by Amanda Patton, Manager, Communications ACCC
In a study from the Fred Hutchinson Cancer Research Center in Seattle, published in the May 15 Health Affairs researchers reported that Washington State cancer patients were found to be 2.65 times more likely to go bankrupt than people without cancer, with younger cancer patients having 2–5 times higher rates of bankruptcy than cancer patients age 65 or older.
Another recent study from Duke Cancer Institute, presented at the June 2013 ASCO Annual Meeting (ASCO Abstract #6506), reported that although most cancer patients would like to talk about the cost of care with their doctors, often they don’t. Reasons why vary from embarrassment, to thinking it was not something that the doctor could help with, to fear that it might compromise the quality of their treatment. Findings come from a survey of 300 insured patients treated at Duke and affiliated clinics in rural North Carolina.
“There’s a real disconnect,” lead study author Yousuf Zafar, MD, MHS, said in a press release. “Even people with the highest needs aren’t bringing up costs as part of the decision-making process.”
Discussion about the financial issues surrounding cancer treatment can be daunting for both patients and providers. However, the Duke study found that many patients felt they gained by having a discussion about finances.
“We found that when patients did talk about costs with their doctors, many felt they gained something from the discussion—that their expenses were reduced,” Zafar said. “This suggests that the perceived barriers to the cost conversation aren’t real, and we need to do more to foster a dialogue around these issues.”
The Association of Community Cancer Centers’ Financial Information and Learning Network project offers resources to help providers who deal with patients on the complex financial issues surrounding their cancer diagnosis and treatment. Among the offerings are online courses on topics such as “Patient Assistance 101,” “Financial Counseling 101,” and “Financial Specialist as Part of the Multidisciplinary Cancer Care Team;” videos; and a practical Financial Assistance Toolkit. Plus, a new Financial Assistance Pre-Conference, being held in conjunction with the ACCC National Oncology Conference, has just been announced.
We hope these resources can help to “foster dialogue” on these complex and difficult issues.
By Chad Knight, MSHA
Zimmer Cancer Center
New Hanover Regional Medical Center
This week actress Angelina Jolie’s New York Times op-ed piece, “My Medical Choice,” focused enormous media attention on genetic testing for breast cancer and treatment options. By sharing her story, Jolie hopes to raise awareness. She writes:
For any woman reading this, I hope it helps you to know you have options. I want to encourage every woman, especially if you have a family history of breast or ovarian cancer, to seek out the information and medical experts who can help you through this aspect of your life, and to make your own informed choices.
At our cancer center, requests for information from local media and the public were immediate and compelling. In response to multiple requests from media outlets and to proactively answer questions from the community, our cancer program scheduled a press conference with the primary goal of educating our community on the importance of cancer screenings, including genetic counseling and testing.
Jolie’s announcement presents the oncology community with an opportunity to raise local awareness of the importance of access to genetic testing and counseling to help with informed, patient-driven decision making. In fact, I agree with Dr. Isabelle Bedrosian of MD Anderson who in a follow-up article expressed her hope that Jolie’s announcement will “focus women on the value of genetic counseling and making informed decisions.”
With opportunity comes challenge. Rest assured we will continue to field questions and address concerns, especially those from patients with a known family history. The challenge will be how we, with limited resources, ensure that our community is informed such that they can make educated decisions about their own health. How do we expand reimbursement models for more genetic counseling and testing and other cancer screenings? How do we make certain our medical community is collaborative and adhering to the most current evidence-based guidelines? Our challenges are great, but our opportunities are greater.
Chad Knight, MSHA, is Administrator, Oncology Services, at ACCC member New Hanover Regional Medical Center, Zimmer Cancer Center, in Wilmington, N.C.
For more on how ACCC member cancer programs responded this week, read “Angelina Jolie, BRCA1 and the Power of Celebrity” from Seattle Cancer Care Alliance.
by Sydney Abbott, JD, Manager, Provider Economics and Public Policy, ACCC
The good news: oral parity legislation has already passed in almost half of the states. The bad news: the laws vary from state to state, and 27 states still have no law at all.
