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An exciting initiative by the OHF to inform the FDA about the patient perspective of living with Primary Hyperoxaluria (PH)

Make Your Voice Heard
A Virtual World-Wide Event

This is your one-time opportunity to share your story of living with Primary Hyperoxaluria with the FDA. It doesn't matter where you live, where you are in your journey, what matters is your story.

The primary goal of this meeting is to hear directly from patients and caregivers about their perspectives on living with Primary Hyperoxaluria and their experiences with treatments, to improve the development of new drugs in the research pipeline. The patient perspective is critical in helping the U.S. Food and Drug Administration (FDA) understand the context in which regulatory decisions are made for new drugs.

Please join us virtually, on Monday, October 5th, to share
your patient perspective on Primary Hyperoxaluria and treatments.

We invite anyone who has PH, lives with or is interested in PH –
patients of all ages, care-partners, families and friends – to attend.

Register

Anyone who lives with or is interested in Primary Hyperoxaluria (PH) - patients of all ages, care-partners, families and friends, healthcare professionals, researchers, industry - are welcomed to attend.

Each person (e.g., child, spouse, etc.) must register separately.

REGISTER

Submit Your Comments

If you are a person living with Primary Hyperoxaluria or a caregiver (e.g., spouse, parent), please share your experiences that relate to the following questions:

Of all the symptoms of the condition, which 1-3 symptoms have the most significant impact?

How do these sypmtoms impact your activities of daily living and quality of life?

What information is important for you to understand when considering participation in a clinical trial?

What are you currently doing to manage your symptoms, and how well do these treat your symptoms?

Are there any downsides to your treatments (e.g., bothersome side effects)?

OHF EL-PFDD

About the OHF EL-PFDD Meeting

The meeting format will be very interactive and include information on current and future PH treatments, hear direct voices from patients on their perspectives living with the disease and hopes for clinical trials and treatments, and a live Q&A with the audience. Audience participants will have a chance to comment during the meeting via phone and email! You can see the full meeting agenda here.

About EL-PFDD Meetings

EL-PFDD meetings bring together patients and their care-partners, representatives from the U.S. Food and Drug Administration (FDA), pharmaceutical companies interested in developing drugs for the disease, and doctors who are experts in the particular disease- all to hear from patients about the disease in question. In these meetings, the patient's experience is brought to the forefront for the FDA and pharmaceutical companies to understand. "Externally-led" refers to PFDD meetings that are led by organizations outside of the FDA.

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 PH.

More information on EL-PFDD meetings can be found at:
http://www.fda.gov /downloads/Drugs/ NewsEvents/UCM493616.pdf

Voice of the Patient Report

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 PH.

Who benefits from EL-PFDD meetings?

Food and Drug Administration (FDA)

  • The FDA gains understanding of what it’s like to live with a particular disease.

  • Inform the FDA 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.

  • Reveals patients needs regarding new drugs, and what their preferences are for clinical trials for their disease.

  • Assists the FDA in knowing if a new drug addresses patient needs.

Patients

  • Patients know the FDA and drug sponsors have heard their voices (in person).

  • Patients’ experiences are validated, reducing feelings of isolation.

  • Hearing other patients voice their experiences and needs helps patients to better self-advocate.

  • All of these points give patients hope that new and effective drugs will become available.

Patient advocacy groups

  • EL-PFDD Meetings help these groups 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 face-to-face with their peers.

Pharmaceutical companies

  • Drug sponsors 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, the FDA can advise pharmaceutical companies on developing potential drugs and therefore help to advance medicines that meet patients’ needs.

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