Nov 7, 2015

Clinical Trials, Big Pharma, the FDA, and Expen$ive Drugs

...turns out this is not always a great combination for patients
Many of us, including this author, are taking one or more of the new and very expensive cancer drugs in our attempt to live longer and have a better quality of life. New and expensive does not necessarily mean we will live longer and better, however. We keep doing this (at least I do) because there is not much in the way of alternatives. We seem to be at the mercy of the most powerful companies in the U.S.

In a new book, Ending Medical Reversal: Improving Outcomes and Saving Lives, Dr. Vinay Prasad looks at ever increasing prices, secretive clinical trial results, questionable procedures, and low drug effectiveness in dozens of the new cancer drugs on the market. Dr. Prasad is an Associate Professor of Medicine at OHSU.

Here are some excerpts from Dr. Prasad’s book in his own words:
"Cancer drugs are outrageously expensive; but they are worth every penny, right?
When you pay a lot for something, you want it to be great. This is true of a new car, a new computer, a new pair of shoes—but it doesn’t seem to be true for cancer drugs.
Recently, we heard that profiteers are raising the price of old, established medicines. For many Americans, Turing pharmaceutical and Martin Shkreli have become four letter words.
But the problem of inappropriate prices is not just a few exceptional cases; it is the norm in cancer medicine.
Before we talk price tag, let’s consider what we get for our money. In medicine, there is always nuance, and it exists here. For example, there are some excellent cancer drugs. Two that come to mind are imatinib—a revolutionary pill that transformed a highly fatal leukemia into a manageable condition—and rituximab—a monoclonal antibody that has improved survival in several cancers. These drugs are expensive, sure, but no one can say they aren’t terrific. But we can say that Novartis, the maker of Gleevec, has raised the price of the drug from an already high $30,000 in 2001 to $76,000 today despite the fact that it costs less than $200 dollars to manufacture a year's supply.
But at least Gleevec is a great drug. Most cancer drugs are not close to being great—they have only a tiny positive impact on longevity and often cause many side effects.
The median improvement in survival for 71 drugs approved by the US Food and Drug Administration for treating solid cancers over the last decade is only 2.1 months. And there are two good reasons to assume that this remarkably short reported extension is in fact an overestimate. First, the average age of cancer patients in the real world is much older than those included in the studies submitted to the FDA —60% of cancer patients are over 65, but they account for just 36% of patients in key trials. Older patients—who are frailer and have other medical problems—experience more side effects from cancer drugs and less benefit.
Second, the majority of cancer drugs approved by the FDA are not included in the 2.1 month figure, because their effects on overall survival are unknown. Amazingly, most new drugs (2/3s) are approved on the basis of improvement of a surrogate endpoint—e.g. a new drug shrinks a tumor on a CT scan. We don't know that this shrinkage actually translates into a longer or a better life.
So, the efficacy of most cancer drugs is minimal and may be even less in the real world. How do we then explain why they are already so ridiculously expensive and increasing in cost at a rate of 10% a year. Cancer drugs now routinely cost more than 100,000 dollars for one year of treatment.
One way doctors measure the value of drugs is to ask how much is the total drug cost to get us 1 year of quality life. Since most drugs just add a few weeks or months, you have to treat many people to get just one year of extra life. By this measure, most cancer drugs are outrageously expensive. A new breast cancer drug costs ~$700,000 for a quality adjusted life year, and a new colon cancer drug costs $900,000. And these figures apply only if you give the drug the benefit of the doubt, and assume it works as well in the real world as it does in FDA studies.
Not surprisingly, Pharma is our most profitable industry—double digit returns ranging from 10% to 42% are routine, despite the overall disappointing benefits of their products.
Reasonable people would say it’s fine to make a tidy profit when you make a great drug—a drug like Gleevec. But we might also say it doesn’t seem fair to keep raising its price over time, as the cost to make it is trivial, and the research and development are already done. Steep price increases feel like profiteering.
Reasonable people might also say that it doesn’t seem right to pay so much for drugs that add so little—and only add that little bit under just right conditions. Especially when the costs to make drugs are so low and profits are so high.
What prevents us from being reasonable people? That is not just a million dollar question, but a tens of billion dollar one. It involves rules that prevent Medicare from negotiating the price of drugs and the intricacies of the patent system. Solutions won’t be simple or easy, but the price of cancer drugs is way too high, and we get far too little for it. The time for reform is now."
As someone who is about to embark on yet another expensive cancer drug experience I applaud Dr. Prasad. I wish him well and hope he can convince enough of the right people (maybe even Congress) to take another look and maybe even take action to decrease drug costs. My life, and many of yours, may depend on it.

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Oct 25, 2015

The World’s Largest Cancer Clinical Trial

The creation of cancer treatment drugs often follows a similar path. Somewhere a researcher or research team independently or as part of a drug company invents a potentially effective drug and a drug company sponsors clinical trials. After many trials at several levels, involving hundreds and maybe thousands of patients, some of the drugs are FDA approved. Conducting clinical trials is expensive and the successful drugs are eventually sold to patients at very high prices. You already know this, of course. But this new clinical trial from the UK has a different twist.

