NephCure Kidney International, Retrophin, AAKP Release Call to Action for Revolutionizing Rare Kidney Disease Diagnosis, Treatment, and Education
The call to action combines insights from leaders in government, industry and patient advocacy
KING OF PRUSSIA, Pa., Oct. 21, 2020 /PRNewswire/ -- NephCure Kidney International and Retrophin, in partnership with the American Association of Kidney Patients (AAKP), today released an expert paper with policy recommendations to coincide with American Society of Nephrology (ASN) Kidney Week 2020 Reimagined. The paper, entitled We Deserve Better: Revolutionizing Rare Kidney Disease, serves as a community call to action, outlining critical policy points for catalyzing innovation in rare kidney disease treatment.
"We're proud to work with Retrophin and AAKP to convene leaders in the community to address the needs of rare kidney disease patients and families," said Lauren Lee, executive vice president, stakeholder engagement for NephCure Kidney International. "The changes proposed in this comprehensive paper are critical to creating a better future for patients who have endured their conditions with limited treatment options."
For the last few decades, there has been little to no innovation in treatment for people living with rare kidney diseases, despite there being more than 150 identified rare kidney diseases. With no approved treatments for the conditions themselves, these patients experience reductions in quality of life and shorter life spans. Each day in the United States, 340 people begin dialysis and 13 patients die waiting for a kidney transplant.
Fortunately, recent receptiveness to new approaches from federal regulatory bodies has been met by a wave of clinical work as companies invest in the development of new treatment options for rare kidney diseases. Motivated communities are engaging in multi-stakeholder initiatives to connect patients to clinical research opportunities and address challenges that prevent families from getting the quality care they need.
In an effort to continue the momentum in rare kidney disease innovation, this community call to action was constructed from unique insights gained from extensive workgroups and a roundtable hosted by NephCure Kidney International and Retrophin last month, Dawn of a New Era in Rare Kidney Disease. The roundtable featured national patient advocates, nephrology community leaders, industry representatives, members of Congress and key staff from the Department of Health and Human Services.
"Patient consumers suffering from rare kidney diseases and their families share an expectation for greater innovation and a demand for policies that expand, not curtail, patient care choice and access to new treatments," stated Richard Knight, President of the American Association of Kidney Patients and a 13-year kidney transplant recipient. "AAKP proudly works alongside allies, including NephCure Kidney International and Retrophin, who embrace the view that the highest quality treatments for kidney patients are those that enable them to pursue the same universal aspirations shared by any other person, including opportunities to have a career, start a family, own a home and retire securely."
The paper proposes five key recommendations to advance the future of rare kidney disease care:
- Rare kidney disease awareness and education need to be dramatically increased to ensure the value of innovation is understood and prioritized.
- Diagnosis of rare kidney disease needs significant improvement through enhanced tools and protocols, potentially incorporating genetic testing.
- Diverse patients need access to community-level information and platforms to connect with healthcare providers and build trust, to communicate with each other for peer support, and to elevate their voices with policymakers and regulators.
- Nephrologists and other providers—including those in the primary care setting and those serving communities of color—need more education on rare kidney diseases to create a standard of care and reduce variable outcomes.
- Patients need earlier access to specialists and designated patient advocates, and providers must be comfortable having hard conversations about disease progression and steroids.
"We are excited to be part of this effort to kickstart a reimagining of how society approaches rare kidney disease education and care," said Eric Dube, chief executive officer of Retrophin. "The workgroups and roundtable discussion presented actionable recommendations to address the gaps in innovation and inclusion in rare kidney disease that have been absent for decades. We look forward to continuing the collaboration with leaders in the space as we aim for a better future for patients affected by rare kidney disease."
The call to action arrives during ASN Kidney Week 2020 Reimagined, the world's premier annual meeting in the practice of nephrology. The paper is intended to provide legislators with timely, actionable recommendations and serve as a launchpad for evolving how patients and providers view rare kidney disease diagnosis and treatment.
To download the paper and learn more about these critical issue areas in rare kidney disease, visit www.rarekidneyrevolution.com.
About NephCure Kidney International
NephCure Kidney International's mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now nearly 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. For more information, please visit www.NephCure.org.
About Retrophin
Retrophin is a biopharmaceutical company specializing in identifying, developing and delivering life-changing therapies to people living with rare disease. The Company's approach centers on its pipeline featuring sparsentan, a product candidate in late-stage development for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), rare disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease. Research in additional rare diseases is also underway, including partnerships with leaders in patient advocacy and government research to identify potential therapeutics for NGLY1 deficiency and Alagille syndrome, conditions with no approved treatment options. Retrophin's R&D efforts are supported by revenues from the Company's commercial products Chenodal®, Cholbam®, Thiola® and Thiola EC®.
About the American Association of Kidney Patients (AAKP)
AAKP is the oldest and largest kidney patient organization in the USA and among the strongest advocates for kidney patient consumer care choice. In 2019, AAKP launched the Decade of the Kidney™, an international effort to place kidney patients and their insights at the center of new innovations in kidney disease biologics, diagnostics and devices. Since 2019, AAKP, in partnership with George Washington University, has conducted the annual virtual Global Summit on Kidney Disease Innovations, which in gained an audience of over 20,000 participants from over 70 countries in 2020. For more information, visit www.aakp.org. Follow AAKP on social media: @kidneypatient on Facebook, and @kidneypatients on Twitter.
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SOURCE NephCure Kidney International
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