**Transcript of Cyberseminar**

**HERC Health Economics Seminar**

**Updated ISPOR Guidelines for Performing Budget Impact Analyses**

**Presenter: Dr. Josephine Mauskopf, PhD, MHA**

**January 15, 2014**

*This is an unedited transcript of this session. As such, it may contain omissions or errors due to sound quality or misinterpretation. For clarification or verification of any points in the transcript, please refer to the audio version posted at www.hsrd.research.va.gov/cyberseminars/catalog-archive.cfm or contact herc@va.gov.*

**Moderator:**I’m very pleased to present Dr. Josephine Mauskopf, PhD, MHA, who is the Vice President of Health Economics at RTI Health Solutions. Dr. Mauskopf has extensive experience both as a consultant and as an employee within the pharmaceutical industry designing and implementing pharmacoeconomic research strategies. She has designed pharmacoeconomic research programs, estimated the cost-effectiveness as well as the budget impact of new health care interventions in a number of therapeutic areas, including infectious diseases and neurodegenerative diseases. She has completed an eight-year term as Editor in Chief of *Value in Health*, and was Co-Chair of the Good Practice II Task Force Guidelines for Budget Impact Analysis. She’s also served for four years as a reviewer on the health care technology and decision sciences study secession at the Agency for Healthcare Research & Quality. Dr. Mauskopf is an expert in the field and we are very pleased that she is presenting today on Budget Impact Analysis. With that I will turn it over to her.

**Dr. Josephine Mauskopf:**Thank you very much and hopefully you can hear me, and I look forward to your questions at the end. Today I’m going to give some background to the new Task Force Report, which I just received a copy of and should be posted on Medline that hopefully will be within the next few couple of weeks or so. So it’s just about to appear. I’m going to give an outline of the report and summarize the key recommendations, and tell you about the publication which I’ve just done. I want to get right into it because I want to leave time for a discussion after the presentation. Many payers include mandates for budget impact analysis, but certainly with a lot of the health technology assessment agencies in North America, Europe, South America, Latin America and Asia, require a submission of a budget impact analyses for new health care interventions if they’re going to reimburse them. But I’m sure a lot of you also have to do this analysis to help you plan your budget. With an aging population and a sluggish economy, concern about the budget impact of new drugs has been increasing recently obviously as well as the monoclonal antibodies and other drugs where the price tag has become significantly higher than used to be the case for drugs. Since Task Force I, which was completed in 2007, many publications appeared reporting the results of budget impact analysis using a variety of approaches. And also several countries have produced country-specific guidelines. So ISPOR felt that it was important after seven years now, has produced an updated methods guidance on the conduct and reporting. That’s what I’m going to be talking about today.

This slide just gives you a snapshot of the old Budget Impact Analysis Paper. And the one thing that I want you to take away from this is if you look at the author list and where they’re from, it’s very apparent that the U.S. and Europe are represented in the group of people who were responsible for passing this Budget Impact Analysis Task Force Report. And so you’ll see one of the differences with second one in just a few minutes. The mission for this second task force that we set up in 2012 was to develop an updated, coherent set of methodological guidelines for those developing or reviewing budget impact analyses. The footnote is very important here. Unfortunately this is not an instruction manual. Maybe it will be nice to have one, but the goal of the Task Force Report itself was to provide methodological guidelines. It’s not a “how to do it,” but to tell you what sort of things you need to think about and what sort of things need to be included in a budget impact analysis. One of the things that I have done recently is that there’s a new Encyclopedia of Health Economics that’s being edited by Tony Culyer of the University of York that’s due to be out in March of 2014. I’ve written a chapter on budget impact analysis that encyclopedia, which is a little bit closer. It’s still not an instruction manual, but it does outline the steps that you need to take and also give some very detailed examples. If you’re looking to the Task Force Report to give you that, then you may be disappointed. Hopefully, it will be helpful. The guidelines are not only methodological for developing the budget impact analysis, but also for presenting the results in a way that’s useful for decision-makers. 

