One of the most intense discussions at ASCO over the past few days has zeroed in on the FDA's new "breakthrough" drug status. The agency is offering developers essentially an open door at the FDA to any program that qualifies, urging on R&D teams with quick responses and help adapting trials and hurrying the review process with the aim of getting more "transformative" drugs to the market faster.
"We have unfettered access to higher-level officials at the FDA," Gary Gilliland, Merck's ($MRK) senior vice president in charge of the oncology franchise, boasts to the Wall Street Journal.
But while the agency says this new designation will speed the arrival of game-changing drugs, a study in Health Affairs suggests that the world's drug R&D engine has been steadily weakening, growing less and less able over the years to usher in true breakthroughs of any kind.
Columbia University's Dr. Mark Olfson and Steven Marcus of the University of Pennsylvania say they reviewed the results of 315 placebo-controlled trials that were published in four big journals between 1966 and 2010. In the 1970s they found that new drugs were on average 4.5 times better than a placebo, and then the trend line pointed straight down. By the time pharma R&D finished the first decade of the 21st century, new drugs were only 36% better than placebo.
And the trends point to a new wave of "incremental" improvements ahead.
"Substantial investments are made in clinical trials," the authors write. "In the United States, for example, more than $100 billion is spent each year on biomedical research, with most of the funding devoted to clinical trials. Concern over a slowdown in the discovery of innovative medical treatments has prompted calls for new public policies to stimulate drug development, including incentives for novelty drugs. During difficult economic times, however, private research sponsors may be tempted to take a cautious approach that favors incremental research focused on follow-on or 'me too' drugs that offer little additional efficacy over more speculative high-risk/high-reward strategies."
Industry insiders are likely to point to the fact that in the '70s and '80s, drug developers were also offered a chance to pluck plenty of low-hanging fruit. No one disputes that R&D productivity levels dropped significantly between 2000 and 2010, but in the past few years there has been a significant spike in new drug approvals. And the oncology field in particular has boasted significant advances in fields like prostate cancer.
The researchers note that drug developers today face a fresh challenge--and opportunity--in new comparative effectiveness research mandates laid out in the Affordable Care Act. But R&D groups have already turned to comparative trials to build a case for new drugs with the world's payer community. And with big players like GlaxoSmithKline ($GSK), Roche ($RHHBY), AstraZeneca ($AZN), Pfizer ($PFE) and others executing major restructuring plans over the past few years, it's unlikely that many will be surprised to hear about the decline in productivity. Fixing that process has become a priority.
- check out the abstract
- here's the report from Reuters
- here's the story from The Wall Street Journal on the breakthrough designation