Submissions received and TGA response: Orphan drug program

18 April 2017

The TGA would like to thank respondents who provided submissions in response to the October 2016 public consultation paper, Consultation: 2015 consultation outcomes and 2016 orphan drug program proposal.

A total of 39 submissions were received. Of those submissions, 10 were from peak bodies (Consumers, Industry or Healthcare Professionals), 20 were affiliated with the pharmaceutical industry and 9 from individuals.

All submissions that gave permission to be published or referenced on the TGA website are now available below in PDF format.



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  • Consultation submission: Mrs Nicole Tulk

TGA response

Submissions received in response to the public consultation were broadly supportive of the general principles of the proposal with some modifications suggested mainly in relation to aspects of the eligibility criteria and particularly the time to lapsing of the validity of the designation.

Stakeholder feedback has informed the policy position going forward and the upcoming changes subject to changes to the Therapeutic Goods Regulations 1990. Medicines, including vaccines and in vivo diagnostic agents will be eligible for orphan designation if they meet all four orphan criteria:

  1. the orphan condition prevalence or a lack of financial viability; and
  2. the 'seriousness' of the condition; and
  3. the absence of registered therapeutic goods for the condition or significant benefit over these therapeutic goods ; and
  4. the medical plausibility of the indication

Policy changes resulting from the feedback are summarised in the Outcome Summary below.

Further information on process details of the orphan drug program will be outlined in guidance documentation, to be released on the TGA website closer to the 1 July implementation date, subject to regulation amendment.

Outcome summary

Table 1: Summary of key changes resulting from public consultation on the 2016 orphan drug program proposal
Change Current situation Proposed change Benefits
Modification of the orphan prevalence threshold

The threshold is relatively restrictive in international comparison:

  • Static number
  • Not more than 2000 Australians

More generous threshold is more aligned in international comparison:

  • Ratio
  • not more than 5/10,000 (currently 12,000 Australians)
  • More diseases may qualify as orphan
  • Change to a ratio takes population growth into account
Introduction of additional orphan criteria

Eligibility for orphan status is established on the basis of meeting the rare disease prevalence, no additional medical criteria need to be met.

Alternatively, eligibility can be based on a lack of demonstrated commercial viability, where the threshold is exceeded.

Additional medical eligibility criteria, which are:

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

An option to apply on the basis of financial viability is retained

  • Targets the orphan program incentive to the greatest unmet need, where it will have the biggest impact
  • Closer alignment with the orphan criteria applied by the European Medicines Agency
Changes to the designation process

Orphan designations are granted indefinitely.

Orphan status is retained even when the criteria are no longer satisfied since the designation is not reassessed.

Orphan drug status will lapse after 6 months. The validity of the designation can be extended by 6 months, up to a total of 12 months, following written justification in certain circumstances. The program incentive (fee waiver) will be granted on the basis of current information to facilitate designation for eligible medicines.

Transition period

We propose to target the orphan drug program to the greatest unmet need. To allow sufficient time for stakeholders to adjust to this change, we will propose that all existing orphan designations will lapse, following a fixed period after introduction of the new orphan regulations, possibly 12 months. A proposed transition plan will be provided to industry for discussion prior to implementation of the reforms to the orphan drug program.