The American Society of Clinical Oncology (ASCO) is very concerned about the escalating cost of cancer care, and since 2007 has been actively pursuing solutions that address

this issue. The unsustainable increase (projected to reach $175 billion per annum by 2020, an increase of 40% from 2010) is the result of:

• an increasing demand for cancer care services (45% new cancer cases estimated by 2030), largely due to aging of the population and due to lifestyle factors

• continued growth of new interventions and treatments without pricing, relative to value.

The overall burden that rising cancer costs place on the national economy translates into enormous strain at the individual level, and may ultimately impede access to lifesaving care. Patients and their families often face crippling expenses during what is already one of the most difficult times in their lives. Furthermore, medical costs are the leading cause of personal bankruptcy, and

people with cancer are 3 times more likely to file for bankruptcy than those without cancer.

It’s important to remember that cost is only 1 component of what really is a question about the “value” of cancer care. Value is defined by more than cost—it includes the clinical outcomes patients can expect from any specific treatment regimen, as well as consideration of costs and side effects. The overall value of care reflects the benefits in quality and quantity of life gained against the physical, emotional, and financial costs of medical intervention.

ASCO has launched a strategic new value initiative to:

• obtain optimal outcomes at lowest cost

• develop a framework or methodology to assess the relative value of treatment options based on:

ASCO believes that medical decision making must be the result of informed discussions between patients and their physicians in the context of the local care delivery system—with the patient’s

preferences and goals at the center. ASCO’s Board of Directors is developing the value framework to help physicians and patients determine the most appropriate evidence-based high-quality treatment option, considering the patient’s clinical status, preferences, and personal and family needs. To achieve the broadest consensus, ASCO plans to solicit input from:

• ASCO members and other oncology providers

• patients and patient advocacy groups. 

Articles

Evidence-Based Oncology

ASCO's Initiative to Define Value In Cancer Care

Clinical care pathways in oncology have been discussed for some time as a strategy for controlling healthcare costs. These “road maps” guide the treatment protocol depending on how the disease progresses. Pathways have supporters and opponents, but they are gaining notice: WellPoint announced a $350 per patient per month incentive on May 27, 2014, in 

 during the annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago.

WellPoint’s pathways initiative began in 6 states on July 1, 2014. With that effort set to begin, 

 convened a panel, with voices from the payer and provider realms, to discuss how pathways are changing cancer care. Seema Sonnad, PhD, associate editor and director of Health Services Research at The Value Institute at Christiana Care Health System, led the discussion.

Joining Sonnad were ASCO President Peter Paul Yu, MD, who is director of cancer research at Palo Alto Medical Foundation; Jennifer Malin, MD, PhD, medical director for Oncology Care Management, WellPoint; Chadi Nabhan, MD, FACP, associate professor of medicine, hematology and oncology section, and medical director, Clinical Outpatient Cancer Center, University of Chicago; and I.W. Tischler, MD, national medical director, oncology, Cigna.

When the president of ASCO says that healthcare spending is “unaffordable” and “unsustainable,” it’s little surprise that both insurers and leading physicians are looking for ways to bend the cost curve. In cancer care, clinical pathways are gaining ground among payers and some providers, although how well they will work and how much they will save remains unknown.

“We have an unaffordable, unsustainable healthcare system,” Yu said at the start of the discussion, describing the challenge that confronts cancer care. With an aging population, the prospect of more cancer patients, and new, expensive drugs to treat them, how does the system allocate limited resources? “We need to get a handle on the rising costs of care,” he said.

What are pathways? Nabhan said there’s a “gray area” between clinical guidelines, such as those developed by the National Comprehensive Cancer Network and ASCO, and pathways, which account for real-world situations like side effects, and, of course, the cost of therapy. Not all pathways are alike, the panelists agreed; some direct toward a “single best treatment,” while others offer 3 to 4 options. And, once a patient progresses past second- or third-line therapy, the judgment of the oncologist starts to replace the protocols. Pathways are still a new concept, and several speakers credited US Oncology with both pioneering the concept and publishing data from the experience.

Pathways differ from guidelines in that they confront cost, a factor that physicians historically avoided; it was not considered ethical to do so. But with the rising costs of cancer care, and the out-of-pocket expenses patients face, doctors and health plans have little choice. The new term “financial toxicity” refers to the effect that these burdens have on patients, and their families, as they undergo treatment. Yu and Tischler agreed that oncologists had to be good stewards of this realm. “This is going to enhance the patient experience,” Tischler said. “We are really stewards not only of our individual patients, but of the population as well.”

Pathways seek to reduce the variability out of the system, not only to control cost, although that is a goal, but also to improve care by bringing more focus to evidence-based guidelines, panel members agreed. That said, pathway compliance will never be 100%, nor should it be; each patient is different, and pathways must provide enough leeway for clinicians to tailor treatment toward individual needs and responses to treatment. The panelists agreed that about 80% compliance to predetermined pathways could be expected.

Yu called for improvements in technology, and better integration of electronic health records with pathways, which he said would allow oncologists to record why they deviate from pathways. This could lead to more frequent updates and improvements in care.

