Thank you for this opportunity to share with you our story and concerns. My purpose today is to help you better understand myeloma, patient and caregiver concerns/stories, and why we are asking you to “walk in our shoes” regarding the report on myeloma treatment options prepared by the Institute for Clinical and Economic Review (ICER). I am not a policymaker, so you will hear that side of the challenge from others at this table today.

My name is Robin Tuohy, and I am a 16-year myeloma caregiver to my husband Michael. I am also Senior Director of Support Groups for the International Myeloma Foundation, assisting more than 140 myeloma support groups across the country representing thousands of patients and caregivers. I can tell you that patients not just in the United States, but around the world are listening, watching and worried. My personal and professional experience in passionately advocating for myeloma patients’ rights is what drives me to ask you to hear and respond to the myeloma community’s concerns.

If you are not familiar with myeloma, here’s a short overview of a very complex disease: Multiple myeloma is the second most common blood cancer. The average life expectancy remains at four years for the third year in a row. However, some people beat the odds and live 10 to 20 years or more. While there are powerful treatments available, all myeloma patients will eventually relapse—which is why it’s so important to have new drugs at hand whenever possible.

Today, we are here to share a bit of our story, and our experience of being part of the public speaking portion of the meeting convened by ICER in St. Louis on May 26th.

In 2000, at the age of 36, my husband, Michael, was diagnosed with multiple myeloma. Our children were only two and seven years old. We were both afraid they might not even remember him.

Michael had a large plasmacytoma which had destroyed two-thirds of his sacrum. Our myeloma expert in New York City immediately had Michael undergo low-dose radiation to shrink the tumor and reduce the pain. The next step was to harvest his stem cells for future use, followed by high-dose radiation to totally eradicate the tumor. Michael continued with monthly treatments and monitoring. Thankfully, his disease was quiet . . . for a while.

In the winter of 2002, Michael started breaking bones just by coughing or picking up our son to play. Myeloma was active again, and Michael prepared for a stem cell transplant by using high-dose steroids alone. Forty milligrams of dexamethasone, four days on, four days off, for five months. It was not easy, but it worked to get the protein low enough to transplant. Again, back then, there were no options; and while dexamethasone came with “the good, the bad and the ugly,” it worked. Today, there are MANY options to prepare for transplant that have much better side-effect management profiles.

The transplant was done in September 2002, and the biggest challenge was that Michael was allergic to every antibiotic under the sun. He had fevers over 105 degrees and was delusional for days, which necessitated him totally going off all antibiotics. Having two young school children and a husband with no immune system and not able to take antibiotics was a real challenge! When the kids would come home from school, I would have them come in, totally change their clothes, take showers, before they could give their dad a “mask” kiss. I took lots of extra precautions. I was thrilled that Michael never got an infection, and the transplant was a success!

Again, in 2002, there were no treatments with overall survival statistics showing benefit of maintenance. So Michael went back to work, enjoyed life, and two-and-a-half years of remission.

During this remission time, it was necessary to repair the damage done to the sacrum from the original tumor and he underwent surgery. Unfortunately, the operation left Michael with irreparable nerve damage and peripheral neuropathy in both his legs and he became totally disabled. He was paralyzed for months and needed to learn how to stand, walk, and perform routine things again as simple as putting on his socks and shoes each morning. Today, Michael is able to walk again, and, although limited, he leads a full life and even coached our son’s baseball team, and currently volunteers to speak all over the country to myeloma patients.

In the summer of 2005, he relapsed again with new lesions. Facing difficult odds, we were very proactive. With the knowledge that we gained through the IMF and the team relationship and trust that we developed with our myeloma specialists, we discussed a plan. Michael’s sensitivity to medications became a major obstacle in fighting myeloma. At the time, Revlimid was in clinical trials and other approved therapies that were available had peripheral neuropathy (PN) issues. This was a real dilemma since Michael already had difficulties with nerve damage and PN from the surgery, and he is allergic to antibiotics, which would make a second stem cell transplant very risky. So Revlimid was not the only choice—but the best choice!

So even back then, you can see the importance of choice in treatment options. With all the options we have today, we can choose what’s best for us based on side effects, existing health challenges/conditions, work schedules, etc. We can have these conversations with our myeloma experts to best decide the course of treatment and adjustments.

Michael started on Revlimid and dexamethasone in November 2005. By March 2006, he had attained complete remission and he continues on Revlimid maintenance today!

During this time, while raising our children and appreciating every moment in life, Michael and I learned all we could through the IMF and became empowered. In the spring of 2001, we went to Washington DC for the blood cancer hearings when Geraldine Ferraro, the first female vice presidential candidate representing a major US political party, announced that she had myeloma. It was the first time we met other myeloma patients. That summer, with the help from the IMF, we started the first myeloma support group in our home state of Connecticut.

In 2005, I was offered a position at the IMF to help their mission to educate patients and caregivers through local support groups. So, as you can see, my life is personally and professionally all about myeloma and helping others to become informed, empowered, and engaged in their treatment decisions.

