FDA 101



first what I'm going to be doing is presenting the FDA 101 section which will be a broad top and there were a broad overview of topics that may be important to patient advocates okay so this is what our campus looks like an aerial view I think that most of the FDA offices are now located at the white oak campus in Silver Spring Maryland and this is the FDA's mission and regulatory philosophy there's two parts the first is protecting Public Health by assuring the safety efficacy and security of human and veterinary drugs biologic products and medical devices and advancing the public health by helping to speed innovations that make medicines and foods more effective safer and more affordable and helping the public to get the accurate science-based information they need to use medicines and foods to improve their health this slide in the next slide shows all the significant days or summer most of the significant states in the history of the food and drug law history it's important to note that major lawmaking activities have focused on the FDA repeatedly over the past century as a result of public health disasters and I think you can see the last major change was in 2009 when tobacco products the oversight of tobacco products was added to FDA's regulatory authority everything that FDA does is dictated by law and can be found in the code of regular several regulation okay so within FDA there are three main centers the first one is the Center for Drug Evaluation research and it's awesome in the cedar cedar regulates the over-the-counter and prescription drugs including biological therapeutics and generic drugs this work covers more than just medicines for example for eye toothpaste antiperspirant dandruff shampoos and sudden screams are all considered drugs the next Center is for biologics and it's called the Center for biologics Evaluation and Research also known see per Sheba regulate biological and related products including blood vaccines allergenic tissue and cellular injury therapy biologics are derived from living sources to human animals and microorganisms and they're not easily fully identified and they're often manufactured using biotechnology the last Center we won't discuss very much but I will raise it periodically throughout my presentation to tell you a little bit about spices but we're not experts in that area so it's it's pretty different from the biologic and the drug evaluation process and that is the Devices and Radiological health CDRH and CDR charge regulates a broad range of medical devices including complicated high risk medical devices like artificial hearts and relatively simple lowest devices such as tongue depressors and then anything that falls in between such as sutures these are some of the other offices that are located with the FDA including the office that Selena and I work out of the office of Health and constituent affairs and our office works with external stakeholders such as yourself and internal stakeholders such as the Centers that I just talked about many of these offices that are listed on this page and others that I didn't include are there to support the three review division what I'm going to do now is I want to make something clear when I speak of a sponsor I'm most of the time I'm speaking in terms of a drug manufacturer such as a pharmaceutical con and when I mention investigators these are the clinicians who actually conducted clinical trials and I'll remind you of that again later on in the presentation so basically what happens is a company the sponsor will come in to FDA will submit an application for biologics and drugs it's called an investigational new drug application and for devices that investigate device exemption application and by regulation the IND is the process under which human trials of investigational drugs are conducted they cannot be conducted until FDA has approved the IND and drugs and biologics often follow the same regulations and investigation of biologic large molecules with biologics which are large molecules are often called they go by investigational new drug application in the same term because as I said the biologics are large molecules them drugs themselves are smaller molecules but there are all drugs essentially and then like I said four devices there's the IDE the ID the IMD's the IDE applications are reviewed by FDA before any clinical trials can begin I think that this is important for location happens to understand the types of meetings that take place between sponsors again that's the drug company in FDA there's the type a meeting he doesn't want meetings means that there's an issue that is actually going to solve drug development if they don't remedy the situation and so FDA usually responds within 30 days and sets up a meeting the type B meetings are probably mostly justification advocates because these are the pre-ind meetings the end the phase 1 meetings etc as you can see on the slide and the idea of this is to look at the data that was obtained from the clinical prior the prior clinical trial and to see if there's any changes that need to take place in the trial design employing sutures need to have a discussion about recruitment of patients that that's an issue these occur within 60 days the types a meeting is just any type of meeting that the sponsor needs to hold with FDA then typing in tight to me okay so we've gone through the IND and the IDE and we've gone through the clinical trial so now the company the sponsor has finished trials and what they do is they pull the data together and they analyze the data and then the sponsor submits the application for approval of the tests that would be amended a or b la now the that is for the drug for the devices as I said I would touch on it a little bit there are two types of devices missions there's the pre-market approval and the premarket notification 5mk and basically the pre-market approval is determined if the if the new device is determined not to be substantially equivalent to an approved device then it must go through clinical trials to get approval if it's a premarket notification in the pre-market notification if the new device is determined by fda to be substantially equivalent and that is there's a predicate then