In total, 16 respondents participated in the survey and completed the questionnaire in its entirety. As listed on the TGA website, 42 sponsors had a PR or PA determination approved as of October 2021. Thirteen of the 16 survey respondents in this survey had experience submitting via PR, PA or both pathways, thus making this sample size of 31% (13/42) representative of the industry segment of companies using the FRPs.

The demographic profile and professional background of the participants are summarised in Table 1, including the age (30 to ≥ 60 years old), gender (M/F, 9/7 [56/44%]), workplace (15/16 [94%] in multinational company), company size in Australia (7/16 [44%] with < 100 employees; 6/16 [37%] with 100–250 employees; 3/16 [19%] with 500–1000+ employees), roles (13/16 [81%] as RA Manager or Director), and duration of employment in the Regulatory Affairs field (13/16 [81%] for 10–15 years or longer).

Table 1 Demographics and professional background of the survey participants

As indicated, all respondents were highly experienced, senior Regulatory Affairs professionals. All 16 respondents had experience submitting via the standard pathway, nine had experience with PR, ten with PA, and three respondents had standard pathway experience only.

Fifteen out of 16 participants 15/16 (93.7%) were working for multinational companies with the number of employees varying from under 100 to over 1000 in Australia, with portfolios covering a wide range of therapeutic areas, including alimentary tract and metabolism, blood and blood forming organs, cardiovascular system, dermatological, genitourinary system and sex hormones, anti-infectives for systemic use, anti-neoplastic and immunomodulating agents, musculoskeletal system, nervous system, respiratory system, or sensory organs. The companies’ portfolios included new chemical entities (NCE), new biological entities, gene therapy and in vitro diagnostics.

All respondents indicated excellent awareness of the Australian regulatory reforms and the MMDR, including the expedited registration pathways (PR, PA) and collaborative arrangements with overseas regulatory authorities, such as Project Orbis (which included the Regulatory Agencies of Australia, United States of America, Canada, Singapore, Switzerland, Brazil, United Kingdom, and Israel), the Access Consortium (Australia, Canada, Singapore, Switzerland, United Kingdom), and Comparable Overseas Regulator reliance pathways (COR)-A and COR-B. Two participants were not aware of the Project Orbis registration option (in one of the two instances, the company was not developing oncology products). Additionally, one participant reported being involved in the development of the reform guidance through TGA working groups.

3.1 Overall Satisfaction with the Pathways

The highest satisfaction was observed towards the PR pathway with 8/9 (89%) respondents indicating positive satisfaction (from extremely satisfied to slightly satisfied), whereas only 1/9 (11%) indicated a slightly dissatisfied experience. In comparison, 11/16 (69%) and 6/10 (60%) respondents showed a positive level of satisfaction with the standard and the PA pathways, respectively (Fig. 1). For PA, the responses were somewhat ambivalent, with 6/10 (60%) respondents expressing satisfaction with the pathway, 1/10 (10%) neutral, 2/10 (20%) slightly unsatisfied and 1/10 (10%) extremely unsatisfied, so 30% of respondents indicated a dissatisfied experience with the use of the PA pathway. By contrast, only 13% expressed dissatisfaction with the standard pathway. Free-text comments were provided by two respondents, who stated that the length of time taken for approval using the standard pathway remains a challenge and could be subject to improvement.

Fig. 1

Overall satisfaction with pathways (left) and ease of use of the pathways (right). PR priority review pathway, PA provisional approval pathway

3.2 Ease of Use

Most respondents found the standard and PR pathways relatively easy to use, with 12/16 (75%) and 7/9 (78%), respectively, indicating a positive level of ease of use, followed by 3/16 (19%) and 1/9 (11%) being neutral, with 1/16 (6%) and 1/9 (11%) being negative. However, the perception of the respondents was less favourable towards the PA pathway. Only 3/10 (30%) were positive whereas 6/10 (60%) were neutral, that is, neither easy nor difficult to utilise, and 1/10 (10%) was negative on this parameter (Fig. 1). A free-text comment was provided by one respondent indicating that the current execution of the standard pathway activities by the Regulator resulted in increased unpredictability.

