In Focus: From the lab to the clinic in New Zealand’s first CAR T-cell therapy clinical trial
Dr Nathaniel Dasyam is a postdoctoral researcher who has been working in the Malaghan Institute’s CAR T-cell therapy programme since 2019. Nathaniel is a self-described ‘Jack of all trades’ when it comes to his role within the programme..
Nathaniel works in the research team, where the CAR T-cells are designed, and supports both the GMP team, where the CAR T-cells are manufactured, and the clinical team, who administer the CAR T-cells to participants in the trial.
“It just so happened that the project I was working on previously at the Malaghan Institute, to test a therapeutic vaccine for melanoma, laid the groundwork for many of the processes that needed to be in place to manufacture CAR T-cells.”
This placed Nathaniel in an ideal position to help the CAR T-cell team develop methods needed to test CAR T-cells for the ENABLE clinical trial – a first of this kind for New Zealand. This was no mean feat given it involves developing a gene therapy in a highly regulated environment.
CAR T-cell therapy works by extracting a patient’s own immune cells and modifying them in the lab to identify and attack cancer cells once they have been returned to the patient. The ENABLE trial uses CAR T-cells to treat patients with certain types of relapsed and refractory B-cell non-Hodgkin lymphoma, who have exhausted other treatment options. Read more here: first-car-t-cell-therapy-clinical-trial
Patient booklet from AbbVie on Venclexta
We’re pleased to be able to share this information and patient booklet from AbbVie on Venclexta.
Their Venclexta website https://venclexta.co.nz/cll-sll/about-cll-sll/ is also another useful source of information on CLL and SCL.
Imbruvica Patient Booklet. This booklet was developed to support people prescribed IMBRUVICA (ibrutinib).
This IMBRUVICA Patient Booklet on ibrutinib/Imbruvica has been provided by Janssen and is also available in hard copy by emailing: clladvocates@outlook.co.nz.
This booklet was developed to support people prescribed IMBRUVICA (ibrutinib). It will help you, and your family and friends,
understand more about your condition and what you can expect during your treatment. It also suggests how to access extra support if required.
NZIER Community Pharmaceuticals Report 2022
NZIER has today released its 2022 Community Pharmaceuticals Report. This report looks at the overall trends in the Government-funded medicines budget for New Zealand in the 15-year period between 2006/07 and 2020/21.
We recommend reading the executive summary of the report as a starting point. Below are some key ideas which have come out of the report.
(1) The report identifies that there is a $332 million investment gap in medicines that are publicly funded and made available to patients through the public health system in New Zealand.
- This is the amount of additional investment that would be required on top of the current budget to have the same level of investment in medicines as existed in 2006/2007 in real terms (i.e. inflation and population adjusted).
- The additional investment is required because, despite some new investments being made, there have also been significant transfers of already existing medicines spending from other parts of the health system into the Combined Pharmaceutical Budget from 2012 onwards, which has hidden the fact that the core funding (community pharmaceutical expenditure) has been dropping [see Figure 2].
- As a result, when compared to the total Health budget which has grown by 1.0% in real terms over the 2006-2021 period, the community pharmaceuticals budget has decreased by 2.9% on an annual compound growth basis [see Table 1].
(2) One solution to this investment gap would be a corrective real terms adjustment of $332 million to maintain stability in pharmaceutical investment relative to other health and social investments [See Table 2].
- This corrective action approach was taken by the Government with the DHB deficits in Budget 2022. The Government has also taken a multi-year approach to funding the health system with a confirmed and committed $14.9 billion over the next four years. It is the first Budget ever to take that multi-year approach.
(3) Looking forward from 2022, the medicines budget received $191 million investment but only over a 2-year period ($72 million (2022/23)and $120 million (2023/24)), rather than a four-year period as for other parts of the system. This lack of funding commitment and stability was raised by the Treasury as the only major health budget issue in their Budget Economic & Fiscal Update (BEFU) for 2022.
- Other near-term issues include the need for the medicines budget to also include investment in COVID-19 vaccines and therapeutics going forward, which will put more pressure on an already stressed medicines budget situation.
Publicly funded community pharmaceuticals are a critical and valuable enabler for both New Zealanders' ability to self-manage health conditions and for the health system to function optimally. They, therefore, require protection from ongoing budget erosion. There also needs to be an alignment of the medicines budget appropriation to the broader health systems budget and planning cycles.
To read the full report, please click here
Help us Help you! Please join one of three CLL focus groups on 3rd or 10th of December on Zoom.
Help us help you!
We’re getting moving on some research to find out what CLL patients would like CLL Advocates NZ to do for them.
Please help us with this by joining one of three focus groups on Zoom to help us understand what issues are of greatest concern and interest to you and how we can help with them.