improve 'el of the device yet if it's equivalent to an approved device FDA will provide clearance for the device to go to the market and there will not be any clinical trials that will be necessary so now the applications have come in and what happens is it goes to the center where it's appropriate whether it's a drug or biologic and then once in the center it goes to the review team that have expertise in this area and what they are doing each member of the review team is reviewing the application based on their expertise and again they're looking at safety and efficacy as well as the proposed use and if the benefits of the drug outweigh the risk and how appropriate the labeling is etc I mean I'm going to talk really briefly about into the members of the team the review team the first one is the project members and the cuz there are two primary roles for the project member and that is facilitate the review process and to be the primary contact for the sponsor every team has at least one medical officer and they review all the clinical studies and again they're looking at safety and efficacy they're looking at the protocol design endpoints they are also looking at the clinical investigators who supply the clinical data to make sure that they followed all the procedures properly so they're going to be looking at anything that's clinically related now you have the pharmacology and toxicology specialist and this team reviews all the non clinical meaning the animal studies which take place prior to the the IND application the animal stays are usually done on two species because the a drug may affect one differently than another and with the pharmacologist historian is looking at the data to determine there is pharmacological action and toxic effects of the drug as it's related to adduce what they are as well as looking at the reproductive and seed oral sex and again these studies are done prior to the IND and the statistician what happens is the sponsor will submit the application with raw data from the investigators and then they will run the sponsor will run the analyses they will send that data to fda yesterday's debited statisticians will review the analysis completed by sponsor they will rerun those analysis and sometimes they will also conduct additional analyses and the next slide should show clinical pharmacology and thought biopharma civics and what these reviewers are doing is looking at the molecular level data they're looking at the background ADME studies and that's the absorption distribution metabolism and the information the bioavailability bioequivalent studies etc and the next slide are the chemists and the biologist and microbiologist and these reviewers evaluate the drugs substance product in the areas of components and composition and the factoring controls batch formulation they want to make sure that all the controls were in place that the batches are produced up to the qualities are supposed to be at and it's all consistent across batches etc so now what I did was I reviewed the the IND s then we went through the clinical trials then we went through the application process so now the application process is with FDA they're going through the review team is going through the application sometimes what happens is there's a complex scientific technical or policy issue that FDA wants some expert opinions on and they will pull together an advisory committee meeting they'll pull together a panel for meeting and for example an advisory committee maybe you need it for providing FDA advice about whether or not the you have to weigh the risks and benefits of the new potential treatment for a disease now an NDA or a B al for something in the area of myotonic dystrophy that requires an advisory committee meeting would might come before the peripheral central nervous system drugs Advisory Committee so now this is the peripheral and central nervous system drugs Advisory Committee and the purpose is to again to look at the data to review evaluate the data concerning safety advocacy and the committee is basically made up of a core of non-voting members and includes the chair and the members are chosen either by the commissioner or designee among societies who are knowledgeable in the fields of neurology nor neuropharmacology nah neuropathology Otolaryngology epidemiology statistics and related specialties and the big thing to remember about this is that the panel puts forth recommendations to the commissioner but these are strictly recommendations they are non-binding also the other important thing to note is that most advisory committee panels do include a patient rep and I believe scaliness and we'll be talking more about that in her presentation now we're on to the approval process a standard review takes ten months and standard reviews starts after FDA has received the NBA or PLA application and decided that the applications file abble meaning that it's complete all sections must be received by FDA before the clock starts before they will start to review the application you will see here the FDA will approve an application after it determines that the drug meets the statutory standards for safety and effectiveness manufacturing controls and labeling FDA's required to exercise scientific judgment to make these determinations and FDA views the one makes its views on drug products and classes of drugs available through guidance documents recommendations and other policy statements and these can be found on the FDA website now besides the regular approval where FDA does have several mechanisms to speed access to promising drugs to treat preview to treat serious diseases and to fill unmet needs so the next slide with the four types of mechanisms accelerate approval Priority Review fast track designation and breakthrough therapy designation even with availability of drugs that treat serious diseases and unmet need are in everyone's interest especially when the drugs are the first available treatment or the drug has an advantage over the standard treatment the existing treatment today 34 mechanisms sponsors need to apply those mechanisms and they can apply to more than just one they can try to one two three they can apply to all for the accelerated approval process the accelerator