- Email: [email protected]
PTH2: MALIGNANT RETROPERITONEAL TUMOURS OF CHILDREN: FIRST RESEARCH OF QUALITY OF LIFE OF 278 SURVIVED PATIENTS
403
Abstracts tal fees will necessarily increase tremendously; proven efficacy for these costs has not been provided in Greece yet.
PID6
TREATMENT OF ACUTE OTITIS MEDIA IN A MANAGED CARE POPULATION McLaughlin T1, Margraf T2, Okamoto L1 1 NDC Health Information Services, Phoenix, AZ, USA; 2 PharMetrics, Inc, Boston, MA, USA OBJECTIVES: To assess treatment patterns and treatment-related costs of acute otitis media (AOM) in a Managed Care population. METHODS: All subjects diagnosed with AOM in PharMetrics’ Integrated Outcomes Database between January 1, 1997 and December 31, 1998 were eligible for inclusion. Subjects were classified as: AOM with major surgery, AOM with minor surgery, or AOM with no surgery. Subject demographics, AOM—related utilization and costs, and antibiotic prescriptions were measured during the study period. RESULTS: 322,626 subjects were included in the study, with 97% falling into the AOM with no surgery category. Approximately 50% of the subjects were 6 years of age or older, while 52% of the subjects were female. The total cost of treating AOM for these subjects was $76 million. Minor and major surgery for AOM was more prevalent in children aged 6 or older than any other age group, and was associated with substantially higher costs. In 1997, the mean cost for subjects undergoing major surgery was $4,114 (std dev 3,290) compared to $142 (std dev 335) for subjects not requiring surgery. The most commonly prescribed antibiotic overall was amoxicillin, followed by sulfamethoxazole/trimethoprim; however, prescribing patterns differed across AOM categories and age groups. CONCLUSIONS: Acute Otitis Media is a prevalent condition associated with significant medical costs.
USE OF PHARMACOECONOMIC AND OUTCOMES RESEARCH IN HEALTHCARE DECISION-MAKING AND PRODUCT DEVELOPMENT
PTH1
USE OF ADMINISTRATIVE DATA FOR POSTMARKET SURVEILLANCE OF THE OUTCOME OF TOTAL HIP AND KNEE REPLACEMENTS Silverman B1, MacDonald T2 Davey P2 1 US Food and Drug Administration, Rockville, MD, USA; 2 Medicines Monitoring Unit, Ninewells Hospital, Dundee, Scotland OBJECTIVES: To use routine data to study outcomes of all knee and hip joint replacements in an unselected population with universal coverage of healthcare.
METHODS: The study population was all resident in Tayside, Scotland from 1989–1995. Joint replacements, and subsequent hospitalizations were identified from a hospital discharge database. Death was recorded from a community health database. The electronic records were validated against primary case records for a random sample. RESULTS: We identified 1,649 people who had first primary hip replacement and 989 first primary knee replacements. Case note validation was performed for 274 patients, of whom 143 had no previous record of hip or knee surgery and 131 had previous surgery. The proportion of valid cases was higher in the patients with no previous surgery (98% vs 57%). The most common mis-classification was revision arthroplasty as primary arthroplasty because of incomplete recording of previous joint replacement. There were 353 deaths in the hip implant cohort (21.4%) and 187 deaths in the knee implant cohort (18.9%); 6.4% of hip recipients and 5.5% of knee recipients died within the first year of implant. Four percent of hip replacements were for management of hip fracture and they had significantly reduced survival compared to the remaining patients (deaths at 60 days, 1.6% vs. 0.8%). Significantly more knee recipients than hip recipients required revision of the joint replacement (4.4% vs 2.4%, p 0.007). Although time to hip implant revision varied with the year of first implant (with 1990 recipients showing the shortest time to revision), this effect did not occur among knee implant recipients. CONCLUSIONS: This study demonstrates the suitability of the Scottish Morbidity record for postmarket surveillance of medical device implants. The electronic data come from a stable population with universal medical coverage and outcomes can be validated by review of primary case records.
PTH2
MALIGNANT RETROPERITONEAL TUMOURS OF CHILDREN: FIRST RESEARCH OF QUALITY OF LIFE OF 278 SURVIVED PATIENTS Zaeva G, Moiseenko E Scientific Research Institute of Oncology and Gematology, Moscow, Russia OBJECTIVES: Research on quality of life (QoL) of patients cured in childhood from malignant tumors. Wide usage of the latest anti-tumor methods of treatment has led to considerable growth of the number of recovered patients during the last years. Only in Moscow between 1978–97 2255 children with oncological diseases were successfully cured, 278 (12,3%) who had retroperitoneal malignant tumors. Among these children 191 were cured from nefroblastoma, 66 – from neuroblastoma, 21 – from adrenal tumors. Antitumor treatment deeply affects the child’s organism, causing not only physical dysfunction, but also dramatic psychological, emotional and social changes in the patient’s life. QoL research of the people who suffered from malignant tumors in childhood is a new area in Russian children’s oncology.
