ExecutiveDirector, RTI Health Solutions, 3040 CornwallisRoad, Research TrianglePark, North Carolina, USA 27709 Tel.: +1 919 541 6996 Fax: +1 919 541 7222 jmauskopf@rti.org www.rtihs.org FutureDrugsLtd. All rightsreserved. ISSN 1473-7167 1 Why study pharmacoeconomics? Pharmacoeconomics research is a flourishing industry with many practitioners, a large research and applications agenda, several journals and flourishing professional societies. Why study pharmacoeconomics?Well the first reason is so that you will know how to spell it!!! You will not get any help from your spell- checker software as I realized when I sat down to write this editorial! Before I try to give a more serious answer, I need to start with a little history when wasthe term pharmacoeconomics first coined and why did it catch on? and an attempt at adefinition of theterm what ispharmacoeconomics? Probably one of the first times that the term pharmacoeconomics was used in a public forum was in 1986, at a meeting of Pharma- cists in Toronto, Canada, when Ray Townsend, from the Upjohn Company, used the term in a presentation. Ray and a few oth- ers had been performing studies using the term pharmacoeconomics within the pharmaceuti- cal industry since the early eighties. Today, pharmac- oeconomics research is a flour- ishing industry with many practi- tioners, a large research and applications agenda, several jour- nals and flourishing professional societies including the International Society for Phar- macoeconomics and Outcomes Research. What had happened just prior to 1986 that prompted Ray to coin the term?Why did the term catch on?Let me give you a little more history, personal this time, that might suggest the answers to these questions. Around this same time, my career in pharmacoeconomics started with a study of the cost-effectiveness of AZT for treatment of persons with AIDS. This was a modeling study based on data from a clinical trial, ACTG 002. The cost for AZT when it was first introduced was $10,000 for 1 year of treatment. The clinical data showed that the drug would increase life expectancy for persons with AIDS by approximately 1 year. My model therefore showed that the incremental cost-effectiveness ratio for AZT was $10,000, which was then and is now con- sidered to be good value for money. However, the idea of having to pay $10,000 each year for a drug was shocking to persons with AI DS and to the public. This was especially so in the US where at that time many people did not have any insur- ance coverage for outpatient drugs. This resulted in a lot of negative publicity for Bur- roughs Wellcome Co. and for the whole pharmaceutical industry. The industry knew that AZT was just the beginning. Drug development costs had risen because of increased regula- tory requirements to protect the pub- lic from ineffec- tive or unsafe drugs following the thalidomide tragedy. New techniques for drug design and new types of drugs engineered using the tech- niquesof biotechnology, meant that the cost of manufacturing drugswasalso likely to rise. Both of these increasesin costsmeant that drug com- panieswould likely be charging more for drugs that successfully made it to market, in order to obtain an acceptable return on their higher investment. I think that these changes may be some of the reasons that the new disipline of pharmacoeconomicsflourished. Rays initial definition of pharmacoeconom- ics was the description and analysis of the costs of drug therapy to healthcare systems and society. Later this definition was Measurement and presentation of a comprehensive set of outcomes that describe the consequences of the use of a new drug. M a uskop f 2 Expert Rev. PharmacoeconomicsOutcomesRes. 1(1), (2001) amended by Ray and some of his colleagues to include both the costs and quality of life consequences associated with the use of a new drug therapy. My own definition of pharmac- oeconomics is the measurement and presentation of a compre- hensive set of outcomes that describe the consequences of the use of a new drug. These outcomes include the impact of the new drug on healthcare costs and individual quality of life but also include the impact of the new drug on individual func- tional status and productivity, as well as the drugs impact on caregivers and families and society. Since 1986, many methodological advances have been made that provide the tools that we need to measure this comprehensive set of outcomes. These advances include strategies for collecting phar- macoeconomic data during clinical trials. Profile meas- ures for measuring changes in general and disease-spe- cific health status have been developed and validated using psychometric techniques. Economic techniques for eliciting preferences have been applied to the measurement of drug value. Statistical techniques have been developed, using both frequentist and Bayesian approaches, for design- ing trials and analyzing both trial and observational data for use in pharmacoeconomic evaluations. The reference case for cost-effectiveness ratios has been developed and methods for estimating confidence limits around these ratios proposed. The concept of budget and population impact estimates has been introduced. All of these advances have given us tools that we need to measure the comprehensive set of outcomes associated with a new drug. They now form the body of knowledge that is pharmacoeconomics. Who should study pharmacoeconomics? Anyone who is involved with healthcare decision-making, including suppli- ers of healthcare products, such as pharmaceutical compa- nies or medical device companies, the consumers of such products and the healthcare providers. All should know enough about the different elements of a pharmacoeco- nomic analysis to be able to understand the results of the analysis. Those who plan to perform the pharmacoeconomic analyses should have a more extensive training. How can you become a practising pharmacoeconomist? What sort of professional