In the summer of 2007, our family embarked on the “Myeloma Mobile” – an idea Michael had that the IMF made a reality. Our whole family – Michael, me, and our children, Ally and Mikey, and even our dog and cat – traveled across the country in an RV. Our goal was to reach out to the myeloma community with current information and to create awareness. We traveled over 3,000 miles, visited more than 20 cancer centers, support group meetings, backyard picnics, and even started a support group!

As you can see from this short summary, our story for the past 16 years has been a roller coaster of remissions and relapses. But through it all, we worked to help others, and that’s why we are here today—to ensure that each of you know more about myeloma and the dangers to anything that diminishes access to treatment.

In 2000, treatment options were extremely limited and we lived with the heavy burden of trying to “keep something in our back pocket,” “a big gun” for when you really needed it.

Today, we are able to treat myeloma in sequence and in combinations that reflect our individual disease markers, chromosomal abnormalities, and yes, even side effect management and quality of life choices . . .  I’ll say that word again . . . choices. I can’t stress that enough.

You’ve heard a bit of Michael’s story and some of the many treatment options we’ve gone through. Today, he’s been on Revlimid for 11 years, has had a good quality of life, and contributes to society. If any kind of “guidelines” stated that he could only be on this drug for a certain time period to save money, would this be a benefit to him or to the payer? Again, please “wear these shoes” and imagine how you would feel and what you would want to happen with guidelines.

Would additional hospitalizations have actually cost more if he were not on treatment? That’s something the ICER report has not addressed.

We have taken the time to read the Final Report from ICER for myeloma relapse therapies, issued in June 9th. The report seemed to admit that they do NOT have the expertise to adequately assess the complexities of new myeloma therapies. ICER even conceded in a statement at the end of the report that a “Fail First” policy is a mistake for myeloma patients. They have also indicated that they have come to understand that each patient is unique and that all therapies will be required during the course of multiple relapses.

I have to say that while I was relieved to read this. I found the draft report to be ambiguous and was concerned that third party payers may use the vague language to their own benefit. While Michael fights myeloma, I fight insurance companies.

My experience with Michael’s health challenges and listening to the stories of patients and caregivers all over the country has demonstrated that while ICER may have good intentions with recommendations, they do NOT always translate to what insurance companies will do. This is a very slippery slope that should not give insurance companies, or any third parties, leverage to limit or deny access to treatment. Only physicians and patients have the knowledge and right to decide together what treatment is best and when to use it. In fact, patients will need access to our entire armamentarium of drugs over the course of this disease. Since there is currently no cure, many patients have numerous remissions and relapses; and each time they relapse, the disease is harder to fight. Why not go with the best treatment plan first, when a patient’s body is stronger and better able to fight?

I truly fear that while ICER’s report is well intended, the content of its conclusions can quickly be used by policymakers and payers to limit treatment options to patients.

The Final report seems to now defer to the expertise of myeloma doctors who work with myeloma therapies every day and also the experience of patients. I can only hope that payers such as CMS/Medicare will not seek ICER guidance. There is lack of transparency, expertise, and trust.

I understand that myeloma drug costs are unsustainably high, and we need to address this. I believe that the IMF’s research arm, the International Myeloma Working Group (IMWG), will produce superior patient-centered and research-supported guidelines to effectively impact drug costs. The IMWG is an international body of more than 200 well respected myeloma experts, who have already issued myeloma guidelines adopted by doctors around the world. This is an urgent issue and at the IMWG Summit last week in Copenhagen attendees focused on how best to protect patients from all processes that might limit or distort access. Two important decisions were already made:

To proceed immediately to prepare access- or cost-stratified guidelines for use of therapies throughout the course of the disease from Frontline to Refractory/Relapse.

Initiate meetings bringing all stakeholders to the table, including, of course, patients, pharma, insurers, central pharmacies, hospitals, and clinics and regulators, who will ultimately need to change the cost and reimbursement landscape.

Consensus statements and guidelines routinely seek the maximum input and feedback to incorporate ALL opinions and suggestions.

I cannot help but think back to this past December, when I was fortunate to attend the annual meeting of the American Society of Hematology. There, I witnessed the elation of researchers, doctors, and patients rejoicing over the approval of FOUR NEW MYELOMA DRUGS.

When we were in St. Louis speaking at the ICER public session, and had to sit through a very frustrating meeting, we painfully witnessed the low of a door closing on the HOPE to have access to these life saving treatments with ICER’s recommendations.

In closing, I’m happy to be able to tell you that my husband, Michael, is here with me today, has seen our children grow up. This year, we just celebrated our daughter graduating magna cum laude from college and our son graduating with honors from high school. As a matter of fact, he’s going into bio-molecular sciences and perhaps some day, he’ll be part of finding a cure for myeloma.

Would Michael have seen these milestones if his treatment decisions were limited? Perhaps not. In my statement to ICER, I ended with this challenge to them: “Your decisions today will certainly affect lives, for better or for worse. I pray you chose for better.” I think they listened, at least a little, and I hope each of you today will also hear our very real concerns. Life is precious; please do not let anyone limit our options, our hope, our lives.

Thank you.

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