We asked, You said, We did

Below are some of the issues we have recently consulted on and their outcomes.

We asked

The 2022 follow up consultation sought stakeholder input into four core challenges for the repurposing of medicines:

  • address commercial and intellectual property issues;
  • identify the best candidates for repurposing;
  • prioritise candidates; and
  • approach and incentivise the market.

You said

Twenty-seven (27) responses were received from four key stakeholder groups: patients and patient groups, health professionals, academia, and the pharmaceutical industry. The feedback we received can be summarised in four areas.

Overcoming commercial constraints and addressing intellectual property issues

  • Incentives will need to counter costs involved in preparing a submission, fees, the burden of post-market surveillance and supply guarantee obligations.
  • Collaborative submissions would require clear, fair, and appropriate incentives for all involved. Responsibilities and liabilities would need to be clearly determined. The Department will also need to consider anti-competition issues.
  • Clarity regarding data sharing and ownership, regulatory, manufacturing, and operational responsibilities, as well as cost sharing will be required when repurposing off-patent medicines.
  • Extension of data or market exclusivity regimes to the repurposed indications may discourage ‘game play’ and strategic timing of new indications to maximise market monopoly through ‘rolling’ periods of data exclusivities.

Identifying potential candidates

  • The Department should make patient voice a priority.
  • Rare diseases and unmet need should be focus areas.
  • Candidates may be identified in current clinical practice documentation such as clinical guidelines and Standards of Care.
  • National and state organisations such as the Council of Australian Therapeutic Advisory Groups (CATAG), as well as specialist medical colleges and compassionate programs may be able to identify potential candidates.
  • Involvement of hospital networks and pharmacies in sourcing pharmacy compounding records, retrospective medication charts and patient records may enable a demand driven model for identifying potential candidates.
  • Horizon scanning for new products that might enter the market and meet unmet patient needs should be conducted in parallel with the repurposing process.

Prioritising candidates

  • Real-world data insights should be considered early in the process of prioritisation.
  • Candidate medicines that have the largest impact on patient care and quality of life should be a priority.
  • An expert advisory panel with representation from all stakeholders and expertise in all the different facets of the repurposing process should be responsible for prioritising candidates.
  • Safety, efficacy, commercial feasibility, and other factors should be considered early in the process for efficient use of time and resources.
  • Clear guidance and criteria should be set by the Department.
  • If the Department chooses to set priority therapeutic foci, National Health Priority Areas (NHPA) should be considered, with flexibility where unexpected changes arise due to shortages, commercial decisions or delisting.
  • The TGA could publish a list of indications prioritised for repurposing to attract sponsors of off-patent product who have interest and capability to pursue a new indication even if they do not currently sponsor the candidate product in Australia.
  • The current EMA pilot of repurposing medicines could be a model for the prioritisation of candidates in Australia.
  • The Government should be transparent about how the decisions are made, which indications may be considered, and the outcomes of applications.

Encouraging sponsors to apply for regulation and reimbursement

  • Fee waivers and exclusivity periods of sufficient length (e.g., up to 5 years) may provide adequate financial returns to cover the ongoing cost of pharmacovigilance activities and financial and legal obligations for local sponsors.
  • A streamlined process with simpler data requirements would reduce the burden on sponsors of applying for the repurposed indication.
  • Detailed guidance on how the TGA and PBAC will evaluate repurposing applications would be beneficial.
  • Active negotiation such as pre-submission meetings between sponsors and government as well as parallel evaluations by the TGA and PBAC may help clarify minimum standards for approval, garner mutual commitment and expedite the process.
  • An initial feasibility assessment of an acceptable price would allow sponsors to explore commercial incentives, and consider international pricing risks.
  • PBS exclusivity rather than regulatory exclusivity may be attractive to some sponsors.
  • Sponsors should be allowed ample time (e.g. up to 3-4 months) to consider their interests and decide to take on a repurposing application.

We did

The Department is now using this and other feedback to shape regulatory reforms and policy. Consultation with internal and external stakeholders continues. Implementation of some options may require government approval (regulatory change) and/or parliamentary approval (legislative change), and the Department will present these to the Government for consideration.

We asked

We asked for feedback on proposed new functionality that would allow sponsors to view and export relevant de-identified medicine adverse event data from TGA systems, using their existing sponsor authentication process (e.g. TGA Business Systems login credentials). We specifically asked about:

  • The type of medicine adverse event data sponsors would wish to view and/or extract from TGA’s Adverse Events Management System (AEMS) and their preferred format.
  • Sponsors’ views on using the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) Enterprise to Business (E2B) R3 standard  for submitting and receiving adverse event reports via the secure Electronic Data Interchange (EDI) service.

You said

We received 44 survey responses from sponsors and research organisations spanning prescription, non-prescription and complementary medicines, and medicinal cannabis products. Respondents broadly supported the initiative and provided valuable feedback on our proposals.

