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After seeing promising effects of CAR T cell therapies in patients with relapsed/refractory leukemia and lymphoma, researchers began to explore their potential in relapsed/refractory multiple myeloma. First, researchers needed to find a target for the T cells —one that was detected in multiple myeloma cells. Scientists found a target in B-cell maturation antigen (BCMA), which is also found in healthy plasma cells, and created a new family of CAR T cell therapies. Now the question is whether these investigational medicines will help prolong survival in patients with relapsed/refractory multiple myeloma.

Dr. Nina Shah, associate professor in the Department of Medicine at University of California San Francisco, discusses how CAR T cell therapy may further transform the treatment of relapsed/refractory multiple myeloma and how researchers are looking to optimize these treatments for this still incurable blood cancer.

DR. NINA SHAH

DR. NINA SHAH FROM THE UNIVERSITY OF CALIFORNIA SAN FRANCISCO BELIEVES THAT CAR T CELL THERAPY HAS THE POTENTIAL TO IMPROVE OUTCOMES FOR SOME PATIENTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA.

What may CAR T cell therapies offer patients with relapsed/refractory multiple myeloma?

“The hope is that CAR T cell therapy may be a way for some patients with relapsed/refractory multiple myeloma to have a chance for a long-lasting treatment response. Clinical trials are still in the early stages.

Currently, multiple myeloma is treated with several repeated cycles of different therapies. Patients would most likely still go through these treatment regimens before receiving CAR T in a later line of therapy, but it would be one of the few single agent treatments these patients would receive.”

Will CAR T cell therapy be as effective in multiple myeloma treatment as it has been in other blood cancers?

“It remains to be seen. Every tumor has different properties. So it will depend on many factors, including whether targeting BCMA kills the cells that drive the cancer and whether the CAR T cells themselves disappear or stick around.

As we continue to see follow up research, we’ll look at the longevity of these CAR T cells as well as the duration of the response. Will finding these CAR T cells in patients long after treatment correlate with efficacy and survival? If so, we would try to use that information to create T cells that last longer in patients.”

What is the toxicity profile of CAR T in multiple myeloma?

“In clinical trials, patients with multiple myeloma have tolerated CAR T cell therapy as anticipated, but further investigation is needed. We can’t compare across disease states as patients themselves may experience the treatments differently in some ways. While it is uncertain why, what we do know is that as toxicities and disease states are better understood, along with further optimization of dosing, we may be in a better position to manage these responses. We have also learned from our colleagues who have been using CAR T cell therapy for longer in cancers such as in leukemia and lymphoma.”

CAR T cell therapy research offers hope for patients with relapsed multiple myeloma who have already been through several lines of treatment.

How would you characterize the early results in multiple myeloma?

“CAR T cell therapy research offers hope for patients with relapsed multiple myeloma who have already been through several lines of treatment. But we shouldn’t be overzealous.

In general, we have to work harder to understand why not all patients respond and which patients are most likely to receive a benefit from CAR T cell therapy. If we can predict that and learn why others don’t benefit and why relapses occur, then maybe we can improve how we engineer CAR T cells.”

Could CAR T cell therapy be combined with other treatments for multiple myeloma?

“There may be the potential for combinations worthy of investigation as we look toward the future with therapies such as immunomodulatory regimens and checkpoint inhibitors. Moreover, further exploration of CAR T cell therapies given simultaneously with other agents may show the potential to boost their activity. In any case, such combinations would require additional studies and careful attention should always be given toward the potential toxicities that may occur in combination strategies due to T cell overreaction.”

What other ways could researchers optimize CAR T cell therapy?

“We see lots of creativity in how we design these therapies. Some researchers are trying to grow CAR T cells under conditions that would allow them to last longer once they are given back to the patients, and others are trying to add an on/off switch to CAR T cells, so they only activate when they encounter signs of a tumor.

Meanwhile, we’re looking at when we use these therapies. Right now, CAR T cell therapies are being studied in patients who have tried and relapsed on multiple treatments. Using it sooner might be useful for patients with high-risk disease who don’t have very durable responses to traditional treatments, but this will require additional research. So I’m looking forward to seeing the results of clinical trials as we learn more about CAR T cell therapies in the multiple myeloma field.”

To learn more about the ongoing research into CAR T cell therapies, read “CAR T Cell Research Continues to Advance.”

Long Island isn’t the best place to train for climbing Iceland’s mountains and volcanoes. But it was here, in his hometown of Oceanside, NY, where Rich Appelbaum prepared for the Moving Mountains for Multiple Myeloma (MM4MM) team Fire & Ice trek across Iceland.* While five straight days of hiking for seven to eight hours a day would be tough for almost anyone, it posed an extra challenge to Appelbaum, who was diagnosed with multiple myeloma in 2016 at the age of 62. Two years later, he found himself on a life-changing excursion.

MM4MM organizes treks that are more than just a physical challenge for its teams, with members who are all directly affected by multiple myeloma as patients, family members, friends, clinicians or advocates. This collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) brings attention to the need for additional research and treatments for the more than 118,000 people in the U.S. living with this cancer and an estimated 32,000 others were diagnosed in 2018. Team members each raised at least $7,500 for the MMRF, and together the Iceland team raised more than $135,000.

Pushing past the diagnosis

Appelbaum’s 2016 diagnosis was unexpected, arising out of a routine blood test for a life insurance application. “I was honestly expecting to receive a preferred status,” he said. Instead, he was not eligible, based on extremely high levels of protein in his blood. After a biopsy and bone marrow test, Rich was perplexed by his diagnosis, because he had never heard of multiple myeloma.

Rich Appelbaum's Team

MULTIPLE MYELOMA DIDN’T STOP RICH APPELBAUM FROM KEEPING UP WITH HIS TEAMMATES. AS THE TOP FUNDRAISER OF THE GROUP, HE RAISED MORE THAN $20,000 FOR THE MULTIPLE MYELOMA RESEARCH FOUNDATION.

He started treatments in January 2017 but continued to encourage himself. “When you’re diagnosed with cancer, certain doubts creep into your mind as to what you’re going to be able to do,” he said. The idea of hiking volcanoes, glaciers, lava beds, gorges and up to waterfalls was a personal challenge that motivated him.

Surpassing expectations

Appelbaum was a novice hiker. “I’d never even carried a backpack before, and I was told I’d need to get used to carrying an extra 30 or 40 pounds on my back,” he said. he filled a backpack with weights, put on hiking boots and walked around Long Island with his wife. Eventually, he joined friends on day trips to nearby trails on the weekend. “My motto has always been, if it’s worth doing – it’s worth overdoing.”

Appelbaum encountered his first hurdle before the trek even began. Initially, the MMRF didn’t respond to his application. As the closing date approached, he sent them videos of himself at the gym with accompanying music from the film “Rocky.” Before long, the organizers offered him a spot on the trip. Good thing for the MMRF, too – Appelbaum not only completed and in some instances excelled on the hike but was the top team fundraiser, bringing in more than $20,000 for multiple myeloma research.

In the process of raising funds, he also raised awareness of multiple myeloma and the MMRF among the thousands of his followers who read his social media updates about the trek. “I made it clear that all the funds would go to the MMRF. That made it understandable for people to support me.”

My motto has always been, if it’s worth doing – it’s worth overdoing.

RICH APPELBAUM BONDS WITH HIS TEAMMATES DURING THEIR TREK ACROSS ICELAND.


RICH APPELBAUM, CENTER, IS MOST GRATEFUL FOR THE BONDS HE MADE WITH HIS TEAMMATES DURING THEIR TREK ACROSS ICELAND.”

 

Forging friendships on the trek

While completing the trek was a major personal accomplishment, Appelbaum’s most valuable takeaways were the friendships he forged with his teammates and the stories they shared while hiking through the craggy landscape. Even eight months later, the teammates are still in touch daily. “I’m inspired by all of them,” he said. “One of my teammates was told she had a 20 percent chance of living five years when she was diagnosed; she has already lived 10.”

Appelbaum is also grateful for the bonds he made with the health care professionals on his team. “They’ve been very supportive and I consider them good friends as well.”

Stepping out of his comfort zone

Appelbaum sees parallels between the excursion and his battle with multiple myeloma. “The trek definitely forced me out of my comfort zone, and I think dealing with cancer does the same,” he said. “You may no longer necessarily be able to do things on your own terms or your own schedule.”

The experience also made him appreciate recent treatment advances, including those supported by the MMRF, which has supported more than 350 research grants worldwide and has helped bring ten drugs to market since its inception. “I’m truly amazed by how often I read about advances in medication for multiple myeloma and ways to identify which medication is best for each patient,” Appelbaum said.

To learn more about the MMRF and how you can support multiple myeloma research, visit the MMRF website.

*The MM4MM Fire & Ice trek across Iceland was partially sponsored by Celgene Corporation.

Since 2006, Medicare Part D has helped millions of American seniors gain access to their prescribed medications. In particular, the federal prescription medication coverage program has been remarkably effective for helping individuals with the blood cancer multiple myeloma in accessing their medicines. According to a new study published in the Journal of Clinical Oncology, not only has the program increased the use of newer or oral anticancer therapies among people with multiple myeloma, but may also be associated with helping those patients live longer.

For this year’s Multiple Myeloma Action Month, study author and Yale University professor Amy J. Davidoff, Ph.D., explains how Medicare Part D may have an impact on patient outcomes and what this means for the future of access to oral therapies for cancer.

AMY J. DAVIDOFF, PH.D.

IN A RECENT STUDY, AMY J. DAVIDOFF, PH.D., FROM YALE UNIVERSITY AND HER CO-AUTHORS FOUND THAT PATIENTS WITH MULTIPLE MYELOMA WHO HAD PRESCRIPTION MEDICATION COVERAGE HAD BETTER SURVIVAL OUTCOMES.

How does Part D impact what treatment a patient receives for multiple myeloma?