What will make pathways successful? Payer and provider representatives on the panel agreed that “buy in” from treating clinicians was crucial. “You have to have people believe that it’s a transparent process of pathway development that they respect and feel they had a voice in,” Yu said. Whether institutions use pathways developed by an outside vendor or build them in-house, it’s essential to have representation from “across the spectrum of academic and community medicine” of care, he said. “That helps build trust into the decisions,” said Yu. He believes that the medical literature doesn’t always reflect the reality on the ground, and providers can bring that reality into pathway design.

Malin said WellPoint considered using an outside vendor for its pathways but ended up using in-house expertise, consisting of oncologists and pharmacists, who developed the protocols in a process not unlike that used to place therapies on a formulary. Internal teams of oncologists and pharmacists put together summaries based on efficacy, quality of life, and cost. The external advisory group members came from community and academic oncology practices, including National Cancer Institute—designated institutions, she said. WellPoint’s pathways are available through an online portal, and forms of feedback come both weekly and quarterly, Malin said.

The essential element of WellPoint’s program is that it offers rewards, not penalties, Malin said. If providers deviate from the pathway, which is anticipated, “They will still get reimbursed,” she said. “But if they are on the pathway, (they are eligible for) additional reimbursement for treatment planning and care coordination.”

Yu said incentives are preferable to penalties at this stage, since pathways are new. “You’re trying to change people’s thinking in a positive manner,” he said.

What about the most expensive drugs? Malin said that while WellPoint’s pathways plan aims to bend the cost curve, it will not keep cutting-edge therapies away from patients if the evidence shows they save lives.

“Our pathway is about rewarding quality care,” she said. “We’re not going to exclude something from the pathway simply because it’s more costly. Pathways, like the rest

of clinical medicine, is an iterative process—a groundbreaking therapy that is going to change the care for a large percentage of patients with a particular tumor type&mdash;that would need to be put in the pathway right away.” By contrast, a therapy that would only affect a small share of patients would be added more slowly; for other therapies, as evidence accumulates, a therapy may have better or worse toxicity or effectiveness, and thus may be added or dropped, Malin said.

Among the challenges, and opportunities, of pathways is the fact that the evidence needed to include therapies in practice might be based on different criteria than in a clinical trial. More emphasis might be placed on toxicity and other side effects, or on “value,” which involves survival-versus-cost calculations, and that’s where judgments get complicated.

“You can’t exclude an expensive drug just because it’s expensive,” Nabhan said. His main concern is that it can take time to gather evidence to determine the value of a new therapy; what is the right amount of time to include a drug in the pathway?

Yu emphasized that pathways must be dynamic, living vehicles that are constantly revised to include new findings. This is where better integration with technology will be critical, he said: if a regimen that was well-followed suddenly falls out of compliance with the pathway, it would be an indication that there’s a new study that should be reflected in a pathway update.

As Nabhan noted, “We don’t have a metric to decide, ‘What is the cost of one life saved for how long?’ We really don’t—that is the elephant in the room&mdash;it’s critical to address at some point.” 

1. Mathews AW. Insurer push to rein in cancer spending. 

. http://online.wsj.com/articles/insurer-to-reward-cancerdoctors-for-adhering-to-regimens 1401220033. Published May 27, 2014. Accessed July 2, 2014.

Panel Discussion led by Seema Sonnad,PhD, that saw participation from ASCO's president Peter Paul Yu, MD; Jennifer Malin, MD, PhD, from WellPoint; Chadi Nabhan, MD, FACP, from the University of Chicago; and I.W. Tischler, MD, from Cigna.

AJMC Panel Asks: Does it Pay to Use Pathways?

The insurer WellPoint made news in late May when it promised to pay $350 per month per patient to those oncologists who adhered to its cancer care clinical pathways.

 While the use of pathways is not new, this particular incentive appears to be a game changer, albeit one that not all think is a good idea.

Smaller oncology practices, which already face competitive disadvantages under healthcare reform, will struggle if they must keep track of multiple pathways for different insurers, said Ted Okon, executive director of the Community Oncology Alliance. “The idea that each payer has a different pathway is not sustainable; it’s totally unworkable,” he said. Okon told 

 he was aware of a practice that now has 5 different pathways it must track; WellPoint’s program alone includes protocols for breast, lung, and colorectal cancer.

The WellPoint program, which began July 1, 2014, in Kentucky, Indiana, Missouri, Wisconsin, Ohio, and Georgia, will expand nationwide throughout 2015.

 The initiative follows the release of studies over the past 2 years that show pathways can help reduce costs without harming the quality of care. In May 2013, 

 published a study by Cardinal Health Specialty Solutions, with Bruce A. Feinberg, DO, as the lead author, that showed an oncology pathways program could trim cancer care costs by 15% and reduce hospital admissions by 7%.

 More recently, UnitedHealth announced results from a 3-year pilot study involving 810 patients that produced a 34% reduction in cancer care costs. The study was published in the 

But because pathways seek to narrow treatment options beyond guidelines published by the National Comprehensive Cancer Network, the prospect of every health plan having its own protocols for reimbursement has unsettled some oncologists, especially those outside major hospital systems. Community oncologists lack the infrastructure to juggle multiple pathways protocols, Okon said, and he warned that this trend could be “another nail in the coffin” of community care. Meanwhile, other studies show that cancer drug costs, which are a prime target of savings in pathways, soar if the patient is treated in a hospital or hospital-owned clinic instead of a community practice.

