GRACE :: Cancer Basics

Frogs in boiling water: On breaking the $10,000 barrier

Note: Novartis has provided funding to GRACE for our recent ALK-positive patient forum.

Last week, the FDA approved Zykadia (ceritinib), the second generation ALK inhibitor.  As I wrote in my post about this new agent its rapid approval as the first effective treatment for acquired resistance to a targeted therapy in advanced lung cancer, there should be little question that it provides a helpful new option.  A couple of days later, I learned the cost: $13,500/month.

I felt some sticker shock over this. After initially being shocked at the price of EGFR inhibitors at around $5000/month, then having then escalate every few years, we saw the approval of XALKORI (crizotinib), setting a new pace in lung cancer, at $9800/month. Though that represents a heady range, we cou;d also potentially justify the cost by saying this was a very limited population and that criotinib provided a profound benefit. 

I was shocked about the cost of Zykadia, at $13,500, which made the $9800/month cost of XALKORI seem quaint, like a relative bargain. I expressed my concerns to the folks at Novartis, saying that I thought the price was aggressive and approaching extortionate, especially after the FDA approved the drug just a couple of years after it began phase II testing, based on just 140 patients, not the typical requirement of large, expensive phase III trials over many years that provided the justification for the high cost of these drugs in the past.

To their credit, they were very responsive and got back to me about my concerns.  They made several points that made me feel as though the question of cost and value for cancer agents is a fair concern, but also that their pricing was a thoutful process, what they estimated as fair market value relative to other agents and not just a simple opportunity to demand the absolute most that could be obtained.

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The Spectrum of Cancer Progression (50 Shades of Progression)

Here’s a general summary of a thoughtful approach to how we might assess progression of disease, recognizing that it isn’t just a simple matter of a “yes/no” question of progression or not.  

And for those who want the pdf to print, here it is: 50 Shades of Cancer Progression

Feel free to leave questions. comments, objections, etc. here.I hope it’s helpful.

 

 


How a Cancer Adapts: Key Clinical Implications from the Evolution of an Advanced Cancer

Key Clinical Implications of how a Cancer Evolves from H. Jack West

The associated, printable pdf is here.


All about subgroups: Why do we value the results in some trial subgroups but not others?

Here’s an important summary of how to interpret results from cancer trials that are increasingly hyped to the general public. You need to be a knowledgeable consumer of this information.

Over the past decade, one of the biggest developments in lung cancer, and in fact many kinds of cancer, has been the identification of important subgroups within the broad categories. Where 10-12 years ago we categorized patients as “advanced NSCLC” and didn’t get much more granular than that, we now routinely look for differences depending on different tumor histologies (adenocarcinoma, squamous, or other), the presence or absence of driver mutations, perhaps even smoking status. We expect to see differences, and in many cases identification of specific subgroups within a larger trial has led to major changes in treatment recommendations. At other times, however, you may see/read cancer news highlights that describe results as being significantly different for one group or another in a trial that was otherwise negative. What makes the results of one subgroup analysis credible and practice-changing, while oncologists view others with far more skepticism?

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Distinguishing clinical from statistical significance: Are cancer trials aiming high enough to make a real difference?

Back in August, a press release came out about a new monoclonal antibody against EGFR, called necitumumab, which is a “humanized” antibody that is expected to lead to fewer infusion/hypersensitivity reactions than Erbitux (cetuximab), which is another antibody against EGFR.  This press release from Lilly, the makers of necitumumab, was about the SQUIRE trial, a randomized phase III trial in previously untreated patients with advanced squamous cell NSCLC, in which patients were randomized between cisplatin/gemcitabine with this new agent or placebo.   The press release noted that the trial was positive and demonstrated a statistically significant survival benefit. GREAT!

So why hasn’t there been any discussion of it? First, this agent is remarkably similar to Erbitux, which has been heavily tested and hasn’t found its way into treatment of lung cancer. But the bigger issue may be related also to Erbitux, ,and that is that it highlighted the distinction between statistical and clinical significance. 

