Roughly 1 in 25 Americans are living with a serious mental illnesses, such as bipolar disorder or schizophrenia, and many of them are also struggling to manage tardive dyskinesia (TD), a movement disorder associated with the long-term use of antipsychotic medications. These involuntary movements, including grimacing, lip smacking, and others involving the mouth and face, carry significant social stigma, can lead to further social isolation for people living with serious mental illness, and exacerbate symptoms of depression and negative self-perception. Just this year, the Food and Drug Administration (FDA) approved the first-ever treatments for TD.
On Dec. 5, 2017, the Institute for Clinical and Economic Review (ICER) presented a draft assessment of value for these new treatments and plans to issue a final report later in the month. In their comments to ICER, mental health advocates have raised significant concerns about the approach ICER has used in evaluating the value of TD treatments.
To learn more about these concerns, NPC President Dan Leonard caught up with Andrew Sperling, Director of Legislative Advocacy for the National Alliance on Mental Illness (NAMI). NAMI is the nation’s largest grassroots mental health organization dedicated to building better lives for the millions of Americans affected by mental illness. Since its start in 1979 by a small group of families gathered around a kitchen table, NAMI has grown into the nation’s leading voice on mental health with hundreds of local affiliates, state organizations and volunteers dedicated to raising awareness and providing support and education.
Dan Leonard (DL): First, tell us about tardive dyskinesia and the significance of these first treatments for it for patients.
Andrew Sperling (AS): TD is a neurological disorder associated with exposure to the older neuroleptic medications used to treat schizophrenia and other psychotic disorders. Symptoms include facial tics and other involuntary movements of limbs and extremities. There is enormous social stigma associated with TD, and patients experiencing moderate to severe symptoms are typically unable to work retail jobs and other work that involves interaction with the general public. Up until this year, we did not have an FDA-approved treatment for TD. However, in 2017, FDA has approved two new medications: Valbenazine and Deutetrabenazine. These two new treatments have enormous potential be “game-changers” for people living with TD, creating new opportunities for overcoming social isolation and encouraging community integration.
DL: Why did you become engaged with ICER on this review process? How have you engaged with ICER to ensure that mental health advocates will be heard in this process?
AS: NAMI began engaging with ICER with the drafting of the initial “scoping” document on these TD treatments outlining how the review would be conducted. We worked hard to offer the perspectives of people living with TD and their family caregivers as ICER designed and conducted this review. NAMI also participated in the public meeting on Dec. 5 in Boston as part of an afternoon Policy Roundtable to share our concerns.
DL: What are your specific concerns with ICER’s approach in considering factors important to patients and their families in this value assessment? How have your concerns factored into the process so far?
AS: Most concerning in the ICER review is the use of Quality-Adjusted Life Years (QALYs) to measure the value of these two new medications to treat TD. In NAMI’s view, QALYs are a poor measure to assess the value of symptom relief for someone living with moderate to severe TD for many years. While it is the case that significant symptom relief does not extend overall life expectancy, it is enormously important for individual patients. Additionally, QALYs fail to capture outcomes that are of high value to TD patients, such as being able to engage in activities of daily living — shopping, interacting with family and friends, engaging in work — free of an awkward glance from a neighbor or a passerby who does not understand what TD is.
DL: Based on your experience, what suggestions do you have for other stakeholders who want to make sure their views are considered? Are there important milestones for advocates to watch for and provide comment?
AS: Engage as early as possible in the process with ICER, before the review is designed and the questions developed. Where possible, provide them with patients and their caregivers as a part of the review process to ensure that they do not rely solely on providers for the clinical content of their review. Be aware that once the initial review is completed, much of work is done and significant changes are difficult to achieve. In other words, the design of the review, the questions developed upfront and the measures drive the result.
DL: What suggestions would you have for improvement to ensure that patient recommendations are considered by ICER and in other value assessments?
We understand that ICER is developing a new outside stakeholder panel to represent the interests of patients and family caregivers in treatment reviews. This is a step in the right direction. ICER has strict rules regarding conflict of interest for patient advocates. While ensuring that input from patient advocates is free of conflicts, there are concerns that this may have limited the diversity of patient voices that will able to serve on this new panel. So long as patient advocacy groups are completely transparent with the sources of their financial support, they should be allowed to fully participate in the work of groups such as ICER. Likewise, organizations such as ICER that engage in clinical and economic reviews should be fully transparent with the public and policymakers.
DL: Thanks for your time, Andrew.