This slide is where I wanted you to contrast with the author list on the first Task Force Report, because one of our concerns when we were choosing the core members for the Task Force was to make sure that we reached beyond the U.S. and Europe. And also to make sure that we included people who were very much involved with health care technology assessment agency people, with designing and creating these budget impact analyses, as well as involved with the use of them for reimbursement decisions. You can see we have at least one representative from Latin America, South America, and also one representative from Asia. So we went beyond the U.S. and Europe. We also have Mark Minchin from NICE in the UK who represents a health technology assessment agency, who’s not using budget impact analysis for making reimbursement decisions, but he’s producing them after they’ve made the decision based on cost-effectiveness analysis for helping their groups within the practice areas within the country to plan their budgets based on whatever NICE decision has been made. Then Karen Lee from Canada was one of the people from the health technology assessment agency and also was responsible for developing Canadian-specific guidelines. We wanted to make sure that the group included people like Ewa Orlewska from Poland who also has been involved in creating country-specific guidelines. So we’ve had people who were familiar with creating guidelines, were using them for decision-making, and were now representative of at least four continents rather than two. 

In terms of the Report Outline, I have an Abstract, something about the Task Force Process, which I’m actually not going to talk about. There’s an Introduction, Recommendations for an Analytic Framework, Recommendations for Inputs and Data Sources. Both these sections were expanded from the first Task Force Report. An then there’s Recommendations for Reporting Format, Recommendations for Budget Impact Computer Program, and a Concluding Statement. I’ll be presenting all of those sections to you. The purpose of the Budget Impact Analysis as stated now in the report is an essential part of a comprehensive economic assessment of a health care technology increasingly required, along with cost-effectiveness analysis, prior to formulary approval or reimbursement. And the purpose is to estimate the expected changes in a health care system’s expenditures after adoption of a new intervention. The intended audience is listed on this slide and I’m not going to read it to you. But they are for health care decision-makers and for other interested groups. 

The context of the new report and to some extent I’ve said some of this before, initially we called it a history, but then we decided not to have history and to use the word context. In the 1990’s Health Technology Assessment agencies as they were being set up started to request budget impact analyses. For example, NICE in the UK, the Academy of Managed Care Pharmacy requiring some sort of economic evaluation in the U.S., and the Pharmacy Benefits Advisory Committee in Australia started to request budget impact analyses along with the cost-effectiveness analyses. Starting in the late 1990’s, publications started to make a distinction between the budget impact analysis and the cost-effective analysis. The budget impact analysis has a different perspective. It takes prevalence, a population perspective. We’re looking not as a cohort going through time, but we’re looking at all of the people currently treating for this particular disease in a particular time period. So it’s a different perspective from that of a cost-effectiveness analysis. And also there’s an emphasis that came out in the literature on the decision-makers perspective. It’s not a societal perspective as there is with the cost-effective analysis, and so there’s no right set of cost to include. It really depends on what the decision-maker is interested in. If they’re only interested the drug budget then that’s what you need to be able to show them. If they’re interested in offsetting disease-related costs then you should design your model to show them that. There was the ISPOR Task Force I in 2007, and then after 2007 what we noticed was that there were an increasing number of publications in research journals between 2007 and 2014. Before 2007 if you went in and did a Medline search on budget impact analysis as a title, you’d find very few papers. After 2007, and I don’t think it was anything necessarily to do with our Task Force Report, there were a lot more. So this led us to think about, “Are there new methods?” What happens with publications once things start to be pushing the research literature, you get a sort of research imperative and new methods developed because that gives you more of a chance of publication. And so we were concerned about that. In addition, there were many country-specific guidelines that were developed. There were some before but many after 2007 that we also felt we should look at and see if our recommendations were consistent, whether they were things we could learn from those guidelines. That is the context in which the ISPOR Task Force II was set up and the background part of the Task Force Report.

