PSC Partners Issues a Call to Action in Response to the FDA Rare Disease Framework Public Meeting

DENVER--(BUSINESS WIRE)--PSC Partners Seeking a Cure commends the U.S. Food and Drug Administration (FDA) for convening the Rare Disease Framework public meeting and engaging directly with the rare disease community. This dialogue is an important opportunity to build on our shared commitment to advancing safe, effective treatments for people living with rare diseases.

PSC Partners is calling on policymakers and the FDA to translate that engagement into decisive action to accelerate the development and regulatory review of treatments for primary sclerosing cholangitis (PSC), a rare, relentless, and potentially life-threatening liver disease. Characterized by chronic inflammation and scarring of the bile ducts, PSC causes progressive liver damage and places patients at risk of devastating complications, including liver failure, certain cancers, and the need for liver transplantation. Despite the severity of this disease, there are currently no FDA-approved therapeutic treatments proven to slow, halt, or reverse its progression or to address the many burdensome symptoms of PSC. The rarity of PSC, combined with an incomplete understanding of its underlying causes and unpredictable disease progression, presents significant challenges for researchers and drug developers. There is an urgent need for greater regulatory flexibility, innovation, and clear pathways to bring effective treatments to patients.

We acknowledge the challenges of reviewing a drug for approval and the need to ensure patient safety. However, there needs to be a full understanding of the risk patients are willing to accept to have treatments for PSC and related symptoms. Multiple published sources show that patients with life-threatening conditions, especially those without a treatment, are willing to accept a level of unknown risk to improve their quality of life and survival. A recent global survey of more than 1,100 PSC patients and caregivers conducted by PSC patient organizations showed that the vast majority would accept additional monitoring requirements—including blood tests, clinic visits, and liver biopsies—to gain access to disease-modifying and symptom treatments. These findings underscore the importance of incorporating patients’ perspectives on risk and benefit into treatment development and regulatory review.

Our community has been an active partner in the discussions with clinicians, researchers, industry and regulators since the initial PSC Endpoints Consensus Conference in 2015. We have consistently ensured that patient priorities have been front and center in multiple forums, including a patient-led FDA sponsored Patient Focused Drug Development meeting in 2020. Despite these activities, the pathway for getting a drug approved remains fraught with significant hurdles. The pathways to approval often change from when a study starts to when a drug is finally reviewed by regulatory authorities. New potential treatments are being delayed, and many drug developers have abandoned promising therapies due to these challenges.

PSC Partners stands with patients, families, clinicians, and researchers, serving as both a vital amplifier of their voices and an active force driving the urgent demand for new treatment options and continued progress against PSC.

“For people living with PSC and their families, the need for new treatment options cannot be overstated,” said Ricky Safer, Founder and CEO of PSC Partners. “Patients deserve a driving voice in the development and review of the treatments that will dictate their futures. We need a regulatory and research environment that aggressively fosters innovation while maintaining rigorous safety standards.”

PSC Partners Seeking a Cure is pressing urgently for:

• A critical, sustained commitment of resources to rare disease research and FDA oversight, ensuring that funding constraints never delay the development and review of life-saving therapies.
• Meaningful inclusion of the patient voice throughout treatment development and regulatory review.
• Clear, consistent, and streamlined regulatory review processes that maintain rigorous safety standards while eliminating unnecessary delays.

Patients with PSC urgently need access to effective treatments, and the regulatory process must reflect the severity and urgency of this disease. While there are no therapeutic treatments to stop PSC, the ultimate intervention is a liver transplant. A transplant is a measure of last resort: if a liver becomes available on time, post-transplant care presents its own severe challenges, and PSC returns in the new liver for 40% of patients. The acute demand for liver transplants in PSC places a severe burden on healthcare systems and further depletes the critically limited supply of life-saving organs available for other patients.

PSC Partners Seeking a Cure maintains that patients must remain at the absolute center of all efforts to advance PSC research and treatment development. Unwavering collaboration among patients, families, clinicians, researchers, industry, and policymakers is essential to aggressively accelerate progress while rigorously protecting patient safety.

About PSC Partners Seeking a Cure

PSC Partners Seeking a Cure is a 501(c)(3) nonprofit dedicated to funding research to discover treatments and a cure for primary sclerosing cholangitis (PSC). Founded in 2005, the organization supports patients, caregivers, and families through education, advocacy, and community connection while advancing scientific progress through its research grants and patient registry. With a global network of more than 35,000 patients, PSC Partners collaborates with leading clinicians, researchers, and the patient community to improve outcomes and drive toward a cure. For more information, visit www.pscpartners.org.