Tough to swallow

WORLD writer Sharon Dierberger pitched me a story about rare diseases several months ago. It sounded ­interesting, but I asked her to keep digging. I wanted to see if she could discover anything really surprising under the surface. Boy, did she. Her story, “Life-saving treatments, blockbuster profits,” in this issue, details the massive scheme pharmaceutical companies use to fleece patients and taxpayers. When she was done digging through all the data, I asked her to reflect on what she’d learned. Here’s what she told me:

What motivated you to write this story? I’d written a few articles before about people with rare diseases. Then a WORLD reader wrote to tell me the story of her infant, who had recently been diagnosed with a rare disorder. Researching his disease, I learned how the Orphan Drug Act resulted in effective new treatments. But then came the downside: greed, companies preying on patient desperation, and seemingly ­endless profits.

These drugs cost a lot, but they work, right? Wrong. Most patients think if a drug is FDA-approved, then of course it works. But multiple studies have shown that’s not true. Even a Big Pharma–backed study last year said half the orphan drugs getting FDA accelerated approval haven’t proven they’re effective. Drug companies promise to verify effectiveness later, after patients are already taking them. But once a company is successfully selling a drug, it has no incentive to follow through.

What did you learn that surprised you? The sheer number of people affected by rare diseases. Collectively, rare diseases aren’t rare. Thirty million Americans—that’s about 1 in every 10 people—have a rare disorder. Two-thirds of them are kids. That’s an astounding number. It’s just that so few have each one. Sitting in church, I sometimes look around and wonder which families are dealing with this and what else we could be doing to help.

What grabbed you about this story? It’s heart-wrenching to hear about the lives of those afflicted and their caregivers. But those I talked with had similar attitudes I find humbling: They’re ever-hopeful for a treatment or a cure. They told me their suffering brought some good things, too—some mentioned a deeper relationship with God and more genuine conversations with loved ones. Another said she’ll always have a greater appreciation for the ordinary, like her baby’s smile, a day without a crisis, a friend’s word of encouragement, or simply being able to sit down for a minute and listen to birds sing. Each spoke of trusting God, even while acknowledging many moments of ­feeling overwhelmed.