parole asked for earlier approvals of drugs to treat serious diseases and that still an unmet medical need based on a surrogate endpoints so that's one of the prime issues with on the accelerated approval it's going to be based on a surrogate endpoint and circuit endpoint of a marker and that is a laboratory measurement or a physical sign that is used clinical trials with a new direct or substitute measure measurement that represents a clinically meaningful outcome such as survival or symptom improvement by going through this mechanism of accelerated approval and using an endpoint instead a surrogate endpoint instead of a longer-term endpoint such as overall survival is really shorten the amount of time that's required prior to receiving FDA approval the other thing to note about accelerated approval is that it's only approved on a condition and that is that post margining clinical trials known his face for clinical trials must verify the clinical benefit so if if there's a clinical benefit that's is that the phase four trials the confirmatory trials show the FDA the grant traditional approval if there's no clinical benefit that they has the regulatory procedures in place to remove the drug from the market or from the indication on the way sometimes these drugs may have three or four indications and maybe two or three of them are the regular approval and one may be the accelerated approval the clinical trials with the not clinical trust the confirmatory trials would be on the accelerate of the approval and it would have to prove clinical benefit it doesn't mean that if it doesn't that the drugs are come out to market it that's why I said either the drug will see the move from the market or in the case where there's more than one indication then just that indication would come off the label the next slide is Priority Review and this is really for our diseases where the the treatment is inadequate or just doesn't exist and so instead of the ten month review you got it down to a six month review is designated again to facilitate the development next about the review of drugs for serious diseases and so on that medical need and the the big thing with fast-track what makes it go faster is that they allow what's called rolling review with the regular approval as you may remember I said that the FDA cannot start the review until they received all parts of the application they have to have a whole application some of them here with fast-track as they get completed sections from the of the MVA FDA will start reviewing those completed sections and the next slide is breakthrough therapies and again it's for serious or life-threatening disease and what happens here is that it's the sponsor notices that the drug is demonstrating a substantial improvement over existing therapies or one more clinical significant endpoints and that this is seen early in the drug development process they can apply for a breakthrough therapy that would help get this drug onto the market faster Samantha lien is going to talk about patient advocacy yay great thanks Deb it was a great overview I think it's really helpful to have a basic understanding of the agency especially when we're talking about the next topic which is really focused on patient engagement and as Deb mentioned we worked within offices of FDA's office of Health and concision Affairs which we call bys acronym polka and today I plan to answer the question that many patients and patient advocates come to visit us with how can we get connected to the agency when I got here a little over two years ago I learned quickly that this agency speaks a distinct language and sometimes it's not easily digestible what I also learned was that if I want to help translate that language I was in the right office you the patients caregivers are in fact our offices primary stakeholders like that's what I'm trying to say is that once you learn how to speak FDA your voice gets louder for sure health understand where we fall in the agency I want to look at the organization chart that is located within the office of External Affairs and that's within the office of the commissioner and our primary job is really to connect with informal relationships with various key stakeholders in communities okay their office is comprised of two teams we have a health professional team on one end and the patient liaison team on the other and although we are two distinct teams we often work together the health professional team really worked hard to make connection with with the health professionals and health professional organizations now the patient team's primary stakeholder of course is you the patient or the Advocate and care giver and I can't tell you how many calls we get from patients who just want to share their experiences or just want any help that I loved one or even get more information on drugs that they happen to learn about on the news so on a daily basis we filled a lot of types of patient increase and I can say with certainty that each of these calls is such a unique opportunity for us to make an impression on the caller and the way I can describe this office if I had to sum it up of three phrases is is that we really listen because sometimes we get calls with someone who's just so frustrated that they all don't want to do is be heard and that's exactly what we do we listen we educate many patients in patient groups need a better understanding of what we do so we also have to serve as quote unquote translators describing how to agency works and how the drug development process works in simple terms and as a result people become more familiar and engaged with the agency's activities and they start asking the right questions so education is a really a big component of what we do and lastly I think a big part of what we do is advocate as I mentioned the reviewers within each Center and division are so focused on their processes that there's often a disconnect between them and the outside world well one way that our office has been described to as a bridge is a bridge that links the FDA reviewers with the patient community and I like that model but another way that I think of our office is is a string so to explain we would be the string but connect you and the