Series Description

FDA's Division of Drug Information in the Center for Drug Evaluation and Research (CDER) sponsors a series of educational webinars targeting the needs of health care professionals and students. The webinars cover a broad range of FDA drug regulation and medication safety topics. These focused webinars support FDA's mission of promoting and protecting public health through interaction and education to strengthen current and future partnerships and relationships with clinicians and researchers.

Lecture Description
The development of drugs to treat rare diseases is challenging on multiple levels. This webinar will examine these challenges and FDA programs to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.

References

• Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products Guidance for Industry. U.S. Department of Health and Human Services Food and Drug Administration. December 2019. https://www.fda.gov/media/133660/download.
• Rare Diseases: Common Issues in Drug Development Guidance for Industry. U.S. Department of Health and Human Services Food and Drug Administration. January 2019. https://www.fda.gov/media/119757/download.
• Accelerating Rare disease Cures (ARC) Program. Content updated: 3/08/2023. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/accelerating-rare-disease-cures-arc-program.
• FDA Patient Engagement Opportunities. Content updated: 7/22/2021. https://www.fda.gov/patients/learn-about-fda-patient-engagement/fda-patient-engagement-opportunities.
• New Drug Therapy Approvals 2022. Content updated: 1/10/2023. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022#:~:text=these%20expedited%20programs.-,CDER's%20Novel%20Drug%20Approvals%20of%202022,been%20approved%20in%20the%20U.S.

Series Objectives

• Explain how to utilize FDA's Drug Information, medication safety resources, and regulatory guidances to improve delivery of patient care and optimize outcomes.
• Describe and inform health care providers of recent labeling, policy and regulatory changes which would impact prescribing and medication management to optimize patient care.

Learning Objectives After completion of this activity, the participant will be able to:

• Describe the challenges involved in developing a drug for a rare disease.
• Identify key aspects of the FDA regulatory framework that are relevant to rare disease drug development.
• Summarize the efforts of the FDA Center for Drug Evaluation and Research to accelerate rare disease drug development through innovation and engagement.

Target Audience
This activity is intended for physicians, pharmacists, pharmacy technicians, nurses, Certified Public Health (CPH), and physician assistants.

Requirements for Receiving CE Credit

Physicians, physician assistants, pharmacists, nurses, pharmacist techs, and those claiming non-physician CME: participants must attest to their attendance and complete the final activity evaluation via the CE Portal (ceportal.fda.gov). For multi-day activities, participants must attest to their attendance and complete the faculty evaluation each day. Final activity evaluations must be completed within two weeks after the activity - no exceptions.

Attention Pharmacists and Pharmacy Techs: Failure to provide your correct NABP AND Date of Birth information, in the required format, may result in the loss of credit for this activity. NABP profile number should be the 6-7 digit profile number assigned by the CPE Monitor and your birth date should be in the MMDD format (e.g. 0721) Do not provide your pharmacy license number. Please click the "My Account" tab and then navigate to "Edit Contact Information" to verify that your information is correct.

Important Note regarding completion of evaluations and receiving credit
Attendees have 14 days from the last day of the activity to log in, complete the required evaluation(s) and attest to your attendance to claim credit.Physicians, physician assistants, and nurses may then view/print statement of credit. Pharmacists should log into the CPE monitor 8 weeks after the last session of the activity to obtain their CE credit.

Disclosure

Faculty

• Winiecki, Scott, MD, medical officer, FDA - nothing to disclose

Planning Committee

• Burke, Kara, PharmD, Team Leader/Pharmacist, FDA/CDER/OCOMM/DDI - nothing to disclose
• Cao, Christian, MPAS, PA-C, Cross-Discipline Safety Advisor, FDA/CDER/OSE/OPE - nothing to disclose
• De Fronzo, Kimberly, RPh, MS, MBA, Consumer Safety Officer, FDA/CDER/OCOMM/DDI - nothing to disclose
• Kapoor, Rama, MD, Senior Physician, FDA - nothing to disclose
• Nguyen-Chu, Thanh, PharmD, Pharmacist, FDA/CDER/OCOMM/DDI - nothing to disclose
• Paraoan, Dianne, MPH, BSN, RN, Associate Director for Regulatory Affairs, FDA/ CDER/ OMP - nothing to disclose

CE Consultation and Accreditation Team

• Littlefield, Jr, Kenneth P., Training Specialist, FDA/CDER/OEP/DLOD - nothing to disclose
• Zawalick, Karen, CE Team Leader, FDA/CDER/OEP/DLOD - nothing to disclose

Registration Information
Registration is complimentary; therefore refunds are not applicable.