Can We Take the Excitement out of the FDA? Agency Could be Turning a Corner

The U.S. Food & Drug Administration (FDA) likes to trot out the statistic that claims it regulates about $100 billion in biomedical R&D each year: investments made by industry (and government) to create new innovations to treat today’s health challenges. But the agency has been hesitant to address another facet of investments: that they should be predictable. As the late, great economist Paul Samuelson said, “Investments should be like watching paint dry. If you want excitement, take $800 and go to Las Vegas.”

The U.S. Food & Drug Administration (FDA) likes to trot out the statistic that claims it regulates about $100 billion in biomedical R&D each year: investments made by industry (and government) to create new innovations to treat today’s health challenges. But the agency has been hesitant to address another facet of investments: that they should be predictable. As the late, great economist Paul Samuelson said, “Investments should be like watching paint dry. If you want excitement, take $800 and go to Las Vegas.”

Anybody who’s watched the stock market the last few years knows we would rather live without this kind of excitement. However, the latest documents issued by the FDA – from Research Use Only/Investigational Use Only (RUO/IUO) guidance to LDT to the latest strategy document on boosting innovation (read coverage from Xconomy) – are inconsistent enough to make drug development resemble a Roulette wheel, rather than a science-based, multi-year, multibillion-dollar strategy to develop therapeutics and diagnostics. Even venture capitalists, not known for their risk-averseness, have gone to Congress to demand more predictable responses from the FDA.

Could the FDA be showing signs—finally—that it is aware it needs to change its stripes? In its recent “Driving Medical Innovation” strategy document, the agency outlines a number of steps to streamline clinical trials for critical treatments, encourage more external input in policy-making, review policies and regulations for unnecessary obstacles, and train its staff to keep up on fast-breaking innovations in biotechnology. All of these are good things—we especially like the new small business liaison program and fellowships for young entrepreneurs. And the tentative list of Entrepreneurs in Residence looks impressive.

Like all of us who work in life science, the FDA faces many challenges, not the least of which is keeping up on new technology. While the agency has always acknowledged its dual roles as regulator and facilitator of innovative products, it now appears to be taking some realistic steps to embrace its latter role. If the reforms result in a more predictable, faster regulatory process, while maintaining safety obligations, then biopharmaceutical companies, investors, patients, health care providers and the U.S. economy can all win. Without the gambling.

What do you think of these FDA reforms? Will recent actions help create a clearer pathway for therapeutics innovation? Is there a broader opportunity for post-market surveillance, especially in assessing diagnostics? We look forward to learning your thoughts.