On March 22, 2021, the PFIC Network has an opportunity to speak to the Food and Drug Administration (FDA) to share our stories as PFIC patients and caregivers. This is a very important time for the PFIC community. There are potential treatment options in development for PFIC, but there are still some huge hurdles to overcome. One of those hurdles is gaining approval from regulating agencies, including the FDA and the EMA. With all of the questions, all of the uncertainty and all of the hopelessness, the PFIC community needs to have more treatment options. We seek to take this opportunity WITH the patient community so that these regulatory agencies understand the needs of PFIC patients. This is an incredible opportunity to share our stories to help accelerate research and treatment development, for both these therapies and all future potential therapies.
Read on to learn more about who the FDA is and why a listening session is an important opportunity for our community.
Click here to learn how you as a patient or family member can participate in the FDA Listening Session
Who is the FDA?
The Food and Drug Administration (FDA) is a federal agency in the USA who is responsible for protecting and promoting public health through the control and supervision of food and medication safety, among other things.
The FDA is a regulatory agency. Regulatory agencies are responsible for balancing rapid approvals of effective therapies while ensuring that appropriate safety measures are met. A large hurdle for these regulatory agencies is to protect the public from unacceptable risk while balancing the need for patients and caregivers to have as fast a development timeline as possible.
Getting new treatments developed for any disease is a process. Being able to provide new therapies to patients requires the involvement of many stakeholders. These stakeholders include academic and government researchers, pharmaceutical and biotechnology companies, government regulators and patients and their caregivers. (View the role definitions of each stakeholder in a collaborative research model)
What is a FDA Listening Session?
An FDA Listening Session is an opportunity for those who experience a disease (such as PFIC) as a patient of caregiver to speak to the FDA. This allows the FDA the opportunity to understand the desperate need for further treatments to be developed. Listening sessions are used as a resource for these regulators to engage with patients or their advocates so that the patient community can share their experience with their disease by talking directly to FDA staff.
The listening session that PFIC Network will participate in will allow the patient community to help educate review staff about specific aspects of PFIC. It can help to inform those involved in research and development, as well as regulatory decision-making.
The Listening session is small, informal, non-regulatory and not public. It is only for PFIC and available to staff in multiple FDA Centers/programs. During the session, the FDA staff may simply listen to better understand our experience with PFIC or they may ask questions. Only FDA, patients, caregivers and advocates participate in the session.
For more information or to view the agendas and notes of other organizations listening sessions, visit the FDA website at this link https://www.fda.gov/patients/learn-about-fda-patient-engagement/patient-listening-sessions
What is the benefit to the PFIC community to participate in the FDA Listening Session?
PFIC patients and caregivers are experiencing urgent and unmet needs. It is difficult to simply sit back and wait for help, that is why we advocate. Only we can recognize first hand these urgent medical needs. It provides us the passion to see ways to improve symptoms and slow or halt disease progression. Our mission is to keep all stakeholders focused on the mission of rapidly developing safe and effective treatment options.
We, the patient and caregiver community, bring important medical insight to the community. We live with the symptoms and progression of the disease. Because of this, we can contribute valuable medical observations to researchers and an understanding of the priorities of the patients to industry and regulatory agencies.
We may value risks and benefits very differently than what is currently set aside by industry and government agencies. Without our perspective, these agencies may not recognize this as a strong enough benefit to patients and caregivers worth risks of therapy.
Main goal: to bring PFIC patients and researchers together so the patients understand research better and the researchers can get the perspective of the patients.
This page was developed using resources from the FDA website, www.fda.gov and from Global Genes, www.globalgenes.org, whose mission is to connect, empower and inspire the rare disease community.