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“Best Supportive Care” or “Therapy According to Physician's Choice” as Appropriate Comparator for German HTA Dossiers – An Underestimated Chance or Risk for the Benefit Assessment?
A494
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
Access Evidence Dossier based on the EU ST which contains clinical value elements of HTA CM domains 1-4 & 6 (Health Problem and Use of Technology, Technical Characteristics, Clinical Effectiveness and Safety, and Ethical Analysis). The current research obtained input from ten affiliates (Australia, Brazil, Canada, China, France, Germany, Mexico, Spain, UK and USA) to determine whether the EU ST provides a framework to generate a robust global summary of the clinical value proposition for a product to inform HTA and payer submissions. Methods: Market Access colleagues in ten major markets were surveyed on the importance and transferability of 102 clinical value elements contained within the EU ST to inform HTA and payer submissions. Results: There was a high level of agreement on the importance and transferability of clinical value elements across the ten affiliates. At least 7 out of 10 countries agreed that 95 clinical value elements were important for payers and 60 of the clinical value elements were considered both important and transferable across affiliates. None were ranked as unimportant. Conclusions: The EU ST was considered to be robust and comprehensive to capture the clinical value proposition for a product and inform HTA and payer submissions across ten key markets. The majority of clinical value elements contained within the EU ST (HTA CM domains 1-4 & 6) were ranked as important and captured clinical value essential for HTA and payers. Roche has developed product-specific pilot Access Evidence Dossiers and will evaluate these with affiliates and EUnetHTA in 2016 to capture ongoing learnings. PHP307 Implemantation of Processes to Estabilishing a HTA Unit in Czech Hospital Enviroment Zavadil M1, Matlonova V 2, Kubatova I2, Rogalewicz V2 University Hospital, Prague, Czech Republic, 2Czech Technical University in Prague, Kladno, Czech Republic
1Motol
Objectives: To shape the implementation process of a hospital HTA Unit for the Motol University Hospital in Prague. Methods: The methodology consists of data collection from hospitals that have recently successfully estabilished HTA Units, organization of their recommendations into comprehensive schemes that serve not only for the purpose of a comparison of these hospitals/countries, but also as a tool to find out key factors needed to model and recommend the implementation scheme and process elements for the HTA Unit in the Motol University Hospital. Results: The recommended model represents a synthesis of experience with HTA Units in hospitals worldwide, while taking into account the available qualifications as well as already well established departments that could cooperate with and use services and support from the future HTA Unit of the Motol University Hospital. Suggested steps of the implementation process have been carefully formulated in a general way, so that the process could be applied also to other teaching hospitals in the Czech Republic, requiring only minor adjustments to the specificity and individuality of each hospital. Conclusions: Hospital-based HTA is the concept, whose main goal is to inform hospital management in situations when they have to make choices or prioritize. Another reason why health technology assessment (HTA) is being performed at the hospital level is that costs, benefits and impacts of used technologies can be directly assessed. Even though, since the concept is new and there is relatively small amount of evaluations containing quantitative data released, it is more than interesting to compare different hospital-based HTA approaches and try to evaluate their impacts on the hospitals as well as health care systems they are being implemented in. PHP308 Institute for Clinical and Economic Review (ICER) VS. Health Technology Assessment (HTA) Agencies: The Case of Multiple Myeloma and PCSK9S Jaksa A, Liden D Context Matters, New York, NY, USA
Objectives: ICER, a newly established HTA organization in the U.S., has been both praised and criticized for its drug assessments. Manufacturers and patient groups have been critical of ICER, but is this criticism justified? ICER’s goal is similar to other established HTA agencies: to inform payers and policymakers on a drug’s value and better tie drug prices to value. How does the ICER’s decisions compare to those of more established HTA agencies? Methods: We compared ICER’s multiple myeloma and PCSK9 evaluations with the corresponding HTAs from NICE, SMC, pCODR/CADTH, G-BA, and HAS to determine how consistent the agencies were in their decision making. We focused on their overall clinical effectiveness and costeffectiveness conclusions. Results: ICER’s assessments were published before all the corresponding HTAs; a comparison of all drugs was not possible. This is because ICER times their reviews based on FDA approvals, which are issued before EMA approvals. There were few differences between ICER and the HTA agencies in their conclusions on clinical efficacy. For example, ICER determined that panobinostat (third-line multiple myeloma) did not demonstrate a net clinical