Designation of Letermovir (TBA) as an orphan drug - Merck Sharp & Dohme (Australia)

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, establishes a framework for the regulation of therapeutic goods in Australia, including medicines, medical devices, and blood products. This Act aims to protect public health by ensuring that therapeutic goods are safe, of high quality, and effective. One of the key provisions of the Act is its ability to designate certain drugs as "orphan drugs" to address unmet medical needs for rare diseases or conditions. The designation of Letermovir (TBA) as an orphan drug on 15 March 2017 under the Therapeutic Goods Regulations 1990 exemplifies this objective. By designating Letermovir (TBA) for the prevention of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant, the Therapeutic Goods Administration seeks to encourage the research, development, and availability of treatments for rare and life-threatening conditions. This aligns with the policy objective of the Act to improve access to therapeutic goods that address significant unmet medical needs.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration within the Department of Health, provides the legislative framework for the regulation of therapeutic goods in Australia. Under this Act, specific therapeutic substances can be designated as orphan drugs to encourage the research, development, and availability of treatments for rare diseases or conditions that affect a small number of people. In the context of this legislation, Letermovir (TBA) has been designated as an orphan drug for the prevention of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant (HSCT). This designation applies to the film coated tablet and powder for injection dose forms of Letermovir (TBA), with Merck Sharp & Dohme (Australia) Pty Limited identified as the sponsor of the drug. The geographic reach of this designation is national, applying across the Commonwealth of Australia, and the designation is specific to the therapeutic use outlined. There are no exclusions, exemptions, or thresholds specified in this designation, and the application of the orphan drug designation may be further detailed or extended through subordinate instruments under the Therapeutic Goods Regulations 1990.

Key Provisions

The Therapeutic Goods Act 1989, as amended by the Therapeutic Goods Regulations 1990, designates Letermovir (TBA) as an orphan drug for the prevention of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients of an allogeneic hematopoietic stem cell transplant (HSCT) (Section 16J of the Regulations). This designation, announced on 15 March 2017 by Dr. Anthony Gill, acting as the Delegate of the Secretary, recognises Letermovir (TBA) for its intended use in a rare medical condition, thereby encouraging research and development for treatments that might otherwise not be commercially viable. The drug is available in two forms: film coated tablets and powder for injection, both intended to meet the specific therapeutic needs of the designated patient group. Entities involved, primarily the sponsor Merck Sharp & Dohme (Australia) Pty Limited, must comply with certain obligations under this designation. This includes ensuring that the drug's manufacturing, testing, and distribution adhere to the highest standards of quality and safety as stipulated by the Therapeutic Goods Administration (TGA). The sponsor is also required to provide ongoing data on the drug's efficacy and safety to the TGA, which may include post-market surveillance and reporting of adverse events. Additionally, any changes to the drug's formulation, manufacturing process, or intended use must be reported to and approved by the TGA before implementation. Failure to comply with the obligations and requirements set forth by the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant legal consequences. Violations may be subject to both civil and criminal penalties. For instance, supplying therapeutic goods that do not comply with the regulations can attract civil penalties, with fines up to $222,200 for individuals and $1,111,000 for corporations, depending on the severity and intent of the breach. Criminal penalties may also apply, with individuals potentially facing imprisonment for up to five years and corporations facing penalties of up to $5.5 million. These stringent measures underscore the importance of adherence to the legislative requirements to ensure public health and safety.