Executive Summary

New orphan drug designations under Australia’s reformed program will lapse after six months, although there is a 12-month transitional period for existing designations.

NEW ORPHAN DRUG RULES CAME INTO EFFECT ON JULY 1

Australia’s Therapeutic Goods Administration has released a step-by-step guide on how rare disease drug sponsors can apply for designation under the country’s reformed orphan drug program, which came into effect on July 1. The TGA has also published guidance on eligibility criteria and supporting documentation requirements for orphan designation.

Under legislative amendments to Australia’s 20-year-old orphan drugs program, all applications for orphan designation made after July 1 must address the new eligibility criteria in order to be considered for designation.

There is a transition period to give stakeholders time to adjust to the changes, the TGA said. All existing orphan drug designations will lapse 12 months after the introduction of the revised program, i.e., on July 1, 2018. The new program retains the current incentive of a 100% fee waiver for orphans.

The reforms create “a fairer program that aligns more closely with international criteria without impeding the availability of drugs for rare diseases” – TGA

Commenting on the revised orphan program, the TGA said the reforms created “a fairer program that aligns more closely with international criteria without impeding the availability of drugs for rare diseases.”

The main changes to the program are:

the validity of the orphan designation lapses after six months with the possibility of a six-month extension in certain circumstances to ensure that the fee waiver of a related registration application is based on information that is “reasonably current”;

the orphan disease prevalence threshold is more generous, potentially allowing additional conditions to classify as orphan;

the proposed orphan condition must be seriously debilitating or life threatening;

there are additional criteria aim to bring orphan products to market that treat conditions for which no therapeutic goods are registered, or that can provide significant benefit over registered therapeutic goods;

the orphan indication must be medically plausible (generally a distinct disease or condition) – subgroups would only be considered appropriate where the product would be ineffective in the remaining population; and

a new pathway is introduced, with separate eligibility criteria for seeking orphan designation for new dosage form medicines.