You are here

Reform of the orphan drug program - End of transition arrangements

7 June 2018

On 1 July 2017 amendments to the Therapeutic Goods Regulations 1990 (the Regulations) came into effect that reformed the Australian orphan drug program, following public consultation in 2015 and 2016. Changes to the program were implemented to create a fairer program that aligns more closely with international criteria without impeding the availability of drugs for rare diseases.

Characteristics of the current orphan drug program

Introduction of new criteria re‑focussed the program to the consideration of greatest unmet need. In particular, the new program provides:

  • A more generous orphan disease prevalence threshold (fewer than 5 in 10,000 individuals in Australia), potentially allowing additional conditions to classify as rare
  • A requirement for the condition to be life-threatening or seriously debilitating
  • A requirement for medical plausibility of the condition (generally a distinct disease or condition, subgrouping generally not appropriate)
  • A requirement that there are no other therapeutic goods that are registered for the treatment, prevention or diagnosis of the same condition or, that if there are, the medicine provides a significant benefit over those other goods in terms of efficacy, safety, or major contribution to patient care
  • Introduction of a new pathway with separate eligibility criteria to seek orphan designation for new dosage form medicines
  • Time limited designation - the validity of the orphan designation lapsing after six months with the possibility of a single six months extension, in certain circumstances. Lapsing ensures that the fee waiver for a related registration application is based on information that is current.

Transition period

A transition period is in place for orphan drug designations which were made prior to the reform of the program, at a time when their status would not lapse. In accordance with regulation 54 of the Regulations, these designations remain in force for a period of 12 months from 1 July 2017 – i.e. until 30 June 2018, to allow sufficient time for stakeholders to adjust to the new program. Specific transition arrangements are also provided in regulation 55 of the Regulations for those applications for orphan drug designation that were pending at the time the amendments which introduced the new program commenced on 1 July 2017.

What this means for sponsors with designation under the new orphan program

Sponsors with an orphan designation made after 1 July 2017 under the new program will not be impacted by the end of the transition arrangements. Please see our guidance on the new orphan drug program for more information.

What this means for sponsors with designation under the previous program

Orphan designations under the previous program will remain published on the TGA website but will no longer be in force after the transition period ends (unless any relate to pending applications as at 1 July 2017). Fees for registration will only be waived for medicines with an orphan designation that is in force. This means that once the transition period ends, medicines with an orphan designation under the previous program will no longer be eligible for a fee waiver for registration fees under regulation 45, unless an orphan designation comes into force under the new program. In summary:

  • Sponsors may re-apply for orphan designation under the new system at any time
  • Sponsors should consider re-applying after considering eligibility under the new program
  • Orphan drug designations granted under the new program will lapse after 6 months, though a single extension of a further 6 months may be requested and approved in certain circumstances
  • There is no requirement for sponsors of expiring designations to re-apply for an orphan designation under the new criteria
  • To be eligible for a fee waiver a related orphan designation must be in force when the relevant fee is payable. The application fee is payable at the time of making an application for registration and the evaluation fee is payable on the day on which the applicant is notified of the amount of the evaluation fee (following preliminary assessment)
  • Only those application types that create a separate and distinct good in accordance with section 16 of the Therapeutic Goods Act 1989 are eligible for an orphan fee waiver (e.g. new chemical entity, extension of indication, major variation).
Key dates
Key Dates  
1 July 2017 to 30 June 2018 Orphan drug designations determined prior to 1 July 2017 continue in force until 30 June 2018.
1 July 2018 Orphan drug designations determined prior to 1 July 2017 will cease to be in force from 1 July 2018.

If you have any questions or need further assistance, please do not hesitate to contact us via email at All inquiries regarding applications to the Pharmaceutical Benefits Advisory Committee should be directed to