“I understand WellPoint’s drive toward trying to get a handle on costs,” Okon said. However, he said, “Pathways are not a panacea. They are a tool.” If too much judgment is taken from individual physicians, “It’s a slippery slope to a regimented formulary.”

Okon warned that with the onset of the Affordable Care Act, there are multiple pressures on community oncologists. Because many newly insured selected the lower-cost silver or bronze plans, pressure will mount to use narrow networks and formularies to control the costs of care. “We’ve only seen the tip of the iceberg,” he said. 

1. WellPoint’s new quality initiative aims to improve cancer outcomes, promote access to evidencebased and cost-effective care [press release]. Business Wire: Indianapolis, IN; May 28, 2014. http://ir.wellpoint.com/phoenix.zhtml?c=130104&p=irol-newsArticle&ID=1934999&highlight=.

2. Feinberg BA, Milligan S, Cooper J, et al. Third-party validation of observed savings from an oncology pathways program. 

3. Study: new cancer care payment model reduced health care costs, maintained outcomes [press release]. Minnetonka, MN: UnitedHealth Group; July 8, 2014. http://www.unitedhealthgroup.com/Newsroom/Articles/Feed/UnitedHealthcare/2014/0708CancerCarePaymentStudy.aspx.

4. Newcomer L., Gould B, Page RD, Donelan SA, Perkins M. Changing physician incentives for affordable, quality cancer care: results of an episode payment model [published online July 8, 2014]. 

5. Rabin RC. Chemo costs in US driven higher by shift to hospital outpatient facilities. Kaiser Health News. http://capsules.kaiserhealthnews.org/?p=28074. Published May 6, 2014. Accessed August 4, 2014.

WellPoint Initiative May Challenge Some Practices

With a consumer-focused approach, the second-largest healthcare provider in the United States has announced plans to change its corporate name to Anthem, Inc, the

 The merger in 2004 between Anthem Inc and WellPoint Health Networks Inc created the giant conglomerate that is WellPoint Inc.

In the press release making the announcement, Joseph Swedish, president and CEO of WellPoint said, “As consumer engagement is heightened, we recognize that brand—an indicator of trust and a predictor of willingness to engage&mdash;is going to be of increasing importance. We believe it is important to call ourselves by the name that people know best&mdash;Anthem. Changing the corporate brand to Anthem is an important expression of our commitment to serve as a trusted partner in health.”

Following the merger, WellPoint used the Anthem brand to cover 14 of its Blue-plan states, while the WellPoint name was used in investor materials and social media activities.

 Following implementation of the Affordable Care Act, insurance marketplaces (exchanges) were created across states, which saw a tremendous rise in WellPoint’s enrollment numbers early this year. “There’s been a major pivot to a more consumer-focused approach. The Anthem brand is better-recognized among consumers, and we’re not going to swim upstream any longer,” said Swedish in an interview with the Wall Street Journal.

1. WellPoint announces intent to change corporate name to Anthem, Inc [press release]. Indianapolis, IN: WellPoint, Inc; August 13, 2014. http://ir.wellpoint.com/phoenix. zhtml?c=130104&p=irol-newsArticle&ID=1958094&highlight=.

2. Company history. WellPoint website. http://www.wellpoint.com/aboutwellpoint/companyhistory/. Accessed August 13,2014.

3. WellPoint changes its name to Anthem. 

 website. http://online.wsj.com/articles/wellpointchanges-its-name-to-anthem-1407877202. Published August 12, 2014. Accessed August 13, 2014.

WellPoint Hopes a Name Change Will Ensure a New Tide of Consumers

It’s a concern that only comes when cancer therapy succeeds: as the length of survival for the typical multiple myeloma patient has nearly tripled, the number of patients living with the disease has also increased.

 Today, this blood cancer that was once a blip on the radar screen for insurers is slowly catching their attention as the cost of care increases, both for individual patients and gradually, across payment systems.

 recently convened a panel that included clinicians and representatives from the payer community and the Multiple Myeloma Research Foundation (MMRF). The group discussed the rapid advances in diagnosis and treatment of the disease. But as patients are living longer and therapy regimens become more complex, the cost of care is rising for this still relatively uncommon blood cancer.

co-editor- in-chief Michael Chernew, PhD, were Shaji K. Kumar, MD, professor of medicine, Mayo Clinic; Jack Goldberg, MD, clinical professor of medicine, Penn Presbyterian Medical Center; Gene Reeder, RPh, PhD, director, Managed Care Networks, Xcenda; and Anne Quinn Young, MPH, vice president, Development and Strategic Partnerships, MMRF.

According to the MMRF, about 24,000 new patients will be diagnosed in the United States in 2014.

 Over the past 2 decades, advances in therapy and the use of stem cell transplants have revolutionized care and increased average survival from 3 years to about 8 years, according to Kenneth Anderson, MD, director of the Jerome Lipper Multiple Myeloma Center at Dana Farber Cancer Institute, who in March gave the multiple myeloma update at the 19th annual conference of the National Comprehensive Cancer Network (NCCN).