Sponsor access to medicine adverse event data

Almost all respondents stated they rely on the public Database of Adverse Event Notifications (DAEN)-medicines and direct email requests to TGA to obtain information on medicine adverse events. Most respondents import this data into their company pharmacovigilance systems to fulfill their TGA pharmacovigilance responsibilities and to supplement their international reporting obligations.

For the proposed new functionality respondents wished to access the same information they receive through their existing mechanisms. They also requested access to additional data, such as data to assist in identifying duplicates, information about the seriousness of an adverse event, concomitant use of medicine and medical conditions, and case type (e.g., post-market, Special Access Scheme (SAS) or under clinical trial).

Viewing/extracting adverse event data held within TGA systems

Most respondents supported the use of the proposed functionality to view adverse event data that relates to both their own products and other products which have the same active ingredient. A large majority stated that they wish to extract data directly into their pharmacovigilance systems. Although it is not mandatory for sponsors to seek historical adverse event cases, many requested access to the full range of available mapped sponsor-product data. Many respondents indicated that automating the ability to extract data would reduce regulatory burden for sponsors in meeting their pharmacovigilance responsibilities. The majority preferred receiving adverse events data via E2B R3 ICSR message, with a flat .CSV file being the second preferred option. Other file format options were pdf and xml.

Most respondents agreed that agents (i.e. nominated officers external to the company who have permission to undertake regulatory correspondence with TGA on behalf of the sponsor) should have the same access as sponsors to this data.  

EDI Gateway

The majority of respondents stated they currently submit cases through the EDI Gateway, with almost all eager to receive data through this functionality. Some respondents who have not yet implemented EDI functionality also showed interest in future adoption to reduce manual processes.

Where use of EDI was not adopted, respondents stated this was due to concerns over lack of expertise in required standards, perceived complexity of the functionality, resource constraints, low volume of reportable cases and need for consistent formats with global partners.

E2B R3 – Current behaviour and transition

Most respondents currently submit case reports to overseas regulators using the ICH E2B(R3) standard and almost all respondents supported a voluntary transition to use of this standard as an input channel. Where not supported, reasons included concerns over company size, need for resources, and complexities involved in adoption.

Most respondents were eager to adopt the updated standard within a year’s time, with the majority stating they could transition within 6 months. Most other respondents are willing to transition within 2 years, with a small proportion stating they would require 3-5 years for implementation.

Product terminology

WHO drug dictionary was the most commonly used terminology for coding product information in sponsors’ internal systems and cases submitted in the E2B R3 format. Some respondents stated they use extended EudraVigilance medicinal product dictionary (XEVMPD) and bespoke terminology.

Some respondents are already using ISO IDMP standards for submitting cases in the E2B R3 format, with a few also planning to adopt these in the near future.

We did

We are considering your feedback

Thank you to everyone who responded to the survey. The feedback received will inform IT requirements for the proposed functionality, as well as our privacy impact considerations. Guidance for sponsors about this new functionality will be published as part of implementation.

Other improvements to how we share adverse event data

We are currently working on restoring the ability for users to extract and save search results from the DAEN‑medicines, as well as other improvements to how information is displayed on‑screen. These improvements are expected to be available on the TGA website in the coming months.

We asked

Public consultation was held between 12 October 2021 and 11 November 2021 on the Delegates’ interim decision in relation to sodium nitrite referred to the June 2021 joint meetings of the Advisory Committee on Medicines Scheduling (ACMS) and the Advisory Committee on Chemicals Scheduling (ACCS) held on the 23 June 2021.

You said

Respondents provided feedback on the interim decision regarding sodium nitrite. We received two submissions in response to the public consultation.

We did

The Delegates considered all submissions prior to making their final decision. The final decision was published on 19 January 2022 on the TGA website.

We asked

The TGA sought feedback from interested parties on the proposed low-negligible risk changes to the Permissible Ingredients Determination. The proposed changes were intended to address the following issues:

  1. Allergen statement for mollusc-derived ingredients.
  2. Peripheral neuropathy associated with lower dose vitamin B6.
  3. Risk to infants from nasal use of benzalkonium chloride.
  4. Artemisinin and pregnancy risk.

You said

A total of 20 submissions were received in response to this consultation. All submissions that gave permission to be published on the TGA website are available through the ‘View submitted responses’ link below.

The TGA received:

  • 6 responses to the proposed mollusc warning statement,
  • 15 responses to the proposed changes for vitamin B6 ingredients,
  • 5 responses to the proposed changes to benzalkonium, and
  • 8 responses to the proposed warning statement for artemisinin-containing ingredients.

There was general agreement for the proposed mollusc label statement and changes to benzalkonium for use in nasal sprays, whereas the submissions concerning vitamin B6 and artemisinin varied in stance and recommendations.

We did

The feedback and recommendations provided by respondents were taken into consideration, and the proposed changes have been finalised. The final changes incorporate some variation compared to the original proposals.

