Orphan Drug Designation: a pathway to accessible treatment for rare diseases

The term ‘Orphan Drugs’ refers to medicines developed to treat medical conditions, considered to be rare. In Australia, if a medicine is intended to treat a condition which affects fewer than 5 in 10,000 individuals, it may fit within the definitions of an Orphan Drug. With a low number of patients who may be eligible for treatment, it would not be financially viable to bring such products through to market, given the considerable development time and cost incurred, without some form of assistance. Thus, many regulatory agencies around the world, including the Therapeutic Goods Administration (TGA), provide companies the incentives to develop and market products where the target population is small.

The initial process of registering an orphan drug involves the submission seeking an Orphan Drug Designation, which will only be granted for:

  • a previously unregistered medicine,
  • an already registered medicine seeking a new indication that meets the Orphan Drug definitions,
  • new dosage form medicine, or a variation application that meets the significant benefit criteria specified by the TGA.

In order for a drug to be eligible for an orphan designation status, there are specific criteria that must be fulfilled, including, the submission is for only one indication for the medicine, the indication must be for a serious (life-threatening or debilitating) condition, it should not be medically plausible that the medicine could effectively treat, prevent or diagnose the condition in another class of patients that is not  covered by the relevant patient class, and the medicine must provide significant benefit over existing therapies (if any). In addition to this, the drug must also meet one of the following orphan drug prevalence thresholds which includes,

  1. The medicine is intended to treat a condition affecting fewer than 5 in 10,000 individuals in Australia when the application is made,
  2. The medicine would not be likely to be supplied to more than 5 in 10,000 individuals in Australia during each year that it is included in the register, or
  3. It is not likely to be financially viable to market the medicine in Australia unless certain fees were waived in relation to the medicine.

In order to objectively support the prevalence claims, we source relevant data from a variety of sources, including (but not limited to) Orphanet database, scientific publications, and from the Australian Institute of Health and Welfare (AIHW) for Australia specific statistics.

The key to a successful orphan drug designation application is presenting clinically relevant information supporting the efficacy and safety.  These data may include the drug development overview, pharmacology data, as well as clinical safety and efficacy of the product, all of which can be sourced from the existing regulatory dossier. We also seek to complete a thorough literature search covering the background of the rare disease, providing the evaluator with a comprehensive story on the disease and how the drug provides a safe and effective option for Australian patients.

Where a drug has been granted orphan designation status, this designation comes into force for six months, during which time the Sponsor must lodge an effective registration application to TGA. As with any other drug registration, these applications are subject to rigorous evaluation processes, which results in a TGA decision on whether the medicine should be registered on the Australian Register for Therapeutic Goods (ARTG).

Recently, through Accelagen’s ongoing collaboration with Recordati Rare Diseases Australia, Accelagen supported the regulatory approval of LEDAGA (Chlormethine hydrochloride) by TGA. This approval means access to treatment for individuals diagnosed with mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL). Accelagen is continuously working with companies within this field, to obtain approvals in Australia for products intended to treat rare diseases.

To learn more about the Orphan Designation process in Australia, please Contact us by email or phone +61 (0) 3 9114 2270


Anzari Atik, PhD

Regulatory Affairs and Study Start-Up Associate

Accelagen Pty Ltd