The analytic framework is the first major section in the report. And what that is doing is basically setting up a set of things that you need to think about and to know about before you make a decision about how you’re going to design your budget impact analysis. It’s a means of synthesizing available knowledge to estimate the likely financial consequences. You’re trying to estimate what checks the health care decision-maker and the budget holder is going to have to write. A budget impact analysis provides a valid computing framework that allows users to apply their own input values and view financial estimates pertinent to their setting. And you’ll see this when we talk about the computer model at the very end. 

Now just to step back for a moment and tell you a little bit about why would a new health care intervention have a budget impact? We have this Figure in the Task Force Report. On the left we have Current Environment. You have a Cost of Illness box at the bottom. The cost of an illness, the cost associated with a condition depends on all the things above it in that Figure. It depends on the size of your population to begin with, and it depends on how many of those people get the condition of interest, so the incidence of prevalence. How many of those are diagnosed and treated? If they’re not diagnosed and treated, you may well have a cost, but you’re not necessarily going to be aware of it or be responsible for it.  And then of those who were diagnosed and treated once their resources are used depending on how they’re currently treated and what’s the unit cost associated with that resource use. When you multiply all of those things together you come out with a cost associated with that condition. The reason why a new health care intervention can make a difference to those costs and therefore have a budget impact is it varies depending on what the intervention is. If it’s a preventative intervention, a vaccine or a prophylaxis of some type, then it’s going to obviously reduce the number of people who actually get sick. If it’s a diagnostic or some sort of a new treatment, a diagnostic might change the number of people who are diagnosed and therefore the number who might be treated. A new treatment could also change the number of people who are diagnosed and treated. For example, when the new immune inhibitors came in for influenza, it probably resulted in more people going to the doctor when they got the flu, because there was now a viral treatment for it. So the number of people diagnosed and treated with influenza I’m sure has increased greatly since that time because of the availability of a new treatment. 

Another way that a new treatment might change the cost of illness is because it changes the probability of a person being hospitalized and visiting the physician. It may substitute for other treatments, either surgery or other drugs. Or it may be added onto other treatments. So the resource utilization for that underlying disease may change. And also of course the new therapy or procedure may have different unit costs from the old ones. If people are just being treated just with generic drugs and now you’re bringing in a branded drug, then clearly the unit costs will change. All of those things can result in a change in the total cost associated with that disease. Some of which will be increases and some of which will be decreases. But there’ll be a net change in the total cost and the difference is the budget impact. So that’s Budget Impact 101 very quickly for you.

Let’s get now to the Task Force Report and the Elements of the Analytic Framework. I’m going to go through each of these and give you very briefly what we said about each of them. One of the things I just want you to note on this slide is that if you look at the third bullet from the bottom it’s the choice of the computing framework. So the analytic framework is not just about the computing framework, because choice of computing framework really depends on all these other things that come above it in this list. You have to think about all of these other things first and then based on your particular indication and disease, then it may help you on what is the appropriate computing framework. So you don’t start with computing framework. You have to start considering all of these other issues, and based on that come up with a computing framework and the other items. 

The first recommendation is that the features of the health care system include those that impact the budget. I probably should have pushed on this slide not only those that impact the budget because all of them do, but they’re all likely to change with the new intervention. If it’s not going to make any difference to certain features then obviously you don’t want to include them. Let me say before I go on that one of things that the Task Force Members were unanimous amount, and this is exciting to me especially, was that we wanted to keep the goal of budget impact analysis as simple as possible.  One of the concerns that we had is that with more budget impact analyses being published, there’s this research imperative and a more complex methodology making it easier to get published in a high tier journal. There was a concern that there’d be a push towards making the methodology more complex and everything that we’ve recommended was pushing in the opposite direction and saying, “We want to keep these things as simple as possible.” So again for the features of the health care system to include only those that are likely to change, but to include all of the things that you are likely to change. The perspective of the analysis is that of the budget holder. And you want to design your computer model to allow presentation of all of the perspectives of interest. So don’t only allow them to look at the drug budget or the drug budget plus the offsetting disease costs, but let them look at elements that they want to look at. 

