The PBS Update April 2026: Expanded Indications + New Rare Cancer Options

Proudly Produced by The Oncology Network

Professor Craig Underhill and Rachael Babin break down the key oncology changes from the April 2026 PBS update.

This month we run through six fresh PBS listings and policy changes that affect Australian oncology and haematology practice, from ROS1-positive non small cell lung cancer to perioperative immunotherapy and late-line blood cancer options. 

We focus on who benefits, what the key trial signals are and what clinicians need to monitor so access translates into safe, real-world outcomes. 

• Repotrectinib listed for ROS1-positive NSCLC, including activity after prior TKIs and in resistant mutations, plus monitoring for dizziness and pneumonitis 
• Durvalumab listed for muscle-invasive bladder cancer using the Niagara perioperative regimen, balancing survival gains with immune-related toxicity 
• Three new pembrolizumab listings across cervical cancer, adjuvant renal cell carcinoma and perioperative head and neck cancer, with discussion of endpoints and eligibility 
• Haematology additions including a BCMA bispecific for relapsed myeloma, an oral kinase inhibitor for higher-risk myelofibrosis and mogamulizumab for cutaneous T-cell lymphoma 
• PBS price cuts via generics and biosimilars, plus expanded supervised prescribing for some medicines by nurse practitioners

Follow The PBS Update for regular discussions of PBS listings and oncology policy changes affecting Australian healthcare professionals.

Visit the Show Notes for links to the updates discussed in this episode and to send us audio feedback or questions for future episodes.

Transcript

Welcome And Series Purpose

SPEAKER_01 0:00

Hello and welcome to PBS Updates, a new short series from the Oncology Podcast where we break down the latest changes to Australia's pharmaceutical benefits scheme and what they mean for oncology practice. This is Rachel Batten from the Oncology Network and I'm joined by Professor Craig Underhill. In this series we take a quick look at the recent oncology and hematology updates, what's changed, which patients may benefit, and what clinicians should know. For more detail and ongoing coverage of cancer medicines, you can also visit oncolynetwork.com.au. So if you want a fast practical overview of the latest PBS developments, you're in the right place. Let's get into it.

SPEAKER_00 0:46

G'day, g'day, g'day. Welcome to PBS Update Episode 2. Rachel, I heard we've actually had some good feedback from the first episode, which is amazing.

SPEAKER_01 1:00

Yes. Nice to be back with you. It is nice.

SPEAKER_00 1:04

Nice to be here with you. And not those people from the other side of the ditch telling us about how poor they are in New Zealand and how they can't have these drugs. So it's actually nice to have a segment just on uh the PBS change in Australia.

SPEAKER_01 1:20

We're very blessed, aren't we, with the uh PBS system that we have in Australia, and that's a very nice, nice reminder before we get into the details. So thank you. And yes, we've had some great feedback. So I'm glad the listeners have been enjoying this new series. So this was episode two, and we promise we will try to keep them short, like the first episode, because we think that that probably helps everybody to listen whilst they're on the go.

SPEAKER_00 1:49

Well, I was amazed that it's only a month later, and we've actually got six new listings to talk about. It just shows how far the whole field's progressing. It's great to see these new listings with innovative molecules.

SPEAKER_01 2:03

I I agree. You know, remember we were uh originally planning this series, we thought, oh, maybe one every two months. But there's been so much, and I think it is a reflection on how things are changing.

SPEAKER_00 2:14

And Rachel, do you I think you were going to mention the Industry Spotlight?

Repotrectinib For ROS1 Lung Cancer

SPEAKER_01 2:18

Just a a quick reminder. So we will include all the papers and press releases and any documentation related to the episode in the show notes. But the Industry Spotlight section on our website has all of our cancer drug news covering the PBS, but also what's happening, FDA updates and updates in general. So that's your go-to spot for information in this space. There's plenty to talk about, so let's get stuck into it.

