ARC Hosts Patient Forum with the FDA

ARC Hosts Patient Forum with the FDA

On November 16th, the Amyloidosis Research Consortium (ARC) hosted an historic event. Over 220 people from all over the nation came to Silver Spring, MD to participate in a patient forum with the U.S. Food and Drug Administration (FDA). The meeting was simultaneously webcast with another 500 people watching it live, while still more are viewing the meeting daily on the ARC website.  This patient forum is one of the many ARC initiatives to facilitate and advance drug development.

The webcast and more information about the meeting is available at:

It is a new dawn for the amyloidosis diseases.  For the first time in history there are drugs in development that specifically target amyloidosis. To advance this research through the pipeline and be able to deliver it to the patient, it is critical for pharmaceutical companies and the FDA, whose job it is to evaluate these therapies, to fully understand both the complexity of the disease and what amyloidosis patients want.


FDA Mtg Audience









The day was structured around successful patient focused drug development meetings previously held at the FDA. There were patients, caregivers and advocates who covered the whole spectrum of the disease; from newly diagnosed to those that have lived with it for years.  Patients talked openly and honestly about their experience of living with AL and ATTR amyloidosis; discussing quality of life, the burden of the disease and existing treatments. There were also important sessions regarding clinical trials and the unmet need of patients. These discussions and the report that will be generated out of this meeting will be an important resource for the FDA and industry to refer to when they design and evaluate therapies for our patients.

Dr. Woodcock at FDA Mtg (2)

Among the many FDA members who attended were regulators from the different divisions who are responsible for evaluating amyloidosis therapies. We were very fortunate to have Dr. Woodcock in attendance, the director of the Center for Drug Evaluation and Research. She spoke about the important role that patients play in the drug development process, the need for collaboration with all those involved in the process, the FDA’s commitment to rare diseases, and how this matters for amyloidosis.

She discussed the role of biomarkers for both early diagnosis and drug development and their vital importance in rare diseases. A biomarker is specific to a disease and can be used to measure response to treatment. Dr. Woodcock emphasized the important work the ARC has done to identify particular biomarkers and the FDA’s commitment to accelerate approval for their qualification.

The ARC was established earlier this year to bring together all those in the field of amyloidosis: From bench scientists, researchers, physicians, pharmaceutical companies, government regulators (FDA) and patients as well as advocates to collaborate and expedite the evaluation of treatments for amyloidosis. The ARC is playing a critical role in the research landscape, and will be launching some major initiatives in the coming year in order to speed clinical trials and advance research. To find out more visit:

Together with the Amyloidosis Foundation programs that support patients, education and awareness, we look forward to a bright future.