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Consultation: Orphan drugs program

Discussion paper

28 July 2015

This consultation closed on 15 June 2015

Invitation to comment

The TGA sought comments from interested parties on the Orphan drugs program discussion paper.

Consultation documents

How to access a pdf or Word document


Document released for consultation on 1 May 2015.

Interested parties responded by close of business 15 June 2015.

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

About the consultation

The TGA is considering whether the Orphan Drugs 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.

The purpose of this paper is to seek your input on this issue and, in particular, on four potential options for program reform if changes are required to ensure the program objectives continue to be met.


When the Orphan Drugs 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 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 Orphan Drugs Program in its current form, with the shifting market focus, is achieving this in the most effective way possible.

This discussion paper was first released in January 2015 to a target group consisting of patient advocate groups, industry groups and sponsors; this initial consultation resulted in a few minor amendments to the paper. The submissions received from the limited consultation will be placed on the TGA Internet site at the end of this full public consultation subject to privacy requests.

Content of submissions

Submissions may address any part or all of the Orphan drugs program discussion paper or other identified issues.

What will happen

All submissions will be placed on the TGA Internet site unless marked confidential or indicated otherwise in the submission cover sheet (see Privacy information).

Submissions will be reviewed by the TGA and feedback on submissions will be provided through the TGA Internet site.

Privacy information

  • The TGA collects 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).
  • The TGA will disclose your name and (if applicable) your designation/work title on the TGA Internet site (i.e. make this information publicly available) if you consent to the publication of your name on the TGA Internet site (please complete the cover sheet).
  • Any text within the body of your submission that you want to remain confidential should be clearly marked 'IN CONFIDENCE'.
  • 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.


Any questions relating to submissions should be directed by email to