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

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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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Objectives

• Educate the public
• Provide advice to FDA

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Agenda

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

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Definitions

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

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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

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Investigational Drug Use

• Clinical investigations
• Treatment use

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Treatment Use of Investigational Drugs

• Single-patient use
• Expanded access

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Treatment Use of Investigational Drugs

• Expanded Access
• Well-defined procedures
• Group C
• Treatment IND
• Other expanded access protocols

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Treatment Use of Investigational Drugs

• Single-patient use
• Single-patient IND
• Single-patient exception

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Requirements for Single-Patient Treatment

• Drug supplier and Sponsor
• Qualified investigator
• IRB approval and informed consent
• FDA concurrence

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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?

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When is Single-Patient Use Appropriate?

• Evidence of safety and/or efficacy
• Stage of drug development (Phase I, etc.)
• Results of studies
• Standard therapy