(Cth) Exempt Orphan Drugs from All PBAC Submission Fees

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Author: Devathri Nanayakkara, Emmaby Barton Grace & Jessica Luu | Publish date: 19/3/2026

• P: A fee waiver only applies to the first submission for an orphan drug (OD) to be listed on the Pharmaceutical Benefits Scheme (PBS), with manufacturers needing to pay fees for all subsequent submissions.

• S: The Minister for Health should amend Section 67 of the National Health (Pharmaceuticals and Vaccines—Cost Recovery) Regulations 2022, made under the National Health Act 1953 (Cth), to exempt ODs from all PBS submission fees.

Problem Identification: 

Section 67 of the National Health (Pharmaceuticals and Vaccines—Cost Recovery) Regulations 2022, made under the National Health Act 1953 (Cth), states that ‘no fee is payable’ for medicines designated as ODs for the first Pharmaceutical Benefits Advisory Committee (PBAC) submission. Section 67 further states that this exemption applies to designated orphan drugs that are either under approval to be included in the Australian Register of Therapeutic Goods (ARTG) or were included in the ARTG in the previous 12 months.

Thus, designated orphan drugs are eligible for ‘a waiver of [the] application and evaluation fee’ associated with the first submission to PBAC. According to Medicines Australia, this means that subsequent PBAC resubmissions require fee payments. They elaborated that this results in higher financial costs for the manufacturer. They have argued that this ‘may have a negative impact on the financial viability … resulting in medicines intended to treat conditions with the highest unmet need … not brought to market.’ OD manufacturers have reported that this can then limit access to treatment for rare disease (RD) patients.

Context: 

Orphan drugs (ODs) refer to ‘a medicine, vaccine, or in vivo diagnostic agent … [that] must be intended to treat, prevent or diagnose [an RD]’. RDs refer to medical conditions affecting fewer than 5 in 10,000 people. According to the Department of Health, this ‘equates to around two million’, or 8% of Australians. Manufacturers can apply to the Therapeutic Goods Association (TGA) for their drug to be designated as an OD. RVA emphasised that ODs are ‘essential’ for ‘improving quality of life and extending life expectancy’.

A PBAC submission refers to an application submitted by a new medicine or medicinal product manufacturer to PBAC to obtain a listing on the PBS or Life Saving Drug Program (LSDP). When medicines are listed on the PBS or LSDP, they are subsidised by the government.

According to the Rare Disease Industry Working Group (RDIWG), RD submissions ‘are rarely recommended following the first [PBAC] submission.’ Specialised Therapeutics Australia (STA) stated that ‘it typically takes two to three submissions for a drug to receive a positive PBAC approval.’ A review by IQVIA, a company focusing on health research and technology, revealed that ‘87% of drugs were not able to take … advantage of the fee waiver’ as they required resubmissions. RD manufacturers stated that ‘the real costs of a successful submission sit between $500 000 to $1 million dollars.’ 

Arguments:

The RDIWG reported that ‘cost-prohibitive’ resubmission fees do not ‘[incentivise manufacturers] to research, develop and commercialise new [ODs].’ The STA noted that ‘to [their] knowledge … [Australia] incur[s] the highest application and administrative fees in the world.’ Rare Voices Australia (RVA) identified that ‘due to small patient numbers, it is not always commercially viable for companies to seek reimbursement for [an RD] indication.’ RVA explained that these fees may therefore delay or prevent access to ODs, stopping ‘Australians living with [an RD from] experienc[ing] the best health and wellbeing outcomes’.

According to STA, it ‘is not an acceptable outcome’ that ‘access to [ODs] is at best delayed, and often completely unavailable.’ RVA noted that this delay contributes to ‘the burden of [RDs] remain[ing] unacceptably high’ in Australia. RVA further explained that ‘as many [RDs] are progressive, time is often critical, making timely and equitable reimbursement [of ODs] essential’ for RD patients. 