Specifically, a trial called FLEX came out several years ago that compared standard chemo with Erbitux to standard chemo with placebo and was actually technically a positive study, showing a survival benefit that was statistically significant. This means that the difference of the two arms  in the primary endpoint, the main variable of the trial, was great enough that there is a less than 5% chance that this is due to random chance alone.

But there is a difference between statistical significance and clinical significance. If a trial is large enough, there can be a statistically significant but actually quite unimpressive in actual terms. Conversely, a smaller trial may fair to be statistically significant but still show results that are extremely promising.  In the case of the FLEX trial, the study showed an improvement in median overall survival of about 5 weeks, which is arguably meaningful, but when you factor in the weekly visits for IV Erbitux, the side effects, and the cost of over $50,000/year for the drug, this agent has failed to lead the world to be sufficiently impressed enough to have Erbitux become a leading consideration in advanced lung cancer.

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Institute of Medicine: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part VI: Translating Evidence Into Practice, Measuring Quality, and Improving Performance

Welcome to part V; part I addressed the current landscape of cancer care, part II addressed patient-centered communication and shared decision-making, part III addressed the work force caring for patients with cancer, part IV addressed the evidence base for high quality care and part V focused on information technology (IT).  The final chapter of the IOM’s report focusses on translating evidence into practice, measuring quality and improving performance.  The reports summarizes existing measures of quality, but notes that none are adequate—“No current quality reporting program or set of measures adequately assesses cancer care in a comprehensive, patient-oriented way.”

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Institute of Medicine: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part V: A Learning Health Care Information Technology System in Cancer Care

Welcome to part V; part I addressed the current landscape of cancer care, part II addressed patient-centered communication and shared decision-making, part III addressed the work force caring for patients with cancer and part IV addressed the evidence base for high quality care.  Part V focuses on chapter 6 of the IOM’s report.  This chapter focuses on information technology (IT).  The potential for IT to improve the quality of health care and the pace of research was dramatic and mostly unrealized.  Medical records systems were not primarily designed to improve the quality of care, improve coordination of care, or facilitate research to advance care in the future.  Rather, they were designed around billing and coding.  Most cannot even speak to each other.  So, as far as information sharing between providers go, IT has not significantly improved the situation of our grandparent’s generation, when paper charts ruled.

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Institute of Medicine: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part IV: The Evidence Base for High-Quality Care

Welcome to part IV; part I addressed the current landscape of cancer care, part II addressed patient-centered communication and shared decision-making and part III addressed the work force caring for patients with cancer.  Part IV, to come, will address the evidence base for high quality care.  The IOM says that the current evidence base is inadequate in that it does not address many pressing clinical questions.  While there is a problem in medicine in general, the complexity of cancer care makes the problem particularly bad in our world.

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Institute of Medicine: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part III: The Workforce Caring for Patients with Cancer

Introduction

Welcome to part III; as you will recall, part I addressed the current landscape of cancer care and part II addressed patient-centered communication and shared decision-making.  Part IV, to come, will address the evidence base for high quality care.

 

The IOM says that coordinated, experienced professionals should deliver cancer care and that current practice falls far short of this gold standard.  Much of the fourth chapter addresses the current workforce shortages among many of the professionals involved in providing cancer care and why the problem will get worse.  Ironically, some of the drivers of the problem are side effects of changes that are otherwise desirable.  The chapter spends the greatest focus on physicians, but also gives attention to nurses, pharmacists, advanced practitioners (nurse practitioners and physician assistants) and family caregivers.

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IOM: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part II: Patient-Centered Communication and Shared Decision Making

The institute of medicine (IOM) who put out this report deserves credit for bringing patient-centeredness to national attention.  They put out a very famous report in 2011 called Crossing the Quality Chasm: A New Health System for the 21st Century that defined patient-centeredness as, “providing care that is respectful of and responsive to individual patient preferences, needs, and values, and ensuring that patient values guide all clinical decisions.”  For those who want to read more about the definition of patient centered care and shared decision making, I refer you to the very beginning of chapter 3 of the report.

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