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.

Background

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.

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.

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

FDA Federal Register Notice