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

Objectives Educate the public Provide advice to FDA

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

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

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

Investigational Drug Use Clinical investigations Treatment use

Treatment Use of Investigational Drugs Single-patient use Expanded access

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

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

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

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?

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