As a patient-led organization, ARC has firsthand knowledge of how amyloidosis affects our community. We engage scientists, policymakers and government regulators as partners in our efforts to break down barriers that slow research and drug development. In order for new therapies to make the journey from the lab to patients’ hands, a joint focus on public policy and research is essential.
At a time when there is great uncertainty around funding for rare disease research, it is of the utmost importance to focus on changing healthcare policy and tax credits for orphan drug development, making sure that patients with rare diseases and the treatments in development are not adversely affected. ARC advocates for government policies that accelerate the development of amyloidosis therapies and increase quality of life for patients and caregivers. We actively work in close collaboration with the federal agencies and lawmakers that further scientific progress and help individuals access care.
Throughout the year the ARC team has the opportunity to represent amyloidosis patients and their unmet needs in many forums. It is a role we see as vitally important and one we take great pride in. These meetings vary from small closed-door meetings with policy makers, to speaking engagements at large public meetings.
ARC puts together patient panels and surveys throughout the year with the goal of integrating the voice of patients into drug development and the regulatory process. The purpose of the meetings can vary from patient perspective on the clinical study design to outlining the benefits versus risks patients are willing to take for the FDA to use in reviewing treatments.
ARC regularly meets with representatives about key policy issues. Every year ARC participates in Rare Disease Week on Capitol Hill. The event aims to bring together the rare disease community to educate them on federal legislative issues and give them the chance to share their stories with legislators. The entire week provides a unique opportunity to raise awareness of key policy issues that affect amyloidosis. Patient organizations, the NIH and other government entities, medical researchers and pharmaceutical companies developing treatments for rare diseases all take part in the event.
Patient-Focused Drug Development (PFDD)
On November 16, 2015, at the suggestion of the US Food and Drug Administration (FDA), the Amyloidosis Research Consortium (ARC) hosted an externally led Patient-Focused Drug Development meeting to share with the Agency and other stakeholders (e.g., industry) the perspectives of people living with systemic amyloidosis, its impact on their daily lives, and their perspectives on approaches to treating amyloidosis. The meeting was conducted in accordance with the Agency’s Patient-Focused Drug Development (PFDD) initiative, an FDA commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition.
Voice of the Patient
As a result of the PFDD meeting, ARC submitted a comprehensive report entitled “The Voice of the Patient” to the FDA in 2016. This is a detailed summary of the patient testimony presented at the meeting. This report will serve a critical function in communicating to both FDA review staff and industry the improvements that patients want and hope to see in their daily lives.
ARC is focused on developing the tools and strategies to represent the perspective and interests of patients, ensuring their needs are effectively addressed across the entire amyloidosis drug development continuum. The collective patient experience is critical to inform amyloidosis research and improve patient outcomes. As an advocate, your voice is powerful. By helping drug developers and policymakers understand what is fundamentally important to those who know amyloidosis personally, you can help us shape their decision-making.
There are many ways to get involved in advocacy. No matter what you choose, you’ll be playing a vital role in supporting the amyloidosis community.