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Orphan drug program reforms

26 June 2017

The objective of the orphan drug program is to provide an incentive to sponsors to bring medicines for a small population to market and make medicines available to patients who would not otherwise be able to access them.

In 2015 and late 2016, we consulted publicly on proposed changes to the orphan drug program. At this time, the orphan drug eligibility criteria had not been reviewed in almost 20 years and changes to the Program were proposed to ensure that it is still fulfilling its intended purpose.

These consultations have resulted in reforms that will create a fairer program that aligns more closely with international criteria without impeding the availability of drugs for rare diseases. Please see the submissions received and TGA response for further information.

Changes to the orphan drugs program

In line with recent amendments to the Therapeutic Goods Regulations 1990 from 1 July 2017, the main changes to the orphan drugs program are:

  • retaining the current incentive of a 100% fee waiver;
  • a more generous orphan disease prevalence threshold, potentially allowing additional conditions to classify as orphan;
  • requiring the proposed orphan condition to be seriously debilitating or life threatening;
  • additional criteria that 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;
  • requiring the orphan indication to 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;
  • introduction of a new pathway with separate eligibility criteria to seek orphan designation for new dosage form medicines; and
  • the validity of the orphan designation lapsing after six months with possibility of six months extension in certain circumstances to ensure that the fee waiver of a related registration application is based on information that is reasonably current.

Please see our guidance on the eligibility criteria for Orphan drug designation for more information.

Applications for Orphan drug designation

Amendments to the Therapeutic Goods Regulations 1990 came into effect on 1 July 2017. All applications for orphan drug designation made after 1 July 2017 must address the new orphan drugs eligibility criteria to be eligible for designation.

A transition period will be in effect for the existing Orphan Drug designations which were made prior to the reform of the program, at a time when their status would not lapse. To allow sufficient time for stakeholders to adjust to this change, all existing orphan drug designations will lapse 12 months after the introduction of the revised program, on 1 July 2018.

For more information on transition arrangements and applying for orphan drug designation using the new program, see the transition plan.

Updated guidance for sponsors on the Orphan drugs designation process is now available on our website.

Please continue to check our website for updates.