Cancer Drugs Fund extended until 2016

Whilst I applaud and welcome the news that the Cancer Drugs Fund (CDF) has been extended until 2016 with a budget of £200m a year it’s important we don’t see this as a win and look for the next battle. It is likely that this ‘new’ £400m has been top-sliced from the NHS budget in a similar fashion to original CDF thereby creating a problem at another level.

Let’s also not forget that the CDF was established in order to provide a means by which National Health Service (NHS) patients in England can get cancer drugs that are not routinely available on the NHS. This includes drugs like dasatinib (treatment for CML – Chronic Myeloid Leukaemia) that effectively saves lives (like mine) but for some misguided reason are rejected by NICE (National Institute for Health and Care Excellence). The new value based pricing (VBP) system that we were expecting next year appears to have been swept under the carpet, presumably because when the government did the sums it was going to cost them more than £400m. So our new £400m is a stay-of-execution. Get the next election out-of-the-way, note that this 2 year extension ties very nicely into the coalition term, and make an unpopular decision at the beginning of the next term if the election is won. In the meantime the Conservative party can bang their drums over the next few days at the party conference to the tune of being saviours of cancer patients everywhere. Not sure how people who are suffering with other illnesses are going to feel about that one.

Even I’m astounded by the level of my own cynicism but in this case I feel it is well founded. The complete and utter failure of VBP to even emerge from the starting blocks and the countdown to the end of the CDF left the coalition with no alternative.

Patients deserve the best treatment. We should not be held hostage by pharmaceutical companies or be pawns in politics. Much of the problem lies with NICE, their flawed processes and their reliance on limited information about the clinical effects of new products supplied to it by the pharmaceutical industry. There is much to unpick. In the short-term we’ll save lives and I can’t be angry about that but in the long-term the system will still be in a mess. What we’ve been presented isn’t a solution, it is another finger in the dam and I’m losing count of the number of holes.

Kris Griffin

BBC NEWS: Cancer drugs fund ‘to be extended’ until 2016

SKY NEWS: NHS’ Life-Extending Cancer Drug Fund Extended

The Guardian: Cash injection to keep cancer drugs fund running for two more years

 

Trial Suggests Imatinib Discontinuation Safe, Some Responses Persist

More positive news on World CML day – we need to keep working towards a cure. K

Trial Suggests Imatinib Discontinuation Safe, Some Responses Persist

Evidence continues to mount that discontinuing imatinib treatment for chronic myeloid leukemia (CML) in the chronic phase is safe. A new phase II Dutch and Belgian study showed only about two-thirds of patients relapsed after discontinuing treatment with imatinib and cytarabine, and all patients remained sensitive to imatinib after relapse.
Tyrosine kinase inhibitors including imatinib have revolutionized CML treatment in recent years, but the need to continue treatment indefinitely is limiting. Several recent studies have begun to suggest that alternate treatment schedules or discontinuation of therapy are feasible among patients with good molecular responses. In a study of the Dutch-Belgian Cooperative Trial for Haemato-Oncology, researchers led by Noortje Thielen, of the VU Medical Center in Amsterdam, 33 patients with a molecular response lasting at least 2 years on imatinib and cytarabine combination therapy were randomized to either continue (18 patients) or cease (15 patients) imatinib treatment. Results were published online ahead of print in July in the European Journal of Cancer.

After a median follow-up period of 36 months, three patients randomized to continue treatment (17%) and 10 patients in the discontinuation arm (67%) had a molecular relapse; all three of the former patients had stopped imatinib treatment after randomization.

The molecular relapse rate at 12 months was 0% in the continued therapy group and 53% in the discontinuation group; at 24 months, those rates were 6% and 67%. In an as-treated analysis (accounting for the patients who ceased treatment in spite of randomization to continue imatinib), the two-year rate was 61% for discontinued therapy and 0% for continued imatinib.
The five patients in the discontinuation group who did not relapse showed a stable molecular response. The 13 patients who relapsed all regained molecular response after a median of 6 months from the restart of imatinib or nilotinib treatment.

“To our knowledge, this is the first randomized trial regarding the discontinuation of imatinib in first chronic phase CML patients who have achieved a durable and stable molecular response,” the authors wrote. “Our results are encouraging.” They noted that the addition of cytarabine to the initial regimen may have contributed to the persistence of response after therapy discontinuation, but that remains unclear.