Again the whole point is not only that it be simple, but it’s meant to be useful for real life decision-makers. Even though the population is one small little simple bullet here, this is the hardest part if you’re actually doing a budget impact analysis. This is the hardest part to get right. You want to include all patients eligible for the new intervention during the time horizon of interest. So this is a population perspective. But what you have to note and to understand and allow for in your computer model is that the new intervention may change the size of the eligible population. I gave you one example with a treatment for influenza, but it can change the size for many reasons. Another big reason is if the disease like cancer is rapidly fatal and this prolongs survival, then obviously you’re going to have more people alive getting treatment at any one time. The HIV infection is another good example of where that has happened. The other thing that it might do is to change its distribution by disease severity, and HIV is a good example for that because not only are more people living with HIV infection now, the proportion of them who are in less severe stages is much higher. They’re being kept very healthy. So when you’re looking at what’s happening when you introduce a new intervention, you’ve got to look at not just the size of the population and distribution by disease severity now, but what’s going to happen to it when the drug is introduced.

For current interventions you want to again unlike cost-effectiveness analysis when you’re usually comparing a new drug with some standard of care, here you’re comparing the mix of all currently used drugs or treatments including surgery. If appropriate, you should also include off-label interventions. And then you’ve got to estimate the uptake of the new intervention and its effects on current intervention mix. What is very important for estimating budget impact is whether its going to replace a current intervention, be added to a current intervention, or be used where there was no previous intervention. You need to create a model that allows the use of flexibility to enter different assumptions about changes in the intervention mix. And also it depends on whether if you’re going to be replacing a generic drug with a branded drug, clearly the budget impact is going to be much larger than if you’re replacing a branded drug with a second branded drug. So you need to consider all of those things, makes reasonable assumptions, but allow the user to change those assumptions if they don’t agree with yours. We’ve had a lot of discussion about off-label use of the new intervention. We said yes to the off-label use of current intervention, because they’re really being used and you might well replace them. You really only need to consider current interventions that you’re likely to make a change in their use. But for off-label use of the new intervention we felt that those should not be included. And that’s specifically requested by the budget holder. Again the budget holder is the key here. You’re trying to do this to help them. And then the cost of the current intervention depends on the budget holder’s prices, multiplied by the eligible population.

Condition-related costs should be included if credible data are available and they impact the budget during the relevant time horizon. If there’s a nice head-to-head random-controlled trial that says that the use of this drug or the use of that drug will have fewer hospitalizations in the year, then you could offset your increase in drug budget costs that save with reductions and condition-related costs. Again, not all budget holders are interested. And many are only interested in drug budgets or some other budget that they’re responsible for. So you should present results with and without the impacts. We said that indirect costs should not be routinely included in a budget impact analysis. It could be if the decision-maker wanted them. The time horizon was that of relevance to the budget holder between one and five years. And time dependencies and discounting is generally not the discount to net present value. Because what we’re interested in is cash flow. So we’re looking at cash flows of the year one, year two, and year three. What is the cash flow estimated to be and not what the net present value is as one might in a cost-effectiveness analysis? So these didn’t change from the previous ones.

