Rare Diseases Working Group
AfPA’s Rare Diseases Working Group is a network of policy-minded health care providers who advocate for patient-centered care.
By participating in advocacy initiatives and the development of educational resources, working group members ensure that the clinician’s perspective informs policy discussions that impact health care for people living with a rare disease.
To learn more, or to join AfPA’s Rare Diseases Working Group, please email info@allianceforpatientaccess.org.
Policy Resources
White Papers & Policy Briefs
- Treatment Options & Access for EBV+ PTLD
Alliance for Patient Access, October 2022 - Addressing Unmet Needs in the PH1 Patient Population
Alliance for Patient Access, October 2022 - In Their Own Voices: The Lived Experiences of IgG4-RD Patients [Report Update]
Alliance for Patient Access, September 2022 - Amyloidosis Initiative Meeting Summary
Alliance for Patient Access, September 2022 - Patient-Centered Care: A Position Statement
Alliance for Patient Access, August 2022 - EBV+ PTLD Initiative Meeting Summary
Alliance for Patient Access, April 2022 - In Their Own Voices: The Lived Experiences of IgG4-RD Patients
Alliance for Patient Access, December 2021 - Genetic Testing & Counseling for Rare Hereditary Diseases
Alliance for Patient Access, September 2021 - 2021 Working Group Meeting Summary
Alliance for Patient Access, May 2021 - In Conversation: A Physician Discusses COVID-19 & Rare Disease
Alliance for Patient Access, August 2020 - Telemedicine: A Position Statement
Alliance for Patient Access, July 2020 - Fast Facts: Clinical Diagnosis Guidelines for Transthyretin Amyloidosis
Institute for Patient Access, July 2020 - Health System Barriers for Amyloidosis Patients
Institute for Patient Access, May 2020 - High Out-of-Pocket Costs: A Position Statement
Alliance for Patient Access, July 2019 - Co-Pay Accumulator Programs: A Position Statement
Alliance for Patient Access, May 2019 - Prior Authorization: A Position Statement
Alliance for Patient Access, April 2019 - Step Therapy: A Position Statement
Alliance for Patient Access, January 2019 - Co-pay Accumulator Programs: What Can Policymakers do to Protect Patients?
Institute for Patient Access, August 2018 - Co-pay Accumulator Adjustment Programs
Institute for Patient Access, June 2018 - Fast Facts: The Institute for Clinical and Economic Review
Institute for Patient Access, April 2019
Videos
- Innovation: A Mother’s Story
Institute for Patient Access, April 2022 - Rare Disease Advisory Councils Give Patients a Voice
Alliance for Patient Access, April 2022 - Patient-Centered Care for Amyloidosis
Alliance for Patient Access, August 2021 - Challenges in Rare Disease Treatment
Alliance for Patient Access, November 2020 - Why Health Care Providers Should Care About ICER
Institute for Patient Access, October 2019 - The Co-Pay Surprise
Institute for Patient Access, May 2018
Infographics
- Accessing Treatment for Amyloidosis
Alliance for Patient Access, October 2022 - The Danger of Prior Authorization for Rare Cancer Patients
Alliance for Patient Access, September 2022 - Accelerated Approval & Rare Disease Patients
Alliance for Patient Access, October 2021 - Rare Diseases & Cost Sharing
Alliance for Patient Access, February 2021 - What Stands Between Patients and Treatment for Rare Diseases?
Alliance for Patient Access, January 2021 - Challenges in Diagnosing Rare Diseases
Alliance for Patient Access, December 2020 - Access Barriers for Patients with Rare Diseases
Alliance for Patient Access, June 2020
Blogs