3.3 Availability and Usefulness of the TGA’s Instructions and Guidelines

Overall, the respondents perceived that there were more guidelines available for the standard pathway than for the PR or PA pathways. For the PR and PA pathways, fewer participants, 4/9 (44%) and 6/10 (60%) respectively, indicated a high amount of guidance, followed by 44% and 40% who indicated a moderate amount only. One respondent stated that only a modest amount of guidance was available for the PR pathway (Fig. 2). In comparison, 14/16 (88%) respondents indicated a high amount (‘a great deal’ or ‘a lot’) of guidance available for the standard pathway, followed by 2/16 (13%) respondents who considered the amount of available guidance was moderate. Most of the respondents found the guidelines for all pathways useful, with 94%, 89% and 80% indicating a positive level of usefulness for navigating the standard, PR or PA pathways, respectively. One participant provided a free-text comment with positive feedback that guidelines and instructions for the standard pathway have improved over the years, becoming more specific and providing steps to navigate the process.

Fig. 2

Availability of instructions and guidelines. PR priority review pathway, PA provisional approval pathway

3.4 Level of Regulatory Burden Experienced in the Use of Regulatory Pathways

Overall, higher regulatory burden was perceived in PR and PA, with 3/9 (33%) and 5/10 (50%) participants, respectively, regarding the burden as high (‘a great deal’ or ‘a lot’) compared with 3/16 (19%) respondents for the standard pathway. For the PR pathway, 4/9 (44%) considered the regulatory burden moderate, compared with 2/10 (20%) for the PA pathway. For the standard pathway, most participants 10/16 (63%) indicated a moderate level of regulatory burden (Table 2). The respondents’ perceptions of regulatory burden with using the PA pathway were ambivalent with opinions ranging from high to no burden. Two factors were noted to contribute to the higher regulatory burden seen in using the PA pathway: the necessity to submit a registration application via the standard pathway if the later stage (typically phase III) data is positive and the necessity to maintain the TGA provisional registration even where the product is not marketed, for instance due to inability to secure reimbursement based on partial clinical data. The higher level of regulatory burden associated with the PR pathway was attributed by one participant from a large multinational company to some unique Australian requirements of the priority review designation. For the standard pathway, the timelines in the later stages of review were noted by one participant from a mid-sized multinational company to be tight and potentially difficult to manage.

Table 2 Level of regulatory burden in the use of a pathway3.5 Regulator's Level of Support and Clarity of Criteria

In this study, most participants considered the level of support from the Regulator adequate across all three pathways, that is, neither too much nor too little (81% for the standard pathway, 67% for the PR and 90% for the PA pathway). This might be attributable to the regulatory authorisation of many therapeutic products in the European Union (EU) or US prior to their registration in Australia. Therefore, the early enhanced regulator involvement occurs in these jurisdictions, making TGA’s level of involvement at later stages sufficient. In Australia, engagement and dialogue between the applicant and Regulator prior to submission for registration is facilitated via pre-submission meetings with the TGA. Such meetings aim to develop a common understanding of the proposed prescription medicine, clarify supporting documentation requirements and help plan the submission activities and resourcing. The TGA provides advice on existing studies, data sets or the proposed dossier content and format. However, unlike the FDA or EMA, the TGA does not engage in enhanced early dialogue at the product development stage or offer scientific advice or protocol assistance. Most respondents indicated that the determination criteria for the FRPs are generally clear: 8/9 (89%) for PR determination, and 9/10 (90%) for PA determination.

3.6 Impact of the Pathway on the Timeliness of TGA Approval

All respondents perceived that the PR pathway was effective in accelerating the approval process for eligible prescription medicines. Fifty-six percent of respondents (5/9) thought the PR pathway extremely expedited the registration timelines while 33% (3/9) thought it did so moderately (Fig. 3). One respondent provided a free-text comment on this parameter based on their experience before and after the COVID-19 pandemic. The PR pathway could be classified as extremely expedited as TGA were delivering an outcome in approximately half the time of the standard pathway prior to COVID-19. However, during the pandemic the TGA was adhering to the milestone plan which only offered a more moderate acceleration (3 months) compared with the standard pathway.

