Patient-Focused Drug Development Meeting on Classical Homocystinuria (HCU)

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Classical HCU Externally Led Patient-Focused Drug Development (PFDD)

An exciting initiative by HCU Network America to inform the FDA and other stakeholders about the patient perspective of living with Homocystinuria (HCU) due to Cystathionine Beta-Synthase Deficiency, better known as Classical HCU.

This meeting will be broadcasted with a NOVEL NEWSCAST FORMAT, with patient testimonies, remote audience participation

The meeting agenda focuses on two patient panels and audience discussion sessions on living with Classical HCU, treatments for the disease, and clinical trials for Classical HCU.

Classical Homocystinuria EL-PFDD

Friday, October 27, 2023 - Virtual


What are EL-PFDD Meetings?

Externally led patient-focused drug development (EL-PFDD) meetings bring together patients and care partners, US Food and Drug Administration (FDA) representatives, pharmaceutical companies, doctors who are experts in the particular disease, and other stakeholders. For the meeting on Classical HCU, the goal is to hear from patients on what it's like to live with the condition so that we can better understand the patient experience. This information can help the FDA to make informed decisions on approvals of potential medicines for Classical HCU, and pharmaceutical companies to design clinical trials that are meaningful for patients.

Event Details
Oct. 27, 2023 | 10:00 am-3:05 pm ET

This will be a virtual meeting. We will email you the link to join the virtual meeting in advance of the meeting date.

Voice of The Patient
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 Classical HCU.


Find Out More
Please stay tuned - we will have a community webinar before the meeting as well as post the recording and the slides after.


Who benefits from EL-PFDD Meetings?

Food and Drug Administration (FDA)
The FDA gains an 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.

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 help to bring new treatments to the market, which benefits patients.


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 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 the 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.


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