Chernew asked how diagnosis of multiple myeloma has changed, and what the optimal practice would be for early screening. Kumar said routine screening of everyone over a certain age was impractical, as it would identify persons for whom there were no therapeutic options, and “would only contribute to anxiety.”

That said, there is a group for whom the disease should be identified well before the onset of the “CRAB” criteria: elevated calcium levels, renal failure, anemia, and bone failure. “At that point it may be too late,” Kumar said. Techniques such as examining circulating tumor cells, while not new, are becoming more precise in their prognostic value. The challenge is finding the optimal way to identify patients with myeloma early. For years, advances in treatment of multiple myeloma stalled because of the very nature of the disease, which presents differently from patient to patient and thus was not a good candidate for investment by the pharmaceutical sector. But the advancement of genomics, in which researchers identify the mutations that cause the varieties of myeloma, is allowing multiple myeloma patients to join clinical trials of therapies aimed at mutations that occur across multiple cancers.

Potentially quite fruitful is the CoMMpass Study, an effort by the MMRF that Anne Quinn Young discussed. CoMMpass seeks to gather tissue from 1000 patients at diagnosis and at each time the patient experience a relapse. The effort, Young said, will yield data about 3 kinds of sequencing: whole genome, exome, and RNA. Hopefully, “this will help give us a better understanding of the natural history of the disease and give us some clues to better precision medicine approaches,” she said.

Getting an early diagnosis does not mean a patient must be treated, Goldberg said. “It means better outcomes,” he said. Even if treatment is not immediate, patients can make lifestyle changes such as eating healthier foods and avoiding alcohol. Finding the fine line in “smoldering” myeloma— the optimal time, after diagnosis but before symptoms appear, when treatment should start&mdash;remains the challenge.

While Goldberg is hopeful that biomarkers may someday help pinpoint that line, thus far some biomarkers under study “are not ready for prime time. “It may take years, he said, “before these experimental markers are going to be able to tell us what the future will be.”

Kumar explained that treatment advances have 2 pathways: changes due to new therapies, and changes due to better understanding of multiple myeloma’s biology. New drugs such as bortezomib and thalidomide have already changed the treatment landscape for the disease. Excitement is building because there aren’t just several new drugs in the pipeline but multiple new drug classes. As at NCCN in March, Kumar spoke animatedly about the clinical trials involving monoclonal antibodies (elotuzumab and daratumumab).

“The second half of the story is the changing understanding of the science,” Kumar said. In the past, the approach to therapy was “hit and run,” but today, clinicians know that “the bottom line is that patients need a couple of years’ worth of therapy, or even more. This is dictated by the underlying biology,” he said, as myeloma must be treated chronically to keep it under control.

That is possible, Goldberg said, because the newer drugs are much less toxic than their predecessors. Longer treatment periods achieve a “deeper” response, and “That’s where the benefit has been seen the most,” he said.

A simple truth, he said, is that to treat a patient with cancer, “You’ve got to be able to deliver the drug.” If the side effects are so awful that the patient can’t tolerate them, then the therapy has limited value.

The challenges today, Goldberg said, often involve dosing, and how to use the growing number of options in combination. The concept of “minimum residual disease” or

MRD, is also new to myeloma, because until recently, patients never got to that point, he said.

The evolution of multiple myeloma from hard-totreat blood cancer to a chronic disease means that it will likely become expensive for payers as patients live longer, Goldberg said, although this may be offset by more years of productivity and quality of life. Kumar noted that some drugs that treat the disease will soon come off patent, which will mitigate costs somewhat. But the clinicians explained that the complexity of the disease and its heterogeneity means that treatment will always be very individualized, and that many patients will be on multiple drugs at once.

Both Goldberg and Kumar said that thus far, insurers have largely not balked at paying for multiple myeloma therapies, with the possible exception of oral therapies. But they said that could change as monoclonal antibodies near approval, and the variety of available options increases again.

“The insurer market is concerned about all these things,” said Reeder. While it’s clear that patients have benefited, he said, payers will likely reach a point of determining which patients will benefit from which therapy, and establishing a “stopping rule” to handle requests to repeat therapies that have already failed.

Young said one outcome of the CoMMpass study will be its guidance for precision medicine. It will show which patients can be helped with drugs that are off-patent, as well as which patients only need perhaps 2 drugs.

The most important task, the panelists agreed, will be making improvements in tackling myeloma at the outset. Said Goldberg, “We have to figure out how to do the best job in the beginning.” 

1. Ramesh N, Haehle M. Multiple myeloma survival increased significantly the past 15 years, but unevenly across ethnic and age groups. The Myeloma Beacon. http:// www.myelomabeacon.com/news/2013/08/31/multiplemyeloma-survival-race-age/. Published August 31, 2013. Accessed June 6, 2014.

2. Newly diagnosed patients: what is multiple myeloma. Multiple Myeloma Research Foundation website. http://www.themmrf.org/living-withmultiple-myeloma/newly diagnosed-patients/whatis-multiple-myeloma/. Accessed June 6, 2014.

3. Caffrey MK. Promising news in treating multiple myeloma. 

4. Bench to bedside translational science doubling myeloma survival. Dana Farber/Harvard Cancer Center website. http://www.dfhcc.harvard.edu/news/news/article/3062/. Accessed June 6, 2014.