benefit, while both NICE and SMC determined the drug was clinically effective. All three agencies noted the same toxicity issue, which would likely be mitigated by the route of administration used in clinical practice. The uncertainty around toxicity lead ICER to determine there was no net clinical benefit. Conclusions: Using established HTA agencies as a benchmark, ICER’s clinical evaluations were similar to other HTA agencies, however, there was a lack of data for comparisons of all drugs. The criticism in the U.S. might be due to the newness of HTA in the US healthcare system and not a deviation from well-established HTA decisions. While HTA decisions from the agencies evaluated have immediate impact on their healthcare systems, the impact of ICER is less well known. PHP309 “Best Supportive Care” or “Therapy According to Physician’s Choice” as Appropriate Comparator for German HTA Dossiers – An Underestimated Chance or Risk for the Benefit Assessment? Eheberg D1, Dannemann S2, Jakovac M2, Bonduelle D1 1IMS Health, Munich, Germany, 2IMS Health Germany, Munich, Germany
Background: The choice of the appropriate comparator is a central aspect of the German HTA dossier. In most cases the appropriate comparator, which is defined by four criteria set in the rules of procedure of the federal joint committee (G-BA), is a list of equally appropriate substances and treatment methods. However, in some cases the G-BA deviates from this approach and determines that either “best supportive care” (BSC) or “therapy according to physician’s choice” (TPC) is the appropriate comparator. Objectives: The aim of our project was to assess how often BSC and TPC were used as an appropriate comparator and to evaluate the consequences in benefit for the submitted dossier. Additionally, we wanted to evaluate what triggered the choice. Methods: We reviewed the decisions published on the G-BA website between 01.01.2011 and 01.06.2016. The submitted dossiers were screened for appropriate comparators referring to BSC or TPC and a descriptive analysis was performed to identify a decision algorithm. Results: We identified 12 substances with 18 subpopulations with BSC as appropriate comparator and 13 substances with 15 subpopulations with TPC. The main reason to determine BSC as appropriate comparator was a lack of alternatives, mostly in the context of palliative indications. For TPC unclear guidelines and uncertain evidence was a key criterion. An additional benefit was granted in nearly half of the subpopulations with BSC as appropriate comparator. For TPC only in four subpopulations an additional benefit was determined. Conclusions: TPC and BSC offer an opportunity for substances without direct comparison trials. Especially BSC is the best way to present data from placebo-controlled trials. In the last years the number of benefit assessments for substances with TPC as the appropriate comparator has increased. TPC is more challenging than BSC and it is hard to fulfill the elaborated standards of G-BA and IQWiG. PHP310 Different Methods, Different Results? Comparing Health Technology Assessments in the United Kingdom and Germany Schaefer R, Schlander M Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany
Objectives: Implementation of health technology assessments (HTAs) by HTA agencies varies internationally, perhaps most profoundly with respect to health economic evaluation methods. Whereas the UK National Institute for Health and Care Excellence (NICE) relies heavily on cost utility analysis, HTAs by the German Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (Gemeinsamer Bundesausschuss, GBA) focus on comparative effectiveness based on a rigorous application of principles of evidence-based medicine (EBM). The present study aimed to explore the extent to which different value judgments and resulting methodological choices lead to different HTA outcomes. Methods: We extracted data from all GBA decisions between January 2011 (when early benefit assessments were implemented) and April 2015 (cut-off date for the present study), as well as all published NICE single technology appraisals (STAs) during this period. We compared health benefit assessment results by IQWiG/GBA and by NICE overall, and by additional criteria including therapeutic area, clinical and incremental cost effectiveness, and patient-relevant endpoints. Results: During the study period, NICE issued guidance for 88 technologies (125 subgroups). GBA completed 105 appraisals (226 subgroups). We identified 37 matched conditionintervention pairs; of these, 24 were evaluated differently by NICE and GBA. NICE recommended 29/37 treatments, whereas GBA confirmed additional benefit for 21/37 only. By therapeutic area, interventions for hematological and oncological diseases were relatively more likely to be evaluated positively by IQWiG/GBA; in contrast, NICE appraisals were relatively more favorable towards treatments for metabolic and cardiovascular disorders. Results including all interventions were consistent with the findings reported for matched pairs. Conclusions: Overall, NICE tends to evaluate new drugs more favorably than IQWiG/GBA. However, treatments for some therapeutic areas like cancer were evaluated more favorably by IQWiG/GBA. Our results lend support to the hypothesis that different HTA methods contribute to systematic differences in decision-making.
