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Pain Management: Opioid Adherence in Cancer Patients

This week, Real World Health Care speaks with Salimah H. Meghani, PhD, MBE, RN, FAAN. Dr. Meghani is an associate professor and term chair in Palliative Care at the University of Pennsylvania School of Nursing. She is also associate director, NewCourtland Center for Transitions and Health. Her main research interest involves palliative care, specifically understanding and addressing sources of disparities in symptom management and outcomes among vulnerable patients.

We asked her about her study on analgesic adherence and health care utilization in outpatients with cancer pain, recently published in Patient Preference and Adherence. We also discussed the role of non-pharmacological approaches in treating cancer pain.

Opioid Adherence Patterns

Real World Health Care: Last year, you published an article: Patterns of analgesic adherence predict health care utilization among outpatients with cancer pain. Can you provide a brief summary of the article and talk about the study’s implications for cancer patients with pain management issues?

Salimah H. Meghani, University of Pennsylvania School of Nursing

Salimah Meghani: This is the first study to understand how opioid adherence patterns, over time among cancer patients, relate to health care utilization outcomes. We used objective measures of adherence (Medication Event Monitoring System – MEMS) and novel adaptive methods recently validated by the co-author, Dr. George Knafl from UNC-Chapel Hill. We found that inconsistent adherence patterns of analgesics over time was significantly associated with hospitalization over a 3-month observation period. The interaction of inconsistent adherence and strong opioids (WHO step 3 opioids) was one of the strongest predictors of health care use. It should be noted that this was a serendipitous finding. We did not plan to study adherence patterns and health care utilization. It therefore needs validation in hypothesis-driven study.

RWHC: Are you currently involved in any new research programs studying pain management in cancer patients? If yes, can you briefly describe?

SM: Yes, I am studying outcomes of opioid adherence and adherence patterns among cancer outpatients. This is an important topic as few recent U.S. based studies exist on the topic despite all the recent guideline contentions (e.g., CDC guidelines for managing chronic pain including chronic cancer pain and ASCO response) and national policy debates on opioids.

How Patients Manage Cancer Pain

RWHC: What do you think are the biggest challenges facing researchers studying pain management in cancer patients? How can those challenges be addressed?

SM: One of the biggest challenges is that we know very little about how patients manage their cancer pain. We know that opioids are widely prescribed, but we also know that there is poor adherence to prescribed opioids. Other treatments such as acupuncture are not consistently covered by health insurance or lack data on clinical effectiveness. There is a need to understand how patients are managing their cancer pain and what health care systems can do better to address the great burden on unrelieved cancer pain. Future work should also include improving access to effective non-opioid treatments for cancer patients. My previous research has also documented racial and ethnic disparities in cancer pain treatment for African Americans, which requires continued attention.

Safe Opioid Use

RWHC: What do you think are the biggest challenges facing clinicians treating pain in cancer patients? How can those challenges be addressed?

SM: There is a lot of confusion among clinicians about the role of opioids and the safe and rational use of opioids among cancer patients. Unfortunately, there is little empirical evidence base about the outcomes of opioid treatment among cancer patients. A look at the recent CDC guidelines on managing chronic pain would indicate that cancer patients frequently, if not invariably, have been excluded from the studies of the outcomes of chronic opioid therapy. More empirical evidence is needed to help clinicians develop comfort in opioid prescriptions.

Non-Opioid Treatments

RWHC: What do you think is the role of non-pharmaceutical pain management therapies for cancer patients? How can clinicians integrate both pharmaceutical and non-pharma therapies for cancer patients?

SM: I think access to non-pharmacological treatments is the biggest problem. While the NCCN guidelines for cancer pain identify a number of non-pharmacological modalities, they are not readily accessible to cancer patients. I have argued this in a recent letter to JAMA Oncology about the CDC opioid guideline that recommends that non-opioid treatments should be the first line therapy for chronic pain. This paradigm assumes easy and consistent access to non-opioid treatments. Also, access to effective non-pharmacological treatments are very different among poor, minorities, those with limited literacy.

Global Disparities

RWHC: What initially attracted you to this field? What continues to inspire you about it?

SM: My original research interest was global disparities in opioid availability for cancer pain management and the role of the International Narcotics Control Board. After migrating to the United States, I became familiar with racial and ethnic disparities in pain care and the toll it has for patients and families. This work continues to inspire me.

