Transcript for:
UCSF Catalyst Program Workshop Notes

second half hour and so um great how about if I share my screen let me just uh here we go just a second yeah this will probably take a couple minutes anyway [Music] um okay great uh can everybody see my screen or I mean can I ask one person can I can yes looks good Charles okay thank you very much okay again my name is Charles Hart I'm with the Catalyst program and what I'm going to do for this first half hour is I'm gonna say a few words about the UCSF Catalyst program and then I'm going to say a few words about the target product profile and the role it plays in um Healthcare product development um and industry government I'm going to um say a bit about the Catalyst summer internship program and I'm going to finish off uh talking about the medical fields of the target product profile which is the first set of fields that all the interns are going to um create this summer and then I'm going to introduce Ray manowski who's gonna uh give the second half of the workshop tonight and so actually I first want to start off off by thanking all of you that have joined the call in particular I want to thank the other uh Catalyst program staffers Nate proo rupar ramam Mory and SOA Shaw that are on the call and uh you're going to get some of you are going to get to know each of them very well and others sort of um peripherally peripherally I want to thank all the Catalyst a principal invest tigators that are on the call for um their projects and and volunteering to participate this summer I want to thank everybody from the UCSF office of innovation Ventures especially the case managers the business development case managers that work on these same projects with the investigators um and finally I and I want to say most importantly I want to thank all the Catalyst interns that have volunteered this summer um we hope this is a a worthwhile um Venture for you we appreciate you participating um if you have any questions or suggestions or comments you can let me know or um the other Catalyst staffers and then for any of the Catalyst industry advisors joining I want to thank you as well especially okay so I'm going to move on and so the Catalyst program is one of six programs within in Innovation Ventures at UCSF which is basically the tech transfer office at large and uh I'm not going to go through all these other programs but they have to do with commercializing um inventions working with the investigators uh entrepreneurship um and the like we're actually part of a larger Network um of efforts involving entrepreneurship and Innovation both on the UCSF campus as well as other uc's and the the Bay Area at large and actually you know regionally and um nationally and internationally as well the Catalyst programs focused on helping UCSF professors and postcss and trainees and staffers and anybody at UCSF that has an idea an invention or discovery that could that could possibly Advance along the translational path to commercialization and we have really have three different um uh sort of programs the Catalyst Awards which again um provide seed funding and um advice and mentorship to the awarded uh principal investigators we have the Catalyst Launchpad that's focused on entrepreneurship that Nate's going to talk about in a couple weeks and we have the summer internship program that we're here for today that have uh students and trainees from both UCSF as well as other local institutions and other actually some that aren't so local and the focus is to create Target product profiles for the Catalyst award teams and the you know the overarching goal of catalyst is to help these projects advance advance along the translational path and hopefully exit as you know Partnerships with industry or startup companies or into other accelerator incubator type programs Catalyst approach projects again across all aspects of healthcare product development primarily drugs tests devices and digital and our advisor span our advisors I already said this absolutely key we have um about a hundred volunteer industry advisers that cover all aspects of healthc care product development I've just put these in the boxes around toxicology Regulatory Affairs statistics prototypes and reimbursement just to highlight a couple um this is the list of this year's Catalyst projects I already saw ree Knight here on the phone with a question um I'm not going to go through these I'll just mention that the ones that the red ask risks those are co-funded by the the wild Institute of neurosciences um at UCSF that we're very appreciative of and the cell therapy projects are uh co-funded by the living Therapeutics initiative at UCSF that we're also very appreciative of um Callis program has been going since 2010 almost 300 projects been awarded they've been awarded um over12 million and those same projects have subsequently raised a lot of money in um followon funding okay great I'm going to change now to the second topic which is the TPP um just across these four bullets a Target product profile is really a description of what you hope the project will look like at the very end and so if you look at the second bullet it really embodies that's funny I was reading about these words that tell you if something's artificially intelligence written if chat GPT wrote it and I thought and the word is Del and so if you think if you see something has delve in it is a good chance chat GPT wrote it actually I used the word delve now I'm gonna stop using it I think embody is probably the same type thing anyway ppp's embody beginning with the end in mind and if you look at the bottom bullet it really identifies the optimal and minimal product attributes and uh in the case of drugs and if you go to the pharmacy and you get your pillbox and you open it up and there's that super thin paper that you can almost see through um that piece of paper is called well it's called the package insert it's also called the product label it's also called the prescribing information all three of those words mean the exact same thing you know I apologized I can I just realized that there was a box blocking stuff and um it was blocking what I was just saying and so this is the um the first three pages of uh triss's package insert these are used by different parties for example in industry they're used to guide um product development Venture calculus use them to help decide if they're going to invest in a company the FDA uses them as a form of communication and the World Health Organization uses them to set targets for um Global Health projects this is just a snapshot sorry of what the target product profile