Ninety Percent of Rare Diseases Do Not Have an Approved FDA Treatment


Dr. Ed Neilan, who is the Chief Medical and Scientific Officer of the National Organization for Rare Disorders (NORD), recently commented that there are approximately more than seven thousand rare diseases, but over ninety percent are without an approved FDA treatment. The focus of the pharmaceutical industry is on somewhere between one to two hundred rare diseases.

According to a recent article in Eureka Alert, attendees at the RDCA-DAP September 2021 annual meeting were among those who hope to draw attention to so many neglected rare diseases.

Attendees included over four hundred providers, patients, clinicians, researchers, regulatory reviewers, scientists, and biopharmaceutical companies.

The meeting, which included a launch and demonstration of data sharing and rare disease innovation, was hosted through a partnership between C-Path, NORD, and the FDA. Currently, it leads the rare disease community in accelerating treatment innovation for rare diseases.

The workshop consisted of thirty-one panelists and speakers from regulatory, academic fields, and industry along with various patient organizations. Also, five profiles of patients were presented.

RDCA-DAP is an acronym for Rare Disease Cures Accelerator-Data and Analytics Platform initiative. It is funded by the FDA and will function as a central base that will share de-identified (identifying information is removed) data about rare diseases and act as a support for treatment innovation. Many other entities have since joined the effort.

Dr. Jeff Barrett, SVP and Lead at RDCA-DAP, said that there is indeed enthusiasm about the program among developers and patients. Their expectation is to develop tools to reduce the risk that has been inherent in decision-making for drug development in rare diseases.

Dr. Barrett further commented that in addition to focusing on the data, the program will also generate solutions through the enthusiastic collaboration of its participants. For more information please visit

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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