PharmExec Blog

Industry and FDA Brace for Change


Janet Woodcock

While the convention floor of the 46th annual meeting of the Drug Information Association bustled with the usual vendors touting the latest and greatest clinical trial technology, the panels (particularly those that featured FDA members) had a remarkably somber tone.

The main refrain was: “Things are going to change and everyone must step up or get trampled.” However, those comments were often followed by: “We are going to have to change, but we have no idea how or how much it will cost.”

“Drug development is in crisis, and there is a demand for better evidence,” said Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research during her panel presentation “The Impact of New Comparative Effective Requirements.”

“Right now, we are at the same point with comparative effectiveness as we were with drug efficacy in 1960,” Woodcock said. “We didn’t know how to do it, and there was a lot of resistance to it. There was a battle over efficacy that almost died.

“In 2010 there is no requirement for comparative evaluation pre-market,” Woodcock continued. “Effectiveness is something different. Pre-market  clinical trials won’t give you that effectiveness, because they aren’t done in a ‘real world’ setting. The preferred methodologies we don’t actually know. There are people out there that think we can just push a button on healthcare data sets and get the answers.”

She also noted that pharmaceuticals, unlike other health technologies, have a tremendous evidence base. “The FDA Amendments Act explicitly calls for additional evidence regarding safety when new safety questions come up after marketing,” she said. “Comparative effectiveness is a call for more outcomes data to guide practice. It’s a trajectory that industry must recognize. This is where we are all going together.”

She pointed out that there are budgetary restraints in government so it will fall into pharma’s lap to perform these new pre and post market trials. “Unfortunately, right now, drug development is in crisis, but this isn’t a good time for the pharma industry to take on new requirements,” Woodcock said. “For the regulators, this is a huge problem. Drug regulators are caught between two societal expectations and demands. On one hand you have the rising desire for certainty, and on the other they want innovative drugs.”

In Europe, they think that the tech assessors such as NICE are going to start planning pre-market, during the drug development process, and give advice on how to fulfill the technology assessors requirements. In that way, the drug development process can be seen as a seamless process that takes care of the regulators needs as well as the payer’s needs.

The Tough Questions
“The problem that we have is that we had 50 years of experience working this out,” Woodcock said. “The technology assessors don’t think the same way regulators do—we have a great deal more experience in trial methodology and analysis. We realize the limitations in data and the methodology in CE has not been worked out.”

She said that there will be tremendous pressure on industry and regulators for more evidence and that’s what’s driving the controversy about safety. The question is: Who in the US will generate this initial evidence, and what evidentiary standards will be applied? FDA just opened a study with the Institute of Medicine on the use of observational studies and other types of databases versus randomized control trials to evaluate safety signals.

Woodcock brought up two more questions: How will the standards be implemented and when in the drug development product should what evidence be generated? How much certainty should we have in any given point during drug development?

“It’s imperative that the United States develop a clinical trial infrastructure,” Woodcock said. “We don’t have a machine to generate all this evidence. We don’t have any mechanisms in place to generate all this evidence. It’s time that we develop the capacity to find out these answers in a way that doesn’t cost hundreds of millions of dollars every time we ask a question.”

The Harder Answers
For certain diseases, like cancer, the answers might be driven from the patient side. The Cystic Fibrosis Foundation has set up such a network for their patients so they can get into trials for new treatments rapidly—and so that those new treatments can rapidly be evaluated.

Another suggestion Woodcock offered is to move on the science of new personalized medicine. “That’s the best way to improve the value of medicine,” Woodcock said. “We must target it towards populations that stand to benefit more and don’t have as much risk. We are there on the science.”

Finally, she said that there’s new science of safety evolving and that has to do with Sentinel and the use of electronic health records. That science will help industry and FDA evaluate the performance of medical products once they are released. “The problem is that the science is also in its infancy, and there are a large number of questions that we’ll have to answer before those types of databases can be used to answer the questions we have about outcomes,” Woodcock said. “Comparative outcomes data are essential for evidence-based medical practice, and they have to be gathered in real world settings so in some ways the premarket world is not the time to get this information. Methodologies for doing this kind of research in the US are lacking. Work is ongoing, but more needs to be done, and we need to incorporate new science so we can understand what we are doing.”

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One Comment

  1. Posted June 17, 2010 at 10:56 am | Permalink

    I am glad the FDA is finally getting around to this problem. Our little start up ( has done a great deal of comparative analysis for our technology (all of which has been positive). We constantly monitor our performance in predicting patient outcome and response to therapy against other technologies. I am glad to hear the FDA is taking this approach for other technology domains

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