Case Studies and Future Trends
Full course description
The Georgia Clinical & Translational Science Alliance- Georgia CTSA and Southern California Clinical and Translational Science Institute -SC-CTSI have collaborated to provide free, high quality educational programs for clinical research professionals at novice to expert levels of experience. At the completion of each course or program, participants earn contact hours recognized by a certificate and/or badge.
Current trends in drug development and various regulatory approval pathways will be discussed.The different approaches in drug development and regulatory approval pathways are highlighted by two case studies.
- Promising Trend: Growing number of drugs being approved
- Forty-six (46) new drugs in 2017 approved by CDER
- Scientific advances pushing this trend forward- Human Genome Technology
- Not all drugs follow the same pathway:
- Regulatory incentives
- Fast track
- Breakthrough therapy
- Accelerated approval (conditional approval process with surrogate endpoint and after being introduced intro market, needs to show benefits)
- Priority review (10 months to six months)
- Drugs can qualify for one or more expedited pathway
- Study sizes vary (orphan drug vs. non-orphan)
- Case study: Bevyxxa
- Action Package Checklist available online and includes what approval tracks requested can be found in that checklist
- One large randomized trial
- Many studies were involved in the overall development
- IND in 2005, End of Phase II in 2011 and approved in June 2017 (12 years total)
- Case study: Brineura
- Action Package Checklist shows it took advantage of the priority designation, orphan drug designation, and breakthrough therapy designation
- Much smaller program in comparison to Bevyxxa (23 patients)
- Single-arm, non-randomized, open-label study
- Control arm: historical control based on registry data
- Stabilized at the 300 doses
- IND in July 2014 and approved in April 2017 (3 years total)
Eunjoo Pacifici, PharmD, PhD, is the Chair and Associate Professor of Regulatory and Quality Sciences and Associate Director of the International Center for Regulatory Science. Dr. Pacifici received a BS in Biochemistry from the University of California Los Angeles followed by a PharmD and PhD in Toxicology from the University of Southern California. She conducted her graduate research in the laboratory of Dr. Alex Sevanian in the Institute for Toxicology where she studied the mechanism of oxidative damage and repair in endothelial cell membrane. Before returning to USC as faculty, Dr. Pacifici worked at Amgen and gained experience in conducting clinical research with a special focus on the Asia Pacific and Latin America regions. She initially worked in the clinical development group managing U.S. investigational sites and central laboratories and then went on to work in the Asia Pacific / Latin America group interfacing with local clinical and regulatory staff in Japan, the People’s Republic of China, Taiwan, and Mexico. She represented regional clinical and regulatory views on therapeutic product development teams and led satellite task forces in order to align local efforts with U.S. activities. Her additional professional experiences include community pharmacy practice in various settings and clinical pharmacy practice at the Hospital of the Good Samaritan in Los Angeles. Her current focus is on developing the next generation of regulatory scientists and pharmacy professionals with the knowledge, tools, and skills to expedite the development of innovative, safe, and effective biomedical products. firstname.lastname@example.org
Learner Level: Beginner
This symposium is designed for Clinical Research Assistant/Associates or Clinical Research Coordinators in academia, clinics, hospitals, industry, or CRO with 1- 2 years of clinical research experience.Individuals may be new to clinical research with limited knowledge of clinical trial conduct, good clinical practices, regulations and common terminology associated with clinical research.
Emory Nursing Professional Development Center (ENPDC) is accredited as a provider of nursing continuing professional development by the American Credentialing Center’s Commission on Accreditation. Attendees to this CNE activity will be awarded 0.5 contact hours by ENPDC. No conflict of interest has been found with the speaker for this CNE activity nor with the members of the planning committee.
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Program Information: This is the fifth course in a five (5)-course program. To complete the entire program and earn a badge CLICK HERE.