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# FDA Needs More Evidence for Good Decisions: Califf
- URL: https://www.fdaweb.com/fda-needs-more-evidence-for-good-decisions-califf/
- Published: 2017-01-03T12:00:00.000Z
- Updated: 2026-09-14T21:56:16.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5137620

In an interview with the *Washington Post* as he prepares to return to Duke University, from which he is on leave, FDA commissioner **Robert Califf** [says](https://www.washingtonpost.com/news/to-your-health/wp/2016/12/29/the-head-of-the-fda-defends-the-importance-of-drug-effectiveness/?utm%5Fterm=.43f55a43f816&ref=fdaweb.com) that for FDA to be able to make scientifically sound decisions, it needs more evidence about a regulated product’s safety and efficacy. He describes how as an intensive care doctor he made decisions in individual patients and then as a researcher always had the luxury of being able to identify areas where further research is needed.

“At FDA, you have a lot of the same science issues but you have to make decisions,” he said. “And it’s very noticeable when we make decisions with good evidence. It’s still emotionally charged because we regulate such a large part of the economy, and there are winners and losers. But when we have good evidence, it’s easy to defend the decisions and the arguments are typically good arguments to have about how you interpret good evidence. But we are often in situations where to have to make decisions because there are time limits and we don’t have the kind of evidence we really need to make a good decision, and then things are not as pretty. Here we are in a time when everything is digitized, we have scads of information, but as a society we haven’t been so good at turning that information into knowledge, particularly in the medical arena.”

Asked specifically about the controversy over FDA approval of the Sarepta Therapeutics Duchenne muscular dystrophy drug Exondys (eteplirsen), Califf said the agency really didn’t have the evidence it wanted and a decision had to be made. (He ended up deferring to CDER director **Janet Woodcock**, who overruled the objections of lower-level scientists and medical reviewers to approve the drug.) Califf says he has laid out steps that he thinks should be taken in the future to reduce the number of times FDA finds itself in such a situation.

“By law,” he told the *Post*, “FDA is given a lot of discretion in cases of serious and life-threatening diseases with no available treatment. We are instructed by law to consider all sources of evidence beyond the traditional and to use unvalidated biomarkers if we think that it’s reasonably likely they will predict a clinical benefit. So as I pointed out … the definition of ‘reasonably likely’ is not defined and ‘all sources of evidence’ includes a lot of possible things.”

He said FDA has been working closely with the National Institutes of Health because there are 5,000 rare genetic diseases and many cures will start coming as we learn more about the functional aspects of the human genome. “I hope people will look at the Serapta case and say there are some things that regardless of whether we think FDA made the right decision, let’s look at what needs to be done for the next 4,999 rare genetic diseases because patients and their families have reason to want to get things done.”

Califf also was asked about the argument voiced by many, including **Jim O’Neill**, who has mentioned as a possible FDA commissioner in the Trump administration, that drugs should not have to be proven effective before the agency approves them. He reviewed the history of proving drug effectiveness dating back to before 1962 and noted that the standard that is written into law is actually quite flexible. He pointed out that almost 90% of drugs that go into Phase 1 testing don’t make it to market either because of toxicity, they actually don’t work, or they can’t be manufactured on a scale that is needed to be safely produced and distributed in a global market. “I think we have pretty clear evidence from the public that they would like to have a system that’s giving them some assurance that the treatments they are given work,” he said. “But again, to get back to the flexible standard, that means in the case of rare genetic disease with no available treatment, ‘working’ means changing a biomarker, it doesn’t mean improving a clinical outcome, and then the proof of that is developed post-market.”

In other comments, Califf said the agency got much of what it was hoping for in the 21st Century Cures bill in terms of Congressional approval for pay increases for scientists and other expert staff and the sections on real-world evidence and modernizing clinical trials. He also said that FDA agrees on the need to move as quickly as possible to approve generic drugs, and noted that drugs in the backlog are almost always compounds that have had multiple applications because of deficiencies that had to be corrected. He said the notion of some members of Congress that there should be a 150-day deadline for generic drug approvals may not necessarily be the right number. “But there’s no objection at FDA to going as fast as we can as long as we keep a quality standard,” he added.