The final changes will commence on 1 March 2022. A 1 year transition period will be instated to allow sponsors to ensure product compliance, ending on 1 March 2023.

We asked

We asked for feedback on potential refinements to the existing regulatory requirements for patient information materials, including the content and form of the materials and exempt devices.

You said

A total of 34 submissions were received with more than half of the submissions from health care professionals and hospitals who are responsible for providing information to patients. Submissions were also received from consumers, professional health related societies and the medical device sector.

Almost all respondents, including the consumers, agreed with the proposals for providing information in a range of formats. This would allow information to be recorded in electronic formats such as electronic patient records and MyHealth records. Although, several respondents considered that regardless of how the information is kept, patients should also have the option to receive a hard copy.  Most were in favour of flexibility in the way information is provided. There were also suggestions for providing patient information materials in different languages and in audio form. The medical device industry respondents noted that electronic formats would reduce cost and some health care providers considered that costs are offset by benefits. However, there were several concerns raised about the burden on hospital staff and administration times if the system is too demanding or complex.

The majority of respondents agreed that it is unnecessary to provide patient implant cards if the device is going to be absorbed by the body within a certain timeframe. Those that stated they did not agree had concerns over delayed reactions to such implants. The suggested timeframes varied from days to 2 years, but the majority of those who responded opted for periods of 3 to 6 months.

The strong majority of respondents agreed that it is unnecessary to provide patient implant cards for pedicle screws, given that many types of screws could be used in one surgery.  There were suggestions that a system implant card is sufficient (that covers all implanted devices from one system), or that a generic patient information leaflet could be provided. In general, the responses indicate that it is unnecessary to provide patient implant cards for each screw or component, but the requirement for a patient information leaflet should be retained.

Although only a few respondents had comments on other devices which could be exempt from the requirements, there was strong representation for exempting reloads with staple line reinforcement, sutures with pledgets, and pledgets alone from requiring patient implant cards. There was also a recommendation to ensure alignment with the European requirements by exempting spinal rods, suture anchors, wedges (augments), laminar hooks, non-expandable cages, interspinous spacers, and bone screws.

There were also some questions about certain devices such as coronary stents and dental crowns, which indicated that there was still some confusion about the regulatory requirements for these devices.

It was noted that there was a need for increased communication about the implementation including education for hospitals, procurement officers and clinicians in relation to the provision of these materials.

We did

Based on the outcomes from the consultation, agreement will be sought from the Government to consider refinements to the Regulations. These refinements would:

  • allow more flexibility in how patient information materials can be provided including in a range of formats such as electronic format and at either pre or post surgery (i.e. not necessarily supplied with the device in the packaging);
  • exempt devices absorbed by the body within a defined time period from the requirement for a patient implant card but maintain the requirement for a patient information leaflet;
  • exempt pedicle screws from the requirement to have a patient implant card. The requirement for a patient information leaflet will be retained, but could be incorporated into a system patient information leaflet where appropriate; and
  • create the power for a TGA delegate to consider exempting other devices where clinically appropriate to ensure alignment with Europe, where possible.

Guidance documents will be updated to address other concerns raised in the consultation. Continued education will also be provided, including clear messages to relevant groups such as hospitals, surgeons, clinicians and procurement officers in relation to the implementation of patient information materials.          

Guidance will be further updated following a decision from the Government on the proposed refinements.

We asked

Public consultation was held between 30 July 2021 and 27 August 2021 on the Delegate’s interim decision in relation to a scheduling proposal about nitrous oxide that was referred to the March 2021 joint meeting of the Advisory Committee on Medicines Scheduling and the Advisory Committee on Chemicals Scheduling.

You said

We received seven submissions in response to the public consultation. Respondents provided feedback on the proposed changes to the Poisons Standard.

We did

The Delegate considered all submissions prior to making a final decision on this proposal. The final decision was published on 8 October 2021 on the TGA website.

We asked

Public consultation was held between 20 July 2021 and 17 August 2021 on the Delegates’ interim decisions in relation to scheduling proposals referred to the March 2021 meetings of the Advisory Committee on Medicines Scheduling and the Advisory Committee on Chemicals Scheduling.

You said

We received 34 submissions in response to the public consultation, including one submission that was received offline. Respondents provided feedback on the proposed changes to the Poisons Standard.

We did

The Delegates considered all submissions prior to making their final decisions on these proposals. The final decisions were published on 9 September 2021 on the TGA website.

We asked

We asked for feedback on potential refinements to the Regulations in relation to classification rules for medical devices that are substances introduced into the human body via a body orifice or applied to the skin.

You said

A total of 45 submissions were received. They comprised of 17 from the medical device or medicine industry (manufacturers, sponsors or peak body associations) and 28 from consumers.

The 28 responses from consumers have been noted but have not been taken into account in this consultation. It appears these respondents misunderstood the intent of the consultation due to social media incorrectly suggesting the consultation was in relation to microchipping individuals.