404
Abstracts
METHODS: Special clinical methods were used for health condition evaluation. Our first research of some of the QoL factors was held in 1996–97. RESULTS: It was conducted on 561 patients with different oncological diseases, including retroperitoneal malignant diseases. The period of remission for 59% of the patients was 5–10 years, for the rest 41%—5 years. Ages of the patients varied from 3–7 years to 15 years and over. Their physical condition was studied (including cardiovascular, urinary, immunological, reproductive, muscular and other systems), as well as their psychological condition and social adaptation. CONCLUSIONS: Only 40% of the patients don’t have significant after-effect from their anti-tumor therapy. Dysfunction of the immune system was found in 78% of the examined patients, of the alimentary canal, 67%, of the central nervous system, 60%, of the muscle and bone and the urinary tract systems, 56% and 50% respectively. Together with psychological and social problems this considerably influences the QoL of such patients. The research was continued by the evaluation of life conditions of 278 patients cured from malignant retroperitoneal tumors, as well as by the analysis of the obtained data in accordance with the international life-level standards. For this purpose we are currently adopting the selected interrogatory sheets EQ-50 (for adult patients) and SP-36 for children. The results will be introduced in future publications.
PTH3
GUIDELINES FOR SUBMISSION OF REQUESTS FOR INCLUSION OF A PHARMACEUTICAL PRODUCT IN THE ISRAELI NATIONAL FORMULARY Shani S, Morginstin T, Shemer J Ministry of Health, Jerusalem, Israel The Israeli National Health Insurance Law enacted in 1995 determines a list of health services including a list of reimbursed drugs (NLRD). Accordingly, each citizen is entitled to receive health services of established quality within the framework of the available resources. In the last decade, significant advances in R&D have resulted in the emergence of numerous expensive drugs in the pharmaceutical market. Hence, the Pharmacoeconomics Unit at the Pharmaceutical Administration of the Ministry of Health established a mechanism for updating the NLRD, based on the evaluation of clinical, epidemiological and economic data. Prior to the presentation of guidelines for the pharmaceutical industry on submission of economic data as part of a request for the inclusion of a pharmaceutical product in the NLRD, a survey was conducted to examine existing pharmacoeconomic knowledge of the Israeli pharmaceutical industry. The results of the survey indicated that the pharmaceutical industry has very little pharmacoeconomic knowledge. These results prompted a lenient approach to the submission of requests for the next two years during which the industry is expected to develop a firm pharmacoeconomic basis. Guidelines on
submission of a request to include a pharmaceutical product in the NLRD were presented to the pharmaceutical industry. The dossier to be submitted by the industry consists of three sections: Clinical—treatment regimens, adverse effects, efficacy and safety in comparison to alternate therapies. Epidemiological—morbidity figures, definition of target population, sales figures. Economic—cost of the suggested treatment compared to that of alternative therapies and an economic evaluation. 260 different requests have been submitted for the 2000 update of the NLRD, according to the guidelines, and are currently being processed. Following the 2000 update, a reassessment will take place, and the guidelines will be changed according to the experience incurred by both the industry and the Ministry of Health.
PTH4
ECONOMIC EVALUATION BY UK PHARMACEUTICAL AND MEDICAL DEVICE FIRMS: GENERAL TRENDS AND POLICY IMPLICATIONS Cookson R LSE Health, London School of Economics, London, UK OBJECTIVES: To survey expert opinion on trends in economic evaluation by UK pharmaceutical and medical device firms, from R&D to product launch and beyond. The background is the move in some EU countries, exemplified by NICE in UK, towards emulating the Australian “fourth hurdle” of mandatory economic data requirements before pharmaceutical purchasing. METHODS: Interviews with a range of experts from UK industry and academia. Industry representatives included both science experts and policy experts from six pharmaceutical firms and four medical device firms; academics included both health economists and pharmaceutical policy specialists. RESULTS: Dramatic increase over the last decade in production of pharmacoeconomic evidence at late development and early marketing stages in response to more aggressive management of pharmaceutical budgets. Medical device firms lag behind, due to smaller size and less aggressive purchasers, but large firms are now starting to design trials with economic evaluation in mind. Limited improvement in scientific quality of pharmacoeconomic evidence in relation to state-of-the-art methodology (“Washington Guidelines”); limited use of effectiveness trials, utility indices, cost-utility analysis, sensitivity analysis. Limited use of prospective cost-effectiveness modeling in R&D go/no go decisions, even though prospective modelling of return on investment is routinely used from early development onwards. Some industry representatives argue that pharmacoeconomics cannot be used to prioritize R&D since “hard” data are not available and uncertainties are great.
