My son Jack spent the first four weeks of his life screaming in pain and crying from hunger. When he was two-weeks-old, the pediatrician told us our baby’s newborn screening test indicated that he had a 50 percent chance for a disease that required a special “sweat chloride” test, but had to wait two weeks for our tiny baby to gain enough weight to take the test.  

For those two weeks Jack suffered and my husband and I worried. We waited for our tiny baby to gain enough weight to take the test. The very next day we were sent to our Children’s Hospital where we first heard the diagnosis that changed everything: cystic fibrosis, a fatal genetic disease.

Cystic fibrosis affects 30,000 people in the United States, 70,000 worldwide. It is a progressive illness. Thick mucus builds up in major organs, creating many health issues and eventually causing lung failure and death. We were floored when doctors told us there’s no cure, and our sweet boy was only expected to live 28 years.

We searched for hope and found it in medicine. I remember watching Jack closely moments after he took his first medication, and I finally saw an expression of relief cross his sweet face for the first time, followed by his very first smile. I knew in that moment our best hope for Jack’s future lay in the possibility that pharmaceutical companies researching new medications might find a breakthrough drug to help people with CF live longer, healthier lives.

I have had good reason to be hopeful. Research and development of therapeutics for cystic fibrosis is advancing rapidly. Years ago, the life expectancy for a child with CF was age 5. Today it is 47. Pharmaceutical companies are creating medications to target the cause of this disease. At every conference I go to, I hear that a cure is on the horizon for my son’s genetic mutation.

But I also have good reason to be worried. Congress is currently debating a tax bill that will remove a tax credit vital to the development of drugs for orphan diseases like CF. Orphan diseases are ones with less than 200,000 patients in the United States, diseases with populations once considered too small for drug companies to invest in research and development to bring new drugs to market to address. In 1983, there were only 38 orphan drugs approved by the FDA, and there was little research into cures or treatments for rare disease because it’s an expensive process with a low profit margin. To address this, Congress passed The Orphan Drug Act, which among other things included a tax credit that created a fiscal incentive for pharmaceutical companies to invest in the development of drugs for rare diseases. Today, there are more than 500 drugs for rare diseases on the market and much more in development.

If Congress succeeds in removing the Orphan Drug tax credit, pharmaceutical companies will lose a key incentive to research cures and treatments for rare diseases. The National Organization for Rare Disorders has stated that they anticipate that if the tax credit is ended, there will be 33 percent fewer orphan drugs developed. That means that Jack, now age 6, will face even greater odds of living to see the cure he needs.

The tax bill that just passed in the House of Representatives gives parents of rare disease patients another reason to worry. Medications and other health related expenses for orphan diseases are expensive. Just one of my son’s many medications is priced at more than $58,000 for a three month supply. To help offset the hardship of such steep costs for critical care, families like mine rely on being able to deduct some of our medical expenses from our taxes. If we can no longer rely on this deduction, we won’t just worry about our children’s health, we also have to worry about our entire family’s financial future.

As the mom of a warrior kid, I can’t sit idly by. I learned how to advocate six years ago when doctors gave us that life changing diagnosis and I was thrown into a scary world of complex medical needs. Earlier this month we joined the Little Lobbyists—a grassroots organization of families just like mine who love their kids with complex medical needs. Since the threats to our healthcare emerged this summer, they’ve been showing up on Capitol Hill to advocate for our children and families, hoping they can change hearts and minds by sharing their stories and introducing their children to our legislators.

Jack and I recently attended a Senate hearing and a rally to advocate against taking access to affordable healthcare and hope for cures away from families like ours in order to fund increases in tax benefits for corporations and the very wealthy. 

This bill could take away our hope for a cure and our financial resources, two of the most important things families of medically complex children need to continue the fight for their children’s lives. Today, I’m writing this article to share our story with you. I hope it moves your heart and mind and gives you the incentive you need to call your legislators and ask them to rethink this tax bill, before it’s too late. As the mom of a child with a terminal illness, I know the clock is ticking.

November 27, 2017

Tasha Nelson is a patient advocate whose son has cystic fibrosis