SPEAKER_00 2:47

Yeah, well, the first one we want to talk about is a drug called repotrechtinib. So this is an oral tyrosine kinase inhibitor, and it's been approved in Australia to treat ROS1 positive non-smell lung cancer, and it also has activities in MTRK positive tumours. It is a next generation compact macrocyclic TKI that inhibits the ROS1 and TRK family proteins. And it's also notable through clinical activity and subsequently clinical activity to be efficacious in tumours that have some resistance to first-line TKIs like chrysotinib or ntrectinib. So this listing was based on the results of the Trident 1 study. Big shout out to Melbourne's own Den Solomon, who is one of the authors in this New England paper from January 2024. So Trident One study was done, it was a phase one and two study done in two cohorts. One was patients who had not previously received a Rosamond TKI, and another cohort in patients who had previously received TKI. So in total, there was 520 patients enrolled in this study. It took five years to enroll that many. This is a kind of 1 to 2% of lung cancer, these ROS-1 positive non-small cell lung cancers. And the top line results were a response rate of approximately 80% in patients who had not previously received ROS-1 TKI, duration response 34 months, progression-free survival 35 months. And in those who had previously received one ROS1 TKI not received chemotherapy, the response rate was 38% median duration of response 14.8 months, progression-free survival nine months. There were 17 patients with a specific ROS-1 G2032R mutation, which confers resistance to the first-line drugs. 10 of 17 or 59% responded. So of the 426 patients who received the phase two dose in the study, the most common side effect in about 60% was dizziness. Although that was quite a high rate, the rate of grade three toxicity was low. But the adverse event of particular interest is newinitis. And so patients need to be monitored for that. And if while other side effects can be managed with a pause and then restarting at a lower dose, pneumonitis was a reason to take people off study. So an active drug in this population, and the conclusions were that this was another treatment option for these patients with a rare cancer.

SPEAKER_01 5:55

And it is a quite a small group of people that we're talking about.

SPEAKER_00 5:59

Yeah, I'm just trying to do the maths in my head, Rachel. It's a one or two percent of lung cancer, but there's tens of thousands of patients a year with lung cancer. So I'm not sure overall what the expectation was from the PBAC or PBS for the number of people that would benefit per year in it. And now there's, you know, this is just one of the treatment options for them.

SPEAKER_01 6:21

Well, it's great. And yes, congratulations to Ben Solomon. Always nice when we see Aussie names on these big international studies.

SPEAKER_00 6:29

And it really highlights the great work that Toga and other groups have done for all patients diagnosed with lung cancer, be it early or late, should again receive the next generation sequencing testing to look for rare mutations. And then if a target's identified, if a drug's available, then that becomes a treatment option for the patient, either upfront or down the track.

SPEAKER_01 6:53

It seems to be the theme of most of conversations that I'm having at the moment. I think the the case for the next generation sequencing for all patients ideally is being made and the nature of oncology practice is shifting so remarkably. I think it's it's a fascinating time.

SPEAKER_00 7:17

Ooh, tea about a recent recent paper by Omic Co. Um talking about the benefit of the testing. So we'll get to that.

SPEAKER_01 7:28

Oh well, I'm I'm I'm looking forward to that. And in the meantime, I think we've seen some changes with develop.

SPEAKER_00 7:36

We have seen some changes with develop. So it's again got another listing. This is time for patients with muscle invasive platicancer on the back of the Niagara regimen, which is giving uh four doses of deverlimab as a neoadjuvant treatment, followed by doses eight cycles afterwards. The neoadjuvant devollimabs combined with neoadjuvant gem cytobine and cisplatin in the study, um, but then the eight cycles post-opis deverlimab on its own. So this was first presented at ESMO 2024. We'll put a link to the paper. Event-free survival has shown hazard ratio 0.68. So at 24 months, 67% of patients are alive without relapse and 59.8 in the comparator arm. Overall survival at 24 months, 82% versus 75, hazard ratio 0.75. So in terms of you know neoadjuvant-adjuvant approach, this is a 7% uh difference in survival at 24 months. Most patients would accept the extra toxicity, which is uh, as we all know now with the immunotherapy drugs, uh can be uh not insubstantial and life-changing and needs to be uh managed um by a multidisciplinary team. But uh on the basis of a study, this drug is now available uh for this group of patients.

SPEAKER_01 9:10

Excellent. Okay. And new indications for Pembroke as well, I believe.

