This archive report was first published on 26 August 2019.
Published on August 26, 2019, a patient advocate, Maria Kefalas, described the situation of high-priced gene therapies 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 basic abilities like crawling, walking, swallowing, and speaking.
Ms. Kefalas worries that these treatments will cost millions of dollars, leaving families struggling to afford them. 'I don’t know who is going to pay for this,' she said, looking at working with families.
Some attribute the trend of high-priced drugs to the Affordable Care Act, which banned lifetime caps and annual limits on patients’ coverage and includes a mandate that employers keep paying for drugs, no matter the cost.
Companies like Alexion hold all the leverage in pricing these one-of-a-kind drugs, offering minimal discounts and controlling the price.
Dr. Jonathan Gavras, the chief medical officer of Prime Therapeutics, recalled a small employer that was forced to cover a worker who needed Strensiq, a gene therapy, a few years ago. Paying for the employee’s drug 'essentially almost put the group under.'
