1. Single-Patient Use of Investigational Drugs and Biologic Products for Treating Cancer Grant Williams, M.D. Medical Team Leader DODP/CDER/FDA

2. Objectives Educate the public Provide advice to FDA

3. Agenda FDA introduction Ethical framework Industry perspective Patient advocate perspective Committee discussion of questions

4. Definitions IND (Investigational New Drug Application) Sponsor Drug manufacturer Investigator

5. Cancer Drug Development Preclinical studies Phase 1: –Few patients –Dose, toxicity Phase 2: –30-100 patients/indication –Anti-tumor activity Phase 3: –Hundreds to thousands of patients –Clinical benefit, comparative findings

6. Investigational Drug Use Clinical investigations Treatment use

7. Treatment Use of Investigational Drugs Single-patient use Expanded access

8. Treatment Use of Investigational Drugs Expanded Access –Well-defined procedures Group C Treatment IND –Other expanded access protocols

9. Treatment Use of Investigational Drugs Single-patient use –Single-patient IND –Single-patient exception

10. Requirements for Single- Patient Treatment Drug supplier and Sponsor Qualified investigator IRB approval and informed consent FDA concurrence

11. Evaluating a Request for Treatment Use Items to consider: –Activity and toxicity of drug –Other treatment options for patient –Is sponsor pursuing drug development? –Will treatment use impede drug development?

12. When is Single-Patient Use Appropriate? Evidence of safety and/or efficacy –Stage of drug development (Phase I, etc.) –Results of studies Standard therapy