“It’s a complicated situation because there are the oral and IV (intravenous) options for multiple myeloma treatment. The out-of-pocket costs depend not only on the medication’s method of delivery but also on whether a patient has supplemental medical coverage (e.g. Medigap), prescription medication coverage or both.

These supplemental coverage programs are vital to protecting patients from large out-of-pocket burdens. In some cases, the types of coverage they have may lead them to choose one therapy over another or enable them to afford a combination of medications, which is common for multiple myeloma.”

Does Part D and other insurance plans impact survival for multiple myeloma?

“Our study revealed that multiple myeloma patients were more likely to receive any myeloma therapy, but less likely to received older IV therapies if they had prescription medication coverage compared to those without such coverage. We could not directly observe, but presume that they were receiving oral therapy. Patients covered by Part D or other creditable prescription medication coverage programs had median overall survivals that were 16 percent higher than the non-covered group.

Prescription drug coverage becomes an important part of the equation when patients have the option to take oral therapies, which is the case with multiple myeloma.

Why is prescription medication coverage particularly important in multiple myeloma?

“Prescription drug coverage becomes an important part of the equation when patients have the option to take oral therapies, which is the case with multiple myeloma. Patients with other cancers, such as diffuse large B cell lymphoma, are most often prescribed IV chemotherapy, which is administered in an outpatient setting and is covered under a patient’s medical benefits. As a result, our study found Medicare beneficiaries with this lymphoma saw no significant difference in survival with Part D coverage.

Oral therapies for multiple myeloma, however, were introduced in 2006, so our study was able to cover an adequate period of their use, from 2006 to 2013. During the last decade, oral therapies have been developed for other malignancies such as breast cancer and melanoma. As time progresses, we could see similar research on prescription coverage and patients with various cancers.”

Why don’t more seniors opt for prescription medication coverage?

“The few seniors who decline Part D coverage may perceive themselves as relatively healthy. They may assess the cost of Medicare Part D coverage compared to the cost of their current medications and decide that they don’t need to enroll. Health care professionals understand that your health status today does not necessarily determine your status tomorrow, but not all people understand the risk of future health problems and may not understand the potential risks of not enrolling.

Many of those seniors regret this decision later on. For example, 41 percent of non-covered patients enrolled in a prescription plan the year after being diagnosed with multiple myeloma. There is definitely an ongoing need to better inform the Medicare population of their coverage options during open enrollment.”

What are your thoughts on the implementation of Part D and its future?

“We need to change the way seniors enroll in Medicare programs. The process as it is now is confusing and far from ideal. There are a lot of opportunities to improve the enrollment site and provide beneficiaries with better information.

In addition, there’s a need to establish better caps on out-of-pocket costs. This would be very useful. The goal is to make prescription medication coverage as affordable as possible for Medicare beneficiaries.”

To learn why policymakers must act to protect prescription coverage for America’s seniors, read “Medicare Part D: Challenges Loom Ahead.”

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At 6:21 p.m. on September 18, 2016, the sun was beginning to set in New York City, and Eric Gelber started his final lap around Central Park. A crowd of supporters were running with him and cheering him on, and cowbells were ringing along the race course. Every step was painful. When he finally crossed the finish line, he broke down and kissed and hugged his wife. He had just run 200 miles and raised over $320,000—bringing his lifetime fundraising total to more than $1.1 million and moving us one step closer to a cure for multiple myeloma.

But for Gelber, the journey is not over yet. Now, he’s inviting other runners from the New York City area and beyond to join him on The Journey Towards a Cure initiative by taking part in a 12-hour endurance run/relay on October 6, 2018, to support the Multiple Myeloma Research Foundation (MMRF).

In anticipation of this new chapter in a personal journey he started more than a decade ago, Gelber explains what keeps him coming back to this cause, how this year’s event will be different than his previous efforts and why it remains vital to support multiple myeloma research.

What did it feel like to finally achieve that 200-mile run?

“It was unbelievable. I was somewhat out of it when I finished, but it felt like a dream. After two failed attempts, it was an amazing feeling to fulfill that goal. I was thinking of what all the people on my support team and I had just accomplished. Together, we pushed our cumulative fundraising total to over $1.1 million.

I’m not sure what would have happened if I did not complete all 200 miles. I decided it would be my final attempt before I did it. The following year, a documentary was released at the Tribeca Film Festival to tell my story and the stories of my friends who have been affected by multiple myeloma. I hope our story inspires more people to support this effort.”

ERIC GELBER AND HIS WIFE DISCUSS HIS PROGRESS AFTER COMPLETING 176 MILES DURING HIS FINAL ATTEMPT TO RUN 200 MILES IN SUPPORT OF THE MULTIPLE MYELOMA RESEARCH FOUNDATION IN 2016.

What kept you coming back year after year to push yourself to your limits and raise money for multiple myeloma research?

“Over time, this became a massive part of our lives, and I keep coming back because I want to do as much as I can to support the MMRF.

I’m not a scientist. I’m not a doctor. But I can run, and I can fundraise. That’s my way of getting us one step closer to a cure for multiple myeloma.”

This year, you are encouraging others to run a 12-hour relay race. Why did you feel it was time to change the event?

“This year, there is a different format. I wanted others to feel like they were actively helping the cause as well. An event that gets people to come out and push themselves—either solo or in a team—expands the idea of building a community. I didn’t expect it would take this format, but it was time to evolve.

There will be the race followed by a celebration with food, kids’ activities and a music festival with four live bands. It’s important to give people a reason to come together, share their stories and celebrate life in a fun atmosphere. I’m looking forward to this new chapter.”

What advice do you have for someone who wants to get involved with this year’s event?

“If you’ve never run a race before, be cautious and set achievable goals. Pace yourself so you avoid getting injured. There is an athlete in all of us, but you don’t have to run for 12 hours. You can a join a five- or 10-person relay team and run for as long as you can. As long as you’re challenging yourself, there’s a lot to be proud of.

If you are unable to run, there’s the option to walk a loop on the course. We also need volunteers to help direct traffic, register participants and manage aid stations. All of that is critical to making this event possible.”

One of my friends has been living with multiple myeloma for 20 years. Part of that is because there are more treatments available now. It’s very encouraging.

How do you feel about the progress made in the treatment of multiple myeloma over the last decade?

“I see progress in the faces of my friends who have multiple myeloma. They’re living longer. One of my friends has been living with multiple myeloma for 20 years. Part of that is because there are more treatments available now. It’s very encouraging.

There’s still a considerable amount of work to be done. We still don’t have a cure. That’s why we’re opening the event to a broader audience. I’m still learning about organizing and putting together an event as big as this. But sometimes you have to go for it and try—just like attempting to run 200 miles. You take it one step at a time.”

To learn more about the upcoming The Journey Towards a Cure event, visit The Journey Endurance Run/Relay website.

While innovative therapies have helped improve the relative survival rate for patients with multiple myeloma, the disease remains incurable with most patients experiencing repeated relapse and ultimately becoming refractory to treatments. Today, research continues to delve into whether treating relapsed multiple myeloma with combination therapies may be an appropriate option for certain patients. More agents could also result in increased toxicities and patients must be monitored closely.

­At the 54th Annual Meeting of the American Society of Clinical Oncology (ASCO), new data regarding the use of triplet therapy for relapsed multiple myeloma were presented. Now four or more drug combinations are being examined as well. In this interview, Dr. Paul Richardson, the RJ Corman Professor of Medicine at Harvard Medical School and clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, discusses the new data presented at this year’s ASCO meeting and how the future of relapsed multiple myeloma treatment could look.

DR. PAUL RICHARDSON

DR. PAUL RICHARDSON FROM DANA-FARBER CANCER INSTITUTE BELIEVES THAT WE WILL CONTINUE TO SEE PROMISING RESULTS FROM COMBINATION THERAPIES FOR RELAPSED/REFRACTORY MULTIPLE MYELOMA.

Why have triplet and quadruplet regimens become more frequently used to treat relapsed multiple myeloma?

“Multiple myeloma is a heterogeneous disease, which means that patients typically have multiple subpopulations of malignant plasma cells with different molecular profiles and characteristics. When patients with multiple myeloma are given a single therapy, some of these subpopulations recede while others may grow. This phenomenon is believed to contribute to treatment resistance and eventual relapse.”

“So the theory that combination therapies provide a multi-pronged attack to target multiple myeloma subpopulations may be a reason why we are seeing more relapsed multiple myeloma patients being treated with combination therapies. Quadruplet regimens are also being investigated for high-risk patients with particularly resistant multiple myeloma.”

How have data presented at this year’s ASCO meeting moved the field forward?

“At ASCO, we saw new data from clinical trials comparing triplet with doublet regimens for relapsed multiple myeloma. Those findings support the idea that if you throw a wider net around the illness early with a triplet, you may achieve greater clinical benefit. At the same time, as we continue to explore new combinations, we are finding more regimens that may be better tolerated by patients.”

Why do many of these combinations include immunomodulators?

“Immunomodulators are an integral part of multiple myeloma treatment in both the newly diagnosed and relapsed/refractory setting. These therapies have numerous effects on tumor cells, the immune system, and the tumor microenvironment which we believe leads to their anti-multiple myeloma activity. In fact, they constitute a foundation for therapy regimens in multiple myeloma.”

“At this year’s ASCO, we saw important new data from trials exploring the interactions between these therapeutic classes in the relapsed setting.”

Although the treatment of relapsed multiple myeloma remains an unmet medical need, we’re making progress. The data presented at ASCO on combination therapies and potential breakthrough approaches like CAR T cell therapy are particularly exciting.

How are researchers taking a more precision-focused approach to treating relapsed multiple myeloma?

“Choosing the most appropriate therapy based on the genetic causes of a disease makes sense but is challenging in multiple myeloma, because the cancer is so genetically unstable. For example, we sequenced the genome of one patient at diagnosis and identified over 5,000 disease-associated mutations. By the time he relapsed, we had found more than 12,000. When you have that number of genetic changes, targeting a single mutation will be unlikely to make much of a difference on its own.”