Now we finally come to the cost of a computing framework. This particular Task Force Report actually is quite different from the first one, which was much less prescriptive about this. But clearly the panel and the person from NICE was very supportive of this to use a simple cost calculator using spreadsheet software and not a disease progression model where possible. And that should be possible for most acute illnesses, but even for chronic illnesses. Unless you really couldn’t manage it you should use a simple cost calculator approach. If there are changes in eligible population size, disease severity mix or intervention patterns, you can still very often capture those using a cost calculator approach. If you can’t do that then you could use the cohort or simulation model to account for those changes over time. But you still need to adapt that model to account for the fact that this is an open population and you’re not just following a cohort as you are in a cost-effectiveness model, but that you’ve got people entering and leaving the eligible population over time. We were much more into the simple one this time than we had been last time. And that’s partly in response to a lot of the publications where they had moved away from the simple one. Not all of them but many of them. We didn’t put examples in the text of this Task Force Report. But we do have a fairly good reference list of both cost calculator models and the more dynamic disease progression models. So we cite several good examples. If you want to get more of a feel for how to do each type, look in the reference list and you’ll find what we consider to be a state-of-the-art good model. There was a bit of pressure again in the literature for probabilistic sensitivity analysis to creed in on the uncertainty or scenario analysis, but we pushed back on that and recommend a one-way sensitivity analyses and also a plausible scenarios of relevance to the budget holder based on alternative assumptions and/or input values. So again we wanted to keep it fairly simple. In the validation we finally said that the face validity and program verification should always be completed. We felt that for additional credibility, initial year budget impact estimates could be compared to current observed costs in the health system if they’re available. So validation is I believe very important and so we did try to say if you’ve got some actual cost data and you can validate it you should do it. I know one of my first budget impact models when I tried to publish it, the reviewer came back and said, “Is it valid?” And so I got some medical data from California and was able to look at the values from that and show that my estimates were close to what was actually being observed. It certainly got the paper published, but I think it also helped make it more valuable for the decision-maker.

Now I’m going to go fairly quickly because I want to leave us some time for questions, and I’m conscious that I’ve been using up a lot of time. What we tried to do in this Task Force Report is to be a little bit more helpful about different input data sources. On the next two slides we have listed some examples, as we do in the Task Force Report, of how to estimate these different data sources in order to be able to estimate the different things that you need to estimate for a budget impact model like the size and characteristics of the eligible population. I’m not going to read this and you have access to the slides, but I think this to me is the hardest part of doing a budget when you’re actually doing the budge impact analysis and maybe the most important. So plan on spending a fair amount of time on it and hopefully our suggestions are helpful. The one thing that I wanted to point out is the last major bullet here, the eligible population for a chronic condition or vaccination program, because there are two population cohorts that you have to consider. You have to consider those who are newly eligible and then a catch-up subgroup because they became eligible before the new intervention was available. And so what this means is that maybe people who passed the eligibility criteria threshold a couple of years ago, let’s say for a vaccine, if you bring in a vaccine for twelve year olds there are thirteen year olds and fourteen year olds and fifteen year olds for whom the vaccine wasn’t available when they were twelve. And so you can have a big catch-up. If you’re a budget holder this can make a big difference to how much you’ve got to shell out in the next year or two. So it should be considered in the budget impact analysis. 

Again we say that if you can get the data from the budget holder’s population directly, then this is obviously the preferred approach. But there are other sources like market research data for example. For the new intervention mix you’re going to have to estimate the uptake of the new intervention. We give you three possible examples of how to do that. Predicting the future is not an accurate science, so none of those are going to be quite right. The very key thing is how to determine the impact on the use of current intervention. And there again, you don’t have a lot of sites. Market research doesn’t really help you. Producer estimates or expert opinions should be used. But here I think the key is flexibility in the program. In the cost of current and new intervention mix you need to think about drug acquisition costs after adjusting for any discounts or rebates. You may want to think about administration and monitoring costs, because those may be different for a new intervention. You want to think about costs of managing side effects or complications, because again they might be different for a new intervention. These are all of the things that you have to think about and suggestions of how you can get those costs are provided in the Task Force Report. If you’re going to include costs of other condition-related conditions, the latest services, where are you going to get that information from? And one of them of course is from the clinical trials, but again you’ve got to switch it to a population perspective. You’re not following a cohort. You’ve got new cohorts entering the population every year. And then other ways to do that are in consultation with physicians to get treatment algorithms that are different. Or if your new interventions don’t change disease severity, then you can have consultation with physicians about what it costs, what treatment algorithms are for the different levels of disease severity. And that will allow you to estimate changes in disease-related costs. 

