ARC is Poised for Great Things in 2020
2019 was a year of great accomplishments for the Amyloidosis Research Consortium (ARC), and we are grateful to all of you who have so generously supported our efforts to accelerate the pace of development of drugs to treat and improve quality of life for amyloidosis patients.
Without your help we could not have achieved the following milestones:
The publication of our overarching 10-year strategy, Bridging the Gap. ARC’s vision is to make a significant impact on the curability of amyloidosis. Our strategic plan outlines our direction and ambitions for the next decade.
The first Amyloidosis Forum meeting at the US Food and Drug Administration (FDA), focusing on Advancing Drug Development in AL Amyloidosis. The meeting was a resounding success, with participants ranging from patients to researchers to physicians to representatives from the FDA convening to discuss how to address the challenges of and develop a pathway forward to accelerating the development and approval of drugs to treat AL amyloidosis. You can watch the webinar from the meeting here, and read more about the meeting and the next steps here.
The initiation of our work to create a Patient Reported Outcomes (PRO) measurement tool in ATTR amyloidosis to be used to understand and measure the impact of the disease on patients from the patient’s perspective. The goal is to measure what matters most to patients, such as how the disease and/or treatment impact their daily lives, physical functioning, and mental health status. The goal is to create a tool that will allow physicians to monitor patients’ progress and may also be useful in clinical trials. You can read more about the PRO here.
The development of clinical data standards through a collaboration with the Critical Path Institute (C-Path). C-Path is an independent, nonprofit organization dedicated to bringing together experts from regulatory agencies, industry, and academia to collaborate and improve the medical product development process. ARC is working with C-Path to create an annotated case report form (aCRF) that can be used by physicians in clinical trials to help standardize the information collected on these trials, which will help increase the ability to share data in a format that can be accessed by many different researchers and organizations.
The publication in the journal of the American Heart Association, Circulation: Heart Failure, of consensus best practices guidelines for the diagnosis of cardiac ATTR amyloidosis. Read more here.
Increasing the resources available to patients and physicians both on our website and in hard copy. We have begun the development of booklets for both hATTR and ATTRwt amyloidosis that provide an overview of amyloidosis, specific information on each type of amyloidosis and a guide to the current treatment landscape. In addition, we have produced – and are continuing to produce – a series of webinars called “ARC Talks,” directed to both physicians and to patients addressing topics of interest to each group. Our recently formed Patient Advisory Group has been instrumental in helping us identify topics of interest for patients. Learn more about the ARC Talks webinars here.
For physicians specifically, we have developed the Amyloidosis Masterclass, a Continuing Medical Education (CME) program, the first of which will take place in January 2020. The Masterclass is an interactive, educational event where attendees will learn about amyloidosis care, the value of multidisciplinary teams, how to build an amyloidosis program to best support patients, and connect with experts in the field. These conferences will be open to all medical specialties but are primarily targeted to practicing physicians who most commonly see and treat amyloidosis patients. Learn more about the Masterclass here.
Laying the foundation for creating a Care Center Network that will improve patient access to care by facilitating the growth of new amyloidosis centers, recognizing centers of excellence and holding centers accountable to maintaining high standards of care. We expect to fully launch the program in 2020.
While the list above is quite full, it is not by any means exhaustive. In 2019 we also presented posters at medical and research conferences, organized symposia, and attended Rare Disease Week in Washington, DC. Without the generous support of our partners – patients, families, industry and other donors – we would be unable to continue this important work. We greatly appreciate your support of our mission, and we look forward to reaching the day where amyloidosis is easily and accurately diagnosed and treated, with a concomitant improvement in quality of life for all amyloidosis patients.