“To help address this problem, we have to take a modified precision medicine approach. Researchers are studying whether combining backbone agents such as immunomodulators and proteasome inhibitors that have broad success in the disease with more targeted agents and evaluating these combination regimens, and in particular with monoclonal antibodies.”

What are your thoughts on the future of relapsed multiple myeloma treatment following this year’s ASCO meeting?

“Although the treatment of relapsed multiple myeloma remains an unmet medical need, we’re making substantial progress. The data presented at ASCO on combination therapies and potential breakthrough approaches like CAR T cell therapy are particularly exciting. While the FDA approved CAR T cell therapies have already shown promise in other cancers, multiple myeloma is a much tougher nut to crack. So we’ll have to wait and see what happens with these trials in the longer term, but early data are encouraging.”

To learn more about the advances discussed at ASCO 2018, read “ASCO 2018 Preview: Precision Medicine, CAR T Cells and Immunomodulators.”

Dr. Richardson regularly provides input as a paid consultant for Celgene.

The cost and value of medical innovation in oncology is one of the hot topics on the agenda for this year’s American Society for Clinical Oncology Annual Meeting (ASCO). One cancer that has seen an increase in relative survival rates over the past decade is multiple myeloma. So it makes sense that multiple myeloma is at the center of a debate at ASCO on the cost and value of new therapies.

As one of the participants in that debate, Dr. Rafael Fonseca, a hematologist, oncologist and chair of the Department of Internal Medicine at Mayo Clinic in Arizona, will argue that society can’t afford not to provide patients with multiple myeloma access to the best care possible. In this Q&A, Fonseca shares his views about the affordability of cancer therapies, why many doctors hold onto the notion that medications are too expensive and the implications for future multiple myeloma treatment.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

In the debate, you will argue that we can’t afford not to provide patients with access to the right treatment for them. How did you come to that conclusion?

“Over the past decade, I’ve witnessed so many patients with multiple myeloma increasingly beating the odds for survival. So I wanted to know what exactly was responsible for this. After some research, I concluded—as many of my colleagues have also—that it was the new medications. These innovative therapies provide tremendous value to our patients and society.”

When you discuss this topic with your fellow oncologists, what is the most compelling evidence supporting your position?

“I usually take a stepped approach to presenting my point of view. I ask them to consider the progress that we’ve made in survival in cancers like multiple myeloma and what they have seen in their own patients. I help them understand the value by walking them through all the new therapies that those improvements are attributed to. You can’t just look at the price tag of a specific medication, which they often focus on most.”

The opposing position is that patients and society cannot afford multiple myeloma therapies. Why don’t you believe that argument?

“The data don’t support the argument. One study found that 98 percent of patients paid $50 or less to fill their prescriptions in 2017. While that could be a hardship for some people, it is far different than the list prices of thousands of dollars that make headlines. So we need to talk about what patients are paying in the real world and what’s best for our patients.”

“Beyond that, there are the ethical considerations. Doctors should prescribe the medications they believe will benefit their patients the most.”

Why do doctors continue to say that new cancer therapies are unaffordable if the data suggest they aren’t for most patients?

“I feel like most cancer doctors are concerned about the cost of prescription medications out of their sense of compassion and responsibility for their patients. They see their role as treating patients responsibly. Prescribing a therapy that may cost them thousands seems inconsistent with that mission.”

“But they are so busy caring for patients that they don’t have the time to research the real world data about what patients actually pay for their prescriptions. As we see in other areas of discourse, facts matter. I believe that misunderstood empathy and baseless rhetoric can have real-world consequences for patients.”

Everyone agrees that today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care.

Have you ever had a patient —or many—who could not access treatments that you prescribed?

“I cannot think of a single patient who could not access a medication due to financial reasons. I’ve had patients who have chosen other treatment options but for other reasons—never financial. In a few instances, we have had to go above and beyond to get them financial help from the manufacturer or non-profit groups. But those cases are the exceptions, not the rule.”

What do those on the other side suggest should be done about the affordability of multiple myeloma care and what are the potential consequences?

“When we say that new therapies are too expensive, what we’re doing is calling for price regulations. But without a doubt in my mind, those regulations will kill innovation. Medical innovation is a high-risk, high-reward endeavor. We should not fool ourselves into thinking that there won’t be consequences; we’ll have fewer new treatments for our patients as a result.”

Is there common ground in this debate over the value of multiple myeloma care that can be used to move forward?

“There is plenty of common ground. I think today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care. So we should make sure that we do not hinder that innovation.”

“Most cancer doctors also agree that clinical trials should be as fast as possible without sacrificing safety, so they cost less and lead to faster approvals. And I think that we all think patients should have access to the best treatment options but have different ideas on how to provide that access.”

To learn more about the how medical innovation has improved the treatment of multiple myeloma, read “A Decade of Progress in Multiple Myeloma, and More to Come.”

Dr. Fonseca has received speaker fees, advisory board fees, travel support in connection with consulting services, and research support from Celgene.

Since Amy Wolverton was diagnosed with multiple myeloma in 2008, she has been on numerous chemotherapy regimens. When one treatment stopped working, her doctor would recommend another. Sometimes the new treatment would be administered intravenously (IV); other times it would be a pill. Like many cancer patients, Amy has faced hurdles with insurance coverage for her cancer treatment.

During this year’s Multiple Myeloma Action Month, Wolverton wants people to know that some insurers are charging patients with cancer more for oral therapies (pills) than for IV therapies due to differences in how the two forms are covered. Intravenous chemotherapy falls under medical benefits, but oral cancer therapies are included in prescription medication benefits, which have much higher cost sharing for patients. As a result, some patients are forced to make treatment decisions based on their out-of-pocket costs rather than what is best for their health and survival.

“It makes little sense to me,” Wolverton said. “When you add up the medical supplies, doctors’ and nursing costs, facility overhead, and costs for complications such as infections from IVs, the insurance companies could be paying more for the IV than they pay for the pills.”

AMY WOLVERTON’S (CENTER) PARENTS, JANICE AND LEE BURKHEAD, HAVE BEEN SUPPORTING HER THROUGHOUT HER JOURNEY WITH MULTIPLE MYELOMA TREATMENT.

AMY WOLVERTON’S (CENTER) PARENTS, JANICE AND LEE BURKHEAD, HAVE BEEN SUPPORTING HER THROUGHOUT HER JOURNEY WITH MULTIPLE MYELOMA TREATMENT.

The Rise of Oral Chemotherapy

Over the past decade, Wolverton has seen the progress made in multiple myeloma and the increased availability of oral therapies. In 2017, nine of the 16 cancer treatments approved by the U.S. Food and Drug Administration were oral therapies. For many oral therapies, there is no IV equivalent. And, for some patients with blood cancer, a pill may be the only option.

When she was on an oral cancer treatment, Wolverton took a pill at home each morning and continued her routine, working full-time as a corporate government affairs executive in the telecommunications industry and spending time with friends and family, including with her mother Janice, her father Lee, her brother Jason, sister-in-law Carla, and her nephew Dylan, who is now 10.

Like many patients with multiple myeloma, Wolverton has had to find new therapies as each consecutive treatment stopped working for her. “It’s frightening and frustrating when a treatment stops working,” Wolverton said. “But, even though we have yet to find a more curative treatment for myeloma, we remain hopeful with all the new treatments, both oral and IV, that have been approved.”

I’m doing everything I can to help others with multiple myeloma and other cancers get the best possible treatment for themselves.

Oral Parity Protects Patients

Wolverton strongly believes that all patients with multiple myeloma should have equal access to all treatments, regardless of how they are taken. Most state lawmakers agree, and they have taken action.

Since 2008, the year that Wolverton was diagnosed, 43 states and the District of Columbia have enacted oral parity laws, which make the patient’s financial responsibility for covered medications equal for IV and oral therapies. Meanwhile, the Cancer Drug Coverage Act  has been introduced in the U.S. House of Representatives to protect the millions of people with plans that are not state regulated, including most plans offered by large employers.

AMY WOLVERTON AND HER NEPHEW DYLAN TAKE A SELFIE TOGETHER.

AMY WOLVERTON AND HER NEPHEW DYLAN TAKE A SELFIE TOGETHER.

With her background in legislative issues, Wolverton knows how those laws can help patients. She continues to advocate for these policies, joining the International Myeloma Foundation in its activities. She believes it’s her job to speak up for everyone who struggles to get affordable access to oral cancer treatments.

Over the years, Wolverton has switched insurance companies and experienced changes in out-of-pocket treatment costs. But, she is doing well on her current medication and remains optimistic about her future. “I’m just so grateful to be here,” she said. “And I’m doing everything I can to help others with multiple myeloma and other cancers get the best possible treatment for themselves.” 

To learn about how Congress can act to protect Americans from outdated insurance policies, read “Why Federal Oral Parity Legislation Is Urgently Needed Today.”

Knowledge is power, and Chad Saward, director of patient advocacy at Celgene, knows as well as anyone that a little knowledge goes a long way for people with multiple myeloma . That’s why he and Celgene are dedicated to helping patients understand their options, not only from a treatment perspective, but also from financial, emotional and psychological perspectives.

At last year’s national summit for leaders of multiple myeloma support groups hosted by the International Myeloma Foundation, Saward was approached by a patient who was struggling to pay for a Celgene medication he had been prescribed. Saward referred the patient to Celgene Patient Support®  , which helps patients who have been prescribed Celgene medications understand their insurance and learn about financial assistance that may be available to help them pay for their Celgene medicine. For this patient, a Celgene program helped to substantially reduce the patient’s out of pocket responsibility for a Celgene therapy and provided some peace of mind.

“At Celgene, we’re committed to improving patient care, and education is an essential component of that goal,” Saward said. “For patients with multiple myeloma, there’s definitely a benefit to being informed and engaged when trying to manage this deadly cancer.”

