YORK — The new year promises change and uncertainty, with health care reform square in the sights of both Democrats and Republicans in Washington. As a parent to a promising 17-year-old who has cystic fibrosis and is soon to be entering college, these are especially troublesome times. Health care solutions for people with rare diseases like cystic fibrosis must never be political – trust me, no one asked to have this dreadful disease.

Over 30,000 children and adults in the U.S. (about 300 in Maine) have cystic fibrosis, a deadly genetic disease. It causes the body to produce thick, sticky mucus that clogs the lungs and leads to respiratory infections and a host of other serious medical problems.

While advancements in cystic fibrosis care continue and hope remains great for new treatments and scientific breakthroughs, the typical cystic fibrosis patient endures a daily treatment regimen that would leave most breathless. It includes two or more hours of chest physical therapy, six to eight breathing nebulizations, each lasting 20 minutes, swallowing a combination of 45 to 50 pills, and much more, including making every effort to naturally live a normal life.

Nonetheless, many cystic fibrosis patients require frequent hospitalizations for intensive, cystic fibrosis-specific therapies, IV antibiotics and other treatments in a relentless and valiant effort to rid the lungs of the bacteria attempting to wreak havoc on the body, causing permanent lung scarring and, eventually, early mortality. Thankfully, with close guidance and leadership from the Cystic Fibrosis Foundation, in concert with a team of “rock star” doctors and nurses from the Barbara Bush Children’s Hospital at Maine Medical Center, our daughter has always received the highest-level care available – the importance of which cannot be underscored nor undervalued.

While crafting a health care system that benefits all Americans, our government leaders must remember Americans with cystic fibrosis and their undeniable need for adequate, affordable and available health care.

Adequate health insurance covers treatments and services delivered by providers who are experienced with the disease in accordance with cystic fibrosis care guidelines.


Affordable plans ensure that patients are able to access needed care in a timely manner from an experienced provider without undue financial burden.

Available coverage means that all people with cystic fibrosis, regardless of employment status or geographic location, should be able to gain coverage through a health plan that provides adequate benefits at an affordable cost, including out-of-pocket expenses.

Additionally, there are proposed health care reform policies that are of particular concern to the cystic fibrosis community, including:

High-risk pools: Before passage of the Affordable Care Act, people with cystic fibrosis and other rare diseases had negative experiences with high-risk pools. Premiums and out-of-pocket costs were too high and there were lengthy waiting lists.

Medicaid funding: Medicaid is a vital safety net for people with cystic fibrosis. Over half of all children with cystic fibrosis get their insurance through the program.

The pipeline of cystic fibrosis therapies is full of promising new treatments that would allow people with this disease to live longer, healthier lives than ever before. Implementing a rigid funding structure such as a block grant or per-capita cap would limit a state’s ability to keep pace with providing their population access to groundbreaking, lifesaving treatments.


 Pre-existing conditions: Coverage for people with pre-existing conditions is critical to people with cystic fibrosis. Coverage must be comprehensive, and people should not be denied coverage because they have a rare disease, or be charged higher premiums because of a health condition.

Annual and lifetime benefit caps: The prohibition on capping benefits on an annual and lifetime basis is critical to ensuring adequate access to health care for people with cystic fibrosis.

I thank U.S. Sens. Susan Collins and Angus King and U.S. Reps. Chellie Pingree and Bruce Poliquin for each signing on to the Cystic Fibrosis Senate Caucus and Cystic Fibrosis Congressional Caucus in 2016 and for their relentless support of the many National Institutes of Health and Food and Drug Administration efforts underway to discover and develop new therapies to treat and, hopefully, someday cure cystic fibrosis and other rare diseases.

Any politics aside, I believe in the goodness in each of our members of Congress. We ask them to again please keep the Maine cystic fibrosis community’s health care needs in mind while working this year to build a health care system that will benefit all Americans.

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