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An Introductive Analysis of the Timeframe from European Drugs Approval to the Sicilian Hospital Formulary Inclusion
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VA L U E I N H E A LT H 2 0 ( 2 0 1 7 ) A 3 9 9 – A 8 1 1
economic problems for the country which is the subject of the sanction, but also cause many other problems in other sanctions such as health. For example, the recent international sanctions against Iran, resulted in many pharmaceutical shortage due to financial barriers restricted the procurement of medicines. However, there are short-cuts and solutions in the case of medicine shortages. So, Iran was highly successful to maintain its quality of the pharmaceutical services by applying economic resilience strategy. Methods: This paper, by reviewing historical data, aimed to evaluate the IFDA strategy in handling crisis during sanctions and recommended successful polices, which could improve access to medicines in such a situation. These policies may be also recommended to other countries in such a situation as effective solutions for today’s vulnerable economics situations. Results: While, the crisis reached its peak in 2012, to reduce the negative impact of the sanctions, some changes were proposed by the IFDA at different policymaking levels from parliament legislation to organizational procedures. Significant price reduction, decreasing market share of imported medicines, facilitating domestic manufacturing, attracting foreign investment for domestic manufacturing, managing drug shortages, and increasing financial protection of patients in general and patients with catastrophic payments in particular were the main achievements of the new strategy of the IFDA, leading to better accessibility and availability in the country. Conclusions: Iran’s experiences on the boost of domestic manufacturing and cost containment approaches so that it withstood and recovered pharmaceutical sector from negative impacts of the sanctions, while the health system was shocked by the international sanctions, could be a successful example of economic resilience strategies. PHP81 The Impact of Scottish Medicines Consortium (SMC) Process Changes on Acceptance Rates for New Medicines for End of Life and Very Rare Conditions McMurray M, Downes N, Brown A, Lee A, Macdonald A, O’Toole B Healthcare Improvement Scotland, Glasgow, UK
Objectives: The SMC advises NHS Scotland on the clinical and cost-effectiveness of all new medicines. Methods changes introduced in 2014 aimed to increase access to medicines used at the end of life and for very rare conditions. This research examines the impact of the changes on the acceptance rates for all full submissions. Methods: A quantitative analysis was undertaken of assessments under the new process up to May 2017. A direct comparison of acceptance rates before and after the process changes is not possible as new criteria for end of life (EoL) and medicines for very rare conditions were introduced in 2014. The acceptance rate for decisions published from January 2011 to December 2013 for orphan and cancer medicines (as a proxy measure) was compared with those for medicines assessed as end of life and orphan under the new processes (decisions published October 2014 - May 2017). The acceptance rates for all medicines during these time periods were also compared. Results: From January 2011 to December 2013 the acceptance rates were: for medicines assessed as a full submission 114/175 (65%); for cancer and orphan medicines 15/31 (48%). From October 2014 to May 2017 the acceptance rates were: for all medicines assessed 154/180 (86%); for medicines assessed under the new process 61/78 (78%). The acceptance rate for EoL medicines was 42/46 (91%) and for very rare conditions medicines it was 52/68 (76%). Note that medicines may be categorised as both EoL and for a very rare condition. Conclusions: These findings show that the changes have met the Scottish Government’s policy imperative of increasing access to new medicines used at the end of life and for very rare conditions that may not have been accepted under the previous assessment process. The analysis was limited by the inability to undertake a direct comparison.
