Orphan drug designation

A step-by-step guide for prescription medicines

26 June 2017

This guidance assists sponsors with the process of applying for an Orphan drug designation of a prescription medicine for registration on the Australian Register of Therapeutic Goods (ARTG).

To assist you understanding the eligibility criteria and supporting documentation requirements for Orphan drug designation, please see the guidance on Orphan drug eligibility criteria and supporting documentation.

Orphan drug designation

Purpose of designation

We will assess your application for designation before you submit an application for registration to determine whether the medicine is eligible for orphan drug designation.

Designation is a formal decision to accept an orphan drug designation application under regulation 16J of the Therapeutic Goods Regulations 1990.

The designation application is the formal application made using a specified form requesting assessment against the relevant eligibility criteria and a decision from TGA.

Granting of an orphan drug designation does not guarantee successful registration on the Australian Register of Therapeutic Goods (ARTG).

You can lodge an application for a designated orphan drug through any available registration pathway, subject to meeting the requirements for that pathway.

Benefits of designation

A valid designation must be held in order for benefits resulting from designation to apply.

Orphan drug designation of a medicine can:

  • facilitate orphan drug access to the Australian marketplace
  • help offset orphan drug development costs
  • provide consistency and transparency to the orphan drug program.

Medicines with orphan drug designation have the same evidence requirements as other prescription medicines and will be evaluated by TGA for quality, safety and efficacy in the same way.

Eligibility requirements

Specific eligibility criteria for the orphan drug program are set out in Section 16J of the Therapeutic Goods Regulations 1990. See the guidance on orphan drug eligibility criteria and supporting documentation for details of the eligibility criteria and associated definitions.

Transition period

As part of the implementation of the revised orphan drug program, we have developed transition arrangements that will apply for one year from the effective date of the new regulation.

Designation process for orphan drugs

See the Process for Orphan drug designation diagram for an overview of the designation process.

Step 1: Arranging a pre-submission meeting

Early notification

If you have not previously contacted TGA about your intended designation application through a pre-submission meeting, you are encouraged to notify us 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.

You can make this notification informally by sending an email to AET.Application.Entry.Team@health.gov.au, including details of the planned:

  • designation/s to be sought
  • the active ingredient
  • the sponsor's name
  • the proposed indication for designation.

Pre-submission meetings

In place of early notification, you may request a pre-submission meeting with TGA to discuss a planned orphan drug designation application and subsequent submission for registration. The content and procedures for these meetings are as per the standard prescription medicines pre-submission meetings process.

Pre-submission meetings can be a useful opportunity for you to obtain advice from us about the intent of the eligibility criteria for orphan drug designation. However, TGA cannot provide binding advice at a pre submission meeting on whether your application will be granted orphan drug designation.

We recommend that pre-submission meetings occur three to six months before the date you plan to lodge your submission for registration.

Details of any pre-submission discussions should be provided in the Pre submission Planning Form (PPF) and the dossier (Module 1.7.1), as per the usual process.

If the pre-submission meeting is greater than six months from the planned date of the submission for registration lodgement, then we suggest that an email is also sent to the Application Entry Team, as per the 'early notification' section.

Step 2: Verifying your access to TGA Business Services (TBS)

If you already have a client ID number and password to access TGA Business Services (TBS) as a Submitter, go to Step 3.

Client identification and access to TGA Business Services

Designation applications are created and lodged through TGA Business Services portal.

You will need both of the following to make an application:

  • a TGA client ID number
  • password access to our TGA Business Services portal
  • Submitter access to the TBS portal

If you do not have a client ID number or access to our Business Services:

Step 3: Submitting your designation application

We recommend that you submit your application for designation at least three months before you plan to lodge your submission for registration.

Using the designation application e-form

Under section 16H of the Therapeutic Goods Regulations 1990, the designation application must be submitted using the appropriate form (the designation e-form) in order for TGA to make a designation decision.

You can access the Prescription Medicines - Designation Application e-form through your TBS account. See the designation e-form guidance for further guidance on completing the form.