The solution: HR 1801, the bipartisan Cancer Drug Coverage Parity Act, introduced recently by U.S. Representatives Brian Higgins (D-NY) and Peter King (R-NY).
Currently, most IV-infused chemotherapies are covered under the medical benefit component of a health insurance plan, while orally administered drugs fall under the prescription drug benefit, which often carries a very high patient-cost-sharing component. The Higgins-King bill would eliminate the cost-sharing disparity by requiring insurance companies to cover orally administered prescription drugs at a cost no less favorable than their infused counterparts. Legislation such as this is particularly important as the oncology drug development pipeline increasingly consists of oral anti-cancer medications. Some estimates are that over the next decade the oncology pipeline will include up to 30% oral anti-cancer drugs.
So why is ACCC pushing for oral parity legislation at both the federal and state level? The answer is simple: we want to achieve parity across every state as quickly as possible. While federal legislation is preferable because it would negate the need for individual state laws and would cover every patient equally from coast to coast, the current political climate on Capitol Hill is such that legislation regulating insurance companies can be unpopular and difficult to move through both houses of Congress. In fact, Representative Higgins introduced this same bill in the last Congress, but the legislation never made it through committee or to the House floor for a vote. Additionally, companion legislation must be introduced and passed in the Senate before the bill can become law. ACCC continues to work with other organizations to identify a Senate sponsor for a companion bill.
State legislation, on the other hand, generally moves faster due, in part, to the fact that most state legislatures are only in session for a portion of the year. Over the past five years, 23 states plus Washington, DC, have been able to pass their own oral parity bills while Congress has been unable to pass one. For this reason, ACCC will continue to advocate for oral parity with a two-pronged approach until every patient in the U.S. has access to the most appropriate anti-cancer medication at the most appropriate time. Please visit the ACCC Legislative Action Center for more ways to get involved in this issue and others impacting cancer care. ACCC will keep members current on HR 1801, as well as progress on state-level legislation.
By Sydney Abbott, JD, Manager, Provider Economics and Public Policy, ACCC
As more oral anti-cancer regimens become the most appropriate form of treatment for cancer patients, the inequality in coverage for oral prescriptions compared with their IV-infused counterparts is becoming clearer. Because health insurance plans cover oral anti-cancer drugs differently than IV chemotherapy and injected anti-cancer medications, patients have to pay far more out-of-pocket for oral drugs than for IV chemotherapy or injected medications. The phrase “oral parity” refers to legislative initiatives intended to protect patients with cancer from the high cost-sharing requirements that are sometimes associated with oral cancer drugs under health insurance plans.
ACCC continues to support oral parity laws in the states, as well as federal legislation. At the state level, efforts by ACCC members and other organizations are having an impact. So far this year, three additional states have passed parity legislation: Massachusetts, Utah, and now, this week, Florida, where legislation is awaiting the Governor’s signature. This brings the tally of states with oral parity legislation to 23* plus the District of Columbia.
The oral parity issue is really a case of technology outpacing policy. As therapies become more targeted, reliance on oral anti-cancer treatments will only increase. Patients receiving IV-infusions through the physician office setting are covered under the medical benefit of their insurance plan, generally requiring about a $30 co-pay. However, patients on an oral regimen are covered under their pharmacy benefit, which often requires a cost-sharing component that is some percentage of the total cost of the drug. In the case of oral anti-cancer medications, that can be thousands of dollars out of pocket every month.
ACCC is proud to support the oral chemotherapy parity bills that recently passed in Massachusetts, Utah, and Florida, and will continue to support federal efforts to pass legislation covering all 50 states. Stayed tuned for an update on federal oral parity legislation.
