Single-Patient Use of Investigational Drugs and Biologic Products for Treating Cancer

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: • 30-100 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

Single-Patient Use of Investigational Drugs and Biologic Products for Treating Cancer