Ibrutinib Combo in Chronic Lymphocytic Leukemia is Superior, but Costly

Ibrutinib Combo in Chronic Lymphocytic Leukemia is Superior, but Costly

This article was originally published on the Cancer Network

In the first-line setting, ibrutinib plus rituximab had a superior survival benefit to standard chemoimmunotherapy for patients with chronic lymphocytic leukemia (CLL), according to an interim analysis of a phase III randomized clinical trial published in the New England Journal of Medicine.

Study researchers also noted that “indefinite” use of ibrutinib therapy has been associated with “substantial expense.” According to the IBM Micromedex, the estimated average wholesale price per month for ibrutinib alone is more than $13,324. A 2018 study found that at current prices, ibrutinib was not cost-effective as first-line treatment for CLL patients older than 65 years of age with a 17p deletion.

The trial enrolled 529 patients between March 2014 and June 2016 who were 70 years of age or younger with treatment-naïve CLL. Patients were randomly assigned in a 2:1 ratio to receive either 6 cycles of ibrutinib plus rituximab followed by continuous administration ibrutinib until disease progression (n=354) or 6 cycles of standard chemoimmunotherapy (n=175), which consisted of fludarabine, cyclophosphamide, and rituximab. Follow-up was a median of 33.6 months.

At 3 years, patients who received ibrutinib plus rituximab had a statistically higher progression-free survival (PFS) rate and 65% lower risk of disease progression or death compared with patients who received standard chemoimmunotherapy (89.4% vs 72.9%; HR=0.35; 95% CI, 0.22–0.56; P<0.001). Patients who received ibrutinib plus rituximab also had a significantly higher overall survival (OS) rate and an 83% lower risk of death compared with patients who received standard chemoimmunotherapy (98.8% vs 91.5%; HR=0.17; 95% CI, 0.05–0.54; P<0.001).

Overall, both the ibrutinib plus rituximab and chemoimmunotherapy groups had approximately an 80% incidence of adverse events of grade 3 or higher. There was, however, a difference in infection complications of grade 3 or higher, with the ibrutinib plus rituximab group having a lower incidence compared with the chemoimmunotherapy group (10.5% vs 20.3%; P<0.001).

The incidence of adverse events of grade 3 or higher (regardless of attribution) was similar in the two groups (in 282 of 352 patients [80.1%] who received ibrutinib–rituximab and in 126 of 158 [79.7%] who received chemoimmunotherapy), whereas infectious complications of grade 3 or higher were less common with ibrutinib–rituximab than with chemoimmunotherapy (in 37 patients [10.5%] vs. 32 [20.3%], P<0.001).

“I don’t think one hundred percent of people should be getting ibrutinib as frontline therapy,” Joshua Brody, MD, director of the Lymphoma Immunotherapy Program at the Icahn School of Medicine at Mount Sinai Hess Center for Science and Medicine in New York, told Cancer Network.

On the basis of the study results, he explained the ibrutinib combination is “definitely” a “reasonable” option and “better” than fludarabine, cyclophosphamide, and rituximab, but it’s not “definitely” better than rituximab plus bendamustine, which is the other chemoimmunotherapy regimen commonly used in this patient population.

previously published trial in treatment-naive CLL patients found a PFS benefit among those who received ibrutinib monotherapy or ibrutinib plus rituximab when compared with rituximab plus bendamustine. However, no OS benefit was seen for the ibrutinib-containing regimens.

Government unveils national cancer agency and $60 million injection into Pharmac

Government unveils national cancer agency and $60 million injection into Pharmac

This article was originally published on the New Zealand Herald

The Government has today unveiled plans to establish a national cancer agency, which will be led directly by experts of the deadly disease, and a $60 million injection into Pharmac.

Key announcements:

• Cancer Control Agency to abolish postcode lottery.• World-leading public health physician and cancer epidemiologist Professor Diana Sarfati has been appointed as the Ministry of Health’s interim National Director of the cancer control agency.

• A $60 million funding boost to Pharmac, $20 million this year and $40 million in 2020/21.

• A new system to fast-tracking Pharmac’s drug funding decision process.

Until today, decisions about how to tackle New Zealand’s cancer “crisis” came from bureaucrats within the Ministry of Health.

By December 1, the Government has promised decisions will come from a team of top cancer specialists who will report directly to Health Minister David Clark. That is the date the agency will officially be up and running.

World-leading public health physician and cancer epidemiologist Professor Diana Sarfati has been appointed as the Ministry of Health’s interim national director of the agency.