Chad Saward, Celgene Patient Advocacy

CHAD SAWARD, DIRECTOR OF PATIENT ADVOCACY AT CELGENE, KNOWS THAT A LITTLE KNOWLEDGE GOES A LONG WAY FOR MULTIPLE MYELOMA PATIENTS.

At diagnosis, multiple myeloma patients often start treatment immediately and have little time to learn about the disease, their prognosis and treatment options. For resources and support around these topics to be useful, they must be readily available and accessible.

At Celgene, we’re committed to improving patient care, and education is an essential component of that goal.

Over the past decade, Celgene has supported patient education initiatives for several multiple myeloma advocacy groups including the International Myeloma Foundation, the Multiple Myeloma Research Foundation, The Leukemia & Lymphoma Society, and Myeloma Crowd.

“The role that these patient organizations play is vital to providing education and support that can make a significant difference in a patient’s life,” Saward said.

Every patient has a different experience based on their age, time of diagnosis and their ability to cope with stressful situations, among many other factors. So the information and programs provided to patients need to recognize those differences. For instance, through its Standing in the Gaap initiative, Celgene has created educational resources to raise awareness about how multiple myeloma affects African Americans differently, including a higher risk of developing the disease when compared to white Americans.

Supporting multiple myeloma patients includes offering resources for the people they rely upon, such as their physicians, caregivers and policymakers. Since doctors and nurses are often a patient’s main source for disease education, Celgene has resources such as the Multiple Myeloma Center for Nurses and Myeloma Central. On the policy side, Celgene and the Patients Equal Access Coalition are working together to educate legislators on the need for oral parity legislation to ensure patients have access to and coverage for the most appropriate therapies, regardless of how they are administered.

In the end, these efforts have brought Saward closer to understanding the unmet needs of multiple myeloma patients. “I have the privilege of talking with patients, many of whom I consider my friends,” Saward said. “Tremendous progress has been made with multiple myeloma treatments, but we need to keep our eye on the ball. I still continue to lose friends to this disease.”1

For more information about myeloma symptoms and treatments, visit the websites of these organizations:

When Reverend Veronica Graves had pain in her leg four years ago, both she and her doctor thought it was an exercise injury that would improve with rest. But the pain persisted, and a second trip to the doctor months later revealed a broken right femur. More tests revealed the fracture was the result of a blood cancer called multiple myeloma.

As an African American, Graves was more likely to be diagnosed with multiple myeloma. Based on population growth projections from the U.S. Census Bureau, the rate of multiple myeloma among African Americans is expected to increase over the next two decades. In 2011, African Americans made up 19 percent of men and 23 percent of women who were diagnosed with multiple myeloma; by 2034, those numbers are expected to jump to 23 percent and 24 percent, respectively.

“I am saddened by the news that anyone—regardless of their race and ethnicity—is diagnosed with this disease,” Graves said. “But that statistic particularly troubles me because I don’t know if research alone will help us address the issue.”

Increased understanding of patient demographics further exposes the racial inequality that exists when it comes to access to care. African Americans are less likely to receive the latest treatments and more likely to face treatment delays than white Americans. If African Americans make up a larger proportion of multiple myeloma patients, then more patients could experience barriers to the most appropriate care.

The root causes of this disparity are not known, but some evidence points to differences in socioeconomic status as a contributor.

Elisa Weiss

ELISA WEISS, PH.D., SENIOR VICE PRESIDENT OF PATIENT ACCESS AND OUTCOMES AT THE LEUKEMIA & LYMPHOMA SOCIETY, BELIEVES THERE NEEDS TO BE A CONCERTED EFFORT TO PROVIDE AFRICAN AMERICANS WITH BETTER MULTIPLE MYELOMA CARE.

“What that would mean is that it’s the poorest African Americans who are experiencing the greatest disparity in care,” Elisa Weiss, Ph.D., senior vice president of Patient Access and Outcomes at The Leukemia & Lymphoma Society (LLS), said. “There needs to be a concerted effort to provide African Americans with better care where they live and access to the latest treatments at cancer centers.”

Earlier this year, the LLS kicked off an initiative to do just that. Working with the National Black Church Initiative (NBCI), the organization launched a pilot program this past March in Washington, D.C., and Atlanta. The Myeloma Link initiative is providing African American communities in these cities with tools and resources to help navigate the multiple myeloma treatment landscape and improve their quality of life.

One aspect of the program is Myeloma Sundays. In participating churches, churchgoers will hear a multiple myeloma expert give a “health sermon” about the African American community’s increased risk for multiple myeloma and what they should know about treatments. During the fellowship hour after the service, LLS staff and volunteers are available to answer any follow-up questions.

“Black churches have historically been a trusted source of information for African Americans,” said Graves, who is working with LLS on this program. “When the community needs to know about something that is happening, they can always trust that their pastor has the latest, most credible information. LLS’s Myeloma Link program is just a great partnership.”

Group at United Methodist Church in Atlanta

AT CASCADE UNITED METHODIST CHURCH IN ATLANTA, THE LEUKEMIA & LYMPHOMA SOCIETY HANDED OUT INFORMATION TO THE AFRICAN AMERICAN COMMUNITY ABOUT THE SYMPTOMS AND TREATMENTS OF MULTIPLE MYELOMA AS PART OF THE MYELOMA LINK: CONNECTING AFRICAN AMERICAN COMMUNITIES TO INFORMATION, EXPERT CARE, AND SUPPORT PROGRAM.

Beyond Myeloma Sundays, churchgoers will be encouraged to connect with LLS information specialists. These experts can provide multiple myeloma patients in the African American community with information about financial support, treatment options and clinical trials.

We want to ensure that African Americans feel empowered to ask their providers about participating in clinical trials.

This support may also help address a serious disparity that exists in participation in clinical trials that evaluate new treatment options. In 2016, African Americans represented just 7 percent of patients enrolled in clinical trials.

“When faced with discrimination in daily life, it’s understandable that you might think you would be treated differently in a clinical trial,” Weiss said. “We work to educate patients about clinical trials, address these kinds of concerns, and remove any stigma associated with participation in a trial. We want to ensure that African Americans feel empowered to ask their providers about participating in clinical trials.”

Churchgoers in Atlanta

CHURCHGOERS IN ATLANTA LISTENED TO A “HEALTH SERMON” ON MULTIPLE MYELOMA GIVEN BY A LOCAL EXPERT

So far, the program has been well received, according to Weiss. LLS is now developing local resource books, support groups and peer-to-peer connections between ambassadors and patients within African American communities.

Reverend Graves believes that providing support for the African American community is only the starting point. “The onus can’t rest only on African Americans to be more proactive. The doctors who serve the African American communities must help everyone get the most appropriate treatments across the board. That’s essential to eliminating these disparities in multiple myeloma.”

To learn more about the increased risk for multiple myeloma that African Americans face, read “Myeloma Is Twice As Likely To Strike African Americans.”

When Michael Tuohy was diagnosed with multiple myeloma in 2000, the only thing he could think about was living long enough to walk his then 7-year-old daughter Ally down the aisle and seeing his 2-year-old son Mikey graduate from college. At the time, multiple myeloma patients had just a 35 percent chance of living five years. But 17 years later, Tuohy is about to see his wishes come true; his daughter is planning her wedding for next year, and his son is now a freshman in college majoring in biomolecular science.

Since his diagnosis, Tuohy and his wife, Robin, have seen a lot of positive advances in multiple myeloma treatment and support. During this year’s American Society of Hematology (ASH) annual meeting, they are giving back to the community by sharing what they’ve learned.

What has changed in the 17 years since Michael’s diagnosis?

Robin: When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them. I also stress to people to stay fit and get all their check-ups. You have to keep your body in good shape. As long as you can stay healthy and take care of yourself outside of your multiple myeloma, those treatment options are your future. Don’t be afraid of your options.

Michael: We’ve been going to the ASH annual meeting for a few years now. Lately, it’s been standing room only. Thousands of participants are attending the presentations to hear about the latest developments. It blows my mind and gives me hope for the future of multiple myeloma.  

Michael Tuohy and wife at ASH in 2014

SINCE MICHAEL TUOHY’S MULTIPLE MYELOMA DIAGNOSIS, HE AND HIS WIFE ROBIN HAVE ATTENDED MEDICAL MEETINGS TO LEARN ABOUT THE LATEST TREATMENTS AND SHARE THAT INFORMATION WITH OTHER PATIENTS.

How did you become involved with the multiple myeloma community?

Michael: After my diagnosis, we searched out the best resources and found the International Myeloma Foundation (IMF) website. Geraldine Ferraro had just announced she had multiple myeloma. The IMF was asking patients to write letters to Congress about battling multiple myeloma and the importance of funding research. Susie Novis Durie, the founder of the IMF, saw our letter and asked us to share our story in Washington that year. We both went, and it was the first time we met any other multiple myeloma patients. I had gone about six months without meeting another patient. We became empowered through the IMF and decided it was time to take action to help others.

Why did you start a multiple myeloma support group?

Robin: Connecticut did not have a multiple myeloma support group, and Susie suggested that we start one. But we weren’t the kind of people who sit around and whine. So we decided our group — the Connecticut Multiple Myeloma Fighters’ Information Group — would focus on empowering multiple myeloma patients and caregivers to have better-informed conversations with their doctors and to learn about treatment, side effects, clinical trials and the importance of seeking out the opinion of a myeloma expert.

In 2005, the IMF hired me to help with its growing Support Group Program. Today, there is a network of over 150 myeloma specific groups in the United States and over 300 worldwide. The IMF has numerous programs specifically geared to ensure support and information are available at the local level. The IMF applauds the efforts and commitment of these groups and conducts an annual summit to offer help, training, and encouragement to support group leaders.

When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them.

How does your support group help new multiple myeloma patients?