center's reviewers by providing information which would be the sound that travels through the string using various communication vehicles and that would be the vibrations on the string and initially I thought maybe be interesting to use probably two iPhones just to make more current but I didn't think 3G network was visually compelling so I left it at this and I think you guys just adjust okay so how does our office bring you to the medical product development process there are so many ways first off there's the FDA patient for patient webpage about three years ago our office worked very hard to get a patient presence on the FDA website which historically has been viewed as an industry centric entity but really we've been engaging at patients for a really long time our website however needed to be able to showcase that so as the results what exists today is a for patients research page so strictly devoted to patients and patient advocates and it really serves as a as a one-stop shop to learn as much as possible about the regulatory process the URL for this page is at the bottom of the slide and the bottom of the next slide as well see so on this page you can get all sorts of information you get information about upcoming agency sponsored meetings public meetings watch recorded webinars with FDA experts you can subscribe to our bi-weekly patient network newsletter which I highly recommend it is an excellent resource for getting announcements in a timely way you can also act as clinical trials to learn about clinical trial processes and find Charles that may be right for you in the event that you or a loved one doesn't respond to current approved therapy for various reasons people learn about treatment options so sites really does a good job of explaining the drug and device approval process so it's really a fact that you want to check out okay so during the year our office with many meetings with patients and their communities and sometimes connects them with their review division so they have some safe to face time with the FDA experts these meetings extremely valuable to us because they're unique opportunities for us to really hear from the patients firsthand the time to share concerns that provide clarity to us of any misconceptions about a condition it's also time for our experts to relay information that will help better understand internal processes and as I mentioned earlier we continuously feel calls from patients on all sorts of issues it's really a big part of what we do in the office so I'm putting a plug that if you have any questions for us I listed our email address at the bottom and I can tell you to please email us now I want to touch on some other ways that are really effective to connect with FDA so as you know FDA the regulatory agency we publish using a Federal Register rules and guidances that establish or modify the way we do business in them we often ask for comments so clearly the business does not mean if I chance in the back to you their forms with public input for example last year in November we issued a FR notice which we call short for the Federal Register notice of our notice that ask the question what more can the agency do to obtain the views of patients during the medical product development process and how can we further consider patients perspective during regulatory decisions as a result we received many comments from organizations and individual patients and we're vetting those right now as we speak if you need more information before patients website on FBA gov is a great resource and you can get more information about that as well okay so another very effective and directly to show yourself in opinions is through meetings for example the advisor 20 meetings at dimensions there's devoted time on the schedule for us to listen his views and opinions from the public from patients as she also mentioned you have the sponsor in the room if these staff you have scientists statisticians and others who are there who are available to hear what you have to say so it's a unique opportunity the FDA also holds periodically meetings to gather input from various stakeholders including patients and these are what we call parts of these public hearings their opportunities for broader public participation and comment on various topics all right the patients of the drug development meetings that yet another way for the patient voice kitty heard the agency begin these meetings in 2013 to really get a systematic approach on obtaining the patient perspective of certain diseases and treatments how did it get nominated well there's a public process if there wasn't nominations that I believed last year there was a Federal Register notice that was published seeking nominations for 2016 and 2017 I believe that is the end of the five-year nomination process for submitting comments or submit for nominating topics the outcome of these meetings is an agency report it's called a voice of the patient and it's really a detailed center of each of the meetings and a document in the patient's own words what matters to them most in terms of the impact of disease and treatment approaches so it really is a piece that informs the agency as you can see the next meeting scheduled for April 2nd and is on breast cancer and again you can learn more about this on the floor pieces website that was listed ok now the real focus of my presentation FDA's patient representative program this program is managed by our office our office of patient liaison team and really is such an exciting part of what we do if there needs the patient voice directly to the table and I mean literally to the table here's a group picture of the patient at the site and I've seen last year from our workshops all right so in order to help you better understand where the intersections lie between a patient reps and the development process I think this graphic is pretty useful the two bottoms there's two squares at the bottom of the slide and one square the one on the left indicates that the patient reps can serve as consultants refer to these as the visual finance because if it brings the patient into discussions early on in the process and connects them directly with the drug sponsor and the FDA reviewers in a closed