“This is huge for New Zealand. It’s the biggest change in cancer care in at least 15 years, if not ever. When the UK appointed a national cancer director there was an unprecedented improvement and survival rates increased dramatically,” Cancer Society of New Zealand medical director Chris Jackson said.

It comes after a powerful public movement started by dying dad Blair Vining who launched a petition calling for a national cancer agency to hold District Health Boards to account and put an end to postcode lottery.

His petition gained more than 140,000 signatures from New Zealanders across the country.

One of the main aims of the plan is to have equitable cancer survival rates across New Zealand by 2030 – including across geographic areas and across ethnicities. Māori cancer outcomes will be one of the first priorities as Māori have higher rates of cancer and poorer survival rates overall.

Funding decisions will be made with an “equity first” methodology, the plan says.

For example, the national bowel screening programme, which is still being rolled out, makes screening free for people aged 60-74.

But because Māori people tend to develop bowel cancer at an earlier age, the plan suggests the screening programme could be made free for Māori ten years earlier, between the ages of 50-74.

Another aim is for New Zealanders to suffer from fewer preventable cancers.

Lung cancer is the top of the list, with prevention strategies including promotion of vaping and the finalisation of a Smokefree 2025 Action Plan.

The plan also highlights improved detection and management of Hepatitis A, B and C, reducing HIV transmission, increasing the uptake of the HPV vaccine and addressing Helicobacter pylori infection in priority populations.

Skin cancer will also be a target, with promotion of sun safety – particularly among children – and sunscreen could also be regulated as a therapeutic product, the plan says.

A programme will also be developed to prevent work-related cancers.

Another aim of the plan is to improve survival rates once people have cancer, with an emphasis on better, earlier screening, and improved treatment once diagnosed.

Strategies including increasing the age of free breast screening from 70 to 74 years old, progressing the national bowel screening programme and exploring bringing in HPV screening as part of the cervical screening programme.

The radiation and oncology workforce could also see a boost to its workforce and resources.

Fast-track diagnostic pathways will be developed for “priority cancers”, and there would be a national agreement on the scope and distribution of specialist cancer services.

“Quality performance indicators” will be introduced under the plan to ensure specific cancers are being diagnosed and treated consistently across New Zealand.

Bowel cancer has been used as a test case for QPIs with the first results published for diagnosis and treatment in March this year. The Bowel Cancer Quality Improvement Report compared DHBs on measures such as death rate after surgery, length of stay in hospital and where at least 12 lymph nodes were examined.

By early 2020 prostate, lung and neuro-endocrine tumours will also have similar reports published.

Health Minister Dr David Clark said the Government has listened to calls for strong central leadership and will deliver the promised Cancer Control Agency by December 1, 2019.

“Cancer care is woven into so much of the work that our public health service does, so while the agency will have its own chief executive, it makes sense for it to be housed within the Ministry of Health.

“I’m also pleased to announce that leading public health physician and cancer epidemiologist Professor Diana Sarfati has been appointed interim National Director of Cancer Control, starting immediately. She will lead work to improve the quality of treatment.

“An immediate priority will be establishing quality performance indicators for specific cancer types. This will mean we can measure progress towards consistent care across DHBs.

“We are also combining the four current regional cancer control networks into a National Network to help remove regional variations in care,” Clark said.

More Pharmac funding welcomed

Pharmac has welcomed the $60m funding injection, which includes $20m for this year and $40m for 2020/21. That’s on top of $10m announced in this year’s Budget.

That brings Pharmac’s annual medicines budget to over $1 billion, Pharmac board chair Steve Maharey said.

In last year’s Budget, the Government increased Pharmac’s funding by more than 13 per cent, from about $870m to $985m. That compares to increases of 2.4 per cent and 6.3 per cent in National’s last two years in government.

Pharmac announced this afternoon it was proposing to funding three new medicines to treat ovarian cancer (olaparib, known as Lymparza), breast cancer (fulvestrant, known as Faslodex) and leukaemia (Venclexta – used to treat chronic pymphocytic leukaemia).

It is also proposing making contraceptives Mirena and Jaydess available to more people, and making the meningococcal ACWY vaccine free for people in close living situations like hostels and army barracks.

The medicines could be available from November if consultation feedback is positive.

Pharmac said it was also looking to get the best deal for New Zealanders for a particular type of advanced breast cancer.

Two registered medicines palbociclib (Ibrance) and ribociclib (Kisqali) could be suitable, experts believe.