Pain Management for Cancer Survivors

This week, Real World Health Care continues our series on Pain Management by speaking with Judith A. Paice, PhD, RN, who is the lead author for the American Society of Clinical Oncology’s guideline, Management of Chronic Pain in Survivors of Adult Cancer. Dr. Paice is Research Professor of Medicine, Hematology/Oncology, at Northwestern University’s Feinberg School of Medicine and a full member of the Robert H. Lurie Comprehensive Cancer Center. Dr. Paice’s clinical work focuses on the management of cancer-related pain, and her research focuses on the study of chemotherapy-induced peripheral neuropathy. We spoke about the ASCO guideline and the need for clinicians to balance pharmaceutical and non-pharmaceutical approaches to pain management.

Real World Health Care: Why did ASCO issue a guideline for the management of pain in survivors of adult cancer?

Judith A. Paice, Feinberg School of Medicine, Northwestern University

Judith Paice: The oncology field has evolved tremendously in recent years. Not only are people living longer with cancer, but they’re being cured of their disease thanks to some fantastic treatments. These treatments provide good clinical responses, but they can also cause significant toxicity, some of which may lead to chronic pain syndromes. The goal of the guideline was to alert oncologists to the presence of these long-term, persistent pain syndromes. A secondary goal was to provide support for chronic pain syndrome treatment.

Clinician Guidance

RWHC: What are the most important take-aways for clinicians?

JP: First are ASCO’s recommendations for screening and assessment. Second are recommended treatment options, both pharmacological and, equally important, non-pharmacological treatments. The guideline also provides insights and risk mitigation strategies for clinicians around the long-term use of opioids.

Today’s oncologists are faced with a very different pain management phenomenon than they were 20 years ago, when opioids were primarily used at end of life. Opioids are now being used for patients with a much longer survival trajectory — 20 to 30 years or more. As clinicians, we need to ask if such long-term use of opioids is appropriate and safe. How do we go about determining that? The guideline helps oncologists with those types of assessments and decision making.

RWHC: What do you feel are the biggest challenges facing oncologists in managing chronic pain in cancer patients, and how is ASCO helping clinicians manage those challenges?

JP: Our society is facing a serious public health problem in the opioid abuse and misuse epidemic. As a result of this problem, we’ve seen regulations at both the state and federal level that are having a chilling effect on the availability of opioids, even for those in desperate need of these medications. ASCO has a position paper on protecting access to treatment for cancer-related pain that I encourage all clinicians to read. ASCO also advocates for better third-party reimbursement for physical therapy, occupational therapy, cognitive behavioral therapy and mental health counseling. These are crucial therapies for patients facing the “new normal” of cancer survivorship, yet most third party payers provide little or no support for these treatments. As clinicians, we need to help our patients maintain function and cope with the fact that their lives are going to be very different. For patients, it’s more than surviving cancer. It’s about finding their own inner strength in survivorship.

New Pain Management Treatments

RWHC: Where do the biggest opportunities lie for new pharmaceutical pain management treatments?

JP: Several new findings in the laboratory may lead to novel agents that do not produce opioid related adverse effects. This is promising, assuming the findings can be translated into a clinical setting. There have also been numerous compounds that proved effective in animal models of pain, but when moved into the clinical setting, they either had adverse effects that weren’t seen in animals, or they didn’t have the efficacy they presented in the lab. It is very difficult to develop a model of cancer pain in animals.

Unfortunately, there haven’t been many completely new drugs. Most of the agents approved recently are slight variations of existing compounds or an update in the delivery method: a spray instead of a tablet, for example. The industry has been more focused recently on abuse-deterrent compounds. This is a somewhat controversial area because while such formulations prevent people from crushing, snorting or injecting the drugs, they don’t keep people from taking more than what is prescribed.

Non-Pharmacologic Therapies

RWHC: What should the role be for non-pharmaceutical pain management therapies in treating cancer patients?

JP: For quite a long time, there was a tendency in medicine to rely only on pharmaceutical therapies. This made sense when patients did not have long-term survival prospects and when managing pain meant helping the patient get from their bed to a chair. Today, cancer patients are living longer. They want to get back to work and function safely without the risk of falls and other complications.

We’ve seen good data around the usefulness of physical therapy, occupational therapy and cognitive behavioral therapy for many chronic pain situations, including cancer-related pain. These non-pharmacologic therapies must go hand-in-hand with pharmaceutical therapies.

Part of the challenge with non-pharmacologic therapies is limited reimbursement. The other big challenge is getting buy-in from patients. Most of us want a quick fix. Redefining expectations can be difficult. Physical and occupational therapy can be demanding, and access to specialists who understand the special needs of those surviving cancer are in short supply. Also, there remains a stigma attached to seeing a mental health counselor. It’s important for cancer patients to know that they aren’t “weak” if they need support to help them cope with the physical and emotional challenges of being a cancer survivor. Our field needs to do a better job educating our patients about the importance of including non-pharmacologic therapies as part of our pain management repertoire.