Michael: The first thing we tell people is to see a multiple myeloma specialist. Their local oncologists are treating many different cancers. Form a team with your local oncologist to work with a myeloma specialist. Myeloma is one percent of all cancers and a very individual disease; what works for one may not work for another. You still need a local oncologist, someone who can do your blood testing and know your history in case you need hospitalization close to home. But you want a multiple myeloma specialist as well. You want someone who is conducting clinical trials and knows the latest treatments and research. It should be a collaboration between your multiple myeloma specialist, local oncologist and other experts. You have a lot of people working for you that you need to coordinate — you’re the captain of the ship.

Michael Tuohy, MM survivor, with his family

MULTIPLE MYELOMA SURVIVOR MICHAEL TUOHY (SECOND FROM THE LEFT) AND HIS WIFE ROBIN (FAR RIGHT) FEEL FORTUNATE THAT HE HAS LIVED LONG ENOUGH TO SEE HIS DAUGHTER ALLY (RIGHT) GRADUATE FROM COLLEGE. SINCE THEN, SHE HAS STARTED HER CAREER AS AN ELEMENTARY SCHOOL TEACHER, AND HIS SON MIKEY (FAR LEFT) HAS BEGUN HIS COLLEGE STUDIES AS A BIOMOLECULAR SCIENCE MAJOR.

How does your attitude make a difference in living with multiple myeloma?

Robin: We’ve never lived our lives waiting for the other shoe to drop. We’ve remained positive. You have to be confident in your doctor and the treatment that you’ve decided on together. Knowledge is power. Patients should learn as much as they can so they can have conversations with their doctors and remain engaged.

How else are you giving back to the community?

Robin: Our whole family has gotten involved in advocating for increased funding for multiple myeloma research, U.S. Food and Drug Administration approval of new therapies and oral parity laws. Those laws push for equal insurance reimbursement for anti-cancer treatments regardless of the delivery method. Michael was on an oral therapy at one point, so we know the benefits; it allowed him to go home, take a pill and get on with his normal routine.

A silver lining to the multiple myeloma journey is seeing and appreciating our children become advocates and doing good things in the world. Our son Mikey is now in his first year of college, studying biomolecular sciences. Our daughter is an elementary teacher.

Multiple myeloma is a growing problem. In Europe, the number of people who will be diagnosed with this blood cancer is projected to increase 17 percent by 2025, according to the World Health Organization based on 2012 figures. With these rising numbers comes a greater burden on healthcare systems and society, not to mention patients.

MICHAEL ZAIAC, VICE PRESIDENT OF CELGENE EMEA, EXPLAINS WHY MULTIPLE MYELOMA CASES ARE ON THE RISE AND WHAT THE FUTURE HOLDS FOR TREATING THE DISEASE.

MICHAEL ZAIAC, VICE PRESIDENT OF CELGENE EMEA, EXPLAINS WHY MULTIPLE MYELOMA CASES ARE ON THE RISE AND WHAT THE FUTURE HOLDS FOR TREATING THE DISEASE.

Fortunately, treatment advances over the past 20 years are helping people with multiple myeloma live longer lives. The number of clinical trials in multiple myeloma has approximately tripled since the early 2000s, and in fact, this cancer stands out as a striking example of what medical innovation can accomplish. Between 1990 and 2011, five-year relative survival rates for patients with multiple myeloma increased more than 60 percent, compared with 15 percent for all cancers. Some innovative therapies may also help patient quality of life for those with multiple myeloma.

As we commemorate Blood Cancer Awareness Month, Dr. Michael Zaiac, vice president Medical Affairs, Hematology/Oncology, at Celgene EMEA, discusses past successes and explains why it’s critical that innovation continues in the search for a cure.

What is driving the increase in multiple myeloma in Europe?

“People aged  65 and older are more likely to get multiple myeloma, and there are more older adults in the world than ever before. This is especially true in the Western world, where multiple myeloma is currently most prevalent. These trends mean we will likely see more and more cases of multiple myeloma.

39K new cases of multiple myeloma are diagnosed in Europe each year.“But patients may be able to have a different experience today than they had 20 years ago. Treatment options introduced since then are extending their lives and helping their quality of life.”

What impact does multiple myeloma have in Europe?

“Nearly 39,000 new cases of multiple myeloma are diagnosed in Europe each year. While most patients are elderly, 30 percent are between the age of 15 and 64 at diagnosis. Over 40 percent of multiple myeloma patients cannot work because of their disease, and another 23 percent were forced to retire early. Those are direct impacts on productivity. But the disease also has an indirect impact on productivity as elderly patients rely on family and friends for care, forcing those caregivers to miss days of work. We have had a real opportunity to reduce this burden with new and effective treatments over the past couple decades.”

How have treatment advances changed patient lives?

“Patients are living longer lives because of advances in the treatment of multiple myeloma. The five-year relative survival rates for multiple myeloma has increased more than 60 percent since 1990, which is a bigger improvement than we have seen for all cancers on average.”

Treatment advances have led to longer remissions for patients, suggesting a cure may one day be possible.

1 out 3 people who are diagnosed with multiple myeloma are working age.What is driving the rapid progress in multiple myeloma treatment?

“Innovation, without a doubt. And that cannot stop now. Even with the innovations over the past few decades, we have more to do. For example, the five-year survival rate (based on 2007-2013 data) for people with breast cancer is 90 percent; for multiple myeloma, it’s 50 percent. We cannot yet guarantee a cure for a single multiple myeloma patient, so medical innovation is a crucial, ongoing task.”

What new treatment approaches are being explored?

“At least 50 new treatment options for multiple myeloma are being studied in clinical trials, which is significantly more than there were a decade ago. We know the immune system plays a role in this blood cancer, and we have seen the success of certain therapies that act on the immune system and have built on it. Researchers are also looking at harnessing the power of the immune system through novel investigational immunotherapies, including CAR T-cells and bispecific antibodies.

“Additionally, combining different treatments allows for the targeting of different cancer processes simultaneously, and there are ongoing studies investigating various combinations of therapies. Finally, researchers are studying the benefits of different types of stem cell transplants.”

17% — The increase of worldwide incidence of multiple myeloma projected by 2025.How hopeful are you that there will one day be a cure?

“I’m very hopeful for a cure. Treatment advances have led to longer remissions for patients, suggesting that a cure may one day be possible. Multiple myeloma cells are not all the same, so treatment combinations may be needed to make sure we eliminate all the cancer cells and do not give the disease a chance to come back. We are hopeful that, as research continues to advance our understanding of disease and the development of new approaches to treatment, the future may bring multiple myeloma patients a cure or may allow them to live very close to their natural lifespan.”

To learn more about how treatment advances are giving patients a reason to be hopeful, read “A Decade of Progress in Myeloma, and More To Come.”

 

RAFAEL FONSECA, MD, MYELOMA EXPERT AND CHAIR OF THE DEPARTMENT OF MEDICINE AT THE MAYO CLINIC IN ARIZONA, AND COLLEAGUES DISCOVERED THAT COST DRIVERS IN THE CARE OF MULTIPLE MYELOMA INCLUDE HOSPITALIZATIONS AND OUTPATIENT SERVICES, NOT INNOVATIVE THERAPIES ALONE.

RAFAEL FONSECA, MD, MYELOMA EXPERT AND CHAIR OF THE DEPARTMENT OF MEDICINE AT THE MAYO CLINIC IN ARIZONA, AND COLLEAGUES DISCOVERED THAT COST DRIVERS IN THE CARE OF MULTIPLE MYELOMA INCLUDE HOSPITALIZATIONS AND OUTPATIENT SERVICES, NOT INNOVATIVE THERAPIES ALONE.

Over the past decade, treatment advances in multiple myeloma are helping patients live longer, better lives. But many doctors are concerned that those advances have made treatment unaffordable for some patients. Leading up to this year’s American Society of Clinical Oncology (ASCO) meeting, oncologists are learning that might not be the case.

Since 2000, outpatient services and inpatient admissions have driven more of the total cost increases for multiple myeloma treatment than new therapies, according to a study published earlier this year in Leukemia.

“That was a bit of a surprise to us,” said Rafael Fonseca, MD, a myeloma expert and chair of the Department of Medicine at the Mayo Clinic in Arizona and one of the study authors. “We just didn’t know exactly how much we were spending on the various components of multiple myeloma care.”

One of the primary drivers of the cost increases for multiple myeloma care is outpatient services, which increased by $5,200 per patient per month from 2000 to 2014. This rise was fueled by an increased use of imaging technologies, diagnostics and other tools needed for the care of myeloma patients.

Meanwhile, hospitalizations and prescription medication costs increased by $4,100 and $3,833, respectively, due to increased utilization of stem cell transplants and other new therapies.

Fonseca and his co-authors dug deep into “real world” data from the Truven Health MarketScan Research Databases and Medicare Supplemental Databases. Those databases provide information about what is paid for health services and medications, including rebates and discounts that insurers and pharmacy benefit managers receive.

In contrast, conversations about treatment-related costs often focus on list prices rather than actual prices negotiated by insurers, according to Fonseca.

Multiple Myeloma Treatment Cost Increases Per Patient Per Month

Of course, the study’s data had some limitations. For instance, it only included people with commercial health coverage or private Medicare supplemental coverage, so the results may not apply to people with other or no health insurance. Also, the identification of multiple myeloma patients relied on diagnosis codes, which may not always be accurate or complete.

“Despite those limitations, it’s fairly representative of reality,” Fonseca said. “Without an objective analysis, the community risks perpetuating the narrative that patients can’t afford treatment. I would argue that’s not true in most cases.”

At the Mayo Clinic, Fonseca treats patients from all walks of life and some on fixed incomes. Most of his patients ultimately get the care they need because their health insurance or Medicare help with the costs and foundations like the Leukemia and Lymphoma Society provide additional copay support. Even though some patients needed help with their efforts to secure financial assistance, Fonseca does not remember a single instance in his practice where a multiple myeloma patient couldn’t access their treatment because of cost.

That is not to say it never happens, but at least not for his practice. “Commercially insured patients get a fixed maximum copay, Medicare patients receive support from foundations, and we can obtain free medications for uninsured patients,” Fonseca said.

