Published May 14, 2019

PKD patients are very well-informed about their disease and its consequences. As a disease impacting multiple generations or appearing unexpectedly as a spontaneous mutation, patients and their families witness first-hand the progression of the disease individually and from generation-to-generation. They recognize how ongoing research and breakthrough medications can help patients live longer, healthier lives.

As a highly motivated group, patients are often looking for opportunities to become involved in research. The statistics are telling. Out of the 600 we asked, an astonishing 64 percent of patients said they already have or would like to sign up for Accelerating Clinical Trials (ACT) alerts that message patients about clinical studies in their geographic area. Sixty percent said they would like to participate in a clinical trial.

Patient-centered outcomes

Today, we’re seeing more opportunities for participation for PKD patients throughout the U.S. because biomedical research and drug development are becoming increasingly patient-centered. Such a focus is affirmed in the vision of organizations like the Patient-Centered Outcomes Research Institute (PCORI) that states, “Patients and the public have information they can use to make decisions that reflect their desired health outcomes.”

This might include enrolling in a clinical trial or joining a database or registry. But current registries often capture only a few data points like contact information or focus on limited research questions. Because information about their eligibility is limited, patients must go to great lengths to see if they qualify for upcoming trials and studies.

The ADPKD Registry is coming

We believe it should be easier for patients to get involved. As a part of the PKD Foundation’s mission, this includes offering opportunities to participate in PKD research in a way that’s meaningful and attainable. Later this year, we will launch a nationwide ADPKD Registry, a collection of individuals with Autosomal Dominant Polycystic Kidney Disease (ADPKD). The patient-centered Registry offers the potential to gain insights into PKD from the individuals living with the disease.

In the Registry, patients can self-report information including:

• Diagnosis
• Family history
• Symptoms
• Medication
• Impact PKD has had on their quality of life

The ability to regularly update a personal profile ensures information is up-to-date and reflects the current status of an individual’s disease progression. With the information provided, we will be able to offer information for health care service planning and help patients connect to clinical studies for which they’re eligible. We will also learn about disease progression from the patient’s perspective rather than as a collection of laboratory values.

“The ADPKD Registry will serve to alert patients to clinical trials for which they’re a best match,” says David A. Baron, Ph.D., Scientific Advisor, PKD Foundation. “This helps the patient as well as the sponsor of the study get the answer as expeditiously as possible. Going forward it will give the medical community a better idea of the issues that are most important to someone with ADPKD.”

By sharing information, patients can fully engage in assisting researchers with the development of PKD treatments by offering insights to how symptoms change as individuals age, providing a clearer overall picture of the lives of PKD patients.

This is the first in a series of blogs exploring the PKD Foundation’s new ADPKD Registry.

Find out more or sign up here for the ADPKD Registry!