Clinical trials on the cheap
The BBC has reported that Cancer Research UK and NIHR (part of the National Health Service) will be sponsoring a 12-year study of the cancer prevention benefits of ASPIRIN. Yes, common aspirin. It is called the Add-Aspirin Trial

There has been some research to suggest that aspirin can delay or slow the growth of some cancers. This trial will help determine if aspirin can be an effective cancer treatment. A proven inexpensive cancer treatment would be a major benefit to millions. 

11,000 people with early stage cancer—the largest number ever in a clinical trial‑‑will be randomly assigned to one of four groups. One group will take a daily placebo and the other three groups will each take aspirin at a different dosage level. This part of the study will last five years. Participants will be monitored for as long as 12 years. Patients with early stage bowel, breast, prostate, stomach, and esophageal cancers will be included in the study. 

Aspirin is one of the cheapest meds on the market. It was originally developed as a painkiller but is now widely used to help reduce the risk of heart attack and stroke. Maybe there is one more chapter in the aspirin story. 

Aspirin has several documented side effects—as do all drugs‑‑ and would not be appropriate for everyone.

Keep an eye on this study. Not all clinical studies have a good outcome but, if successful, this one could be a ‘game changer.’

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Sep 28, 2015

Cancer Treatment Drugs Approved in 2014 and 2015 by the FDA

Looking  for the latest and greatest medications to treat your cancer? There are a number of drugs approved by the FDA in the past two years and listed by CenterWatch. Never heard of of them? Check them out, talk to your oncologist...

 Drugs Approved in 2015

Farydak (panobinostat); Novartis; For the treatment of multiple myeloma, Approved February 2015
Ibrance (palbociclib); Pfizer; For the treatment of ER-positive, HER2-negative breast cancer, Approved February 2015
Lenvima (lenvatinib); Eisai; For the treatment of thyroid cancer, Approved February 2015
Lonsurf (trifluridine and tipiracil); Taiho Oncology; For the treatment of metastatic colorectal cancer , Approved September 2015
Odomzo (sonidegib); Novartis; For the treatment of locally advanced basal cell carcinoma, July 2015
Opdivo (nivolumab); Bristol-Myers Squibb; For the treatment of metastatic squamous non-small cell lung cancer, Approved March 2015
Unituxin (dinutuximab); United Therapeutics; For the treatment of pediatrics with high-risk neuroblastoma, Approved March 2015
Varubi (rolapitant); Tesaro; For the prevention of delayed nausea and vomiting associated with chemotherapay, Approved September 2015
Drugs Approved in 2014
Akynzeo (netupitant and palonosetron); Helsinn; For the prevention of chemotherapy-induced nausea and vomiting, Approved October 2014
Beleodaq (belinostat); Spectrum Pharmaceuticals; For the treatment of relapsed or refractory peripheral T-cell lymphoma, Approved July 2014
Blincyto (blinatumomab); Amgen; For the treatment of Philadelphia chromosome-negative relapsed /refractory B cell precursor acute lymphoblastic leukemia, Approved December 2014
Cyramza (ramucirumab); Eli Lilly; For the treatment of gastric cancer, Approved April 2014
Imbruvica (ibrutinib); Pharmacyclics; For the treatment of chronic lymphocytic leukemia, Approved February 2014
Keytruda (pembrolizumab); Merck; For the treatment of unresectable or metastatic melanoma, Approved September 2014
Lynparza (olaparib); AstraZeneca; For the treatment of previously treated BRCA mutated advanced ovarian cancer, Approved December 2014
Opdivo (nivolumab); Bristol-Myers Squibb; For the treatment of unresectable or metastatic melanoma, Approved December 2014
Zydelig (idelalisib); Gilead; For the treatment of relapsed CLL, follicular B-cell NHL and small lymphocytic lymphoma, Approved July 2014
Zykadia (ceritinib); Novartis; For the treatment of ALK+ metastatic non-small cell lung cancer, Approved April 2014

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Sep 7, 2015

ASCO 2015 Prostate Cancer Updates from a Roundtable of Experts

What's new and encouraging in prostate cancer treatment and research? This panel of experts, Dr. Bruce Montgomery,Moderator, Dr. Emanuel Antonarakis, Dr. Tomasz Beer, and Mr. Tom Kirk who is President and CEO of Us Too International, explain advances in research and treatment in 2015.
Prostate cancer patients and cancer patients in general will find some hopeful news in this discussion.

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Aug 25, 2015

Cancer Clinical Trials and the Elderly—an Unlikely Combination

Did you know?

Q         What age group is most likely to have Cancer?

A         People older than 65. They represent 13% of the population and more than 60% of all those with cancer.

Q         Who is most likely to die of cancer?
A         Seniors. About 80% of all cancer deaths occur in those older than 65.

Q         What age group is least likely to be represented in cancer clinical trials?
A         Seniors. 

