Individuals with pulmonary hypertension (PH) and staff from the Pulmonary Hypertension Association (PHA) weighed in during a recent public meeting about living with a rare disease. The meeting was hosted by the U.S. Food and Drug Administration (FDA) and was designed to give people living with rare diseases and their family caregivers an opportunity to share their perspective. Participants responded to questions about rare disease symptoms, barriers to taking medication, barriers to participating in clinical trials and what risk-benefit considerations are important when seeking treatment for a rare disease.

When drug manufacturers want to test a new product, the FDA uses information from meetings like this to influence the clinical trial design. For example, the FDA uses input from these meetings to ensure the clinical trial tests the new therapy’s impact on symptoms patients deem important.

Patients with PH and caregivers who were unable to participate in the meeting can still share their experience with the FDA until May 30.

For assistance with contributing to this comment period, contact Katie Kroner at 301-565-3004 x749 or Advocacy@PHAssociation.org.