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Standardisation and Compatibility Between HTA Networks: what Impacts on Quality?
A504
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
The Appraisal of Guideline for Research and Evaluation II (AGREE II) tool will be used to create a systematic appraisal in six domains to determine the guidelines fulfilling the criteria of methodological rigour and transparency. Additionally the specification of treatment methods will be issued basing on drugs and lines of treatment described in the individual guidelines. The consistency of recommended treatment options among the guidelines will be checked. The conclusions of such a study will be of double benefit: they will provide an overview of the stronger and weaker clinical management recommendations in terms of underlying evidence. It is expected as well that the difference between evidence based and expert opinion based guidelines will be shown what may play an educational role for the medical practitioners of the countries where the role of EBM is still unappreciated. PHP362 Standardisation and Compatibility Between HTA Networks: what Impacts on Quality? Pacheco L GfK Market Access, London, UK
Objective: HTA agencies have structured into 3 international networks: EUnetHTA in Europe, RedETSA in Latin America and HTAsiaLink in Asia. To increase the efficiency of collaborative work, HTA networks develop standards. The level of quality is dependent on the standard in place; hence this study aims to identify what drives the choice of standards and degree of compatibly by HTA networks from an economic perspective, and the implications on quality. Methods: Network economics provides a set of analytical frameworks that was used to analyse standardisation within HTA networks. The Katz & Shapiro model specifies how the degree of compatibility between standards impacts the growth dynamics of competing networks. While standardisation between networks may allow attracting customers of compatible standards, it reduces quality differentiation opportunities. Depending on their size, installed base (number of members) and marginal cost, there can be 4 situations faced by 2 networks as to whether they will choose to be compatible or not. Results: Under the assumption that a certain form of competition may exist between HTA networks (to develop size, academic reputation, public health impact etc.), networks could be incline to make a choice of standardisation that may not support HTA collaborations at an optimal quality level. Conclusion: In spite of the development of best practice in HTA, to support their growth international of HTA agencies may make standardisation and compatibility choices that do not support collaboration at an optimal quality level. This poses the question of quality control within HTA networks. PHP363 Challenges for HTA Submissions Concerning Conditions Lacking Established Treatment Pathways Adkins E, Fountain DL, Bovens SM PHMR Ltd, London, UK
During health technology assessment (HTA) submissions, the manufacturer must specify the decision problem that their evidence submission is addressing. This involves defining the scope of the appraisal, including the population and possible subgroups, comparators, outcomes of interest, equality issues, and innovation. Whilst some HTA bodies such as the National Institute for Health and Care Excellence (NICE) in England pre-specify the scope of the evaluation, others, such as the Scottish Medicines Council (SMC), require the manufacturer to define their own decision problem. This can be particularly challenging in diseases where the therapeutic pathway is not well defined, for example in rare diseases or where treatment depends on a number of patient characteristics (health, age, prior treatment etc.). However, choosing the correct comparator(s) is key to HTA success; a common reason for failure with the SMC is not choosing the most appropriate comparator. Furthermore, the sequencing of treatments in the treatment pathway can have profound effects on the cost-effectiveness of therapies, and therefore in the absence of established pathways, manufacturers must establish these carefully and perform appropriate sensitivity analyses as required. We have explored how the lack of a well-defined treatment pathway can result in challenges for defining and addressing the HTA decision problem using examples from recent HTA submissions for treatments in orphan oncology, neuropathic pain, and scleroderma, in England, Scotland, and France. Factors such as collaboration with payers and clinicians and analysis of real world evidence are often used and we will summarise how different methods used to determine treatment pathways have been used by manufacturers with different HTA agencies to demonstrate areas of similarity and difference between countries. PHP364 The Evaluation of Innovative Drugs Applying Business Valuation Concepts Nuijten MJ1, Vis J2 1A2M, Amsterdam, The Netherlands, 2Talanton, Amsterdam, The Netherlands