HEALTH CARE USE & POLICY STUDIES – Formulary Development PHP82 An Introductive Analysis of the Timeframe from European Drugs Approval to the Sicilian Hospital Formulary Inclusion Aiello A1, D’Ausilio A1, Pizzimenti V2, Formica D2, Sultana J2, Lucchesi S3, Ientile V2, Toumi M4, Trifirò G3 1Creativ Ceutical, Milan, Italy, 2A.O.U. Policlinico “G. Martino”, Messina, Italy, 3University of Messina, Messina, Italy, 4Aix-Marseille University, Marseille, France
Objectives: Drugs centrally approved by the European Medicine Agency (EMA) are automatically granted marketing authorization by Italian Drug Agency (AIFA), which is responsible for pricing and reimbursement (P&R) negotiation at national level. In most Italian regions, a Drug Formulary Committee (DFC) must admit those drugs in the Regional Hospital Formulary before use at regional level. Some authors have argued that the timing from National to Regional formularies inclusion may lead to important disparities in patients’ access to new drugs. The aim of this work is to present data on the timeframe/lag time between EMA, AIFA and Sicilian DFC (SDFC) for newly approved drugs in Europe. Methods: Newly approved drugs, for which dossiers were presented to SDFC, were identified from 1st January 2013 to 1st April 2016. Publicly available sources were used to retrieve relevant information on approval decisions. Both median and mean values were used in the analysis to estimate the timeframe/lag time. Results: On 117 drugs, 100 newly approved medicines were included in the analysis while 17 were excluded due to missing data. The median lag time from EMA to AIFA (LTEA) was 448 days (1st quartile = 315 days; 3rd = 635 days) and 112 days (54 – 231) from AIFA to SDFC (LTAS). The data showed the presence of some outlier drugs, in particular for LTAS, which impacted results when considering mean values. Indeed, the mean value of LTEA was 484 days with a standard deviation (SD) of 225 days, but for LTAS it was 249 days with an almost double SD (400 days). Conclusions: Homogeneity lack on admission of new drugs in the regional formularies may lead to access disparity among patients across Italy. However, our analysis shows that such timeframe/lag time mostly depends on AIFA P&R negotiation at national level.
Health Care Use & Policy Studies – Health Care Costs & Management PHP83 Haemorrhoidal Artery Ligation Procedure For The Treatment of Symptomatic Grade II–III Heamorrhoids: A Trial-Based And Long-Term Model-Based Economic Evaluation Alshreef A1, Wailoo A2 1University of Sheffield, Sheffield, UK, 2EuroQol Office, Rotterdam, The Netherlands
Objectives: To evaluate the cost-effectiveness of haemorrhoidal artery ligation (HAL) procedure versus rubber band ligation (RBL) for the treatment of symptomatic grade II–III haemorrhoids using data from the HubBLe study, a UK-based, multicentre randomised controlled trial. Methods: An economic evaluation was undertaken from the UK National Health Service (NHS) perspective including short-term trial-based and long-term model-based analyses. In the primary trial-based analysis, a seemingly unrelated regression model was fitted for estimating the mean difference in total cost and quality-adjusted life-years (QALYs) over 12-month time horizon. Cost-effectiveness results were expressed in terms of cost per QALY gained and cost per recurrence avoided. A parametric analysis and various deterministic sensitivity analyses were performed to address uncertainty around the primary cost-effectiveness result. In the long-term analysis, a three-health-state Markov model was built to extrapolate the analysis for a 4-year time horizon using data from HubBLe (costs, utilities and recurrence) and external studies (long-term recurrence). A probabilistic sensitivity analysis was performed to address uncertainly around the long-term cost-effectiveness result. Results: In the short-term trial-based analysis, the difference in total mean cost for HAL versus RBL was £1027 (95% confidence interval [CI], £782–£1272), p< 0.001). The difference in mean QALYs was 0.01 (95% CI, -0.02–0.04). This led to an incremental cost-effectiveness ratio (ICER) of £104,427 per QALY gained. The cost per recurrence avoided was £4882 (95% CI, £3628–£6135). The primary cost-effectiveness results were robust to all sensitivity analyses. In the long-term analysis, the probabilistic ICER was of £21,798 per QALY, generated from an incremental total mean cost of £1125 (95% CI, £1117–£1133) and incremental mean QALYs of 0.05 (95% CI, 0.048– 0.055). Conclusions: HAL procedure was not cost-effective compared with RBL for the treatment of symptomatic grade II–III haemorrhoids at a cost-effectiveness threshold of £20,000 per QALY. PHP84 Empirical Studies On The Economic Value Of A Statistical Life Year (VSLY) In Europe: What Do They Tell US? Schlander M1, Schaefer R2, Schwarz O3 of Heidelberg/German Cancer Research Center (DKFZ), Heidelberg, Germany, 2Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany, 3Hochschule Heilbronn, Wiesbaden, Germany
1University
Objectives: Health economists and health care policy makers often conceptualize value for money in terms of cost effectiveness or cost per (quality-adjusted) life year gained. Whether used in isolation or alongside further drivers of social value (such as priority for younger or more severely impaired patient groups), for example within a multi-criteria decision analysis framework, a benchmark or anchor “value of a statistical life year” (VSLY) will be required. Ideally, the VSLY should be based on the observed preferences of the population(s) in question. Here we report insights from a systematic review of empirical European economic studies, which were published during the last two decades (i.e., from 1995 to 2015). Methods: Our systematic literature search (using the EconBiz and EconLit databases) identified 41 European studies reporting original data, yielding 49 estimates for the value of a statistical life (VSL). We classified studies by methodology (revealed preference, RP; stated preference, SP: contingent valuation, CV; discrete choice experiment, DCE; cross-sectional or panel analysis). We transformed VSL estimates into VSLY expressed in year 2014 Euros, using