AccesstoInvestigationalDrugs.PDFVIP

Access to Investigational Drugs ___________________________ Terry Toigo Richard Klein Office of Special Health Issues Food and Drug Administration Outline ? What is expanded access? ? 1997 FDA Modernization Act ? Expanded access programs – Benefits and Risks ? Implementing the process – Who is responsible for what? ? Questions/Answer 2 For Patients Seeking Treatment… ? FDA-approved drugs should be the first option ? If patients do not respond to, or are intolerant of approved therapy, investigational drugs may be available through two potential pathways designed to help protect patients from unknown potential risks to patients, where we do not know if a drug is effective. – Some may be eligible to receive an investigational drug as a participant in a clinical trial. – Some may pursue an expanded access program (EAP) also known sometimes as “compassionate use.” 3 What is Expanded Access? ? Use of an investigational drug or biologic to treat a patient with a serious disease or condition who does not have comparable or satisfactory alternative therapies to treat the disease or condition. ? Contrast with investigational drug in a clinical trial where the primary intent is research (systematic collection of data with the intent to analyze it to learn about the drug) 4 1997 FDA Modernization Act Amended § 561 of the FDC Act to say an individual patient may obtain an investigational drug for treatment use when: 9 The patient’s physician determines that the patient has no comparable or satisfactory alternative therapy; 9 FDA determines that there is sufficient evidence of safety and effectiveness to support use of the investigational dru