Designation of Drisapersen as an Orphan drug - GlaxoSmithKline

Overview

The Therapeutic Goods Act 1989, enacted to regulate the quality, safety, efficacy, and timely availability of therapeutic goods in Australia, addresses various gaps in the healthcare system by providing a comprehensive legislative framework. The Act was introduced by the Parliament of Australia to ensure that therapeutic goods available in the country meet necessary standards and are appropriately monitored. One of the Act's key policy objectives is to facilitate access to innovative and life-saving treatments, particularly for rare diseases that affect small patient populations. In line with this objective, the designation of Drisapersen as an orphan drug on 10 December 2013 by the Delegate of the Secretary, Dr Anthony Gill, under the Therapeutic Goods Regulations 1990, is an example of the Act's application in addressing specific medical needs, such as the treatment of Duchenne muscular dystrophy in patients with certain mutations.

Scope and Application

The Therapeutic Goods Act 1989 designates Drisapersen as an orphan drug for the treatment of patients with Duchenne muscular dystrophy (DMD) bearing specific mutations that are amenable to exon 51 skipping. This designation is made by Dr Anthony Gill, a delegate of the Secretary, under subregulation 16J(2) of the Therapeutic Goods Regulations 1990, effective 10 December 2013. Orphan drugs are intended for rare diseases, and this designation acknowledges the limited patient population and the need for targeted therapeutic interventions. The application of this Act is confined to the Commonwealth jurisdiction, with the Therapeutic Goods Administration overseeing the approval and regulation of therapeutic goods, including Drisapersen, within Australia. The Act does not specify exclusions or exemptions for orphan drug designation, but it extends its application through subordinate instruments, ensuring comprehensive coverage and regulation of therapeutic goods designated under this category.

Key Provisions

The main operative section of the legislation, subregulation 16J(2) of the Therapeutic Goods Regulations 1990, provides the authority for designating a drug as an orphan drug. In this instance, Drisapersen is designated as an orphan drug for the treatment of patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations suitable for exon 51 skipping therapy. This designation was made on 10 December 2013 by Dr Anthony Gill, the Delegate of the Secretary for the purposes of section 16J of the Therapeutic Goods Regulations. The specified dose form for Drisapersen is a solution for injection, and the sponsor of this drug is GlaxoSmithKline. The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, imposes several obligations on the parties involved in the designation and use of orphan drugs. Primarily, the sponsor, in this case GlaxoSmithKline, must ensure that the drug is manufactured and supplied according to the prescribed standards and regulations. Furthermore, they must provide accurate and complete information about the drug, including its use, safety, and efficacy. The Act also requires that the sponsor applies for and obtains necessary approvals for the drug, including any clinical trials and therapeutic use. Breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in various civil and criminal penalties. For instance, supplying a therapeutic good that does not comply with the standards set by the Act can lead to substantial financial penalties. In the case of corporations, the maximum penalty can be up to $5.5 million for each offence under section 31AB of the Act. Additionally, individuals involved in the manufacture or supply of non-compliant therapeutic goods may face imprisonment, with penalties varying based on the severity and intent of the breach. The Act provides a framework for enforcement actions to ensure compliance and protect public health.