According to health and medical researchers Adachi et al. (2023), ‘affordability of drugs to treat RDs is one of the major issues’ contributing to ‘greater inequities’ for RD patients and families. According to RVA, ‘without government reimbursement, many [ODs] are unaffordable’. IQVIA reported that between January 2018 and June 2023, only 38% of designated ODs were PBS-listed. According to the Noonan Syndrome Awareness Association, ‘45% of parents [of children with an RD] ... are not coping financially with the demands of their child’s disease with payment for medical care being the number one concern.’ Further, Adachi el al. (2023) observed that ‘there are cases where medicines that can be used to treat an RD are only subsidi[s]ed under [PBS] for a common disease, meaning RD patients must pay more for the same medicine.’ For example, Rare Cancers Australia reported that patients may pay up to $10,000 per round of medicine for a rare cancer. However, under the PBS, a patient would not pay more than $25 for the same medicine.

Advice/Solution Identification:

Medicines Australia and Dr Falk Pharma Australia have called for ODs to be exempted from all PBS submission fees. Medicines Australia has said that this could ‘help mitigate [the] risk’ of ODs not being accessible to patients due to companies not being able to afford submission fees. 

Precedent:

There is international precedent for exempting ODs from regulatory fees. The Czech Republic has waived administrative, regulatory, and scientific fees for ODs.

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Public Support:

1. Medicines Australia

2. Dr Falk Pharma Australia

Broad Support: 

1. Specialised Therapeutics Australia (STA) (STA called for fee waivers to be extended beyond the current 12-month timeframe, and for smaller companies (with revenues of less than $50 million annually) to be exempted from paying fees upfront for at least two applications (with fees only being paid if PBS expenses exceed $3 million annually.)

2. Rare Disease Industry Working Group (RDIWG) (RDIWG called for timeframes for fee exemptions to be extended and for fees to be considered based on manufacturer budgets.) 

3. Recordati Rare Diseases Australia (RRDA) (RRDA called for fees to be waived for the first 2-3 submissions.)

4. Amicus Therapeutics (Amicus Therapeutics called for fee waivers to be extended from 12 months to 5 years.)

This list reflects publicly stated positions and should not necessarily be taken as endorsement of this specific brief.

News Coverage:

• ABC News - “Care for people with rare and less common cancers ‘delayed and fragmented’, new report reveals”. This article discussed the high costs of and difficulty obtaining life-saving medication for rare cancers. By: Stephanie Dalzell and Paige Cockburn | Mon 1 Sep 2025 - Read the article here.

• McKell Institute - “NSW Government urged to develop rare disease strategy”. This article discussed how a new report by the McKell Institute found that an RD strategy is urgently needed in NSW as more than 600,000 people continue to face fragmented and inconsistent care. By: McKell Institute | Wed 3 Dec 2025 - Read the article here.

• ABC News - “Rare cancer patients push for better medication access, say current PBS framework is unfair”. In this article, ABC News interviewed a patient with a rare cancer who is unable to receive life-saving medication as it is not available for his condition under the PBS. By: ABC News | Tue 18 Oct 2016 - Read the article here.

Where to go to learn more: 

1. (2021) The New Frontier - Delivering Better Health for All Australians | House of Representatives Standing Committee on Health, Aged Care and Sport - This report was written as part of the Inquiry into approval processes for new drugs and novel medical technologies in Australia. Section 8 provided an overview of submission responses regarding RDs, including the cost of OD submissions. Read the full report here.

   • (2021) House of Representatives Standing Committee on Health, Aged Care and Sport - This page provides links to all of the submissions received by the Committee, including those from STA and RVA. See all of the submissions here.

   • (2020) Specialised Therapeutics Australia - This submission provided a thorough overview of STA’s concerns and recommendations regarding the approval process for new drugs for RDs in Australia. Read the full submission here.

   • (2020) Rare Voices Australia - This submission explained why medicines may be available under the PBS for common conditions, but the same medicine may not be available under the PBS for RDs. Read the full submission here.

2. (2020) National Strategic Action Plan for Rare Diseases | Australian Government Department of Health and Rare Voices Australia - This Action Plan provided guidance and direction around key goals and priorities for Australians living with an RD to improve health and wellbeing outcomes. It also provided an overview of the current Australian and international context. Read the full Action Plan here. 

3. (2021) Funding Rare Disease Therapies in Australia - Ensuring Equitable Access to Health Care for All Australians | McKell Institute - This report provided an overview of how rare disease therapies are funded in Australia. It included a summary of current mechanisms such as the PBS, LDSP and OD designation, as well as recommendations for improving RD funding, and comparisons of what other countries are doing. Read the full report here.

