When Patience Pays Off

Published on: 
Pharmaceutical Technology, Pharmaceutical Technology-04-02-2011, Volume 35, Issue 4

PhRMA efforts of industry's R&D scientists.

When chemist Ann Weber saw data from the first clinical trial of a molecule she'd helped develop, she was so excited that she worried about herself.

John Castellani

"For four days," she says, "I felt like I was flying."

She had good reason for feeling emotional. She'd earned a PhD because she dreamed that education could lead to her finding new medicines that could help people. The data she saw was preliminary, but provided evidence that maybe, just maybe, this molecule could be a new weapon in the battle against diabetes—a devastating disease of epidemic proportions.

When I hear of our industry being criticized, I think of people like Ann Weber. Our industry is filled with brilliant researchers who have dedicated their lives to seeking new medicines. If you've ever questioned the motivation of people working for biopharmaceutical companies, you need only talk to someone who has worked on a successful medicine.

Nancy Thornberry, who worked with Weber on the diabetes project, says she cannot begin to describe the "absolute thrill, the incredibly satisfying feeling" of learning that the first new oral medication for diabetes in a decade had been approved by FDA.


Thornberry and Weber are winners of this year's annual Discoverers Award in recognition of their leadership in the development of JANUVIA at Merck. For the first time in the award's 24-year history, two women have won the competition which recognizes researchers whose work has been of special benefit to mankind. The award is the highest honor presented by the Pharmaceutcial Research and Manufacturers of America (PhRMA) to biopharmaceutical scientists.

To those who perpetuate the myth that our industry focuses on me-too and lifestyle drugs, I offer Weber and Thornberry as an example of the drive and enthusiasm of thousands of biopharmaceutical scientists.

Last year, nearly 3000 medicines were in development in the US, either in clinical trials or awaiting FDA review. That figure includes 235 medicines to treat diabetes and related conditions, more than 400 for rare diseases, more than 800 for cancers, and about 250 each for cardiovascular diseases and mental disorders.

Our successes save and enhance the lives of millions. Thanks in large measure to new medicines, for example, AIDS deaths in the US have dropped more than 70% since 1995. From 1995 to 2005, heart-disease death rates in the US dropped more than 34%.

These achievements come despite the tremendous difficulty of discovering new medicines. Weber and Thornberry, in fact, represent not only the successes of researchers in our industry, but also their dogged determination in the face of failure.

Woody Allen once said, "If you're not failing every now and again, it's a sign you're not doing anything very innovative."

By that measure, among others, America's biopharmaceutical companies are incredibly innovative. On average, of 250 drugs tested in preclinical trials, one becomes an FDA-approved drug.

Just before she started on the JANUVIA project, Weber had spent 10 years working on a weight-loss drug. She and her colleagues felt hopeful when preclinical data showed that the compound slimmed down fat rats, fat dogs and fat monkeys. Only problem was, it simply didn't work on people.

Although JANUVIA was developed in record time, by no means did this diabetes program sail through research and development (R&D). The researchers were exploring the theory that patients with diabetes could control glucose in their blood by inhibiting the action of an enzyme called DPP-4.

In addition to in-house efforts, Merck also acquired a compound from a small biotechnology company that showed promise for inhibiting DPP-4. Unfortunately, preclinical safety trials showed that the compound wasn't well tolerated, and it had to be abandoned.

Their work eventually proved that inhibiting DPP-4 was the right idea. To find a DPP-4 inhibitor that was well tolerated, they tested more than 800,000 compounds. From those they found two with promise. Making variations of those two, they created more than 2000 new molecules. Of those, one turned out to be the compound that became JANUVIA. Today, it has been prescribed to millions of patients with diabetes.

Our industry exists to reduce human suffering, but the ancillary benefits deserve recognition as well. Medicines help the entire healthcare system by reducing hospital and nursing home admissions and by keeping people productive.

And in a competitive world where innovation is the key to economic survival, our industry invests more than 10 times more in R&D than manufacturing industries overall and supports 3.1 million jobs in the US.

Weber and Thornberry are part of a grand enterprise. As we honor them, we acknowledge the importance of an entire industry.

John Castellani is president and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA).