Robin Tuohy's testimony at CEPAC on Treatment Options for Relapsed and Refractory Myeloma
On May 26, 2016, the International Myeloma Foundation’s Senior Director of Support Groups and spouse of Michael Tuohy, a sixteen-year myeloma survivor, testified at the Comparative Effectiveness Public Advisory Council’s Public Meeting (CEPAC) on Treatment Options for Relapsed and Refractory Myeloma. In her compelling testimony, Robin shared how her husband Michael’s survival as a myeloma patient may not have been possible without access to a clinical trial, an oral therapy regimen, he has been on for the past eleven years. She pointed out, “Today we are able to treat myeloma in sequence and in combination that reflects our individual disease markers, chromosomal abnormalities, and yes, even side-effect management and quality of life choices. I’ll say that again: choices!” She urged the Institute of Clinical and Economic Review (ICER), that while their recent report was “well-intended,” when it comes to patient care, “This is a very slippery slope that should not give anyone leverage to limit or deny access to treatment.”
Thank you first of all for hearing the public testimony today. My husband Michael Tuohy is a sixteen-year myeloma survivor, and that’s makes me part of this process. As Senior Director of Support Groups for the International Myeloma Foundation, I’m in touch with over 140 myeloma support groups across the US. And 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. When my husband Michael was diagnosed with myeloma in 2000, at the very young age of 36, life expectancy ranged between 18 months to 5 years. Our children were two and seven years old. We were both afraid that they may not even remember Michael. 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 combination that reflects our individual disease markers, chromosomal abnormalities, and yes, even side-effect management and quality of life choices. I’ll say that again: choices! Our goal from the moment Michael was diagnosed was to search out the best treatment options available, to connect with the top myeloma experts, and to become empowered with knowledge, and to form a team with our doctors to make critical decisions that could save Michael’s life. Michael has been through many treatment options, including a clinical trial, which has since been FDA-approved. He has been on this oral therapy for eleven years. He has had good quality of life and contributes to society. If guidelines stated that he could only be this drug for a certain time period to save money, would this benefit him or a payer? If he had to follow ICER recommendations back then, he may have been put on an inferior, less expensive first treatment; then need to fail which absolutely would have a negative impact on his quality of life l and his ability to productively contribute to society.
My experience with Michael’s health challenges and listening to the stories of patients and caregivers all over the country has demonstrated the reality that your good intentions with recommendations do not always translate into what third parties will do. This is a very slippery slope that should not give anyone leverage to limit or deny access to treatment. Only physicians and patients have the knowledge and right to decide together, what treatment, when to use it, is the best treatment. In fact, patients will need to access 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 we 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 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. I do understand that myeloma drug costs are unsustainably high, and we need to address this. I believe the IMF’s research arm—the International Myeloma Working Group—will produce superior, patient-centered and research-supported guidelines to effectively impact drug costs at their Annual Summit this June. 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 the IMWG plans to issue formal recommendations within two months. I cannot help to think back to this 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 the approval of four new myeloma drugs. Today I am painfully witnessing the low of a door closing on the hope to have access to these life-saving treatments. In closing, I am happy to be able to tell you that my husband Michael is here with me today. We’ve seen our children grow up and this year we’ll celebrate our son graduating high school and our daughter graduating college. Would Michael have seen these milestones if his treatment decisions were limited? Certainly not. Your decisions will certainly affect lives: for better or for worse. I pray you choose for better.