DIA 2018: Regulatory Convergence and Challenges of Precision Medicine



hello I'm sued at Parikh senior vice president and managing director is responsible for the dia Americas and I'm joined by dr. Sandra Queen deputy director and liaison to the EMA Office of International Programs OSI FDA that's an that's a mouthful sandy yeah what you really need to know is that she is a dia fellow and then also joined by dr. Peter marks director of the Center for biologics Evaluation and Research at the FDA we're really fortunate to be here at the DIA 2018 global annual meeting in Boston and dr. Klieger is gonna chair the EMA FDA question time diamond session tomorrow and dr. marks will be a panelist in the FDA town hall session the same day dr. marks is also participated in several other panels including a really live one lively one today on precision medicine thank you so much for joining us both of you the sessions tomorrow are gonna take a closer look at the EMA FDA relationship you know I'd love to explore that just a little bit what you think is important what you think is happening what's what's on what's what's gonna be happening in the future dr. clear in just a few words can you tell us what areas are gonna be covered in the session yeah this session this is actually a fun session because the session is all about the questions of the audience we give our speakers very strict instructions we have a panel of eight people four pairs of experts from four topic areas in FDA and the European Medicines Agency about we give them seven minutes in one slide to give an introduction so that we can get through everyone and then the audience gets to ask the questions that's pretty so it's a little bit of a free-for-all they get very nervous but then it's always fine that's great but the topic the general topic areas that we'll be covering our transparency you know data transparency pediatrics the third one is rare diseases activities and rare diseases and the fourth one is PD I should say Pediatrics did I just say Pediatrics why am i I'm blanking there's a fourth one and it'll come to me okay you know it's interesting because that's a great format did you give them a font size for their song manufacturing CMC quality product quality how can I forget that one seven minutes you know the font size if it's small that might might be hard yeah we're we're it we're tough we're tough one topic that we'll probably discuss tomorrow also is that is how both agencies can be engaged constructively and effectively at the sports development of new globally accessible medicines particularly in the area of biologics dr. marks what any thoughts on what might be discussed in that time you know I what I what I suspect people may want to talk about is how we achieve some regulatory convergence between the Europeans the FDA and perhaps other global regulators I think everyone's looking I think to figure out how they can streamline development programs and not having to have different regulatory hurdles in different jurisdictions is probably one of the ways we can help with that absolutely you know the challenge of course is that you've got a Europe you know European system and a u.s. system that have different points of origin and each system has grown up and built regulations around its original laws and yet we're looking at the same information for the most part so our challenge is to maintain scientific integrity meet the laws of our land but within that space find ways to come together so that we don't have a lot of redundancy that clinical trial resources including patients aren't wasted really I kind of a term I don't like to use lightly but that we can do things in a way that meets everyone's needs takes a lot of work I you know I understand that some of the first harmonization efforts started in the 80s of them fun yeah I think they did actually late 80s we started looking at ways to just very simple things like inspections sharing you know who inspected what when it didn't mean we were sharing the details of those inspections but we've evolved to a point where we are beginning to do more of that and then one of the other big areas that we've been working on for a long time since the 90s a little bit since the 90s but more more like about 15 years ago is Pediatrics yeah how far do you think we've come in that time that's that's a long time that's 30 years have we made significant progress enormous progress enormous progress and my observation is if you look back at the history of these engagements when people first started having conversations across Europe and and FDA and the u.s. it was well we're gonna just share information we're not sure about this alignment or harmonization we're going to share information and as the scientists start to talk to each other and realize that the challenges are the same it really feeds their creativity and their willingness to really roll up their sleeves and solve tough problems together and it's a wonderful thing to think we can't always do it you know sometimes we there are barriers and they may be regional practices regional laws really strong differences of opinion you're gonna see that but where we can particularly plan in advance for more streamlined development or almost we almost always have it an all-around win for everyone it seems like there was a point of real progress with the it's a mutual recognition of inspections and that that was it was that last year I think that was last year it feels like that was that was momentous that there was there was something real on paper that we actually mutually recognized this do you think anything will slow down due to brexit mhm alright place quite a role not related to this no no not related to this and this particular is this particular the MRA the mutual recognition agreement that you're talking about really speaks to sharing information about certain types you know it's a selected cohort but certain types of manufacturing inspections the ones that are routine surveillance inspections with the idea of trying to prevent duplication where we possibly can and learn from each other it's really good it's the first time the US has ever done this Europe has does this Oh has has had many of these they