This rare kidney disease is symbolic of a bigger problem in the US health care system | Opinion

It is National Kidney Month, a period when lawmakers, health care organizations and communities unite to raise awareness about kidney disease and its associated risks. More broadly, Congress makes kidney disease a priority by supporting legislative measures that will bolster the kidney disease workforce and increase kidney care education and awareness, yet that barely scratches the surface in spotlighting the nuanced challenges faced by those with rare and specific kidney diseases, such as polycystic kidney disease, also known as PKD.

PKD, though classified as a rare condition, stands as a poignant example of a disease lacking the tailored policy solutions necessary to address its complexities. Too often, the voices of PKD patients and other rare kidney disease groups are overlooked on Capitol Hill. It’s imperative that Congress listens now.

PKD is a chronic, life-threatening condition characterized by the uncontrolled growth of fluid-filled cysts on the kidneys, which will inevitably lead to kidney failure. PKD is heredity, and often casts a long and somber shadow on generations of families who grapple with the heavy knowledge that they have a 50% chance of passing PKD onto their children. Imagine, for a moment, knowing that the intense struggles you have fought throughout your life may eventually become the same battles your children must experience.

PKD patients can fall through the cracks in America’s health care system. With limited treatment options, these patients are forced to hunt for a physician well-versed on PKD who can provide the information necessary to help them manage their condition. This is a daunting journey as there are only a limited number of clinicians who currently provide specialty care for the estimated 600,000 people living with PKD.

Ultimately, PKD’s disease trajectory leaves patients with two treatment options: dialysis or a kidney transplant — the latter of which is challenging for many to come by. According to the Health Resources and Services Administration, each day 17 people die while waiting for an organ transplant, 12 of whom are waiting for a kidney.

Beyond its health implications, PKD also comes with a hefty price tag for patients and Medicare. As the fourth leading cause of kidney failure, PKD represents approximately 5% of end-stage renal disease patients, translating to roughly $1.75 billion of the estimated $35 billion this condition costs to Medicare.

Despite PKD’s costly and unique challenges, policymakers have historically employed a one-size-fits-all approach to kidney disease management. While effective in addressing broader issues contributing to kidney disease, this approach falls short in tackling the underlying mechanisms responsible for PKD.

As we enter the fiscal year 2025 appropriations season, our dedicated advocates are again set to engage Capitol Hill on April 9. They will aim to highlight the unique challenges and needs of those affected by PKD. Our ongoing advocacy efforts aim to secure PKD’s inclusion as a topic area within the Department of Defense’s Peer-Reviewed Medical Research Program, which has allocated millions of dollars toward PKD research funding over the past five years — a testament to our commitment to advancing understanding and treatment of this disease.

Furthermore, the PKD Foundation has consistently championed the inclusion of inherited kidney diseases, such as PKD, in the surveillance research conducted by the Centers for Disease Control and Prevention’s Chronic Kidney Disease program. The inclusion of PKD-specific data sets would not only deepen our understanding of the disease’s prevalence and its broader impact, but could spur research, draw critical investments, and foster advancements in diagnosis and treatment strategies.

The PKD community’s battle is symbolic of a broader challenge within our health care system to recognize and address the unique needs of those with rare diseases. The PKD Foundation remains committed to this cause, advocating tirelessly for our patient community. But we cannot do it alone. We need the collective effort of policymakers, clinicians, researchers, those living with PKD and those who care and love for them to join us in this fight, ensuring no one is left in the shadows. By standing united, we can turn hope into action and make a lasting impact on the lives of those living with polycystic kidney disease.

Susan Bushnell is president and chief executive officer of the PKD Foundation in Kansas City, Missouri, the only organization in the United States solely dedicated to finding treatments and a cure for polycystic kidney disease.