Introduction

The Rhizomelic Chondrodysplasia Punctata (RCDP) Patient-Focused Drug Development session is a landmark event being hosted by RhizoKids International during their 2024 RhizoKids Family Conference! This hybrid (in-person and online) meeting calls on 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. We need your voice to make this happen!

The information gathered at this session will be 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 will follow 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 will not be 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.

To help us make the PFDD meeting as impactful as possible, RhizoKids International and their collaborators request that the community attend the meeting in-person or virtually and make their voices heard. You can register to attend here.

Meeting

The RCDP PFDD session will be a hybrid event held on July 20, 2024 at the Children’s Harbor Camp on Lake Martin in Alabama. A live stream of the event, with the opportunity to participate by submitting comments, will be available on this site on the day of the event.

The meeting will include brief presentation on the purpose of the PFDD, as well as overview of the clinical aspects of RCDP given by Dr Michael Bober. This will be followed by two sessions with panelists, live polling, and comments by participants both on-site and attending virutally.

A recording of the meeting will be available after the meeting has completed 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.