Finally for alternative values for uncertainty and scenario analyses, arbitrary ranges plus or minus twenty percent or fifty percent are not recommended, but unfortunately are often used. It’s of concern because plus twenty percent might work for some variables and might be realistic, but might not be realistic for others. So I would hesitate before you do that. In the scenario analyses I want to point out is if adherence or persistence is included in a scenario analysis. We haven’t talked about that, but also in some chronic diseases people don’t adhere to therapy or don’t persist with therapy and that makes a difference to the budget holder. If you’re meant to get twelve prescriptions a year of one every month, but in fact you only take your medicine once a day instead of twice so you only fill six prescriptions. And it’s not so difficult to estimate the impact on costs, but of course it might affect the efficacy as well. So if you’re looking at offsetting disease-related costs, you might want to consider that as well. That’s a bit tricky, but you need to think about it. 

For the reporting format I’m really not going to explain very much. Basically we say you should present detailed information on the main parts of what you’ve done with tables. You should present all model assumptions, all input values, and results you should present with disaggregated outputs. So that if you’re presenting it in a print form rather than presenting it in a computer. The thing that I want you to look at when you look at the report is the reporting combined cost-effectiveness and budget impact analyses. We had a real concern with these because apart from the fact that often when these are reported or even done and not published, the budget impact analyses are often done incorrectly in the sense that their focusing on a cohort rather than a population. But also what you find in most publications is that because of space limitations, the cost-effectiveness model gets described very thoroughly and the budget impact analysis doesn’t get much description at all and there’s very little sensitivity analysis. We really recommend publishing them separately. We’re not too keen on together. But there should be a full description even if you have to use a supplemental appendix. For the computer model you want to create a simple spreadsheet program if possible. You want to default input parameter values with a text description of each parameter and reference to data sources and any calculations. The user should be able to change all input parameter values, because the point is the framework and they’re going to have costs that are unique to them. They want to be able to change it, but of course you want them to be able to restore defaults if they want to. Present in tabular and graphical formats and different levels of aggregation. The user should be able to change the scope of the analysis including the time horizon and the cost components included. This slide shows how it was reviewed. We had not only the Task Force Report people, but we had some primary reviewer people with a lot of expertise in this area, again representing U.S., Europe, and Asia. It was also reviewed by the ISPOR Membership as well. So we had eighty-four people review it. It was a lot of work. I’m done and ready and happy to take any questions.

**Moderator:**Great. Thank you very much Dr. Mauskopf. We do have one question about U.S. payers requiring budget impact analyses. Do any U.S. payers currently require them? If so, which ones? 

**Dr. Josephine Mauskopf:**I think the AMCP Guidelines require an economic model. A lot of U.S. payers use that, and WellPoint also. They’ll all require economic models, but I’m not sure that they require a budget impact analyses. But what I’ve found is that some of them may require that, but even if they don’t, there’s a general sense that U.S. payers actually prefer budget impact analyses. It’s not so clear how useful or how interested they are always in cost-effectiveness analyses. So I would always do one for a U.S. payer even if they didn’t say they required it. Sometimes in the AMCP dossiers that we do, we only present a budget impact analysis and don’t present a cost-effectiveness analysis. But I don’t think that we ever just present a cost-effectiveness analysis without a budget impact analysis. So that’s not a very definite thing in terms of a requirement. I think usually what you submit to them is what a company wants to submit. But most companies I work with want to submit a budget impact analysis.

**Moderator:**Thank you. I don’t see any other questions from the participants right now and I would encourage you all to submit a question through the online interface. I myself do have a couple of other questions that I’ll post to you and we’ll see if any others trickle in. One of the questions is about the characteristics of eligible population. This is a slide that you presented maybe five or six ago. You had mentioned to get some information from efficacy data from clinical trials regarding the change and the size and the characteristics of the eligible population. I see cross-sectional data from registries and efficacy data from clinical trials. I’m wondering how much of an emphasis you would place on getting the data from a comparative effectiveness research versus efficacy data from randomized-controlled trials?

**Dr. Josephine Mauskopf:**Are you talking about data based studies for comparison of effectiveness research? 

**Moderator:**It could be that or it could be just observational data interventions that have more patient reported outcomes and collective as well.