Please see the eligibility criteria and supporting documentation guidance for information on meeting the eligibility criteria and the requirements for supporting documentation to be attached to your designation application.

For any attachments, use the format outlined in Section 2.2 (Formatting for documents) of the general dossier requirements for prescription medicines.

Payment of the designation fee

There is no designation fee for Orphan drug designation applications. If you are applying for Orphan drug and Priority review designation simultaneously, see the Priority review designation guidance for information on the designation fee rules that apply.

Step 4: TGA assessment of the designation application

TGA will assess your designation application and make a decision as quickly as possible.

After you have submitted your designation application using the e-form, we will check the information provided and confirm whether it is sufficient to allow us to assess the application.

We will check that your application:

  • form has been completed in full, including declarations
  • meets the administrative requirements
  • has separately addressed each criterion of the relevant designation/s that have been requested with clear, robust and scientific justifications
  • contains justifications that are up to date
  • includes supporting data and references where required.

You will not be provided with a formal notification of the results of this checking process, however, if required you may receive requests for further information.

If you have any questions during the designation application assessment period, please contact AET.Application.Entry.Team@health.gov.au.

How we will assess your designation application

The focus of our assessment is to determine whether your application and supporting documentation meet the eligibility criteria.

Applications for designation are reviewed against the relevant criteria set out in regulation 16J of the Therapeutic Goods Regulations 1990, depending on whether you have applied for a new dosage form medicine or a non-new dosage form. This assessment will be completed by the relevant Clinical Evaluation Unit.

TGA may seek independent expert advice on aspects of the assessment if required.

A recommendation from the relevant Clinical Evaluation Unit will be referred to TGA's Chief Medical Advisor, who is the delegated decision-maker for designation applications.

Requests to the sponsor for additional information

You may receive requests for additional information or clarification from TGA during the course of the assessment. Such requests will include a timeframe for response and TGA contacts for you to provide your response. The standard timeframe for your response is five working days.

It is your responsibility to provide TGA with sufficient evidence to allow the decision-maker to be satisfied that the eligibility criteria have been met.

Any time taken for you to respond to requests will extend the overall assessment and decision-making timeframe. If no response is received in the specified time period, TGA will make a decision based on the available information.

If we find that your application is likely to be ineligible for designation, you will have the opportunity to provide comment on the decision prior to finalisation. In this circumstance, you will have a period of five working days to provide a statement of perceived errors of fact or omission to TGA.

Withdrawing your designation application

You may withdraw your designation application at any time before we issue a decision.

To withdraw the application, email AET.Application.Entry.Team@health.gov.au.

Your email should include:

  • a statement that you wish to withdraw a designation application
  • the designation application number
  • the active ingredient
  • the sponsor's name
  • the proposed indication for designation.

We will not publish applications for designation that are withdrawn before a formal designation decision is issued on our website.

Step 5: Notifying sponsors of the designation decision

If TGA assesses that your application for designation is eligible, the designation will be approved.

After the delegate has made a decision, we will advise you of the outcome as soon as practicable in writing via a notification letter issued through eBS which will include:

  • details of designation(s) eligible/ineligible and the relevant indication
  • a statement of reasons for designation decisions (for ineligible decisions only)
  • the date on which the designation (if approved) lapses (six months after the date of the designation decision)
  • details of your appeal rights.

How to appeal the designation decision

Ineligible designation decisions are appealable under Regulation 48 of the Therapeutic Goods Regulations 1990.

Further information on how to seek internal review by TGA or external review by the Administrative Appeals Tribunal is available on our website.

Publication of designation outcomes

If your designation application is approved, TGA will publish details of the eligible designation decision on its website.

Publication will include:

  • the name of the medicine
  • the sponsor's name
  • the eligible Orphan drug indication
  • the dosage form
  • designation decision date and lapsing date
  • designation status (i.e. active or lapsed).

Designation decisions will be published as soon as we have made a decision and you have been notified.

We will not publish details of designation applications that are assessed as not eligible or those that are withdrawn before we make a decision.