fields are this just for two of them this is just for the drug projects or the Therapeutics projects on the left and medical device projects in the right and if you note the top and bottom in bold black text on both sides and then you know the the fields in the middle they're gray and faint are gray texts and not and bold the ones in black and gold are sort of common across all tracks regardless of what the modality is where it's a drug or a test or a medical device and those are things like you know what's the clinical indication what's what's the need what is this product doing to help people what's the competitive landscape about either other products doing the same thing or other people trying to do the same thing you're doing and and if you look at the four at the bottom those are things like the opportunity to get patents or trademarks or copyrights and uh the um that's extremely important for a drug it can be important for a a digital Health um and it ranges in the next three the regulatory path to get it approved by the FDA in the United States and the other regulatory bodies elsewhere in the world the question of who's going to pay for it is it's super important and in fact the the second talk tonight excuse me the second talk today by Ry is going to touch on this and then finally the uh the market opportunity and what's the commercial potential and then the ones in the middle the gray ones are sort of specific to whether it's a drug or a test or a device or a digital Health thing um and then the other thing I want to say at the you know a TPP it's sort of jargony and sort of specific but and there's other instruments or documents tools that do the same that are basically the same thing and if you look at the top half of this these are three other things excuse me that are basically the same thing and they have different names in fact this top one sorry I got to take another sip excuse me if anyone's wondering I actually do have covid-19 I'm here by myself actually I feel fine but I've got this cough that you've already noticed and um I'm sorry but I got a big jug of water here so minimum viable product MVP many of you have made many of you have heard this term probably and it's actually even more commonly used especially in the areas of medical devices uh digital health and I think Diagnostics as well but my background as Therapeutics and um my personal experience twice has been with Target product profiles construction um a product development plan a PDP is analogous a design and development plan which is used in medical device development and it's actually the the more common term is a design and development plan and then at the bottom I want to introduce something or not introduce it necessarily I want to say something which is two partner documents for those the top the ones at the top being TPP MVP PDP two companion documents are a clinical development plan which is a much more detailed plan for getting your product through um if it needs clinical trials or if it needs clinical profiling and then finally the risk management plan and a risk register they're really powerful documents that uh help you advance things into development and keeping your eye on the things that could go wrong and just in passing just in passing it turns out that two of our projects this summer um two of the awards actually already have Target product profiles and for those two teams they're thinking about pulling uh excuse me putting together one of these um ancillary documents rather than a DPP okay let me ask this well I take a sip are there any questions before I move to uh and I'm almost done before I move to strategies to fill the medical fields of the TPP which are the fields that are somewhat in common across all projects which are the fields that we're first going to sink our teeth in starting I think this week or next week um any questions Nate I can't really see the screen so or just shout them out or if not it's completely fine no question you can keep going I'm gonna keep going thank you Nathaniel okay um since I've got this screen here I want to mention something that even though I emailed this to all of you and and actually you might have gotten it more than once from more than one person what we're doing this summer so we've been doing this for like six I don't know six years now or something and every summer we try to learn from the previous Summer from the summer before on you know what worked well what could we do what could we do anything different this summer we always sort of tweak things a bit and actually this is only relevant for people that have been involved before because to the rescue it's like oh it's this summer it's this summer I don't care about years past but what we're doing this summer and I don't really want to say is an experiment because we think this is a better way to do it but we'll see is we're going to fill the TPP field we're going to create excuse me we're going to create the content for the TPP Fields sort of sequentially by sort of area and so the first area is the medical fields of the TPP and so those are the ones that you see on the slide here if you look to the the left column these are the one yeah and then you can see in the right column like let's say the details or just explaining what the left column's saying and so the medical fields of the tppp you know vary a bit from product type to product type this is sort of an example that's sort of generic I think um although having said that I should say that some of the digital Health projects you know they're not really therapeutic necessarily and so there's not necessarily a standard of care so I'm just acknowledging that because I I know that but if you look at the left column that these different fields of the medical fields of the TPP are like the indication I mean is this for cervical cancer um you know what is the disease or what is the setting etc etc and is that is it subdivided by um you know the stage of the grade or the extent or the like etc etc and then the next field is how is this indication currently taken care of I mean what do what if what are doctors and nurses and other health care providers doing right now for patients that have it and given what they're doing right now the Third Field is well what do they need you know what improvements can be made and presumably your product is one of those and will hopefully become part of the standard of care and the fourth one it's really really important in 2024 it was less so in 2014 or 2004 1994 is that are there a way to identify the ideal patient for your uh treatment there's this ter there's this expression