PV Patient Shares Experiences from Workshop at the FDA

The International Pemphigus & Pemphigoid Foundation (IPPF) quarterly readers are about RARE patients and I am one of them.
So many of patients shared their stories. Some of us were lucky: the lucky ones found a good dermatologist and got the correct diagnosed right away. Some of us didn’t have to fight with the insurance company to get the very expensive rituximab infusions.
We know how important for us is to get the newest and best for us medication, but first, those treatments have to be developed and approved for marketing.
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.

Our participation in the natural history study is very important, since we are providing valuable information about our disease.
What is a natural history study?
Natural history studies follow subjects who have or may develop a specific disease. Many of the 7,000 rare diseases are poorly investigated, so the progression of symptoms are not well understood.
For many diseases, researchers do not yet have biomarkers and drug targets that can be used to help make clinical trials more successful. Targeted drugs account for 60% of the orphan drug approval.
Knowledge of the disease’s natural history is important when researchers are planning to develop a treatment. Data collection and management determines the use of the data for meeting the goals of the study, which in turn, leads to faster treatments.
Why is patient voice important to FDA?
Engagement with CDER means patient focused drug development. Engagement helps identify what matters and what is important to the patients. Patient input aids in development of clinical trials that are meaningful and realistic.
FDA works very closely with orphan drugs developers to design trials for small patient population. Flexible trial designs are used in 80% of rare disease approvals. 60% of the trials are done abroad. Finding a cure and a treatment for rare diseases depends on the collaborations of researchers and scientists from all over the world.
The 2017 IIPF scientists conference in Lübeck demonstrated the importance of international cooperation to study Pemphigus and Pemphigoid diseases.
Currently PV patients do not have adequate treatment options, but there is hope that soon a pill will be available. Principia Biopharmaceutical company announced that PRN 1008 has been granted orphan drug designation. PRN 1008 is currently being evaluated in Phase 2 clinical trial.
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.

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