Drug pricing for a novel treatment for wet macular degeneration: using incremental cost-effectiveness ratios to ensure societal value. Economic Evaluation of a Problem Solving Intervention to Prevent Recurrent Sickness Absence in Workers with Common Mental Disorders Economic evaluation of an intensive home visiting programme for vulnerable families: a cost-effectiveness analysis of a public health intervention. The Cost-Effectiveness and Cost-Benefit of Screening and Brief Intervention for Unhealthy Alcohol Use in Medical Settings More money, fewer lives: the cost effectiveness of welfare reform in the United States. A pharmacoeconomic comparison of UFT and 5-FU chemotherapy for colorectal cancer in South America. Economic Evaluation of Bone Morphogenetic Protein Versus Autogenous Iliac Crest Bone Graft in Single-Level Anterior Lumbar Fusion: An Evidence-Based Modeling Approach Economic evaluation of a workplace intervention for sick-listed employees with distress Cost-effectiveness of pazopanib: an example of improved transparency and accessibility of industry-sponsored economic evaluations through publication in peer-reviewed journals.
OBJECTIVE Health economic models can assist policy-makers in determining the value of novel treatments from the viewpoint of society . In this context , value is defined as the benefit of treatment , given its cost . A new treatment for wet age-related macular degeneration ( AMD ) , juxtascleral administration of anecortave acetate , 15 mg for depot suspension ( Retaane ) , is now in a late-phase clinical trial . In a theoretical analysis , we sought to determine the cost at which this treatment might offer economic value to society , using incremental cost-effectiveness ratios ( ICERs ) . METHODS A series of 1-year cost-utility models was created for the investigational treatment and st and ard treatment ( photodynamic therapy [ PDT ] with verteporfin [ Visudyne ] ) . Value to society was defined in terms of theoretical associated ICERs ( in US dollars ) : $ 100,000 per quality -adjusted life-year ( QALY ) , $ 50,000/QALY , $ 20,000/QALY and $ 0/QALY , the point of economic indifference . Models were created from the societal perspective and included a patient-derived utility assessment involving regression equations to estimate time trade-off preferences , event probabilities derived from a r and omized clinical trial comparing the safety and efficacy of anecortave administration and PDT with verteporfin , decision analysis and relevant costing information . RESULTS An ICER of $ 100,000/QALY would be associated with an anecortave cost of $ 3022/vial , an ICER of $ 50,000/QALY with an anecortave cost of $ 2986/vial and an ICER of $ 20,000/QALY with an anecortave cost of $ 2964/vial . The point of economic indifference between anecortave administration and st and ard therapy would occur with an anecortave cost of $ 2950/vial . INTERPRETATION In theory , an anecortave cost of $ 2986/vial is associated with an ICER of $ 50,000/QALY , the threshold used by many health technology assessment and reimbursement agencies Objectives Workers with common mental disorders ( CMDs ) frequently experience recurrent sickness absence  but scientifically evaluated interventions to prevent recurrences are lacking . The objectives of this study are to evaluate the cost-effectiveness and cost-benefit of a problem solving intervention aim ed at preventing recurrent sickness absence in workers with CMDs compared to care as usual  . Methods An economic evaluation was conducted alongside a cluster-r and omised controlled trial with 12 months follow-up . Treatment providers were r and omised to either a 2-day training in the SHARP-at work intervention , i.e. a problem solving intervention , or care as usual . Effect outcomes were the incidence of recurrent sickness absence and time to recurrent sickness absence . Self-reported health care utilisation was measured by question naires . A cost-effectiveness analysis ( CEA ) from the societal perspective and a cost-benefit analysis ( CBA ) from the employer ’s perspective were conducted . Results The CEA showed that the SHARP-at  work intervention was more effective but also more expensive than care as usual . The CBA revealed that employer ’s occupational health care costs were significantly higher in the intervention group compared to care as usual . Overall , the SHARP-at work intervention showed no economic benefit compared to care as usual . Conclusions As implementation of the SHARP-at work intervention might require additional investments , health care policy makers need to decide if these investments are worthwhile considering the results that can be accomplished in reducing recurrent sickness absence BACKGROUND Recent review s have shown that home visiting programmes that address parenting have the potential to improve long term health and social outcomes for children . However there are few studies exploring the cost-effectiveness of such