AJMC Panel: Advances in Multiple Myeloma Therapy Extend Survival for Patients, Raising Cost Concerns

In 1996, Kathy Giusti was a 37-year-old rising star in the pharmaceutical industry, with a young child. When she learned she had multiple myeloma, the news floored her. Not only did she not resemble the typical profile of someone with this rare blood cancer, but she quickly discovered that treatments for the disease had barely

Neither the projected 3-year life expectancy nor the state of treatment were acceptable to Giusti, and she took action. With her twin sister, Karen, who would become her donor for a life-saving stem-cell transplant, the pair founded the Multiple Myeloma Research Foundation (MMRF).

 Since 1998 MMRF has raised more than $250 million,

 and from 2003 to 2007 the foundation helped win FDA approval for 4 drugs that are mainstays of current multiple myeloma therapy: bortezomib, lenalidomide, thalidomide, and doxorubicin.

 More recently, MMRF offered research support for

By bringing the same principles she learned in her business career to finding treatments for multiple myeloma, Giusti has beaten the odds, in scientific and personal terms. She’s lived with the disease for 18 years, and the organization she founded has helped trim approval times for a host of treatments, in turn increasing the average survival from 3 to 8 years.

It’s an important time for the foundation and for those who study and treat persons with the disease, said Walter M. Capone, who in December 2013 became MMRF president after several years as chief operating officer. Capone, who came to the organization after years in the pharmaceutical sector, said he expects the next few years to resemble the shift he witnessed from 1996 to 1998 in HIV treatment, with the arrival of both new drugs—protease inhibitors and non-nucleoside analogue inhibitors&mdash;and new viral load detection tests. The ability to both detect HIV more accurately and attack it through multiple channels, Capone said, ended the disease’s status as a certain death sentence. By 2012, HIV qualified as a

“chronic condition” under the Affordable Care Act.

Multiple myeloma treatment is headed in the same direction, he said. “This couldn’t be a more exciting time…the advances that are going on are going to be quite significant and  transformational,” Capone reported. Although the declaration of a “cure” for multiple myeloma is well in the future, doctors are much closer to finding one than when the MMRF started.

Researchers still don’t completely understand what sets the disease in motion, but they know that in a healthy person a delicate balance properly regulates the transition of B lymphocytes into plasma cells as they leave the bone marrow. When the immune system is damaged, this process produces oncogenes instead. Healthy blood cells get crowded out, causing a host of health

problems such as fatigue, kidney damage, and calcium deposits. The skeletal system is compromised, and multiple myeloma patients may suffer broken bones from no obvious cause.

The MMRF sees progress in how multiple myeloma has expanded its presence on the scientific agenda. Advances have taken center stage at recent meetings of the American Society of Hematology

 (ASH) and the National Comprehensive Cancer Network (NCCN). At NCCN’s spring meeting, Kenneth C. Anderson, MD, director of the Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute, and chair of the steering committee for the MMRF’s scientific arm, the Multiple Myeloma Research Consortium, proudly told his colleagues, “I want you to get excited about the future…we think we’re about to get a monoclonal antibody or two.”

A central feature of multiple myeloma, and a challenge of treatment, is how the disease behaves differently from patient to patient. Even in a given person, it must be attacked on multiple fronts. Thus, the MMRF supports research into many different types of drugs, while also spearheading the science that examines myeloma’s genetic underpinnings. In the necessary diversity of

approaches, “It’s like an investment portfolio,” said Capone. Drawing on the HIV comparison, he foresees breakthroughs in both the diagnostic and therapeutic areas: next-generation

sequencing and circulating tumor cell detection strategies, as well as whole new classes of drugs. There are elotuzumab and daratumumab, the antibodies that Anderson referenced at NCCN.

Capone also sees promise in the histone deacetylase inhibitors, which are being studied in combination with bortezomib and dexamethasone in patients with relapsed myeloma.

Capone also pointed to Millennium’s late phase 3 trials on what would be the first oral proteasome inhibitor. Results presented at ASH 2013 in New Orleans showed a combined complete response and very good partial response rate of 76% (46/62), and a 94% overall response rate (58/62 ≥ partial response.)

MMRF’s most ambitious project to date may be its CoMMpass Study, an effort to track 1000 multiple myeloma patients for at least 5 years each.

 Patients enrolled in the study will provide

a tissue sample when they are newly diagnosed, and then provide additional samples each time their disease relapses. The goal is to examine how each patient’s molecular profile affects

the progression of the cancer, with the hope of not only tailoring treatment for that individual patient but also developing an increasingly rich database, one that will propel the discovery of more effective targeted therapies with each passing year.

CoMMpass patients come from a variety of treatment settings, and its data are open to clinicians and researchers. All this is by design, Capone said, to facilitate clinical trials and to aid  treatment for patients far from academic research centers. When asked if MMRF had a position on whether pharmaceutical or diagnostic testing companies should share patients’ genetic data,

Capone strongly supported making data available for all researchers: “I’ve got a particularly strong emotional and practical position,” he said. “It’s the patients’ data,” and, thus, does not belong to any one company.

