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Research Priorities for the Growing Population of Childhood Cancer Survivors

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About Our New Blog Series:
Regardless of political affiliation, most cancer survivors can agree that the specific concerns of survivors received little attention during the presidential campaign. With the election now over, NCCS is inviting cancer survivors from around the country to highlight those specific needs, and propose a cancer care and research agenda to the new Administration and the new Congress.
Guest Post by Greg Aune, MD, PhD

Guest Post by Greg Aune

Post by Greg Aune, MD, PhD
@gregoryjaune

In the aftermath of 2016’s historic Presidential election, one fact remains unchanged: cancer continues to be a devastating array of diseases that has touched or will touch the lives of virtually every American. As a 25-year survivor of childhood cancer, I want to insure that the health challenges faced by patients like me are emphasized as research priorities for the coming years are established.

Cancer is most certainly not a Republican or Democratic issue, yet solving the challenges facing cancer patients in the United States will require ongoing collaboration from our newly elected executive administration and the 115th Congress. Going forward, it is imperative that we continue to capitalize on the recent momentum established by the National Cancer Moonshot and other evolving collaborative efforts in the cancer research and clinical care umbrellas.

Pediatric cancers include an array of biologically unique cancers that are rare and many are exclusively seen in children. For these diseases, it is imperative that we continue to study the scientific underpinnings of these cancers and use that knowledge to develop novel, targeted less toxic therapies. But, it is becoming clear that the development of novel therapies for pediatric cancers is not by itself likely to solve the problems faced by the growing numbers of childhood cancer survivors.

“As our new leaders begin to set their policy priorities, it is important to highlight critical research areas that have the potential to improve the quality of life for childhood cancer survivors.”
In the field of pediatric oncology, we are at the beginning of a new era where five-year survival rates from pediatric cancer are becoming a less important measure of our success. Quality of life is receiving an increasing amount of attention from pediatric oncologists, patients, and their families.

Because the development of new drugs for pediatric cancers lags behind efforts in adult cancers, it is additionally important to invest in new research areas that focus on perfecting the administration of our existing chemotherapy and radiation regimens. Currently, there is a dearth of knowledge pertaining to the effects pediatric chemotherapies and radiation have on normal organs such as the heart, brain, liver, and kidneys.

Survivors of childhood cancer are a growing population that is expected to number 500,000 in the United States by 2020.[1] This growing number is the product of steadily increasing 5-year survival rates for childhood cancer that began in the 1940s when Sidney Farber first introduced systemic chemotherapy to the anticancer armamentarium.

In the first decade of the 21st century, epidemiologic evidence began to emerge confirming what was long expected—that curative regimens for childhood cancer are associated with tremendous long-term cost and increased risk of severe morbidity and mortality. Numerous analyses from childhood cancer cohorts in the United States and Europe have highlighted the shortcomings of successful childhood cancer therapy.

In the United States, the Childhood Cancer Survivor Study (CCSS) has served as a central resource to quantify the health problems faced by childhood cancer survivors. Analyses of outcome data collected from over 34,000 long-term survivors have clearly established a link between childhood cancer therapy and an increased risk of severe health effects including secondary cancers, cardiovascular disease, strokes, endocrinopathies, infertility, neurocognitive deficits, and pulmonary diseases. More important are the numerous studies that have clearly shown that long-term survivors of childhood cancer have a markedly increased mortality rate when compared to age-matched control groups.

Our new leaders must understand that the plight of childhood cancer survivors has become a major priority with patients and their families. With increasing numbers of children surviving their cancer, the devastating long-term consequences of successful treatment are now quantified. A recent study of survivorship patient cohorts indicated that by age 50, 60% of childhood cancer survivors will be either dead or suffering from a life-threatening health complication caused by their pediatric cancer therapies.[2]

As our new leaders begin to set their policy priorities, it is important to highlight critical research areas that have the potential to improve the quality of life for childhood cancer survivors. With this in mind, I would like to identify three broad research areas that I believe, if funded and expanded, would lead to an overall healthier population of childhood cancer survivors.