the life expectancy of the populations studied, a discount rate of 3%, the national CPI for inflating, and purchasing power parities for currency conversion. We calculated confidence intervals by means of nonparametric bootstrapping. Results: The European median VSLY was € 158,448. Our VSLY estimates showed large heterogeneity by methodology (median: RP studies, € 231,422; DCE, € 187,857; CV, € 137,413; we did not identify studies using the human capital approach) and by regional origin (continental Europe, € 158,448; United Kingdom, € 117,956; Nordic countries, € 161,052). In contrast to the larger worldwide dataset, differences by method and regional origin were not statistically significant in our European sample. Conclusions: Our results suggest that the empirical willingness-to-pay for a statistical life year might be substantially higher than the thresholds currently used by the international HTA community. PHP85 Cost-Utility Analysis Of Rosuvastatin Compared To Atorvastatin In Primary Prevention Of Myocardial Infarction Among HIV-Positive Patients On Haart In Uganda Serunjogi B, Lamorde M Infectious Diseases Institute, Kampala, Uganda
Objectives: Cardiovascular disease is the leading cause of death worldwide. Three-quarters of these occur in low-middle income countries. HIV-positive patients are living longer because of anti-retroviral treatment (ART). However, the HIV infection and the ART has put them at twice the risk of cardiovascular disease events compared to non-HIV-infected people. Statins have been proved to be both effective and cost-effective in reducing this risk. This research aims to carry out a Cost-utility analysis of Statins for primary prevention of myocardial infarction in this group of people, who have double the baseline risk than the general population. Methods: A Markov model was used to assess the cost-effectiveness of Rosuvastatin compared to Atorvastatin. A hypothetical cohort of 1000 HIV-positive patients, stable on ART, was simulated through 4 Markov states over a lifetime. The treatment effectiveness and cost parameters were derived from published studies. The primary outcomes are QALYs, and ICER. A deterministic and probabilistic
VA L U E I N H E A LT H 2 0 ( 2 0 1 7 ) A 3 9 9 – A 8 1 1
economic problems for the country which is the subject of the sanction, but also cause many other problems in other sanctions such as health. For example, the recent international sanctions against Iran, resulted in many pharmaceutical shortage due to financial barriers restricted the procurement of medicines. However, there are short-cuts and solutions in the case of medicine shortages. So, Iran was highly successful to maintain its quality of the pharmaceutical services by applying economic resilience strategy. Methods: This paper, by reviewing historical data, aimed to evaluate the IFDA strategy in handling crisis during sanctions and recommended successful polices, which could improve access to medicines in such a situation. These policies may be also recommended to other countries in such a situation as effective solutions for today’s vulnerable economics situations. Results: While, the crisis reached its peak in 2012, to reduce the negative impact of the sanctions, some changes were proposed by the IFDA at different policymaking levels from parliament legislation to organizational procedures. Significant price reduction, decreasing market share of imported medicines, facilitating domestic manufacturing, attracting foreign investment for domestic manufacturing, managing drug shortages, and increasing financial protection of patients in general and patients with catastrophic payments in particular were the main achievements of the new strategy of the IFDA, leading to better accessibility and availability in the country. Conclusions: Iran’s experiences on the boost of domestic manufacturing and cost containment approaches so that it withstood and recovered pharmaceutical sector from negative impacts of the sanctions, while the health system was shocked by the international sanctions, could be a successful example of economic resilience strategies. PHP81 The Impact of Scottish Medicines Consortium (SMC) Process Changes on Acceptance Rates for New Medicines for End of Life and Very Rare Conditions McMurray M, Downes N, Brown A, Lee A, Macdonald A, O’Toole B Healthcare Improvement Scotland, Glasgow, UK
Objectives: The SMC advises NHS Scotland on the clinical and cost-effectiveness of all new medicines. Methods changes introduced in 2014 aimed to increase access to medicines used at the end of life and for very rare conditions. This research examines the impact of the changes on the acceptance rates for all full submissions. Methods: A quantitative analysis was undertaken of assessments under the new process up to May 2017. A direct comparison of acceptance rates before and after the process changes is not possible as new criteria for end of life (EoL) and medicines for very rare conditions were introduced in 2014. The acceptance rate for decisions published from January 2011 to December 2013 for orphan and cancer medicines (as a proxy measure) was compared with those for medicines assessed as end of life and orphan under the new processes (decisions published October 2014 - May 2017). The acceptance rates for all medicines during these time periods were also compared. Results: From January 2011 to December 2013 the acceptance rates were: for medicines assessed as a full submission 114/175 (65%); for cancer and orphan medicines 15/31 (48%). From October 2014 to May 2017 the acceptance rates were: for all medicines assessed 154/180 (86%); for medicines assessed under the new process 61/78 (78%). The acceptance rate for EoL medicines was 42/46 (91%) and for very rare conditions medicines it was 52/68 (76%). Note that medicines may be categorised as both EoL and for a very rare condition. Conclusions: These findings show that the changes have met the Scottish Government’s policy imperative of increasing access to new medicines used at the end of life and for very rare conditions that may not have been accepted under the previous assessment process. The analysis was limited by the inability to undertake a direct comparison.