**Dr. Josephine Mauskopf:**Okay. So what I was going to say is the problem with most data based studies is they don’t have data bases, administrative data bases especially as in electronic medical records, they don’t have a lot of information about the characteristics of the population in terms of disease severity. And the severity of the disease, certainly for a chronic illness, but even for an acute illness, makes a big difference for how much it cost to treat it. And you can’t usually get that information from a lot of data bases if you’re talking about perspective observational studies that have that sort of information included in them. One of the things that you do need to know for budget impact analysis when you’re estimating the eligible population is that you need to know is what proportions of it are needed and at what levels of severity. I can give you two examples of some budget impact models that I’ve done. If you’re doing for HIV infection, clearly the annual costs associated with treating someone with a very low CD4 cell count is much higher than the annual cost of treating someone with a high CD4 cell count. And similarly with schizophrenia if the annual costs of treating someone who has refractory disease and has to be institutionalized, it’s obviously a lot higher than someone who has occasional exasperation, and so it relapses. But it’s quite hard to find that information. You might go to a database and know how many people have a diagnosis code of schizophrenia, and you could find out if they were in the hospital the whole year. But for HIV you wouldn’t know what their CD4 cell count was unless it was in their electronic medical record. So a clinical trial or a registry will generally have that data. 

**Moderator:**Okay. Sure. And in the VA we have now a new corporate data warehouse that does have a lot of outcome data, so even our administrative data-based analyses are able to pull some outcomes. So in that sense I guess we would be able to get some comparative effectiveness type work with patient outcomes or patient severity. In that situation, would you still prioritize the data from the clinical trial or do you feel the real world effectiveness would be a greater value to the budget impact analysis?

**Dr. Josephine Mauskopf:**Yeah and two bullets before I said the preferred approach is in the budget holders population directly. So the preferred approach would be to use your own data if you’ve got data that has that level of detail. I just didn’t put that bullet into the third one. Often you’re evaluating a budget impact analysis when the product has just been approved. So the problem with your own data is that you don’t have any data on the new product. You’ve got it on the old one. If the product’s been on the market for four or five years, you’ve got some good internal data. But you simply do not have data on what’s going to happen. The only data you have for what the new product’s going to do at a time you’re making a decision is from a clinical trial, “Do we put it on the formulary? Do we use it? What restrictions do we put on its use?” That’s why we suggested that maybe the first and only possible source for the new product and not for the old one. 

**Moderator:**Thank you. Another question is about the types of costs to include. For example, if we have a new drug that’s coming out that provides a curative therapy, which allows people to live longer without the original disease, and then through now living longer they use more health care, can you give us some feedback on what the panel thought about including those other subsequent health care costs associated with increased longevity?

**Dr. Josephine Mauskopf:**I would try to bring that up occasionally at our meetings, but somehow it never got traction to be discussed as to whether we should include those costs or not. I think the general sense was no we shouldn’t, even though it appears that if people don’t die of cancer then they live long enough and have a heart attack or something that you’re going to have to pay for that. I guess maybe a part of the sense is that they’re paying some premiums in that extra time period that they’re alive. So I just think we skipped that. I do bring it up occasionally. It’s not that people ignored it, but the people just didn’t want to make a definitive recommendation on. This comes into cost-effectiveness analysis too and there was a whole school of cost-effectiveness analyses who include if you increase life expectancy, but then you’ve got production versus consumption and you should include I know in Europe in Sweden they have expanded ways that you account for the cost of an extra year of life, the net cost. The people are producing but they’re also consuming. So it gets very complex and I think especially in the U.S. I’ve not seen that much done that way. But for all of the work that I did on HIV, certainly we always were estimating the total health care costs of the person in different stages of HIV when it was a more acute disease, but now it’s become a chronic disease. I think we’re still doing that for HIV, so the standard budget impact model for HIV does account for all the costs because more people with HIV are now surviving and getting heart disease and cancer. So in those budget impact models we do include just total cost for those people. I don’t believe there’s anything in the Task Force Report, and if there is it’s very non-prescriptive I think.