Pembrolizumab Expands Across Three Cancers

SPEAKER_00 9:15

Yeah. So the the Pembroke people, of course, have been a bit disappointed because they haven't got a trim reagnostic listing like the Volume Ab and Hippie does. But I understand following the listing of these drugs that there's still ongoing um discussion about some kind of listing. But three new listings for Pembroke, overall about 10,000 patients a year expected to benefit. So one is in high-risk locally advanced cervix cancer, the other renal cell carcinoma and patients at intermediate or high risk of recurrence following surgery as an adjuvant treatment, and locally advanced head and neck squamous cell carcinoma. And so again, we just can mention the the papers that back this. The um listing in high risk, likely advanced cervix cancer uh was in chemo A18 study. So it's giving Pembroke and chemo radiotherapy followed by Pembro and showing a survival advantage in this group of patients. In Australia, this has become a pretty rare cancer, but this is now a standard approach in those patients. In clear cell renal cell carcinoma and high-risk patients, this listing is based on keynote 564. So, in that study, patients received typically for one year doses of pembulismab if they're condemned as high risk for recurrence following the anaphrectomy. In the locally advanced head and neck squamous or carcinoma listing. This is based on keynote 689. Again, this is a perioperative approach, like we just heard about devoluum ab in muscle invasive body cancer. Again, we talked about recurring themes, Rachel. This perioperative approach with doses pre and post-OP seems to be perhaps a more effective approach than um standard adjuvant treatment. So this keynote 689 showed hazard ratios 0.73 with an event-free survival of 51 versus 30 months. Interestingly, there's no overall survival data. So this drug's being listed on the basis of event-free survival. Christopher Jackson would be horrified. But just to note that the parallel sort of study with the volume out called Nevo Post OP, which was giving just post-operative treatment, didn't show the same advantage and hasn't been listed as yet as a treatment strategy. So again, maybe suggesting that this perioperative approach is the way to go. So giving doses of immunotherapy when someone's immune system is still intact.

SPEAKER_01 12:07

Yeah, it's a it's a clever approach, isn't it? It's very nuanced in that respect. And if we see this shift occurring, I think the comments by the health minister were were quite incredible on this one. I don't know if you saw that. He said that this is one of the most extraordinary drugs available in the country. Quite incredible commentary, really, from the minister, highlighting that 10,000 people per year are expected to be affected.

SPEAKER_00 12:36

There was previously 18 different listings. This is an additional three, so 21 different listings. So you can understand why there's a push to maybe get a tumor agnostic listing for this drug as well.

SPEAKER_01 12:48

And so, hematology. There's a few new things as well.

SPEAKER_00 12:51

There is a few new things. So three drugs listed in hematology. The first one, this is where we're going to stumble on the names. LRENATONAB for patients with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy. So, you know, relapsed multiple myeloma used to have such a dismal prognosis. And the study behind this drug, uh, which is a bi-specific V cell maturation anti-child BCMA directed C D3 T cell engager, um, showed quite considerable activity in patients that had three previous lines of treatment with a response rate of about 60% and a median progression-free survival of 17 months. This is fourth line people surviving 17 months on average, progression-free survival 17 months. That probably exceeded the one to two years prognosis of people when they were diagnosed some 10, 15 years ago. So it highlights the benefit we're seeing from these innovations and why it's so important that we need to make studies on these drugs available to as many patients as possible, including in regional Australia or in other priority populations, so that they can all benefit. So, like all the biospecific drugs, the most important side effects include the cytokine release syndrome. And people who are using these drugs obviously will need to become adept at managing um the side effects of these agents.

SPEAKER_01 14:38

Interesting development. And the next unpronounceable drug, Craig.

SPEAKER_00 14:43

It's itiratinib. You'd have known. So this is a listing for patients intermediate to or high-risk primary myelophobosis, post-polycythemia vira myelfibrosis or post-essential thrombocythemia myelfibrosis. So the hematologists or hematology nurses listening will know all about that, and probably the medical oncologists will be a bit perplexed. This is another uh oral kinase inhibitor. It works by inhibiting both JAC-2 and FLT3, reducing spleen size and disease-related symptoms. Common side effects include diarrhea, nausea, vomiting, and anemia. So these need to be well managed because patients are on these drugs for considerable periods of time. And those even low, low-grade toxicities can have a significant impact on quality of life. But interestingly, one uh particular adverse event of note is Wernicke's and kephalopathy, usually associated with um thymine deficiency, uh causing potentially fatal neurological toxicity and of note. It's often um seen in people with severe alcoholism. So it's suggested that thiamine levels are monitored.

