alpha1 voice of the patient report fda

Is alpha-1 antitrypsin a patient-focused drug?

On September 29, 2015, FDA conducted a public meeting on Patient-Focused Drug Development for Alpha-1 Antitrypsin. FDA was interested in obtaining patient perspectives on the impact of Alpha-1 Antitrypsin on daily life and patient views on treatment approaches.

When did FDA conduct a public meeting on breast cancer drug development?

On April 2, 2015, FDA conducted a public meeting on Patient-Focused Drug Development for Breast Cancer. FDA was interested in obtaining patient perspectives on the impact of Breast Cancer on daily life and patient views on treatment approaches. On April 28, 2015, FDA conducted a public meeting on Patient-Focused Drug Development for Chagas Disease.

When did FDA conduct a public meeting on pulmonary arterial hypertension?

On May 13, 2014, FDA conducted a public meeting on Patient-Focused Drug Development for Pulmonary Arterial Hypertension. FDA was interested in obtaining patient perspectives on the impact of Pulmonary Arterial Hypertension on daily life and patient views on treatment approaches.

How can I contact FDA’s CDER patient-focused drug development program?

FDA was interested in hearing perspectives from individuals with vitiligo on the health effects and daily impacts of their vitiligo, treatment goals, and decision factors considered when seeking out or selecting a treatment. To contact FDA’s CDER Patient-Focused Drug Development Program Staff, please email patientfocused@fda.hhs.gov.

When was the FDA meeting on hereditary angioedema?

On September 25, 2017 , FDA is conducting a public meeting on Patient-Focused Drug Development for Hereditary Angioedema. FDA is interested in obtaining patient and caregiver perspectives on the on the impact of Hereditary Angioedema on daily life and patient views on treatment approaches.

When was the FDA meeting on Chagas disease?

On April 28, 2015, FDA conducted a public meeting on Patient-Focused Drug Development for Chagas Disease. FDA was interested in obtaining patient perspectives on the impact of Chagas Disease on daily life and patient views on treatment approaches.

When was the FDA meeting on stimulant use disorder?

On October 6, 2020 , FDA hosted a public meeting on Patient-Focused Drug Development for Stimulant Use Disorder. This meeting provided FDA the opportunity to obtain input from individuals with stimulant use disorder and other stakeholders on the impact of stimulant use disorder and views on treatment goals and approaches. FDA was interested in hearing perspectives from individuals with stimulant use disorder on the health effects and daily impacts of their stimulant use disorder, impact (if any) of opioid and polysubstance use on their stimulant use disorder, treatment goals, and decision factors considered when seeking out or selecting a treatment.

When is the FDA meeting for vitiligo?

On March 8, 2021, FDA hosted a public meeting on Patient-Focused Drug Development for Vitiligo. FDA was interested in hearing perspectives from individuals with vitiligo on the health effects and daily impacts of their vitiligo, treatment goals, and decision factors considered when seeking out or selecting a treatment.

When was the FDA meeting for systemic sclerosis?

On October 13, 2020 , FDA hosted a public meeting on Patient-Focused Drug Development for Systemic Sclerosis. FDA was interested in hearing perspectives from individuals with systemic sclerosis on the health effects and daily impacts of their systemic sclerosis, treatment goals, and decision factors considered when seeking out or selecting a treatment.

When was the Alport Syndrome meeting?

On August 3, 2018 , the National Kidney Foundation (NKF) and Alport Syndrome Foundation (ASF) held . an Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting on Alport Syndrome (AS). The . broad goals of the meeting were to inform the Food and Drug Administration (FDA) and other .

What is the 569C report?

Pursuant to section 569C of the Federal Food, Drug, and Cosmetic Act to: . Center for Drug Evaluation and Research (CDER) . U.S. Food and Drug Administration (FDA) . This report reflects the National Kidney Foundations and Alport Syndrome Foundations accounts of the .

What is Alport syndrome?

Alport syndrome is a rare, inherited disorder of type IV collagen, caused by mutations in the COL4A3, . COL4A4, andCOL4A5 genes. Under physiological conditions, the collagen subunits produced by each of . these genes (α-3, α-4, and α-5, respectively) come together to form a triple helix, the building block for .