Objective: Many pharmaceutical companies are now commercialising innovative expensive medicinal products, e.g. biologicals, with an incremental cost-effectiveness ratio (ICER), which will probably exceed the accepted threshold values for reimbursement. The goal of this research is to propose an additional methodology to evaluate innovative drugs from a broader perspective by applying concepts from business valuation, when the ICER exceeds the threshold. Methods: In a pure free health market, the price of the new innovative drug would be determined by demand and supply mechanisms, and all considerations about the use of costeffectiveness data would be redundant. On the other hand, the health authorities leave the responsibility for medical innovation to the market. Therefore medical innovation relies on the market mechanisms in the finance market of biotechnology including the incentives of the capital providers, who demand a required return of investment. The justification of the drug price can be based on the Discounted Cash Flow method. The Discounted Cash Flow method is based on the expected free cash
flows and the required cost of capital, can be used to validate the price of the new drug from a narrow investor’s perspective. The free cash flow represent the sales from the pharmaceuticals, and cash necessary for capital expenditures represent the costs for research & development (R&D) and marketing. The cost of capital refers to the opportunity cost of making a specific investment, which is the rate of return required to persuade the investor to make a given investment. Conclusion: We propose a policy approach for the evaluation of innovative drugs by bridging concepts from health economics and business economic valuation. This approach may justify a drug price from an investor’s perspective when the ICER exceeds the threshold and may be used in negotiations between governments and companies. PHP365 Unlocking Established Products Patient and Societal Value in Additional Indications Nayroles G1, Gabriel S1, Toumi M2, Kornfeld A3, Frybourg S3, Antonanzas F4, Espin J5, Jommi C6, de Pouvourville G7, Tolley K8, Wasem J9 1IPSEN Pharma, Boulogne-Billancourt, France, 2Aix-Marseille University, Marseille, France, 3Creativ-Ceutical, Paris, France, 4University of La Rioja, Logroño, Spain, 5Andalusian School of Public Health, Granada, Spain, 6Cergas, Bocconi University, Milano, Italy, 7ESSEC Business School, Cergy-Pontoise, France, 8Tolley Health Economics, Buxton, UK, 9University Duisburg-Essen, Essen, Germany
Background: New indications for established products (EPs) – marketed for 8 years or more - may represent a high societal value; but pharmaceutical manufacturers criticize the disincentives to expand indication of EPs and deliver the potential value for the society, such as lack of intellectual propriety or data protection, price cuts for new indication, generic competition, internal reference pricing. Incentive policies for orphan drugs or pediatric indications have driven EP development in these specific areas. Capturing the societal benefit of EP development is on the European Commission agenda. The objective of this research was to propose new potential incentives for the development of EPs through an international expert panel. Discussion: The expert panel suggested the following incentives for EPs that provide an added value in new indications: To extend the market protection period following introduction of a new indication To develop differential pricing regulation for different indications of the same active ingredient. Three processes may help achieving differential pricing: Differential market access agreement such as rebate or other financial-based or outcome-based managed market entry agreements for different indications Electronic prescription including indication to ensure the right product is used for each specific indication when generic do not match extension of indication To avoid or delay systematic therapeutic reference pricing Conclusion: Current regulations can represent a serious disincentive to develop new indications for EPs. EPs may offer treatment options with well-known safety. They have a potential to reduce off-label use and contain prescription escalation toward expensive products. Policy changes, both regulatory, through data protection and market exclusivity, and pricing, by rewarding the effort to bring new indication for EPs, are needed to capture the full societal value of EPs. PHP366 Pharmaceutical Tracking System: An Improvement in Data Accuracy and Uses Afify Y Ministry of Health, Cairo, Egypt