meeting and this is typically done in a comp as a conference call more commonly we use the patient reps as panel members on the Advisory Committee meetings and that's the square on the right and that's typically during the final stage of subjects okay so because patient reps are temporary voting members on a panel and there's usually only one patient rocker meeting although that hasn't always been the case there's generally open to the public like division all privates patients have a critical role and that is to share with the agency their own experiences and those of their communities so that we can make the final decision on whether to approve a product or it should change a product or to completely pull it off the market so along with other specialties in the panel like the physicians and scientists that I mentioned but they should have to really bring a unique perspective to the table incorporating constructs into the process extremely values a part of what we do in our office is responsible for making sure the right patient patient rep is assigned to each meeting and last year alone we helped identify and apply a chance for about close to 60 divides your committee meetings okay now just to touch on the divisional assignments briefly I see a review staff can call on a patient up at any point earlier in the process to get their perspective and this is extremely important part of the process as well because it's another opportunity to engage of the patient and incorporate their input before a final decisions made now I should mention that section 11 37 of FDA safety and innovation Act known as padega which recognized the value of patient input in the entire drug development process was signed into law in 2004 has been including patients in their processes for years and I just want to take a detour and talk about the timeline so these are some of the historical milestones of how these involve patients in the past the initial milestone was back in 1988 when the FDA formed our office which was originally established to work with patient advocates focusing mostly on HIV and AIDS community and as a result in 1991 the first patient ride was called to serve on the antiviral drugs Advisory Committee for HIV in 1994 transportation advocates were recruited into the patient program which then expanded to include patients and caregivers of serious and life-threatening diseases and in 96 of patient reps received voting privileges as members of SDS advisory committees the role of patient reps expands to serve as consultants for divisional assignments in 2001 and about 10 years later the agency created a patient network network which I refer to as the for patients website but it still can be referred to as teacher Network a year after that as I mentioned vajayjay section 1137 came into effect after which a working group has started to develop the procedures for implementing 1137 across the agency and finally in November of last year we issued a Federal Register notice as part of 1137 to capture patient advocacy group perspectives on suggestions for how FDA can increase improve patient participation earlier and the process and I think I mentioned that earlier as well okay so back to the Patriot program how how do we pick the patients how do we select them how to where identify them there is a recipe we want an effective patient rep and one of the things we really look for is is the patient hat or a patient web has to have personal experience with the disease or condition that could be either as a patient or as the primary care giver and ideally we do like to see actual patients on board but we understand this can't always be possible say for example if it's a pediatric condition or if the condition that so debilitating that the caregiver really is only the best option for relating experiences the peak of the major community awareness is also an important part of being patient rep we really want someone who's active in their respective organizations and really understands the issues that their communities are facing we also would like someone who's been a political in the end of justice who doesn't necessarily need to be a scientist but could be able to understand basic scientific principle finish in issues because some of the information that is presented is complex and decisions do have to be made using that information so right now we currently have about 190 patron reps and they're savvy they're smart influential other communities and because of this there was a risk of conflicts of interest which we as needed be paid very close attention to so bringing the rep on board who's minimally conflictive is what we need for and last but not least we want someone who's got great communication skills this is so important in serving especially when you're a large room full of experts it can be a little bit intimidating so here's a list of some of the conditions of which we have about 120 120 conditions on right now represented represented by 190 Pynchon reps and we often have multiple multiple folks on the condition which is why that numbers off conditions include a HIV cystic fibrosis Parkinson's and muscular dystrophy and obviously I wish we could say that we have representation for every medical condition however we do not and the reason for this it's not because we don't have a manpower or any other reason it's simply because we recruit based on meet the medical product it's not coming up for review and all will be discussed at a meeting there really isn't a need to need to recruit at this point however what we do is work really closely with the reviewers and the division to try to predict and get a better sense of what's the Kama had so that we can plan ahead and recruit the appropriate person earlier in the process and of course this takes time but once you're on board we begin the training so this is how we train and keep the patient reps active and engaged with other ways to serve with each mutation that we make it a point to really emphasize the significance of the program and hold an individual FDA 101 which basically provides overview similar to what they have had provided now I mentioned earlier that serving can be intimidating for some so we've learned that by having more seasons