Designation of Pomalidomide as an Orphan Drug - Celgene Pty Ltd

Overview

The Designation of Pomalidomide as an Orphan Drug, gazetted on 17 October 2012, was enacted to address the need for specialised treatments for rare diseases, specifically targeting multiple myeloma in patients who have not responded to at least two prior therapies. This legislation, issued under the authority of the Therapeutic Goods Regulations 1990 by Dr Anthony Gill, a Delegate of the Secretary, aims to facilitate the availability and development of treatments for conditions that affect a small number of people in Australia. The sponsor for Pomalidomide, the drug designated for this purpose, is Celgene Pty Ltd. The overarching policy objective is to encourage the development and registration of orphan drugs, which are critical for patients suffering from rare diseases that otherwise might not have viable treatment options.

Scope and Application

The designation of Pomalidomide as an orphan drug under the Therapeutic Goods Regulations 1990 applies to the specific therapeutic use of this drug for the treatment of multiple myeloma in patients who have failed two or more prior therapies. This designation is made for the capsule dose form of Pomalidomide, with the sponsor of the drug being Celgene Pty Ltd. The application of this designation is specifically within the scope of the Commonwealth of Australia, governed by the Therapeutic Goods Regulations 1990, which are administered by the Therapeutic Goods Administration (TGA). The purpose of this designation is to encourage the development of treatments for rare diseases by providing certain incentives, including expedited assessment pathways and potential market exclusivity. This particular legislation does not specify any exclusions or exemptions from its application, and its scope is limited to the therapeutic use of Pomalidomide as outlined. The designation can be extended or refined through subordinate instruments under the Regulations, allowing for adjustments to the scope and conditions of the orphan drug status as necessary.

Key Provisions

The main operative sections of this legislation designate Pomalidomide as an orphan drug under section 16J(2) of the Therapeutic Goods Regulations 1990. Specifically, section 16J(2) allows the Delegate of the Secretary to designate a drug as an orphan drug when it is intended for the treatment of a rare disease or condition, in this case, multiple myeloma in patients who have failed two or more prior therapies. The drug is specified to be in the form of capsules. This designation was made on 17 October 2012 by Dr Anthony Gill, who is acting as the Delegate of the Secretary, and the sponsor of the drug is identified as Celgene Pty Ltd. The Act imposes certain obligations on the parties involved. The Delegate of the Secretary, in this case, Dr Anthony Gill, is required to make the designation based on the criteria outlined in the Regulations. The sponsor of the drug, Celgene Pty Ltd, must ensure that the drug meets the necessary standards for safety and efficacy, and comply with any further requirements that may be imposed under the Regulations. This includes providing any necessary data and information to support the orphan drug designation and any subsequent applications or approvals. There are potential consequences for breach of the obligations under the Act. While the specific offences and penalties are not detailed in this particular piece of legislation, breaches of the Therapeutic Goods Regulations 1990 in general can lead to both civil and criminal penalties. Civil penalties can include fines and the possibility of court-ordered actions to correct non-compliance. Criminal penalties can include fines of up to $222,000 for individuals and $1,110,000 for corporations, as well as imprisonment for up to five years for serious offences. The specific penalties would depend on the nature and severity of the breach, as well as any relevant precedents set by previous cases.