Medicines are strategic health commodities that governments are required to provide for people. Tracking systems are new technologies emerged in the different fields of logistics. In pharmaceutical sector, many countries had begun implementing the track and trace system. While implementation in some countries around the world ranges from drafting the legislations and partial testing of the system, others are fully implementing such technology in more than successful fashion. The technology depends on identification of every single pack/unit of medicines of any type once manufactured using a combination of serial number and code in addition to the batch number and expiry date, then recording every transaction applied to the pack in the market using its identity. The last transaction applied to the pack is the dispensing to patient. When the governments integrate the patients’ health records and different registries with the tracking system, every pack can be tied to the patient name and identity. In such system, pharmacoeconomic studies can accurately utilize the accurate data of medicines dispensed and linked to patients. Better epidemiology data will be available for any geographic area using patterns of consumption of different therapeutic classes of medicines. Data provided by tracking of pharmaceuticals can improve the monitoring of supply and demand of medicines and hence pricing. Health technology assessment can be based on real data from the market and precise comparisons are then feasible using different models of decision trees and Markov Model. Big data are easily generated to assess different patterns and trends in the pharmaceutical market. Moreover the technology allows control of the market by preventing counterfeit and smuggled drugs. The new system can effectively supports the rational use of medicines and helps take instant and precise actions with more effective and accurate market surveillance. PHP367 Mapping of The Biosimilar Drug Policy in 10 Central Eastern European Countries Inotai A1, Csanadi M1, Petrova G2, Bochenek T3, Tesar T4, York K5, Fuksa L6, Kostyuk A7, Araja D8, Lorenzovici L9, Egyed K10, Kaló Z11 1Syreon Research Institute, Budapest, Hungary, 2Medical University-Sofia, Faculty of Pharmacy, Sofia, Bulgaria, 3Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, Poland, 4Department of Organisation and Management in Pharmacy, Faculty of Pharmacy, Comenius University, Bratislava, Slovakia, 5Independent, Innsbruck, Austria, 6Department of Social and Clinical Pharmacy, Faculty of Pharmacy, Charles University in Prague, Hradec Králové, Czech Republic, 7Kazakh Agency for Health Technology Assessment, Astana, Kazakhstan, 8Riga Stradins University, Riga, Latvia, 9Syreon Research Romania, Tirgu Mures, Romania, 10Egis Pharmaceuticals, Budapest, Hungary, 11Eötvös Loránd University, Budapest, Hungary
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
The Appraisal of Guideline for Research and Evaluation II (AGREE II) tool will be used to create a systematic appraisal in six domains to determine the guidelines fulfilling the criteria of methodological rigour and transparency. Additionally the specification of treatment methods will be issued basing on drugs and lines of treatment described in the individual guidelines. The consistency of recommended treatment options among the guidelines will be checked. The conclusions of such a study will be of double benefit: they will provide an overview of the stronger and weaker clinical management recommendations in terms of underlying evidence. It is expected as well that the difference between evidence based and expert opinion based guidelines will be shown what may play an educational role for the medical practitioners of the countries where the role of EBM is still unappreciated. PHP362 Standardisation and Compatibility Between HTA Networks: what Impacts on Quality? Pacheco L GfK Market Access, London, UK
Objective: HTA agencies have structured into 3 international networks: EUnetHTA in Europe, RedETSA in Latin America and HTAsiaLink in Asia. To increase the efficiency of collaborative work, HTA networks develop standards. The level of quality is dependent on the standard in place; hence this study aims to identify what drives the choice of standards and degree of compatibly by HTA networks from an economic perspective, and the implications on quality. Methods: Network economics provides a set of analytical frameworks that was used to analyse standardisation within HTA networks. The Katz & Shapiro model specifies how the degree of compatibility between standards impacts the growth dynamics of competing networks. While standardisation between networks may allow attracting customers of compatible standards, it reduces quality differentiation opportunities. Depending on their size, installed base (number of members) and marginal cost, there can be 4 situations faced by 2 networks as to whether they will choose to be compatible or not. Results: Under the assumption that a certain form of competition may exist between HTA networks (to develop size, academic reputation, public health impact etc.), networks could be incline to make a choice of standardisation that may not support HTA collaborations at an optimal quality level. Conclusion: In spite of the development of best practice in HTA, to support their growth international of HTA agencies may make standardisation and compatibility choices that do not support collaboration at an optimal quality level. This poses the question of quality control within HTA networks. PHP363 Challenges for HTA Submissions Concerning Conditions Lacking Established Treatment Pathways Adkins E, Fountain DL, Bovens SM PHMR Ltd, London, UK