Of the remaining 17 respondents, the majority (11) did not support the proposal to refine the Regulations. While, the other six respondents supported the proposed refinements.

There seemed to be a general misunderstanding of the consultation and types of devices covered by the relevant classification rule. Some respondents were unsure how their product would be regulated under the proposed refinement, and some did not support the proposal if it meant their products would become a medicine. Some respondents raised issues that were not directly related to the questions in this consultation, including concerns about the interface between medical devices and foods. The intent of the proposed refinement to the classification rule was not for products to change regulatory pathways, but rather to provide clarity about which pathway the product should take (i.e. medicine or medical device).

Some respondents believed there would be an increased regulatory burden should the classification rule be amended. They believed that Australia would not align with Europe. Where sponsors may not have the resources to support a product to be regulated as a medicine in Australia, whilst the same product would be regulated as a medical device in Europe. Some respondents also noted that Australian patients may miss out on a number of products if there was a change in the regulatory pathway. Not all sponsors would continue to supply the product if it deviated from other major markets in its classification and regulatory pathway.

Importantly, some respondents did not appreciate that:

  • any product that is a substance-based medical device will be a Class IIb device instead of a Class III device (so it represents lower regulatory burden than in Europe); and
  • the mode of action will be reviewed should the product appear to not meet the definition of a medical device as defined in the Therapeutic Goods Act; and
  • without the proposed refinement, from 25 November 2021, some products would be reclassified to Class III devices and the regulatory burden increased.

This consultation strongly highlighted that clarification was urgently needed on this topic, including on a number of other matters in relation to this classification rule and definitions. There is an urgent need to clarify and provide a definition for absorption, chemical action and physical barriers, both of which help determine whether a product should be regulated as a medicine or medical device.

We did

Whilst industry concern over misalignment with Europe and their Medical Device Regulations were noted, the Australian regulatory framework and definitions are different to those in Europe. The proposed refinements to the Medical Device Regulations (as set out in the consultation paper) would not have the effect of pushing any of these products to be considered as medicines – the proposed refinements would principally reduce some of the confusion in the language of the device classification rules and avoid any such products being up-classified to the Class III level.

Based on the outcomes from the consultation, agreement will be sought from the Government to consider refinements to the Regulations. These refinements would remove reference to products that are systemically absorbed and provide clarity around the regulation of these products.

Guidance will be further updated post a decision from Government on the proposed refinements and to address other concerns raised in this consultation as well as continued education in relation to the regulation of products containing substances.

We asked

We asked for feedback on proposals to remake some of the legislative instruments relating to human cell and tissue (HCT) products (including blood and blood components), which sunset in October 2021.

You said

We received 33 submissions in response to the consultation, including from government, sponsors, industry organisations, as well as from individual consumers. There was broad support for the remaking the legislative instruments relating to human cell and tissue (HCT) products (including blood and blood components), with proposed amendments. Responses from the eye banking sector did not initially support some proposed changes.  

In addition, stakeholders provided a range of views and suggestions to improve the standard, including:

  • Label requirements too onerous for biologicals not for supply in Australia.
  • Introduce risk-based assessments as an alternative for HTLV serological testing for donor screening requirements.
  • Donor history requirements specified in TGO 108 as burdensome for ‘export only’ plasma for fractionation (PFF).

The changes proposed in the public consultation and above referenced feedback have been incorporated in the final standards.

Some feedback was not accepted as matters either affected or were beyond scope of the quality, safety, efficacy of therapeutic goods.

Thank you to everyone who provided feedback and helped in the development of the standards.

We did

Most changes proposed in the standards are deregulatory or the current sponsors already comply with the new requirements.

To allow sponsors to meet the quality system requirements a 12-month transition period will be allowed for all standards from 1 October 2021 to 30 September 2022.  During this time the former Orders may be conformed with, despite being repealed, as an alternative to the new standards.

The exception to this are donors whose ocular tissue will be released for supply solely for the purpose of corneal transplantation (‘cornea only donors’). TGO 108 will now mandate additional serological testing for human T cell leukaemia virus types 1 and 2 (HTLV-1/2) and syphilis, with nucleic acid amplification testing (NAT) for human immunodeficiency virus types 1 and 2 (HIV), Hepatitis C virus (HCV) and Hepatitis B virus (HBV) for these donors. An extended transition timeframe of 3 years for ‘cornea only donors’ from additional donor testing requirements in TGO108 to allow adequate time for availability of appropriate testing facilities in some states and territories.  

The new orders TGO107, TGO 108 and TGO109 come into effect on 30 September 2021.The Therapeutic Goods (Biologicals - Specified Things) Instrument 2021 also comes into effect on 30 September 2021.

 

 

 

We asked

We asked for feedback on proposals for improving the Therapeutic Goods Advertising Code (No.2) 2018 (the Code). The proposals included changes to provisions of the Code that had been identified as being unclear, inconsistent, or otherwise difficult to work with. The proposals were designed to improve advertising compliance, minimise advertising compliance costs, and ensure Code provisions work as intended.