NSCLC: Targeting What Drives People’s Cancer

This week, Real World Health Care talks with Edward B. Garon, M.D., Associate Professor of Medicine at the David Geffen School of Medicine at UCLA Health. He specializes in hematology and oncology, with an interest in lung cancer and chest malignancies.

Dr. Garon’s research focuses on the testing and development of targeted therapies and immunotherapies in the treatment of non-small cell lung cancer (NSCLC), including the development of a class of drugs known as PD-1 (programmed cell death-1) inhibitors, which allow immune cells to eliminate cancer. We spoke with Dr. Garon about checkpoint inhibitors, immunotherapies and targeted therapies for NSCLC.

Real World Health Care: Describe your role at UCLA’s David Geffen School of Medicine, especially as it relates to research of non-small lung cancer.

Edward Garon, MD, UCLA Health

Edward Garon, MD, UCLA Health

Edward Garon: I serve as director of thoracic oncology and conduct both clinical and laboratory research with a focus on translational research to determine lung cancer patient subgroups that are most likely to respond to certain therapies. Instead of looking at NSCLC as one disease, it’s important to personalize therapy and give people the therapies that are appropriate, not just for the disease site origin, but for a disease that’s driven by a particular set of molecular events.

RWHC: What do you think are the biggest challenges relating to NSCLC research and how are those challenges being addressed?

EG: We’ve seen some real progress in NSCLC research, especially in terms of immune checkpoint inhibitors, which unleash a patient’s own T cells to kill tumors. But we’re not yet where we want to be. One of the biggest priorities is identifying more people who will respond to therapies and connecting the right research with the right patient population, especially since targeted therapies currently only apply to a small percentage of the patient population. Although early-phase lung cancer studies in non-metastatic patients have hinted at the potential to use biomarkers to select patients, data from clinical studies have tempered expectations.

RWHC: What do you think are the biggest challenges relating to current NSCLC treatment and how are those challenges being addressed?

EG: In the past 10 years, there has been a push to individualize care for NSCLC, to evaluate individual tumors on individual patients and determine if there are molecular changes or abnormalities in the tumor itself that can dictate whether there are certain therapies that are more or less likely to be effective in any given patient. Treatments for NSCLC have improved somewhat over time, but in patients whose tumors have progressed during or after their initial therapy, the outcomes for additional treatment have been quite poor.

Another challenge for clinicians is the emergence of checkpoint inhibitors, immunotherapies and targeted therapies. We currently have two PD-1 inhibitors available for treating advanced NSCLC, both of which are well-tolerated among patients. The quality and duration of responses to anti-PD-1 therapy can be profound in NSCLC, but some clinicians are not overly familiar with them and how to use them. Much of the experience with these drugs is concentrated in select academic centers. We need wider clinician awareness of which patients are most likely to benefit from therapy, when therapy should be stopped and how toxicity should be managed.

RWHC: Where do you think the biggest opportunities for future advances in NSCLC research and treatment lie?

EG: We will soon see a tremendous amount of data on the combination of checkpoint inhibitors and additional agents. It will be interesting to see both what the data from randomized studies show and how researchers interpret that data in terms of what constitutes a signal and what doesn’t. Careful selection of patients, doses of each agent, and information supporting strategies — concomitant or sequential — is still needed. Another exciting avenue is the potential incorporation of immunotherapy in early-stage disease, locally advanced disease and in first-line therapy for metastatic disease. These agents could become the frontline choice for select patients with stage IV disease versus standard chemotherapy.

RWHC: Why did you get into this field and what continues to inspire you about it?

EG: I became involved in lung cancer as a young physician coming from fellowship training. While there was not a lot of excitement in the field at that exact moment, I saw a good opportunity to be on the leading edge of therapy development. I am fortunate here at UCLA to be part of many of the studies of new drugs that have changed the course of patients’ disease and don’t have the toxicity associated with many chemotherapies. It’s certainly been gratifying to see how new therapies can positively impact patients. Just a few short years ago, NSCLC was seen as a disease that wasn’t particularly immunogenic. Ten years from now, I hope to look back on this exciting time and realize that we have come much farther still.

NSCLC: The Promise of Immunotherapy

As part of our series on non-small cell lung cancer (NSCLC), Real World Health Care spoke with Hossein Borghaei, D.O., in the Department of Hematology/Oncology at Fox Chase Cancer Center, which is part of the Temple Health System. Dr. Borghaei serves as Chief, Thoracic Medical Oncology; Director, Lung Cancer Risk Assessment; and Associate Professor. He specializes in endobronchial disease, lung cancer, lung metastases, mesothelioma and thymoma and conducts research in molecular therapeutics.