During health technology assessment (HTA) submissions, the manufacturer must specify the decision problem that their evidence submission is addressing. This involves defining the scope of the appraisal, including the population and possible subgroups, comparators, outcomes of interest, equality issues, and innovation. Whilst some HTA bodies such as the National Institute for Health and Care Excellence (NICE) in England pre-specify the scope of the evaluation, others, such as the Scottish Medicines Council (SMC), require the manufacturer to define their own decision problem. This can be particularly challenging in diseases where the therapeutic pathway is not well defined, for example in rare diseases or where treatment depends on a number of patient characteristics (health, age, prior treatment etc.). However, choosing the correct comparator(s) is key to HTA success; a common reason for failure with the SMC is not choosing the most appropriate comparator. Furthermore, the sequencing of treatments in the treatment pathway can have profound effects on the cost-effectiveness of therapies, and therefore in the absence of established pathways, manufacturers must establish these carefully and perform appropriate sensitivity analyses as required. We have explored how the lack of a well-defined treatment pathway can result in challenges for defining and addressing the HTA decision problem using examples from recent HTA submissions for treatments in orphan oncology, neuropathic pain, and scleroderma, in England, Scotland, and France. Factors such as collaboration with payers and clinicians and analysis of real world evidence are often used and we will summarise how different methods used to determine treatment pathways have been used by manufacturers with different HTA agencies to demonstrate areas of similarity and difference between countries. PHP364 The Evaluation of Innovative Drugs Applying Business Valuation Concepts Nuijten MJ1, Vis J2 1A2M, Amsterdam, The Netherlands, 2Talanton, Amsterdam, The Netherlands
Objective: Many pharmaceutical companies are now commercialising innovative expensive medicinal products, e.g. biologicals, with an incremental cost-effectiveness ratio (ICER), which will probably exceed the accepted threshold values for reimbursement. The goal of this research is to propose an additional methodology to evaluate innovative drugs from a broader perspective by applying concepts from business valuation, when the ICER exceeds the threshold. Methods: In a pure free health market, the price of the new innovative drug would be determined by demand and supply mechanisms, and all considerations about the use of costeffectiveness data would be redundant. On the other hand, the health authorities leave the responsibility for medical innovation to the market. Therefore medical innovation relies on the market mechanisms in the finance market of biotechnology including the incentives of the capital providers, who demand a required return of investment. The justification of the drug price can be based on the Discounted Cash Flow method. The Discounted Cash Flow method is based on the expected free cash
flows and the required cost of capital, can be used to validate the price of the new drug from a narrow investor’s perspective. The free cash flow represent the sales from the pharmaceuticals, and cash necessary for capital expenditures represent the costs for research & development (R&D) and marketing. The cost of capital refers to the opportunity cost of making a specific investment, which is the rate of return required to persuade the investor to make a given investment. Conclusion: We propose a policy approach for the evaluation of innovative drugs by bridging concepts from health economics and business economic valuation. This approach may justify a drug price from an investor’s perspective when the ICER exceeds the threshold and may be used in negotiations between governments and companies. PHP365 Unlocking Established Products Patient and Societal Value in Additional Indications Nayroles G1, Gabriel S1, Toumi M2, Kornfeld A3, Frybourg S3, Antonanzas F4, Espin J5, Jommi C6, de Pouvourville G7, Tolley K8, Wasem J9 1IPSEN Pharma, Boulogne-Billancourt, France, 2Aix-Marseille University, Marseille, France, 3Creativ-Ceutical, Paris, France, 4University of La Rioja, Logroño, Spain, 5Andalusian School of Public Health, Granada, Spain, 6Cergas, Bocconi University, Milano, Italy, 7ESSEC Business School, Cergy-Pontoise, France, 8Tolley Health Economics, Buxton, UK, 9University Duisburg-Essen, Essen, Germany
