meetings Attendees at the meeting are asked to maintain order and not display behavior that is disruptive to the meeting (i.e., shouting from the audience, loud outbursts) The committee Chair or Designated Federal Officer will note on the record any disruptive behavior and will ask the person to cease the behavior or else leave the meeting room We ask that attendees not approach the advisory committee table area before, during, or after the meeting without permission from a Designated Federal Officer/Executive Secretary 21
How much time will I have to speak? At least an hour will be set aside for public comment Time is allotted based on the number of requests to speak FDA asks people with similar messages to consolidate if possible In rare instances, time may be allotted by lottery Speakers are randomly selected from requests to speak. Comments of speakers will be included in the meeting transcript 22
What is the value of the meeting to FDA? Engage outside experts from various fields Variety of perspectives for consideration Input from the public on important regulatory decisions In person Written submissions Allows public access to data and decision- making Often the only opportunity for the public to see the data and hear exchange of viewpoints 23
What is the value of the meeting to FDA? While most advisory committees include a vote on the questions, the discussion between committee members is the most important contribution to the decision- making process 24
How can I prepare for an advisory committee meeting? Background materials related to the meeting will be posted on the FDA web site prior to the meeting Meeting agenda Instructions for providing your comments Roster of committee participants Briefing information submitted by the sponsor and FDA Presentation slides Questions for the committee Webcast information 25
Can I watch past advisory committee meetings? Webcasts of past committee meetings are archived on the FDA website for example http://www.fda.gov/downloads/ad visorycommittees/committeesmee tingmaterials/drugs/peripheraland CentralNervousSystemDrugsAdviso rycommittee/ucm352967.pdf 26
Where is the meeting held? FDA White Oak Campus, Silver Spring, MD Open to the public No registration or fees required to attend Directions on FDA s website 27
What if I need special accommodations? A contact person will be specified in the meeting announcement Let that person know if you need special accommodations due to a disability at least 7 days before the meeting date Every effort will be made to make your accommodate your needs 28
Do I need to attend in person? Advisory committee meetings held at the FDA campus are webcast live A link to the live webcast is provided on the FDA web site http://www.fda.gov/advisorycommittees/committeesmeeting Materials/Drugs/PeripheralandCentralNervousSystemDrugsAd visorycommittee/default.htm Viewers are able to watch the proceedings, and see the slides during presentations Webcasts are view only and not interactive 29
Some important Take Aways Meetings are an integral part of regulatory decision- making Meetings are open and transparent The public is invited and encouraged to participate Advisory committee recommendations are carefully considered, but non- binding Offer a window into the complexity of drug development FDA, and the public need to consider the totality of the data 30
Thank you If you have additional questions, please contact us at PatientNetwork@fda.hhs.gov 31
Question & Answer ParentProjectMD.org
Thank you If you have additional questions, please contact us at PatientNetwork@fda.hhs.gov 33
To learn more about ways that our unified Duchenne community is preparing for the upcoming AdComms and to join our army, please contact : ryan@parentprojectmd.org ParentProjectMD.org 34