called Precision medicine and it refers to you know the right drug at the right time with the right dose for the right patient and so it really brings together both Diagnostics and Therapeutics sometimes it's referred to as thetics but it's really uh if you think about 23 and me you think about you know sequencing the human genome there's just going to be a lot of work in the next 10 years trying to figure out exactly what you have as a patient and as opposed to one siiz fits all and so uh if there's the opportunity to come up with a a biomarker a diagnostic you know you want to know it and you want to factor that in and the FDA is just all about that not surprisingly is as they should be and then the last one's clinical trials and so the medical Fields the TPP are what we're going to do first you know this week next week week after and then we're going to move into the more technological Fields the more technical fields of the TPP and those vary widely across all for tracks right and so for example in if you look at the left here in drugs for drugs you think about oh what are the the cell-based models you know what are the predictors of toxicity what are the predictors of efficacy what are the clinical trials and then if you look at the right it's for medical device you know what are the performance requirements what are the safety aspects what are the material requirements how easy is it going to make how much is it going to cost sorry oh my God okay and so how are you interns going to get the data or excuse me the information needed to to fill these fields well one importantly maybe most importantly maybe this is all you need is um hopefully you've been circulated both the application of the the professor from September that was the basis for his uh award application and then as well the PowerPoint finalist presentation from a few months ago and so both of these documents have the information you need but you might want to go into greater detail and that's admirable and fine and actually relevant because you do want to have you know current information and databases around incidents and prevalence get updated every year now that it's already June they might be updated for 2023 in terms of how many patient in the United States you know have this Affliction or this this this uh condition how many in the world have it and those databases might be updated now that weren't updated for the professors uh last year now one of the things that I think you've been provided and I'm taking a chance hold on a second I'm taking a chance I'm gonna click on this oh I should have practiced no actually I'm not going to take a chance because I should have practice anyway one one of the items that you've been provided and don't worry I can't see this either so it's actually this is only funny to me I realized um you've been provided a list of you provided this uh database that we put together and if and this is sort of like a a great thing for all of you both now and in the future assuming the hyperlinks are still hot in the future and what these are is that for your particular disease let's just say it's type one diabetes that you can click on the metabolic disease you know Health incidents and prevalence and facts and figures and find out you know how currently treated how many people are getting it etc etc and so I just want to point out that um the focus of these uh these workshops these five workshops today and then in a couple weeks and then in a couple weeks and then in a couple weeks they're really intended to help you pill out the fields of the next set of fields that are to be filled out and I say it for the third time the medical fields are the first fields that you're going to fill out and this and um again they're pretty straightforward for you to fill out because the Prof the principal investigator already completed them in her his uh app and her presentation but importantly if you interns have questions about the information and where you find it um don't hesitate to reach out to myself or SOA or to Nate or Rupa whoever's been assigned to you to help you to help facilit um what I'd say F what I what I want to say is I just flashed on this is it's better to reach out to us first before um reaching out to the professor I think something else I want to say that's super important that I should have said before that I'm going to say now is and I did write it in an email to my teams is the first thing that we want to happen after this thing after tonight or after today is and actually this is already happen for some projects I happen to know it hasn't happened for any of mine is you want to have a meeting there there's going to be a meeting between the professor the members of her or his team at UCSF working on this project um the summer interns working on this project um The Catalyst staffer facilitating this project hopefully the office of technology management and advancements business development person the the tto the tech transfer officer that's working on this project and maybe some ringers actually a couple of these projects have out outside consultants and so maybe they would show up too goodness knows but that first meeting I want to say this that first meeting is super important now it might work out that some of you can't make it but don't worry you won't have missed anything important no I'm kidding don't worry if you can't make it you'll get brought up to speed because it's important to have these earlier rather than later and it's really hard to get a a meeting on the calendar that everybody can make but at that meeting why it's so important is the professor will give an overview of the project each of you will introduce yourselves to each other and you'll find out you know what you're interested in working on um what your career interests are you know what you're currently doing um in school or out of school uh and we'll just talk about how to divide and conquer on completing these tpps and that'll be a great opportunity to discuss the sources for information and so um each Workshop is going to highlight the upcoming fields that will be completed and the resources for completing those um but anyway uh chances are what each of the projects are going to do is circulate one of those um when to meet W W2 M polls or when to meet polls or a doodle poll or something like that or one of those Google polls um and that first meeting will really be a chance for everyone to meet each other and really kick things off that's the real kickoff this sort of the soft kick off okay I'm done and I'm going to turn it over to the next speaker speaker and it's my pleasure