We canvassed stakeholder preferences for different options for some provisions. We also invited stakeholders to identify other areas where the Code could be improved.

You said

We received 67 submissions in response to the consultation, including from sponsors, manufacturers, advertisers, marketers and sellers of therapeutic goods, peak industry bodies and associations, regulatory affairs firms, policy advocates, and health professional and consumer organisations. There was broad support for amending the Code to reduce complexity and increase clarity for users. Stakeholders also advocated for a review of current guidance on the Code to ensure it remains contemporary and easily accessible.

On some proposals, there were widely divergent views:

  • There was broad support for clarifying the requirements around claims in advertising, including ‘therapeutic’ and ‘non-therapeutic’ claims, and the evidence requirements for each. But there was a divergence of views as to whether this should be done in the Code, or in guidance.
  • The proposal to strengthen the rules around the use of language and images in advertising that may invoke a sense of fear or distress in consumers also produced a range of views.
  • Stakeholder views about who should and who should not be allowed to make product endorsements and testimonials for use in advertising were widely divergent.
  • There was also a range of views around the rules relating to advertising involving the offer or provision of free samples of goods, and the criteria that should apply when determining what types of goods should be exempted from any general restrictions on this practice.

However, stakeholders broadly supported the introduction of alternative mandatory statements in advertisements for typically ‘non-consumer’ therapeutic goods. Many stakeholders also indicated they wanted simpler rules around the use of existing mandatory statements and health warnings in advertisements.

Thank you to everyone who provided feedback.

We did

In response to this feedback, we have decided to re-write the Code using a simplified structure and plainer language. We will also ensure the guidance on the Code is more accessible and user-friendly.

On specific Code issues, we are reflecting on feedback and undertaking further targeted consultations with members of the Therapeutic Goods Advertising Consultative Committee.

We aim to prepare and publish the new Code by the end of 2021. Please subscribe to the TGA Advertising email list to be notified of progress.

We asked

Public consultation was held between 27 April 2021 and 27 May 2021 on scheduling proposals referred to the June 2021 meetings of the Advisory Committee on Chemicals Scheduling (ACCS) was held on 22 June 2021.

You said

Respondents provided feedback on the proposed changes to the Poisons Standard. We received 10 submissions in response to the public consultation.

 

We did

The Delegate considered all submissions prior to making an interim decision on these proposals. The interim decision was published on 13 October 2021 on the TGA website, with the eugenol interim decision published alongside other substances considered at the Advisory Committee on Medicines and Chemicals Scheduling meeting.

We asked

Public consultation was held between 27 April 2021 and 27 May 2021 on scheduling proposals referred to the June 2021 meeting of the Advisory Committee on Medicines Scheduling (ACMS) held on the 23 June 2021 and 24 June 2021.

You said

Respondents provided feedback on the proposed changes to the Poisons Standard regarding oral contraceptive substances. We received 27 submissions in response to the public consultation.

We did

The Delegate considered all submissions prior to making an interim decision on these proposals. The interim decision was published on 30 September 2021 on the TGA website.

We asked

Public consultation was held between 27 April 2021 and 27 May 2021 on scheduling proposals referred to the June 2021 meetings of the Advisory Committee on Medicines and Chemicals Scheduling held on 23 June 2021.

 

You said

Respondents provided feedback on the proposed changes to the Poisons Standard regarding sodium nitrite. We received seven submissions in response to the public consultation.

We did

The Delegate considered all submissions prior to making an interim decision on these proposals. The interim decision was published on 12 October 2021 on the TGA website.

We asked

Public consultation was held between 27 April 2021 and 27 May 2021 on scheduling proposals referred to the June 2021 meeting of the Advisory Committee on Medicines Scheduling (ACMS) held on the 23 June 2021 and 24 June 2021.

You said

Respondents provided feedback on the proposed changes to the Poisons Standard. We received 11 submissions in response to the public consultation.

We did

The Delegate considered all submissions prior to making an interim decision on these proposals. The interim decision was published on 30 September 2021 on the TGA website.

We asked

Respondents provided feedback on the proposed changes to the Poisons Standard. We received six submissions in response to the public consultation.

You said

Respondents provided feedback on the proposed changes to the Poisons Standard. We received six submissions in response to the public consultation.

We did

The Delegate considered all submissions prior to making an interim decision on these proposals. The interim decision was published on 13 October 2021 on the TGA website.

We asked

We asked for feedback on proposals to help ensure the ongoing, reliable supply of important medicines in Australia. Four proposals were consulted on which sought feedback on medicine shortage mitigation or management strategies that would:

  • prioritise the evaluation and registration process for certain important generic prescription medicines, to reduce the risk of shortages
  • encourage registration of more generic versions of medicines known to be affected by shortages, to mitigate the impact of those shortages
  • support a more reliable supply of overseas-registered medicines imported into Australia as substitutes through an annual charge waiver when the Australian-registered medicine is in longstanding or repeated shortage.