interventions . The objective of this study was to evaluate the cost-effectiveness of an intensive home visiting programme directed at vulnerable families during the antenatal and postnatal periods . METHODS The design was an economic evaluation alongside a multicentre r and omized controlled trial , in which 131 eligible women were r and omly allocated to receive 18 months of intensive home visiting ( n=67 ) or st and ard services ( n=64 ) . Due to the public health nature of the intervention a cost-effectiveness analysis was undertaken from a societal perspective . RESULTS The mean ' societal costs ' in the control and intervention arms were 3874 pounds and 7120 pounds , respectively , a difference of 3246 pounds ( p<0.000 ) . The mean ' health service only ' costs were 3324 pounds and 5685 pounds respectively , a difference of 2361 pounds ( p<0.000 ) . As well as significant improvements in maternal sensitivity and infant cooperativeness there was also a non-significant increase in the likelihood of the intervention group infants being removed from the home due to abuse and neglect . These incremental benefits were delivered at an incremental societal cost of 3246 pounds per woman . CONCLUSIONS The results of the study provide evidence to suggest that , within the context of regular home visits , specially trained home visitors can increase maternal sensitivity and infant cooperativeness and are better able to identify infants in need of removal from the home for child protection . The extent to which these benefits are ' worth ' the societal cost of 3246 pounds per woman however is a matter of judgment SUMMARY Economic evaluation can be a valuable tool for assessing the efficiency and value of health care programs . To examine the literature on the economic evaluation of  alcohol screening and brief intervention in medical setting s , relevant studies were identified in the MEDLINE data base ( 1966 through November 2006 ) and by h and - search ing the references of identified articles and relevant journals . The 15 identified studies used a range of economic evaluation methods , including cost analysis , cost-benefit , cost-effectiveness , and cost-utility . Nearly all of the studies supported the use of alcohol screening and brief intervention . The studies that prospect ively collected cost and effect data and /or conformed closely to method ological guidelines demonstrated a strong economic benefit of alcohol screening and brief intervention when compared to usual care . Overall , the review ed studies support alcohol SBI in medical setting s as a wise use of health care re sources and illustrate the usefulness of economic evaluation for assessing alcohol prevention and treatment programs OBJECTIVES We evaluated the economic benefits of  Temporary Assistance to Needy Families ( TANF )  relative to the previous program , Aid to Families with Dependent Children ( AFDC ) . METHODS We used pooled mortality hazard ratios from 2 r and omized controlled trials-Connecticut Jobs First and the Florida Transition Program , which had follow-up from the early and mid-1990s through December 2011- and previous estimates of health and economic benefits of TANF and AFDC .  We entered them into a Markov model to evaluate TANF 's economic benefits relative to AFDC and weigh them against the potential health threats of TANF . RESULTS Over the working life of the average cash assistance recipient , AFDC would cost approximately $ 28000 more than TANF from the societal perspective . However , it would also bring 0.44 additional years of life . The incremental cost effectiveness of AFDC would be approximately $ 64000 per life-year saved relative to TANF . CONCLUSIONS AFDC may provide more value as a health investment than TANF . Additional attention given to the neediest US families denied cash assistance could improve the value of TANF The escalating role played by managed care organizations in the health-care system is reflected in the increased dem and for cost-effectiveness analyses ( CEAs ) to assess the balance between economic impact and clinical efficacy . For example , the high incidence and costs associated with colorectal cancer in Latin America calls for a comprehensive economic evaluation to ensure appropriate allocation of limited health-care funds . In addition , the current call for a " societal " perspective of such analyses indicates the need for increased consideration of the concerns of both patient and health-care provider . The introduction of oral tegafur and uracil ( UFT ) provided the opportunity to evaluate the pharmacoeconomic advantage of the new agent compared with the st and ard fluorouracil ( 5-FU ) . Results of this study indicated an economic advantage for oral UFT vs a 5-FU-based regimen in the treatment of colorectal cancer in Brazil and Argentina . It was further noted that the mild toxicity profile of UFT reduced both the number of clinic visits and the need for venipuncture procedures . Noting that oral UFT may have a positive impact on quality of life in addition to its estimated economic benefit , it was concluded that prospect i ve economic research and quality -of-life evaluations are needed to fully assess the pharmacoeconomic impact of oral UFT Study Design . Economic evaluation provides a framework to explicitly measure and compare the value of alternative medical interventions in terms of their clinical , health-related quality -of-life , and economic outcomes . Comput-erized economic models can help inform the design of future prospect i ve studies by identifying the cost-drivers , the most uncertain parameter estimates , and the parameters with the greatest impact on the results and inferences . Objective . An economic analysis of bone morphogenetic protein versus autogenous iliac crest bone graft for single-level anterior lumbar fusion poses several method ologic challenges . This article describes how such an economic evaluation may be framed and design ed , while enumerating challenges , offering some solutions , and suggesting an agenda for future research . Summary of Background Data . An evidence -based modeling approach can incorporate epidemiologic , clinical , and economic data from several sources including r and omized clinical trials , peer- review ed literature , and expert opinion . Sensitivity analyses can be conducted by varying key parameter estimates within a reasonable range to assess the impact on the results and inferences . Results . Preliminary results suggest that from a payer perspective , the upfront price of bone morphogenetic protein is likely to be entirely offset by reductions in the use of other medical re sources . That is , bone morphogenetic protein appears to be cost neutral . The cost offsets were attributable largely to prevention of pain and complications associated with autogenous iliac crest bone graft , as well as reduction of the costs associated with fusion failures . Conclusions . Future research should focus on quantifying the health-related quality -of-life impact of bone morphogenetic protein relative to autogenous iliac crest bone graft , as well as the impact on lost productivity Objectives To evaluate the cost effectiveness , cost utility and cost benefit of a workplace intervention compared with usual care for sick-listed employees with distress . Methods An economic evaluation was conducted alongside a r and omised controlled trial . Employees with distress and who were sick-listed for 2–8 weeks were r and omised to a workplace intervention ( n=73 ) or to usual care ( n=72 ) . The workplace intervention is a stepwise process involving the sick-listed employee and their supervisor , aim ed at formulating a consensus-based plan for return to work ( RTW ) . The effect outcomes were lasting RTW and quality -adjusted life years ( QALYs ) . Healthcare utilisation was measured over 12 months . Cost effectiveness analyses ( CEA ) and cost utility analyses ( CUA ) were conducted from the societal perspective and cost benefit analyses ( CBA ) from the employer perspective . Bootstrapping techniques were used to estimate cost and effect differences , related CIs , and cost effectiveness and cost utility ratios . Cost effectiveness planes were presented and subgroup analyses were performed . Results CEA and CUA revealed no statistically significant differences in lasting RTW , QALYs or costs . The CBA indicated a statistically significant higher cost of occupational health services in the workplace intervention group . The workplace intervention was not cost effective according to the CEA , CUA and CBA . Conclusions Widespread implementation of the workplace intervention for sick-listed employees with distress is not recommended because there was no economic benefit compared with usual care . Future trials should confirm if the workplace intervention is cost effective for the subgroup employees who intended to return to work despite symptoms . This trial has been registered at the Dutch National Trial Register IS RCT N92307123 We congratulate Amdahl et al.1 on publishing their paper about the cost-effectiveness of pazopanib compared with sunitinib in metastatic renal cell carcinoma ( mrcc ) in Canada . The article reports an analysis similar to the one su bmi tted to the pan-Canadian Oncology Drug Review to support the funding request for pazopanib in mrcc , up date d with final overall survival ( os ) data from the pivotal trial2 . Economic evaluations in oncology are increasingly important to guide both policy and practice . We commend the efforts of these authors to put their su bmi tted analyses into the public domain for transparency and to support and inform future research . The base-case analysis ( using the list prices of pazopanib and sunitinib ) by Amdahl et al.1 found that , compared with sunitinib , which is the st and ard of care for first-line therapy in mrcc , first-line pazopanib is likely cost-saving . The base case analysis also suggested that pazopanib might be slightly more effective than sunitinib numerically , with an incremental gain of 0.057 life-years and 0.059 quality -adjusted life-years . Before concluding that pazopanib is dominant ( that is , it provides more benefit for a lower cost ) with respect to sunitinib , it is important to review the clinical data comparing pazopanib and sunitinib in mrcc to assess the comparative efficacy and preference-based measures of health-related quality of life ( hrqol ) associated with those agents — that is , their health utilities — to examine the face validity of the economic model in its base case . The efficacy differences between the pazopanib and sunitinib in the comparz study 3 were not statistically significant , median progression-free survival for pazopanib and sunitinib being 8.4 months [ 95 % confidence interval ( ci ) : 8.3 to 10.9 months ] and 9.5 months ( 95 % ci : 8.3 to 11.1 months ) respectively , with a hazard ratio of 1.05 ( 95 % ci : 0.90 to 1.22 ) . The study met its primary endpoint of non-inferiority in progression-free survival . No os difference was observed in the comparz study , in which median os was 28.4 months for pazopanib ( 95 % ci : 26.2 to 35.6 months ) and 29.3 months for sunitinib ( 95 % ci : 25.3 to 32.5 months ) , with a hazard ratio of 0.91 ( 95 % ci : 0.76 to 1.08 ) . At the up date d analysis , os was similar in the two groups ( hazard ratio for pazopanib vs. sunitinib : 0.92 ; 95 % ci : 0.79 to 1.06 ; p = 0.24)2 . With similar survival results for pazopanib and sunitinib based on the comparz study , one might ask , “ How did the model project an incremental gain of 0.057 life-years in favour of pazopanib ? ” The model actually projected a shorter life expectancy while patients were taking pazopanib than while their counterparts were taking sunitinib ( 0.013 life-years lost in the progression-free state ) . However , the model also projected that , after patients progressed and stopped taking pazopanib , their life expectancy was longer than it was for patients who received sunitinib ( 0.070 life-years gained in the post-progression state ) . Together , the loss and the gain result ed in a net gain in life expectancy for pazopanib , implying that the survival benefit of pazopanib acted only after progression , when patients were no longer taking pazopanib . There is no biologic basis for that phenomenon , and it likely simply reflects small numerical differences between the pazopanib and sunitinib progression-free survival and os curves , without accounting for uncertainty . Interestingly , the model also predicted that pazopanib would be more effective in at least 80 % ( or possibly all ) of the simulations despite the nonsignificant result . Although the comparz study 3 collected some hrqol measures , it did not collect preference-based measures of hrqol to allow for the calculation of quality -adjusted life-years for the purpose of economic evaluation . The pisces study 4—a r and omized , double-blind trial with a crossover design ( that is , pazopanib→sunitinib vs. sunitinib→pazopanib)—had , as its primary endpoint , an examination of patient preference for either pazopanib or sunitinib , which suggested that patients preferred pazopanib over sunitinib . However , the study had a few limitations 5 , including a relatively high proportion of the r and omly assigned patients ( 33 % ) not being evaluable for the primary endpoint , and the analysis of the pazopanib preference being based on a “ modified ” intention-to-treat population . The EQ-5D ( EuroQoL Group , Rotterdam , Netherl and s ) was also collected as the preference-based measure of hrqol in the pisces study 4 , but the EQ-5D results would have been subject to similar limitations . The data from the pisces study were used in the su bmi ssion to the pan-Canadian Oncology Drug Review . In the up date d analysis 1 , the authors conducted a regression based on patients in a trial comparing pazopanib with placebo6  , assuming that the effect of adverse events ( aes ) were driving the differences in hrqol , regardless of actual treatment received . For the analysis , the aes were grouped by severity and whether the aes were more commonly occurring in the sunitinib or the pazopanib group of the comparz trial . Using that approach , the regression model found that the health utility decrement was larger for the group of aes more commonly occurring in patients who used sunitinib than for the remaining aes more frequently occurring in patients who used pazopanib in the comparz trial . The results of the regression model were combined with the incidence and duration of aes in each treatment arm to derive the mean utility values used in the model , producing mean utility values of 0.7089 ( st and ard error : 0.0193 ) for pazopanib and 0.6832 ( st and ard error : 0.0236 ) for sunitinib , an incremental difference of 0.0257 in the progression-free period1 . Given that the comparz