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A Model For Managed Introduction Of New Medicines In Sweden
Clinical Therapeutics/Volume 38, Number 10S, 2016 3-ECONOMICS 3.1 Pharmacoeconomics: How It Can Be Applied To Support Decisions In Health Care? D. Vitezic University of Rijeka School of Medicine and Rijeka University Hospital Centre, Rijeka, Croatia The problem of the limited health care resources is worldwide present, and this logically leads to increased interest in assessing the value for money, or economic efficiency, of new medicines and therapies. The cornerstone in this process of assessment belongs to pharmacoeconomics, which is defined as a scientific discipline that analyses the values of one medicine or medical intervention to another. Pharmacoeconomic evaluation is relevant to decisions about the pricing and reimbursement of different procedures and health technologies, since it offers a way of estimating the additional value to society of a new intervention (e.g., medicine) in comparison to existing therapy. The most frequently used pharmacoeconomic analysis includes cost-effectiveness (CEA) and cost-utility analyses (CUA). These techniques are most appropriately applied when a choice must be made between two or more competing options for which the expected health gains can be expressed in terms of a common outcome measure. It is necessary to emphasize that pharmacoeconomic evaluation as a part of health technology assessment (HTA) is applicable to review therapeutic areas and especially with significant budget impact, including the revision of therapeutic guidelines and financing protocols and delisting of previously reimbursed pharmaceuticals. The importance of such an evidence-based health policy is widely acknowledged among health care policy-makers all over the world. Pharmacoeconomics as part of HTA is a transparent process that provides health care decision-makers with valuable information. This is used in a decision concerning the optimal health care resource allocation in a standardized and scientifically consistent process.
economic evidence, while the full SMC committee is able to take a wider perspective and consider societal values, areas of unmet need, and “special situations,” such as medicines for orphan diseases. Up to 1/3 of new medicines have been found not to represent “value for money” and have therefore not been accepted for routine use; roughly another 1/3 have been accepted only for restricted use, with limitations either around the patients in whom the medicine should be used or the clinicians who should be allowed to prescribe the medicine. Increasingly, medicines not found to represent “value for money” are re-submitted for review with a “patient access scheme,” usually a simple discount on the medicine acquisition cost, often allowing the medicine to be accepted for use but at a significantly lower cost. SMC has shown that rapid, robust HTA of new medicines is possible to very tight timelines – submission to final decision is usually around 18 weeks. Considerable effort has been put in to ensuring implementation of SMC advice. National clinical guidelines (produced by SIGN – Scottish Intercollegiate Guidelines Network) may only recommend medicines accepted by SMC, and local prescribing protocols and formularies are also limited to medicines with SMC approval, so the potential for “mixed messages” to prescribers is minimized. Monitoring of adherence to advice has been undertaken locally and generally with a “light touch,” increasingly using routinely collected prescription information. The collaborative processes have been evolutionary and took over 10 years to get from first steps to the present integrated system. They have been characterized by full involvement of clinicians throughout, with clinician leadership and ownership of the individual components, such as formularies, SMC, and SIGN. Other stakeholders, including the public, patient advocacy groups, and the pharmaceutical industry, are involved in parts of the process as appropriate. The expenditure required to develop and maintain the processes has been modest compared to the gains achieved and thus may represent a useful model for other small/medium-sized health care systems.
3.3 3.2 Rapid Health Technology Assessment Of New Medicines: Lessons From 15 Years Experience In Scotland K.R. Paterson University of Glasgow, Glasgow, United Kingdom Fifteen years ago, the challenges of new, and often expensive, medicines were recognized in Scotland and were addressed by the creation of a national health technology assessment organization (Scottish Medicines Consortium – SMC) to undertake rapid assessment of the clinical effectiveness and cost-effectiveness of all new medicines. SMC involves clinicians (doctors and pharmacists) from both primary and secondary care and from all across Scotland, ensuring short lines of communication to and from the prescribing “front line.” To achieve rapid assessment and minimize the health care system resources required to undertake assessment, the SMC process is based on review of a written submission prepared by the manufacturer of each new medicine to demonstrate the clinical value and costeffectiveness of the medicine. The “burden of proof” of both clinical value and cost-effectiveness lies with the medicine manufacturer. SMC has pharmacist and health economic reviewers with the necessary critical appraisal skills to assess submissions. The SMC process then involves 2 stages – its New Drugs Committee reviews the submission and makes its recommendation based on the clinical and
October 2016
A Model For Managed Introduction Of New Medicines In Sweden Y. Böttiger Linköping University, Linköping, Sweden Many of the new medicines that are now introduced into the market and approved by medical authorities are expensive and aimed at treating diseases confined to a rather narrow patient population, such as selected forms of cancer or autoimmune diseases. The era of blockbusting drugs for large patient groups, such as depression or hypertension, seems to have passed. These new and expensive drugs pose new challenges to the health care organization, public financing of medicines, and the equal distribution of new treatment possibilities throughout the country. In Sweden, the Medical Product Agency approves new medicines (within the EU collaboration), whereas subsidizing of prescription medicines is decided by the Dental and Pharmaceutical Benefits Agency. Furthermore, a national organisation for the managed introduction of new medicines, including those that are only used in the hospital setting, into health care has been created. The aim of this organization is to ensure the equal and cost-effective use of new medicines throughout the country. The procedure of horizon scanning, decision on what new products to focus on, protocols for the introduction and follow-up, economic evaluation, negotiating with manufacturers, recommendation for usage, and evaluation of outcome will be described in this presentation.
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Clinical Therapeutics 3.4 How To Assess The Value Of The Drug? An Overview On Producing Economic Evidence In The R&D Process Z. Kaló Health Economics Research Centre, Eötvös Loránd University and Syreon Research Institute, Budapest, Hungary Due to scarcity of public resources, assessment of the economic value is increasingly important in the pricing and reimbursement process of new pharmaceuticals. As a consequence, pharmaceutical R&D faces the need for a new type of development strategy. Innovators should prove – ideally in a real-world situation - that their new product is worth public financing against the best and most widely used alternative procedures. In addition to efficacy and safety end points, a new type of data must be collected alongside clinical trials, which increases the risks and the expected budget of the R&D program. The value proposition of new medicines can be strengthened by burden of disease and cost of illness studies, naturalistic health economic studies, and economic modelling. Health economists are involved in the pharmaceutical R&D process. They initiate and optimize pharmacoeconomic data collection during the clinical development program so that the cost-effectiveness of investigational medical products can be presented at the time of launch. Appropriate timing of their contribution is essential. During the early development phase, the health economic profile of target indications has to be established. Economic modelling could provide a reasonable estimate of the economic value of the target product profile, which is a key input into the expected net present value calculations at internal R&D decision points. Economic modelling may contribute to “go-no-go” decisions (including business development and licensing, selection of the order of target indications, and strategic pricing of new drugs). The health economic profile defines the major value drivers of the investigational product, based upon which health economic plan can be established. The health economic plan determines activities during the clinical development program between phases I and III. Before launch, the global health economic dossier is prepared by the global health economic team in parallel with the adaptation of results to most important countries or markets. Local health economists in affiliates prepare country-specific health economic dossiers and risk-sharing plans in order to obtain reimbursement and formulary listing in all countries. Health economists in the global teams and affiliates are also involved in the planning and pharmacoeconomic assessment of major phase IV and observational studies to extend the economic evidence base in a constantly changing clinical and economic environment. Finally, in the case of new indications, a new health economic dossier has to be prepared. In order to support the value proposition of new medicines, clinical teams in the pharmaceutical R&D process have to be familiar with the principles of pharmacoeconomics, including selection of health policy-relevant comparators, analytical techniques, and measurement of heath gain by QALYs.