to introduce Ray macowski who happens to be a good friend and a former co-worker of so la shha which is how we got turned on how we got introduced to Ry Ry and I have talked on the phone I'm really looking forward or I guess talked on a zoom I know those are different trust me uh we talked on a zoom and I think I'm really looking forward to the second half Ray is a MD PhD from Puffs University in Boston and he spent much of his career in Industry he worked at um Bristol Meers squib he worked at genim he worked at EP he worked at Fantastic companies sovian and he got into the the thing that we're all doing which is helping project teams Advance her discoveries along the translational path and he works for uh a a Consulting a medical and Management Consulting Group called nitz it's called the nemit group and they do exactly this which is their you know Consultants that help projects Advance along the translational path to commercial calization and patient benefit and so without further Ado I'm going to turn the zoom over to uh Raymond Kowski I see him right on the call well thanks Charles can you all hear me okay that was a really nice introduction I appreciate that someone can hear me yes can you can hear me yes yes okay fabulous now the next question is can you see my slides yes ah even better I have two for two today that's awesome thank you um so it's my pleasure to to have an opportunity to share with you um uh to delve into the chat she PT word to delve into uh the reimbursement considerations and pharmacoeconomics field of the TPP um as Charlie mentioned just a you know my bio is up here but as he mentioned um my consultancy specializes a lot in um helping companies do the soul searching that is really what becomes their TPP um often times my biotech companies will have a Target and they'll have a drug that activates that or stops that Target but they're not sure like which disease to um to use for treatment or where they should go or where they should launch or how they develop their clinical development program um and so we help them a lot with that and I was just thrilled when Charles said at the beginning um that you start with the end in mind because um the ultimate end in mind of the target product profile and in my perspective is is is someone actually going to pay for what you developed um and I think five or 10 years ago maybe 10 or 15 years ago a lot less attention was paid to this uh and there was just a lot more um sort of um prange biotechnology development I would say without a lot is being considered um but it's nice to see that this is on a TPP um and there are a lot of examples um that are showing up around drugs which you are approved but there are challenges to get reimbursement for it and I will say that even before that step um investors when they are looking at companies and again at some of my small biotechs even early on in development they will ask the question how do you think this is going to get reimbursed who is going to pay for it and why would they pay for it I see this with a lot of frequency so I thought it might be helpful um for today just to talk a little bit about the landscape like what that means in terms of reimbursement and reimbursement strategy and how companies think about managing that and how that evolves over um the development of the asset so in the United States um it is still kind of fre range I think but but all therapies and this is you know primarily talking about um therapeutic products drug products and so forth there's a committee on every insurance plan or in every hospital depending on the situation then there's also the government payers um where a drug a therapeutic is provided information about it is provided to this committee and they make a decision on whether it's something they would pay for or not pay for or something in between and um frequently now and within regulation companies are going to these payers long before their phase three trial is done to ask them about what is the economic value that they expect from their drug and the things on the left here the criteria for formulary consideration are the kinds of things that the payers are going to want to know about and they're the kinds of things that Charles mentioned early on is is there a need do we need this drug you tell me this is a great drug for firstline treatment of X but I've already got a firstline treatment for X that treats 95% of the patients and it's generic and it's cheap so do I need it does it work do I agree with the end points in your study um it's not just because an FDA approves a drug based on a study doesn't mean a payer has to provide that drug um necessarily based on the evidence in the stud St Europe is especially like this oncology and rare disease are a little bit different because some regulations is it safe is it tolerable how is it going to fit in to the current treatment Paradigm but most importantly how much is the thing going to cost th this is the the primary question that the payers are going to have and and you can have a great drug that treats a lot of people but if it's going to completely stretch uh of payers budget then they're not going to be able to pay for other things and so the people who are on these pnt committees are always weighing their options in terms of if I pay for this can I not pay for this if I don't pay for this can I not pay for this if I do pay for this and it's got a bad adverse effect effect profile like what other new costs am I going to gender and then what payers in the US can do is they have all these little levers that they can pull in terms of um getting access to a drug or not so for example they can say you know what we need to have a healthcare practitioner say that you got the disease or we're going to restrict it to the label or we're going to restrict it access to the um the patients who in your clinical studies doesn't mean you can't get it you can still pay out of pocket for a lot of these drugs um but that's very very cost prohibitive and they can go all the way down the line to saying you know you know what um we'll give it to you but you're going to have to stop it in six months to see if it's still working or you can only get it at the hospital or you're going to review it Case by case now if you think that sounds restrictive Europe ises a whole another ball game when it turns uh when you're talking about getting access to drugs so in Europe it not including the UK in Europe you get a regulatory approval um from the EMA and that's great but then every country although this is kind of centralized now has what they call a health technology assessment