Australia Wants Input on Orphan Drugs Program as it Conducts Review

Regulatory NewsRegulatory News | 01 May 2015 |  By 

Regulators in Australia today released a discussion paper intended to solicit comments on its orphan drugs program, in light of changes in the landscape for rare diseases since the program's inception in 1997.

Orphan Drugs Regulation in Australia

Orphan drugs are products intended to treat, diagnose or prevent rare diseases. Because the potential treatment populations for these drugs are small, regulators often offer incentives to encourage the development of treatments for rare conditions.

In Australia, orphan drugs are defined under Regulation 16H of the Therapeutic Goods Regulations 1990, which states:

  1. "A medicine, vaccine or in vivo diagnostic agent is an orphan drug if it complies with this regulation.
  2. It:
    • must be intended to treat, prevent or diagnose a rare disease; or
    • must not be commercially viable to supply to treat, prevent or diagnose another disease or condition."

Rare diseases are defined in the act as "a disease, or condition, likely to affect not more than 2,000 individuals in Australia at any time."

To promote the development of orphan drugs, the fees paid to the Therapeutic Goods Administration (TGA) are waived for designating, evaluating and registering qualified orphan drugs under Regulation 45(12), which states:

  1. The Secretary must waive the following fees:
    • a fee that would have been payable, but for this subregulation, for applying to the Secretary under subregulation 16I(1) to have a medicine designated as an orphan drug;
    • a fee that would have been payable, but for this subregulation, for the Secretary considering the application under regulation 16J;
    • a fee that would have been payable, but for this subregulation, as part of the registration of a designated orphan drug."

Under the current 2014/2015 fee schedule, this can amount to a savings of $221,400 (AUD), or roughly $173,600 (USD).

According to TGA, the agency has waived roughly $35 million (AUD) in fees since 1998, when the country made its first orphan drug designation. In total, the agency has granted orphan drug designation to 287 products, at a pace that has increased from 14 designations per year in the program’s first decade to 27 designations per year between 2008-9 and 2012-13.

Changing Medical Landscape

Since 1997, the landscape for rare diseases has changed dramatically, and TGA has had time to reflect on the successes and challenges met by its orphan drugs program.

Despite the increase in the number of drugs granted orphan designation in recent years, it has become more difficult for a disease to be classified as "rare" in Australia. This is because the current regulations use a fixed amount of patients per year as the threshold for a disease being considered rare. While other countries, including the US, Japan and Singapore, also use a fixed number for disease prevalence, other areas such as the EU, Columbia and Ukraine factor for population fluctuation by setting the prevalence at a percentage of the population.

Additionally, the threshold for orphan designation in Australia is far lower than most other regulators, regardless of whether they use a fixed population or one that accounts for population fluctuation. TGA estimates that its current population threshold works out to approximately 0.009%, compared to 0.05% in the EU and roughly 0.06% in the US.

In the chart below, the figures demonstrate how the threshold for rare disease has changed over time in the US and Australia.

CountryPop. When Orphan Drug Laws EnactedPop. (Most Recent Census)Pop. 2013 World Bank Est.)Orphan Drug Designation (total Pop.)


% of Pop. (Orphan Law, Recent Census, 2013 Est.)


18, 532,200 ( 1997)

21,507,717 ( 2011)



0.0108%, 0.0093%, 0.0086%

United States

234,721,000 ( 1983)

308,745,538 ( 2010)



0.0852%, 0.0648%, 0.0633%

European Union




5 per 10,000


TGA has also found that as treatments for rare diseases become more effective, patients with rare diseases are living longer, leading to an increase in their populations. TGA demonstrates this by looking at population estimates for patients with hemophilia A, which have increased from 1,793 to 1,954 between 2009-10 and 2012-13.

Potential Reforms

In light of the changes in the rare disease landscape, TGA is asking for input from the public on potential reforms to its orphan drugs program.

Some of the questions TGA is asking include how it should apply fees and waivers in the future, and if it should change how it defines rare diseases. The agency is also considering varying the definition of orphan drugs to accommodate for the recent trend of targeting narrow patient subsets or specific disease stages in the indication of a drug.

TGA also acknowledges that the current population threshold for orphan drug status is "considerably lower" than other regulators, and calls for suggestions on how the definition could be altered to account for population growth.

Based on the factors TGA is considering for reform, the agency is presenting four reform packages that encompass the possible combinations of answers to its questions. The agency asks that interested parties submit their input by 15 June 2015.


TGA Discussion Paper


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