Section outline

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  • Wednesday, April 9, 2014; 10:30 AM - 12:00 PM

    Discussion Seminar: Introduction
    History and phases of drug development, role of regulatory agencies; ethics of drug development; program design; intellectual property.

    Faculty:  Richard Chin

    Location:     China Basin 6702

    • Session Slides:

    • DrugDev Lecture1-1 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading/Viewing:

    • Guidance documents URL
    • FDA guidance documents URL

    • Resources:

    • Zepeda EPI260 Case Study File
    • Dronaderone Case Study File
    • Epi260 - Case Study knishimura File

    • Assignment: Read a guidance document from FDA or EMA on an indication of your choice and write up your thoughts on the indication, the endpoints, magnitude of clinical benefit that the agency expects, and what you see as strengths and weaknesses of the document.

       

    • Assignment Due Date:  Due April 16: Students should email their assignments to Janel Long-Boyle at long-boylej@pharmacy.ucsf.edu and bring a copy to this lecture.

       

  • Wednesday, April 16, 2014; 10:30 AM - 12:00 PM

    Discussion Seminar: How to Assess an Early Stage Drug Candidate and Drug Development Lessons
    Characteristics of a good drug; target and validation: assessment; biology and animal models; chemical characteristics; pharmacokinetics; regulatory path; case studies. Case studies on drug development including: CAST; FIAU; TGN-1412; gene therapy.

    Faculty:  Menghis Bairu, MD

    Location:     China Basin 6702

    • Session Slides:

    • Lecture 2-1 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading/Viewing:

    • Drugs at the FDA URL

    • Assignment: Homework Assignment due April 23: Pick a drug that has an FDA review posted, and read the clinical review, the approval letter, and the package insert. Based on this, and knowledge of the indication, please write a target product profile. Students should email their assignments to Janel Long-Boyle and bring a copy to this lecture.

    • CaseStudyAssign 2012 File

  • Wednesday, April 23, 2014; 10:30 AM - 12:00 PM

    No Attendance required: What you need to file and IND/IMPD

    The preclinical studies, information and the process that are required to get approval to carry out clinical studies in humans.

    Faculty:  Richard Chin

    Location:     China Basin 6702

    • Session Slides:

    • Epidemiology 260 Spring 2012 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading/Viewing:

    • Principles and Practice of Clinical Trial Medicine (2008), Chapter 2, pages 31-37.

    • FDA website info on the IND Application URL

  • Wednesday, April 30, 2014; 10:30 AM - 12:00 PM

    Discussion Seminar: Phase I Trials: dosing and design
    Basic principles of Phase I designs; definition and assessments of Phase I endpoints; conventional and novel dose escalation models; design of pharmacokinetic and pharmacodynamic correlative studies.

    Faculty:  Les Benet

    Location:     China Basin 6702

  • Wednesday, May 7, 2014; 10:30 AM - 12:00 PM

    Discussion Seminar: Late phase clinical trials
    Good Clinical Practice; protocol design and writing; case report forms; investigator brochure; regulatory filings; budget; staff training; data management; safety reporting; site closeout; data analysis; report writing; record retention; regulatory requirements for approval; NDA/BLA and pre-NDA meeting.

    Faculty: Richard Chin

    Location:     China Basin 6702

    • Session Slides:

    • MB4 25UCSF Lecture 6(FINAL) File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Assignment: Go to http://www.ich.org/products/guidelines.html and download two of the documents, one of which should be E6, and another of your choice. You should read them, and discuss how the guidelines might 1) protect patient safety and 2) protect data integrity. Homework assignment due: May 15, 2014.

    • Assignment Due Date:
      Case Study Assignment, Part I: Due May 5: Email assignment to Dr. Chin and cc: Janel Long-Boyle by Monday, May 5.

      May 7: Bring a copy of your current draft case study.

      Be prepared to present your case study in a small group.

  • Wednesday, May 14, 2014; 10:30 AM - 12:00 PM

    No Attendance Required: Vaccines
    What are vaccines and how does their development and use in practice differ from therapeutics. Vaccine safety and public perception; chance association vs. cause and effect. Importance of other governmental (e.g. CDC) and multinational (e.g. WHO) organizations. Evolution of antigen discovery and 'smart' adjuvants. Illustrative case studies including vaccines against (a) meningococcus ACWY, (b) meningococcal B, (d) influenza and pandemic influenza, (e) group B strep. Commercial considerations.