Further studies on this issue will need to define predictive factors for successful discontinuation of imatinib, as well as of other TKIs including nilotinib and dasatinib, the researchers wrote. “Although imatinib treatment was previously expected to be life-long, our data suggest that, under close PCR monitoring, discontinuation of imatinib is safe in CML patients with a long-lasting molecular response,” they concluded.

http://www.cancernetwork.com/chronic-myeloid-leukemia/content/article/10165/2157668

FOI results on dasatinib for CML patients

I wanted to share the results of the Freedom of Information (FoI) request today. The results speak for themselves but it is concerning that there is such disparity between prescribed medication. For the East Midlands to have funded 74 cases of dasatinib and the East of England to have prescribed none is worrying. This means that depending on which part of the country you live the clinician you see will have a different opinion on the type of drug you receive, perhaps some don’t even bother with dasatinib because of the NICE decision. This has truly become a postcode lottery.

Whilst I am in favour of personalised treatment this level of disparity is not good for patients. We are finalising our Cancer Drug Fund document that provides patients and clinicians information on how to apply for drugs through the CDF. It saddens me to think that there are areas of the UK where patients are not receiving the full range of CML treatments but I will continue to do all I can to ensure that our voice is heard.

Kris

Trusts who have dasatinib on their approved list.

Number of applications for dasatinib on CDF

 

FDA approves Synribo for chronic myelogenous leukaemia

More fantastic news regarding another drug to treat CML. More hope for those who fail on other TKIs. We must ensure our UK health system funds drugs like this as they become available. Kris

The U.S. Food and Drug Administration today approved Synribo (omacetaxine mepesuccinate) to treat adults with chronic myelogenous leukemia (CML), a blood and bone marrow disease.

An estimated 5,430 people will be diagnosed with CML in 2012, according to the National Institutes of Health. Synribo is intended to be used in patients whose cancer progressed after treatment with at least two drugs from a class called tyrosine kinase inhibitors (TKIs), also used to treat CML.

Synribo blocks certain proteins that promote the development of cancerous cells. It is injected just under the skin (subcutaneously) twice daily for 14 consecutive days over a 28-day cycle until white blood cell counts normalize (hematologic response). Synribo is then administered twice daily for seven consecutive days over a 28-day cycle as long as patients continue to clinically benefit from therapy.

“Today’s approval provides a new treatment option for patients who are resistant to or cannot tolerate other FDA-approved drugs for chronic or accelerated phases of CML,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Synribo is the second drug approved to treat CML in the past two months.”

On Sept. 4, 2012, the FDA approved Bosulif (bosutinib) to treat patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies.

Synribo is approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical studies to confirm the drug’s clinical benefit and safe use. Synribo also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.

The effectiveness of Synribo was evaluated using a combined cohort of patients whose cancer progressed after previous treatment with two or more TKIs. All participants were treated with Synribo.

The drug’s effectiveness in chronic phase CML was demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation found in most CML patients. Fourteen out of 76 patients (18.4 percent) achieved a reduction in an average time of 3.5 months. The median length of the reduction was 12.5 months.

In accelerated phase CML, Synribo’s effectiveness was determined by the number of patients who experienced a normalization of white blood cell counts or had no evidence of leukemia (major hematologic response, or MaHR). Results showed five out of 35 patients (14.3 percent) achieved MaHR in an average time of 2.3 months. The median duration of MaHR in these patients was 4.7 months.

The most common side effects reported during clinical studies include a low level of platelets in the blood (thrombocytopenia), low red blood cell count (anemia), a decrease in infection-fighting white blood cells (neutropenia) which may lead to infection and fever (febrile neutropenia), diarrhea, nausea, weakness and fatigue, injection site reaction, and a decrease in the number of lymphocytes in the blood (lymphopenia).

Synribo is marketed by Frazer, Pa.-based Teva Pharmaceuticals. Bosulif is marketed by New York City-based Pfizer.