We must develop and emphasize:

  1. Preclinical laboratory models of cancer survivorship. – The bulk of our knowledge in health outcomes for childhood cancer survivors has been obtained from retrospective cohort studies that have largely focused on correlating specific treatment exposures with the development of specific health problems later in life. Thus, we currently have minimal knowledge that details how chemotherapy and radiation damage normal tissues and lead to late effects. While efforts to develop targeted less toxic therapies are ongoing, in the near term this strategy is unlikely to eliminate our use of the current curative regimens. Therefore, going forward, it is vital that we develop robust preclinical research models to address this major gap in our oncology research portfolio.
  2. Research exploring the premature aging phenotype observed in many childhood cancer survivors. – There is emerging evidence that suggests survivors of childhood cancer age prematurely. Several studies of survivor cohorts have documented elevated risks of age-related conditions such as cardiovascular disease, secondary cancers, and possibly dementia. The biological basis of these epidemiologic observations is mostly unknown. Thus, we must invest in research that is aimed at understanding how early chemotherapy and radiation exposure alter normal aging processes in long-term survivors.
  3. Applying precision medicine concepts to late treatement-induced toxicities. – With the announcement of the Pediatric MATCH (Molecular Associated Therapy Choice) Trial, efforts to harness the promise of genomic sequencing to deliver tumor-targeted therapy for pediatric cancers have moved to the forefront. These efforts are largely focused on utilizing rapid sequencing technology to identify targetable mutations in pediatric cancers and thereby deliver treatments targeting these changes within an individual patient’s tumor. In contrast, the same sequencing technology can be utilized to identify the underlying germline background that mediates how an individual patient responds to standard chemotherapy regimens and results in toxicities. Studying our current range of pediatric cancer therapies from this perspective must be systematically and prospectively initiated in all new childhood cancer patients.
Watch Dr. Greg Aune Share His Survivorship Story

Video: Dr. Greg Aune Describes His Treatment Experience »
“I think my story kind of makes people take pause.”

If adequate funding is applied to these areas, the life trajectory for our childhood cancer survivors can be changed for the better. In fact, the National Cancer Institute (NCI) has already identified these three areas of research as major priorities—preclinical models and aging research are included in the recently established list of pediatric provocative questions and precision medicine has been a major focus of the NCI for over two years. With these areas clearly identified, our new leaders could send a powerful bipartisan message of support for cancer survivors, by setting aside additional funds specifically for these research priorities.

Researchers, clinicians, patients, families and advocates must continue to diligently pursue a new day in cancer care—one where curative regimens are available for all childhood cancers and all survivors are living a life free from health problems caused by successful cancer treatment. Our new leaders have the power to make this day a reality.

About the Author

Gregory J. Aune, MD, PhD, is a 25-year survivor of Hodgkin’s Lymphoma. He is a policy advisor for NCCS, a St. Baldrick’s Foundation Scholar, and the Stephanie Edlund Distinguished Professor in Pediatric Cancer Research at Greehey Children’s Cancer Research Institute in San Antonio, TX.

Twitter: @gregoryjaune
Website: https://ccri.uthscsa.edu/GAune.html
Research Gate: www.researchgate.net/profile/Gregory_Aune

The views & opinions expressed in any guest post featured on our site are those of the guest speaker and do not necessarily reflect the opinions & views of the National Coalition for Cancer Survivorship. Read our blog and comment policies here.

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WCOE: Traveling for Treatment, Bipartisan Cancer Policy, Some Tumors Evade Targeted Therapy, Treatment vs Hype, Clinical Trials Info

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What Caught Our Eye (WCOE)
Each week, we recap the cancer policy articles, studies, and stories that caught our attention.