Q         Can seniors respond to cancer medications in a different way than younger patients? (For example, having more toxic side effects.)
A         Yes

Q         So, WHY are seniors with cancer not included more in clinical trials?
A         It’s a long story……

Improving the research base for treating older adults with cancer was a focus of the 2015 ASCO conference. An expert panel presented the problem and possible solutions. The Journal of Clinical Oncology reprinted the complete report and recommendations.

Factors such as meeting eligibility requirements can create hurdles. Many older patients are limited in mobility, have numerous previous treatment regimens, have a more limited predicted lifespan, and take multiple medications (the average is more than 9 for patients over 65). Multiple medications can interfere with trial medications. More than 80% of older patients have a chronic condition (such as diabetes or arthritis) which can complicate cancer treatment. 

Doctors often do not even recommend older patients for inclusion in clinical trials, although Doctor recommendation is the number one predictor of older people choosing to participate in clinical trials.

Many oncologists, such as Stuart Lichtman, MD, Memorial Sloan Kettering Cancer Center in NY, believes that seniors with cancer are being short changed when it comes to cancer treatment because they are under-represented in research.

ASCO makes five recommendations to improve evidence generation in the 65+ population:

(1) Use clinical trials to improve the evidence base for treating older adults (older than 65) with cancer,

(2) Leverage research designs and infrastructure for generating evidence on older adults with cancer (change trial requirements to fit older patients),

(3) Increase US Food and Drug Administration authority to incentivize and require research involving older adults with cancer,

(4) Increase clinicians' recruitment of older adults with cancer to clinical trials (educate doctors to recommend older patients for trials), and

(5) Use journal policies to improve researchers' reporting on the age distribution and health risk profiles of research participants.

Doctors currently have no choice but to use results from clinical trials conducted with people in their 50s to treat people in their 80s.

Research results from clinical trials with older patients could provide a better scientific basis for treating cancer in the elderly. As someone who is elderly and has cancer, this author wholeheartedly supports these recommendations.

Click on the links in this article for a more comprehensive discussion of this issue.

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Aug 13, 2015

Prostate Cancer Research at OHSU--Interview

 Tom's interview with Reggie Aqui at KGW Channel 8 in Portland.

He covered prostate cancer basics, risk, types, and when to get tested.


Post Text Here
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Aug 4, 2015

Advances in Prostate Cancer Treatment this Past Year-ASCO 2015

Dr. Tom Beer attended the 2015 ASCO (American Society of Clinical Oncology) Annual Meeting in Chicago. He wrote a paper about some of the  recent discoveries and important advances in prostate cancer treatment. This is a summary of that paper. You can find the original in the Oncology Journal.
  •  A trial called STAMPEDE (Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy) recruited men starting hormone therapy for high-risk, locally advanced prostate cancer. Multiple arms of the study are testing the addition of various agents to standard hormonal therapy. The results of adding docetaxel, zoledronic acid, or both were reported at ASCO. Nearly 3,000 men have participated in the study. The use of docetaxel, with or without zoledronic acid, significantly increased overall survival. The bottom line‑the use of chemotherapy with hormonal therapy in patients with advanced metastatic prostate cancer was supported.

  • In Radiation Therapy Oncology Group study 0521, the early use of chemotherapy was also evaluated, along with radiation and hormonal therapy, in high-risk localized disease.[2] Six hundred twelve patients with localized prostate cancer and features consistent with a high risk of relapse were treated with radiation therapy and 2 years of hormonal therapy with or without 6 cycles of docetaxel. The addition of chemotherapy resulted in an improvement in the 4-year overall survival rate and improvement in 5-year disease free survival. These results support earlier trials which found that using chemotherapy in the initial management of metastatic prostate cancer increased survival time. Additional follow-up is necessary before this becomes standard treatment.

  • A French study, the GETUG-AFU 16 trial, used radiation in men with a PSA rise after a prostatectomy. A six month regimen of hormonal therapy was added for some of the patients. Progression-free survival was significantly improved in those patients receiving the combination therapy.

  • How soon to begin hormonal therapy in men with rising PSA levels was the focus of another study. Men were randomly assigned to a group that started hormonal therapy in two years or four years. The overall survival rate was significantly higher in the men who began therapy at two years. A concern in using hormonal therapy is toxic side effects. Early study results suggest earlier use of hormonal therapy may save lives in spite of side effects. Larger, more definitive studies are needed to test these results.

  •  A comparison of the use of intermittent hormonal therapy versus continual hormone therapy was the focus of a study conducted by the Southwest Oncology Group (SWOG). One group of older men received continual ongoing hormonal therapy and the other group intermittent treatment. Early results suggest the intermittent treatment group had more medical events over the time of the study than those on continual hormonal therapy.

  • Hormonal therapy is routinely combined with radiation for the treatment of high-risk and most intermediate-risk patients. But in intermediate-risk patients, the combination is not as well studied and questions about the need for hormonal therapy have been raised. A randomized trial that involved 600 participants showed that biochemical and disease-free survival with 6 months of hormonal therapy plus radiation were superior even to results with dose-escalated radiation, providing support for the widely practiced approach of combining these two treatment modalities.


Post Text Here
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker