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Submissions received: Provisional approval pathway for prescription medicines

1 September 2017

The TGA would like to thank respondents who provided submissions in response to the March 2017 public consultation paper, Consultation: Provisional Approval pathway for prescription medicines.

A total of 30 submissions were received. Of those submissions, 17 were from industry and regulatory consultants, 1 from an industry representative group, 2 from individuals, 3 from health professional groups and 7 from consumer, academic and other professional representative groups.

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

A separate public consultation paper sought comments on the implementation of a range of enhancements to the TGA medicines Vigilance Framework. Submissions received in response to this consultation process can be found here



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TGA response

Feedback has been considered from the 30 submissions received and from a TGA workshop held with representatives of industry, healthcare professional groups and consumer groups. Stakeholders welcomed the new registration pathway for prescription medicines and provided overall support for the TGA's proposed implementation approach. There were mixed views on some issues. The TGA has sought to balance the differing stakeholder views by ensuring appropriate alignment with the approach taken by international regulators and the Government’s intention of the pathway, which is to facilitate faster access to certain promising medicines while maintaining patient safety.

Stakeholder feedback has informed the policy position going forward and upcoming changes to the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. Policy changes as a result of this public consultation process are summarised in the Outcome Summary below.

Further information on process details of the Provisional Approval pathway, which have been informed by this consultation, will be included in guidance documentation to be released on the TGA website closer to the pathway’s implementation.

Outcome Summary

Change Table 1: Summary of changes resulting from public consultation: Provisional Approval pathway for prescription medicines
Current situation Proposed changes Benefits
Pre-submission and designation process

There is no formal mechanism for registering prescription medicines on the basis of early clinical data, which is out of step with comparable overseas regulators.

In exceptional circumstances, some medicines have been granted full registration on the basis of early clinical evidence on efficacy and safety where there is potential for significant benefit to patients with unmet needs.

Sponsors of medicines with early clinical data are strongly encouraged to request a pre-submission meeting approximately six to seven months prior to submission.

Eligibility for the Provisional Approval pathway will be determined through a formal designation process. The sponsor must demonstrate the seriousness of the condition, comparison against existing registered therapeutic goods and promising evidence of a major therapeutic advance.

Provisional Approval designations will remain valid for six months, with a possible six month extension at the TGA's discretion.

Provides a formal and transparent process for the registration of certain promising medicines on the basis of early clinical data. These medicines may reach patients with unmet needs up to two years earlier than under the current framework.

Sponsors can discuss their application with the TGA to determine the appropriate registration pathway.

Aligns with similar processes offered by overseas regulators and fulfils the Government’s objectives of providing earlier access to certain medicines while maintaining the TGA’s regulatory standards.

Pre-market registration process

Full dataset must be submitted at the time of lodgement, unless further data is requested by the TGA or is related to safety.

Sponsors must provide a scientifically robust justification if they are unable to fulfil the adopted scientific guidelines for clinical, quality and non-clinical safety modules of the dossier.

Statutory timeframe of 255 working days for evaluation.

Applications for the Provisional Approval pathway must be accompanied by a valid designation at the time of submission. The TGA will not provisionally register a medicine that has been submitted through the standard pathway.

Comprehensive non-clinical data on safety, quality and Good Manufacturing Practice must be submitted at the time of lodging a Provisional Approval registration application.

Provisional Approval registration applications will be prioritised within the existing statutory timeframe. Rolling submissions of clinical data can be agreed upfront, but this may impact on the evaluation timeframe. There may be higher fees and/or annual charges in line with cost recovery arrangements.

Proposed legislative amendments will allow certain medicines to be provisionally registered in the Australian Register of Therapeutic Goods (ARTG) for a limited duration on the basis of early clinical data.

Sponsors will benefit from upfront agreement of rolling submissions of clinical data and prioritisation of Provisional Approval applications. The TGA will ensure that there is clear guidance and predictability of timelines as requested by industry stakeholders.

The TGA will ensure that Provisional Approval applications are subject to the appropriate level of scrutiny and deliberation to weigh up the benefits and risks of earlier availability of the medicine in the absence of full clinical data.

Post-market requirements  for provisionally registered medicines

Registration in the ARTG is subject to any conditions imposed by the TGA.

Registered medicines are subject to routine post-market monitoring and compliance activities.

The TGA has enforcement powers to suspend or cancel registration if concerns arise about the medicine’s efficacy, quality or safety; or if registration conditions are not met.

Sponsors will be required to collect and submit confirmatory data on the medicine’s efficacy and safety. In exceptional circumstances, this may be through 'real world' observational data (e.g. from a patient registry).

Provisionally registered medicines will be subject to additional risk minimisation activities and prioritised for the TGA’s enhanced post market monitoring.

Conditions of provisional registration will be generally consistent with those imposed by comparable overseas regulators, unless Australian specific activities are required to monitor or mitigate safety concerns.

Additional enforcement powers have been proposed to allow the TGA to vary the approved patient population or directions of use for a provisionally registered medicine if there is reasonable concern about its efficacy or safety.

Post-market requirements to collect additional clinical data will ensure that these medicines will meet the TGA standards before full registration is granted.

Given that provisionally registered medicines may be of higher risk, enhanced post-market monitoring will ensure that efficacy and safety issues are identified early. It will also ensure that sponsors meet their obligations within a maximum provisional registration period of up to six years.

Additional risk minimisation activities will ensure that information about provisionally registered medicines will be clearly communicated to patients and health care professionals.

Post-market requirements  for provisionally registered medicines Medicines are registered in the ARTG until they are withdrawn by the sponsor, or suspended or cancelled by the TGA.

Provisional registration will automatically lapse after a two year period unless the sponsor has applied for full registration or the TGA has granted a two-year extension, up to a maximum period of six years.

Where appropriate, patients may be able to continue accessing a medicine after provisional registration has lapsed through existing avenues for unapproved therapeutic goods.

The two-year validity will provide regular checkpoints for the sponsor to demonstrate that they are on track with fulfilling post-market conditions.

Other implementation considerations

The Australian Public Assessment Report (AusPAR) provides information about the evaluation of a prescription medicine and the rationale for TGA decisions.

A person whose interests are affected by certain TGA decisions may seek an internal review of the decision or appeal to the Administrative Appeals Tribunal.

A medicine cannot be listed on the Pharmaceutical Benefits Scheme (PBS) prior to inclusion in the ARTG.

In the interests of expediting Provisional Approval applications, appeals will be limited to certain TGA decisions and to the sponsor only. This will be balanced with the increased transparency, including publication of Provisional Approval designations; a rapid publication of TGA decisions relating to provisional registration; and full details of decisions relating to provisionally registered medicines in the AusPAR. It is proposed that information will be published on the progress of fulfilling provisional registration conditions; automatic lapsing; extensions; modifications to provisional registration; suspension or cancellation; withdrawal by the sponsor; and transition from provisional to full registration.

Provisional registration will fulfil the PBS requirement of ARTG inclusion. Sponsors may apply for PBS listing of a provisionally registered medicine, including through a Managed Entry Scheme (MES) where criteria are satisfied.

Health professional and consumers will have transparency of TGA decision making processes, to inform their treatment decisions and maintain confidence in the TGA’s regulatory standards.

The Pharmaceutical Benefits Advisory Committee (PBAC) may consider applications for the reimbursement of provisionally registered medicines. The Government is working to ensure alignment of regulatory and reimbursement processes where appropriate.