*Oregon (2008), Indiana (2009), Iowa (2009), Hawaii (2009), District of Columbia (2009), Vermont (2010), Connecticut (2010), Kansas (2010), Colorado (2010), Minnesota (2010), Illinois (2011), New Mexico (2011), Texas (2011), New York (2011), Washington (2011), Nebraska (2012), New Jersey (2012), Maryland (2012), Virginia (2012), Delaware (2012), Louisiana (2012), Massachusetts (2013), Utah (2013), Florida (2013 – pending Governor’s signature)
by Matt Farber, MA, director, Provider Economics and Public Policy, ACCC
In the past week, two bills important to oncology care have been introduced in the United States Congress. The first, introduced by Reps. Israel (D-NY) and Tiberi (R-OH), is HR 1661. This bill would provide reimbursement for a one-hour chemotherapy teaching session by nurses in the physician-office setting. This legislation has been introduced in previous Congresses, with the primary support of the Oncology Nursing Society (ONS). ACCC has supported this legislation in the past and will be supporting it again this year.
The second bill is the “Patient Centered Quality of Life Act” (HR 1666). This bill would support the growing demand for palliative care, which is specialized medical care that focuses on care coordination and relief from pain, stress, and other symptoms of treatment for a life-threatening disease such as cancer. The bipartisan bill, co-sponsored by Reps. Cleaver (D-MO) and Bachus (R-AL), would facilitate and expand federal research into palliative care; support training for nurses, nurse practitioners, and other allied health professionals to effectively practice palliative care; and convene health professionals, patients, public and private payers, and state and federal health officials for a national summit to develop tools and model best practices for providing palliative care.
Both pieces of legislation support oncology care and would improve the overall quality of care that patients receive. These bills address issues that have been identified as key to ensuring quality care as part of ACCC’s Grassroots Advocacy Campaign. ACCC is working with advocacy organizations, such as ONS, the American Cancer Society Cancer Action Network (ACS CAN), and other organizations to increase the support for these bills. We encourage you to write and call your elected officials and ask them to support these important measures. Visit ACCC’s Legislative Action Center.
By Virginia Vaitones, MSW, OSW-C
Having a cancer diagnosis is very expensive even for those with insurance. For those who are underinsured or uninsured, the cost of care can be a strong deterrent to accessing treatment. For patients with a chronic disease, such as multiple myeloma, the cost of annual deductibles, office visit co-pays, and prescription drug co-pays can quickly mount up. Many of the newer drugs used in treating multiple myeloma are available only through specialty pharmacies, which require preauthorization processes to access medications.
Patients eligible for transplant face additional financial, logistical, and psychosocial issues, for example, having to travel significant distance to a transplant center for care, which adds the cost of transportation, lodging for caregivers, and being away from home into the mix.
Fortunately, for the individual with multiple myeloma there are resources available to help with co-payments, medication assistance (including free medications), transportation, and lodging costs. Among resources to help with paying for medications are the Chronic Disease Fund (1-877-968-7233); the Leukemia & Lymphoma Society (1-877-557-2672); the Patient Access Network Foundation (1-866-316-7263); and the Patient Advocate Foundation Co-Pay Relief Program (1-866-512-3861).
The following anticancer drugs have pharmaceutical company programs that will help walk you through all the benefits that your patient may be eligible for throughout their treatment for multiple myeloma:
- Velcade (bortezomib) (1-866-835-2233) www.velcade.com/payingfortreatment.aspx
- Revlimid (lenalidomide) (1-800-931-8691) www.celgenepatientsupport.com
- Pomalyst (pomalidomide) (1-800-931-8691) www.celgenepatientsupport.com
- Kyprolis (carfilzomib) (1-855-669-9360) www.kyprolis.com/resources
In addition to financial assistance information, each of the above has educational materials written specifically to help the patient and their caregivers understand the disease and treatment.
Here’s an important fact: For those men and women seeking consultation for the first time for multiple myeloma, a key question to ask during the initial consult is, “Have you served in the military?” Due to exposure to chemicals such as Agent Orange, ionizing radiation exposure, and—most recently discovered—contaminated drinking water from Camp LeJeune from January 1, 1957, to December 31, 1987, veterans diagnosed with multiple myeloma may be eligible for benefits and care from the U.S. military. You can find more information on these benefits and how to access care at www.publichealth.va.gov.
Visit ACCC’s Improving Quality Care in Small-Population Cancers: Multiple Myeloma project webpage for additional information on patient and provider resources.