Brett's share their experiences it's really helped those who are unsure of their role and new at serving becoming more informed and in turn confident at serving it's part of the sharing information we go into detail what the room looks like with the panel members are using the meaning he's going to be attending outside of that then those are really key pieces that are valuable to them our office office regular webinar strictly for patient reps so that they can continually stay engaged because often times during their four-year term they don't always serve so we do try to keep them ready to serve by consistently feeding them information then of course the approximate length about the for patient site which we created to be a resource for patients and that includes the patient map finally each year about the middle or end of summer our office holds the annual t-strap workshop which is strictly for patient reps there it is only really includes the patient reps who have just been brought over the last year it's really a great one and a half day excellent immersion course really that's highly interactive the closed meeting so they can ask whatever questions they want and we feel comfortable sharing information as well ok so what is the value provided to FDA what is what is important why is it important to engage patients in a regulatory process I mean why do we why do we do this at all and there are many because the patient point of views are simply mandatory issues and concern the issues that concern you the what you're the ones living with the condition this is one that is critical and there's a engage to shape the outcome of our decisions secondly it brings the human element into the picture after all the patients are the end users patients bring diversity of opinion and viewpoint and experience engaging patients makes them feel as though they have a vested interest in the concept patients bring opportunity to increase quality of life such as reporting adverse events from the product is already out so in the end office is committed to really creating a patient and patient advocate community of ambassadors and educators so that you can continue to share what you've learned with others with that in mind dev and I will pick some equipment that we have tied we do have a few questions from our participants the first question is kind of what's an average amount of time that it takes to get a drug approved I think that the average time is about 10 to 15 years and that's all the research that that includes all the research that's done in the laboratory before it even gets to humans before it even gets to animals so it does it is a timely or not family but a very time-consuming process with the caveat that you have those other accelerated ways of getting Africa's those accelerate ways are after it's been through the initial development through the animal studies and your into the clinical trials and you see that the drug is for a very serious disease and there's a need to fill an unmet needs and that's when those four mechanisms would come into play so but usually we say about 10 10 15 years so what is more important to the FDA safety or evidence that the drug works they're equally important and we follow them through the clinical trials safety and fix efficacy we follow them in post market studies the confirmatory trials we follow them once they're on the market we keep track on the effectiveness and safety of these drugs through the MedWatch system which we didn't talk about because we just had so much today so they're equally important is it faster to get drugs approved in the US or in Europe the last piece of data that I saw was just looking at oncology drugs and it was put out by the Friends of cancer research the study that they did and actually the drugs are getting the proof faster in the u.s. right now but and I see that this is a question further down about working with the EMA and others FDA works very closely with the EMA they work closely with health Health Canada and Japan in fact I know in oncology they have monthly meetings where they discuss what applications are coming up etc so that because now a lot of the data the clinical trials are being helped growth in global rotations and so that data is being used by all of us you know me and Mary FDA Health Canada etc to approve clinical trials so we really do keep in touch with what's happening and any kind of decisions that are made and just to add to that most recently we did have an EMA fellow come visit one of the things were really focused on right now is to learn from each other's processes especially on how they're engaging patients and their processes which I think they're just really starting to take notice and they've learned a lot coming here within that two weeks it was quite an intense two weeks that fellow did develop a voice blog and it's on our website if you wanted to read more about her experience here and I think that would be really helpful for you to understand you know some of the lessons learned a couple years before Selina got here we had a group from Japan who came in with Han flavors and we talked to them about what we do and they talked to us about what they did over in Japan and kind of continuing on that does does approval from the EMA European Medical Association or you know Japanese Medical Association or any other foreign bodies does approval over there make gaining approval in the u.s. from the FDA any easier or harder or does it affect it at all like I said a lot of the same is used to approve a drug here in the United States and in other countries but the United States to prove you know they they have to still see the raw data they have to see the evidence just like the other countries they insist on seeing all that data as well and doing their own analyses so I think that I think it yeah yeah yeah exactly so I think that you know every country has to make sure the drugs are safe effective for their citizens so of course they're going to take a look at the data intensely we do talk to one another where the drug is being approved often time you know in the EMA oftentimes USA will know about it and they will have had discussions on why the EMA is proving it versus you know FDA a lot of