Background: New indications for established products (EPs) – marketed for 8 years or more - may represent a high societal value; but pharmaceutical manufacturers criticize the disincentives to expand indication of EPs and deliver the potential value for the society, such as lack of intellectual propriety or data protection, price cuts for new indication, generic competition, internal reference pricing. Incentive policies for orphan drugs or pediatric indications have driven EP development in these specific areas. Capturing the societal benefit of EP development is on the European Commission agenda. The objective of this research was to propose new potential incentives for the development of EPs through an international expert panel. Discussion: The expert panel suggested the following incentives for EPs that provide an added value in new indications: To extend the market protection period following introduction of a new indication To develop differential pricing regulation for different indications of the same active ingredient. Three processes may help achieving differential pricing: Differential market access agreement such as rebate or other financial-based or outcome-based managed market entry agreements for different indications Electronic prescription including indication to ensure the right product is used for each specific indication when generic do not match extension of indication To avoid or delay systematic therapeutic reference pricing Conclusion: Current regulations can represent a serious disincentive to develop new indications for EPs. EPs may offer treatment options with well-known safety. They have a potential to reduce off-label use and contain prescription escalation toward expensive products. Policy changes, both regulatory, through data protection and market exclusivity, and pricing, by rewarding the effort to bring new indication for EPs, are needed to capture the full societal value of EPs. PHP366 Pharmaceutical Tracking System: An Improvement in Data Accuracy and Uses Afify Y Ministry of Health, Cairo, Egypt
Medicines are strategic health commodities that governments are required to provide for people. Tracking systems are new technologies emerged in the different fields of logistics. In pharmaceutical sector, many countries had begun implementing the track and trace system. While implementation in some countries around the world ranges from drafting the legislations and partial testing of the system, others are fully implementing such technology in more than successful fashion. The technology depends on identification of every single pack/unit of medicines of any type once manufactured using a combination of serial number and code in addition to the batch number and expiry date, then recording every transaction applied to the pack in the market using its identity. The last transaction applied to the pack is the dispensing to patient. When the governments integrate the patients’ health records and different registries with the tracking system, every pack can be tied to the patient name and identity. In such system, pharmacoeconomic studies can accurately utilize the accurate data of medicines dispensed and linked to patients. Better epidemiology data will be available for any geographic area using patterns of consumption of different therapeutic classes of medicines. Data provided by tracking of pharmaceuticals can improve the monitoring of supply and demand of medicines and hence pricing. Health technology assessment can be based on real data from the market and precise comparisons are then feasible using different models of decision trees and Markov Model. Big data are easily generated to assess different patterns and trends in the pharmaceutical market. Moreover the technology allows control of the market by preventing counterfeit and smuggled drugs. The new system can effectively supports the rational use of medicines and helps take instant and precise actions with more effective and accurate market surveillance. PHP367 Mapping of The Biosimilar Drug Policy in 10 Central Eastern European Countries Inotai A1, Csanadi M1, Petrova G2, Bochenek T3, Tesar T4, York K5, Fuksa L6, Kostyuk A7, Araja D8, Lorenzovici L9, Egyed K10, Kaló Z11 1Syreon Research Institute, Budapest, Hungary, 2Medical University-Sofia, Faculty of Pharmacy, Sofia, Bulgaria, 3Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, Poland, 4Department of Organisation and Management in Pharmacy, Faculty of Pharmacy, Comenius University, Bratislava, Slovakia, 5Independent, Innsbruck, Austria, 6Department of Social and Clinical Pharmacy, Faculty of Pharmacy, Charles University in Prague, Hradec Králové, Czech Republic, 7Kazakh Agency for Health Technology Assessment, Astana, Kazakhstan, 8Riga Stradins University, Riga, Latvia, 9Syreon Research Romania, Tirgu Mures, Romania, 10Egis Pharmaceuticals, Budapest, Hungary, 11Eötvös Loránd University, Budapest, Hungary