Live from BIO: When it comes to future drugs, think smaller but more effective

At his annual state of the industry report yesterday, venture capitalist Steve Burrill tossed out a statement that got a lot of people talking: 90 percent of drugs work in just 30 percent to 50 percent of individuals. That fact underscores one of themes that's emerged here at BIO 2011: the industry needs to shift its efforts from drugs that treat a lot of people to drugs that treat fewer people, but more effectively.

Nadine Cohen, Biogen Idec's senior VP in charge of of regulatory affairs, echoed Burrill's assessment of the future, noting that the FDA is very receptive to drugs that have been tailored to a specific patient population. Referring to drugs with a biomarker component, Cohen observed that "your benefit/risk equation is so strong that it increases your chances of FDA approval. It may save your drug." She delivered her remarks at FierceBiotech's Executive Breakfast on regulatory approval issues.

As the number of older Americans continues to rise, so too will healthcare costs. And payers will become much less tolerant of high-priced drugs that only work for a small segment of the population, warned Burrill. Efficacy will be king. Therefore, biotech and Big Pharma would do well to develop biomarkers and companion diagnostics to target patients who will have the best response to their drug. Developers that can prove their drug effectively treats a defined group of patients--even a small one--are the ones that will build real value.