**Moderator:**Okay. Thanks for the feedback. A couple of other questions more on logistics. One of the participants would like to know the name of the book chapter you mentioned that you had recently written?

**Dr. Josephine Mauskopf:**Yeah, it’s *Difficult Budget Impact Analysis*. It’s in an Encyclopedia of Health Economics. It’s being edited by Tony Culyer, C – U – L – Y – E - R from the University of York and Elsevier is publishing it. That is a huge encyclopedia. I still haven’t figured out because I asked, “Can I use my inspection? How can I distribute it?” They weren’t very positive about that. But I’m sure it will be a reference work that you can access in the library. 

**Moderator:**Great. There is one other question about modeling. Can you share your thoughts on whether or not including incident cases in addition to prevalent cases while capturing the size of the eligible patient population, should you also get incident as well as prevalent cases?

**Dr. Josephine Mauskopf:**Yeah, because again when you’re modeling the population you want to consider it to be what we call an “open population.” So in any given year you’ve got two sets of people. You’ve got those who started the year already with the condition if it’s a chronic condition, and then you’ve got those who newly acquired the condition during the year. And both of those need to be included in your population size. And then you’ve got to subtract from that those who left the population during that year. Some of whom might have died, but some of whom might have left it because they were cured or because they moved out of that eligible population by getting a more serious disease. Sometimes people when they move to a later stage of the disease are no longer eligible for certain treatments. So you need to think about incident people, prevalent people that are already there, and people leaving, so that you have what we call an open population. And often to keep things simple, you might want to if you can assume that the number of people entering the population is equal to the number leaving because you’ve got a stable incident rate over time and the treatment over time has been stable, then your prevalent population will remain constant. So you don’t have to specifically count the incident and prevalent population. You can just look at how people are there and assume that it stays constant. But if you’re going to change the size of the population by changing the mortality rate or the disease progression rate, then you need to consider it more explicitly. 

**Moderator:**The last question is also just one that’s logistic. It’s asking about the new publication. I believe the participant is referring to the new ISPOR Task Force Report. When will it be available and will it be available in *Value in Health*?

**Dr. Josephine Mauskopf:**Yeah, it will be in Value in Health. I don’t know exactly when it’s in the January 2014 issue, but with journals, sometimes those come in February. So I’m not sure. They just sent me a copy, but I’m not allowed to circulate it, otherwise I would have been happy to share it with you. It means it’s all printed and it’s all proofed and it’s all done. So it should be online at any moment. I don’t know but I can let you know. I can find out but I don’t know. It should be very soon. 

**Moderator:**Great. Well thank you Dr. Mauskopf. That wraps up all of the questions that we have from our participants, and I want to thank you very much for a wonderful presentation. Again, we really appreciate your expertise and walking us through these new guidelines.

**Dr. Josephine Mauskopf:**My pleasure and good luck with them. If anyone has any questions, I get e-mails from people asking for advice and help on budget impact models and I’m happy to give it at any time, because I really enjoy it and they’re challenging. 

**Moderator:**Thank you. And for the audience this is a monthly series. Our next session in this series will be on February 19th and Ann Borzecki and Amresh Hanchate will be presenting Hospital Profiling with Enhanced Risk Adjustment Based on Laboratory Tests and Vital Signs Data. You all received in announcements to register for that earlier today. Take a look at your e-mails. And I will be putting up a feedback form for all of you in just a moment. If you could hang out and fill that out we would definetely appreciate it. Once again Dr. Mauskopf, thank you for presenting at today’s Cyberseminar. We very much appreciate the time you gave us today.  

**Dr. Josephine Mauskopf:**My pleasure. I’m going to hang up now.

**Moderator:**That’s just fine. Thank you.

**Dr. Josephine Mauskopf:**Thank you. Bye bye.

**Moderator:**Thanks. Bye. I hope that everyone is able to join us at a future HSR&D Cyberseminar. Thank you.

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