Virginia Vaitones, MSW, OSW-C, is an oncology social worker at Pen Bay Medical Center in Rockport, Maine, and president of the Association of Community Cancer Centers.
by Amanda Patton, ACCC Communications
One way to ensure a place at the table? Invite everyone to lunch. On March 8 the Association of Community Cancer Centers (ACCC) did exactly that with its lunchtime Town Hall Meeting “Shift in Site of Care, Real or Fiction?” during the ACCC 39th Annual National Meeting.
Moderator William T. McGivney, PhD, asked panelists Christian Downs, JD, MHA, ACCC Executive Director; Joseph Bailes, MD, FACP, healthcare policy expert; and Eli Lilly & Company’s Byran Litton, MBA, to explore issues around the shift in site of care and its implications, as well as the move away from fee-for-service reimbursement to shared-risk models such as bundled payments, episode-based payments, and ACOs. Here’s what the panel had to say.
Shift in site of care—it’s real and it’s already underway.
Citing ACCC’s Cancer Care Trends Survey, now in its fourth year, Downs noted the continuing trend of office-based physicians looking at creating arrangements with hospitals. The answer to why this is happening and what this shift will mean for cancer care is complicated, requiring a multifactoral analysis, Downs said. However, two forces are clearly at work: financial pressures and a demographic shift.
Well-known financial pressures squeezing physician practices include increasingly expensive anticancer therapies, more patients who are uninsured and underinsured, and rising patient copays. With reimbursement decreasing in the physician office setting, there’s a sense that joining forces with the hospital may provide opportunities to continue to provide community-based care in the physician office.
Another driver of change is a demographic trend in favor of employment, Downs said. Younger healthcare providers, in general, are comfortable with hospital employment. This is a different scenario from 10-15 years ago, when providers seemed to have more entrepreneurial interest in establishing their own practices.
It’s not déjà vu all over again.
Although the healthcare community did go through a similar shift in the 1990s, what’s happening today is a little different, commented Bailes. “For the medical oncology community, I think [the shift in site of service] is an opportunity to look at innovative partnerships. I think it’s going to be accelerated by ACOs to a certain extent.” Currently there are about 300 approved ACOs, and many of these are hospital-centric systems, Bailes pointed out. “…I think it’s going to be incumbent on the oncology community to be part of this hospital-centric approach.”
It’s too soon to know exactly how this shift will impact cancer care.
“I think the trend started [over] the last several years for many reasons…and we don’t know the end result,” Bailes said. What he has heard consistently from medical oncologists is that they want to do the right thing for the patient and ensure that an individual with cancer can get access to new therapies that are being developed.
Downs echoed that sentiment, adding that at this stage, it’s important to be wary of broad generalizations about the “best” practice environment. “It’s got to be a case-by-case analysis,” he said. In some locations partnering with a hospital will work, in other locations it may not. Much depends on the specifics of the community.
Protecting the cancer care delivery infrastructure in this time of flux is critical.
Downs pointed out that the cancer care delivery infrastructure is “very fragile fabric. If we pull too hard on one string, it could unravel.” He emphasized the importance of looking at the factors in each community. Bailes concurred. “It’s going to be an individualized decision.”
One area in which the oncology community has done a good job is laying the groundwork for discussion with both policymakers and payers. Despite this, “it’s a big challenge,” Downs said. “Policymakers look to us for answers. I don’t know if the answers are ripe yet. We know we want to end up doing what is best for the patient and the provider.”
It’s important to reserve a patient’s ability to choose.
From the industry perspective, rather than choosing sides—hospital vs. practice—“We really want to reserve choice in site of care for the patient,” said Litton. “Whatever’s best for that patient and that community, we want to support.”
Along with the shift in site of service is the trend away from fee-for-service toward risk-based models.
What are the implications of this move toward increased risk-bearing for physicians and hospital groups? For providers bearing risk is complicated, Downs pointed out. From a business standpoint, few providers may find it worthwhile to adopt this model.
Panelists agreed that while some markets might be appropriate for risk models, others would not be a good fit.
In some communities a risk model may not be the best use of resources that could be otherwise employed to improve care in the community, Litton said.
