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Consultation: Orphan drug program

2015 consultation outcomes and 2016 orphan drug program proposal

14 October 2016

This consultation closed on 25 November 2016.

Invitation to comment

We sought comments from interested parties on the 2016 TGA Orphan drug program proposal.

Consultation documents

How to access a pdf or Word document


Document released for consultation on Friday 14 October 2016.

Interested parties should respond by close of business on Friday 25 November 2016.

Feedback will be released following consideration of submissions. (see 'What will happen').

About the consultation

We are considering whether the TGA Orphan Drug Program is still fulfilling its intended purpose: to provide an incentive to sponsors to bring medicines for a small population to market, and in doing so make medicines available to patients that otherwise would not be available. An initial public consultation addressing possible reform options for the orphan drug program was conducted in 2015. The proposal for consultation incorporates feedback received in the initial consultation and proposes changes to the Australian orphan drug program.

The purpose of the 2016 consultation is to:

  • present the outcomes of the initial orphan drug consultation conducted in 2015
  • discuss proposed changes to the Australian orphan drug program
  • seek feedback regarding specific aspects of the proposed program, which will be used to inform changes to the Therapeutic Goods Regulations 1990 which are required to implement the changes to the Australian orphan drug program.


When the TGA Orphan Drug Program was established it was generally recognised that support was required to bring these products to market due to low demand and the lack of financial incentive to develop or market a medicine for such a small patient population. At the time, this support was in the form of waiving fees for evaluation and registration.

Since the inception of the TGA Orphan Drug Program there have been developments in the definition and recognition of 'rare diseases'. These have included improvements in technology to diagnose and identify rare diseases and subtypes of diseases, and in the ability to tailor therapies for these diseases and disease subtypes. Consequently there has been a significant increase in the number of applications for orphan drug designation and applications for market authorisation for such drugs.

While the priority of ensuring availability of treatments for rare diseases remains, it is timely to consider if the TGA Orphan Drug Program in its current form, with the shifting market focus, is achieving this in the most effective way possible.

Content of submissions

Submissions may address consultation items 1 to 6 which are included in the proposal (full details on page 4/5/ of the consultation paper).

Please specify:

  • whether or not you support the proposed changes to the TGA orphan program. If you do not support the change/s, you may make suggestions for an alternative
  • an assessment of how the proposed change will impact on you, and what you see as the likely benefits or costs to you (financial or non-financial)
  • if possible, please attempt to quantify these costs and benefits.


Any questions relating to submissions should be directed to the the Business Review and Reporting section by email to

What will happen

All submissions will be placed on this website unless marked confidential or indicated otherwise in the submission form (see Privacy information).

We will review submissions and provide feedback through this website.

Privacy information

  • We collect your personal information in this submission in order to:
    • contact you if the TGA wants to seek clarification of issues raised in your submission or to check whether you consent to certain information that you have provided being made publicly available.
    • help provide context about your submission (e.g. to determine whether you are an individual or a director of a company or representing an interest group).
    • seek feedback about how the consultation was undertaken.
  • Please do not include personal information about other individuals in the body of your submission. Personal information in this context means information or an opinion about an individual whose identity is apparent, or can reasonably be ascertained, from the information or opinion.
  • More information on consultations and privacy is included in the submission form and on our website.