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

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Presentation on theme: "Single-Patient Use of Investigational Drugs and Biologic Products for Treating Cancer Grant Williams, M.D. Medical Team Leader DODP/CDER/FDA."— Presentation transcript:

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: – 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


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