Externally-Led Patient-Focused Drug Development Meeting for ARPKD

August 28, 2023 Virtual Meeting for ARPKD Patients and Their Families

We’re incredibly grateful for everyone who participated in the Externally-Lead Patient Focused Drug Development meeting on ARPKD. Your input and participation will help the FDA make informed decisions on approvals of potential medicines for ARPKD and help pharmaceutical companies to design therapies and clinical trials that are meaningful for patients. 

The meeting brought together the ARPKD community — patients, parents, and stakeholders — to make a change. Many families and individuals bravely shared their stories because of their commitment to accelerate a cure for PKD.  

You can also submit comments for the next 30 days, if you’d like to share details of the impact of ARPKD, CHF, or other related complications.  

Again, thank you for your passion, participation, and commitment. 

Additional Resources

Voice of the Patient Report

PKDF will write the Voice of the Patient Report which will summarize the meeting. This report will be publicly posted on PKDF’s website.
FDA will post a link on their website to the report as well.


What’s the difference between ARPKD and ADPKD?

There are two types of PKD: autosomal dominant PKD and autosomal recessive PKD. ADPKD is the more common type and affects more than 600,000 Americans and 12.4 million people worldwide. ARPKD is a rare form of the disease that occurs in 1 in 25,000 children worldwide.

Why is the FDA coming to this meeting?

The PFDD initiative started in 2012 as part of FDA’s commitments under the Prescription Drug User Fee Act (PDUFA) V. After conducting FDA-led PFDD meetings, FDA recognized there are many more diseases/conditions that can be addressed beyond those that were planned and conducted by FDA.

To help expand the benefits of FDA’s PFDD initiative, in 2015, FDA announced the opportunity for externally-led (EL-PFDD) meetings. EL-PFDD meetings are planned and hosted by patient organizations, with the input of FDA staff, and use the process established by FDA-led PFDD meetings as a model. Learn more here https://www.fda.gov/industry/prescription-drug-user-fee-amendments/externally-led-patient-focused-drug-development-meetings

What will the EL-PFDD meeting look like?

Watch a recording of a similar EL-PFDD meeting from the National Kidney Foundation.

Additionally, an informational webinar in advance of the meeting on ARPKD will be held on August 8, 2023. Stay tuned for details on how to register.

Who benefits from EL-PFDD meetings?

Patients: know the FDA and drug sponsors have heard their voices. Patients’ experiences are validated, reducing feelings of isolation. Hearing other patients voice their experiences and needs helps patients to better self-advocate. EL-PFDD meetings can also help to bring new treatments to the market, which benefits patients.

FDA: gains understanding of what it’s like to live with a particular disease. The FDA becomes informed of side effects and risks patients may be willing to accept to gain a certain level of symptom relief or slowing of their disease progression. The FDA learns about patients’ needs regarding new drugs, and what their preferences are for clinical trials for their disease. EL-PFDD meetings assist the FDA in knowing if a new drug addresses patient needs.

Patient advocacy groups: EL-PFDD Meetings help these groups (like the PKD Foundation) identify what needs exist for patient education and advocacy. More effective advocacy increases public awareness and knowledge of the disease. In addition, these meetings help patient advocacy groups connect patients with their peers. 

Pharmaceutical companies: gain insights into the major concerns of patients. This helps the companies develop treatments and design clinical trials that match patients’ needs and preferences. Drug sponsors learn which disease symptoms or treatment side effects are, or are not, tolerable by the patients. This helps the companies develop drugs that matter to patients. With knowledge gained from EL-PFDD Meetings, pharmaceutical companies receive advice from the FDA on developing potential drugs and therefore help to advance medicines that meet patients’ needs.

What does it cost to attend the meeting?

This meeting is free and open to the public.

How do I register for the ARPKD EL-PFDD meeting?

Click here to register for the meeting. Each person (e.g., child, spouse, etc.) must register separately.

Can pediatric patients attend the EL-PFDD meeting on ARPKD?

Yes! We welcome individuals with ARPKD of all ages.

What happens after the meeting?

PKDF will write the Voice of the Patient Report. This will be sent to the FDA and will be used to help in their decisions to approve potential new treatments for ARPKD. The Voice of the Patient Report will also be publicly posted on PKDF’s website.

Where can I find more information about ARPKD and PKDF’s efforts to support the community?

Click here to explore patient and physician resources, consider advancing research by participating in a clinical study, learn more about the PKD Foundation’s investment in ARPKD research, and mark your calendar for national awareness events. https://pkdcure.org/what-is-arpkd/arpkd-resources/


Still have questions? Email research@pkdcure.org


Meeting organizers


Meeting sponsors