Access to Treatment

Regulatory Approval

ARC has an exceptional relationship with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and other regulatory bodies around the world. We partner closely with regulators to ensure they have a thorough understanding of amyloidosis and to improve the design of registration clinical trials. We also work with regulators to ensure their decisions about whether a new treatment should be approved, based on an assessment of both its benefits and risks, take into consideration the views of amyloidosis patients and their families.

Download the Voice of the Patient report to learn more about our Patient-Focused Drug Development initiative with the FDA.


Government and private payers in the United States make their own decisions about whether they fund and how to fund new treatments, sometimes using health technology assessment reports to guide their decisions. ARC works closely with groups like the Institute of Clinical and Economic Review (ICER) providing detailed reports to educate payers to help them understand amyloidosis and the value of new treatments to patients and their families. We also encourage them to make it as easy as possible for patients to understand the process for accessing a treatment and to minimize the personal costs for patients and families.

Read our latest submission to ICER about the value of Patisiran and Inotersen, two new treatments for hATTR amyloidosis.

European Health Technology Assessment

After gaining marketing authorization, a new treatment is sometimes evaluated under a process called health technology assessment (HTA) before it is funded by national health systems or payers. This process looks at how ‘cost-effective’ a new treatment is compared to other treatments that may already be available. ARC works closely with HTA bodies around the world to find solutions to approve and fund promising amyloidosis treatments so that patients can benefit. We also work with the pharmaceutical companies, advising them on how to meet the specific data needs of HTA bodies, and find ways to make effective treatments available to patients as quickly as possible.

Guidance for Patients and Families

ARC provides information and helps patients and their families naviagate accessing treatments through clinical trials, expanded access programs and once a treatment is approved.

320 Nevada Street, Suite 210 Newton, MA 02460 USA | 617-467-5170

ARC is a 501(c)(3) nonprofit organization. Tax ID: 47-2589708
©2024 ARC – Amyloidosis Research Consortium, Inc. All rights reserved.

Guidestar Platinum Transparency 2023 2021 Top Rated Great Non Profits Charity Navigator Four-Star Rating