More From “What Caught Our Eye” »

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Video Post: Dr. Keith Flaherty Discusses Genetics of Cancer, Targeted Therapies, and Clinical Trials

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Dr. Keith T. Flaherty gave the keynote talk at our 2015 Spring Cancer Policy Roundtable where he addressed the issues of the genetics of cancer and personalization in cancer treatment. Dr. Flaherty later joined us to further discuss these topics and the need for understanding the biologic basis of an individual patient’s cancer.  While we have defined cancers based on their site of origin (e.g., lung cancer, breast cancer, colon cancer, etc.) for decades, Dr. Flaherty notes that we must redraw the lines based on molecular features and shared biology in order to make advancements in targeted therapy.

VIDEO TRANSCRIPT:

I’m Keith Flaherty, a medical oncologist at Massachusetts General Hospital Cancer Center in Harvard Medical School, where I direct the Termeer Center for Targeted Therapy. As we deliberate in this forum on the topic of precision medicine and what progress we’ve made and progress yet to make in terms of clinical care and clinical research in this new era, there are a couple of realities that we have to wrestle with. One is that the notion of trying to understand the biologic basis of an individual’s cancer and assigning them to therapy means that we need to largely dissolve the definitions that we’ve used for decades now—defining cancers based on their site of origin—treating lung cancer, breast cancer, and colon cancer as distinct entities and increasingly think about classifying these cancers based on their molecular features or their shared biology.

In other words, we find that there are subsets of certain cancer types, so a ten percent subpopulation of breast cancer that have a similar biology and even genetic makeup to five percent of colon cancer, and maybe a handful of patients with a certain type of sarcoma. When we think about redrawing the lines, or the definitions of cancer along those lines, and assigning patients to, at first, investigational therapy in the context of clinical trials, what we’re learning over and over again is that defining cancers based on therapeutic opportunity, as opposed to site of origin, is a much more useful or operational principle, to think about the idea of making advances in cancer outcomes in the present era.

Much of the progress that’s been made in this regard is with genetic testing. In the first ten years or so of the so-called targeted therapy era in cancer, going from about the year 2000 to 2010, single genes of interest were assayed using research methods to try to point patients to clinical trial opportunity. But as we’ve understood the cancer genome in more depth and as technology has been brought to bear so that we can take what were laboratory-based methods into a routine pathology clinical setting, we’re able to decode the cancer genome in a more complete way, and not only look at single genetic alterations, but really large scale number of genetic alterations that would point a single patient to a unique therapeutic option and another patient with the same cancer type to a potentially totally different opportunity.

Now this is a liberating moment to be thinking about moving to this notion of looking broadly for genetic alterations and as we think about other molecular definitions of cancer, we’ll proceed down the same path of not just wanting to look with the blinders on at a single analyte. It also has forced a change in clinical-trial design. Where we increasingly find the need to set up a portfolio of trials, or even a single umbrella trial that basically will receive patients regardless of which genetic or molecular feature they have. In other words, triage patients based on these next-generation diagnostic strategies to one of many different therapeutic opportunities.

It used to be that clinical trials were really walled off one from another, and increasingly we need to break down those walls, and not just by redefining cancer from site of origin to molecular definition, but also really thinking about how to capture or offer clinical trial participation to a broader audience.

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About the Contributor: Dr. Keith Flaherty is director of the Henri and Belinda Termeer Center for Targeted Therapies at the Mass General Cancer Center and Associate Professor of Medicine at Harvard Medical School. Dr. Flaherty’s research and clinical focus is therapies for melanoma, with a particular expertise in targeted therapies.

The views & opinions expressed in any guest post featured on our site are those of the guest author and do not necessarily reflect the opinions & views of the National Coalition for Cancer Survivorship. Read our blog and comment policies here.

