Description
As part of the global observance of Rare Disease Week, we will host FDA's Rare Disease Day 2024, a virtual public meeting, on Friday, March 1, 2024, 9:00 am – 4:30 pm ET. This year's theme is "FDA's Rare Disease Day 2024-Dedicated to Patients and Providers."
This event is for patients with rare diseases, their families, caregivers, patient advocates, and their local healthcare providers, e.g., physicians, pharmacists, pharmacy tech, nurses, physician assistants. The goals of this year's RDD are fourfold:

1. In the future, to improve diversity and inclusivity, it is expected that more clinical trial investigations will have decentralized designs in which some or all of the trial-related activities occur at locations other than traditional clinical trial sites. Local health care providers will play a greater role in decentralized trials (DCTs). To help prepare, this event will stimulate awareness of DCTs, provide general information on clinical trials, and describe how FDA reviews products to ensure the legal standards for approval are met.
2. To empower patients and providers with information related to clinical trials and medical products that may be difficult to find.
3. Describe various ways for the rare disease patient community to connect with FDA and get involved with our outreach and engagement programs. Collaborating with our stakeholders is critical to ensure the patient voice informs medical product development and regulation.
4. Familiarize the audience with FDA's cutting-edge initiatives to improve accelerate product development for rare diseases.

Learners will have the opportunity to submit questions to panelists using the chat feature or by emailing OOPDOrphanEvents@fda.hhs.gov.

References

• FDA, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Guidance for Industry (Dec 2019) (accessed at https://www.fda.gov/media/133660/download).
• FDA, Decentralized Clinical Trials for Drugs, Biological Products, and Devices, Guidance for Industry, Investigators, and Other Stakeholders (May 2023) (accessed at https://www.fda.gov/media/167696/download).
• FDA’s Labeling Resources for Human Prescription Drugs for Industry (Jul 2023) accessed at (accessed at https://www.fda.gov/drugs/laws-acts-and-rules/fdas-labeling-resources-human-prescription-drugs).
• FDA, Informed Consent: Guidance for IRBs, Clinical Investigators, and Sponsors (Aug. 2023), (accessed at https://www.fda.gov/regulatory-information/search-fda-guidance-documents/informed-consent).
• FDA, Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products: Guidance for Industry(Aug 2023) (accessed at https://www.fda.gov/media/171667/download).
• FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making, (Apr 2023) (accessed at https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical).

Learning Objectives

• Describe the legal framework for approving medical products at FDA and the differences between those for drugs and biologics versus devices.
• Define what decentralized clinical trials are and why such trials may improve diversity and inclusivity in clinical trials and improve trial efficiency.
  
• Identify where to find important information and documents related to clinical trials and medical products in the United States.
  
• Define the types of information that can be obtained from medical product labels.
  
• Explain, in general terms, the legal and ethical requirements for consent forms in clinical trials.
  
• Recall key FDA initiatives intended to advance and accelerate medical product development for rare diseases.

Target Audience
This activity is intended for physicians, pharmacists, pharmacy technicians, nurses, and physician assistants.

Registration Information
Registration is complimentary; therefore refunds are not applicable. For information on how to register to attend this activity, please contact the Activity Coordinator(s) listed above.

Requirements for Receiving CE Credit

All learners claiming credit must attest to their attendance and complete all required activity evaluation(s) in the FDA CE Portal (ceportal.fda.gov) within 14 days after an activity ends. Upon completion, learners may view/print statement of credit.

Attention NABP Pharmacists and Pharmacy Technicians: The FDA CE Team will report your credit to the National Association of Boards of Pharmacy (NABP) provided you add your NABP ID and date of birth to your profile in the FDA CE Portal. The only official Statement of Credit is the one you pull from CPE Monitor®. If you do not see your credit reflected on CPE Monitor®* after 45 days of attestation, please contact FDACETeam@fda.hhs.gov.
*CPE Monitor® sets a strict 60-day limit on uploading credits.

Disclosure

Faculty

• Bell-Vlasov, Andrea, PhD, Science Policy Analyst, Food & Drug Administration - nothing to disclose
• Brodsky, Eric, MD, Associate Director, FDA/CDER/OND/Labeling Policy Team - nothing to disclose
• Bugin, Kevin, PhD, RAC, Deputy Super Office Director of Operations, US FDA - nothing to disclose
• Califf, Robert M., MD, Commissioner, Food and Drug Administration - nothing to disclose
• Donoghue, Martha, MD, Associate Director, Pediatric Oncology and Rare Cancers, Oncology Center of Excellence, FDA - nothing to disclose
• Gray, Tracy, RN, MS, MBA, Patient Engagement Lead, FDA/CDRH - nothing to disclose
• Kraus, Stefanie, JD, MPH, Senior Regulatory Counsel, FDA - nothing to disclose
• Lee, Kerry Jo, MD, Associate Director for Rare Diseases, FDA - nothing to disclose
• Liang, Wei, PhD, Regulatory Operations Staff Chief, FDA - nothing to disclose
• Marston, Hilary, MD, MPH, Chief Medical Officer, Food and Drug Administration - nothing to disclose
• Mercer, Kelly, PhD, Staff Fellow, NCTR - nothing to disclose
• Morales, Jose, MD, Senior Medical Advisor, FDA Office of Good Clinical Practice - nothing to disclose
• Myers, James, JD, Associate Director for Policy, FDA, CBER - nothing to disclose
• Pattee, Suzanne, JD, Regulatory Counsel, FDA/OC/OCPP - nothing to disclose
• Pepe, Salvatore, PharmD, MS, Senior Regulatory Review Officer, FDA/CDER/OTS/IO - nothing to disclose
• Presto, Ryan, PharmD, Regulatory Officer, FDA - nothing to disclose
• Retzky, Sandra, DO, JD, MPH, Director, OC/OCPP/OOPD - nothing to disclose
• Sacks, Leonard, MD, Associate Director for Clinical Methodology, CDER/FDA - nothing to disclose
• Summers, Jeff, MD, Associate Director Translational Sciences OOD, FDA - nothing to disclose
• Tomar, Eli, JD, MPH, Associate Director, Guidance & Legislation, FDA Center for Devices and Radiological Health (CDRH) - nothing to disclose
• Viviano, Charles, MD, PhD, Supervisory Medical Officer, FDA - nothing to disclose

Planning Committee

• Bent, Robyn, BSN, MS, Supervisory General Health Scientist, FDA - nothing to disclose
• Retzky, Sandra, DO, JD, MPH, Director, OC/OCPP/OOPD - nothing to disclose
• Serrano, Michael, PA-C, Investigator, Office of Regulatory Affairs - nothing to disclose
• Vaillancourt, Julienne, MPH, R. Ph, Rare Disease Liaison/Policy Advisor, FDA/CBER/OD - nothing to disclose
• Welsh, Cynthia, MD, Medical Officer, FDA - nothing to disclose

CE Consultation and Accreditation Team

• Bueide, Rachel E., MPhil, Training Specialist, FDA/CDER/OEP/DLOD - nothing to disclose
• Bryant, Traci, M.A.T., Lead Training Specialist, FDA/CDER/OEP/DLOD - nothing to disclose
• Wood, Sara, Accreditation Program Administrator, CECAT, FDA/CDER/OEP/DLOD - nothing to disclose

Requirements for Certificate of Completion (Non CE)
Must attend 80% of the activity.