The International Pemphigus & Pemphigoid Foundation (IPPF) quarterly readers are about RARE patients and I am one of them.
On October 30th at 8:00, I signed to attend the Center for Drug Evaluation and Research (CDER) rare diseases public workshop at the FDA. It was my first visit in Silver Spring, MD.
The FDA’s public health mission is to ensure the safety, effectiveness and security of human and animal drugs, biological products and medical devices. CDER has more than than 4,000 employees.
I was familiar with the agenda and was prepared to be overwhelmed with information. There are 7,000 know rare diseases and 30 million people in USA are living with rare disease. 95% of the individuals with rare diseases are still waiting for a treatment. For many rare diseases, basic research is lacking, and scientists have a limited understanding of what causes the condition.
The FDA is collaborating with NIH, industry, academia and patient groups to develop new information about rare diseases, biomarkers and outcome measures for clinical trials. In USA there are 40,000 PV patients and 170,000 worldwide. We are not so rare!
I learned how important it is to raise awareness about our disease.
We, the patients with chronic diseases, are the experts. We feel the burdens of the disease, the burden of the treatments, the benefits and the risks. The pharmaceutical companies, NIH and FDA need us.
What is a natural history study?
Why is patient voice important to FDA?
We all know how difficult it is to live with a rare diseases.
We must make sure that our stories are heard.
Ask your Senators, your Representatives to join the Rare Disease Congressional Caucus. We need their support.
“ENGAGEMENT is about DIALOGUE” John Whyte, MD, director CDER
About the Author: Iva is a PV patient, diagnosed in 2006. She’s had 6 rituximab infusions, but she’s not in complete remission.
Iva was born in Sofia, Bulgaria and now lives in Maryland. Iva is short of Ivanka (like the first daughter). The Pemphigus gene primarily effects people from the Mediterranean and Jews from Eastern Europe.
Her husband is German, their son was born in Edison, NJ and their daughter was born in Erlangen. Her son now works for a biotech company in Gaithersburg, MD and their daughter now works in the same hospital.
Iva and her husband moved to the Eastern Shore of Maryland 15 years ago and still like it there.