Pharmac said it had issued a request for proposals and one of these medicines could be available from April next year.

Prime Minister Jacinda Ardern said from next year Pharmac will also speed up its decision-making by considering applications for funding while Medsafe assesses the safety of new medicines.

“Rather than waiting until that work is complete as it does currently. Work on options for early access to new cancer medicines is also progressing well,” Ardern said.

New Zealand’s history of tackling cancer:


– Labour Government announces plans are under way for first ever cancer plan.

2003 – Cancer control strategy released included cancer prevention, screening, early detection, treatment, rehabilitation, support and palliative care.

2005 – Labour Government establishes national agency known as Cancer Control Council, formally Cancer Control of New Zealand, to provide independent advice free from political interference.

2014 – National Party-led Government introduces three-year cancer plan, including a faster cancer treatment target requiring most patients to start treatment within 62 days of diagnosis.

2015 – National disestablishes Cancer Control Council, saying it is no longer necessary as it has been superseded by new plan.

July 2017 – Pre-election, Labour promises to set up a National Cancer Agency, with $10 million to establish the agency and a further $10m to get work under way.

2018 Labour-led Government ends public reporting of DHBs’ faster cancer treatment target results, although the data is still collated.

Jan 31, 2019 – Melissa Vining blasts Health Minister David Clark for not following through on promise to establish cancer agency.

Clark announces the Government’s plan to tackle cancer is under way and he expects to see a draft by the end of June.

May 6, 2019 – Herald starts investigation revealing hundreds of Kiwi cancer sufferers have received large taxpayer-funded payouts after being let down by the public health system.

June 28, 2019 – Blair Vining launches petition calling for a national cancer agency to hold District Health Boards to account and improve the health care system.

June 29, 2019 – Herald reveals Prime Minister’s letter to Blair’s daughter Lilly Viningsuggesting agency is not part of the Government’s plan.

More than 650 New Zealanders gathered in Invercargill to join Vining as he handed over a petition to National’s Michael Woodhouse.

July 7, 2019 – Vining’s petition closes to the public.

Today – Government’s unveils plans to establish a national cancer agency lead by experts of the deadly disease.

New Study Reports “Curative Potential” of a Combination Therapy for Some Leukemia Patients

New Study Reports “Curative Potential” of a Combination Therapy for Some Leukemia Patients

This article was originally published on Dana-Farber Cancer Institute

Chemoimmunotherapy combined with a targeted drug given for two years has achieved undetectable minimal residual disease (MRD) for a high proportion of younger patients with chronic lymphocytic leukemia (CLL), Dana-Farber scientists report.

The phase 2 clinical trial results are so favorable that they represent a step toward “a new standard of care with curative potential for a broad population of younger, fit patients” with CLL, say the investigators, led by Matthew Davids, MD, MMSc, and Jennifer Brown, MD, PhD. “Younger” in this setting is 65 years of age or below.

The report in The Lancet Haematology describes the regimen administered to 85 patients with CLL whose median age was 55 years. The study looked at individuals whose cancer carried a mutated IGHV gene — which confers a higher likelihood of a long-lasting response to chemotherapy — and those with an unmutated IGHV gene — who rarely have durable responsesto chemotherapy.

The study examined the combined effect of two different treatment options for CLL patients. The first, chemoimmunotherapy consisting of fludarabine, cyclophosphamide, and rituximab (FCR), can improve overall survival for younger fit patients with CLL who have an IGHV mutation. The second, the targeted drug ibrutinib, can be effective for CLL patients irrespective of their IGHVstatus, but requires continuous treatment. The clinical trial examined if FCR plus time-limited ibrutinib could achieve long-lasting responses in younger, fit patients with either mutational status.

The patients, who were previously untreated, received oral ibrutinib for seven days, then up to six 28-day cycles of FCR along with continuous ibrutinib. Responders continued on maintenance ibrutinib for up to two years, and those who had undetectable MRD in bone marrow after two years were able to stop treatment.

Thirty-three percent of patients achieved the primary endpoint — a complete response with undetectable minimal residual disease in bone marrow two months after the last cycle of ibrutinib plus FCR. A best response of undetectable minimal residual disease in bone marrow was achieved by 84 percent of patients at any time during the study. “Many of the patients had deepening responses while on the two years of ibrutinib maintenance, so later converted to undetectable MRD,” explains Davids.