Abstracts tal fees will necessarily increase tremendously; proven efficacy for these costs has not been provided in Greece yet.
PID6
TREATMENT OF ACUTE OTITIS MEDIA IN A MANAGED CARE POPULATION McLaughlin T1, Margraf T2, Okamoto L1 1 NDC Health Information Services, Phoenix, AZ, USA; 2 PharMetrics, Inc, Boston, MA, USA OBJECTIVES: To assess treatment patterns and treatment-related costs of acute otitis media (AOM) in a Managed Care population. METHODS: All subjects diagnosed with AOM in PharMetrics’ Integrated Outcomes Database between January 1, 1997 and December 31, 1998 were eligible for inclusion. Subjects were classified as: AOM with major surgery, AOM with minor surgery, or AOM with no surgery. Subject demographics, AOM—related utilization and costs, and antibiotic prescriptions were measured during the study period. RESULTS: 322,626 subjects were included in the study, with 97% falling into the AOM with no surgery category. Approximately 50% of the subjects were 6 years of age or older, while 52% of the subjects were female. The total cost of treating AOM for these subjects was $76 million. Minor and major surgery for AOM was more prevalent in children aged 6 or older than any other age group, and was associated with substantially higher costs. In 1997, the mean cost for subjects undergoing major surgery was $4,114 (std dev 3,290) compared to $142 (std dev 335) for subjects not requiring surgery. The most commonly prescribed antibiotic overall was amoxicillin, followed by sulfamethoxazole/trimethoprim; however, prescribing patterns differed across AOM categories and age groups. CONCLUSIONS: Acute Otitis Media is a prevalent condition associated with significant medical costs.
USE OF PHARMACOECONOMIC AND OUTCOMES RESEARCH IN HEALTHCARE DECISION-MAKING AND PRODUCT DEVELOPMENT
PTH1
USE OF ADMINISTRATIVE DATA FOR POSTMARKET SURVEILLANCE OF THE OUTCOME OF TOTAL HIP AND KNEE REPLACEMENTS Silverman B1, MacDonald T2 Davey P2 1 US Food and Drug Administration, Rockville, MD, USA; 2 Medicines Monitoring Unit, Ninewells Hospital, Dundee, Scotland OBJECTIVES: To use routine data to study outcomes of all knee and hip joint replacements in an unselected population with universal coverage of healthcare.
METHODS: The study population was all resident in Tayside, Scotland from 1989–1995. Joint replacements, and subsequent hospitalizations were identified from a hospital discharge database. Death was recorded from a community health database. The electronic records were validated against primary case records for a random sample. RESULTS: We identified 1,649 people who had first primary hip replacement and 989 first primary knee replacements. Case note validation was performed for 274 patients, of whom 143 had no previous record of hip or knee surgery and 131 had previous surgery. The proportion of valid cases was higher in the patients with no previous surgery (98% vs 57%). The most common mis-classification was revision arthroplasty as primary arthroplasty because of incomplete recording of previous joint replacement. There were 353 deaths in the hip implant cohort (21.4%) and 187 deaths in the knee implant cohort (18.9%); 6.4% of hip recipients and 5.5% of knee recipients died within the first year of implant. Four percent of hip replacements were for management of hip fracture and they had significantly reduced survival compared to the remaining patients (deaths at 60 days, 1.6% vs. 0.8%). Significantly more knee recipients than hip recipients required revision of the joint replacement (4.4% vs 2.4%, p 0.007). Although time to hip implant revision varied with the year of first implant (with 1990 recipients showing the shortest time to revision), this effect did not occur among knee implant recipients. CONCLUSIONS: This study demonstrates the suitability of the Scottish Morbidity record for postmarket surveillance of medical device implants. The electronic data come from a stable population with universal medical coverage and outcomes can be validated by review of primary case records.
PTH2
MALIGNANT RETROPERITONEAL TUMOURS OF CHILDREN: FIRST RESEARCH OF QUALITY OF LIFE OF 278 SURVIVED PATIENTS Zaeva G, Moiseenko E Scientific Research Institute of Oncology and Gematology, Moscow, Russia OBJECTIVES: Research on quality of life (QoL) of patients cured in childhood from malignant tumors. Wide usage of the latest anti-tumor methods of treatment has led to considerable growth of the number of recovered patients during the last years. Only in Moscow between 1978–97 2255 children with oncological diseases were successfully cured, 278 (12,3%) who had retroperitoneal malignant tumors. Among these children 191 were cured from nefroblastoma, 66 – from neuroblastoma, 21 – from adrenal tumors. Antitumor treatment deeply affects the child’s organism, causing not only physical dysfunction, but also dramatic psychological, emotional and social changes in the patient’s life. QoL research of the people who suffered from malignant tumors in childhood is a new area in Russian children’s oncology.