HEALTH CARE USE & POLICY STUDIES – Patient Registries & Post-Marketing Studies PHP311 Impact Of Post-Inscription Studies On Reassessment In France: A Case Study On Orphan Drugs Jubert H, Ouali I, Kolahi C IMS Health, La Défense, France
Objectives: Orphan designation is a status assigned to a drug intended for use against rare condition. The drug must fulfil criteria for designation as an orphan medicine so that it can benefit from incentives like protection from competition once commercialized. In France, HAS (French National Authority for Health) reinforces requests for post-inscription studies, providing a better understanding of innovative drugs. This study aims to analyze their impact on reassessment focusing on orphan medicines. Methods: We conducted an analysis of ASMR (added medical benefit) reassessment between 2006 and 2016, for orphan drugs. For each product, we have pointed out by indication: ASMR rating between two evaluations, therapeutic area, authority requests, post inscription study types and their methodology. Results: Six out the 21 reassessments faced a decrease in ASMR rating, five faced an increase and 10 kept the same ASMR. Among decreases,the HAS challenged the drug effectiveness (1 product), requested for more exhaustive real life data in post-inscription study (3 products), but also the therapeutic benefit for all treatments of a therapeutic area (2 products). For increases not due to reevaluation of therapeutic class (2), the HAS gave an ASMR upgrading for companies providing new real life data (1) or additional comparative study (2). Furthermore, we observed a positive correlation between exhaustive data provided (real world data or indirect comparison), a good methodology or practice behavior from the marketing authorization holder, and positive opinion from HAS. Conclusions: Post-inscription
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
Access Evidence Dossier based on the EU ST which contains clinical value elements of HTA CM domains 1-4 & 6 (Health Problem and Use of Technology, Technical Characteristics, Clinical Effectiveness and Safety, and Ethical Analysis). The current research obtained input from ten affiliates (Australia, Brazil, Canada, China, France, Germany, Mexico, Spain, UK and USA) to determine whether the EU ST provides a framework to generate a robust global summary of the clinical value proposition for a product to inform HTA and payer submissions. Methods: Market Access colleagues in ten major markets were surveyed on the importance and transferability of 102 clinical value elements contained within the EU ST to inform HTA and payer submissions. Results: There was a high level of agreement on the importance and transferability of clinical value elements across the ten affiliates. At least 7 out of 10 countries agreed that 95 clinical value elements were important for payers and 60 of the clinical value elements were considered both important and transferable across affiliates. None were ranked as unimportant. Conclusions: The EU ST was considered to be robust and comprehensive to capture the clinical value proposition for a product and inform HTA and payer submissions across ten key markets. The majority of clinical value elements contained within the EU ST (HTA CM domains 1-4 & 6) were ranked as important and captured clinical value essential for HTA and payers. Roche has developed product-specific pilot Access Evidence Dossiers and will evaluate these with affiliates and EUnetHTA in 2016 to capture ongoing learnings. PHP307 Implemantation of Processes to Estabilishing a HTA Unit in Czech Hospital Enviroment Zavadil M1, Matlonova V 2, Kubatova I2, Rogalewicz V2 University Hospital, Prague, Czech Republic, 2Czech Technical University in Prague, Kladno, Czech Republic
1Motol
Objectives: To shape the implementation process of a hospital HTA Unit for the Motol University Hospital in Prague. Methods: The methodology consists of data collection from hospitals that have recently successfully estabilished HTA Units, organization of their recommendations into comprehensive schemes that serve not only for the purpose of a comparison of these hospitals/countries, but also as a tool to find out key factors needed to model and recommend the implementation scheme and process elements for the HTA Unit in the Motol University Hospital. Results: The recommended model represents a synthesis of experience with HTA Units in hospitals worldwide, while taking into account the available qualifications as well as already well established departments that could cooperate with and use services and support from the future HTA Unit of the Motol University Hospital. Suggested steps of the implementation process have been carefully