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It's known as the "blockbuster," and it's the Holy Grail in the world of medicine – a Big Pharma drug that generates big yearly and lifetime sales because it targets a big opportunity in maladies that afflict millions of people.
Indeed, the three biggest blockbusters of all time target high cholesterol (Lipitor), inflammatory diseases (Humira), and digestive afflictions (Nexium) and have pulled in a combined $350 billion – and counting.
But what about people suffering from rare diseases? Here we're talking about such afflictions as cystic fibrosis, acute myeloid leukemia, multiple myeloma, renal cell carcinoma, ovarian cancer, Duchenne muscular dystrophy, glioma, and pancreatic cancer.
Many of these diseases are genetic, appear early in life, and are tough to treat. Indeed, while more than 7,000 rare diseases have been identified, about 95% have no approved therapies.
As many as 30 million Americans live with some type of rare disease, says the National Center for Advancing Translation Sciences Genetic and Rare Disease Information Center (GARD).
Compare that with diabetes – which all by itself affects more than 30 million Americans.
Given that drug firms and biotechs alike are striving for that Holy Grail blockbuster – and thus are targeting the maladies with the biggest number of sufferers – what's the outlook for rare-disease sufferers? Will drug companies develop drugs to ease their suffering?
Thankfully, the answer increasingly is "yes."
Here in America, Washington understands the need for these "orphan drugs" targeting rare diseases – and actually created financial incentives to encourage drugmakers to develop them. The "Orphan Drug Act of 1983" offers a seven-year window of tax reductions and the exclusive right to market a drug for a particular rare disease.
(You'll find somewhat similar regulatory designations in Europe, Japan, Singapore, and Australia to encourage the development of drugs to treat orphan diseases.)
The incentives are helping. North America leads the world in orphan drugs. Here in the United States, the number of orphan drugs coming to market has zoomed from a single digit in 1983 to 40 the next year to 121 in 2007.
To date, more than 600 orphan drugs have been approved by the U.S. Food and Drug Administration (FDA).
And the global market for orphan drugs targeting rare diseases is growing at double the rate of the non-orphan market. In 2017, $125 billion worth of rare disease drugs were sold – a total that will grow at a compound annual growth rate (CAGR) of 11.3% between now and 2024, when it reaches $262 billion.
That total would account for 21.8% of the $1.2 billion prescription drug market.
More needs to be done. And that "more" will create a massive investment portal – provided you pick the best-positioned companies.
About the Author
Michael A. Robinson is a 36-year Silicon Valley veteran and one of the top tech and biotech financial analysts working today. That's because, as a consultant, senior adviser, and board member for Silicon Valley venture capital firms, Michael enjoys privileged access to pioneering CEOs, scientists, and high-profile players. And he brings this entire world of Silicon Valley "insiders" right to you...
- He was one of five people involved in early meetings for the $160 billion "cloud" computing phenomenon.
- He was there as Lee Iacocca and Roger Smith, the CEOs of Chrysler and GM, led the robotics revolution that saved the U.S. automotive industry.
- As cyber-security was becoming a focus of national security, Michael was with Dave DeWalt, the CEO of McAfee, right before Intel acquired his company for $7.8 billion.
This all means the entire world is constantly seeking Michael's insight.
In addition to being a regular guest and panelist on CNBC and Fox Business, he is also a Pulitzer Prize-nominated writer and reporter. His first book Overdrawn: The Bailout of American Savings warned people about the coming financial collapse - years before the word "bailout" became a household word.
Silicon Valley defense publications vie for his analysis. He's worked for Defense Media Network and Signal Magazine, as well as The New York Times, American Enterprise, and The Wall Street Journal.
And even with decades of experience, Michael believes there has never been a moment in time quite like this.
Right now, medical breakthroughs that once took years to develop are moving at a record speed. And that means we are going to see highly lucrative biotech investment opportunities come in fast and furious.
To help you navigate the historic opportunity in biotech, Michael launched the Bio-Tech Profit Alliance.
His other publications include: Strategic Tech Investor, The Nova-X Report, Bio-Technology Profit Alliance and Nexus-9 Network.