The researchers say the results of the trial demonstrated that the combination of chemotherapy and two years of ibrutinib maintenance leads “to the best of our knowledge, to the highest proportion of patients achieving undetectable minimal residual disease in the bone marrow ever published for any regimen used to treat this disease.” The approach, they add, could increase the potential to “functionally cure patients with mutated IGHV and, importantly, might for the first time extend this potential to patients with unmutated IGHV.”

Survival increased in chronic lymphocytic leukemia patients treated with ibrutinib and rituximab

Survival increased in chronic lymphocytic leukemia patients treated with ibrutinib and rituximab

This article was originally published on 2 minute medicine

1. Previously untreated chronic lymphocytic leukemia (CLL) patients experienced increased progression-free and overall survival when treated with ibrutinib-rituximab compared to current standard chemoimmunotherapy.

2. Rates of high grade adverse events were similar between treatment groups.

Evidence Rating Level: 1 (Excellent)  

Study Rundown: CLL is a common hematologic malignancy often treated with a combination of fludarabine, cyclophosphamide, and rituximab. While efficacious, there are notable side effects to the chemoimmunotherapy regimen. The Bruton’s tyrosine kinase (BTK) inhibitor ibrutinib has shown efficacy in patients with relapsed or refractory CLL, though how it performs as a first line treatment is not yet determined. This phase 3 trial evaluated ibrutinib-rituximab in comparison to standard chemoimmunotherapy in untreated CLL patients and found both progression-free and overall survival to be improved relative to standard therapy at almost 3 years after treatment initiation. The incidence of grade 3 or 4 adverse events was similar in the treatment groups, though infections were more common in the ibrutinib-rituximab group.

This randomized trial provides support for use of a new regimen as a first line treatment in CLL patients. Strengths of the study include its randomized design, mutational analysis of immunoglobulin heavy-chain variable region (IGHV) patients, and extensive subgroup analysis. The utility of this study is limited by its discordance with other trials evaluating ibrutinib in CLL.

Click to read the study in NEJM

Cirmtuzumab-Imbruvica Combo Trial Enrolling CLL, SLL Patients for Phase 2

Cirmtuzumab-Imbruvica Combo Trial Enrolling CLL, SLL Patients for Phase 2

This article was originally published on Lymphoma News Today

Following promising early efficacy results for a cirmtuzumabImbruvica treatment combo, a Phase 2 clinical trial has now been opened to people with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).

The opening of this randomized trial was based on the promising results seen in the prior Phase 1 stage, in which 100% of the first nine CLL/SLL patients responded to treatment with Oncternal Therapeutics‘ investigational therapy cirmtuzumab in combination with Imbruvica (ibrutinib).

The Phase 2 clinical trial is now open to an additional 90 patients with CLL/SLL. Participants will be randomly selected to receive the combination of cirmtuzumab plus Imbruvica, or Imbruvica alone. More information on enrollment can be found on the trial’s page here.

Imbruvica, by Janssen and AbbVie, is the standard of care for people with CLL/SLL and mantle cell lymphoma (MCL) who have received at least one prior therapy. The molecule binds permanently to the Bruton’s tyrosine kinase protein, which is key in the growth and proliferation of B-cell cancers.

Cirmtuzumab is an antibody that blocks the ROR1 receptor in tumor cells. ROR1 is rarely seen in healthy cells, but in tumor cells it works as a receptor for growth signals, helping the tumor grow and survive.

Researchers have been testing a combination of these two treatments in patients with CLL/SLL and MCL who have failed prior therapies but have not received a prior BTK inhibitor.

The CIRLL Phase 1b/2 trial (NCT03420183) was first designed in three parts. First, participants received ascending doses of cirmtuzumab alone, followed by a combination of cirmtuzumab and Imbruvica. The goal was to determine the optimal dosing regimen for further testing.

The recommended dosing regimen then was further tested in part 2 (Phase 1b). In total, 66 patients were included in these two Phase 1 parts.

Preliminary data from these parts were presented in a poster, “Phase 1/2 Trial of Cirmtuzumab and Ibrutinib: Planned Analysis of Phase 1 CLL Cohorts,” at the American Society of Clinical Oncology (ASCO) annual meeting, held in the spring in Chicago.

The analysis included the first 12 CLL/SLL patients, for whom the treatment was safe and well-tolerated. The therapy led to an objective response rate of 91.7%. Three of the 11 patients who responded had clinical or confirmed complete responses.

Researchers noted that all nine patients who completed 12 weeks of treatment in part 2 — in which participants received a recommended dose of 600 mg cirmtuzumab plus 420 mg Imbruvica — achieved a response to treatment.

