You are here

TGA Internet site archive

The content on this page and other TGA archive pages is provided to assist research and may contain references to activities or policies that have no current application. See the full archive disclaimer.

Reform of the Orphan Drug Program - Transition arrangements

1 May 2017

This letter was emailed to sponsors of medicines that have received Orphan Drug designation since the commencement of the Orphan Drug Program in Australia. If you are a sponsor of an orphan medicine and have not received a letter, please access the electronic copy provided below.

Dear Sir/Madam,

Reform of the Orphan Drug Program - Transition Arrangements

You have been identified as a sponsor of a medicine that has received Orphan Drug designation since the commencement of the Orphan Drug Program in Australia.

The Orphan Drug Program provides an incentive for sponsors to bring medicines for a small population to market that might otherwise not be available in Australia. The current Orphan Drug criteria have not been reviewed in almost 20 years and changes to the Program are required to ensure that it is still fulfilling its intended purpose.

In 2015 and in late 2016, we consulted publicly on options for changes to the Australian Orphan Drug Program. These consultations have resulted in soon-to-be introduced reforms that will create a fairer Program that aligns more closely with international criteria without impeding the availability of drugs for rare diseases.

Summary of the proposed changes

As part of the Orphan Drug Program reform we are proposing to re focus the program to the consideration of greatest unmet need. This requires introduction of additional eligibility criteria, as follows:

  • Life threatening or seriously debilitating condition; and
  • No existing therapeutic goods for prevention diagnosis or treatment, or significant benefit over existing therapeutic goods; and
  • Medical plausibility

To ensure that the fee waiver is granted on the basis of meeting the eligibility criteria, the validity of the designation is proposed to lapse after 6 months, with the option to apply for a 6 month extension. This is different to the current Orphan Drug designation that does not involve the re assessment of eligibility over time.

What this means for you

A transition period is proposed 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, we propose that all existing Orphan Drug designations will lapse 12 months after the introduction of the revised Program.

  • Sponsors with lapsed Orphan Drug designation may re-apply under the revised system
  • Orphan Drug designations granted under the revised system will lapse after 6 months, an extension of a further 6 months may be requested and approved by the TGA

A proposed transition plan has been distributed for targeted industry consultation, including amongst industry peak bodies for broader dissemination to interested parties, with responses due by close of business on Friday, 19 May 2017.

The implementation of the proposed transition plan will involve the following (dates subject to necessary regulations being in place):

Key dates
Date Description
1 July 2017 The new Orphan Drug regulations come into effect.
1 July 2017 to 30 June 2018 The TGA recognises the validity of approved Orphan Drug designation applications that were lodged prior to 1 July 2017.
1 July 2018 All Orphan Drug designations based on an application lodged prior to 1 July 2017 lapse.

If you have any questions about the project or need further assistance, please do not hesitate to contact us via email at

Thank you for contributing to the reform of the Australian Orphan Drug Program to ensure it addresses the greatest unmet need for patients.

Yours sincerely

(Signed Electronically)
Dr Larry Kelly

Acting Deputy Secretary for Health Products Regulation
Department of Health
1 May 2017