Introduction
The Rhizomelic Chondrodysplasia Punctata (RCDP) Patient-Focused Drug Development session was a landmark event hosted by RhizoKids International during their 2024 RhizoKids Family Conference! This hybrid (in-person and online) meeting allowed those who have experience with RCDP (previous or past) to communicate “what matters most” about the disease, it’s management, and caring for patients. The FDA is continuing to put more and more emphasis on the voice of the caregiver with respect to what would be considered clinically meaningful improvement, and considers PFDDs and PFDD-like events a high priority in the drug development review process. Thank you to everyone who leant their voice to make this happen!
The information gathered at this session is currently being used to generate a “Voice of the Patient” report that can then be used by the Food and Drug Administration (FDA), drug developers, and other stakeholders to better understand the wants and needs of the community.
The meeting followed the model developed by the FDA for their internally and externally-led PFDD meetings, although due to the ultra-rare nature of RCDP and the urgency of capturing the needs of RCDP patients, the FDA was not in attendance at this meeting. By following the roadmap set out by the agency we are confident that they will still consider the valuable insights gained at the meeting.
Meeting
The RCDP PFDD session was a hybrid event held on July 20, 2024 at the Children’s Harbor Camp on Lake Martin in Alabama.
The meeting included a brief presentation on the purpose of the PFDD, as well as overview of the clinical aspects of RCDP given by Dr Michael Bober. This was followed by two sessions with panelists, live polling, and comments by participants both on-site and attending virtually.
A recording of the meeting will be available soon for those who want to rewatch the session, or those who were not able to attend live.
Agenda
9:30-9:35 a.m. Welcome
Tim Franson, M.D., Meeting Moderator
Principal, Faegre Drinker Consulting
9:35-9:40 a.m. Opening Remarks
Melinda Holladay
President of the Board, RhizoKids International
9:40-9:50 a.m. Overview of Patient-Focused Drug Development
David R. Zook, J.D.
Chair, Faegre Drinker Consulting
9:50-10:05 a.m. Overview of RCDP Symptoms and Treatment Approaches
Michael B. Bober, M.D.
Skeletal Dysplasia Program, Nemours Children’s Hospital – Delaware
10:05-10:25 a.m. Discussion Format Overview and Demographic Polling
Tim Franson, M.D., Meeting Moderator
10:25-10:35 a.m. Break
Topic 1: Symptoms of RCDP and Impact on Daily Life
10:35-11:00 a.m. Panel Remarks
11:00-11:45 a.m. Facilitated Discussion
11:45 a.m.-1:00 p.m. Lunch Break
Topic 2: Views on Approaches to Treatment and Future Treatments
1:00-1:25 p.m. Panel Remarks
1:25-2:10 p.m. Facilitated Discussion
2:10-2:30 p.m. Closing Remarks
Tim Franson, M.D., Meeting Moderator
Voice of the Patient Report
The Voice of the Patient report will summary the key points discussed at the meeting, as well as the information collected as part of the pre-meeting survey and in-meeting polling. The focus of the report will be to provide an overview of the challenges faced by individuals living with RCDP and the views of caregivers on current and future treatment options.
The report will be made publicly available here and on the RhizoKids International website when available and will be an incredible resource for the RCDP community in their interactions with health providers, therapists, and other stakeholders.
Who is organizing the RCDP PFDD Session?
The meeting is being held as a collaboration between RhizoKids International and Med-Life Discoveries. While Med-Life has been involved in the overall planning of the PFDD session, they have had no input on the perspectives shared by panelists or attendees and will attend this session as an observer.
The consulting and underwriting costs associated with the meeting are being covered by Med-Life Discoveries.