Drug Breakthroughs Need Human Touch

Ask any drug company executive why his medicines cost so much and you’ll get a standard answer: research and development is expensive. If Americans want wonderful new drugs, they have to be willing to pay for the research.
This argument is getting worn, but it still carries weight. Goodness knows we are desperate for better treatments for Alzheimer’s disease, cancer, AIDS, multiple sclerosis, kidney failure, strokes and even heart disease.
The pharmaceutical industry maintains that it takes over $800 million to develop a new drug. Last year R&D spending hit $33 billion, an all-time high.
There’s only one problem with this picture. Where are all the breakthroughs? In 1996 the FDA approved more than 50 new medicines. After roughly $200 billion in R&D spent over the last decade, the number of new compounds approved annually has dropped to half that number.
What went wrong? Developing new medicines is always difficult. But a decade ago the industry embraced a new technology called “high-throughput screening.” This robotic testing process was supposed to deliver thousands of exciting therapeutic compounds with minimal human effort and maximal efficiency. Instead, this expensive revolution has been disappointing.
Many companies invested hundreds of millions of dollars in drug testing machines. But robots don’t think, and issues like whether a drug dissolves in water were sometimes overlooked.
Perhaps more damaging, mechanizing the drug discovery process may not have left enough room for hunches and serendipity. These human qualities have often been crucial in the search for new compounds.
Just think about one of the most important breakthroughs in modern medicine. Alexander Fleming discovered that something was killing the bacteria he was growing for an experiment. But instead of discarding the contaminated culture, he investigated more closely and eventually isolated penicillin from the mold that had invaded it. Penicillin has saved millions of lives as a result of his accidental discovery and persistence.
Some of the most successful medications today are called ACE inhibitors (Accupril, Altace, Capoten, Lotensin, Prinivil, Vasotec, Zestril). These blood pressure pills have helped millions and new benefits are still being found.
The history of their development reads like a mystery thriller. A Brazilian scientist noted that when people were bitten by the poisonous jararaca snake their blood pressure dropped dramatically. Investigation of the chemical activity of the venom led to the development of captopril, the first drug in this class. A robot could never have figured this out.
Sometimes human foibles are key in drug discovery. Researchers investigating a heart drug called sildenafil wondered why so many men were reluctant to hand over their left-over pills at the end of the clinical trial. The drug hadn’t worked all that well against heart problems. But it has made billions for the company under the brand name Viagra.
If drug companies expect Americans to continue subsidizing their research and discovery process they had best get back to basics. Sometimes luck and intuition are essential ingredients in drug breakthroughs.