trial demonstrated similar efficacy and a more favourable toxicity profile for pazopanib , it is not unreasonable that the point estimate of the utility score could slightly favour the pazopanib group . However , these mean utility values were not directly derived and might be neither statistically nor clinical ly different , especially because the difference was smaller than the reported estimates of minimal important differences for health utilities ( which are in the range of 0.05–0.08 for the EQ-5D)7,8 . Thus , it is somewhat difficult to conclude from those data that pazopanib is associated with better utility than sunitinib . It might be reasonable to conservatively expect that the utilities of pazopanib and sunitinib are fairly similar given the size of the st and ard errors of the two utilities , subject to a reasonable amount of uncertainty9,10 . Given that clinical trials did not demonstrate a statistically significant survival benefit or EQ-5D health utility benefit for pazopanib over sunitinib , we feel that an appropriate interpretation could be that the study findings suggest similar efficacy and health utility for the two drugs . Reasonably , the pan-Canadian Oncology Drug Review ’s Expert Review Committee also concluded that it is reasonable to assume similar efficacy and to focus on the cost comparison between pazopanib and sunitinib in this setting 11 . We appreciate that the sensitivity analyses by Amdahl et al.1 address all the considerations discussed here , including the importance of the relative price of the two drugs . The authors demonstrate as part of their additional analyses that — assuming similar efficacy , health utility , and daily price of the two therapies — only minor cost differences remain . Thus , interpreting the analyses in light of the evidence , it appears that these two options for first-line therapy in mrcc might be largely similar and , taking into account the relative prices of the two drugs , the hope would be that , after confidential negotiations , they will also be similar in cost . In that case , having both drugs available would give patients the option to choose based on individual preference and side effect profile , especially in the case of intolerance to one of the drugs , and would ensure that health care re sources are being efficiently used . The generalizability of the study findings remains to be seen in practice . Verification from experience is needed to know whether the comparative outcomes as laid out will be realized . In Canada , jurisdictions have an important opportunity and challenge to evaluate the real-world effectiveness and cost-effectiveness of therapies in clinical practice after drugs are funded . Moreover , post-market research can provide valuable insights into practice patterns when two alternatives are funded and can possibly inform continued uncertainties such as sequencing and long-term adverse effects . We applaud the approach of the authors to share their analyses with the broader clinical and research communities , and we encourage more authors of economic evaluations created and su bmi tted to review bodies ( for example , the Canadian Agency for Drugs and Technologies in Health , the pan-Canadian Oncology Drug Review ) to follow suit . We expect that this activity will build on the principle of transparency being increasingly championed by the review organizations and will improve the rigour of the economic evidence used to inform policy . Finally , we call on the community to continue to pursue both academic merit and clinical and policy relevance by supporting the availability of peer- review ed publications of economic evaluations to inform the reimbursement review process . Ideally , it would be helpful if economic evaluations were to be published before decision-making so as to engage more stakeholders in the discussion 12 . In doing so , the clinical and economic evidence bases supporting the health technology assessment processes in Canada might be brought into better alignment , improving the robustness of decision-making and ultimately helping health care systems in Canada to provide effective and appropriate care to patients
Overall , the cohort Markov models remain the most popular approach , followed by decision trees , Individual based model and System dynamics model ( SD ) .
Conclusions This review paper informs policy makers about the availability of modelling techniques that can be used to enhance the quality of economic evaluations for drug and alcohol treatment interventions
Background The overarching goal of health policies is to maximize health and societal benefits .
Economic evaluations can play a vital role in assessing whether or not such benefits occur .
This paper review s the application of modelling techniques in economic evaluations of drug and alcohol interventions with regard to ( i ) modelling paradigms themselves ; ( ii ) perspectives of costs and benefits and ( iii ) time frame .