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Executive Summary

Participation of FDA advisory committee members in end-of-Phase II and pre-NDA meetings with the FDA can be very helpful to the eventual progress of an NDA application, Merck, Sharpe & Dohme Director of Regulatory Affairs James Molt, PhD, told a Regulatory Affairs Professionals Society (RAPS) meeting Sept. 19. "If you're having an end-of-Phase II or a pre-NDA meeting, suggest to FDA that an advisory committee member be present," Molt recommended. "This represents a third party . . . and having the advisory committee member agree to, for example, Phase III ]protocols[, will ensure that at least one member of the advisory committee thinks that you've studied the drug properly," Molt said. The MS&D regulatory affairs exec discussed a blueprint for approaching an advisory committee meeting. He disclaimed his comments as his own and not representative of an MS&D approach. However, based on MS&D's recent string of favorable reviews at FDA, the observations of the firm's regulatory staff probably deserve close attention. Molt also suggested that companies attempt to include participation of an advisory committee member "if you're going down to the agency to discuss (BRACKET)a] Phase IV program." Molt explained that the presence of an advisory committee member at the meetings "maintains some sort of continuity as you proceed with further development of your drug. They have more or less witnessed an agreement." He added: "That person is there to say, 'this is what we agreed to and this is the way we decided that the drug should be developed.'" FDA also appears to be looking for a drug review process that would bring about more exchange between advisory committee members, FDA reviewers and NDA sponsors. For example, the recent review of ICI's Dipravan anesthetic in late August included FDA reviewers, a large contingent of representatives from the sponsor (on-site and by hook-up back at company's facilities), and advisory committee members called together for an intensive review of labeling for the application. FDA is calling the intensive sessions "NDA Days" ("The Pink Sheet" Sept. 4, p. 3). Asked whether an advisory committee member's participation at an industry/FDA negotiating session would preclude the member from serving objectively on the panel, Molt maintained: "I think not. I think what they're doing is just saying early on, 'Yes, you're studying the drug right . . . You don't know what the outcomes of these trials are going to be yet, but certainly the trials are properly designed to ask the appropriate questions. And if [the data show safety and efficacy], then I can go ahead and approve the drug.'" The regulatory affairs director emphasized that a company's preparation for a panel meeting should begin well in advance of the session. "It's a good idea to have knowledge of the advisory committee even if you don't anticipate having a drug come before it for many years. It's a good idea to attend all the meetings to get an idea of how the advisory committee and FDA are run, and it's also a good way to gain intelligence into what's going on with your competitors," Molt noted. Molt suggested that companies "learn about the committee members themselves, know what their interests are, know what their publications are. This certainly will help you anticipate how they're going to react and the kinds of questions that will be asked of you." As the panel meeting day approaches, firms should "prepare and submit a comprehensive yet readable and understandable background package," Molt said. "You're in a much better position if the advisory committee has read some background on the product rather than coming into the meeting cold . . . Understand that these people are busy with their own duties and can't spend a lot of time trying to figure out a complicated or convoluted package," he continued. A couple of days before the meeting, FDA typically drafts questions for the committee to address. Companies "should anticipate [from their previous experiences with the FDA reviewers who are working on the drug] what kinds of questions these will be from the agency, and put together your background package accordingly because you want to focus in on the kinds of things FDA will be asking the advisory committee," Molt pointed out. The firm should also anticipate what questions the panel and FDA will ask at the meeting. The company "should determine whether or not your own personnel are capable of answering" questions from the floor, Molt suggested, "or [if] it [would] be better to bring in some outside consultants." Consultants, Molt said, "are generally a good idea" even though "in some cases you may invite a consultant who will just sit there and never get called on . . . it's far better to have that person there and not even answer a question than have a question asked that no one can answer." Molt suggested that companies bring approximately 15 people with preclinical, clinical, statistical and regulatory expertise to field questions at the meeting. Molt also recommended that companies rehearse prior to a panel meeting. "From the period that you know you're coming to the advisory committee until the meeting day, you have to rehearse and . . . have your presenters stand up on their feet and give their presentations over and over. You have to have people in the audience act as advisory committee members and FDA reviewers and ask tough, hard questions. You have to have backup slides, Molt told the RAPS group. "Any questions that you anticipate being asked you must have the answer to. You can't over-rehearse and you cannot be overprepared for an advisory committee meeting." Molt also presented a strategy for addressing the advisory panel at the meeting. "You need to communicate [your data] effectively so that people can understand it and can process it and remember it," Molt advised. "You need to have effective, clear slides, . . . your presentations very clear and understandable . . . and straight forward with enough data to get the point across without being overcomplicated," he added. "The advisory committee members for the most part are going to be processing it on the spot . . . and making their judgments based on what you are saying on that day," Molt noted. Presentations, he suggested, "should be scientific. It's obvious that you are an advocate of the drug, but you should not come into this meeting with an emotional component." Molt also suggested that companies be candid when presenting to advisory committees, but also be circumspect when responding to questions from committee members. "You should be open," he advised. "You certainly can't give the appearance of withholding information or being evasive. The opposite of that, though, is answer the question that's asked, and answer it concisely. Don't expand upon what they are saying because you think that the questioner might want to hear more. This may get you into territory that neither you nor the questioner wanted to get into, and it can get you into trouble," he said. Molt further advised against arguing with the committee. "We all know that reasonable people will disagree on interpretations of scientific data, [but] there is no point, especially in this public forum with little time, to even get into a scientific [argument]," he said. At the conclusion of a committee meeting, "follow-up action is necessary," he noted. For example, "You should obtain the official transcript of the meeting. The testimony contained in that transcript can be very useful to you in your subsequent dealings with FDA."

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