404
Abstracts
METHODS: Special clinical methods were used for health condition evaluation. Our first research of some of the QoL factors was held in 1996–97. RESULTS: It was conducted on 561 patients with different oncological diseases, including retroperitoneal malignant diseases. The period of remission for 59% of the patients was 5–10 years, for the rest 41%—5 years. Ages of the patients varied from 3–7 years to 15 years and over. Their physical condition was studied (including cardiovascular, urinary, immunological, reproductive, muscular and other systems), as well as their psychological condition and social adaptation. CONCLUSIONS: Only 40% of the patients don’t have significant after-effect from their anti-tumor therapy. Dysfunction of the immune system was found in 78% of the examined patients, of the alimentary canal, 67%, of the central nervous system, 60%, of the muscle and bone and the urinary tract systems, 56% and 50% respectively. Together with psychological and social problems this considerably influences the QoL of such patients. The research was continued by the evaluation of life conditions of 278 patients cured from malignant retroperitoneal tumors, as well as by the analysis of the obtained data in accordance with the international life-level standards. For this purpose we are currently adopting the selected interrogatory sheets EQ-50 (for adult patients) and SP-36 for children. The results will be introduced in future publications.
PTH3
GUIDELINES FOR SUBMISSION OF REQUESTS FOR INCLUSION OF A PHARMACEUTICAL PRODUCT IN THE ISRAELI NATIONAL FORMULARY Shani S, Morginstin T, Shemer J Ministry of Health, Jerusalem, Israel The Israeli National Health Insurance Law enacted in 1995 determines a list of health services including a list of reimbursed drugs (NLRD). Accordingly, each citizen is entitled to receive health services of established quality within the framework of the available resources. In the last decade, significant advances in R&D have resulted in the emergence of numerous expensive drugs in the pharmaceutical market. Hence, the Pharmacoeconomics Unit at the Pharmaceutical Administration of the Ministry of Health established a mechanism for updating the NLRD, based on the evaluation of clinical, epidemiological and economic data. Prior to the presentation of guidelines for the pharmaceutical industry on submission of economic data as part of a request for the inclusion of a pharmaceutical product in the NLRD, a survey was conducted to examine existing pharmacoeconomic knowledge of the Israeli pharmaceutical industry. The results of the survey indicated that the pharmaceutical industry has very little pharmacoeconomic knowledge. These results prompted a lenient approach to the submission of requests for the next two years during which the industry is expected to develop a firm pharmacoeconomic basis. Guidelines on
submission of a request to include a pharmaceutical product in the NLRD were presented to the pharmaceutical industry. The dossier to be submitted by the industry consists of three sections: Clinical—treatment regimens, adverse effects, efficacy and safety in comparison to alternate therapies. Epidemiological—morbidity figures, definition of target population, sales figures. Economic—cost of the suggested treatment compared to that of alternative therapies and an economic evaluation. 260 different requests have been submitted for the 2000 update of the NLRD, according to the guidelines, and are currently being processed. Following the 2000 update, a reassessment will take place, and the guidelines will be changed according to the experience incurred by both the industry and the Ministry of Health.
PTH4
ECONOMIC EVALUATION BY UK PHARMACEUTICAL AND MEDICAL DEVICE FIRMS: GENERAL TRENDS AND POLICY IMPLICATIONS Cookson R LSE Health, London School of Economics, London, UK OBJECTIVES: To survey expert opinion on trends in economic evaluation by UK pharmaceutical and medical device firms, from R&D to product launch and beyond. The background is the move in some EU countries, exemplified by NICE in UK, towards emulating the Australian “fourth hurdle” of mandatory economic data requirements before pharmaceutical purchasing. METHODS: Interviews with a range of experts from UK industry and academia. Industry representatives included both science experts and policy experts from six pharmaceutical firms and four medical device firms; academics included both health economists and pharmaceutical policy specialists. RESULTS: Dramatic increase over the last decade in production of pharmacoeconomic evidence at late development and early marketing stages in response to more aggressive management of pharmaceutical budgets. Medical device firms lag behind, due to smaller size and less aggressive purchasers, but large firms are now starting to design trials with economic evaluation in mind. Limited improvement in scientific quality of pharmacoeconomic evidence in relation to state-of-the-art methodology (“Washington Guidelines”); limited use of effectiveness trials, utility indices, cost-utility analysis, sensitivity analysis. Limited use of prospective cost-effectiveness modeling in R&D go/no go decisions, even though prospective modelling of return on investment is routinely used from early development onwards. Some industry representatives argue that pharmacoeconomics cannot be used to prioritize R&D since “hard” data are not available and uncertainties are great.