Accessing Unapproved Therapeutic Modalities In The USA And Australia

By Gabriel Kremmidiotis, Chief Scientific Officer and Alex Kavros, Executive Vice President, Scientific & Regulatory Affairs

The United States and Australia have developed regulatory frameworks to provide access to unapproved therapies for patients with serious or life-threatening conditions. In the U.S., the Food and Drug Administration (FDA) oversees the Expanded Access (EA) program, offering three pathways: Individual Patient, Intermediate-Size Population, and Treatment Protocol. The EA process balances patient needs with regulatory safety standards and requires manufacturer consent, which can present challenges such as supply limitations or liability concerns.

In Australia, the Therapeutic Goods Administration (TGA) manages the Special Access Scheme (SAS) and the Authorised Prescriber Scheme (APS). The SAS includes Category A for immediate access in life-threatening cases, Category B for pre-approved access, and Category C for streamlined pathways involving low-risk products. The APS allows healthcare providers to prescribe unapproved therapies for multiple patients but requires endorsement from ethics committees or specialist colleges.

Both systems face challenges, including cost burdens and administrative complexities, which may delay access to critical therapies. Despite these issues, the FDA’s EA pathways and Australia’s SAS and APS frameworks play essential roles in addressing unmet medical needs while balancing patient safety, ethical considerations, and the integrity of clinical research.