Lapsing or revocation of designation

Eligible Orphan drug designations will automatically lapse six calendar months after the date of the designation decision, as outlined in Section 16K of the Therapeutic Goods Regulations 1990.

To be eligible for the Orphan drug fee waiver, the designation must be active at the time of lodging your submission for registration (this refers to the dossier, not the PPF).

TGA reserves the right to revoke the designation before the end of the six month lapsing period under section 16M of the Therapeutic Goods Regulations 1990. This may occur if the delegate is satisfied that the medicine no longer meets the relevant eligibility criteria (either new dosage form, or non-new dosage form). However, we will not routinely review designation decisions during their validity.

If TGA decides to revoke your designation, you will be advised in writing as soon as practicable after the decision has been made.

Decisions regarding revocation of designations are appealable under regulation 48 of the Therapeutic Goods Regulations 1990.

You may also apply for revocation of Orphan drug designation before the lapsing date. The process for applying for revocation of a valid Orphan drug designation before lapsing is as per withdrawing your designation application.

Applying for extension of designation

If you are unable to lodge your submission for registration within the six-month lapsing period, you may apply to TGA in writing for one extension of your Orphan drug designation only. Approved extensions will be granted for a further six months, as set out in Section 16L of the Therapeutic Goods Regulations 1990.

You must submit applications for extension of Orphan drug designations at least four weeks before the lapsing date.

Applications for extension must use the 'Application for extension of orphan drug designation validity' form on the TGA website. The form should be scanned, signed and sent via email in PDF format to AET.Application.Entry.Team@health.gov.au.

In the application form, you must:

  • explain why you are seeking an extension and provide justification of why the extension will allow you to lodge a registration application within the extension period, AND
  • provide an updated current justification to establish that:
    • there are no existing therapeutic goods for diagnosis, prevention or treatment of the condition in question, OR
    • if such therapeutic goods exist, that the medicine will bring improved efficacy, safety or a major contribution to patient care to those affected, AND
  • provide a current update on the approval status of the medicine by the regulatory agencies specified in section 16J of the Therapeutic Goods Regulations 1990. The medicine may no longer be eligible if an application has been refused by one of these regulatory agencies due to the medicine's safety.

Notification of outcomes - Application for extension

You will be notified of the outcome of your request for extension via email as soon as possible after the decision has been made by TGA.

Approved extension letters will include details of your revised Orphan drug designation lapsing date.

If your request for extension is not approved, you will be provided with a statement of reasons for the decision.

Decisions regarding a refusal to extend an Orphan drug designation are appealable under regulation 48 of the Therapeutic Goods Regulations 1990. However, a decision to approve extension of an Orphan drug designation is not appealable.

You need to lodge a new designation application if the Orphan drug designation has lapsed and extensions have lapsed or are not approved.

Re-lodgement of lapsed designation

If you wish to seek Orphan drug designation following a lapsed designation application (for a medicine and indication that has been previously designated but where the validity of the designation has lapsed) you will need to lodge a new designation application.

We will re-assess the new designation application against the eligibility criteria, as some aspects such as comparison against existing therapeutic goods may have changed since the original designation decision was made.

For re-lodged applications, you are required to provide the reference number/s for previous designation applications.

Further assistance

If you have read the guidance and still require assistance, please contact: AET.Application.Entry.Team@health.gov.au.

Step 6: Submitting your application for registration

You will have until the lapsing date nominated in the designation decision letter (or extension approval letter, if applicable) to provide a submission for registration.

Designated Orphan drugs can be lodged through any available registration pathway that the Orphan drug is eligible for, including the standard prescription medicines registration process or the Priority Review pathway (subject to priority designation).

The Provisional Approval pathway for the registration of prescription medicines is not envisaged to be available until early 2018. Eligibility criteria for Provisional Approval designation (and therefore access to the pathway) will be available closer to the time.

Guidance on submitting your PPF is available on the TGA website.

Process for Orphan drug designation

Diagram showing the orphan drug designation process

Version history

Version history
Version Description of change Author Effective date
V1.0 Original publication Policy and Reform Facilitation Section, Prescription Medicines Authorisation Branch June 2017