had a number of em Ras with other regions Canada for instance but we've not done it before this is new for us yeah one of things I've noted is just a great relationship between you all at the US FDA and your counterparts that has to take time to build that trust yeah it does and I think Peter would agree you know as we get to know each other and understand people's perspectives and where they come from it's just it's natural that people do that one of the things I always say is is it when you're a regulator in the given agency you're sort of alone I mean you have your colleagues around you but that's for the most part the extent of your peer group and when all of a sudden you find you have an opportunity to really engage with other regulators it's exciting it's a it's it really is you can you hear fresh perspectives and realize that well maybe there are other ways to look at this or consider that and it's fun yeah that's terrific I turned a different subject Peter we just came out of a precision medicine diamond session and it was really I thought was fantastic I learned a lot you know one of the things that you said that they caught my attention was that we don't know the answers we we don't have a path I can speak more to that in terms of you talked about the risk and benefit yeah so I think the issue here in precision medicine there are some things we know a lot about and other things we still are really learning about and then there are still other things where we just don't know where we don't know and and I think gene editing is an area where there's still a tremendous amount to be learned we've made tremendous progress and there's tremendous promise for helping patients but there's a lot we need to learn still and so our goal I think is to move forward help people move forward to get therapies towards the clinic and in the clinic and through Dotel licensure and at the same time making sure that we're keeping you know at least the appropriate level of safety that we need to see so that the benefit risk is maintained for these because there is we do recognize that for different diseases the benefit risk is different you know for very life-threatening diseases people will potentially and the agency understands that more risks can be taken whereas when you have you know diseases that are not life-threatening or less life-threatening or less severe it's it's less of an issue so we I think one of the good things from that session I learned is certain words we never want to use as regulators never use the word slow we never want to be slow we want to be we're cautious calculating but and that's where I think through the approach has to be we have to be cautious as we move forward but obviously want to move things forward as fast as we can I thought that was a really telling moment actually you know words mean things and having gotten to know you all over time I think first of all but FDA is so much more open and transparent than it's ever been in the past you all are you all have become humanized if I can that's not a rude thing to say part of that is knowing your own backgrounds do you mind if I ask you just a bit just quickly about your own backgrounds how you got to FDA I know that's not part of what we put in the questions here but I would I'd love to ask that question interesting so I I have I have kind of a strange background perhaps for getting to FDA I I started out as a medical scientist training program so I'm an MD PhD trained at NYU as a before I got my degrees then went on to do a an internal medicine residency hematology oncology fellowship here in Boston at Brigham Women's Hospital then after that went on actually to do work in industry for a period of time and work for a large large pharmaceutical firm and then came back to academics went back to academics and worked at Yale for several years helping to direct the clinical service in the Cancer Center and then about six and a half years ago came to FDA's deputy center director of the Center for biologics and I took over as director a little over two years ago so it's been incredible for me because it's been I find the FDA is a wonderful way to synthesize a lot of the things I've done in the past yeah and what I think it really speaks to is you've been in the shoes of the physician trying to treat a patient yeah and also the physician try to treat a patient and the person in industry trying to interact with us and understanding I think part of what I know is that if we don't speak an intelligible language at FDA the people who are on the receiving end don't understand it but I remember being an industry and sometimes trying to read tea leaves because we got FDA speaking and I just would rather not be putting people in that place that's terrific I think that's so important and the fact that you're able to be self aware the agency not just to you person to the self aware stuff where it's the agency is really important just to describe this background of you know could have come out because of the moving in and out of academia or regulatory but one of the things you've said when you were describing this session that you just were in was what I learned in this session yeah and I think what's great about some of these sessions for me is you know even when we come in is supposedly the experts right you know you're up there talking or you're putting all the experts up there you almost always learned something and you've just got to be open to that and when you're particularly and it goes back to my point about interacting with other regulators we all have things to learn from each other and they come up with the most surprising time sometimes no but that's but it's wonderful when it does it yeah that's terrific well I really appreciate your time I want to continue this conversation another time maybe you'll agree to it but for our purposes of our videocast in our podcast we keep them short and sweet but you guys are just you know you're warm you're you're normal people and you're doing great great work at FDA and I've made them laugh by saying that so I'd love to end there thank you so much thank you thank you very much come to our sessions

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