The process for approving new drugs in the U.S. takes a long time and costs a lot of money. But in trying to speed things up too much, Congress runs the risk of allowing drugs to reach the market that aren’t necessarily safe.
Some lawmakers — led by Energy and Commerce Committee Chairman Fred Upton, a Republican from Michigan, and Diana DeGette, a Colorado Democrat — assert that drugs are slow in coming because the Food and Drug Administration has been failing to innovate quickly enough. But last year the agency approved more new drugs than in any other year in almost two decades. In 2013, the median approval time for a new drug was about a year, an improvement over prior years and faster than that of the FDA’s counterparts in Europe or Japan. Most new drugs in the U.S. now qualify for some form of expedited review. And two-thirds of drugs approved worldwide last year were first approved in the U.S.
That doesn’t mean the FDA can’t be improved. The agency says it could do its job better if it had greater authority to hire staff directly. It also wants greater freedom from congressional rules that cap executive salaries at about a third of what drug companies pay. A bill that may soon come up for a vote in the House would give the FDA both, as well as push to create still more avenues for expedited drug reviews.
But changing how the FDA operates can do nothing to shorten the long time — almost 13 years — that it takes a new drug to arrive at the FDA’s doorstep. The discovery and development phase lasts an average of six and a half years, and clinical trials take six more.
Speeding up the first of these pre-FDA phases takes an investment in research. To encourage faster drug discovery, the House proposal would fund more research, granting the National Institutes of Health an extra $10 billion over five years — a timely injection of new money for an agency whose budget has been flat for a decade. But how can clinical trials be made to move faster without increasing the risk that unsafe drugs might be approved?
Congress wants to make it easier for drug companies to use changes in so-called biomarkers, such as cholesterol levels or plaque concentrations in the brain, as proxies for more traditional health outcomes that take longer to measure. It also wants the FDA to be more willing to accept clinical trials with fewer participants, and put more weight on evidence from outside the trials and testimony from patient groups.
Some of these ideas have promise. But the balance between political pressure and scientific caution is easily upset. Lawmakers need to give the FDA more flexibility without pushing it to weaken specific aspects of the approval process, especially clinical trials, in ways that the agency considers unsafe.
The legislation needs to be written to reflect this principle, and Congress will have to acknowledge that the gains from reform could well be modest and incremental. Modest change is the only safe kind.