Without an objective analysis, the community risks perpetuating the narrative that patients can’t afford treatment. I would argue that’s not true in most cases.

The false narrative that cancer therapies are unaffordable to patients can have very negative consequences. Fonseca knows the stories; one oncologist in Arizona feared treatment might be a financial burden for one newly diagnosed myeloma patient and his family, so he sent that patient to hospice instead.

“That’s just mind-boggling,” said Fonseca. “At the Mayo Clinic, we have always found a way to get affordable treatment for our patients. Narratives, true or not, can indeed have real-world consequences.”

That narrative has also given credence to the increased use of strategies like prior authorization and specialty tiers that insurers and pharmacy benefit managers use to reduce costs by restricting or discouraging the use of medications.

But many of these strategies, which are not guided by clinical evidence, affect patient care, according to a recent ASCO policy statement. ASCO believes that these strategies should follow evidence-based clinical pathways.

“It’s unethical to ask sick patients to pay more when they are faced with expensive medical costs,” Fonseca said. “The consumerism trend in health care is exerting pressure to prevent utilization. I am pleased that ASCO decided to speak out against these practices. They recognize these innovative therapies for multiple myeloma are worth the cost.”

To learn more about how new treatments are giving multiple myeloma patients hope, read “A Decade of Progress in Myeloma, And More to Come.”

When Michael Tuohy was diagnosed with multiple myeloma in 2000, the only thing he could think about was living long enough to walk his then 7-year-old daughter Ally down the aisle and seeing his 2-year-old son Mikey graduate from college. At the time, multiple myeloma patients had just a 35 percent chance of living five years. But 17 years later, Tuohy is about to see his wishes come true; his daughter is planning her wedding for next year, and his son is now a freshman in college majoring in biomolecular science.

Since his diagnosis, Tuohy and his wife, Robin, have seen a lot of positive advances in multiple myeloma treatment and support. During this year’s Multiple Myeloma Action Month, they are giving back to the community by sharing what they’ve learned.

How did you become involved with the multiple myeloma community?

Michael: After my diagnosis, we searched out the best resources and found the International Myeloma Foundation (IMF) website. Geraldine Ferraro had just announced she had multiple myeloma. The IMF was asking patients to write letters to Congress about battling multiple myeloma and the importance of funding research. Susie Novis Durie, the founder of the IMF, saw our letter and asked us to share our story in Washington that year. We both went, and it was the first time we met any other multiple myeloma patients. I had gone about six months without meeting another patient. We became empowered through the IMF and decided it was time to take action to help others.

MULTIPLE MYELOMA SURVIVOR MICHAEL TOUHY (SECOND FROM THE LEFT) AND HIS WIFE ROBIN (FAR RIGHT) FEEL FORTUNATE THAT HE HAS LIVED LONG ENOUGH TO SEE HIS DAUGHTER ALLY (RIGHT) GRADUATE FROM COLLEGE. SINCE THEN, SHE HAS STARTED HER CAREER AS AN ELEMENTARY SCHOOL TEACHER, AND HIS SON MIKEY (FAR LEFT) HAS BEGUN HIS COLLEGE STUDIES AS A BIOMOLECULAR SCIENCE MAJOR.

MULTIPLE MYELOMA SURVIVOR MICHAEL TOUHY (SECOND FROM THE LEFT) AND HIS WIFE ROBIN (FAR RIGHT) FEEL FORTUNATE THAT HE HAS LIVED LONG ENOUGH TO SEE HIS DAUGHTER ALLY (RIGHT) GRADUATE FROM COLLEGE. SINCE THEN, SHE HAS STARTED HER CAREER AS AN ELEMENTARY SCHOOL TEACHER, AND HIS SON MIKEY (FAR LEFT) HAS BEGUN HIS COLLEGE STUDIES AS A BIOMOLECULAR SCIENCE MAJOR.

Why did you start a multiple myeloma support group?

Robin: Connecticut did not have a multiple myeloma support group, and Susie suggested that we start one. But we weren’t the kind of people who sit around and whine. So we decided our group — the Connecticut Multiple Myeloma Fighters’ Information Group — would focus on empowering multiple myeloma patients and caregivers to have better-informed conversations with their doctors and to learn about treatment, side effects, clinical trials and the importance of seeking out the opinion of a myeloma expert.

In 2005, the IMF hired me to help with its growing Support Group Program. Today, there is a network of over 150 myeloma specific groups in the United States and over 300 worldwide. The IMF has numerous programs specifically geared to ensure support and information are available at the local level. The IMF applauds the efforts and commitment of these groups and conducts an annual summit to offer help, training, and encouragement to support group leaders.

How does your support group help new multiple myeloma patients?

Michael: The first thing we tell people is to see a multiple myeloma specialist. Their local oncologists are treating many different cancers. Form a team with your local oncologist to work with a myeloma specialist. Myeloma is 1 percent of all cancers and a very individual disease; what works for one may not work for another. You still need a local oncologist, someone who can do your blood testing and know your history in case you need hospitalization close to home. But you want a multiple myeloma specialist as well. You want someone who is conducting clinical trials and knows the latest treatments and research. It should be a collaboration between your multiple myeloma specialist, local oncologist and other experts. You have a lot of people working for you that you need to coordinate — you’re the captain of the ship.

When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them.

How does your attitude make a difference in living with multiple myeloma?

Robin: We’ve never lived our lives waiting for the other shoe to drop. We’ve remained positive. You have to be confident in your doctor and the treatment that you’ve decided on together. Knowledge is power. Patients should learn as much as they can so they can have conversations with their doctors and remain engaged.

How else are you giving back to the community?

Robin: Our whole family has gotten involved in advocating for increased funding for multiple myeloma research, Food and Drug Administration approval of new therapies and oral parity laws. Those laws push for equal insurance reimbursement for anti-cancer treatments regardless of the delivery method. Michael was on an oral therapy at one point, so we know the benefits; it allowed him to go home, take a pill and get on with his normal routine.

A silver lining to the multiple myeloma journey is seeing and appreciating our children become advocates and doing good things in the world. Our son Mikey is now in his first year of college, studying biomolecular sciences. Our daughter is an elementary teacher.

SINCE MICHAEL TOUHY’S MULTIPLE MYELOMA DIAGNOSIS, HE AND HIS WIFE ROBIN HAVE ATTENDED MEDICAL MEETINGS TO LEARN ABOUT THE LATEST TREATMENTS AND SHARE THAT INFORMATION WITH OTHER PATIENTS.

SINCE MICHAEL TOUHY’S MULTIPLE MYELOMA DIAGNOSIS, HE AND HIS WIFE ROBIN HAVE ATTENDED MEDICAL MEETINGS TO LEARN ABOUT THE LATEST TREATMENTS AND SHARE THAT INFORMATION WITH OTHER PATIENTS.

What has changed in the 17 years since Michael’s diagnosis?

Robin: When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them. I also stress to people to stay fit and get all their check-ups. You have to keep your body in good shape. As long as you can stay healthy and take care of yourself outside of your multiple myeloma, those treatment options are your future. Don’t be afraid of your options.

Michael: We’ve been going to the American Society of Hematology annual meeting for a few years now. Lately, it’s been standing room only. Thousands of participants are attending the presentations to hear about the latest developments. It blows my mind and gives me hope for the future of multiple myeloma.

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Robert Lasco knows first-hand the impact that multiple myeloma can have. So he’s turning his personal experience with the disease into something positive for fellow African-American myeloma patients who have lacked awareness of proper treatment. Since being diagnosed with myeloma three years ago, Robert started the Lasco Cancer Fund and is supporting Celgene’s Standing in the Gaap initiative to help raise awareness about myeloma within the African American community during this year’s Multiple Myeloma Awareness Month.

How did you discover that you had multiple myeloma?

In 2012, I was seeing a chiropractor because I was experiencing back pain, and my feet went numb during one adjustment. After that, I visited my general practitioner who ordered x-rays but couldn’t find anything. In February 2013, I was in so much pain that my wife had to call the EMTs. They rushed me to the hospital. Around midnight, the doctors told me they had to perform an emergency surgery to remove a tumor on my T8 and T9 vertebrae. It wasn’t until five days later that I was officially diagnosed with myeloma.

What has happened since you were diagnosed?

That surgery left me paralyzed from the waist down; I left the hospital in a wheelchair about six weeks later. Through rehab I eventually began to walk again.

After the surgery, I went through about 12 bouts of radiation therapy before going into remission seven months later. I received a stem cell transplant and was in remission until 2016. Now I’m receiving treatment to continue to help me fight the disease.

Why are you raising awareness of myeloma within the African American community?

Anytime I ask someone about myeloma, they have no idea what I’m talking about. We need to educate ourselves, especially because African Americans are twice as likely to be diagnosed with myeloma compared with other races. We need to learn about all our treatment options. We can’t just fold because we don’t like the hand that we’ve been dealt. We have to play it. We have to be proactive and take control.

Who else are you trying to reach with education?

We’re getting in front of specialists, oncologists and general practitioners to help educate them about myeloma. We see that as a big key to improving the lives of African Americans with myeloma. It isn’t like the other big cancers; it affects a small number, but those numbers are growing. Doctors need to have a better understanding of the disease.

What interests you about the Standing in the Gaap initiative?

The initiative is completely aligned with my personal mission to spread the word about myeloma within the African American community. Standing in the Gaap provides information that is easy to understand and the best that I’ve ever seen on this topic. Right now, we’re making great strides with the initiative. My organization participated and provided information about myeloma at a basketball tournament last year, and the Standing in the Gaap initiative participated in the same event. We’re putting good information in the hands of people who need it most.

When you meet someone who has just been diagnosed with multiple myeloma, what do you say to them?

Live. That’s my message. When they first hear the word “cancer,” all they can think about is death. I encourage them to live and to hope. Have faith in God and believe that you are going to live and not die. I told myself that, especially when I thought that I was going to be bound to a wheelchair for the rest of my life. You have to live.