training is needed?You need training in Pharmacy, Medicine, Psychometrics, Economics, Statistics, Epidemiology, Decision Analysis, Survey Techniques, Opera- tions Research and a whole host of other disciplines. Clearly, it is not practical to obtain formal training in all of these disci- plines, so most practitioners get training in one or two of the key disciplines and then learn something about the other disci- plines on-the-job. In practice, most pharmacoeconomic projects are completed by a team of people who have training in the different disciplines so that the results of a pharmac- oeconomic study are not dependent on one person being an expert in all the relevant disciplines. How can you learn to be a consumer of pharmacoeconomic studies?For those who only need to understand the results of pharmacoeconomic studies, the simplest way to learn this skill would be by taking a single pharmacoeconomics course over a 1 year time period during university training in the primary discipline or asa postgraduate course. This course would cover the main topics in each of the disciplines. Many such courses have been developed. Why should these people all study pharmacoeconomics? The main reason for studying pharmacoeconomics is to be able to estimate and understand the full impact of a new ther- apy. This impact will be on the individuals health and safety but also on their use of healthcare services and the cost of healthcare, on their quality of life and functional status, on their families and friends and on society as a whole. Phar- macoeconomics extends the measurement of the impact of a new drug beyond safety and efficacy to all these additional outcomes. If we truly want to understand the value of a new drug, we need to understand its impacts on this broad range of outcomes. The price of the drug will then determine whether the drug is good value for money, depending on its impact on this broad range of out- comes and on the value that the decision-maker places on these changes in outcomes. Why do we care about the impact of a new drug?Primarily we care because the healthcare decision-makers (governments, doctors, payers, patients) all care. They are vitally interested in better treatments for our diseases as well as better preventive strategies. However, they have realized that, as we have become more successful in preventing and treating disease, we have had to devote more and more of our scarce resources to health- care. Thus, we now need to question more closely than ever whether each new drug is good value for money compared with current treatment. Moreover, as countries try to control the rising costs of healthcare in their aging populations, they have introduced requirements for economic evaluations of new drugs. They are all asking the question, is the new drug good value for money and what is society willing to pay for it? Guidelines for how to perform these economic analyses and how to present the information were developed first in Aus- tralia and Canada and then in many European countries and, most recently, by managed care organizations in the US. All of these guidelines require that a pharmaceutical company esti- mate a comprehensive set of changes in outcomes associated with the new drug. The careful study of the body of knowledge that ispharmac- oeconomicsand further development of that knowledge are par- ticularly important because of the many controversies still remaining in the use of pharmacoeconomic analysesfor the allo- cation of funding to the use of new treatments. For example, there isno agreement on what should be the benchmark value for The main reason for studying pharmacoeconomics is to be able to estimate and understand the full impact of a new therapy. Why study p ha rma c oe c onomic s? www.future-drugs.com 3 a cost-effectivenessratio and how to restrict delivery of healthcare that hasa cost-effectivenessratio above the threshold value. There is still no agreement on how to incorporate uncertainty of the resultsinto decision-making and how to makedecisionswhen only poor quality data are available. The choiceof comparator, popula- tion subgroups, time horizon and perspective all make a big difference to the results and require many more data than are generally available. It isalso hard to find a way to present the data from a pharmacoeconomic anal- ysisto decision-makersthat addressesthese issueswithout making it completely incomprehensible to them. Finally, there are contro- versiesabout who should fund the studiesand whether publication biasresultsin inefficient use of healthcare resources. For all of these reasons, it is critical that all healthcare decision-makers have as much education in pharmacoeco- nomics as possible. This will ensure that the methodological and policy uncertainties still present in the pharmacoeco- nomic analyses are properly accounted for when the results of such analyses are used to help make healthcare decisions. Formal training in pharmacoeconomics will ensure that the methodologi cal and policy uncertainties still present i n the pharmacoeconomic analyses are properly accounted for when the results of such anal- yses are used to help make healthcare decisions. Finally, to illustrate the fields complexity, how many pharmacoeconomists does it take to change a light bulb? The answer is four! One to estimate the cost of the new light bulb, one to estimate the life expectancy of this new light bulb, one to estimate the quality of life associated with the light from the new light bulb and one to package the infor- mation so that it convi nces the healthcare decision-maker to take out the old light bulb and put in the new one! It is critical that all healthcare decision-makers have as much education in pharmacoeconomics as possible...
(Metals and Related Substances in Drinking Water Research Rep) M. Ferrante, G. Oliveri Conti, Z. Rasic-Milutinovic-Health Effects of Metals and Related Substances in Drinking Water-IWA Publishing (201