NATIONAL HARBOR, Maryland — Simon Frost knows all too well what it’s like to have a child with a rare disease, and how incredibly expensive it is to come up with a cure.

His daughter, Annabel, has alternating hemiplegia of childhood (AHC), a genetic disorder that affects only 900 children around the world. Simon and his wife, Nina, have invested $150,000 of their own money to create the nonprofit Hope for Annabel Foundation toward developing a gene therapy to treat their baby’s potentially fatal disease.

“Over the past 3 years, I’ve been working on developing therapies, things like AAV gene editing, and advancing the gathering of open-source genotype, phenotype, and natural history data,” said Frost, a resident of Washington, DC, and CEO of Tiber Capital Group, an investment firm. “It’s definitely not business for me. It’s personal, and it’s about as personal as it gets.”

Frost spoke at an August 26, 2021, roundtable during the 3-day World Orphan Drug Congress USA 2021.

According to Frost — who’s also a director at a second nonprofit, Cure AHC, and a board member of Rare-X, a patient data collection platform — the total US economic burden of rare disease in 2019 came to $966 billion, or roughly $30,000 per patient per year. 

“We spent that on hospital visits, doctors, medicines, and lost productivity, but we don’t spend it on R&D,” he said. “For AHC, all we can afford is $250,000 a year, or about $800 per patient per year. Why is there such a disparity? Because that $1 trillion falls on the shoulders of the rare disease community. We’re broke, and we can’t afford to do the job industry should be doing. Tax dollars are going to treat symptoms instead of cures.”

That’s because developing those therapies is extremely risky for pharmaceutical companies, he said.

“The manufacture and sale of products is a very low-risk business. You’re selling to a captive audience for whom it’s a matter of life and death,” Frost said. “But the discovery and development of drugs is entirely unpredictable. This high degree of risk causes drug companies to shy away from R&D, leaving us with a giant void for about 30 million rare disease patients in this country.”

However, he said, “if can reduce that risk, then we as a society can pay for these therapies, using the same financial methods that Wall Street uses. The only way is a coordinated public and private response.”

Instead of addressing diseases one at a time, he said, “we can build a big machine to address homogenous therapies, so that laymen like me don’t need to reinvent the wheel every time we need a new therapy.”


The economics of paying for expensive therapies: how can we make the process more efficient? Presented at: World Orphan Drug Congress USA 2021; August 26, 2021; National Harbor, Maryland.