HEALTH CARE USE & POLICY STUDIES – Formulary Development PHP82 An Introductive Analysis of the Timeframe from European Drugs Approval to the Sicilian Hospital Formulary Inclusion Aiello A1, D’Ausilio A1, Pizzimenti V2, Formica D2, Sultana J2, Lucchesi S3, Ientile V2, Toumi M4, Trifirò G3 1Creativ Ceutical, Milan, Italy, 2A.O.U. Policlinico “G. Martino”, Messina, Italy, 3University of Messina, Messina, Italy, 4Aix-Marseille University, Marseille, France
Objectives: Drugs centrally approved by the European Medicine Agency (EMA) are automatically granted marketing authorization by Italian Drug Agency (AIFA), which is responsible for pricing and reimbursement (P&R) negotiation at national level. In most Italian regions, a Drug Formulary Committee (DFC) must admit those drugs in the Regional Hospital Formulary before use at regional level. Some authors have argued that the timing from National to Regional formularies inclusion may lead to important disparities in patients’ access to new drugs. The aim of this work is to present data on the timeframe/lag time between EMA, AIFA and Sicilian DFC (SDFC) for newly approved drugs in Europe. Methods: Newly approved drugs, for which dossiers were presented to SDFC, were identified from 1st January 2013 to 1st April 2016. Publicly available sources were used to retrieve relevant information on approval decisions. Both median and mean values were used in the analysis to estimate the timeframe/lag time. Results: On 117 drugs, 100 newly approved medicines were included in the analysis while 17 were excluded due to missing data. The median lag time from EMA to AIFA (LTEA) was 448 days (1st quartile = 315 days; 3rd = 635 days) and 112 days (54 – 231) from AIFA to SDFC (LTAS). The data showed the presence of some outlier drugs, in particular for LTAS, which impacted results when considering mean values. Indeed, the mean value of LTEA was 484 days with a standard deviation (SD) of 225 days, but for LTAS it was 249 days with an almost double SD (400 days). Conclusions: Homogeneity lack on admission of new drugs in the regional formularies may lead to access disparity among patients across Italy. However, our analysis shows that such timeframe/lag time mostly depends on AIFA P&R negotiation at national level.