Downs also drew a distinction between risk-bearing models and bundled payments. “As we start to see some of this shift in site of service, hospitals may be a little more comfortable with bundled payments than practices,” he said.
For hospitals to be able to engage in bundled payments or episode-based payments, they’ve got to be able to understand and define the resources used, McGivney added. This means facilities must have the infrastructure in place to do this.
He noted that more than 500 participants—not-for-profits, for profits, practices, academic programs, etc.—have signed up for CMS’s recently announced bundled payment initiative that includes 48 different areas. (However, most are related to orthopedics, with only one area related to oncology.)
Oncology doesn’t lend itself to bundling; the odds of bundled payments becoming common in oncology are low.
While CMS has looked at creating a bundled payment for oncology, the archetypes for bundling have been around procedures and it’s difficult to establish a defined set of resources for oncology. Cancer care involves stages, variances related to co-morbidities, different lines of therapy, etc., and that makes bundled payment a difficult fit.
Oncology is clinical trial driven. The rapidity of change in oncology and the rapidity of new drugs makes it difficult to set bundled payments that will last a decade and provide economic predictability, noted Bailes. “That’s one of the challenges being faced by Medicare. From a fiscal side…to provide economic predictability over a decade…you can’t do that if you have to make a bundled payment with no provision or separate payment for new technology.”
New technology carve outs are necessary to protect innovation.
Molecular testing is a prime example of the importance of new technology in oncology and the accompanying challenges innovation brings. “They [new molecular diagnostic tests] are going to be sophisticated diagnostics that will [need to] have a more sophisticated reimbursement associated with them,” Bailes said.
Bringing the discussion back to the current oncology environment, McGivney asked each panelist to conclude by citing one event or policy that they would like to see happen that would have a real-world impact on cancer care in the next year.
Downs: “I’d like to see us do a better job of educating our patients on the financial responsibilities for their cancer care. Right now—not just [for] our uninsured and underinsured—even for patients with good insurance policies, I don’t think we do the best job of explaining what [these costs] will mean.”
Bailes: “Congress should streamline the FDA regulatory process and get drugs to the patients [faster], because we are trying to turn cancer into a disease that’s treatable and lets people live long and active lives.”
Litton: “Only 3 to 5 percent of oncology patients go on clinical trials, and I think it’s a black mark on the legacy for anyone in the decision-making seat in oncology. We’re too siloed [in terms of the drug development process]… if we could go from 10 to 15 years and $1 billion to develop a drug to 4, 5, or 6 years [and] $400 million to develop a drug—that changes our ability to invest more deeply. It changes our pricing structure. It brings innovation to patients faster.”
For all panelists the bottom line is making sure patients have access to quality care.
*This program was supported by PACE.
Highlights from the American Society of Hematology (ASH) Annual Meeting, December 8-11 2012
By Edward J. Gorak, DO, MS, FACP
The medical and scientific communities continue to advance our understanding of the pathophysiology of multiple myeloma. With that, new targets are identified and novel therapies are developed. The 2012 ASH Meeting was filled with a host of new targeted or “smart bomb” therapies for the treatment of multiple myeloma. The early showings of these agents are promising but not quite ready for prime time. Other drugs have moved into the prime time slot and continue to strengthen their position as single agents and in combination with other drugs in the fight against myeloma. Finally, some of the differences in our established therapies still find us with unresolved controversy.
Daratumumab is a monoclonal antibody that targets the CD38 molecule (a key surface protein on myeloma cells) and stimulates the immune system to kill myeloma cells. An earlier abstract from the American Society of Clinical Oncology (ASCO) June 2012 Meeting showed a 24% partial response in patients that had previously received multiple therapies (median of 6). The ASH update strengthened the drug’s ability to bind the CD38 target on myeloma cells. The drug was also well tolerated by patients.
