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Transition plan - Orphan drug designations

26 June 2017

Introduction and overview

The Orphan drug legislation was introduced in 1997. Following almost 20 years of operation a review of the program commenced in 2015. The objective of the review was to create a viable Orphan drug program that is fair and provides an incentive for sponsors to bring drugs for small populations to market that might otherwise not be available.

Major changes include the introduction of a more generous orphan disease prevalence threshold and additional criteria that focus the program to where there is the greatest unmet need. The changes bring a closer alignment with orphan criteria implemented by the European Medicines Agency (EMA).

As part of the reform, the designation process has also been modified. The key change was a move from a previously indefinite Orphan drug designations to a process under which the orphan designation lapses after a specified time. This was introduced to align the lodgement of the related registration application more closely with the date of designation, to encourage early application and to ensure that the application and evaluation fee waiver is based on information that is relatively current.

The new eligibility criteria aim to focus the program on the greatest unmet need. For the success of the proposed reform, it is important to transition all current orphan designations into the new scheme.

Consistent with commonly accepted good regulatory practice, a transition period is proposed for the existing orphan designations which were made at a time when their status would not lapse.

To allow sufficient time for stakeholders to adjust to this change, all existing orphan designations will lapse, following a period of 12 months after introduction of the new orphan regulations.

The transition timeline has been developed to clearly communicate the details of the transition and describes the key milestones and associated implementation dates.

Key points in the timeline

This implementation plan specifically applies to Orphan drug designations. Guidance material is available on the TGA website to assist applicants in compiling and submitting designation applications.

The TGA will monitor and report on the number of designation applications and the designated Orphan drugs will be published on the TGA website. Designation applications will no longer be gazetted under the new regulation.

Implementation plan

Implementation plan

Milestone Description

You are strongly encouraged to notify us of your planned designation application at least one month prior to lodgement. Early notification will assist us with resource planning which contributes to the timely assessment of your designation application and submission for registration.

At this stage, TGA will commence accepting early notifications of the intent to lodge an Orphan drug designation application as part of the implementation arrangements. Early notifications will be particularly important during the first stages of implementation of the new Orphan drug regulations.


The new Orphan drug regulation comes into effect

TGA provides a new designation application e-form

TGA guidance on Orphan drug designations published:

  • All relevant Orphan criteria must be met for the application to be eligible.
  • Decision outcomes on eligible Orphan drug designations will be published on the TGA website.
  • The validity of the Orphan drug designation will lapse six months after the designation is granted (possibility of six month extension) if no related registration application has been lodged.
  • Decisions on pending designation applications lodged prior to the regulation amendments taking effect will be made under the current orphan regulations. Decisions for designation applications lodged on or after the day the regulation amendments take effect will be made under the proposed new regulations. Orphan drug designations granted for applications lodged prior to the Orphan drug regulation amendments taking effect will lapse 12 months from the date the new legislation is effective.
C Applications are expected to be in the correct format and contain all relevant information as indicated in the guidance documents and must be lodged using the new designation application form.
D First Orphan drug designation decisions possible under the new Orphan drug regulation.
E Monitoring of the number of submitted designation applications and decision outcomes and the time from designation application lodgement to decision.
F The impact of changes will be reviewed considering designation application numbers, designation outcomes and stakeholder feedback. Guidance material will be reviewed and updated if required. You may be contacted by TGA in the future to follow up in regard to a previous application as part of monitoring the program.
G The validity of all orphan designations lodged prior to July-August 2017 lapses.
H Medium-term review of the Orphan drug program (to assess the program once designated orphans have had sufficient time to complete the registration process)