    Faculty:  Richard Chin

    Location:      China Basin 6702

    • Session Slides:

    • Scharschmidt Vaccines - Part 1 - 2012 File
    • Scharschmidt Vaccines - Part 2 - 2012 File
    • Scharschmidt Vaccines - Part 3 File
    • Scharschmidt Vaccines - Part 4 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading:

    • Guidance for Industry: General Principles for the Development of Vaccines to Protect Against Global Infectious Diseases URL
    • Margarit, I. Preventing bacterial infections with pilus-based vaccines: the group B streptococcus paradigm . J Infect Dis. 2009 Jan 1;199(1):108-15. File
      Not available unless: Your ID number contains 02
    • Reverse vaccinology: developing vaccines in the era of genomics. Sette A, Rappuoli R. Immunity. 2010 Oct 29;33(4):530-41. Review. PMID: 21029963 File
      Not available unless: Your ID number contains 02

    • Assignment:

      The assignment for the Vaccine lecture is to download a freedom of information summary from the FDA - http://www.fda.gov/BiologicsBloodVaccines/ucm133705.htm and comment on the development program.

    • Assignment Due Date:  May 21, 2014

  • Wednesday, May 21, 2014; 10:30 AM - 12:00 PM

    Discussion Sessions: Cell, tissue and gene therapies
    History and regulatory framework for cell, tissue, and gene therapies. How these products are different from drugs and conventional biologics. Challenges for CMC development (donor sources, biologic variability, stability, assays), preclinical animal studies, and clinical trials. For gene therapies: gene transfer methods, special safety considerations for preclinical studies and clinical trials. Product examples including stem/progenitor cells, gene-modified cells, and cell/device combinations.

    Faculty:  Richard Chin

    Location:     China Basin 6702

    • Session Slides:

    • Epi 260 Read lecture 1 050912-1 File
    • Epi 260 Read lecture 2 050912 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading:

    • Carpenter MK, Frey-Vasconcells J, Rao MS. Developing safe therapies from human pluripotent stem cells File
      Not available unless: Your ID number contains 02

    • Optional Reading:

    • Dunbar CE, Larochelle A File
      Not available unless: Your ID number contains 02

    • Assignment:

      The assignment for gene therapy is to read the S-1 for Bluebird Bio - http://www.nasdaq.com/markets/ipos/filing.ashx?filingid=8882306  and comment on one or more of the following topics:

      1. How can Bluebird price its therapy?
      2. What are the issues Bluebird might face regarding patents
      3. Why did it take so long for Bluebird to get to where it is today?

       

    • Assignment Due Date:  May 28, 2014

  • Wednesday, May 28, 2014; 10:30 AM - 12:00 PM

    No Attendance Required: Devices and drug/devices combinations
    Medical device development is as varied as the thousands of different types of devices, ranging from diagnostics, to implants, to the equipment used to deliver care. Medical device regulation is risk based and relies as much on design principles and performance standards as on clinical trials. The device life cycle is more likely to be iterative, with long duration of use, and safety and efficacy is often closely linked to training and skilled users.

    Faculty:  Richard Chin

    Location:      China Basin 6702

    • Session Slides:

    • UCSF Device Regulation 2012 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading:

    • Maisel, W File
      Not available unless: Your ID number contains 02

  • Wednesday, June 4, 2014; 10:30 AM - 12:00 PM

    Lecture: Discussion Seminar: Use of biomarkers in clinical development
    The use of diagnostic biomarkers and surrogate biomarkers in drug development will be covered. Review of case studies of how these different types of biomarkers have been utilized in drug development.

    Faculty: June Lee

    Location:     China Basin 6702

    • Session Slides:

    • Biomarker Lecture 2012 Final File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading:

    • Buyse M, Michiels S, Sargent DJ, Grothey A, Matheson A, de Gramont A. Integrating biomarkers in clinical trials. Expert Rev Mol Diagn. 2011 Mar;11(2):171-82. File
    • Frank R, Hargreaves R. Clinical biomarkers in drug discovery and development File
      Not available unless: Your ID number contains 02

    • Assignment:  Case reports are due on June 4th.  Please email a copy of your case report to Janel Long-Boyle before coming to the class discussion.