To learn more about the Standing in the Gaap initiative, download the Standing in the Gaap brochure or visit the initiative’s Facebook page.

In 2005, just three years after retiring from his professional career as an NFL player, Elijah Alexander was diagnosed with multiple myeloma at 35 years old. Up until his death five years later, he not only battled the blood cancer but also worked hard to raise awareness of the disease.

For his wife, Kimberly, Elijah’s passing was just the beginning of her work. Inspired by her husband’s advocacy, she vowed to stay connected with the cancer community. And during this year’s Multiple Myeloma Awareness Month, she is helping to educate the black community about the facts, including risks and treatment options, as an ambassador for Celgene’s Standing in the Gaap initiative.

“I was so very proud of my husband’s work, raising awareness and funds for patients who needed help, as well as sharing his experience with cancer,” Alexander said. “So I’m keeping his legacy going. Not many people know about myeloma and the increased risk among the African American community.”

Kimberly recently joined Liesel Wabnig, a Celgene registered nurse to talk about how multiple myeloma affects African Americans differently via Facebook Live.

KIMBERLY ALEXANDER HAS CONTINUED TO BE ACTIVE IN THE MULTIPLE MYELOMA COMMUNITY BEYOND HER HUSBAND’S PASSING. SHE CHAIRED THE 2016 NORTH TEXAS LEUKEMIA & LYMPHOMA SOCIETY MAN OF THE YEAR EVENT WHERE DR. BRIAN BERRYMAN, ELIJAH’S ONCOLOGIST, WON THE TOP HONOR.

In fact, African Americans are twice as likely to be diagnosed with this deadly blood cancer compared to white Americans. However, because African Americans are often diagnosed with a less aggressive form of myeloma, they can do as well as, if not better than, white Americans when they get timely diagnoses and treatment.

Unfortunately, African Americans often face barriers to early diagnosis and proper treatments.

“Elijah’s diagnosis came as a real surprise to us because he was young and seemed healthy,” Alexander said. “Cancer can affect anyone at any time in their life, so everyone needs to be aware.”

While myeloma is usually associated with older adults, African Americans are diagnosed at a younger age on average (66 years) than are white Americans (71 years).

Getting an annual physical and knowing the most common symptoms—which include bone pain, fatigue and poor kidney function—can increase the chances of catching myeloma early.

Before his diagnosis, Elijah didn’t notice many symptoms other than some pain in his feet. Although treatments helped keep Elijah alive for five years, Alexander believes that he may have survived longer if they had known more about myeloma before he was diagnosed.

“We only discovered his disease through a blood test,’” Alexander said. “It’s important to have an annual physical and blood screening, which can help detect myeloma earlier.”

The more African Americans participate in clinical trials, the better and faster we can understand why myeloma impacts African Americans differently.

Many mysteries still surround the disease. For example, scientists don’t know why African Americans are at a higher risk for myeloma. One thing that can accelerate scientists’ understanding is increased enrollment in clinical trials, of which Alexander is now working to raise awareness.

“Today, clinical trials are the best way to access the most innovative treatments,” Alexander said. “The more African Americans participate in clinical trials, the better and faster we can understand why myeloma impacts African Americans differently.”

While the future of myeloma treatment continues to improve with new approaches such as immunotherapy, a cure has remained elusive. To do her part, Alexander plans to keep supporting the myeloma community any way she can, until the disease becomes a footnote in history.

“Helping those affected by myeloma has truly become a passion in my life,” Alexander said. “I will never forget what the disease did to my husband, and that’s why I will continue to be involved in the community.”

To learn more about multiple myeloma and the African American community, visit the Standing in the Gaap Facebook page and Myeloma Central.

As a modern class of therapeutics, antibodies continue to change the treatment landscape for cancer. Antibodies are naturally produced Y-shaped proteins whose “arms” grab hold of bacteria, viruses and other possibly dangerous cells while the “leg” recruits immune cells to destroy the invaders. Researchers have leveraged this ability of antibodies into a treatment approach called immunotherapy, in which antibodies, or pieces of antibodies, are designed in the lab to better recognize cancer cells as invaders and call in the immune system to kill them.

Immunotherapy has proven successful, with many drugs approved over the past decade. Now researchers are trying to improve on that success by splicing together portions of two antibodies with distinct targets to create what’s known as a bispecific antibody. The hope is that two targets will be better than one.

Bispecific antibodies may work two ways: either by blocking two pathways cancer cells use to thrive or by blocking one cancer pathway and simultaneously recruiting immune cells called T cells to tumors.

Bispecific Antibodies: Redirecting Immune Cells to Fight Myeloma

The concept of bispecific antibodies isn’t new (the idea has been kicked around since the 1970s), but interest has been renewed as the techniques to produce large quantities of these proteins have improved substantially over the past decade.

As a result, researchers are now able to explore their potential to combat many different cancers. In fact, the U.S. Food and Drug Administration has already approved two bispecific antibodies, and more than 30 others are being tested in clinical trials.

One cancer being tested is multiple myeloma, a disease that affects blood cells. Although researchers have made significant progress in the treatment of myeloma over the past decade, most patients will eventually relapse, meaning new therapies are in demand.

CAR-T cells and bispecific antibodies as single agents may offer new hope to patients with myeloma, but they also may provide the opportunity for new combinations with other novel targeted therapies under investigation.

Myeloma cells display a promising target for immunotherapy called B-cell maturation antigen (BCMA), which is found in 60 to 70 percent of multiple myeloma patients. So a new potentially successful bispecific antibody therapy might, for example, have one arm that binds to myeloma cells via BCMA and another that binds to T cells, one of the immune system’s top cancer assassins. While typical antibodies that bind to just one target also recruit immune cells, they generally do not specifically bring in T cells. A bispecific antibody with this ability is expected to have greater cancer-killing effects than conventional antibodies.

BCMA is also used in another immunotherapy approach for myeloma called chimeric antigen receptor T cells (CAR-T) therapy. In this strategy, a patient’s T cells are extracted, engineered in the lab to recognize and bind to BCMA, and then placed back into the patient. The goal is for CAR-T cells to home in on and destroy myeloma cells.

CAR-T cells and bispecific antibodies as single agents may offer new hope to patients with myeloma, but they also may provide the opportunity for new combinations with other novel targeted therapies under investigation. Ultimately, the aim with both tactics, alone or in combination, is to cure myeloma, a goal that seems more and more possible with every scientific advance.

To learn more about how immunotherapy is changing how we treat blood cancers such as myeloma, read “Immunotherapy May Be the Future of Myeloma Treatment.”

 

When recruiting patients for a clinical trial, it is important that researchers select the right mixture of people—in terms of age, gender and ethnicity—who they’re seeking to treat with a new therapy or treatment intervention. This variety is critical for researchers to understand how treatments impact patients differently. Unfortunately, achieving diversity in clinical trials continues to be a challenge.

While African Americans make up over 20 percent of newly diagnosed multiple myeloma cases in the United States, they represent only 8 percent of cancer clinical trial participants. When it comes to multiple myeloma, a cancer of a type of white blood cells called plasma cells, African Americans are twice as likely to be diagnosed with the disease, which makes it even more important that they are better represented in myeloma clinical trials.

Meanwhile, African Americans are more likely than any other racial group to be diagnosed with and die from cancer. African Americans have also benefited less from advances in myeloma treatment over the past two decades than whites, suggesting unequal access to or response to treatments.

African Americans & Myeloma Clinical Research

“Without a doubt, African Americans are underrepresented in myeloma trials,” Brendan Weiss, an assistant professor of medicine at the University of Pennsylvania who specializes in myeloma, said. “Now that new tools like next-generation DNA sequencing can shed some light on the genetic diversity in myeloma, studies should absolutely do the best they can to try to get diverse representation.”

Understanding the genetic differences between African American patients and other patients with myeloma could go a long way in explaining the differences among different patient groups in how the disease progresses and responds to treatment. As a result, doctors may get more insight into how to treat the disease most effectively for each group.

Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University, explains why minorities have a mistrust of the American medical system.

Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University, explains why minorities have a mistrust of the American medical system.

But recruiting African Americans for trials has been difficult. In one 2013 survey, African Americans were more likely than any other racial group to cite lack of trust as a reason not to participate in a clinical trial and to believe that patients are sometimes enrolled in trials without their consent.

“African Americans have a historical mistrust of the medical system,” said Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University who studies disparities in clinical trials. “A lot of that mistrust comes from people feeling like they’re not treated equally in the health care system.”

To boost enrollment of African Americans in clinical trials, including those of myeloma therapies, clinicians have to build an environment of trust. Clinics must be proactive in communicating the value of clinical trials consistently—with pamphlets in the waiting room, for example, not just when recruiting patients—so people can begin to learn why participation is so vital to improving the understanding of disease and treatments for everyone.

The message we want clinicians to get across to African American patients is: you are needed, you are important, and we need to know how these medications impact people like you.

For instance, one study found that providing patient navigators to help guide patients and answer their questions about the process raised the enrollment rate of eligible African American cancer patients to about 80 percent. Another group found that African Americans who participated in a clinical trials education program through their local church were almost three times as likely to enroll in a clinical trials registry.

“The message we want clinicians to get across to patients is: you are needed, you are important, and we need to know how these medications impact people like you so that we can help other people like you,” said Breland-Noble.

Diversity among researchers, clinicians and clinic staff can also help patients of various backgrounds feel more comfortable throughout their care. It’s a sentiment that Celgene’s Standing in the Gaap initiative is already embracing, with efforts to boost the numbers of students pursuing cancer research and medicine at historically black colleges and universities.

By making the field of myeloma care and research more diverse, the campaign aims to make African American patients feel more included and more likely to participate in clinical trials. It’s only then that researchers can study the full links between genetics and myeloma.

Discover more information about how myeloma affects African Americans on the Standing in the Gaap Facebook page.

