When my son was born with Down syndrome, I learned quickly that the smallest details of medical care can have an extensive effect on the quality of life.
Clinicians who understood Down syndrome, insurance coverage policies that took my son’s unique health profile into account, and even the bedside manner of the health professionals caring for my son had a tremendous effect on our experience with the healthcare system. Today, as a leader of a national advocacy organization, I’m watching a seemingly minor legal provision put new treatments out of reach for children like mine.
Here’s the issue from the perspective of a mother and a healthcare advocate.
The largely well-intended Inflation Reduction Act passed in 2022 imposes price caps on an expanding roster of drugs. However, before these price controls kick in, new medicines get a “grace period” that gives developers time to earn a return on their investments and further incentivize the drug development children need.
The IRA sets different grace period lengths for two broad categories of drugs. Biologics, which are administered by injection or infusion in a clinical setting, get a 13-year exemption. Small-molecule drugs, usually taken at home in pill form, get a nine-year break.
The implications of that four-year gap are profound and misaligned with the needs of patients and families. Drug developers should decide on avenues of research based on science and patient needs, and small-molecule drugs are essential for some children. I want my son to be able to access the treatments he needs in the least invasive and medically burdensome way. Now, facing shorter timelines to recoup investments in small-molecule drugs, the pharmaceutical industry is shifting resources toward the development of biologics, which are more invasive to administer and must be done in a clinical setting. Over the next 20 years, it is estimated that this will result in 188 fewer small-molecule treatments reaching patients.
This is troublesome for the Down syndrome community, as small-molecule research is advancing some of the treatments we need most. I can’t help but wonder if one of those 188 small-molecule treatments could be one that would prevent the onset of Alzheimer’s disease and related dementia for my son. The Down syndrome community has an over 90 percent chance of developing Alzheimer’s disease over a lifetime, a much higher rate than for the general population.
Small-molecule drugs represent one possible treatment option that could greatly affect these patients. Unlike biologics, these medications can cross the blood-brain barrier, targeting the root causes of dementia. Clinical trials of small-molecule drugs to prevent cognitive decline have been encouraging and have brought hope to a community that desperately needs it. Just as the research is showing promise, the IRA threatens to deter investment by penalizing small-molecule drug development as a result of the shorter grace period.
The effect goes far beyond treatments for Alzheimer’s. People with Down syndrome also suffer from hypothyroidism, heart anomalies, blood disorders, infections and epilepsy at higher rates than the general population — all areas that could benefit from small-molecule research.
The reality is that small-molecule drugs make life easier for everyone — not just individuals with Down syndrome. Without the cost and inconvenience of travel, patients can maintain daily activities. For individuals with Down syndrome, who may already struggle with invasive medical procedures and unfamiliar environments, this additional complexity can be overwhelming.
Thankfully, three lawmakers have proposed a solution to the IRA’s “pill penalty.” The bipartisan EPIC Act — short for Ensuring Pathways to Innovative Cures — was reintroduced earlier this year. The bill would eliminate the discriminatory treatment of small-molecule drugs by creating parity between pricing schemes. I support this bill because I do not believe that drug developers should be incentivized to develop drugs that are more invasive and less accessible to people with disabilities.
The Down syndrome community has made remarkable progress in recent decades. Each step forward came through hard-fought battles for access to innovative, life-saving medical innovations, thereby increasing the quality of care. This misguided policy threatens to reverse this progress by reshaping research incentives away from the treatments patients need and may prefer.
Passing the EPIC Act would give people renewed hope to live long and healthy lives.