Health Care Use & Policy Studies – Health Care Costs & Management PHP83 Haemorrhoidal Artery Ligation Procedure For The Treatment of Symptomatic Grade II–III Heamorrhoids: A Trial-Based And Long-Term Model-Based Economic Evaluation Alshreef A1, Wailoo A2 1University of Sheffield, Sheffield, UK, 2EuroQol Office, Rotterdam, The Netherlands
Objectives: To evaluate the cost-effectiveness of haemorrhoidal artery ligation (HAL) procedure versus rubber band ligation (RBL) for the treatment of symptomatic grade II–III haemorrhoids using data from the HubBLe study, a UK-based, multicentre randomised controlled trial. Methods: An economic evaluation was undertaken from the UK National Health Service (NHS) perspective including short-term trial-based and long-term model-based analyses. In the primary trial-based analysis, a seemingly unrelated regression model was fitted for estimating the mean difference in total cost and quality-adjusted life-years (QALYs) over 12-month time horizon. Cost-effectiveness results were expressed in terms of cost per QALY gained and cost per recurrence avoided. A parametric analysis and various deterministic sensitivity analyses were performed to address uncertainty around the primary cost-effectiveness result. In the long-term analysis, a three-health-state Markov model was built to extrapolate the analysis for a 4-year time horizon using data from HubBLe (costs, utilities and recurrence) and external studies (long-term recurrence). A probabilistic sensitivity analysis was performed to address uncertainly around the long-term cost-effectiveness result. Results: In the short-term trial-based analysis, the difference in total mean cost for HAL versus RBL was £1027 (95% confidence interval [CI], £782–£1272), p< 0.001). The difference in mean QALYs was 0.01 (95% CI, -0.02–0.04). This led to an incremental cost-effectiveness ratio (ICER) of £104,427 per QALY gained. The cost per recurrence avoided was £4882 (95% CI, £3628–£6135). The primary cost-effectiveness results were robust to all sensitivity analyses. In the long-term analysis, the probabilistic ICER was of £21,798 per QALY, generated from an incremental total mean cost of £1125 (95% CI, £1117–£1133) and incremental mean QALYs of 0.05 (95% CI, 0.048– 0.055). Conclusions: HAL procedure was not cost-effective compared with RBL for the treatment of symptomatic grade II–III haemorrhoids at a cost-effectiveness threshold of £20,000 per QALY. PHP84 Empirical Studies On The Economic Value Of A Statistical Life Year (VSLY) In Europe: What Do They Tell US? Schlander M1, Schaefer R2, Schwarz O3 of Heidelberg/German Cancer Research Center (DKFZ), Heidelberg, Germany, 2Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany, 3Hochschule Heilbronn, Wiesbaden, Germany
1University
Objectives: Health economists and health care policy makers often conceptualize value for money in terms of cost effectiveness or cost per (quality-adjusted) life year gained. Whether used in isolation or alongside further drivers of social value (such as priority for younger or more severely impaired patient groups), for example within a multi-criteria decision analysis framework, a benchmark or anchor “value of a statistical life year” (VSLY) will be required. Ideally, the VSLY should be based on the observed preferences of the population(s) in question. Here we report insights from a systematic review of empirical European economic studies, which were published during the last two decades (i.e., from 1995 to 2015). Methods: Our systematic literature search (using the EconBiz and EconLit databases) identified 41 European studies reporting original data, yielding 49 estimates for the value of a statistical life (VSL). We classified studies by methodology (revealed preference, RP; stated preference, SP: contingent valuation, CV; discrete choice experiment, DCE; cross-sectional or panel analysis). We transformed VSL estimates into VSLY expressed in year 2014 Euros, using the life expectancy of the populations studied, a discount rate of 3%, the national CPI for inflating, and purchasing power parities for currency conversion. We calculated confidence intervals by means of nonparametric bootstrapping. Results: The European median VSLY was € 158,448. Our VSLY estimates showed large heterogeneity by methodology (median: RP studies, € 231,422; DCE, € 187,857; CV, € 137,413; we did not identify studies using the human capital approach) and by regional origin (continental Europe, € 158,448; United Kingdom, € 117,956; Nordic countries, € 161,052). In contrast to the larger worldwide dataset, differences by method and regional origin were not statistically significant in our European sample. Conclusions: Our results suggest that the empirical willingness-to-pay for a statistical life year might be substantially higher than the thresholds currently used by the international HTA community. PHP85 Cost-Utility Analysis Of Rosuvastatin Compared To Atorvastatin In Primary Prevention Of Myocardial Infarction Among HIV-Positive Patients On Haart In Uganda Serunjogi B, Lamorde M Infectious Diseases Institute, Kampala, Uganda
Objectives: Cardiovascular disease is the leading cause of death worldwide. Three-quarters of these occur in low-middle income countries. HIV-positive patients are living longer because of anti-retroviral treatment (ART). However, the HIV infection and the ART has put them at twice the risk of cardiovascular disease events compared to non-HIV-infected people. Statins have been proved to be both effective and cost-effective in reducing this risk. This research aims to carry out a Cost-utility analysis of Statins for primary prevention of myocardial infarction in this group of people, who have double the baseline risk than the general population. Methods: A Markov model was used to assess the cost-effectiveness of Rosuvastatin compared to Atorvastatin. A hypothetical cohort of 1000 HIV-positive patients, stable on ART, was simulated through 4 Markov states over a lifetime. The treatment effectiveness and cost parameters were derived from published studies. The primary outcomes are QALYs, and ICER. A deterministic and probabilistic