Companies seeking approval for new drugs or biologics must navigate the clinical trials process, which can take over a decade and hundreds of millions of dollars to complete. Only a small fraction of compounds discovered in the preclinical stage will go on to received FDA approval, and in recent years the agency has become even more rigorous in its reviews.

Phases of drug development
In the pre-clinical or drug discovery phase of the approval process, researchers look for potential new compounds to treat targeted diseases. Promising compounds are tested in animals and living tissue until they are refined enough to give to humans. When all tests are completed, the drugmakers file an investigational New Drug Application (IND) with the FDA. In Phase I testing, the new drug is given to a small group of healthy people to see if the drug is safe and well-tolerated. Safe drugs are advanced into Phase II trials, where companies see if their compound is actually effective in a small number of people who have the disease they're targeting. If evidence of efficacy is observed, the drug is moved into large-scale Phase III studies. Positive results at this stage prompt drugmakers to file a New Drug Application (NDA) or Biologics License Application (BLA). An expert panel reviews all the trial data and makes a recommendation to the FDA, which in turn decides whether or not to grant approval.

Low rate of new-drug approval
Following a string of high-profile recalls and safety warnings, the FDA is increasingly rigorous in the review process. As a result, fewer drugs are being approved. Only 21 new drugs got the green light in 2010--fewer than both 2009 and 2008, when 25 and 24 were approved, respectively. However, 2010 was better than 2007, when regulators just 18 medicines-an all-time low. The agency is particularly focused on drug safety. Regulators also want to see evidence that new drugs not only work, but offer real benefits compared to older and less-expensive medications already on the market. Oncology and obesity drugs face the most scrutiny from the agency, while large molecule drugs (biologics) are twice as likely to be approved than than small molecule drugs.


FDA approval

Latest Headlines

Latest Headlines

An ominous trend resurfaces as new drug approvals plunge in 2013

The wave of new drug approvals that had been building at the FDA has broken. According to the official tally of new drug and biologics approvals at the agency, the biopharma industry registered only 27 OKs for new entities in 2013--a sharp plunge from 2012's high of 39 that once again raises big questions about the productivity and sustainability of the world's multibillion-dollar R&D business.

J&J, Pharmacyclics win early approval for 'breakthrough' cancer drug ibrutinib

The FDA has come through with an early approval of the BTK drug ibrutinib, a likely new cancer blockbuster from Pharmacyclics (and Johnson & Johnson.

Helpful regulators, blockbuster drug strategy deliver a crop of major breakthroughs

So far this year the FDA has approved 27 new drugs, fewer than last year's total. But a greater focus on real medical breakthroughs will make this year's crop far more valuable than the harvest of 2012, according to EP Vantage.

2013's story: Fewer drug approvals, better-selling drugs

Despite fewer drug approvals overall compared to last year, this year will have turned out a new crop of blockbusters whose combined revenues 5 years out are forecast to reach $18.7 billion, besting last year's record of $16.4 billion in projected sales, according to a report released today by EP Vantage.

Bayer wins key FDA approval for lung disease drug Adempas

Just hours after Bayer laid out some ambitious plans to accelerate development of a new wave of cancer and cardiovascular therapies, the FDA came through with a key new approval of riociguat--to be sold as Adempas--for a devastating lung disease.

Government shutdown would likely roil FDA's drug-approval process

So what happens at the FDA in the event of a government shutdown next Tuesday? For now, the agency isn't saying, referring reporters to an OMB statement guiding government agencies to prepare to execute "an orderly shutdown."

With $29B in deals so far, 2013 shaping up as big M&A year

With Valeant Pharmaceuticals forking over $8.7 billion for eye-care giant Bausch + Lomb and Amgen's $10.4 billion deal for Onyx Pharmaceuticals, this could be a comeback year for pharma M&A, even as the drug approval rate remains healthy.

The FDA OKs 5 blockbusters in slow start to 2013 drug approvals

Few productivity metrics in biopharma measure up to the annual rate of new drug approvals. So now that the FDA has come through with only 13 novel approvals in the first half of the year, well off the pace of 39 new approvals in 2012, there's some handwringing going on.

New GlaxoSmithKline lung drug nabs thumbs-up from FDA experts

The agency advisers weren't unanimous in backing GSK's follow-up to now-off-patent Advair, however.

Manufacturing problems keep Allergan from drug approval

Manufacturing problems are again standing in the way of a drug launch, with the FDA holding off approval of Allergan's ($AGN) new migraine treatment until problems at an inhaler plant are sorted out.