For more information:

• FDA: Office of Hematology and Oncology Products
• FDA: Approved Drugs: Questions and Answers
• FDA: Drug Innovation
• NIH: Chronic Myelogenous Leukemia

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm325895.htm

More Than a Decade of CML Experience at OHSU Knight Cancer Institute

For the past decade, hematologist/oncologist Dr. Michael Mauro has treated Chronic Myelogenous Leukemia (CML) patients at the OHSU Knight Cancer Institute and worked alongside Dr. Brian Druker on research that has made this a manageable disease for most patients. With rapid diagnostics, a “palette of clinical trials” and a team approach with community oncologists all at his fingertips, Dr. Mauro says it is a joy to see his patients in clinic, to offer them the best care possible and to see them living well with CML long term.

More Than a Decade of CML Experience at OHSU Knight Cancer Institute VIDEO

Credit: Patient Power

Interview with patient advocate Jan Geissler

Cancer survivor Jan Geissler, who lives in Germany, is an active patient advocate globally. In this interview, he shares the reasons he became an advocate and steps he feels others can take to have a voice in their own health care. He also talks about actions being taken in Europe and the progress being made there in giving more patients input into the clinical trial process. Geissler, a chronic myelogenous leukemia (CML) patient, points out the success made in developing new treatments for CML patients and the benefits that knowledge is providing for many other types of cancers. He says great progress has been made, but there is still much more to be done. He advises patients to become informed, join patient support groups and to seek second medical opinions when they feel they are needed.

http://www.patientpower.info/video/a-european-patient-advocate-speaks-out-advice-on-how-to-be-a-powerful-patient?autoplay=1

FDA approves Pfizer leukeamia drug – Bosutinib / Bosulif

Whilst I desperately want to celebrate a new CML drug coming into the marketplace I find it hard when I think about the dasatinib debacle we currently find ourselves in here in the UK. If Pfizer do not offer a PAH we will find ourselves lagging even further behind in CML treatment, this being said even a PAH will not guarantee availability.

This does prove to us that developments are still being made and that we must do all we can to ensure patients get access to the drugs they need, regardless of where they live.

I’ve started off with an excellent video interview with Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan:

In this video interview from ASH 2011, Dr. Carlo Gambacorti-Passerini, a prominent CML researcher from the University of Milan Bicocca, gives us an update on bosutinib, a newer Tyrosine Kinase Inhibitor (TKI) that he has studied in-depth.  Once approved, bosutinib may provide one more option for patients and their physicians to better treat the disease while managing side effects.

WASHINGTON | Tue Sep 4, 2012 5:33pm EDT – LINK TO ORIGINAL ARTICLE

(Reuters) – Health regulators on Tuesday approved a Pfizer Inc pill for a rare type of leukemia, another step in the company’s effort to expand its oncology business.

The medicine, called Bosulif, treats chronic myelogenous leukemia (CML), a blood and bone marrow disease that usually affects older adults. About 26,000 Americans live with the cancer, and 5,430 people in the United States expected to be diagnosed with it annually, the U.S. Food and Drug Administration said.

Most people with CML have a specific type of genetic mutation called the Philadelphia chromosome. This mutation causes bone marrow to make an enzyme that triggers the abnormal growth of white blood cells. Bosulif, known generically as bosutinib, blocks the enzyme’s signal that causes the white blood cells to grow.

“We are seeing improvements in the treatment of CML based on a better understanding of the molecular basis of the disease,” Dr. Richard Pazdur, head of the FDA’s cancer drugs center, said in a statement.

Pfizer’s drug is meant for people who have CML with the Philadelophia mutation who cannot tolerate other medicines, such as Novartis AG’s Gleevec, or whose cancer has stopped responding to the older treatments.

The FDA gave Bosulif orphan status, which means it treats a condition or disease that affects fewer than 200,000 people in the United States. The designation comes with a seven-year period of marketing exclusivity.

The medicine is expected to reach peak global sales of $341 million in 2016, according to the average forecast of analysts polled by Thomson Reuters.

Bosulif is the second Pfizer cancer drug to get the FDA’s nod this year, after its kidney cancer drug Inlyta. Investors are looking for signs of the company’s research productivity, to help replace lost revenue from its cholesterol fighter Lipitor. The world’s top-selling drug began facing generic competition last year.

Shares of Pfizer were largely flat at $23.85 in after-market trading on Tuesday.

(Reporting by Anna Yukhananov, additional reporting by Bangalore equities newsroom; Editing by Leslie Gevirtz)