which is a group of payers getting together and saying okay EMA approved the drug do we agree with their opinion in terms of the efficacy and the safety um regulatory bodies typically don't worry about the approval of a drug and the impact of the Health Care system at large a health technology assessment will say how does this drug fit in the Paradigm of other medicines that I'm paying for and if they agree to that great then you still have to go to pricing and Contracting negotiations with the various countries and say we think the drug should cost X and they will say you think the drug should cross why and then you enter this Loop of um whether or not negotiating what your price is going to be something like this kind of happens in the United States uh with respect to Contracting and rebates and all that kind of things but the United States has an incredibly opaque and difficult to understand Healthcare reimbursement system so it all is kind of something that's happening behind the curtain but in Europe it's it's right out there in front and then this is important when it comes to TPP I'll come back to all this some markets like the UK will want to know what am I getting for my buck or my pound rather and and is it worth it like I've said that your you have said that your drug will reduce mortality by three years and it improves quality of life for patient by this number and you say it's going to cost 30,000 pounds per year per patient for this number but we set a threshold at 20 so you're going to have to negotiate that with us and these are these are countries working in a fixed budget other markets like Germany for example will say um how relevant is this to the patient and they will involve patients early on in the conversation around um should a drug be reimbursed or not or approved or in France they'll talk about the clinical relevance of it so all this comes back to the TPP because again with the end goal of mind just because you get approval at the FDA or EMA or some of the other um outside of us markets doesn't mean it's actually going to find its way to patience and there are examples of companies um BL is a recent one that have pulled out of Europe because they couldn't Reco the cost of their clinical studies so how do you fill the Box in especially if you've got you know you're doing most of your work in a lab right now you may not have a therapeutic idea uh in mind or reimbursement strategy um you can guess and and guessing is okay to start because you're going to change your TPP over time some good guesses that if you have a small molecule and the patient population is not big there won't be a lot of restrictions I think we're about to learn that that doesn't always hold true for the gop1 molecules that are out there there's being considerable amount of push back you can imagine that you have a biologic in a small patient population that you might get a few restrictions on it and if you have a gene therapy for a tiny patient population you might find the compound to be very highly restricted and have to enter into things like what are called value based agreements which are starting to show up in the US's potentially U inflation reduction act about like you have to prove that a drug works and after six months if it only works to a certain percent the Pharma compan is paying that back to the payer of the government but you can't guess forever um investors will only tolerate so much guessing and so what you often will do with the TPP is you put it in front of actual payers and you'll ask them what do you think what would your restrictions be so you can go back you can plan for your restrictions for commercialization and you can also go back to your investors this is an actual slide we used uh about two weeks ago for a biologic for rare neuromuscular disease in the US and we do the same thing you do for Europe but we we got a bunch of payers together and here's just a list of like who they were in their organization what their organization does and what their plans cover and how many lives each plan covers and we put the TPP in front of them for this product and we said what do you think we said what do you think you would do in terms of reimbursing it what do you think you would do in terms of restricting it we didn't ask about the price but they always give you their opinion anyway which is fine what they would um what they would potentially reimburse up to for example and you get information back from them like like this they will say to you well you know what great therapy highend met need nothing effective and safe is out there but here are some things we'd like to see we'd like to see your study be Placebo controlled this is very important in Europe also we want to know like more about the scale that you use in your study agnostic of what your thought leaders might think we think that this is an acceptable improvement over the drug of versus placebo we'd like you to not have to patients to not have to come into Clinic to administer the drug but have a self Administration we'd like to have greater control over how we pay for it meaning putting it under a pharmacy benefit instead of a medical benefit which is just a way of categorizing how um payments are made in the US and then we're going to want to know things like how hard is this going to be in our budget and should we um allow it for a year and if we do allow it for a year then would you have to reauthorize it after that or should we have our health care practitioners do this scale at the beginning to show that patients are severely effective enough to for us to reimburse it and then do the scale again six months later to see it's actually working and these are the kinds of things that they will do to control the levers hey Ray KN yeah thanks I got a quick question this is this is uh this was really really interesting that especially the previous slide about um the same topic um asking all these um payers or payer decision makers their thoughts I'm just curious what was the format was it so I have two questions one when you provide the TPP I mean how extensive is it I told you I've already I worked when I was in Pharma I worked twice on these ad hoc TPP committees and they can be pretty long and detailed I'm just Cur and such that it would take quite a while to read unless you had an executive summary and so my first question is what are they provided what kind of how r or deep is the information that they're getting from you and then the second question which is related but different is what was the Forum do you just email it to them and they email you their answers or did you have like