Designation of Obeticholic Acid as an orphan drug - AU Pharma Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, was established to regulate therapeutic goods in Australia, ensuring they meet safety, quality, and efficacy standards. A specific problem the legislation aimed to address is the need for targeted support and incentives for the development of treatments for rare diseases, which often do not attract sufficient commercial interest due to their limited patient populations. The designation of obeticholic acid as an orphan drug under this Act, announced by Dr Anthony Gill, a Delegate of the Secretary, on 21 April 2017, exemplifies the policy objective of facilitating the development and availability of treatments for rare conditions like primary biliary cholangitis. This action underscores the commitment to enhancing access to necessary therapies for patients with orphan diseases, aligning with broader public health goals of improving health outcomes for all Australians.

Scope and Application

The Therapeutic Goods Act 1989, as amended and applied through the Therapeutic Goods Regulations 1990, pertains to the regulation and administration of therapeutic goods in Australia, encompassing a broad range of products including medicines, medical devices, and blood products. This particular designation of obeticholic acid as an orphan drug under the Act is specific to its application in the treatment of primary biliary cholangitis (PBC) in certain patient groups. The Act applies to the sponsor of the drug, in this case, AU Pharma Pty Ltd, and its provisions are enforced by the Therapeutic Goods Administration. The geographic reach of the Act is national, applying across the Commonwealth of Australia. The designation as an orphan drug under this legislation recognises the drug's use in treating a rare condition, PBC, and provides incentives such as priority assessment and potential benefits in the registration process. Exclusions or exemptions from the Act are not explicitly mentioned in this context, but it is understood that the designation process is stringent, ensuring the therapeutic benefits and safety of the drug for the intended patient population. Subordinate instruments may further refine the application of the Act and its regulations, extending or restricting the scope as needed.

Key Provisions

The primary operative sections of the Therapeutic Goods Act 1989, as evidenced by the designation of obeticholic acid as an orphan drug, involve the authority and process by which certain drugs are designated under specific conditions. Section 16J of the Therapeutic Goods Regulations 1990 provides the legal basis for the designation of orphan drugs, with Section 16J(2) specifically enabling the delegate of the Secretary to make such a designation. In this instance, Dr. Anthony Gill, as the Delegate of the Secretary, has exercised this power by designating obeticholic acid for the treatment of primary biliary cholangitis (PBC) in particular scenarios (Section 16J). This designation is critical as it formalises the drug's status under the orphan drug framework, facilitating specific regulatory benefits and incentives aimed at encouraging the development of treatments for rare diseases. Under the Act, the designation of an orphan drug imposes several obligations on the parties involved. The sponsor, in this case, AU Pharma Pty Ltd, must ensure that the drug is developed and marketed in a way that aligns with the specified therapeutic indications. The sponsor is also responsible for maintaining compliance with all relevant regulatory requirements, including the submission of necessary data and information to the Therapeutic Goods Administration (TGA). Furthermore, the sponsor must ensure that the drug is manufactured to meet quality standards and that all promotional materials accurately reflect the approved indications and usage conditions. Failure to comply with the provisions of the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990 can lead to various civil and criminal consequences. Offences under the Act can result in significant penalties. For example, knowingly supplying a therapeutic good that does not comply with the regulations can lead to substantial fines, with the maximum penalty often reaching up to several thousand Australian dollars for corporations and even higher for individuals depending on the severity and intent of the breach. Additionally, persistent non-compliance can result in court-ordered injunctions or the suspension of the therapeutic good's registration, severely impacting the sponsor’s ability to market the drug. In cases of serious misconduct, criminal charges may be pursued, leading to further penalties including imprisonment for individuals involved in the breach.