Vaccines, Blood & Biologics

Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease and other Heritable Bleeding Disorders

Public Meeting on Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease and other Heritable Bleeding Disorders
September 22, 2014
9:00 a.m. to 5:00 p.m.

The Food and Drug Administration (FDA) is announcing a public meeting on Patient-Focused Drug Development for Hemophilia A, Hemophilia B, von Willebrand Disease, and other heritable bleeding disorders such as other factor deficiencies (including I, V, VII, X, XI), and platelet disorders. 

The public meeting is intended to provide FDA with patients’ perspectives on the impact on daily life of Hemophilia A, Hemophilia B, von Willebrand Disease and other heritable bleeding disorders such as those listed above.  FDA also is seeking patients’ perspectives on the available therapies for these disorders. 

Patient-Focused Drug Development is an FDA performance commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V).  For more information on the Patient-Focused Drug Development Initiative, please visit:

Purpose and Scope of the Meeting
The purpose of this Patient-Focused Drug Development meeting is to obtain input on the symptoms and other impacts that matter most to patients with Hemophilia A, Hemophilia B, von Willebrand Disease and other heritable bleeding disorders.  FDA also intends to seek patients’ perspectives on current approaches to treating these disorders.  FDA expects that this information will come directly from patients, caregivers, and patient advocates.

Heritable bleeding disorders are a diverse group of diseases and some involve lifelong defects in the clotting mechanism of the blood.  The most frequently occurring of these disorders include Hemophilia A, Hemophilia B and von Willebrand Disease.  Less frequent yet also serious heritable bleeding disorders include Factor VII deficiency, Factor XIII deficiency, α2-antiplasmin deficiency and platelet disorders such as Gray platelet syndrome.  Symptoms of heritable bleeding disorders include frequent nose bleed; prolonged and heavy menstrual bleeding, prolonged bleeding from cuts, trauma, dental extractions, and surgical procedures as well as bleeding into internal organs, muscles and joints.  Intracranial hemorrhage is a particularly serious and life-threatening manifestation.  Specific treatment recommendations are determined by the type and severity of the disorder; but in general, therapies such as factor replacement, platelet transfusion, fresh frozen plasma and cryoprecipitate are utilized.

The questions that will be asked of patients and patient stakeholders at the meeting are provided below.  For each topic, a brief patient panel discussion will begin the dialogue.  This will be followed by a facilitated discussion inviting comments from other patient and patient stakeholder participants. 

Topic 1:  The effects of your bleeding disorder that matter most to you

  1. Of all of the symptoms that you experience because of your condition, which one to three symptoms (bleeding or non-bleeding) have the most significant impact on your life? (Examples may include joint damage/pain, infections, prolonged and heavy bleeding with menstruation, fatigue, etc.)
  2. Are there specific activities that are important to you, but that you cannot do at all, or as well as you would like, because of your condition?  Please describe, using specific examples. (Examples may include participating in physical activities, attending work/school, and family/social activities, etc.)
  3. How have your condition and its symptoms changed over time?
  4. What worries you most about your condition?

Topic 2:  Perspectives on current approaches to treatment

  1. What are you currently doing to treat your condition or its symptoms?  (Examples may include blood transfusions, replacement therapies, over-the-counter products, and/or other therapies).
    1. How well do these treatments work for you?
    2. What are the most significant disadvantages or complications of your current treatments, and how do they affect your daily life?
    3. How has your treatment changed over time and why?
    4. What aspects of your condition are not improved by your current treatment regimen?
    5. What treatment has had the most positive impact on your life?
  2. If you could create your ideal treatment, what would it do for you (i.e., what specific things would you look for in an ideal treatment)?
  3. If you had the opportunity to consider participating in a clinical trial studying experimental treatments, what things would you consider when deciding whether or not to participate?

The public meeting will be held at the following location:

FDA White Oak Campus
10903 New Hampshire Ave.
Building 31, Room 1503B and C (Great Room)
Silver Spring, MD 20993

Please visit the following website for location, parking, security and travel information:

For those unable to attend the meeting in person, FDA will Webcast the meeting.  You will receive instructions on how to join the Webcast when you register for the meeting.

If you wish to attend this meeting, visit

Please register by September 12, 2014. 

Those who are unable to attend the meeting in person can register to view a live Webcast of the meeting.  You will be asked to indicate in your registration if you plan to attend in person or via the Webcast.  Your registration will also contain your complete contact information, including name, title, affiliation, address, email address, and phone number.  Seating will be limited, so early registration is recommended.  Registration is free and will be on a first-come, first-served basis.  However, FDA may limit the number of participants from each organization based on space limitations.  Registrants will receive confirmation once they have been accepted.  Onsite registration on the day of the meeting will be based on space availability. 

If you need special accommodations because of disability, please contact Henry Allen (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the meeting.

Panel Participation
Patients and patient stakeholders who are interested in presenting comments as part of the initial panel discussions should register by August 22, 2014.  You will be asked to indicate in your registration which topic(s) you wish to address.  You will be asked to send a brief summary of responses to the topic questions to

Panelists will be notified of their selection soon after August 22, 2014.  FDA will try to accommodate all patients and patient advocate participants who wish to speak, either through the panel discussion or audience participation; however, the duration of comments may be limited by time constraints. 

Interested members of the public, including those who attend the meeting in person or via the Webcast, are invited to provide electronic or written responses to any or all of the questions provided above. 

Submit either electronic or written comments by November 28, 2014.  Submit electronic comments to    Submit written comments to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

Comments may be submitted until November 28, 2014.  Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday, and will be posted to the docket at

Contact Person
Henry Allen
Office of Communication, Outreach, and Development
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave.
Bldg. 71, Rm.1125
Silver Spring, MD  20993
FAX:  301-595-1243

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