The tax bill will either cut or reduce the tax credit for companies developing drugs for rare diseases. Approximately one in 10 Americans has a rare disease. This means that these cuts could ultimately cost the country millions of dollars, while taking away hope from patients like me.
In 2014, I became one of some 40,000 people in the U.S. diagnosed with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), a rare neurological disorder. By the time I was admitted to the hospital, nine months after my first symptoms, I had no feeling from my legs to my torso, could not move my left hand, and was losing the ability to eat.
I was lucky that it took less than a year to diagnose my condition: on average, it takes seven years to correctly diagnose a patient with a rare disease, according to the Shire Rare Disease Impact Report. The burden this places on patients, their families, their caregivers, and the U.S. healthcare system is enormous. By definition, a rare disease affects fewer than 200,000 people in the U.S. But with some 7,000 rare disease identified, that means approximately 30 million Americans — 1 in 10 people — have one.
Thanks to regular infusions of immunoglobulin, my symptoms are now under control. But while my symptoms can be managed, there is no prospect for a cure — unless there are incentives to not treat "rare diseases" as though they were rare.
The new tax bill will eliminate or curtail those incentives. Since 1983, the orphan drug credit has helped more than 500 drugs come to market. The National Organization for Rare Disorders calculates that eliminating the credit would result in 33 percent fewer orphan drugs developed for rare diseases.
This tax bill is a broad scale attack on U.S. health care. It will repeal the individual mandate of the Affordable Care Act, it could trigger cuts to Medicare, and it will stifle investment in rare disease research. True, the orphan drug tax credit could be reformed to reduce concerns over abuses, but this blunt act endangers millions of lives.
There are ways to fix the U.S. health care system. Removing tax incentives to develop life-saving therapies and cures for millions of Americans is not one of them. Rare diseases are a critical component of the U.S. health care system that require more than a Band-Aid.
Instead, we need more funding for powerful, consolidated approaches such as the Rare Diseases Clinical Research Network to transform the theory of genomic medicine for rare diseases into a reality.
We need more examples of patients and their families who, in the face of a life-threatening diagnosis with no cure, were able to use the orphan drug tax credit to develop therapies that prolong and improve quality of life. The benefits of these research initiatives will yield new discoveries that extend far beyond the 30 million patients with a rare disease.
Developing therapies for rare diseases is expensive, but not compared to the costs of managing symptoms that progressively worsen with chronic disease. My infusions cost my insurance company more than $100,000 a year: and could total more than $4 million over the course of my lifetime alone. The costs can be staggeringly higher for some of the 30 million rare disease patients whose therapies cost more than a million dollars a year.
This is not, and should not be, a partisan issue; over 200 patient groups support the orphan drug tax credit. We cannot afford financially or morally to continue to treat rare diseases as though they were rare. Repealing or cutting the orphan drug tax credit undermines the hope that a treatment or cure is possible. And for some patients and their families, hope is all they have.