Dr. Borghaei was the lead investigator of the CheckMate 057 study, which helped to introduce a new immunotherapy paradigm in lung cancer treatment.

Real World Health Care: Tell us about your role at Fox Chase Cancer Center, especially as it relates to the research and treatment of non-small cell lung cancer (NSCLC).

Dr. Hossein Borghaei, Fox Chase Cancer Center

Dr. Hossein Borghaei, Fox Chase Cancer Center

Hossein Borghaei: I’m a medical oncologist by training, with a special concentration in lung cancers. I treat patients at all stages of the disease and have run a number of clinical trials. Some of those trials have been investigator-driven, while others have been funded by the industry. I’m also involved in the Eastern Cooperative Oncology Group which does NCI-funded translational and clinical research. I also have a small research lab that does pre-clinical investigations, working with other investigators to find new ways to treat cancer patients with new or existing drugs.

RWHC: Can you share some highlights of your recent NSCLC research?

HB: The most interesting, impactful and attention-getting study I’ve been involved with recently is related to immunotherapy. This was a Phase III study in which we found that non-squamous NSCLC patients can live significantly longer with an immunotherapy drug called nivolumab than they can with single agent chemotherapy. The immunotherapy treatment has been approved, allowing physicians to use it to manage patients when there is a progression of the disease after platinum doublet chemotherapy. We also found that this immunotherapy resulted in fewer grade 3 or 4 adverse events.

We recently presented a follow-up to the study in which we found that, after a two-year time point, nearly double the previously treated non-squamous NSCLC patients and nearly triple the previously treated squamous NSCLC patients were alive compared with those treated with chemotherapy.

RWHC: What do you think are the biggest challenges in NSCLC research?

HB: We need more funding. NSCLC is a disease that affects a large population. It’s the number one cause of cancer deaths in the U.S. and it’s a very difficult disease to treat. Having adequate funding to study NSCLC is important. There are a number of drugs being investigated to treat NSCLC, so we also need patients who can participate in rationally designed clinical trials that can address specific questions and help to bring new treatments to the marketplace. There is certainly a tremendous amount of interest in evaluating new treatment options, but investigators running clinical trials are struggling in some cases to find the right patient population to study.

RWHC: What do you think are the biggest challenges relating to current NSCLC treatment?

HB: One of the biggest challenges relating to treatment comes back to the ability of patients to participate in clinical trials. Many trials are conducted in academic centers like Fox Chase Cancer Center, making it difficult for patients in remote geographic areas to participate. Even for patients who live close to a clinical trial location, they may have co-morbidities such as emphysema or COPD, making it physically challenging to participate.

Another challenge we face as clinical researchers is our ability to obtain biopsies from NSCLC patients. Biopsied tissue from tumors at different phases of the disease is critical for our ability to understand why some treatments work on some patients but not on others, and every biopsy has its risks. I’m hopeful that the emerging field of liquid biopsy — which will allow us to do molecular-level testing on blood samples — will help us overcome this challenge.

RWHC: What do you think have been the most important advances in NSCLC treatment over the past decade?

HB: Molecularly targeted therapies that allow clinicians to personalize cancer treatments have been successful for about 25 percent of lung cancer patients. Our ability to understand what’s going on in a tumor at a molecular level lets us better target specific drugs to treat and manage the disease.

RWHC: Why did you get involved in this field?

HB: As an oncology clinician, I really get to know my patients on a personal level. A cancer diagnosis is life-altering, and as a treating physician, I get to address my patients’ concerns and fears. I find that closeness extremely rewarding. From a research standpoint, there is such a huge need to understand the disease process and so many patients that we can’t yet cure. I want to contribute to our overall understanding of this disease and why it’s so difficult to treat. The research opportunities in NSCLC are almost limitless.

Non-Small Cell Lung Cancer Focus of New Real World Health Care Series

I am not a clinician, a cancer survivor, or a research expert, however I know the bad hand cancer deals to people like you and me every day.  While I have seen the devastating effects of cancer diagnoses through my work with HealthWell, I am hopeful that the combined efforts of advocates, researchers, and clinicians will continue to move treatment options forward and remission rates up.