Preliminary data also was presented for the first six MCL patients. One participant, whose disease had returned after a stem cell transplant, had a complete response after three months of treatment, which was maintained at least until month 11. The treatment also led to the resolution of a tumor in the chest.

The findings in CLL/SLL participants led researchers to open a third part (Phase 2), to compare the safety and efficacy of the combination versus Imbruvica alone. The main goal is to compare the percentage of patients achieving a complete response.

“We are very pleased to be opening the randomized Phase 2 portion of the CIRLL study for patients with CLL and continue to be encouraged by the interim results from the study for both patients with CLL and patients with mantle cell lymphoma,” James Breitmeyer, MD, PhD, president and CEO of Oncternal, said in a press release.

Ibrutinib, Rituximab for Patients With CLL Superior to Standard Chemoimmunotherapy

Ibrutinib, Rituximab for Patients With CLL Superior to Standard Chemoimmunotherapy

This article was originally published on The Center For Biosimilars

A combination of ibrutinib and rituximab for patients with chronic lymphocytic leukemia (CLL) is superior to current treatment, according to results of a phase 3 trial published recently.

As compared with standard chemoimmunotherapy with fludarabine, cyclophosphamide, and rituximab, the ibrutinib-rituximab combination led to longer progression-free survival, as well as overall survival. The authors also said the combination treatment is better tolerated and is less toxic that standard chemoimmunotherapy. While the current CLL treatment standard works for many patients, it is also toxic, causing severe myelosuppression and creating a risk of infections due to immunosuppression of T cells.

In the trial, 89.4% of those treated with the ibrutinib–rituximab combination had not seen their disease progress after 3 years, compared with 72.9% of those who had the traditional combination.

The study randomized a total of 529 patients, giving 354 patients the ibrutinib-rituximab combination and 175 the chemoimmunotherapy regimen. After 33.6 months, the hazard ratio (HR) for the study drug combination for PFS or death was 0.35 (95% CI, 0.22-0.56; P <.001). For OS, the study drug combo had an HR of 0.17 (95% CI, 0.05-0.54; P <.001). All patients were aged 70 years or younger.

Infections were less common in the patients taking ibrutinib–rituximab (37 patients compared with 32 patients). Both groups had a similar rate of serious adverse events. Patients taking the 2-drug combination also reported less fatigue.

The ibrutinib–rituximab combination was especially beneficial for patients without immunoglobulin heavy-chain variable region mutation, a group that historically did not respond as well to the standard treatment regimen.

Patients will likely see a new standard of care, according to the lead author.

“These results will fully usher the treatment of chronic lymphocytic leukemia into a new era,” Tait Shanafelt, MD, professor of medicine at Stanford, said in a statement. “It seems likely that, in the future, these patients will be able to forego chemotherapy altogether.”

Pharmac's dismal ranking little surprise to cancer patients

Pharmac's dismal ranking little surprise to cancer patients

Republished with permission from Graham Adams. Sourced from NOTED.co.nz

By Graham Adams
Aug 16, 2019

New Zealand has the worst record of funding new treatments among 20 OECD countries.

The news this week that New Zealand has been rated the worst among 20 OECD countries for funding modern medicines and pharmaceutical investment overall will come as a surprise to many people but perhaps not to cancer patients.

Cancer treatment gets so much media attention that patients are often acutely aware of what ground-breaking drugs would be available to them if they had the good fortune to live in other developed countries — including Australia. As a result, there is a regular parade of cancer-stricken supplicants to MPs and Parliament begging for their lives and alleging that the funding system overseen by Pharmac is deficient.

The news of our dismal performance came in a report commissioned by Medicines New Zealand, a lobby group for the pharmaceutical industry. It showed that of 304 modern medicines for a range of diseases funded internationally between 2011-17, only 17 were publicly funded in New Zealand.

Australia funded 83, the UK 174, and Germany 178.

And of 70 new cancer drugs funded across the 20 nations in that period, New Zealand had funded only six. Australia’s tally was 26.

Arthritis, asthma, diabetes and cardiovascular disease, among other conditions, fared poorly too.

For all the claims made about Pharmac’s unique approach to funding drugs much more cheaply than in other countries, it is increasingly apparent its strategy of delaying buying drugs until their prices come down is not fit for purpose — particularly when revolutionary cancer treatments are being developed at an astonishing speed.