formulated in a general way, so that the process could be applied also to other teaching hospitals in the Czech Republic, requiring only minor adjustments to the specificity and individuality of each hospital. Conclusions: Hospital-based HTA is the concept, whose main goal is to inform hospital management in situations when they have to make choices or prioritize. Another reason why health technology assessment (HTA) is being performed at the hospital level is that costs, benefits and impacts of used technologies can be directly assessed. Even though, since the concept is new and there is relatively small amount of evaluations containing quantitative data released, it is more than interesting to compare different hospital-based HTA approaches and try to evaluate their impacts on the hospitals as well as health care systems they are being implemented in. PHP308 Institute for Clinical and Economic Review (ICER) VS. Health Technology Assessment (HTA) Agencies: The Case of Multiple Myeloma and PCSK9S Jaksa A, Liden D Context Matters, New York, NY, USA
Objectives: ICER, a newly established HTA organization in the U.S., has been both praised and criticized for its drug assessments. Manufacturers and patient groups have been critical of ICER, but is this criticism justified? ICER’s goal is similar to other established HTA agencies: to inform payers and policymakers on a drug’s value and better tie drug prices to value. How does the ICER’s decisions compare to those of more established HTA agencies? Methods: We compared ICER’s multiple myeloma and PCSK9 evaluations with the corresponding HTAs from NICE, SMC, pCODR/CADTH, G-BA, and HAS to determine how consistent the agencies were in their decision making. We focused on their overall clinical effectiveness and costeffectiveness conclusions. Results: ICER’s assessments were published before all the corresponding HTAs; a comparison of all drugs was not possible. This is because ICER times their reviews based on FDA approvals, which are issued before EMA approvals. There were few differences between ICER and the HTA agencies in their conclusions on clinical efficacy. For example, ICER determined that panobinostat (third-line multiple myeloma) did not demonstrate a net clinical benefit, while both NICE and SMC determined the drug was clinically effective. All three agencies noted the same toxicity issue, which would likely be mitigated by the route of administration used in clinical practice. The uncertainty around toxicity lead ICER to determine there was no net clinical benefit. Conclusions: Using established HTA agencies as a benchmark, ICER’s clinical evaluations were similar to other HTA agencies, however, there was a lack of data for comparisons of all drugs. The criticism in the U.S. might be due to the newness of HTA in the US healthcare system and not a deviation from well-established HTA decisions. While HTA decisions from the agencies evaluated have immediate impact on their healthcare systems, the impact of ICER is less well known. PHP309 “Best Supportive Care” or “Therapy According to Physician’s Choice” as Appropriate Comparator for German HTA Dossiers – An Underestimated Chance or Risk for the Benefit Assessment? Eheberg D1, Dannemann S2, Jakovac M2, Bonduelle D1 1IMS Health, Munich, Germany, 2IMS Health Germany, Munich, Germany
Background: The choice of the appropriate comparator is a central aspect of the German HTA dossier. In most cases the appropriate comparator, which is defined by four criteria set in the rules of procedure of the federal joint committee (G-BA), is a list of equally appropriate substances and treatment methods. However, in some cases the G-BA deviates from this approach and determines that either “best supportive care” (BSC) or “therapy according to physician’s choice” (TPC) is the appropriate comparator. Objectives: The aim of our project was to assess how often BSC and TPC were used as an appropriate comparator and to evaluate the consequences in benefit for the submitted dossier. Additionally, we wanted to evaluate what triggered the choice. Methods: We reviewed the decisions published on the G-BA website between 01.01.2011 and 01.06.2016. The submitted dossiers were screened for appropriate comparators referring to BSC or TPC and a descriptive analysis was performed to identify a decision algorithm. Results: We identified 12 substances with 18 subpopulations with BSC as appropriate comparator and 13 substances with 15 subpopulations with TPC. The main reason to determine BSC as appropriate comparator was a lack of alternatives, mostly in the context of palliative indications. For TPC unclear guidelines and uncertain evidence was a key criterion. An additional benefit was granted in nearly