Krista Zodet, President, HealthWell Foundation

Krista Zodet, President, HealthWell Foundation

HealthWell has been honored to assist patients receiving treatment for lung cancer since 2006 and this cancer, like many, continues to surprise me with its twists and turns.  According to the American Lung Association, lung cancer is the leading cancer killer in both men and women in the U.S. It has surpassed breast cancer to become the leading cause of cancer deaths in women, and it accounts for approximately 27 percent of all cancer deaths. Almost everyone knows someone who has been affected. That’s why RealWorldHealthCare.org has decided to focus on non-small cell lung cancer (NSCLC) as our next topic.

Non-small cell lung cancer (NSCLC) accounts for about 80-85 percent of all lung cancers and afflicts about 180,000 people in the United States each year. A number of factors can increase risk of developing NSCLC. Smoking cigarettes or being exposed to secondhand smoke is a primary risk factor for the disease. Exposure to asbestos, radon and certain paints or chemicals may also increase risk.  The scariest stories, though, are those where no clear risk factors exist.

NSCLC has five stages, from stage 0 to stage 4 in order of increasing severity. Outlook and treatment is based on the stage, and because stage 4 cancer is typically not curable, treatment is usually aimed at relieving symptoms. However, targeted therapies have been developed that attack specific aspects of the cancer cell, like growth factors or blood vessels that feed the tumor. Each year, tens of thousands of people are cured of NSCLC in the U.S.

Over the next couple months, we will be focusing on some of these targeted therapies and other therapies designed to treat NSCLC. We’ll be interviewing top researchers in the field as well as leaders of patient advocacy organizations dedicated to helping patients and their families manage the disease.

We invite you to check back to learn more about NSCLC research priorities and challenges. You can also sign up to receive email alerts when new interviews are posted. Just enter your email address under the sign-up message to the right.

If you or someone you love is on Medicare and suffering from NSCLC, HealthWell may be able to help with medication copayments and insurance premiums. Visit our eligibility page to learn more.

Big Data in Health Care: Speaking with Dr. Clifford Hudis

Real World Health Care is pleased to bring you the final interview in our series on Big Data and its impact on health care. Here, we spoke with Dr. Clifford Hudis about how Big Data will impact cancer care. Dr. Hudis is Chief, Breast Medicine Service, Department of Medicine, Memorial Sloan Kettering Cancer Center; Vice President for Government Relations and Chief Advocacy Officer for MSKCC; and Professor of Medicine, Department of Medicine, Weill Cornell Medical College. He also serves on the Board of Governors of the American Society of Clinical Oncology’s CancerLinQ project.

Real World Health Care: In a recent article, you write that big data represents a new opportunity to increase our understanding of cancer care. How is that so?

Clifford A. Hudis, MD Chief, Breast Cancer Medicine Service

Clifford A. Hudis, MD
Chief, Breast Cancer Medicine Service

Clifford Hudis: The ongoing conversion of medical record keeping in oncology from paper-based records to electronic format means that for the first time in history we have potential access to the treatment and outcomes for the vast majority of adults with cancer who are not treated on prospective clinical trials. This means that we can explore treatment effects including both efficacy and toxicity in patients who might not have participated in the usual, tightly controlled, prospective studies that are used to gain regulatory approval. For example, older (or younger) patients, those with co-morbidities, other malignancies, and so on — all of whom are frequently under-represented in prospective drug-development trials — can be studied.

RWHC: What sort of knowledge gaps do you think big data will be able to identify in the area of cancer care?

CH: Key gaps include toxicities and efficacy in special populations, but also use of drugs “off label” based on either classical histopathologic tumor features or newer genomic testing. Another key area is to study drug-drug interactions or drug-genotype interactions.

RWHC: Can you give us an example of how big data has overcome a known limitation of randomized clinical trials in evidence development?

CH:         In other disease areas, such as interventional cardiology, large registries have allowed clinical investigators to refine their understanding of the benefits and harms of specific approaches without the use of conventional prospective randomized trials.

RWHC: What are some of the biggest challenges facing the health care industry in terms of its ability to use big data to improve health care delivery, treatment optimization, and cost containment?

CH:         They key challenges may be outside the realm of big data per se. We have a societal challenge in the uniform definition of benefit, efficacy and ultimately value. This is especially true in oncology where drug development costs are high, many diseases are life-threatening, and the pace of innovation has to continue to accelerate. It is possible that big data will allow us to gain deeper and faster insights into some of these issues as new treatments first permeate the treatment arena. At a more mundane level, we would benefit from even greater interoperability and standardization of data storage and access.

RWHC: Much of the literature published on the use of big data in health care focuses on cancer care. Why is cancer care such a ripe area for implementing big data initiatives?