4. (2023) Access to Orphan Drugs in Australia | IQVIA - This report provided an overview and analysis of the Orphan Drug program in Australia. It provided key statistics about the submission process. Read the full report here. 

5. (2025) Therapeutic Goods Administration - This page provides an overview of how to apply for a prescription medicine via OD designation, including information about the eligibility criteria and each step in the submission process. Read the full page here.

6. (2025) The Pharmaceutical Benefits Scheme - This page provides an explanation of cost recovery fees for PBS listings, which are the fees manufacturers are required to pay for resubmissions for ODs. Read the explanation here.

7. (2022) Huang-Ku, E & Laba, T - This article provided an overview of the Life Saving Drug Program, a scheme that complements the PBS to increase access to high-cost drugs for RDs. Read the article here.

8. National Health (Pharmaceuticals and Vaccines—Cost Recovery) Regulations 2022 (Cth) - Read the Regulations here.

Human Perspective: 

Trigger Warning: Childhood illness

Emily, a ten-year-old with adrenocortical carcinoma, a rare cancer, used to count time by school terms and birthdays, but lately she measured it in waiting rooms and pills swallowed. For her family, hope had arrived as a newly developed orphan drug, one her doctor said was already helping children overseas. Emily didn’t understand why it couldn’t help her as well. She only knew that it had been sent to important people for assessment, but it had come back with a ‘no’. Now, every time her parents and doctors talked about it being sent again, their voices grew heavier. She didn’t understand all the words the adults used – submission, review, resubmission, subsidies, rebate, loans – only that the medicine her doctor and family once spoke about with cautious excitement kept slipping further away. As months blurred into years, Emily’s body grew weaker and school days disappeared. She noticed how her mum’s smile tightened whenever official letters about treatment and bills arrived, how her dad stayed up late at the kitchen table, quietly doing sums that never seemed to add up. Emily felt a dull, aching frustration – it didn’t make sense that something made for children like her existed somewhere else, but not here, not when she needed it most. Over time, she stopped asking when the medicine would come. Instead, she learned how to live with waiting, even as she watched the idea of treatment remain just that – an idea – while her childhood slowly shrank around her.

To protect the anonymity of those involved, this is a fictionalised account drawn from an amalgamation of real-life stories, experiences and testimonials gathered during the research process for this brief. Any resemblance to actual individuals is purely coincidental.

Conflict of interest/acknowledgment statement: 

N/A

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Reference list: 

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Basu, A., Thomas, S. K., Chapman, R. H., & Spangler, J. (2025). HTA evidence in rare diseases: Just rare or also special? PharmacoEconomics 43(11), 1271-1279. https://doi.org/10.1007/s40273-025-01538-4 

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Department of Health, Disability and Ageing. (2026, January 1). About the PBS. Australian Government. https://www.pbs.gov.au/info/about-the-pbs 

Department of Health, Disability and Ageing. (2026, January 1). Pharmaceutical benefits: Fees, patient contributions and safety net thresholds. Australian Government. https://www.pbs.gov.au/info/healthpro/explanatory-notes/front/fee 

Department of Health, Disability and Ageing. (2026, January 30). About the Life Saving Drugs Program. Australian Government. https://www.health.gov.au/our-work/life-saving-drugs-program/about-the-lsdp?language=en 

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House of Representatives Standing Committee on Health, Aged Care and Sport. (2021, November). The new frontier - Delivering better health for all Australians: 8. Rare diseases. Parliament of Australia. https://www.aph.gov.au/Parliamentary_Business/Committees/House/Former_Committees/Health_Aged_Care_and_Sport/Newdrugs/Report/section?id=committees%2freportrep%2f024755%2f77601 

IQVIA. (2023). Access to orphan drugs in Australia [White paper]. https://www.iqvia.com/-/media/iqvia/pdfs/australia-and-new-zealand/white-papers/access-to-orphan-drugs-in-australia.pdf

Medicines Australia. (2022). HTA and rare diseases. https://www.medicinesaustralia.com.au/wp-content/uploads/sites/65/2022/11/HTA-DP-Rare-Disease.pdf

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