SPEAKER_01 16:02

And I believe there is one more, and this one I think is quite unpronouncedable. See how you go.

SPEAKER_00 16:08

Yeah, it's called Mogamelazimab. How's that? Well, Mogamelazimab. And so this is for patients with relapsed or refractory cutaneous T cell lymphoma. Again, I'm just gonna have to be really careful how I say this, but this is a defucacylited humanized immunoglobulin G1 kappa monoclonal antibody selectively binding to CC motive chemoreceptor 4.

SPEAKER_01 16:38

Woof.

SPEAKER_00 16:38

Well and there you are. So this is another become another important treatment option uh in patients with uh relapse or refractory uh cutania still see T cell lymphomas. That's a really quite a niche drug, expected about 100 people a year will benefit though. And isn't it great? All these people with rare cancers are now getting treatment options. And a big shout out to people from Rare Cancers Australia who have been really advocating very hard to get as many drugs as possible listed in rare cancers.

SPEAKER_01 17:12

Absolutely, that's a fantastic organization.

Price Cuts And Nurse Prescribing

SPEAKER_00 17:16

That's really important. And then a couple of other little changes to note, big price cuts for drugs, some common drugs, aberterone, anestrazole, bevisismab, and azocytadine, as we see the impact of generics and biosimilars, and also an opening up of the listing for ruxolitonib and momolotonib. Uh now some nurse practitioners are able to prescribe that under supervision. So big shout out to any of the nurse practitioners listening.

SPEAKER_01 17:47

Yeah, fantastic. We know we have a good section of listeners that are nurse practitioners and readers. So absolutely big shout out. Do you think that this is something that's going to happen more and more?

SPEAKER_00 17:58

Yes, I do. I think it's going to have to for us to cope with the workload of uh the cancer tsunamis upon us with an aging population and more and more treatment options, people staying under treatment longer. So certainly, you know, we need to look at appropriate changes in new models of care, including training up the nurse practitioners who are able to prescribe under supervision.

SPEAKER_01 18:20

Yep, absolutely. It's a really interesting point that we're getting to this apex of having so many new therapies and so much information and so much education that's required around the prescribing of all these second, third line therapies around specific molecular targets.

Listener Voice Notes And Next Steps

SPEAKER_00 18:39

Well, it's been wonderful to chat again, Rachel, and uh share the information about these new innovations. We're delighted to hear feedback and Rachel's will explain how you can do that on WhatsApp.

SPEAKER_01 18:52

Yep, absolutely. So in the show notes page, you'll see a little icon that you can press and it will open up WhatsApp and you can send us a voice note and we can include your lovely voice alongside ours in the next episode. So please do get in touch.

SPEAKER_00 19:07

Fantastic. So we'll see you all next month, hopefully for some more exciting listings. And if you're not a listener of the OGC today, uh also check that out.

SPEAKER_01 19:16

Brilliant. Thanks so much, Craig. Looking forward to speaking to you soon.

Safety Disclaimer And Where To Learn

SPEAKER_00 19:20

Thanks, Rachel. Bye, everybody. Please don't take what we say at face value. Please do your own research. Do your own new diligent check of drug indication, be across the efficacy and toxicity data. Discuss it with your patients before embarking on prescribing any of these bloody difficult to say drugs.

SPEAKER_01 19:35

That's all for this episode of PBS Updates. For more information on the listings we discussed and for ongoing coverage of cancer medicines, visit oncologynetwork.com.au where you'll find news, analysis, and resources for oncology professionals. If you're working in industry and have details of compassionate access schemes or upcoming PBS changes you'd like us to share with the oncology community, feel free to get in touch. And don't forget to check out the Oncology Journal Club podcast if you'd like to hear more analysis and puzzles of wisdom from Professor Craig Underhill. This podcast is primarily produced by the Oncology Network. Thanks for listening.