CH: Among the reasons are the myriad diseases — and therefore complexity — that comprise cancer, the acuity of the illness, the broad reach, and the large price we pay in overall public health. In the face of this massive set of challenges, only three percent of adults participate in clinical research that defines and advances the standards of care. To accelerate progress, we need to innovate in the area of data development. Big data is one key opportunity in that regard as it simultaneously offers to provide new insights, broaden the distribution of evolving knowledge, and improve the efficiency of the entire drug development enterprise.

RWHC: How has the use of big data impacted you personally in your practice?

CH: We increasingly have access to patterns of care, treatment decision-making, and patient outcomes across a large and geographically distributed group of clinicians and investigators working in one traditional disease are.  All of this can be used to improve patient care in an iterative fashion.

 

Categories: Big Data, General

Big Data Declares a War on Cancer

In 1970, cancer was the second-leading cause of death in the United States. President Nixon made fighting this disease a priority in his 1971 State of the Union address: “I will also ask for an appropriation of an extra $100 million to launch an intensive campaign to find a cure for cancer, and I will ask later for whatever additional funds can effectively be used. The time has come in America when the same kind of concentrated effort that split the atom and took man to the moon should be turned toward conquering this dread disease. Let us make a total national commitment to achieve this goal.”

DataSocietyLots of great progress has been made over the past 45 years. Many challenges remain, but the technological capabilities have vastly improved.

In his last State of the Union address, President Obama re-iterated Vice President Joe Biden’s plea for a concerted effort to use the brightest minds in the U.S. to cure cancer, and announced the creation of a national cancer moonshot. President Obama asked Vice President Biden to be “in charge of Mission Control.” “For the loved ones we’ve all lost, for the family we can still save, let’s make America the country that cures cancer once and for all,” Obama said.

The good news is that today there is a massive amount available for cancer researchers to use in their mission. The challenge is that due to the lack of reporting standards and the disparate databases, much of the data is left un-analyzed, which can lead to lots of missed opportunities for breakthroughs.

Since President Obama’s declaration, Vice President Biden has met with leaders of the MD Anderson Cancer Center at the University of Texas, which in 2012, launched the Moon Shots Program aimed at reducing cancer mortality. There are many types of cancers. While they are all driven by gene mutations in various cells, every type of cancer requires a targeted approach. The Moon Shots Program has many mini-projects, or Moon Shots, aimed at treating specific cancers.

The program’s innovation is driven by the multitude of specialists involved in the project, from clinicians to biostatisticians and programmers. The Moon Shots include research into B-cell lymphoma, glioblastoma (brain cancer), cancers caused by the human papillomavirus (HPV), high-risk multiple myeloma, colorectal and pancreatic cancers, breast and ovarian cancers, chronic lymphocytic leukemia, lung cancer, melanoma, myelodysplastic syndrome/acute myeloid leukemia and prostate cancer. It covers an unprecedented number of diseases by one effort.

Cancer is a complicated ailment with complex treatments. A single tumor can have more than 100 billion cells and each cell can have different genetic mutations. The mutations are not constant over time, which requires an evolving treatment. To understand each cancer, clinicians need to understand the kinds of mutations that are driving it. There are 3 billion code letters, or amino acids, in each cell so understanding the mutations expressed in each tumor is no small task. There are as many as 300 billion opportunities for mutation in just one tumor.

With so much complexity, there are many ways to approach cancer research. For example, scientists at the NIH have used network analysis methods to map out protein interactions to discover new biomarkers and significant players in the cell’s architecture. These discoveries help guide clinical studies and other research on gene expression.

Researchers across Moon Shots programs are using machine-learning models to predict whether a patient has various types of cancer based on the expression levels of specific genes. Implementation for thyroid cancer has been especially fruitful. Thyroid cancer usually causes a lump at the base of the neck, and around 5 to 15 percent of these lumps are malignant. By measuring gene expression at the lump the machine-learning model is able to predict with greater than 90 percent accuracy whether it is malignant or benign. The work was published in Clinical Cancer Research in 2012.

Protein data is not the only kind of information used by researchers. Scientists at Case Western University have used machine-learning techniques on Magnetic Resonance Images (MRIs) of breast cancer patients to predict if a patient is suffering from aggressive triple-negative breast cancer, slower-moving cancers or non-cancerous lesions with 95 percent accuracy. Today’s capabilities of image analytics can significantly augment the insights gleaned from lab tests. The challenge with cancer is getting a full picture.

Text stored in medical records is another powerful source of relatively untapped data. Modern natural language processing capabilities can analyze massive amounts of unstructured data and combine the results with structured research and clinical information. Combining doctors’ notes versus numerical lab tests, for example, can give context to the condition and symptoms of the patient at various stages of different cancers.