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Guest Post Series: Dr. Michael Ybarra Offers Innovator Perspective on Oncology Care Model

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The Centers for Medicare and Medicaid Services (CMS) recently announced the Oncology Care Model, a new payment model for physician practices administering chemotherapy that aims to provide higher quality, more highly coordinated oncology care at a lower cost to Medicare. This week, we’re presenting several different perspectives on the Oncology Care Model and how it will impact patients, providers, and cancer therapies. Dr. John Cox also shared his thoughts earlier this week, as did NCCS CEO Shelley Fuld Nasso. Dr. Michael Ybarra, Senior Director, Alliance Development, PhRMA is the author of this post.

New Approaches to Paying for Cancer Care Will Benefit from a Patient Advocacy Perspective

The words “you have cancer” strike fear into our hearts. For patients, that news is often followed with emotional discussions of treatment options and confusing insurance lingo, which adds to the enormous stress patients and their families already suffer with a cancer diagnosis.

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Post by Dr. Michael Ybarra, Senior Director, Alliance Development, PhRMA

With an emotional diagnosis like cancer in particular, patients want to rest assured that they are receiving the best, evidence-based care and have access to clear information about their treatment. Insurers and health care providers are experimenting with new models of care with the aim to improve quality and reduce the costs of cancer care. Medicare is now testing several of these models through its Center for Medicare and Medicaid Innovation (CMMI), including a recently announced model for cancer care. While these reforms have the potential to generate savings and improve quality, there is also a risk they will reduce quality and access to treatments. Patient advocates need to engage in the development and assessment of these models to ensure they support the high quality individualized care for patients.

Earlier this year, CMMI began developing the Oncology Care Model for Medicare as part of the federal government’s goal to transition Medicare payment from fee-for-service to “alternative payment models” or “value-based payments.” In our current health care system, doctors are paid based on what they do, or “fee for service.” If a physician sees a patient in the office, and he or she does a procedure, they get paid for performing that procedure. Alternative payment models instead seek to pay providers based on the quality of care they provide and its cost.

Thanks to the advocacy of groups like the National Coalition for Cancer Survivorship, the Oncology Care Model includes several requirements that are intended to improve patient experience in cancer care. These include requirements designed to promote care coordination and keep patients out of the hospital, like providing access to a clinician 24 hours, 7 days a week and designing care plans according to the standards endorsed by the Institute of Medicine. However, there is still work to be done to ensure that the aspects of the model that are designed to reduce the cost of cancer care also support better care for patients.

“One of the challenges with this approach is that each patient is different, and CMMI will be setting cost benchmarks with only limited information about the type of cancers treated by the practice.”
CMMI’s new Oncology Care Model will still pay physicians fee-for-service, but with incentives to reduce the cost of care. When a cancer patient starts chemotherapy, a six month “episode” would be triggered. During the episode, the doctor will be paid an additional $160 per month for care management to support the enhanced services they are required to provide, like 24/7 patient access. Over the course of treatment, they will also be eligible for bonus payments based on their ability to reduce the cost of care during the episode.

One of the challenges with this approach is that each patient is different, and CMMI will be setting cost benchmarks with only limited information about the type of cancers treated by the practice. Oncology is a rapidly evolving field, and research shows that many cancers are different on a molecular level. New precision medicines increasingly allow us to target treatment, but what works for one patient may not work for another. Cost benchmarks will need to be flexible to recognize these differences.

The short, 6 month window to achieve savings also raises some concerns because physicians will have limited tools at their disposal to reduce costs and will only get “credit” for reducing costs in the episode, not in the long term. For example, if a new drug comes to market that’s twice as expensive as the old drug but it’s potentially better, the doctor may choose not to prescribe that one because the cost is higher. Ideally, the Oncology Care Model would be designed to accommodate advancements in cancer care and recognize oncology practices that make changes that improve their patients’ quality of life over the long term.

Medicare’s new Oncology Care Model is an experiment, and several questions remain about how the model will work and what the impact will be on patient care. The decisions that CMMI will make as it finalizes the Oncology Care Model over the next few months will have implications for patients, and we want the patient voice to be heard. We applaud groups like National Coalition for Cancer Survivorship for their advocacy on this issue, particularly around care coordination and patient involvement in decision making on new policies. As Medicare tests and evaluates the OCM, continued involvement by patient advocates is critical to ensuring that reforms balance patient access, quality, and cost control.