Fig. 3

Timeliness of approval for provisional approval (left) and priority review (right) pathways

The responses for the PA pathway were more ambivalent, with 6/10 (60%) respondents stating that the timelines were expedited. On the contrary, 40% (4/10) of respondents indicated the pathway did not offer an advantage over the standard pathway, and one (10%) participant felt it was even slightly slowing the process down (Fig. 3). Two respondents noted that the legislated timelines using the PA pathway were identical to those for the standard registration pathway.

3.7 Impact on Submission Strategy of Companies

All respondents reported that the introduction of the new pathways in Australia had affected their companies’ submission strategies, with over 90% (15/16) indicating they had used one or both FRPs (Fig. 4). Fourteen respondents (88%) indicated that their companies planned to use or continue the use of the TGA’s FRPs in the future.

Fig. 4

Impact of FRP implementation on company (left) and extent of use (right). PR priority review pathway, PA provisional approval pathway

Most participants reported a positive impact on their company from the introduction of these new pathways: 7/9 (78%) for PR and 7/10 (70%) for PA. Approximately one-fifth of the respondents in each pathway thought it made neither a positive nor negative impact on their company. One of the respondents who indicated an extremely positive impact commented this was due to their company being able to supply urgent medicines for the current pandemic. This may also be the case for other respondents who indicated a positive impact, as there was a dramatic increase in COVID-19-related products registered via the provisional pathway in 2021 [20]. One respondent indicated that the PA pathway provided no advantage in terms of market access or availability to patients and lacked commercial viability due to the inability to use the TGA provisional approval to secure reimbursement. It was noted that using the PA pathway offers only a modest Intellectual Property (IP) advantage, as the data exclusivity period of a specific product commences upon TGA’s provisional approval and would typically be approaching expiration by the time a full approval is achieved.

Just over half of the respondents (8/15 [53%]) indicated that they always, or actively, looked for opportunities to use either of the FRPs to obtain registration of new products and new indications, as they considered the best strategy was to maximise the speed to market and access for patients. One respondent stated that eligibility for FRPs has become a strategic decision-making process. Another participant stated that every new asset or indication was assessed for potential against the new pathways, which were then utilised where eligibility criteria were met and the company was willing to expend the required internal resource to make use of these pathways. On the contrary, 2/8 (25%) respondents indicated their company’s experience with the PA pathway was negative; in one case the Regulatory Affairs department generally advises against the use of the PA pathway.

Eight out of fifteen (53%) respondents indicated that the use of FRPs resulted in shorter review timelines, which affected their submission strategy. The PR pathway was sought where possible, particularly where it leads to an earlier Pharmaceutical Benefits Advisory Committee (PBAC) cycle of review for potential reimbursement. One participant stressed specifically the value of the TGA’s Comparable Oversees Regulator (COR)-A process, which had been used multiple times with the fastest approval of a new indication within 5 months. The FRPs were reported by respondents to provide more choices and options for global regulatory collaboration via the Access consortium and Project Orbis initiatives. They also resulted in increased planning, including strategies to launch, and allowed Australian submissions to be prioritised due to reduced resource burden needed to support applications.

3.8 Further Improvements

We received several suggestions for further improvements to the PR and PA pathways (Table 3). Improvements proposed for the PR pathway were classified into those related to the determination step, and those pertaining to the evaluation process. The improvements proposed for the PA pathway indicate its usefulness and desirability are reduced due to the lack of viable reimbursement options since the HTA bodies are reluctant to accept positive review outcomes due to higher uncertainty associated with these expedited pathways. Multiple respondents proposed modifications to the delivery of post-approval commitments and provided suggestions for shortening review timelines.

Table 3 Priority review and provisional approval pathways: proposed improvements