Designation of Inotuzumab Ozogamicin (Besponsa) as an orphan drug - Pfizer Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, serves to regulate the quality, efficacy, and safety of therapeutic goods, including medicines and medical devices. In an effort to address the specific challenges and limited treatment options for rare and life-threatening conditions, the Act was amended to include provisions for the designation of orphan drugs. This was achieved through the Therapeutic Goods Regulations 1990, which allow for the identification of drugs intended to treat rare diseases or conditions affecting a small population, thereby incentivising the research and development of treatments for these underserved medical needs. The policy objective of this regulatory framework is to ensure that patients with rare conditions have access to potentially life-saving therapeutic innovations, as evidenced by the designation of Inotuzumab Ozogamicin (BESPONSA) for the treatment of B-cell acute lymphoblastic leukaemia by the Therapeutic Goods Administration on 19 December 2016.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA) under the Commonwealth of Australia, includes a provision for designating certain therapeutic goods as orphan drugs. This designation applies specifically to therapeutic goods intended for the treatment, prevention, or diagnosis of rare conditions. In the context of the provided gazette, the Act applies to Inotuzumab ozogamicin (besponsa), a drug designated as an orphan drug for the treatment of B-cell acute lymphoblastic leukaemia (ALL). This designation is intended to encourage the development of drugs for rare diseases that otherwise might not be commercially viable. The application of the Act in this instance is geographically comprehensive, covering the entire Commonwealth of Australia, and applies to Pfizer Australia Pty Ltd as the sponsor of the designated drug. The scope of the Act allows for the extension and restriction of its application through subordinate instruments, although the specific details of such extensions or restrictions are not elaborated in this gazette.

Key Provisions

In the document, section 16J of the Therapeutic Goods Regulations 1990 is invoked to designate Inotuzumab ozogamicin (besponsa) as an orphan drug, specifically for the treatment of B-cell acute lymphoblastic leukaemia (ALL). This designation, which took effect on 19 December 2016, is intended to encourage the research, development and availability of treatments for rare diseases. The drug, Inotuzumab ozogamicin (besponsa), is specified in its powder for injection form for this indication. The sponsor of this drug is identified as Pfizer Australia Pty Ltd. The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, imposes certain obligations and requirements on the entities it governs. For instance, the sponsor, in this case Pfizer Australia Pty Ltd, must ensure that the orphan drug designation is used appropriately and only for the specified indication. Additionally, the sponsor must comply with all relevant regulatory requirements, including those related to the marketing and distribution of the drug. Furthermore, the sponsor is expected to provide sufficient information and data to support the safety and efficacy of Inotuzumab ozogamicin (besponsa) for the treatment of B-cell acute lymphoblastic leukaemia. The legislation also outlines potential consequences for non-compliance. Breaches of the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990 can lead to both civil and criminal penalties. The exact penalties depend on the nature and severity of the breach. For example, knowingly supplying therapeutic goods that do not comply with the standards set out in the regulations can result in substantial fines and, in serious cases, imprisonment. The maximum penalties for such offences can include fines of up to $2.1 million for corporations and $420,000 for individuals, along with potential imprisonment terms. These stringent penalties underscore the importance of adhering to the regulations and highlight the regulatory authority's commitment to ensuring the safety and efficacy of therapeutic goods in Australia.