While Pharmac’s CEO Sarah Fitt is undoubtedly correct in claiming that a “simple count of [funded] medicines won’t tell you how effective a health system is”, she is glossing over the fact that her organisation’s tardy funding decisions amount to rationing by delay for drugs that have proven to be very effective in a battery of clinical trials and medical practice.

As the report noted, the OECD average for approving new treatment from the time of a drug’s registration is 233 days. In New Zealand, it is 512.

As a cancer patient myself, I am very aware of the life-saving drugs available for free in public health systems overseas. I have chronic lymphocytic leukaemia (CLL) and one thing is starkly apparent to me. If my New Zealand-born brother who lives in Brisbane is diagnosed with the same aggressive form of the disease as me, he will be eligible for free access to two wonder drugs — ibrutinib and venetoclax — through Australia’s hospital system. However, New Zealanders with CLL like me can only obtain these drugs through a clinical trial, a drug company’s compassionate access scheme or personally paying more than $130,000 a year (which often means begging from strangers on Givealittle).

Ibrutinib and venetoclax are targeted drugs that have transformed the treatment landscape for those with CLL. Just how revolutionary ibrutinib has been is something Tauranga consultant physician Dr Neil Graham knows only too well from personal experience. Speaking to the Health select committee last week, he had to fight back tears as he recounted how ibrutinib — which he has been able to access through a compassionate access scheme — had saved his life after he had transfusion-dependent bone marrow failure as a consequence of CLL.

He told the committee that ibrutinib has put him into remission and he has been able to continue working productively for the past five years — time that otherwise would have been denied to him, and to the patients he serves.

Writer and TV presenter Clive James is undoubtedly the most famous face of the revolution in CLL drugs. In 2015, he said he was “embarrassed” to still be alive after he had predicted a year earlier that his death — from the double-whammy of chronic lymphocytic leukaemia and emphysema — was likely within months.

Such a prognosis was undoubtedly correct at the time he signalled his imminent demise in 2014 but, soon after that, he began treatment with ibrutinib. His health rallied and he is still alive and writing.

In a book of poems published in 2017 — titled for obvious reasons Injury Time — he described ibrutinib as a “little cluster-bomb of goodness”.

My own experience of coming back from death’s door is similar to that of Dr Graham and Clive James. I knew in 2015 that I was in very serious trouble when a doctor told me rather brutally: “I’ve just read your oncologist’s report. It makes for pretty grim reading, doesn’t it?”

I had been aware that my prospects weren’t very good but the oncologist clearly had been far franker writing to his colleague about my dire prospects than he had been to me in person. I understood the aggressive variant of CLL I have — due to a chromosomal aberration termed “17p deletion” — was difficult to treat but I didn’t realise just how slim the chances of conventional treatment working were.

I had naively assumed there would be something in the oncologist’s armoury that would help. In fact, I learned later that conventional chemotherapy treatment has only a five per cent chance of working for 17p deleted patients. As one haematologist told me: “Standard chemotherapy works well for some 17p patients, but the numbers are very, very small.”

By extreme good luck, I was put in a clinical trial for an experimental drug combination. It held back the progression of my disease for only six months but it made me eligible for another clinical trial of a second-generation version of ibrutinib.

Now, nearly four years later, I am still in remission.

When the drug stops working, if I’m very lucky I’ll get access to a clinical trial for venetoclax and the drug will be provided free. But if I don’t manage to be accepted onto a trial, I’ll have to bankrupt myself to stay alive — as many New Zealand cancer sufferers are doing right now.

However, if I were a citizen of Australia, as my brother is, I would be eligible to have the drug funded for a cost of around $33 a month.

Ibrutinib was first registered by Medsafe in New Zealand in 2015 and subsequently prioritised for funding by Pharmac in 2016.

Venetoclax was registered in 2017 and, last September, Pharmac’s Pharmacology and Therapeutics Advisory Committee recommended venetoclax “be funded with a high priority” for CLL patients with aggressive disease, including 17p patients.

In April this year, the recommendation was repeated as a high priority for funding.

Now, nearly a year later after the recommendation was made last September, the drug remains unfunded.

It is worth noting that Pharmac would undoubtedly be able to buy ibrutinib and venetoclax at a much lower price than its “rack rate”, which could make it not much more expensive per patient than hospital-based dialysis over a year.

Dr Graham wants Pharmac to fund ibrutinib and venetoclax for patients with aggressive CLL disease. He told NOTED: “The timeframes are simply unacceptable for treating malignant diseases generally, and CLL specifically. People are dying waiting for a decision on funding.”