half of the subpopulations with BSC as appropriate comparator. For TPC only in four subpopulations an additional benefit was determined. Conclusions: TPC and BSC offer an opportunity for substances without direct comparison trials. Especially BSC is the best way to present data from placebo-controlled trials. In the last years the number of benefit assessments for substances with TPC as the appropriate comparator has increased. TPC is more challenging than BSC and it is hard to fulfill the elaborated standards of G-BA and IQWiG. PHP310 Different Methods, Different Results? Comparing Health Technology Assessments in the United Kingdom and Germany Schaefer R, Schlander M Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany
Objectives: Implementation of health technology assessments (HTAs) by HTA agencies varies internationally, perhaps most profoundly with respect to health economic evaluation methods. Whereas the UK National Institute for Health and Care Excellence (NICE) relies heavily on cost utility analysis, HTAs by the German Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (Gemeinsamer Bundesausschuss, GBA) focus on comparative effectiveness based on a rigorous application of principles of evidence-based medicine (EBM). The present study aimed to explore the extent to which different value judgments and resulting methodological choices lead to different HTA outcomes. Methods: We extracted data from all GBA decisions between January 2011 (when early benefit assessments were implemented) and April 2015 (cut-off date for the present study), as well as all published NICE single technology appraisals (STAs) during this period. We compared health benefit assessment results by IQWiG/GBA and by NICE overall, and by additional criteria including therapeutic area, clinical and incremental cost effectiveness, and patient-relevant endpoints. Results: During the study period, NICE issued guidance for 88 technologies (125 subgroups). GBA completed 105 appraisals (226 subgroups). We identified 37 matched conditionintervention pairs; of these, 24 were evaluated differently by NICE and GBA. NICE recommended 29/37 treatments, whereas GBA confirmed additional benefit for 21/37 only. By therapeutic area, interventions for hematological and oncological diseases were relatively more likely to be evaluated positively by IQWiG/GBA; in contrast, NICE appraisals were relatively more favorable towards treatments for metabolic and cardiovascular disorders. Results including all interventions were consistent with the findings reported for matched pairs. Conclusions: Overall, NICE tends to evaluate new drugs more favorably than IQWiG/GBA. However, treatments for some therapeutic areas like cancer were evaluated more favorably by IQWiG/GBA. Our results lend support to the hypothesis that different HTA methods contribute to systematic differences in decision-making.
HEALTH CARE USE & POLICY STUDIES – Patient Registries & Post-Marketing Studies PHP311 Impact Of Post-Inscription Studies On Reassessment In France: A Case Study On Orphan Drugs Jubert H, Ouali I, Kolahi C IMS Health, La Défense, France
Objectives: Orphan designation is a status assigned to a drug intended for use against rare condition. The drug must fulfil criteria for designation as an orphan medicine so that it can benefit from incentives like protection from competition once commercialized. In France, HAS (French National Authority for Health) reinforces requests for post-inscription studies, providing a better understanding of innovative drugs. This study aims to analyze their impact on reassessment focusing on orphan medicines. Methods: We conducted an analysis of ASMR (added medical benefit) reassessment between 2006 and 2016, for orphan drugs. For each product, we have pointed out by indication: ASMR rating between two evaluations, therapeutic area, authority requests, post inscription study types and their methodology. Results: Six out the 21 reassessments faced a decrease in ASMR rating, five faced an increase and 10 kept the same ASMR. Among decreases,the HAS challenged the drug effectiveness (1 product), requested for more exhaustive real life data in post-inscription study (3 products), but also the therapeutic benefit for all treatments of a therapeutic area (2 products). For increases not due to reevaluation of therapeutic class (2), the HAS gave an ASMR upgrading for companies providing new real life data (1) or additional comparative study (2). Furthermore, we observed a positive correlation between exhaustive data provided (real world data or indirect comparison), a good methodology or practice behavior from the marketing authorization holder, and positive opinion from HAS. Conclusions: Post-inscription