FDA to Focus on Blood, Platelet Disorders at Upcoming Meeting With Patients

Regulatory NewsRegulatory News | 08 July 2014 |  By 

US regulators have announced they will soon hold their tenth patient-focused drug development meeting, this time focusing on a collection of blood and platelet disorders that includes hemophilia A, hemophilia B, and von Willebrand disease.


The meetings are required under the Food and Drug Administration Safety and Innovation Act (FDASIA), Section X of which called for the US Food and Drug Administration (FDA) to meet with patients in order to better understand their perspectives on the safety and efficacy of therapies.

In particular, the legislation envisions FDA focusing on disease areas with inadequate treatment options or diseases with no recognized treatments. The thinking of legislators and advocates of the law was that some patients might be more willing to accept risky or unproven treatments given a dearth of acceptable alternative options.

Including today's announcement, FDA has announced patient-focused drug development meetings for ten disease groups:

  • Chronic Fatigue Syndrome (CFS) and Myalgic Encephalomyelitis
  • Pulmonary Arterial Hypertension (PAH)
  • Narcolepsy
  • Lung Cancer
  • Human Immunodeficiency Virus (HIV)
  • Fibromyalgia
  • Sickle Cell Disease (SCD)
  • Neurological manifestations of inborn errors of metabolism
  • Idiopathic Pulmonary Fibrosis (IPF)
  • Hemophilia A, Hemophilia B, von Willebrand Disease, and Other Heritable Bleeding Disorders

New Meeting

FDA's newest meeting will focus on a "diverse group" of heritable blood and platelet disorders, such as hemophilia A and B, von Willebrand disease, factor VII and XIII deficiencies, α2-antiplasmin deficiency and Gray platelet syndrome.

As with other disease groups, FDA has a specific list of questions it intends to ask of patients—a list of questions that shares much in common with other disease-specific meetings.

FDA's Questions for Blood and Platelet Disorder Patients
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?
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?
How have your condition and its symptoms changed over time?
What worries you most about your condition?
What are you currently doing to treat your condition or its symptoms?
How well do these treatments work for you?
What are the most significant disadvantages or complications of your current treatments, and how do they affect your daily life?
How has your treatment changed over time and why?
What aspects of your condition are not improved by your current treatment regimen?
What treatment has had the most positive impact on your life?
If you could create your ideal treatment, what would it do for you?
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?

Interestingly, the current batch of questions also bucks a recent trend toward FDA asking fewer questions. The last two meetings announced by FDA, focusing on Idiopathic Pulmonary Fibrosis (IPF) and Inborn Errors of the Metabolism, had asked just eight and six questions, respectively. FDA's blood disorders meeting will ask 12 questions of patients.

Questions Asked of Each Group by FDA
Blood/Platelet DisordersCFSPAHNarcolepsyLung CancerFibromyalgiaSCDHIVInborn Errors of MetabolismIPF


The meeting will take place on 22 September 2014 at FDA's White Oak Campus in Silver Spring, MD.


FDA Federal Register Notice


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