a did you email it to them then you have a phone call and they get to just answer off the top of their heads rather than having to write something down I'm just worried interested in like the mechanics yeah getting this questions customer Discovery from five different customers thanks good good question so um the first one is they're blinded so we so they have no idea who the company is who the product is who whatever so as much as we can they don't know exactly who it is now if if they were smart enough they could kind of figure it out sometimes because there's one product out there in clinical development but it's blinded um what we give them we do send them materials in advance so that they have an opportunity to look through it and think about it in the context of their plan and often times if you tell them the disease State they'll go back into their own plan and say oh if our 20 million members we've got 100,000 with this disease and so they're already starting to think about like what the impact of the drug could be and the information you give them there's kind of a core set of basic information like uh you know what's the indication what's the patient population what's the current treatment flow what's the standard care things like that they'll go back to their own formularies and see what's preferred what's not preferred for example and then a lot of it then it becomes very specific for the company so this particular company was wondering about adoption of um Administration in the office versus at home and so you ask very specific questions what are your thoughts on that and the way you do it is when you you mail out the you mail out you email the CPP usually you give them access to a data room so they can't you know distribute um and they're all held under confidential disclosure and they're all paid by the way they're paid advisors um and then you either meet with them oneon-one for an hour hour and a half and you go through everything you talk to them or you meet with them in Europe we do this more often you meet with them as a group so you meet with them as like five or six people from representative countries usually UK France Germany Spain and Italy and then you throw in in Nordic just because you do I guess um and you get feedback from them got it and then that's what turns into this thank you and and and why that's important and and and why we do this early is because you want to get this feedback early enough to make a change in your program if you need to so if like on the last slide they said you know this is really interesting but we'd like to see a placebo controlled study and you say oh yes we okay maybe we should do a placebo control study what I often see in Europe is the the European Health Technologies assessors will say we want to see these two measures these two scales in your study because that will help us understand what the quality adjusted life year is it's a metric they use to determine like how much they should be paying for things across all therapeutic areas diabetes depression cancer whatever and they'll say put this in your trial so if you hear that from the payers early on you put that in your trial sometimes they'll say perform additional studies um I had the unfortunate experience once of feedback with the US wanted to see one type of study and Europe wanted to see another type of study one was a Pao controlled and the other was inferiority against standard of care um it was Europe who wanted noninferiority against standard of care because that's how they think and so you had to reboot the clinical program and then the other one I think that we're starting to see a lot more of um is payers want to see real world evidence because their membership are members of the real world they are not in clinical studies I mean some of them are but but the the people who will be getting the drug are not those people and a question we frequently is what's a standard of care and how well does the standard of care work in the real world and there are lots of studies that you can do that we do do using patient charts using claims databases using a lot of other different Registries for example to develop that evidence um these types of studies take years and they cost millions of dollars and so for a company to know early on in their development that they need to do it is better and the other thing because of that is when investors come in and they see you haven't done any of this they'll realize that number one they probably have to take on this work and number two as well-intentioned as some of these biotech companies may be they hadn't prepared for the payer question and now they're going to be behind getting reimbursed in Europe certainly so just to conclude I wanted to make sure I left time for more questions you know when you're when when you're putting together your tpps and you get to the reimbursement um part you know depending on where you are in your clinical development program you may want more or less information in there if you're still in the lab probably less if you're about to start enrolling patients you'll probably want more investors will want definitely want to know um the landscape is consistently changing the HTA regulations are changing um I actually think they're changing for the better but they're going to make it um a lot more work for these companies to do to get reimbursement which is in my opinion a small price to pay to get reimbursement um us payers typically tend to not worry about this so much basically they're focused on something called the per member per month which is they'll look at the 20 million patients in their plan realize they've got a 100,000 of them your drug cost $5,000 a year their annual member will have to pay four cents more per month and then importantly once you get the feedback it's being ready to adjust the development program or generate new evidence um and and this is something that we actually recommend to our clients that they do uh more than once that they get feedback from the payers and then they change their program and they get feedback again and they get feedback again oh sorry you know Charles one other thing I should mention when you ask me about how you do this um we we don't actually so in in the US it's a little bit different in Europe we're not actually allowed to talk to actual payers so what we do is we talk to people who have been on these payer committees who are now themselves consultants and so they are no long they no longer have decision-making Authority in their country but they can tell us how their country thinks oh that's really I'm glad you added