times it doesn't really have an impact because FDA can't approve the drugs unless the application comes in and so sometimes companies sponsors will submit applications over in the EMA or Japan and they'll approve the drug and we don't approve it and people will say well why are you approving it it looks great over there and it's because we didn't get an application for whatever reason the response of chose not to apply here in the United States so and that's something that a lot of people don't know about we've have to have an application we can't just look at a drug study and say oh that looks good we're going to go ahead and prove it let's see um how how different should the approval process be for rare disease drugs orphan drugs versus kind of blockbuster our mass use drugs the rare drugs but what happens is you when you have it disease where it's rare you have fewer people that get into the clinical trials and so I'm not an expert in the rare disease area but we do have a department in office of rare diseases we have an office of orphan drug product and what FDA does is tries to provide some financial benefits to sponsors because a lot of times sponsors they need that incentive to make the drug because they know they're not going to make very much money from the drug deserve fewer people who get disease so FDA tries to provide that that kind of incentive as well as there are other ways in the trial design that are that have taken place whether they accept fewer people or they make other exceptions FDA is very sensitive to these groups and it's very trying to work with them as closely as possible to get these because that definitely an unmet need but a lot of these diseases mm-hmm why are there so many different agencies and departments within the FDA well because each office is so different it's for example with the Center for biologics biologics are very unique you need to have experts who are reviewing the applications who are experts in biologics and same with devices etc then you need all these support agencies and I or a offices not really ATP as I put a slide up before and talked about the other offices like you have an office of compliance and what compliance does is it keeps track of clinical trials and make sure that the investigators are being compliant with the regulations the statutory laws that say how the the trial should be held medical office of medical policy again they all these offices support the three major centers of cedar cedar and CDR a office of minority health of course is to make sure you know and sponsors are coming in to talk about clinical trials and recruitment that they think about these minority groups and make sure yeah that they are identifying the same thing with women's health and pediatric therapeutics you know that office is Jerry needed because in the past we were afraid to do clinical trials on children and we're now realizing we have to do clinical trials on children children are not just small adults they really do metabolize drugs differently and they have a different impact on kids so as I said there are a lot of different offices and they're all they're not totally Navy but most of them are there to support so the three centers to help them do their job and it's just after that although they are separate centers and it looks very like we're divided we work well together on various issues there's a lot of cross-cutting issues but I think one of the things that I mentioned is the food age 1137 which is how to involved a patient earlier on in the process and we have every one of these centers really thinking about how it ways to do this once we get the formalized and organized the comments that we've received we will enter them into the Centers and really think about how the best you know what's the best next steps are so you know there are situation situations as we go to meeting those representation across the board so it's not like we fit in our silo but they're adduced it does need to be structure and I think that was the set point and we and they're often products that come in for review their combination products and that's when the two two or maybe even three to the Centers worth together think in oncology I'm sorry I specialize in oncology which is why I keep bringing oncology up is the idea of these in Diagnostics with the treatments being approved together there was all of the work that's being done in the genetics field as camp of cancer and finding mutations and treating them accordingly and so there they all appear separate but I can't tell you how many meetings occur every day where someone from almost all the different offices are in the same room working together so they have expertise in other in different areas last question are we have a our patient advocates are going to be in Washington DC this September for the mdf conference and so somebody asked is the FDA open to the public can my family visit when we are in DC later this year the the advocates we often have the Advocate groups come in and we set up a meeting ouka sets up the meeting we try to invite people from the different offices who would be interested or who have products that are just to the advocacy group or whatever I know we were sitting here discussing this before we started I don't we do make special arrangements for advocacy groups and different groups to come in I've never really seen a family come in where offices here and there's really not a whole lot to look at it umm.we and then we also there's a lot of security because as you can imagine the campus is filled with proprietary sensitive information okay well we would be glad to sponsor you guys to come on in it would be great well great well thank you ladies so much I'm sure you know on behalf of everybody Thank You Selina and Deb for for taking your time and teaching us about the FDA this has been really helpful and really important to the myotonic dystrophy community as we kind of move forward with our first-ever clinical trial and just it's really good to know about the whole regulatory process this has been really helpful so thank you both and thank you everybody for participating in the webinar Thanks thank you the opportunity

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