There are about 2000 CLL patients in New Zealand. Dr Graham says most will respond very well to standard chemotherapy and may get remissions of up to 10 years, which is counted as the nearest thing to a cure in treating cancer.

But for the small number who don’t respond to such treatment, their disease quickly becomes a life-or-death matter.

The brutal truth, as he puts it, is that while Pharmac vacillates, people die.

Dr Neil Graham's talk - presented to the Health Select Committee

Dr Neil Graham's talk - presented to the Health Select Committee

The following talk was presented by Dr Neil Graham to the Health Select Committee on 7 August 2019 to support the submission calling for the Select Committee to strongly recommend funding of two life-saving treatments.

“My written submission was to request funding for ibrutinib and venetoclax for CLL, two examples of the new era of cancer treatment medications.

In early 2019 the Cancer at the Crossroads conference was held in Wellington, a major national conference to assess and review the status of cancer treatment in NZ.
An editorial was published in April this year in the NZMJ, reviewing the conference.

To quote from the first paragraph of that editorial:

“Cancer is the leading cause of death in Aotearoa/NZ. The number of those affected by cancer is forecast to increase by 50% in the next fifteen years…..

Our survival rates from cancer lag behind those of Australia, Canada, and Scandinavian countries, and are not improving at the same rate as elsewhere “.

This is a red flag for how we manage the commonest cause of death in our country. We are not doing well by international standards.

In the last decade or so there has been a revolution in cancer medication development internationally, of a magnitude greater, perhaps, than any one area of disease ever. It has been likened in effect to the development of antibiotics for infectious diseases almost a century ago. And it is growing exponentially. It has been predicted that cancer will become controllable, even curable, in the next decade or so.

Some NZers have had their lives saved by these new medications – two of those (Ben and I) sit in front of you today. For me, five years ago, I had transfusion-dependent bone marrow failure. I was likely to die. I was given access to ibrutinib, went into remission, and have been well ever since, working, contributing to society, paying taxes, and living a full life. Ibrutinib saved my life.

Other NZers have not been so lucky.

Most new era life-saving cancer drugs are not funded. If you are wealthy, sell your house to pay, beg in its various guises, move to Australia, or get into a drug trial, you live. If not, you die. And die of a treatable disease. And NZers ARE dying, unnecessarily, because they can’t access these life-saving medications.

Pharmac can’t, or won’t fund them. This is in contrast to Australia, UK, Canada etc, where these medications, including ibrutinib and venetoclax, are funded.

This is still not widely known nationally, but there is an increasing awareness because of this May’s march on Parliament, Blair Vining’s case publicity, and others. When this becomes widely known, it is likely that the NZ population will be critical of NZ health services for allowing this to happen. It is unlikely to be tolerated. You may remember the outcry over hepatitis C in the early 1990’s.

Two other points:
Pharmac has said recently that medications have a relatively small role in cancer treatment. Whilst overall this has some truth, it is not the case for leukaemias, where prevention and screening for early detection have no role, and surgery and radiotherapy have a minimal role. Treatment is all chemotherapy/medications.
And it is the mainstay of treatment in the context of attempted cure in the case of serious cancer in the form of metastatic disease.

Secondly, international guidelines, written by world authorities and specialist societies are not adhered to in NZ, as medications when not funded are generally not able to be used. In CLL, venetoclax, and, even more so, ibrutinib are the first choice medications in international guidelines for severe forms of CLL. But they are not used when not funded.

So people in NZ die unnecessarily.

NZ will increasingly stand out as doing badly in cancer medicine because of this lack of funding – what has been described as third world medicine in a first world country.
So, to finish, the current Pharmac model is not working for modern oncology practice.
My submission makes suggestions regarding this (5 – 9).

The Pharmac model needs to change for modern oncology.

Current Pharmac processing of applications for oncology medications takes almost two years. When you’re dying of cancer, this is woefully inadequate. A rapid access programme for cancer treatments must be developed for these patients.

I would ask/beg this committee to recommend review of Pharmac’s model in modern oncology practice, particularly funding. We spend 5% of our health budget on medications. Other similar countries spend 10 – 15%+.

Involved groups (clinicians, policy makers, Pharmac, patients, and the industry) need to get together and develop a new model to stop the disgrace of NZers dying unnecessarily.”

Health Select Committee hears pleas from survivors and sufferers

Health Select Committee hears pleas from survivors and sufferers

This story was originally sourced here from RNZ

Politicians heard emotional pleas today from cancer survivors, people with disabilities and health agencies to boost Pharmac’s funding for life saving drugs.

