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CRO Leadership Awards 2014

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LIFESCIENCELEADER.COM THE CRO LEADERSHIP AWARDS 2014 8 ROUNDTABLE leaders saying, it would make sense if there were more clarity, because this subjectivity fuels the 'I need to collect more, I need to be ready for any ques- tion a regulatory authority asks for.'" Heyrman: "It's really not the health authori- ties; that's just a nice smoke screen we use to protect ourselves. If my scientist cannot explain to the data manager why we need to collect this piece of data, it probably isn't worth collecting. If you can't explain to somebody not in your field why you want to have what you want to have, question yourself as to whether you really need to have it." I have heard similar debates in other forums, with some folks advocating for specific regulatory clarity as to what is required. This is not likely going to happen — and for good reason. Though the FDA's remit is safety and efficacy, it also needs to create an environment where people feel comfortable being able to question and challenge convention in order to spark innovation. Rather than seeking greater regulatory specificity handed down from the FDA, instead, the goal should be to strive to create a greater degree of collaboration and transparency between industry and regulatory agencies. L Fiore: "As long as you demonstrate that you thought about it and had good reasoning." Anderson: "But the fear is, they're not going to approve it, and you'll have to do another study. That's the issue companies struggle with in designing studies." Katz: "Agencies sometimes give contradictory recommendations or direction. They tell you one thing once, and then eventually, as they learn more, they'll change their minds. It became very obvious to me that these agencies need to part- ner with industry. The regulatory agencies may not have the bandwidth to fully understand the implications and consequences of their changing decisions." Anderson: "We've got to change our way of think- ing. It would be great for our government and other regulatory agencies around the world to say, 'We want it this way.' We are a conforming industry. If you tell us something, you'd be sur- prised how fast we're willing to get behind it and align to it. I have yet to see something where guidance comes out and somebody says, 'It is too difficult to comply with that guidance, so we're going to submit the information the way we've collected it.'" Fiore: "We tolerate extreme subjectivity in the industry, and Mark [Anderson], with what you're During our recent clinical trial thought-leader- ship roundtable, one thing became clear — sim- plifying the clinical trial process is a desirable goal. To do so, members of the roundtable sug- gested that we need to ask the right questions — sparking the following spirited debate. Katz: "So we put everything but the kitchen sink into a trial, and we realize at the end of the day we've collected a lot of rubbish. It causes us a lot of discrepancy resolution problems and costs us a tremendous amount of money. You've got to get simple." Krusinska: "You were mentioning complex designs leading to operational difficulties. It's not only operational difficulties. Actually it does not work for science, either. If I have 200 end points, what value does it bring statistically?" Anderson: "What we need to look for from the beginning is — are we asking the right questions to start with?" Katz: "But the questions have to be aligned with what the regulatory agencies are looking for. The reason we collect everything is because we anticipate that eventually the FDA is going to ask us a question about the data." Heyrman: "But honestly, you can give them the answer, 'We didn't collect that. Sorry.'" to try to answer too many questions," he states. "Louis Lasagna [respected expert in clinical pharmacology] taught us that if you ask too many questions, you are going to lose the focus of your trial." Anderson agreed, adding, "If we don't get that under control, we could be asking a lot of ques- tions that really don't gain us much infor- mation about the actual effectiveness of the drug or what could be commercialized as we go forward." The initial thought behind asking lots of questions in a study was that it would reduce costs, because it would decrease the likelihood of having to do an additional study. Though intuitively this makes sense, it does not fit with the reality of what hap- pens in the field. "What we see coming back from our sites is that the more data you ask for, the more involved the trial will be," says Heyrman. "This is not con- ducive for either patients or science, and it results in higher costs." (For an additional example of how industry practices are not always conducive to patients, see sidebar, "Cookie-Cutter Informed Consent Creates Confusion, Not Comprehension.") She advises always keeping in mind exactly what is needed for the successful registra- tion and launch of a drug — probably in more regions of the world than just one. A simple technique taught to Heyrman by one of her mentors regarding clinical trial development was, "Ask yourself what exactly you think you're going to do with every single piece of data. If you can't answer that straightforwardly, the next question should be: 'Why exactly then are you even collecting it?'" For example, while working at a different company more than 10 years ago, Heyrman relates that one of the clinical trial procedures involved the collection of concomitant medications. "While we found out what concomitant medication was being used, we never col- lected the dose or administration route," she states. This changed when she went to another company that considered it heresy not to collect dose, mode of delivery, and frequency of concomitant medications, though she admits that in the absence of an adverse event, little, if any, data analysis was ever conducted. Much of the "collect-and-monitor-every- thing" mantra that has existed in clini- cal trials was driven by the legacy of Big Pharma deep pockets. Katz believes indus- try is shifting toward simplifying clinical studies, and this shift is being driven by smaller companies with limited resources making smarter decisions. "Because you have one shot," he says, "you have to pick the best choices for your primary and sec- ondary efficacy end points." That being said, during some of Krusinska's recent consultancy work she found the reverse to By R. Wright HOW TO IMPROVE CLINICAL TRIALS – SOME GOOD OLD-FASHIONED WISDOM Simplifying Clinical Studies Provides Spirited Debate 0 3 1 4 _ R o u n d t a b l e _ I m p r o v e . i n d d 3 0314_Roundtable_Improve.indd 3 2 / 1 9 / 2 0 1 4 1 : 4 1 : 1 0 P M 2/19/2014 1:41:10 PM

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