“The fact that industry in its various forms might to try optimize it, well, that’s only wonderful if it’s for the patients’ benefit. When that becomes secondary and subordinated, that’s a fundamental

problem. And that’s why we put the data on a public portal.”

MMRF’s efforts in genomics predate the 2011 launch of the CoMMpass study. In 2005, the foundation started its Multiple Myeloma Genomics Initiative, which gathered 250 tissue samples

 Those efforts bore fruit  in March 2011 with the publication of an article in Nature.

 Researchers examining the first batch of tumor genomes collected by the MMRF observed

mutations of the kinase BRAF in 4% of the patients, suggesting that BRAF inhibitors should be studied in multiple myeloma patients in clinical trials.

As a result, new therapies that have been highly successful in treating metastatic melanoma patients—vemurafenib, trametinib, and dabrafenib&mdash;are now at various trial stages for multiple myeloma patients with BRAF V600E and V600K mutations.

The diversity or heterogeneity of multiple myeloma means that what works in one patient might fail in another. As Giusti discovered shortly after she was diagnosed, that’s what kept pharmaceutical companies from investing in multiple myeloma for so long.

The rise of genomic research is the game changer. It offers the ability to build clinical trials around therapies that have already worked in other cancers, or to include multiple myeloma patients

in trials for new therapies aimed at a particular mutation seen in many cancers.

This is already happening with the BRAF mutations. But as this strategy unfolds, it will mean attacking the disease on many fronts. That will mean many drugs. And that could get very expensive.

 (see related story, below) discussed the cost implications for payers as multiple myeloma patients live longer with the disease.

One clinician, Jack Goldberg, MD, of Penn Presbyterian Medical Center in Philadelphia, said it was essential to get better at treating the disease earlier. Not everyone agreed that screening for

multiple myeloma made sense, since it might not be treated right away, causing patients undue stress.

Capone, however, sees a future for screening among persons older than 60 years. Screening could become routine, just as colonoscopies and prostate-specific antigen (PSA) tests have, he said. Identifying biomarkers for multiple myeloma, and certainly catching more cases at the “smoldering” stage, while figuring out ways to prevent full-blown disease, makes tremendous

“From an intervention standpoint, therapies that involve the priming of, or strengthening of, the immune system to identify and mitigate aberrant cell development offer the best benefit for patients,” Capone said. “It’s much less expensive to treat it early.” 

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6. Blake E. Addition of Zolinza slightly improves Velcade treatment for relapsed melanoma patients. Myeloma Beacon. http://www.myelomabeacon.com/news/2013/10/1 /zolinzaslightly-improves-velcade-treatment-for-relapsedmultiple-myeloma/. Published October 16, 2013. Accessed June 5, 2014.

7. Walker T. Updated data with oral proteasome inhibitor reported in newly diagnosed multiple myeloma patients. Formulary Journal. http:// formularyjournal.modernmedicine.com/formulary-journal/news/updated-data-oralproteasome-inhibitor-reported-newly-diagnosedmultiple-myel?page=full. Published January 9, 2014. Accessed June 5, 2014.

8. The Multiple Myeloma Research Foundation (MMRF) and the U.S. Department of Veterans Affairs (VA) create unprecedented private/public partnership to advance the development of personalized medicine [press release]. Norwalk, CT, and Washington, DC: Multiple Myeloma Research Foundation and US Department of Veterans Affairs; November 26, 2012. http://www.themmrf.org/about-the-mmrf/powerful-news/pressreleases/mmrf-landmark-commpasssm-studyexpands-to-include-va-sites.html.

9. Genomics backgrounder. Multiple Myeloma Research Foundation website. http://www. themmrf.org/assets/research-programs/genomics-initiative/genomics backgrounder.pdf. Accessed June 5, 2014.

10. Chapman MA, Lawrence MS, Keats JJ, et al. Initial genome sequencing and analysis of multiple myeloma. 

11. Lohr JG, Stojanov P, Carter SL, et al. Widespread genetic heterogeneity in multiple myeloma: implications for targeted therapy. 

12. Bohn OL, Hsu K, Hyman DM et al. BRAF V600E mutation and clonal evolution in a patient with relapsed refractory myeloma with plasmablastic differentiation. 

13. ClinicalTrials.gov. A study of trametinib and GSK2141795 multiple myeloma. National Cancer Institute. http://www.cancer.gov/clinicaltrials/ search/view?cdrid=753717&version=HealthProfessional&protocolsearchid=11368643. Updated May 9, 2014. Accessed June 5, 2014.

14. ClinialTrials.gov. Efficacy and safety of the combination therapy of dabrafenib and trametinib in subjects with BRAF V600E-mutated rare cancers. https://clinicaltrials.gov/ct2/show/NCT02034110?term=%22dabrafenib%22+and+%22myeloma%22&rank=1. Updated May 29, 2014. Accessed June 5, 2014.

MMRF Efforts Attack the Disease From Every Angle; "Transformation" on the Horizon

While the incidence of cancer increases, survival rates continue to improve for even the deadliest of cancers thanks to the ongoing development of novel and targeted therapies. However, innovations in cancer diagnosis and treatment are associated with high cost. Cancer therapy constitutes nearly 11% of the total healthcare budget, and the number continues to trend upward.