that that's really interesting the the Restriction against talking to actual yeah thanks for clarifying that thanks you're welcome so that's just a a nutshell run through I'm more than happy to answer any questions or if people have as I mentioned I'm also a catalyst advisor so if people have questions for me random ones I'm happy to answer them I meaning now or later like you know tomorrow next week two months from now I see people have hands up and I'm I think we've reached the end of my um Zoom skills here so let me see uh I think it's an has a hand up yeah uh so this is a pretty like rudimentary question I think for the same slide with payer and um like payer 1 2 3 4 five who are those agents are they they Pharma companies are they um like who are they representing are they representing the investor side are they representing the uh drug and development um that team uh are they representing The Regulators who are them uh is your question who is who the when we talk to payers who are we representing ourselves as in the company oh no I'm asking who are those payers oh so it's in in the United States it's insurance companies so it can be Kaiser it can be Cardinal it can be any sort of insurance company in the United States that is responsible for for paying um we try to find someone who has previously worked at um the CMS centers for Medicare Medicaid so they'll have some sense um in Europe uh each and it's people who have we we try to find people who have um had SE leadership positions in those companies so they can speak to what the expectation would be and then in Europe every country has their own um body responsible for payment C it's it's almost always completely centralized although some countries you can get insurance outside of that um and it's a government body so you actually by law have to go through first getting the drug approved and then go through these payer associations to get it reimbursed that's a good question and U I think Vincent had his hand up yeah did that answer your question by the way H yeah and I have a followup but feel free to answer Vin question first oh you can you can go ahead and get that question out of the way uh yeah so you mentioned one point about um drugs or devices that are still in the lab might want to disclose less for their PPP what is the incentive for that so it's it's usually confidenti oops it's usually confidentiality on the on the part of the client so the client would prefer that payers because they're still kind of formulating an opinion about what their drug is going to do and they're going to still talk about they're going to go back and evaluate the payer feedback and put you know more information to that they just want it to be confidential um it also protects the um the respondents in case they have potentially a financial interest in whatever company is um providing the TPP to them so it's really just a protection measure okay for both y you're welcome yeah so my question was um I was really interested to hear about how different the landscape in Europe is for approval and reimbursement so I assume that because of of the high cost of developing drugs or Healthcare devices there's the goal to get it into as many markets as possible but because of the restrictive or different nature of different countries approval processes are some drugs developed today only for certain markets and they give absolutely I'm just really interested in this absolutely there is there's something that we call Market sequencing market and and and the price the the the price question is fascinating because in Europe if if you get approved in Germany and Germany says your price is X that becomes what's known as a reference price and so every other Market will look at it and say well wait a second you said Germany was X why are you charging us why and so and and there's an entire work stream in Pharma around and there are countries that you want to go to first because the negotiations for the price are either shorter duration or they're easier or there's an expectation that given your therapeutic area you're going to get more favorable price see your reference price is higher on the higher end than it is on a lower end in the US what we typically do is we do analoges so this neuromuscular drug that I mentioned before when we get ready to talk about pric we'll take four or five drugs that are kind of in the same space kind of have the same patient population kind of have the same duration of treatment there may be a biologic or something and we'll Benchmark against all of them and we'll say Okay payer you're paying 10,000 a year for drug a 12 for B 13 for C where do you see this drug fitting in that's a whole separate work stream on the commercial side of the organization but it's it's a great question no that's that's so fascinating and then I could imagine there is a lot of strategy and like you just brought up a lot ideas in my head about choosing these different countries or um finding different analoges thank you so much yeah you're very welcome great question um alza I think had the next hand that I can see yes uh so thank you for uh the nice presentation my question was that so uh based on what I understood the insurance companies wouldn't make money on accepting reimbursing a new drug so they're actually like paying more so maybe if on the earliest stage of developing a new medication if we focus on or if you focus on reducing the secondary complications of diseases or maybe reducing this complications of the current stand of standard of care do do you think this would uh give us more more negotiation power in this them later because we then we can tell them if you accept reimbursing this drug and your customers uh or people who are like in this insurance uh take this drug they will have they would need dress less drugs later for other drugs later so you're going to make money more later they got to save more money for them is this you good understanding or I'm wrong I think it was kind of breaking on me a little bit but I I'll try and answer what I thought I heard um which was about um so so when you're developing drugs there are a lot of drugs out there that treat the symptoms of disease it's very compelling to find a drug that treats the actual cause of the disease and and payers will almost always go for that um what a payer will do though for every single drug and you give them what's called a budget impact mod and you will tell them this is the price we're going to set and they plug it into what's essentially a very fancy Excel spreadsheet using their own um plan and their own tiering and they'll spit out a number