The sessions will be very friendly, café-style discussions with groups of 6 – 8 people and a professional facilitator, and you’ll be able to join by one simple click on an email we’ll send you. The groups will be held on :
- Saturday 3rd December 10:00am – 11:30am
- Saturday 3rd December 1:30pm – 3:00pm
- Saturday 10th December 10:00am – 11:30am
We have limited resources so we’re very keen to start the year with a fresh agenda focused on what really matters to patients and their whanau.
Please contact Melanie (our admin assistant) on clladvocates@outlook.co.nz with any questions, and to let us know if you can join one of these groups.
The information we gather from these sessions will be strictly confidential to CLL Advocates NZ and you would not need to show your full name online if you prefer.
Please help us to help you!
Dr Gillian Corbett’s submission to Pharmac
On behalf of CLL Advocates Trustees
Dear friends,
Below is Dr Gillian Corbett’s submission to Pharmac on the proposal to fund Ibrutinib, submitted today. We warmly encourage you to make your own submission - send it to consult@pharmac.govt.nz by 4pm on Thursday. It doesn’t need to be long or detailed, but every voice counts!
With thanks
CLL Advocates NZ
27 September 2022
Submission to Pharmac on the proposal to fund ibrutinib for people with Chronic Lymphocytic Leukaemia
Introduction
CLL Advocates NZ (CLLANZ) is a charity set up by my colleague the late Dr Neil Graham to advocate for the optimal treatment for patients with CLL. I am a retired haematologist with a strong interest in the management of CLL, and I am a Trustee of CLLANZ. I write this submission on behalf of the Trustees and friends of our organisation and in the spirit of the objectives Dr Graham set out in establishing it.
A personal note
As some Pharmac personnel may recall, Dr Graham was a passionate advocate for gaining funded access to ibrutinib for CLL patients, and gave a great deal of his time and energy to give voice to the desperate need for this treatment. This included, among many other things, meetings, and correspondence with Pharmac, submissions, a petition, an appearance before the Health Committee, and an appearance before the Independent Pharmac Review Panel, using the ibrutinib saga as an example of many of the things we believed need to be addressed in Pharmac’s funding approval processes.
We are very sorry Dr Graham did not live to hear the news of your proposal to fund ibrutinib, although like us he would have been disappointed with the narrow criteria being proposed.
- For urgent future consideration
I appreciate that your request for feedback concerns the current proposal specifically, and I provide that in II. below. But for the record I ask you to give urgent consideration to widening the availability of ibrutinib to these further categories of patients with CLL as follows:
- a) For the upfront management of older or less fit patients who are ineligible for fludarabine, cyclophosphamide, rituximab (FCR) treatment who do not have del17 or Tp53 mutations.
The RESONATE study as updated recently, Barr et al, Journal of Clinical Oncology, Vol 39, Issue 15 supplement, 2021 compared upfront ibrutinib with chlorambucil in patients with CLL aged >65. With a follow up of 6.5 years the PFS for ibrutinib was 61% compared to 9% for chlorambucil. For many older patients chlorambucil and obinutuzumab is the only option for upfront treatment. Ibrutinib is an oral therapy with generally manageable side effects. More recently in the COVID era it is desirable that patients should be treated as outpatients without the need for intravenous therapy which is required for chlorambucil and obinutuzumab.
- b) The other mode of treatment we support for older or less fit patients is the combination of venetoclax and ibrutinib. We believe that at present this is the optimal approach.
The GLOW Study, Kater et al NEJM Evidence, May 13, 2022, evaluated the efficacy of ibrutinib and venetoclax for older and unfit patients with CLL. The study (N=211, median age 71 years) is randomised evaluating first line fixed duration ibrutinib and venetoclax versus chlorambucil and obinutuzumab in elderly patients, >65 years or younger patients with CIRS >6 or creatinine clearance of <70 ml/min. The patients were randomised to either 3 cycles of ibrutinib followed by 12 cycles of ibrutinib and venetoclax (n=106) or 6 cycles of chlorambucil and obinutuzumab (n=105). This study demonstrated that the combination of ibrutinib and venetoclax demonstrated superior PFS (HR 0.216, CR 0.131 to 0.357 and response, Table 1). This study is for a fixed duration of therapy with, to date, excellent results.
- c) We continue to advocate for ibrutinib for relapsed/refractory patients asan alternativeto venetoclax-containing regimens (as opposed to only after they have had venetoclax); likewise for previously untreated patients with 17p deletion, TP53 mutation, and unmutated IgVH.
- Feedback on Pharmac proposal to fund ibrutinib
As noted above we welcome the recommendation of Pharmac to fund ibrutinib for people with relapsed/refractory CLL following treatment with venetoclax. We see this as a recognition of the value of this drug in the management of patients with CLL.
