Meeting Goals, Objectives & Key Learnings
Externally Led Patient Focused Drug Development Meeting
It is critical to showcase the profound impact that classic CAH has not just on the lives of affected individuals, but also on families who suffer because of a lack of safe, effective therapies. (The goal of the EL-PFDD meeting is to provide the FDA and product developers with the unique perspectives of both individuals living with CAH and their caregivers.) Affected individuals will share the impact of their condition on quality of life and the challenges with current treatment options.
Our objective of the meeting is to educate FDA and other meeting attendees on the impact that classic CAH has on diagnosed individuals’ every day life, as well as to document disease severity among those living with CAH. The voices of people living with CAH, as well as their caregivers, will contribute to a summary of the day’s proceedings in the form of a “Voice of the Patient” (VoP) report that can be used by FDA staff as well as other stakeholders in the drug development ecosystem. This meeting will enable the FDA, industry, and stakeholders to become more sensitive to disease manifestations and progression and the risk-benefit tolerance of people with CAH and their caregivers.
Both FDA-led and EL-PFDD meetings have key characteristics that set them apart from other public meetings. PFDD meetings target disease areas for which there is:
- an identified need for patient input
- a condition area that is chronic, symptomatic, or affects functioning and activities of daily living
- a condition area for which aspects of the disease are not formally captured in clinical trials
- a condition area for which there are currently no therapies or very few therapies, or the available therapies do not directly afect how a patient feels, functions, or survives
- condition areas that have a severe impact on identifiable subpopulations (such as children or the elderly)
PFDD Potential Outcomes
- EL-PFDD meetings are a great networking and community building opportunity for the patient community and EL-PFDD meetings can help to channel patient engagement and create momentum in the patient community. Several host patient organizations have hosted follow-up meetings to build on this momentum and have synergized their efforts with other patient organizations in the space.
- EL-PFDD meetings may also serve to identify opportunities for further discussions with patients about their condition and may also generate new research questions.
- EL-PFDD meetings offer an opportunity to foster relationships between patient organizations and industry/medical professionals.
CONTACT
2414 Morris Avenue, Ste 110
Union, NJ 07083
Phone: (908) 364-0272
Toll Free: (866) 227-3737
Fax: (908) 686-2019
contact@caresfoundation.org
