The $6 Million Drug Claim

Maria Kefalas, a patient advocate whose daughter, Calliope, has a rare form of leukodystrophy, described the situation as a “slow-moving train collision.”

At least two companies are developing gene therapies for leukodystrophy, a deadly group of genetic disorders that cause damage to the brain and central nervous system, robbing children of the ability to crawl, walk, swallow and speak. Treatments, Ms. Kefalas worries, will cost millions of dollars. “And I’m sitting here, as a mom, looking at working with families and saying, ‘I don’t know who is going to pay for this.’”

Some attribute the trend of drug companies charging very high prices to the Affordable Care Act, the federal law that banned lifetime caps and annual limits on patients’ coverage. It includes a mandate that employers keep paying for drugs, no matter the cost.

“There was no market for this,” Mr. Fuerstenberg said. “Now there is.”

Because these drugs are one of a kind, companies like Alexion hold all the leverage. They offer minimal discounts and control the price.

Dr. Jonathan Gavras, the chief medical officer of the pharmacy benefit manager Prime Therapeutics, recalled a small, self-insured employer that was forced to cover a worker who needed Strensiq a few years ago. Paying for the employee’s drug, he said, “essentially almost put the group under.”

The Pattersons’ bills forced the boilermaker union’s leaders to consider raising its workers’ premium contributions for the first time in eight years.

Ms. Jasperson sought help from officials — reaching out to her congressman, the Labor Department and even President Trump, who has made consumer drug prices a major talking point.