The Bakersfield Californian
By Eric Butcher
When calling myself an alpha male, there is a deeper meaning that goes beyond the stereotype.
I was diagnosed with Alpha-1 Antitrypsin Deficiency 14 years ago. It caused liver and lung disease. I already had fatty liver disease and cirrhosis. Sadly, doctors didn’t show concern or offer follow-up instructions. The first Alpha-1 doctor predicted I would die within five years. He advised to “get my affairs in order.” I grieved profusely.
But when I found my alpha-1 community and educated myself about my condition, the “alpha male” in me offered strength for surviving a decade past my diagnosis. My mission: helping others who deal with these issues and systemic misinformation.
Alpha-1 is a rare genetic disorder caused by a deficiency or malfunction of the alpha-1 antitrypsin (AAT) protein — created by the liver and then released into the bloodstream to help protect the lungs and liver from bacteria. The protein can get stuck in the liver and accumulate, leading to inflammation, fibrosis and eventually cirrhosis, which is what happened to me. Without AAT in the bloodstream, it can’t do its job of protecting the lungs, so I developed emphysema as well.
Researchers are hoping to cure Alpha-1 within the next 10 years, but I — and others living with rare diseases — need timely access to treatments. One that helps me is plasma-derived infusion therapy, which raises my AAT protein levels. While the weekly infusions can’t reverse damage already done to my liver or lungs, they help slow disease progression. There are currently no approved treatments for Alpha-1 Liver Disease, but some are in advanced clinical trials.
I’m grateful to be able to receive these infusions, and especially thankful for plasma donors, but I can’t stop myself from hoping that a cure will be discovered tomorrow.
We’re lucky in the United States that the best scientists are developing and testing new modalities to ensure people can live longer lives. But decades of progress could soon be at risk if newly proposed price-control proposals are implemented.
Don’t get me wrong; we all want lower prices for essential medications. If I didn’t have Medicare, I would have to pay $20,000 every single month for my weekly infusions. But government-mandated price setting is not the solution.
It costs billions of dollars to bring a drug to market years of clinical trials. And, these investments prove fruitful. Earlier this year, a novel gene therapy emerged that could cure Alpha-1 and its lung co-morbidities. And, just a month ago, another gene therapy successfully slowed the progression of Huntington disease, one of the most devastating brain disorders with no available treatments.
If flawed policies like “Most Favored Nation” pass, these breakthroughs may never come to market, let alone be developed. Why? Because Most Favored Nation and similar policies won’t actually lower what patients and consumers pay. Instead, MFN would tie U.S. drug prices to the prices of countries with government-mandated price controls — the very countries reliant on U.S. innovation.
Drug pricing provisions of the Inflation Reduction Act created a disincentive to invest in small molecule drugs, like antibiotics, despite the urgent need for improved versions.
According to the World Health Organization, one-sixth of bacterial infections are resistant to antibiotics, and rates of drug-resistance are rapidly growing, yet development of new antibiotics slowed. If MFN were adopted alongside the IRA, rates of antibiotic resistance would rise, fewer cures would be developed, drug shortages would skyrocket, and prices would Likely stagnate.
There are more than 7,000 known rare diseases, affecting more than 30 million patients (about the population of Texas) in the United States alone. That equates to about 1 in 10 Americans. All of us together are a powerful voice in patient advocacy and health care reform, and it is imperative that legislators listen to our call for increased access and transparency.
MFN will hold us back. The only way forward is through patient-centered policy proposals that help reduce what consumers pay, particularly those on commercial health plans. Patient-centered and patient-led must be the way of the future.
Eric Butcher is co-leader of the Bakersfield Alpha-1 Support Group and founded the global Alpha-1 Liver Disease Support Group, educating, advocating and supporting those affected by Alpha-1 Antitrypsin Deficiency. He actively partners with patients, caregivers and policymakers to ensure no one faces Alpha-1 alone.