We submit that:
- Patients who relapse later than 3 years after chemo-immunotherapy or treatment with venetoclax, should be eligible for funded ibrutinib.
We are not aware of any evidence or rationale for denying ibrutinib to this patient group, and believe this inclusion in the Special Authority is an arbitrary restriction that would create inequity among patients in virtually identical situations.
It is possible to repeat FCR in some patients, but this is not always feasible because of age or previous toxicity. Second line options for older patients who relapse after bendamustine and rituximab or chlorambucil and obinutuzumab are very limited.
- Patients who are self-funding ibrutinib on their clinician’s recommendation, or receiving ibrutinib on a compassionate access programme on their clinician’s recommendation, should be eligible to transition to funded treatment.
Specifically excluding these patients in the criteria in the Special Authority is manifestly unfair, affecting as it does the exact patient group for whom the application for ibrutinib was originally intended. The change to today’s CLL treatment landscape could not have been anticipated at the time by clinicians or patients.
We submit that there needs to be a pathway available for these patients to move onto funded ibrutinib at the time the medicine is listed. This could take the form of some type of special request from a clinician or the ability to waive the exclusion in respect of these patients.
We look forward to your response to this submission and are happy to discuss it with you further.
Gillian Corbett MBChB, FRACPath, MRCP, FRACP
Trustee, CLLANZ
Pharmac is proposing to fund Ibrutinib!!!
I’m very pleased to advise that Pharmac has today asked for feedback (Follow this link) on a proposal to fund ibrutinib for New Zealanders with CLL. At last!
It’s seven years since Janssen (the supplier) first applied to Pharmac for funding for ibrutinib for patients with relapsed or refractory CLL.
CLLANZ as a patient advocacy group, and our founder the late Dr Neil Graham in particular, have battled hard over the last few years to highlight the desperate need for this treatment. I’m only sorry that Neil did not live to hear this news.
While it’s great that Pharmac is finally moving on this, it is disappointing that they’ve significantly narrowed the criteria first sought for this treatment. These were to fund it as an alternative to venetoclax-containing regimens, and for previously untreated CLL patients for whom chemoimmunotherapy is inappropriate, and notably those with 17p deletion, and TP53 and unmutated IgVH.
However the Pharmac proposal now up for consultation is to fund ibrutinib for relapsed/refractory CLL patients only after they’ve had venetoclax, or for patients where venetoclax is intolerable. So it’s not available as an alternative to venetoclax, an option clinicians have been calling for urgently for some time. Importantly, it also means it won’t be funded for patients who are currently self-funding ibrutinib, or patients receiving it on a compassionate access programme or on a clinical trial. This seems unreasonable and unfair, and we want to seek clarity from Pharmac on what will happen with these patient groups for whom treatment with ibrutinib remains a high priority.
Pharmac is inviting feedback on their proposal until 4pm on Thursday, 29 September. CLLANZ will be providing feedback and we strongly encourage you as patients and whanau to email Pharmac with your own feedback on this to: consult@pharmac.govt.nz. But we feel that it would be more effective to have a coordinated response from CLLANZ friends and supporters on this and propose to come back to you with some guidance, once we have had time to absorb the detail of the proposal and its ramifications.
Let’s not detract from the fact that today’s development is undoubtedly great news, and I’m sure we will all want to support the proposal. But we might also wish to comment on the eligibility restrictions. It may be, with sufficient encouragement and evidence from the CLL community, that Pharmac will consider extending funding of ibrutinib for all relapsed/refractory CLL patients and ideally, as a first line treatment, finally putting us in step with the rest of the world.
As you may be aware, following the highly critical report by the independent Pharmac Review Panel, Pharmac has undertaken to be more open to patient voices, and we want to take this opportunity to be heard!
With best wishes
Dr Gillian Corbett (On behalf of CLLANZ Trustees)
World CLL day
It is World CLL Day today which is held on the first day of Blood Cancer Awareness Month.
On World CLL Day we also wish to send a message of THANKS to those with healthy immunities for considering our community and showing understanding. During the COVID-19 pandemic many people experienced isolation, fear and the difficulty of keeping safe – an insight into the life of a CLL patient.
https://www.wclld.org/about-world-cll-day/
Rachel Smalley: 1,000 kiwis will die while Pharmac takes eight months to make a decision
OPINION: How long does it take you to make a decision about whether or not to buy something?
You’ve got all the information in front of you. You know it’s a good product. It works. You need it. And you’ve got the money to buy it. So how long will it take?
What if I said that every week that passes where you don’t make a decision, 26 New Zealanders will die? What then? Do you think you could focus or add some urgency to that decision?
I’m pretty sure you could.
Why is it, then, that the same level of urgency doesn’t apply to Government – and in particular, to Pharmac, our drug-buying agency?