The National Cancer Institute estimated the costs of cancer therapy at $124.6 billion in 2010, a number projected to touch $207 billion in 2020.

 With an aging population and improved insurance coverage, that figure will almost surely stay on the rise. The result: patients and providers are very often restricted from choosing the highest quality treatment option due to the burden of associated costs.

The problems and possible solutions were the focus of an opening-day Extended Education Session at the 50th annual meeting of the American Society of Clinical Oncology (ASCO), held in Chicago from May 30 through June 3, 2014. The session—titled “Can We Find Common Ground? Stakeholder Perspectives on Value in Cancer Care”&mdash;began with remarks from session chair Neal J. Meropol, MD, of Case Comprehensive Cancer Center, Case Western Reserve University.

Meropol recently coauthored an article on best practice approaches for designing decision-support tools.

 The article described the rigorous development process and initial feedback of the PRE-ACT (Preparatory Education About Clinical Trials) Web-based intervention, designed to improve preparation for decision making in cancer clinical trials. In his talk, “Value in Cancer Care:

What’s the Problem?”, Meropol defined value as the ratio of benefit over cost. Benefit, he said, is a combination of survival, progression, quality of life, and avoidance of toxicity, while cost is determined by biomarkers, the therapy received, therapy avoided, management of toxicity, and other indirect costs. “Value versus cost-effectiveness is the elephant in the room,” he said.

Meropol laid out the problems currently facing the United States:

• Drug approvals based on “safety and efficacy,” without cost considerations

• Producer sets the price for new innovation

• Financial protection, historically offered by insurance, is eroding as cost burden shifts to the patient.

“The current system is unsustainable, as it prevents access to high-quality cancer care,” said Meropol. However, he pointed out that economic recession, increased cost sharing, payment reductions to providers, increased provider efficiency, and slowed innovation have somewhat moderated healthcare spending. An uptick in spending growth is anticipated, however, as the economy improves and insurance coverage improves, enabling the aging population to increase healthcare spending.

Oncology drug expenditures lead the way in spending among the various therapeutic areas. In this country, $27.9 billion was spent on oncology drugs in 2013, representing an annual increase of 9.2%, according to a recent IMS Institute for Healthcare Informatics study (

). High cost of care can increase disparities in care and patient outcomes, exacerbated by steep co-pays, tiered formularies, the part D donut hole in Medicare, and oncology drug shortages.

The result? Patients delay consultations and treatment, and limit or alter their treatment, to the detriment of their health. Hospitals also reduce their spending on charity care. “The stakeholders need to work together,” concluded Meropol, “to find solutions, drive policy, and achieve reform.”

Next, Lowell E. Schnipper, MD, presented “Defining Value: An ASCO Initiative,” to offer insight into ASCO’s strategic initiative to define value in cancer care. Schnipper, chief of hematology/oncology at Beth Israel Deaconess Medical Center in Boston and the chair of ASCO’s Value in Cancer Care Task Force,

 spoke about unsustainable trends in the United States: Medicare, he postulated, is a major reason for deficit projections, in addition to cancer costs, which are rising by 15% to 18% annually.

Schnipper encouraged the oncologists in the audience to discuss the financial implications of therapy with their patients, asserting that patient-centered care, both financial and health outcomes based should be a key element when defining value of care. The main threats to value in healthcare, Schnipper said, include unwarranted variation in quality and outcome; harm to patients; wastefulness of resources and failure to maximize value; health inequalities and inequities; and failure to prevent disease.

A major value initiative introduced by ASCO, the Quality Oncology Practice Initiative (QOPI),

 aims to remedy these ills by evolving a transparent, clinically driven method for defining and assessing the relative value of cancer care options and finding a balance among clinical benefit, toxicity, and cost. The objective of the task force behind QOPI is to afford providers with the necessary skills and tools to meet those goals, and to give patients ready access to essential information, including how to cope with co-pays that are potentially bankrupting.

“We are not caring about the dollars spent, but rather how well we are caring for the patient. Patient-centric is the best way to go,” concluded Schnipper. Representing “The Patient Perspective,”

as her talk was titled, was Diane Blum, MSW, FASCO, a former executive director at CancerCare, an organization that provides emotional and financial support to cancer patients and their

Blum defined value in various terms. Her favorite was crafted by Scott Ramsey, MD, PhD, of the Fred Hutchinson Cancer Research Center in Seattle: An intervention in cancer care can be described as having value if patients, their families, physicians, and health insurers all agree that the benefits afforded by the intervention are sufficient to support the total sum of resources

“Value is a dynamic process—hopes and expectations change through the continuum of illness and must be assessed regularly, said Blum. “Perception changes from cure to palliation. Value rests not just in clinical response, but also in trying.”

How do you help the patient assess value? ASCO’s Choosing Wisely campaign aids the process, said Blum. Encouraging ongoing conversation between physician and patient, as well as a shared decision-making process, are usually very beneficial as well.

Lee N. Newcomer, MD, MHA, senior vice president of oncology, genetics, and women’s health at UnitedHealthcare, spoke next, providing a payer viewpoint on the increasing cost of diagnosing and treating cancer patients. Newcomer recently published his perspective on innovative payment models and measurements in cancer care, in which he introduced the models being evaluated by payer and physician groups for cost-effective care.

