CEO defends $300,000-per-year cost for a drug for a rare form of muscular dystrophy

[Judi Lynn]

Source: CNBC


Sarepta's Doug Ingram responds to a question about the high cost for a drug for Duchenne muscular dystrophy.
"We have already invested $1 billion fighting" this rare disease, he says. "And we're not done yet."
Shares of Sarepta soar after the company reported positive results from a clinical trial of another experimental DMD medicine.
Matthew J. Belvedere | @Matt_Belvedere
Published 18 Mins Ago



Source: CNBC
Doug Ingram, CEO, Sarepta

Sarepta Therapeutics CEO Doug Ingram told CNBC on Wednesday that accessing reimbursement from insurance companies for expensive, rare disease treatments is "always challenging."

But responding to a question about the $300,000-per-year cost for a Sarepta drug for Duchenne muscular dystrophy, Ingram defended the price tag.

"Sarepta is a small company. We have already invested $1 billion fighting Duchenne muscular dystrophy. And we're not done yet," he said on "Squawk Box."

DMD occurs in 1 in every 3,500 to 5,000 males worldwide. Symptoms generally start in early childhood, usually between ages 3 and 5. It rarely affects girls.

Read more: https://www.cnbc.com/2017/09/06/sarepta-ceo-doug-ingram-defends-high-cost-for-muscular-dystrophy-drug.html

[mopinko]

1. can you do math, dude? 1/5,000 is hardly a rare disease.

[Massacure]

5. Here is some math for you

There are just under 4 million births in the United States every year and 1/5000 males will be diagnosed with Muscular Dystrophy. The disease is rate in females. That means that approximately 400 people will be diagnosed every year. The average life expectancy for someone with Muscular Dystrophy is 26 years. So at any given time you are looking at about 10,400 people in the United States with it.

Then there is the fact that no one will actually pay $300,000 a year for it. Insurance companies will refuse to pay for it unless Sarepta can justify that price point. Sarepta may or may not provide charity care for the uninsured, but that's the result of cat and mouse games that insurance companies and drug manufacturers play with each other and is a topic for a different time.

[Cicada]

2. 4000 sales covers their costs

But how many drugs pan out? So to break even do they need 20,000 paying for one year? 40,000?

Patients now live 27 years?

Say 10 year usage? So 4000 patients? Say a million boys born each year in US? 200 per year?

That price seems maybe reasonable if so.

[shraby]

3. If it's on the rare diseases list, doesn't the government subsidize the research?

[BlueIdaho]

4. Jesus H Christ

Why do these CEOs all look like mafia "made guys?"