Elotuzumab is another monoclonal antibody that is showing promise. This drug was also presented at ASCO 2012, where a Phase II study showed that elotuzumab in combination with lenalidomide (Revlimid) and dexamethasone (Decadron) demonstrated an overall response rate of 84%. An update at the ASH meeting reported that the response rate had improved to 92%. The patient population was typically older (median age=63 years) and the majority had received multiple prior therapies. These encouraging results have led to the development of Phase III trials in newly diagnosed and relapsed patients. Look for this drug to be in a prime time slot in the future!
A new novel agent, ARRY-520, acts by disrupting dividing cells through inhibition of kinesin spindle protein. A Phase II trial of ARRY-520 alone or in combination with dexamethasone was conducted in heavily pre-treated patients. Partial response rates in the single agent and combination arms were 16% and 22% respectively. These results warranted moving forward with further clinical trial development.
Carfilzomib (Kyprolis) is a proteasome inhibitor that gained FDA accelerated-track approval in July 2012. The labeled indication is for patients that have had at least two prior myeloma therapies including bortezomib (Velcade) and an immunomodulatory agent such as lenalidomide (Revlimid) or thalidomide (Thalomid). The main toxicities of this drug are hematologic. Be aware of a small but potentially serious risk of cardiac and pulmonary complications. Carfilzomib is now being studied in combination with other established agents such as lenalidomide, dexamethasone, thalidomide, and cyclophosphamide. Additional studies with novel agents such as those mentioned previously are also underway. This new prime time drug should be in a favorable spot for years to come.
Pomalidomide (Pomalyst) was approved by the U.S. FDA on February 2013. This oral drug is in the class of immunomodulatory agents (lenalidomide and thalidomide). Like carfilzomib, this drug is now being combined with other agents and results have been impressive thus far. In particular, the combination of pomalidomide, carfilzomib, and dexamethasone in relapsed patients yielded excellent response rates. Expect a long showing of this drug as well.
The role of maintenance chemotherapy remains undefined in patients with multiple myeloma. Some previous studies have suggested a benefit whereas others suggest no benefit. Bortezomib-Melphalan-Prednisone-Thalidomide followed by Bortezomib-Thalidomide maintenance (VMPT-VT) was compared with Bortezomib-Melphalan-Prednisone (VMP). Notably, VMPT-VT prolonged overall survival. It is unclear if the true benefit in the VMPT-VT arm is due to maintenance therapy or the more intensive induction regimen. Notably, the VMPT-VT group experienced increased toxicity resulting in a discontinuation rate of 12%. The risk of secondary malignancy (SPM) is low with immunomodulatory agents (thalidomide, lenalidomide) but real. Physicians must balance efficacy with toxicity especially in the maintenance setting. The increased toxicity coupled with uncertainty about efficacy leaves maintenance therapy undefined. Stay tuned as the maintenance arena continues to evolve.
There has not been much talk about peripheral blood stem cell transplantation for multiple myeloma of late. Newer, efficacious and less toxic regimens have made transplantation appear less attractive in some circumstances. There continues to be controversy surrounding the timing of transplant, sequence of transplants (single autologous, double transplant, autologous-allogeneic), and the role of consolidation and maintenance therapy following transplant. Hopefully, we will see results from a large randomized trial conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) that addresses these questions in an upcoming meeting.
In 2012 we saw some changes in programming in the care of the myeloma patient. Well-studied agents were brought into routine clinical practice and novel compounds completed pilot testing and are ready to assume a more active role. We look forward to new reasons to tune into the care of the myeloma patient in 2013.
Edward J. Gorak, DO, MS, FACP, is an associate in Hematology and Principal Investigator at the Geisinger Health System, Geisinger Cancer Institute, in Danville, Pa. Dr. Gorak is a member of the accrual working group for the NCI Myeloma Steering Committee. He is a member of the Advisory Board for ACCC’s Improving Quality Care in Small-Population Cancers—Multiple Myeloma education project.
By Sydney Abbott, JD, Manager, Provider Economics and Public Policy, ACCC
Yesterday, Congresswoman Renee Ellmers (R-NC) introduced a bill that would eliminate the sequester on chemotherapy drugs that must be administered in the physician office setting (HR 1416). It would also direct Medicare to repay providers for any reduced payment since April 1.