 These include (1) pay-for-performance (P4P) programs that reward physicians for meeting prespecified goals, (2) bundles or episode payments, and (3) capitation.

Newcomer emphasized the fact that currently, health insurance premiums equal 50% of the average household income, and by year 2033, they are estimated to surpass the average household income (

Addressing insurance benefit design issues, Newcomer said that benefits are not cancer specific, value-based insurance design is hard to implement, and that the site of service distorts benefit. He further added that the valuebased insurance (VBI) plan is trying to create a differential between high-value and low-value services.

Noting that barriers to VBI include access to an external, independent source of measurement lto define value, Newcomer pointed out that the Value in Cancer Care Task Force, spearheaded by ASCO, is working to surmount this obstacle. Emphasizing that consumer literacy is extremely important, Newcomer said that value-based programs compel consumers to digest a lot of information that can be overwhelming, especially when survival decisions are in question. Although demarcating boundaries is extremely difficult, it’s drawing those lines that will help establish value. The most difficult option to choose, he said, would be not to pay at all. For example, the payer could say: no PET surveillance post therapy, no PSA testing for men with less than a 10-year life expectancy, or no targeted therapy without a predictive biomarker.

“Resources are limited, so using high-value resources and/or eliminating resources with limited or no value is the need of the day,” said Newcomer. These decisions, he concluded, are unavoidable. Speaking next and representing the pharmaceutical industry was Gregory P. Rossi, PhD, vice president of payer evidence at AstraZeneca UK, who oversees coverage and reimbursement submissions and is responsible for outcomes studies as well as fulfilling payer evidence requirements.

 In “Industry Perspective: Drug Development, Costs, and Return on Investment,” Rossi discussed the costs of innovation, research and development (R&D) investment decisions, value-based pricing, and patient affordability. He characterized himself

as a proponent of collaboration between research communities and patient communities to advance cancer treatment, he said.

What drives innovation costs and decisions about investments? Rossi noted that success rates in pharmaceutical drug development are about 5%, driving costs per new molecular entity to $3 billion (1 molecule per year for every $3 billion spent). Technical risk, time, and market assumptions all impact investment decisions. Obviously, Rossi noted, treatments directed at small patient populations require higher drug prices to gain returns.

A related issue: value-based pricing. The last 17 oncology drugs approved in the United States offered, on average, 3 months of improvement in survival, but the average monthly cost of each drug, said Rossi, was $65,000. Can this be deemed beneficial? Rossi opined that the National Institute for Health and Care Excellence in the United Kingdom would definitely say “No!” Rossi

explained that cost- effectiveness and the willingness-to-pay threshold are very difficult matters, and suggested developing innovative means to evaluate the cost-benefit ratio of new drugs.

“Patient affordability is obviously a big issue,” said Rossi. “It is inappropriate to tax the patients with high copays.

The pharmaceutical industry has set up assistance programs, but this bandage is not sufficient to fix the current broken system.” Rossi concluded with a slide on ways to find common ground. To confront the burden of illness and the complexity of cancer, he proposed maintaining R&D investment to help discover new targets and develop novel technologies.

For cancer-related reimbursement issues, he had 3 suggestions:

• Maintain reward for meaningful innovation

• Develop a payment process based on indication and clinical pathway adherence

• Continue to refine insurance benefits to reduce the financial risk for patients

The insightful session concluded with “How Should Oncologists Become Value-Based Providers?”, presented by Ezekiel J. Emanuel, MD, PhD, vice provost for Global Initiatives and chair of the Department of Medical Ethics and Health Policy at the University of Pennsylvania’s Perelman School of Medicine. Said Emanuel: “Let’s be honest: it is about cost. We are concerned about value, but we are also concerned about cost. Let’s all be honest as a community about where the real cost of cancer care is.”

Emanuel—also an oncologist&mdash;alleged that expensive treatments are being prescribed despite the availability of cheaper treatment options. The obligation of an oncologist, he said, is to avoid unnecessary testing&mdash;when therapeutically equivalent treatment is available, prescribe the cheaper one. Additionally, oncologists should exclude options that do not add value. “This is not a victimless crime&mdash;to choose the expensive treatments when cheaper alternates are available,”

Emanuel said. “I do think we have common ground, but we can’t reach it without payment reform and taking steps to choose value-based options.”

1. Newcomer LN. Innovative payment models and measurement for cancer therapy. 

2. Study: new cancer care payment model reduced health care costs, maintained outcomes [press release]. Minnetonka, MN:

How Do You Establish Value in Cancer Care?

A clinical trial is a massive investment for the drug manufacturer—an investment of time, effort, and funds that are channeled into preclinical research to identify a target and the right molecule for the target. Then, of course, the actual costs of conducting a trial are enormous.

August 2014

ASCO's Initiative to Define Value In Cancer Care

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MMRF Efforts Attack the Disease From Every Angle; "Transformation" on the Horizon

How Do You Establish Value in Cancer Care?

Reducing Risk and Improving Efficacy of Clinical Trials: the Adaptive Design

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In New Jersey, Focus in Fight Against Cancer Changes, and Care Comes Closer to Home

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