You said

We received 47 submissions in response to consultation from industry organisations and peak bodies, health professionals or heath professional organisations, consumer groups and individuals. Respondents provided a range of views and suggestions.

There was general support to enable import of overseas substitutes for discontinued medicines by granting annual charge waivers (proposal 4). Concerns were raised that the charge waiver may negatively impact fairness and sustainability of the TGA cost recovery system by shifting the waived annual charge cost onto other medicine sponsors. A concern was raised that this proposal will increase confusion for patients and healthcare professionals since more discontinued products would be included on the Australian Register of Therapeutic Goods (ARTG), in order to allow the import of overseas substitutes.

Views were mixed on incentivising registration of medicines known to be in repeat or longstanding shortage (proposals 2 and 3). Some stakeholders raised that incentivising registration is unlikely to address the larger issue of shortages. Once registered, there is no guarantee of medicine supply, nor will the proposals overcome other barriers to supply, such as manufacturing issues. Concerns were raised that long term stability of supply is driven by commercial viability, and that TGA fees are not the major factor driving commercial viability. Waiving the fees would therefore have limited impact on supply of medicines. Some stakeholders identified evidence requirements as a barrier to registration, whereas others raised that changes should not result in reduced TGA submission standards.

Overall support for prioritising the registration of new generic medicines was poor (proposal 1). Stakeholders raised that prioritisation of new generics might displace existing products from the market thus failing to address the aim to create a more robust medicine supply. In addition, the TGA evaluation time was not seen as an impediment to the availability of generic products in Australia.

We did

Since public consultation commenced in March 2021 two complementary strategies have been introduced that directly address the issue of medicine shortages.

  • The Medicine Supply Guarantee was announced by the Health Minister late in 2021 to better protect patients against supply chain volatility. Medicine companies have committed to hold additional onshore stock of certain medicines, for at least four to six months’ supply.
  • The Serious Scarcity Substitution Instruments (SSSIs) were introduced in 2021 and are a powerful tool to help patients receive their medicines from their pharmacist without delay, and also provide better treatment continuity during a medicine shortage. Additionally it relieves workload pressure on prescribers and pharmacists. The instruments allow community pharmacists to substitute specific medicines without prior approval from the prescriber.

In response to the consultation feedback, and the changed regulatory environment we have decided:

  • We will initiate amendments to the Therapeutic Goods Act 1989 which, subject to policy approval and passage of legislation, would support import of overseas substitute medicines if the Australian medicine has been discontinued and cancelled from the ARTG. Currently the Australian medicine must be registered to allow the import of a substitute from overseas. This change would ensure continued supply of substitutes for discontinued medicines without the additional regulatory burden of requiring registration or an annual charge waiver for a medicine that has been discontinued.
  • We will address barriers to registration by creating a web-based information hub to assist sponsors, including those less experienced in registration of prescription medicines that are subject to, or vulnerable to shortage. The hub will feature access to individual pre-submission advice such as early scientific advice. Existing requirements and standards for applications continue to apply.
     
  • In certain circumstances prioritising evaluation of prescription medicine registration applications vulnerable to shortage can prevent or reduce the period of shortage. We will prioritise the start of evaluation for registration applications that are identified to prevent or address a medicine shortage if it is in the interest of public health. Prioritisation will occur following application lodgement on a case-by-case basis, within constraints of existing processes and resources. Existing legislated time frames for evaluation continue to apply to all registration applications.

We asked

We asked for feedback on a proposed standard for nicotine vaping products that have not been assessed by the TGA (unregistered nicotine vaping products). While it is possible that in the future there may be nicotine vaping products that have been assessed and approved by the TGA as medicines, we expect that in the meantime there will be a significant number of prescriptions written for unapproved goods for smoking cessation. We proposed a set of minimum safety and quality requirements for unregistered nicotine vaping products to support health professionals and consumers in knowing what is in the product being prescribed and used and to reduce the risks of accidental poisonings.

In this consultation we also asked for feedback on a proposal to exempt those unregistered nicotine vaping products that meet the requirements in the proposed standard from having to meet certain other default standards in the European Pharmacopoeia and the United States Pharmacopeia. 

You said

We received 103 submissions in response to the consultation, including from research, government, health professional, consumer and industry organisations, as well as from individual consumers. There was broad support for the creation of a standard for unregistered nicotine vaping products. Respondents provided a range of views and suggestions to improve the standard, including:

  • more strict labelling requirements, such as the need for a consistent measure of the concentration of nicotine in the product and clear rules about which label warning statements (e.g. ‘KEEP OUT OF REACH OF CHILDREN’, ‘Avoid contact with eyes’, ‘Avoid contact with skin’)
  • the need for accurate labelling of nicotine concentration or content in the product
  • information about additional ingredients associated with known inhalation harms
  • diverse views on whether the standard should specify limits on nicotine concentration, container volume and flavours
  • a preference that the standard also apply to products that may be listed on the Australian Register of Therapeutic Goods (ARTG) in the future as ‘Export Only’ medicines
  • suggestions about different types of information manufacturers and others should be expected to hold about the products
  • requests for the regulation of vaping devices.