As you know, as part of the sequester, on April 1 the Centers for Medicare and Medicaid Services (CMS) reduced Medicare reimbursement for all drugs and services by 2%. As providers begin feeling the impact from claims submitted April 1 or later, some are being forced to turn patients away. Cancer care providers are impacted disproportionally because of the way care is reimbursed. In addition to services, the 2% sequester cuts are applied to drugs, which have fixed costs. Given the high cost of cancer care therapies, the sequester is putting many cancer care providers underwater.
The cancer care community is working diligently to remove the sequester from all drugs, or cancer drugs, at a minimum. Congress recognizes that such cuts restrict providers’ ability to care for cancer patients. Therefore, ACCC supports HR 1416, the “Cancer Patient Protection Act” that would eliminate chemotherapies from the 2% sequester on Medicare.
We must raise the volume on how these cuts are impacting care! ACCC is joining with various state oncology societies and other organizations to send a letter to members of Congress. Please take a few minutes to send your own letter using ACCC’s template. We are also signing on to a petition that will go to President Obama urging him to exempt cancer drugs from the sequester. We invite you to sign the petition as well. ACCC will continue to keep members informed of developments with the sequester.
The Nebraska Medical Center is serving as a Community Resource Center (CRC) for ACCC’s Improving Quality Care in Small-Population Cancers: Multiple Myeloma education program. Dr. Faber is a member of the Advisory Board for this initiative. ACCCBuzz asked Dr. Faber to provide an overview of this small-population cancer and some perspective on advances that have been made in treating this disease.
Q: Can you provide a snapshot of the progress that has been made in treating multiple myeloma?
Dr. Faber: Arguably, very few other cancers have enjoyed the success of new therapies that have been derived from great translational research. These successes include new therapies and numerous combinations of therapies, which may or may not be combined with stem cell transplant strategies.
Two classes of chemotherapeutic agents, proteasome inhibitors and IMiDs, have solidified the era of “novel agents” by increasing progression-free survival and overall survival. Median five-year survivals for multiple myeloma are over 60 percent. I would anticipate this number to be better once the newest SEER data is reported.
From a certain view point, treatments for another small-population cancer, chronic myeloid leukemia (CML), might best represent our greatest advancement in the last 25 years. In the case of CML, we’ve taken a disease that was almost universally lethal without allogeneic transplantation and turned it into a chronic disease requiring a pill. For this simple reason, the strategy of targeted therapy developed for CML has ushered the era of “targeted” or “individualized” therapy. Many very intelligent people believe that’s where we’re headed with multiple myeloma.
Q: How has a better understanding of the disease biology and pathophysiology led to improvement in care for multiple myeloma?
Dr. Faber: Key pathways such as proteasome inhibition are vitally important for multiple myeloma and, to a lesser degree, mantle cell lymphoma. For reasons that are not clear, proteasome inhibition does not seem to be an important target for other cancers. Understanding the relationship between proteasome inhibition and multiple myeloma has led to drugs like bortezomib and carfilozomib.
The second class of medications is immunomodulatory agents, called IMiDs. Agents such as thalidomide, lenalidomide, and pomalidomide are the direct results of understanding the pathophysiology. Furthermore, many new therapies now available have been first proven in the lab. Direct science correlatives are now quite common, and have led to new drugs and new combinations of drugs both with and without the use of the novel agents.
Q: What is the progression of multiple myeloma?
Dr. Faber: We have made significant strides to improve up-front response rates for both transplant-eligible and non-transplant-eligible patients. Even though we are achieving deeper and more durable responses which lead to better quality of life, ultimately myeloma will relapse. We have made some progress to increase progression-free survival (PFS) and responses, but generally speaking, each successive treatment will have a shorter and shorter PFS, until finally the disease becomes refractory. A few researchers have demonstrated the heterogeneity of the disease. If I may elaborate, when we look at the myeloma genetically at relapse, it can present as different clones, which may or may not have been present at initial diagnosis. So maybe we can consider myeloma to be a series of clones. Each successive treatment addresses one or a combination of the clones, but ultimately there is one clone that becomes resistant to whatever we have and eventually claims the patient’s life.