Medical records include a treasure trove of data. Factors such as family histories, clinical test results and genomic data are stored in repositories across the world. The challenge is combining all that data in one database.

“Big data is not just big. The term also implies three additional qualities: multiple varieties of data types, the velocity at which the data is generated, and the volume seen within MD Anderson,” says Keith Perry, associate vice president and deputy chief information officer.

One of the ambitious objectives of the MD Anderson Cancer Center is to collect and combine patient information including a profile of their genetic makeup, clinical histories, test results, treatment courses and treatment responses. This data will be interpreted by the massive data analytics, which provide real-time decision support to rapidly improve clinical outcomes. This is a much more challenging task than meets the eye.

When the startup Flatiron Health launched with an ambitious goal to improve cancer treatment, one of the largest obstacles they faced is the inconsistency of records from various Electronic Health Record systems (EHRs).

With over $100 million in backing from Google Ventures, Flatiron is facing this basic problem: when measuring the level of a single protein commonly tested in cancer patients, a single EMR from a single cancer clinic showed results in more than 30 different formats. There are over 100 different kinds of protein and genetic tests, biopsies, and other diagnostic methods used in cancer care. And all the various EMR systems out there report these metrics in different ways. This is an incredibly complex data integration problem. So much so that Flatiron purchased Altos Solutions, which makes an EMR service for oncology practices. This allows the company to control the data collection process.

Finding cures and treatments for various types of cancer is truly a Big Data problem. And the ability to collect, store, share and analyze the data cohesively is still in relevant infancy. This isn’t a problem you can solve with just one approach. Whether using network analysis, text mining or other machine learning techniques, the task is a true inter-disciplinary challenge that requires numerous types of expertise and really Big Data.

Big Data and machine-learning don’t hold all the keys, human analysis and contextualization is key. Yet these technologies are starting to shine the light on how humanity will fight one of the most potent killers on the planet. President Nixon’s initiative gave us the Frederick Cancer Research and Development Center, an internationally recognized center for cancer research, and has achieved many breakthroughs. President Obama’s initiative has the potential to revolutionize the state of cancer treatment. We’ll make a comparison in 45 years!

What Happens When an Investigational Drug Cures Some Patients, but not Most?

David Sheon

David Sheon

To date, 179,158 clinical studies have been registered on ClinicalTrials.gov. Though the majority of these trials result in unsuccessful attempts to bring a drug to market, an untold number of lives have been saved or extended as a result of the successes.

But how many lives were saved by drugs that did not meet their clinical endpoint? Recently, the attention of the medical community has been drawn to “exceptional responders.” These are patients who, contrary to expectation, respond extremely well to drugs that are not found to be effective (or even safe) for use in the general patient population studied in a clinical trial.  The study doesn’t meet its endpoints, but a small number of patients thrive.

With the advent of relatively cheap and easy genetic sequencing, researchers are beginning to better understand why exceptional responders do so well on drugs that have little to no benefit for most patients.

In one case, featured recently in an article in The New York Times, a patient has been living four years with a cancer she was told was untreatable, because she responded well to an experimental drug. Of the seven people given the drug, she was the only one who responded. Sequencing her tumor’s genes, the researchers were able to determine that her response was due to the natural presence of a protein that her cancer needed in order to grow.  The experimental drug stops the production of the same protein. Doctors and researchers have been perplexed by these types of cases for years, yet have only recently been able to examine why this is occurring. Researchers at the National Cancer Institute have begun a study to “understand the molecular underpinnings of exceptional responses to treatment.”

Regardless of the science behind it, there is a very real, ethical dilemma raised by the existence of these exceptional responders. If the drug does not get approved, and especially if the company producing the drug  loses investors and goes out of business because the drug trial didn’t meet its endpoints, exceptional responders will have no way of accessing a treatment that could well save their lives just because of an anomalous genetic makeup. There is nothing in place to guarantee that these patients will be able to access their treatments, even if there are no other alternatives.

According to Wayne Pines, President of Regulatory Services and Healthcare at APCO Worldwide and former Associate Commissioner for Public Affairs at the FDA, however, this issue is not so clear-cut.

“There are a lot of factors that go into a decision as to what an individual patient should do.  Each decision has to be individual,” he said. “We must also take into account the fact that clinical trials are essential to determine if a new drug works and is safe, and there are limits to how much of an experimental product can be produced.”

All of this would seem to indicate that a blanketing policy for dealing with individual patients is not where we have to focus. The presence of the exceptional responder makes it difficult to determine a drug’s efficacy, especially when researchers cannot explain the patient’s response.

