ARPKD/CHF Alliance
The ARPKD/CHF Alliance is a nonprofit, 501(c)(3), public charity. The diagnosis of ARPKD and CHF can be devastating and the psychosocial stresses overwhelming.
We are the only organization in the world solely comitted to Autosomal Recessive Polycystic Kidney Disease (ARPKD) and Congenital Hepatic Fibrosis (CHF). The ARPKD/CHF Alliance has served patients and families since 2001, providing leadership on patient needs, thereby changing research priorities. We have far exceeded our initial goals, which was simply to generate more interest for both ARPKD and
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On August 29, 2023 the PKD Foundation will hold an Externally Led Patient-focused Drug Development (EL-PFDD) Meeting on Autosomal Recessive PKD (ARPKD) and Congenital Hepatic Fibrosis (CHF).
The goal of the meeting is provide to the U.S. Food and Drug Administration (FDA) the perspectives of patients and caregivers living with ARPKD, highlighting:
- The symptoms people living with ARPKD experience
- Which symptoms are the most important to families impacted by ARPKD
- What methods are people currently using to treat the symptoms of ARPKD
- How effective are these methods
- What they’re looking for in an ideal drug
- The tradeoffs (side effects) they’d be willing to accept in a treatment when thinking about the potential benefits, and preferences for clinical trials.
PFDD meetings bring together patients and care partners, FDA representatives, pharmaceutical companies, doctors, and researchers who are experts in the particular disease.
For the meeting on ARPKD, the goal will be to hear from patients and their caregivers about what it’s like to live with a kidney and liver disease such as ARPKD and CHF, so the FDA and pharmaceutical companies can understand the patient experience.
This patient input can 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.
After the meeting, a report titled "Voice of the Patient" will be sent to the FDA. This will be a reference for future decisions about potential medicines for ARPKD.
The ARPKD PFDD meeting will take place from 10am-3pm EST on Tuesday, August 29, 2023. This meeting will be virtual in order to allow as many of our ARPKD community to join as we can. This means that, even if you aren’t a panelist, you can still participate through live online polls, telephone call-ins, and by providing written comments in advance.
Agenda:
1. Welcome (Elise Hoover, PKDF Vice President of Research Programs)
2. FDA Opening Remarks (Dr. Kirtida Mistry)
3. ARPKD clinical overview (Dr. Erum Hartung, University of Pennsylvania)
4. Session 1 – Living with ARPKD Symptoms and Daily Impact
1. Patient/Caregiver Panel 1
5. Audience polling and moderated discussion
6. ARPKD treatment overview (Dr. Max Liebau, University Hospital of Cologne)
7. Session 2 – Current and Future Treatments for ARPKD
1. Patient/Caregiver Panel 2
8. Audience polling; moderated discussion
9. Closing Remarks (Elise Hoover, PKDF Vice President of Research Programs)
Ways to participate:
Pre-register. Provide your name and contact information to keep you informed about the activities surrounding this meeting. Anyone who lives with or is interested in ARPKD, including patients of all ages, care-partners, families, and friends are welcomed to attend. Each person (e.g., child, spouse, etc.) must register separately.
Survey:
To prepare for the upcoming meeting on ARPKD, we need your input on how ARPKD and CHF affect you/your child’s life. All information you provide in the survey will be kept strictly confidential.
You will need the following information to take the survey:
- Approximately how old you were when you were diagnosed with ARPKD
- Your latest eGFR (kidney function number)
- A list of medications currently taken to treat ARPKD, CHF, or other related conditions
FAQs
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
Who benefits from PFDD meetings?
Patients and families: know the FDA and drug sponsors have heard their voices.
Patients and 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.
The FDA gains understanding of what it is 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 and the ARPKD/CHF Alliance) 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 patient 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 patient 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 PFDD meeting?
Click here to register for the meeting. Each person (e.g., child, spouse, etc.) must register separately.
Can pediatric patients attend the 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 [email protected]
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