About the Author: Michael Ybarra, M.D. is a board-certified emergency physician and Senior Director of Alliance Development at PhRMA. In his capacity at PhRMA, Dr. Ybarra leads alliance outreach to provider, multicultural, and LGBT organizations. His issue areas include communications with health care professionals and delivery reform. In addition to his work at PhRMA, Dr. Ybarra works clinically in the Emergency Department at MedStar Georgetown University Hospital. The views & opinions expressed in any guest post featured on our site are those of the guest author and do not necessarily reflect the opinions & views of the National Coalition for Cancer Survivorship. Read our blog and comment policies here.

 

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WCOE: Patient-Centered Care, “The Fault in Our Stars” and Genetic Data

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What Caught Our Eye (WCOE) Each week, we take a closer look at the cancer policy articles, studies, and stories that caught our attention.
“A focus on the patient and support for continued progress needs to be part of the equation.”
Three articles caught our eye at NCCS this week.  The one that raises the most questions for us in terms of policy development is from our friend, Amy Abernathy, appearing as a blog post for the June 10th issue of The Hill. Her argument in support of patient-centered care is something we have advocated for over 20 years.  Keeping the patient at the center of cancer care is recognized as a common-sense approach to best practices, as well as a way to achieve payment reforms for oncology that reward health care providers for delivery of such care.  Dr. Abernathy’s critical point about the cost/value equation is even more compelling, as the word “innovation” is often used as a term to suggest very expensive new therapies are akin to scientific breakthroughs. In fact, the science to back up these words is often more seductive than the hoped-for benefit, not to mention the “sticker shock” from the price of many of these new therapies.  Dr. Abernathy argues, “The promise of these advances doesn’t mean we should not address cost – progress comes at a price. But it does mean that, as we work on this important issue, a focus on the patient and support for continued progress needs to be part of the equation.  Value = Quality/cost x appropriateness for the individual.” We agree with this assessment and would add that payers (Medicare and private insurers) must set standards that will cover evidence-based care that is responsive to individuals.  The use of these “targeted” therapies must not have the consequence of financial toxicity for families.  One way to help manage this difficult conversation is to equip physicians with ways to talk about cost and value.  This begs another question which is to determine if it is the role of an individual patient in the midst of making a critical decision about their care to be engaged in the value debate, or is this the role for patient advocates to tackle?

Susan Gubar continues to give us much food for thought as she contributes another in a series of Well Blogs for The New York Times.  In the June 12th blog, Susan writes about the film “The Fault in Our Stars,” a highly anticipated movie made following the enormous popularity of a young adult book of the same name.  Those of us at NCCS who read the book and now will see the movie, can only hope that this story which was so well told by author, John Green, will leave viewers with more than wet gobs of Kleenex as they leave the theater.  As Susan says, “the cast of characters in this movie speaks to all ages.”  We agree, and encourage you to read Ms. Gubar’s blog posts.

The June 10th publication of Nature draws our attention to the public/private debate over databases that contain genetic information on individuals that serve as essential elements for the future development of targeted therapies for many diseases. The importance of the accuracy of this information to inform medical decisions based on the validity of this genetic material cannot be overstated. Issues over the proprietary nature of privately held data have been debated for well over a decade.  The article points out, “In genetic testing, the size of the reference database matters: the bigger it is, the more useful it becomes for interpreting the results of any individual gene test… Only by collecting data from many different people can scientists observe the same variants often enough to make these kinds of calculations with confidence.”  This doesn’t clear up the confusion over recent rulings in court cases dealing with whose genes are these, but it raises the larger questions regarding data sharing where full cooperation among companies is encouraged.

Post by Ellen Stovall.

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