Clinical trials are studies of drugs and biologics that are still in development and have not yet been approved by the FDA. Many patients enroll in clinical trials to gain access to investigational therapies and contribute to finding out how well an investigational therapy works, and how safe it is for patients. Obtaining a drug or biologic under an expanded access program may be an option for some patients who are not able to enroll in clinical trials. The FDA has allowed expanded access to experimental drugs and biologics since the 1970s. That access has allowed tens of thousands of patients with HIV/AIDS, cancer, and other conditions to receive promising therapies when no approved alternative is available.
The Final Rule, “Expanded Access to Investigational Drugs for Treatment Use,” makes investigational drugs more widely available to patients by clarifying procedures and standards. The Final Rule, “Charging for Investigational Drugs Under an Investigational New Drug Application,” clarifies the specific circumstances and the types of costs for which a manufacturer can charge patients for an investigational drug when used as part of a clinical trial or when used outside the scope of a clinical trial.
Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs Access to Investigational Drugs
For Patients and Patient Advocates
FDA Issues Final Rules to Help Patients Gain Access to Investigational Drugs[ARCHIVED] FDA Forms FDAAA Certification to Accompany Drug, Biological Product, and Device Applications or Submissions