“If reasonable endpoints have not been met, then the question is whether the drug has the potential to work,” Mr. Pines said. “Again, this is a decision that has to be made on specific facts, not on the basis of a set policy. A general one size fits all answer to these kinds of questions does not work.”

Ultimately, more research must be done on these cases before making decisions about how to deal with them. With recent advances in molecular testing, hopefully the day is near when we can understand what causes a patient to respond exceptionally, and where to go from there.

What do you think? Should there be policies set in place to protect these patients? If not, how can we ensure that patients can access the drugs they need, when they need them? Let us know in the comment section.

Categories: Access to Care, General

Cancer Doesn’t Care. Please Help on #GivingTuesday.

ECRFjumboCancer Doesn’t Care.

It doesn’t care that a patient can’t afford pain medication or the expense of getting to a treatment center. Cancer Doesn’t Care that some patients are forced to drain their child’s college savings fund or choose between an imaging scan and buying groceries.

Cancer Doesn’t Care. Do you?

Today is #GivingTuesday, a global day where families, individuals, and businesses will come together with a common purpose: giving thanks and helping others.  #GivingTuesday is an annual opportunity to celebrate generosity and donate or volunteer for your charity of choice.  Show that you care about cancer patients by giving to HealthWell’s Emergency Cancer Relief Fund (ECRF).

Whether or not you can make a donation, we’d like your help to build awareness for the Emergency Cancer Relief Fund. One of the easiest ways to help is to share our graphics and messaging (below) with your networks.

1. Please join me in support of @HealthWellOrg’s Emergency Cancer Relief Fund. Click here to donate today: http://bit.ly/cancerrelief #CancerRelief

2. Join us on #GivingTuesday to help #cancer patients cover critical out-of-pocket expenses. http://bit.ly/cancerrelief #CancerRelief

3. Provide meaningful comforts to cancer patients by supporting the Emergency Cancer Relief Fund: http://bit.ly/cancerrelief #CancerRelief

With your support, we will be able to open the Emergency Cancer Relief Fund to assist qualified cancer patients so they can better manage their road to recovery…when waiting another day is not an option.

We hope that you will consider helping HealthWell. Together, we can make the Emergency Cancer Relief Fund a reality. Learn how you can make a difference in the life of a cancer patient.  Join us in showing that you care by donating and reaching out to friends and family through your social network.

This Holiday Season, Give Emergency Relief to Cancer Patients

ECRF_Facebook_wig_boost“You have cancer.”

Those devastating words send chills through us all. As the initial shock subsides the reality of unanticipated expense deals a second, unwelcome blow.

We know from assisting more than 70,000 cancer patients with the cost of their treatments just how financially overwhelming and destructive a cancer diagnosis can be. Cancer Doesn’t Care that a patient can’t afford their pain medication or the expense of getting to a treatment center.  Cancer Doesn’t Care that some patients are forced to drain their child’s college savings fund or choose between an imaging scan and buying groceries.

Cancer Doesn’t Care, but we do.

That’s why during this season of giving, we are proud to announce the launch of our “Cancer Doesn’t Care” giving campaign to raise the remaining funds needed to launch the Emergency Cancer Relief Fund (ECRF). The ECRF will allow us to provide immediate grants to qualified cancer patients to assist them with meaningful comforts, such as anti-nausea medicine, travel to and from an appointment, wigs and other hidden expenses.  Giving is as easy as clicking here.

The out-of-pocket critical costs of cancer can be financially devastating. According to a Duke University Medical Center and Dana-Farber Cancer Institute study, out-of-pocket, cancer-related costs averaged $712 a month. Further, the study found that about 30 percent of respondents said their expenses were a “significant burden” and 11 percent called those expenses a “catastrophic problem.”

The “Cancer Doesn’t Care” campaign asks individuals, celebrities, and corporations for financial and social media support. We’ve created powerful social media graphics and messaging that can be shared to highlight the many ways that cancer affects patients financially.

Cancer patients must pay for so much more than treatment, and every little bit makes a difference. Please help make the holidays a little brighter by donating, and reach out to friends and family through your own social media networks. Share the messages we’ve prepared or create your own.  Even a small donation can make a difference.

Please help ease the burden for someone who has received these three devastating words by asking your social networks to give. On behalf of HealthWell and the thousands of cancer patients we serve, I thank you for caring.

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The HealthWell Foundation is an independent non-profit that provides financial assistance to underinsured Americans to help them afford life-changing medical treatments (and sponsor of this blog).