Thank you to everyone who provided feedback and helped in the development of the standard.

We did

In response to this feedback, the standard includes updated labelling requirements for nicotine concentration (including requiring nicotine concentration to be specified in mg/mL and warning statements to be provided), the prohibition of three additional ingredients with established inhalation risks, record keeping requirements for sponsors, a limit of nicotine concentration of 100 mg/mL in products and a requirement for actual nicotine concentration/content of a product to be within +/- 10% of what it says on the label. The standard has also been expanded to cover ARTG-listed ‘Export Only’ products.

Although the standard does not restrict the flavours of nicotine vaping products (other than those containing prohibited ingredients) or container sizes, it is important to remember that these factors, and others, are restricted by the person’s prescription. This allows the prescribing health professional and patient to work together to make sure that the right product is supplied to support nicotine and smoking cessation.

The new Therapeutic Goods (Standard for Nicotine Vaping Products) (TGO 110) Order 2021 was published in May 2021 and comes into effect on 1 October 2021. This standard will apply to all unregistered nicotine vaping products in Australia (including compounded and clinical trial products and ARTG-listed as ‘Export Only’ products).

The Therapeutic Goods (Exempt Monographs) Determination 2021 (“the Determination”) was made at the same time as the standard. The purpose of the Determination is to exempt unregistered nicotine vaping products from certain pharmacopoeial standards that automatically apply to therapeutic goods.

To help explain the new requirements, TGA has published final guidance on the requirements of TGO 110 and other matters related to nicotine vaping products and vaping devices. The final guidance incorporates stakeholder feedback on the draft guidance published in May 2021.

We asked

We asked for your feedback to understand potential obstacles and/or incentives to repurposing of medicines that may influence a sponsor’s decision to extend an indication for an existing medicine – in particular for those indications that are: 

  • already approved overseas 

  • for a less common disease 

  • already accepted clinical practice albeit ‘off-label’ 

  • likely to be less commercially profitable. 

The public consultation proposed options and ideas that may reduce barriers or encourage sponsors to seek registration of new indications. The three key areas are reducing regulatory burden (fee reduction, simplified submissions, and exclusivity); supporting the development of repurposed drugs through improving access to information (off-label use, and consolidated international regulatory information); and actively pursuing new indications (e.g. using non-commercial entities). 

You said

We received 59 submissions in response to the consultation, including from research, government, health professional, consumer and industry organisations, as well as from individual consumers. There was broad support for repurposing of medicines but some issues and obstacles were raised. You said: 

  • Regulatory burden reduction: Respondents were supportive of the TGA providing fee relief for certain repurposing submissions and streamlining simultaneous submission for regulatory and reimbursement evaluation. 

  • Use of real world data: Most respondents welcomed the use of real world data. There were concerns over a lack of guidance documents and information about the type and quality of real world data that the TGA will accept as part of a submission. 

  • Intellectual property rights and limitations: In the event that a medicine has patent protection, the sponsor runs the risk of infringing on another company’s patent. When a medicine is off-patent, there is a lack of incentive for generic companies to apply for a generic extension of indications. 

  • Enabling exclusivity periods: Respondents raised issues over the feasibility of an exclusive period for the new indication of a repurposed medicine. An exclusive period could infringe on the PBS current price disclosure and pricing policies. 

  • Erosion of ‘comparator’ medicine subsidisations: Respondents raised that if a proposed medicine was benchmarked to an existing comparator medicine on the PBS, the price of proposed medicine may be driven down on PBS. 

  • Clinical equipoise: Respondents highlighted that when a medicine has been used off- label for a prolonged period of time, and there is sufficient observational evidence to suggest its effectiveness, it would be difficult to conduct clinical studies that meet TGA’s criteria for evidence of efficacy and safety. In addition, large randomised trials are not feasible or ethical for rare diseases. 

  • Allowing/encouraging a non-commercial entity acting as sponsor: Many respondents discussed the feasibility of non-commercial entities acting as sponsors.1 The main issue lies in which stakeholder is responsible for the financial liabilities and legal responsibilities for the repurposed medicine. 

  • Alignment to company strategy: Respondents raised the potential impact of repurposed medicines on the company’s business strategies and image, particularly if they supply multiple treatment options for an indication. 

  • Compelling a sponsor to register: The vast majority of industry respondents did not support compelling a sponsor to submit an application to add indications. It was clear this was seen as against the principles of a free market. Beyond the efforts involved in an application, it was suggested this would impose additional pharmacovigilance responsibilities on an unwilling sponsor and this could have significant medicolegal, resourcing and financial consequences for the sponsor.  