After 56 hours of running, New York City businessman and ultramarathoner Eric Gelber realized he was barely moving. It was his second attempt to run 200 miles around Central Park to raise awareness and funds for research towards a myeloma cure, and the last 2.5 miles took four hours to complete. At that pace, he’d be running for another 24 hours.

After 176 miles in two and a half days, he was done. Although the effort beat the 164 miles he ran during his first attempt the previous year and raised $240,000 for myeloma research, Gelber felt defeated. “I feel like I failed,” Gelber admitted. “But I know that no one else feels that way.”

So to commemorate this year’s Blood Cancer Awareness Month, Gelber is making one last attempt to run 200 miles around Central Park and raise an additional $250,000 for myeloma research during September 16-18, 2016. Hitting that goal will put him past $1 million in total money raised on behalf of the Multiple Myeloma Research Foundation (MMRF), an organization whose sole mission is finding a myeloma cure, since his efforts began nearly a decade ago.

In 2007, Gelber’s long-time family friend Anita Sorrell received a stem cell transplant as part of her treatment for multiple myeloma, a rare cancer of plasma cells that help our bodies fight infection. The disease can lead to low blood counts, bone and kidney damage and infections. While advances in our understanding of the disease have lead to progress in treating myeloma over the past decade, a cure has remained elusive. “Although the disease affected her physically, it never dampened her spirit,” Gelber recalled.

Gelber wanted to support his friend during her journey with myeloma. He signed up for the New York City Marathon that year to raise money for the MMRF. He raised over $6,000 in that first effort.

She told me that when she wore the medal, she felt the meds going straight into her veins. That motivated me to do more.

After the marathon, he gave Sorrell his medal. She carried it around everywhere in her purse and wore it during her chemotherapy treatments. “She told me that when she wore the medal, she felt the meds going straight into her veins. That motivated me to do more.”

He realized that by running ultramarathons—races longer than the traditional marathon length of 26.2 miles—more people donated, and previous donors gave more. A 2011 155-mile solo run in the Catskill Mountains in New York raised $35,000. A year later, he competed in the Badwater Ultramarathon, a 135-mile race from Death Valley to Mount Whitney in California, and raised $65,000.

With every mile, Eric has taken strides to raise greater awareness and funds in the hopes of finding a cure for myeloma. By fundraising that drives research through the MMRF, Eric feels he is doing all he can to support patients and find the quickest way to a cure.

In 2012, Sorrell lost her battle with myeloma. Devastated by the loss, Gelber’s resolve to help find a cure was strengthened. He decided to bring his efforts to New York City where more people could see and participate. That’s when the idea for “The Journey Towards a Cure,” 200-mile ultramarathon, was born.

Myeloma patient Pam gives Eric Gelber a hug during his 2014 attempt to run 200 miles around Central Park. She flew from the Midwest to New York to support the event that raised funds towards a myeloma cure. Source: Multiple Myeloma Research Foundation

MYELOMA PATIENT PAM GIVES ERIC GELBER A HUG DURING HIS 2014 ATTEMPT TO RUN 200 MILES AROUND CENTRAL PARK. SHE FLEW FROM THE MIDWEST TO NEW YORK TO SUPPORT THE EVENT THAT RAISED FUNDS TOWARDS A MYELOMA CURE. SOURCE: MULTIPLE MYELOMA RESEARCH FOUNDATION

While exhausting, the first two attempts have been very rewarding for Gelber beyond the funds and awareness raised. During the runs, he has met people living with myeloma who come out just to shake his hand, thank him or run a lap with him. They tell him that his endeavors give them hope.

Gelber is looking forward to seeing their familiar faces and new ones during his third and final attempt this month. He knows that it may be his most challenging yet. It will be his first ultramarathon after undergoing hip surgery last December.

“People ask me about the parallels between what I do and those fighting cancer. That’s one I struggle with because I have a choice. They don’t,” he said. “People have good and bad days. You have to get up and fight.”

To find out how you can support Gelber’s final attempt by signing up to run a mile with him, cheering him on or donating to the cause, visit the Multiple Myeloma Research Foundation’s website.

Myeloma doctors, patients and advocates are speaking out about a new report being developed by the Institute for Clinical and Economic Review (ICER) to determine whether innovative myeloma therapies are worth the money. The institute claims the report will help insurers make more cost-driven reimbursement decisions, but critics say that it will only limit treatment options and shorten lives.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

The American Society of Hematology, the Cancer Support Community and the Multiple Myeloma Research Foundation (MMRF) are among the many organizations in the community that have voiced their concerns about the ICER report since the initial draft was released in April and at a public meeting held to discuss the report last month. A subsequent version was issued in May, and the final report is expected to be published in June.

Rafael Fonseca, M.D., Chair, Department of Medicine at Mayo Clinic in Arizona has written a formal critique of the report. “The important question here is, ‘Value for whom?’ It’s certainly not the patient,” he said. “The institute has framed the report around the notion of how insurance companies can get the best value for treatments, so the intent here is to ultimately limit treatment options.”

The institute claims its report will incorporate perspectives from all health care stakeholders, but whether they will follow through remains to be seen. The MMRF, for example, expressed “serious concerns” with the institute’s efforts back in March—about a month prior to the report’s release, in response to a direct inquiry from ICER for feedback. The draft version of the report did not address these concerns, and the MMRF has gone out of its way to clarify its limited involvement.

While ICER revised its report to clarify some stakeholder input and data sources, many concerns have largely been disregarded. The institute claims that patients, doctors and payers must make decisions today based on the evidence at hand. But for doctors, that evidence includes not just published data but also ongoing trials and their own clinical experience.

ICER & the True Value of Innovative Myeloma Therapies

“Overall, the report in its current state includes minimal input from stakeholders within the myeloma medical or patient communities,” Fonseca said. “By no means does this report accurately reflect current clinical practices in myeloma today.”

Over the past decade, new therapies for myeloma have helped patients live longer, but doctors are still trying to figure out how to best treat patients with all the options now available, including testing new combinations.

“Anyone would be really challenged to assess the value of myeloma individual therapies at this point in time,” Fonseca said. “We are still learning about these treatments on the medical side, so pretending that we could begin factoring in economics into the equation is pure nonsense.”

We are still learning about these treatments on the medical side, so pretending that we could begin factoring in economics into the equation is pure nonsense.

Brian Durie, a multiple myeloma specialist at the Cedars-Sinai Medical Center and chairman of the board of the International Myeloma Foundation, believes that myeloma medical experts—not ICER—should be providing treatment recommendations. He notes that the International Myeloma Working Group, which consists of over 200 experts, will meet this June to develop guidelines that will allow for an individualized treatment approach, based on a patient’s unique characteristics and preferences, that evolves as the disease progresses.

Adaptation and adjustment are essential in myeloma treatment today. It’s a complex disease with multiple genetic factors that make each patient’s case unique, and it requires an individualized, malleable approach. Attempting to generalize the effectiveness—let alone cost-effectiveness—of a therapy across the board for all myeloma patients doesn’t account for the realities of the disease.

The ICER assessment also fails to capture the total cost of care and the economic benefits of these medicines. New therapies can reduce costs for doctor’s visits and hospitalizations and allow patients and caregivers to be more productive and to contribute to society. Last year, a study found that providing the most appropriate myeloma treatment option delayed relapses and the accompanying spikes in costs, making those therapies cost-effective in the long run.

“Total cost of care is almost never looked at,” said Fonseca. “We need to start defining value in other ways than just costs. We need to focus on the value of myeloma therapies to individual patients.”

Learn more about our take on the method used by ICER to assess the value of innovative treatments in our related story.

As Multiple Myeloma Awareness Month comes around again, the myeloma community has good reason to celebrate the past decade, which has brought new treatment options for today and a better understanding of how to treat this disease in the future.

A patient who is diagnosed with myeloma has an about 47 percent chance of living five years or longer, which is up from 31 percent twelve years earlier. Meanwhile, although people are being diagnosed with myeloma at an increasing rate each year, their survival chances are improving.

“The progress has been quite dramatic,” Dr. Paul G. Richardson of the Dana-Farber Cancer Institute Hematology Oncology department said. “Although we may have seen similar improvements in other cancers, the difference with myeloma has really been the volume of new treatment options that have been approved in the past decade and their impact on outcomes as reflected by the improvements we have seen in survival.”

Between 2006 and 2015, the U.S. Food and Drug Administration approved 13 new therapies for the treatment of myeloma, which is especially remarkable considering myeloma accounts for less than 2 percent of new cancer cases in the United States each year. During the same period, the FDA approved 20 treatments for lung cancer, which accounts for 13 percent of all new cancer cases, and 12 for breast cancer, which accounts for 12 percent.

Multiple Myeloma: A Decade of Progress

These new myeloma therapies have already helped patients live longer lives, and the best may be yet to come as doctors explore how to use them most effectively. According to data released during the 2015 Annual Meeting of the American Society of Clinical Oncology (ASCO), researchers believe that by 2022 at least half of myeloma patients will live 6 years after being diagnosed, which is 140 percent longer than was expected in 2001.

Over the past decade, researchers have improved our understanding of the immune system’s role in myeloma, which may transform the way we treat this cancer in the future. For instance, one study of 74 myeloma patients who live for 10 years or longer found that their immune systems may be better equipped to recognize and attack harmful entities—such as cancer cells—than others.

While immunotherapies are certainly exciting because of their new approaches, we should not underestimate the therapies approved over the past decade.

Over the next decade, immunotherapies, which leverage our body’s immune system to bring cancer under control, may help further extend the lives of myeloma patients. These approaches include antibody-based therapies, chimeric antigen receptor (CAR) T-cells and checkpoint inhibitors.

“While immunotherapies are certainly exciting because of their new approaches, we should not underestimate the therapies approved over the past decade,” Richardson said. “There’s going to be great value in combining the next generation of myeloma therapies with those we have today.”

To learn more about how myeloma treatments are allowing patients to continue living active lifestyles, read our “Patients Not Letting Myeloma Hold Them Back” story.