Petitions were presented to the Health Select Committee at Parliament – calling on medicines to be funded – including for breast, lung, ovarian cancers, along with multiple myeloma.

Emily Beswick and her daughter, Stella, travelled 500 kilometres from Cambridge to today’s select committee.

Stella has spinal muscular atrophy and uses a wheelchair.

She sat quietly at the back of the select committee room – listening – smiling from time to time.

While medication to help her condition is available overseas – here in New Zealand – it’s not.

And for Ms Beswick that’s devastating.

“We feel that all hope is gone – we have been waiting 13 years for a treatment to arrive and now that it’s finally here – it is out of reach,” she said.

Spinal muscular atrophy is a childhood version of motor neurone disease but the drugs to halt it are not funded here.

MS Beswick said her daughter’s quality of life could be improved.

Neil Graham has been in remission from chronic lymphocytic leukemia for five years.

He broke down as he told the select committee he was given compassionate access to a drug that probably saved his life.

“Since then, I’ve been working, making a useful contribution to society, paying taxes and having a full and enjoyable existence,” he said.

Mr Graham said the Pharmac model needed to change.

“Current Pharmac processing of applications for approval for cancer medications take almost two years – and if you’re dying of cancer – that’s just not acceptable,” he said.

Joy Wilkie is in remission from the blood cancer myeloma.

No new anti-myeloma medicines have been funded here in the last five years.

“When I relapse – and I know I will – our world class clinicians know what best international treatment I need to give me more years of meaningful life,” she said.

Pharmac wasn’t available for an interview and it did not did not appear at the select committee today.

In statement, it said funding decisions take time and it is aware that not everyone has time on their side.

But late this afternoon, it announced on its website a proposal to fund Kadcyla for some breast cancer patients, Alectinib for lung cancer and Ocrelizumab for multiple sclerosis from 1 December.

Meanwhile, a Cancer Action Plan is expected to be announced by Health Minister David Clark later this month.

Leukaemia advocacy group to tell Health Select Committee NZ’s current cancer drug funding scheme is a ‘national disgrace’

Leukaemia advocacy group to tell Health Select Committee NZ’s current cancer drug funding scheme is a ‘national disgrace’

Dr Neil Graham, founder and executive director of CLL Advocates NZ, a new patient support group for New Zealanders with Chronic Lymphocytic Leukaemia (CLL), will appear before the Health Select Committee today in support of their submission to fund life-saving treatments for suffers of CLL.

The Health Select Committee hearing follows the CLL Advocates NZ’s petition, signed by over 1,000 New Zealanders, fighting for funding of ibrutinib (Imbruvica) and venetoclax (Venclexta™).

Neil Graham is calling on the members of the Select Committee to strongly recommend the funding of these two life-saving treatments, to prevent the unnecessary deaths of New Zealanders with the aggressive form of this cancer.

“These treatments are widely funded in many similar countries to NZ, such as Australia, Canada, UK, and Scandinavia, where many lives have been saved. They are not funded in NZ however, and most patients in NZ are unable to afford their cost. As a result, New Zealanders are dying unnecessarily from lack of access to these medications. Those who can pay live, those who can’t die.” said Dr Graham.

Dr Graham is also asking the Select Committee to urgently review the Pharmac model which is patently unable to accommodate the new wave of drugs.

“It is a national disgrace. The current Pharmac model for cancer medication funding is no longer appropriate for providing modern life-saving cancer treatment. It needs to be changed. Funding applications to Pharmac for cancer medications currently take almost two years to be processed. For people dying of cancer, this is woefully too long” said Dr Graham.

Dr Graham, who is living with CLL, had compassionate access to one of these drugs, ibrutinib, and nearly five years later continues to work and live life to the full. He says Pharmac’s continuing refusal to fund the drug is not only a death sentence for New Zealanders with aggressive CLL, but flies in the face of international clinical opinion and best practice.

“Ibrutinib has been registered in NZ since 2015, and is funded in 44 of 45 countries of similar or lower wealth, NZ being the exception. Evidence for the effectiveness of this treatment is incontrovertible, for example overall survival for patients on ibrutinib at first line is 88%. It’s hard to imagine why the evidence that was so compelling for 44 other countries has not persuaded Pharmac to the same position,” said Dr Graham.

Dr Graham will also be joined by Dr Ben Schrader and Dr Rob Weinkove to present in support of this submission.