  • Deeming an indication: Most industry respondents did not support the suggestion of TGA ‘deeming’ an indication without sponsor agreement. There was more support for TGA to do so with consent and agreement, particularly if the noted pharmacovigilance responsibilities could be limited or alleviated. 

Thank you to everyone who provided feedback and helped us understand some of the obstacles and issue you see around repurposing of medicines in Australia  

We did

We will use the feedback to shape regulatory reforms and policy options. It is likely that there will be further consultation as the Department considers the feedback and formulates options. Implementation of some options would require government approval (regulatory change) and/or parliamentary approval (legislative change). 

We asked

Public consultation on the Delegate’s interim decision, in relation to scheduling proposals referred to the November 2020 meetings of the Advisory Committee on Medicines and Chemicals Scheduling, was held between 3 February 2021 and 4 March 2021.

You said

We received 807 submissions in response to the public consultation, including one submission that was received offline. Respondents provided feedback on the proposed changes to the Poisons Standard. We appreciate all of the feedback that we received.

We did

The Delegate’s final decisions on psilocybin and MDMA were deferred on 7 April 2021, pending an independent expert review and additional committee advice. The Delegate will consider all evidence provided, including the public submissions received, ahead of making the final decisions on these substances.

The final decisions for all other substances considered at the November 2020 meetings were published on 22 April 2021 on the TGA website.

We asked

We asked for feedback on a proposal to streamline how we enter information about the formulations of therapeutic goods into TGA electronic systems when an applicant is seeking market approval. To help make the application process more efficient, we proposed to stop entering three categories of formulations into a subordinate database of ingredient mixtures (known as the Proprietary Ingredients, or PI Table) before they are selected into an application onto the Australian Register of Therapeutic Goods (ARTG).

This proposal was limited to ingredient mixtures that contain an active ingredient (known as ‘Active Premixes’ and ‘Active Herbal Extracts’), and non-specific ‘Excipient Mixes’, which do not provide sufficient information on the purpose of the mixture.

Under the proposal there would be no change to how medicine and other therapeutic goods applications are evaluated or the amount of information publicly available about these therapeutic goods.

You said

We received 14 submissions in response to the consultation. The majority of respondents supported the proposal to cease processing new Active Premixes, Active Herbal Extracts and non-specific Excipient Mixes into the PI Table, some stating that it was a sensible removal of redundant administrative processes, provided greater transparency and streamlining of administrative processes, was consistent with red tape reduction, and would reduce regulatory burden. However, there was concern about what expectations there would be for sponsors of existing ARTG entries that already use PI numbers within their formulation, i.e. whether existing ARTG entries would need to be updated and any cost implications. Many respondents also noted that sponsors can experience difficulties in obtaining formulation information from ingredient suppliers.

Some respondents requested a broader review of the administrative PI notification process, noting that there are broader issues associated with the use of PI numbers in formulations of medicines on the ARTG.

We did

We appreciate all the feedback received and considered all responses before making a decision about this activity.

Based on our consideration of the issues raised in this feedback, we have agreed to stop processing notifications for new ingredient mixtures with an active ingredient or a non-specific purpose into the Proprietary Ingredients Table as of 20 July 2021. This means that no new PI numbers will be allocated to these types of formulations. Ingredient suppliers can still sell these mixtures to sponsors intended for use in medicines, but sponsors will need to select the individual ingredients into their application at the same time as they enter the rest of their medicines’ formulation details.

To help maintain the currency of information in our Proprietary Ingredients Table, we will also be inactivating (i.e. ‘hiding’) historic PI numbers for these types of mixtures where the ingredient mixture:

  • is not linked to current ARTG entries, and
  • was entered into the Proprietary Ingredient Table prior to 1 July 2020.

We will write to ingredient suppliers to advise them where we propose to inactivate their PI numbers, as part of our standard process for inactivating historic PI numbers that are not linked to current ARTG entries.

To address concerns raised about what impact this proposal may have on existing ARTG entries that use the affected PI numbers within their formulation we are:

  • Keeping existing PI numbers active in the Proprietary Ingredient table if the ingredient mixture is included in current ARTG entries.
  • Not requiring sponsors to make changes to existing ARTG entries, unless they choose to do so. In this case the sponsor can choose to correct the formulation of their ARTG entry, using existing variation processes.
  • Updating relevant information on TGA website to support sponsors and suppliers in managing the change.

For more information see Streamlining proprietary ingredient categories.

In response to the call for a broader review of the administrative proprietary ingredients system, this activity is not expected to address the wider-scale challenges associated with use of PI numbers in ARTG entries. There are various categories of mixtures within the Proprietary Ingredients Table. The benefits and challenges associated with entering mixtures into the Proprietary Ingredients Table are different depending on the mixture’s purpose and formulation. Consequently, we propose to approach these challenges in a targeted and step-wise manner.