The Pharmaceutical Benefits Advisory Committee (PBAC) has rejected Lilly's application for PBS listing of donanemab (Kisunla), marking a significant setback for Alzheimer's disease treatment access in Australia. The decision, announced following the July 2025 PBAC meeting, means patients seeking this breakthrough therapy will need to pay approximately $4,700 per infusion out of pocket.
Clinical Evidence and PBAC Concerns
The PBAC acknowledged "a high clinical need for effective disease modifying treatments for Alzheimer's disease" and noted that existing treatments have "only modest effectiveness." However, the committee expressed concerns about the clinical significance of donanemab's benefits.
Clinical trials demonstrated that donanemab can potentially delay the progression of early Alzheimer's disease by around six weeks over 18 months. The drug showed a 35% reduction in clinical decline compared to placebo and resulted in 40% less decline in the ability to perform daily activities. Despite these results, the PBAC noted "a lack of consensus among clinicians over whether these results would translate into meaningful improvements for patients."
The committee concluded that "the potential benefits too small and uncertain to justify the burden of this treatment on both patients and the health system."
Treatment Requirements and Patient Burden
Donanemab represents the first disease-modifying therapy for Alzheimer's disease registered in Australia in 25 years. The treatment works by helping immune cells clear amyloid plaques from the brain, addressing the underlying cause of the neurodegenerative disease.
To be eligible for treatment, patients must have early symptoms of Alzheimer's disease, a specific genetic profile, and confirmed presence of amyloid plaque in the brain. The therapy requires monthly infusions over 18 months, with magnetic resonance imaging (MRI) scans before and during treatment to monitor for brain bleeding and swelling.
The PBAC considered the specialist diagnostic testing required to determine patient suitability "too great a burden, especially for those who did not ultimately qualify for it."
Industry and Clinical Response
Tori Brown, General Manager of Lilly Australia & NZ, expressed disappointment with the decision, stating it "doesn't reflect the true value of Kisunla for patients, their families, the health system and society as a whole." Brown emphasized that the negative outcome "must prompt a national conversation about the importance of investing more in innovative medicines for our rapidly ageing population."
Dr. Marita Long, the first GP appointed as an Honorary Medical Advisor for Dementia Australia and Clinical Associate Professor for the Wicking Dementia Education and Research Centre, provided clinical perspective on the decision. While understanding the disappointment felt by patients and families, she noted that "the studies only go for 18 months, and so we probably are looking for a little bit more real-life data on how this pans out."
Access and Equity Implications
The rejection creates significant equity concerns, as patients meeting the criteria can only access donanemab through private prescription. Dr. Long highlighted this disparity: "So, people who have got the capacity to access this drug can, and people who don't can't. That's where it becomes very difficult, particularly for us in primary care where we have to share that disappointment with our patients."
The cost barrier is substantial, with treatment requiring approximately $4,700 per infusion every four weeks over 18 months, making it prohibitive for most patients.
Future Prospects
Despite the setback, clinical experts remain optimistic about future developments. Dr. Long noted that "there are still trials going on, so for people who can't afford it, there is still the option of trials and there are other disease-modifying drugs that are being looked at as well."
She suggested that Lilly will likely reapply once more real-world data becomes available, particularly from American experience where the drug has broader access.
Alzheimer's disease represents the leading cause of dementia in Australia, accounting for approximately two-thirds of cases. The manufacturer has confirmed it is working "to fully understand the implications" of the PBAC's decision and potential next steps.
Broader PBAC Decisions
The donanemab rejection was part of broader PBAC outcomes that also saw MSD's application for pan-tumour funding of pembrolizumab (Keytruda) rejected after eight years of consideration. However, the committee did approve several other treatments, including abaloparatide for osteoporosis, guselkumab for severe Crohn's disease, and tacrolimus ointment for atopic dermatitis.