that says how much this drug is going to cost um for for for drugs that treat very rare diseases that are very expensive uh a me a a plan may have two patients in their entire membership and they're not even going to notice a million dollar drug so to speak this is where the gp1s are getting really interesting because there's a lot of people out there who could benefit from these drugs and these drugs are very expensive and so the first wave was great we'll put these on formulary and now they're looking at the impact and saying hold on a second I don't think we can afford this so I again I'm sorry I couldn't I had a little trouble hearing the question so I hope I maybe someone heard it better than I could or did that kind of get to what your question was yes I think I got the idea and then the keyboard that I can search for thank you yeah you're welcome so Ray we're over time but I had a quick question along those lines around you know you hear a lot about uh pricing strategy around how much is is it costing the insurers now right so then you kind of try to position yourself you're going to save X doll at what point do you guys do that exercise do you present that when you're doing the he opinion leader discussions or later so for the you you you kind of do it um you do it fairly early to get a sense of where they're where they're going where their heads are at and where the analoges are um but you really do it like maybe six months before launch when you're really start to kind of nail down the price because you'll always keep like a a ballpark price in the back of your head or where it's going to be based on the analoges but when you feel really comfortable about what your price is going to be then you would start to do more detailed research and that becomes super secretive because the that's when you really kind of lock down like what you're going to say about the price and everything because then it it's kind of revealed at the end and there have been some really remarkable failures of companies lately who have completely gotten the price wrong and the backlash against them was was incredible and those of us who have been doing this for a while look at them and say what were they thinking like why did they why did they go in with that um but it's it's kind of an ongoing process throughout development I would say but really the benchmarking is is the thing and it's funny because the same is sort of true with a lot of FDA approvals like your benchmarking is what has the FDA approved before and easier if they're working with something they know than something that's new although the FDA I lately especially with the real world evidence is doing an incredible job of being Cutting Edge with a lot of work that they do in my opinion right thanks okay P hang uh go for your question thank you okay sweet thank you um yeah so you just mentioned about like um around maybe six weeks prior to to launch you start looking at your price but I think at some point you mentioned oh you probably want to look into like real world evidence but um to my knowy I feel like real world evidence something you conduct like post launch or like post market right um so given that if you you if you have like real world evidence post launch post Market launch how are you still able to adjust your price after that ah good question so the first question you actually start looking at your price a lot sooner than that um you you start to have an idea around so this company that I mentioned is probably not going to launch for two or three more years um but but they know that um they've already started to get an idea of what will be an acceptable price and you they will refine that price over time so they might get feedback later to say um that uh the price should be different or there's been a new market entr or it's in a different line of therapy um so the pricing work actually starts pretty early but you but weeks before you really start to kind of nail down what the exact price is going to be with respect to rear World evidence um rear World evidence is important not necessarily for the the drug that's approved but for all the other standard of care that's around it so for example um I have one client where I'm actually setting up a registry for them where their drug is not going to be approved for four more years but they don't know how well the standard of care works because it's pretty rare disease and so they need to generate some evidence on how well the standard care works because once they get to Ema and definitely once they get to the payers the payers are going to want that evidence and they're going to want to say okay your drug reduces this by two points what does in this case IVIG do to the same patient population the other thing that rear World evidence is important for is once you know what the standard of care is that patients are getting you can know how much that costs so so you can go back to a payer and say for your patients with XYZ for example their cost of payment is this and because they have this adverse event and our drug shows that you don't have this adverse event and so therefore our drug is this much more cost effective and that's that's what a lot of the Europeans like I think um the uh inflation reduction Act some language in there around value which is probably where things are going to head a little bit in the US which I honestly think is a pretty good idea um to keep some of the healthc care cost down but yeah you collect and and the other thing I would say about rear World evidence too um is that for a lot of the drugs I work on I work on rare disease and oncology mostly most of the clinical trials for that are are single arm not Placebo controlled and so you you you can't know um what the standard of care is and how it Compares that unless you go out and get it okay sweee thank you so much you're welcome okay uh on behalf of the catalst program I just want to thank Ray for really thought-provoking I learned so much from that talk and it's great um so thanks so much Ray pleasure joining and the next Workshop will be next Wednesday we have three guest speakers uh all of which are great I happen to know firsthand so Linda mallister David Kim and AAA K kurahara are gon to talk about Diagnostics uh digital health and medical devices and finally Gene and cell therapy the focus of next week's Workshop is how do you fill out the the technical science engineering design pre clinical clinical fields for like the the nuts and bolts of how things work yeah I guess that's it so thank you all very very much and we'll see you in a week and two days if not sooner okay bye