Q: In your presentation at the ACCC 29th National Oncology Conference, you commented that multiple myeloma is not a “what you see is what you get” disease. Can you elaborate?
Dr. Faber: We know that all multiple myeloma patients relapse at some point. The question is why do patients who have achieved a complete response then relapse? Where did the disease go? Where does it hide?
With the advent of more sensitive testing, the term “minimal residual disease” is now often used. In my presentation I recalled an iceberg analogy, which I remembered hearing in a presentation by Sagar Lonial, MD, from Emory University. What we used to see with multiple myeloma in the bones, bone marrow, and the blood work was above the water—like the tip of an iceberg. Just as with the hidden underwater area of an iceberg, there was a significant disease burden that was unable to be seen or tested. And I think many of us, as we work with the more sensitive tests, realize that the newer agents eradicate not only what we see (the tip of the iceberg) but also what we had previously been unable to see. But, ultimately, there is disease deep down that you still can’t see. That’s where much of the research is currently focused. Finding a way to detect minimal residual disease is a great way to begin to discover how we can eradicate it.
Q: Ultimately multiple myeloma outsmarts the available treatments?
Dr. Faber: Correct. There are so many pathways that cells can use to signal growth in cancer cells. The pathways can ebb and flow to avoid the toxic effects of our available therapies. The current targeted agents are very good at shutting down certain pathways, but the cell then learns how to see other pathways to continue its growth advantage. That is exactly what I mean by the cell starting to outsmart the therapies. This is coupled with the fact that patients can develop side effects to treatments, which is disheartening, especially when the drug is still working but the patient can no longer have the drug because of the side effects.
Q: Today what do you see as major challenges for providers treating this disease?
Dr. Faber: Drugs that we can use for long periods of time without side effects. Finding the laboratory tests that can help us assess minimal residual disease and are easy enough to be translated into the community. We can do a lot of good things at the big centers, but sometimes it’s not feasible to translate them to the community setting because they are complicated and are not cost effective.
In terms of newer agents which have targets that may work when the available drugs stop working, it would also be nice to have specific laboratory tests to assess for when we should use certain drugs. Maybe this is where we are headed with genomics and gene expression profiling, which would lead to individualized therapy. I want to believe that multiple myeloma can be a disease that you get diagnosed and treated; then, when it relapses, a test would help characterize it so that the “right” drug(s) can be given. In other words, try to give a drug to patients when they need it, while sparing them drugs that may not be as useful at the time.
Q: What interests you most in working with multiple myeloma?
Dr. Faber: The complexity of the disease draws me—its effects on the bones, the kidneys, the heart, and the bone marrow. It truly requires a multidisciplinary approach today in order to try to coordinate the efforts of radiation oncologists, spine surgeons, orthopedic surgeons, kidney specialists, and so forth. And I think the success of the newer therapies allows us to treat patients longer while maintaining a good quality of life. The newer treatments and the newer combinations really do help quality of life.
Multiple myeloma is a very heterogeneous disease. Not all the patients are alike and few are similar. It is that variety that makes multiple myeloma challenging to treat. When you have patients that are failing the new therapies or relapsing harshly, finding the right therapy for these patients is similar to “righting the ship.” This success, of course, is very rewarding.
Q: How can physicians who may see just a few patients with multiple myeloma a year keep up with rapidly changing treatment options?
Dr. Faber: As with most cancers today, the literature can be behind. The advances happen at American Society of Hematology (ASH) and American Society of Clinical Oncology (ASCO) meetings, as well as during discussions in the conference rooms. ASH and ASCO can be overwhelming, which is why I think it is important to prepare by “attending the conference” before you actually attend the conference.
Establishing a relationship with a referral center with someone specializing in myeloma is also important. Establishing a network that you can call when you have questions or problems is also important. I don’t know very many people at referral centers who would be unwilling to help. The Community Resource Centers established through ACCC’s Improving Quality Care in Small-Population Cancers program are one way for ACCC members to access providers with expertise in treating multiple myeloma, chronic myeloid leukemia, and acute promyelocytic leukemia.