The drug pricing debate continues to rage on, both at the federal level – look to H.R. 3, seen in the House in 2019 that imported foreign pricing models, which a recent Congressional Budget Office report acknowledges relied on the Quality-Adjusted Life Year to score savings – and in the states, like in my home state of Massachusetts, where our Health Policy Commission has signed a contract with the Institute for Clinical and Economic Review to develop a framework to determine the “value” a drug. What perplexes me about this debate is that though there is messaging about “helping patients,” patients are consulted by policymakers or the press.
I am the mother of four children, three of whom have rare diseases and a full-time patient advocate. Since no one has asked us what we think, I feel compelled to ensure the voice of the patient is heard, since the outcome of this debate impacts us deeply.
Eighteen years ago, when my sons were diagnosed with Duchenne muscular dystrophy, there were no treatments. I was told to take my sons home and love them, because they would not survive until adulthood. My family has been lucky. We have lived through a major shift for Duchenne. We now have treatments, but what we are learning is that access to these new treatments has become the next frontier.
Recently, in their quest to lower drug costs, policymakers and payers keep looking to easy and simplistic formulas to determine the value of a drug. Typically, this comes in the form of cost-effectiveness analyses, which utilize a metric called the QALY. The QALY is a concerning metric, as it places a lower value on treatments that extend the lives of people with disabilities and chronic illnesses, like my sons. Other countries, like Canada and the United Kingdom, rely on this type of assessment, and in these countries, rare disease patients generally do not receive access to novel therapeutics.
Historically, the United States has been against the use of the QALY, and a ban on its use in Medicare was included in the Affordable Care Act. In 2019, the National Council on Disability, an independent federal agency, cautioned against relying on the QALY in any federal program, releasing a report that found that relying on the QALY to make coverage decisions would violate United States disability and civil rights laws.
Despite the discriminatory nature of QALY-based assessments and the ban on its use in Medicare, some nongovernmental groups continue to conduct these assessments and showcase them as a solution for state Medicaid programs to lower their drug prices. The preeminent of these groups is the Institute for Clinical and Economic Review, which is based in Boston and refers to the QALY as the “gold standard.”
I personally engaged with ICER on its review of Duchenne treatments. My son Max was enrolled in a clinical trial for exondys 51, which was approved in 2016 and assessed by ICER immediately upon approval. It isn’t a cure, but we see value in it. Max has less fatigue, and he is able to move around in bed on his own. These are huge quality-of-life improvements for both Max and me as his caregiver.
ICER’s assessments are one-dimensional and fail to capture the quality-of-life outcomes that matter to patients, like my son’s ability to move in bed on his own. Families living with health conditions understand that incremental benefits make a difference. You may not be so concerned when your child stops walking but when they stop being able to feed themselves, roll in bed — those things have grave implications. ICER’s value models fail to capture these critical benefits and reverts to broad categories like “ambulatory or wheelchair bound.”
I am concerned that federal and state policymakers continue to reference ICER as they look to lower the cost of drugs. For example, the recent CBO report on H.R. 3 directly references ICER’s QALY-based assessments and notes that savings are based on “avoidance of coverage.” Let me be clear: This means denial of care to patients like my sons. This is leading us in a direction where rare disease patients will have even greater difficulty accessing needed treatments.
I do not want any other family to be in the situation I was 18 years ago, when my doctor told me there was no hope for my children. These treatments now exist, and we must preserve patient access to them.
Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate; her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013, a nomination for the Global Genes Champion of Hope award in 2014 and the prestigious 2017 Meyer-Whalley Instrument of change award.
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