3.5 Evaluation Of Medicines For Reimbursement By The National Social Insurance Institutions In Austria T. Griesbacher1,2; and R. Sauermann3 Medical University of Graz, Graz, Austria; 2Medicines Evaluation Commission, Vienna, Austria; and 3Main Association of Austrian Social Security Institutions, Vienna, Austria
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Hundreds of new medicinal products enter the market each year and apply for reimbursement by national public health services. In order to keep public health systems affordable, it is of paramount importance that the amount of money spent on medicinal products is kept in proportion to the therapeutic benefit of these products for the patients. In Austria, all medicinal products for which the responsible company applies for inclusion in the Code of Reimbursement (EKO), a positive list of drugs reimbursed by the public social insurance institutions, undergo a three-step evaluation performed by the Main Association of Austrian Social Security Institutions. The first step (“pharmacological evaluation”) classifies the product according to its degree of innovation in eight categories, from product with the same active ingredient to product that allows for treatment of a disease for the first time; in addition, based on the drug’s mechanism of action and the indication, the “therapeutic alternatives,” which are already reimbursed and are pharmacologically most similar to the drug, are determined. The second step, the “therapeutic evaluation,” determines the additional therapeutic benefit of the drug in comparison to the therapeutic alternatives, categorized in six categories from no additional benefit to significant additional benefit for the majority of patients. As third step, the “economic evaluation” determines acceptable prices based on the outcome of the therapeutic evaluation: among other criteria that have to be fulfilled (external reference pricing), acceptable prices are determined based on the therapeutic benefit of the new drug compared to that of the therapeutic alternatives. If a significant additional benefit has been determined, a pharmacoeconomic study must demonstrate costeffectiveness for the defined group of patients; for all other benefit categories, strict rules set the acceptable prices in relation to the costs of already reimbursed therapeutic alternatives.
3.6 Current Situation And Future Directions For Health Technology Assessment In Europe F. Meyer Haute Autorité de Santé, Saint Denis, France The first bodies performing Health Technology Assessment (HTA) were set up in the 1980s-1990s in Western Europe. Since then, HTA has been expanding all over Europe and has taken a growing importance. Decision makers for the organization of health care systems face increasingly difficult issues and need to be adequately informed by a sound, solid, well-accepted assessment and appraisal of health technologies. To achieve this, HTA has evolved over time with regard to its scope, organization, objectives, processes, and procedures. HTA is organized at the national (and/or regional) level, and criteria and processes vary from one country to another. Despite these differences, HTA systems have a lot in common, notably a shared priority on the assessment of relative effectiveness, i.e., the benefit over existing therapies. Whether cost-effectiveness is applied or not, and for what purpose, is, in contrast, more country-specific. Since the early 2000s, cooperation between HTA bodies in Europe has been developing, with the support of EU-funded projects. The European Network for HTA (EUnetHTA) is currently starting a new Joint Action that will last 4 years. This operational and scientific level of cooperation has been, since 2013, reinforced by the set-up of a governance body, the HTA Network, as a consequence of the entry in force of the European Directive on cross-border health care. Objectives are to reduce duplication of work, increase quality and consistency of European assessments, and interact with industry to improve the quality of data produced during technology development. At a time when all health care systems are challenged by the arrival of personalized
Volume 38 Number 10S
economic evidence, while the full SMC committee is able to take a wider perspective and consider societal values, areas of unmet need, and “special situations,” such as medicines for orphan diseases. Up to 1/3 of new medicines have been found not to represent “value for money” and have therefore not been accepted for routine use; roughly another 1/3 have been accepted only for restricted use, with limitations either around the patients in whom the medicine should be used or the clinicians who should be allowed to prescribe the medicine. Increasingly, medicines not found to represent “value for money” are re-submitted for review with a “patient access scheme,” usually a simple discount on the medicine acquisition cost, often allowing the medicine to be accepted for use but at a significantly lower cost. SMC has shown that rapid, robust HTA of new medicines is possible to very tight timelines – submission to final decision is usually around 18 weeks. Considerable effort has been put in to ensuring implementation of SMC advice. National clinical guidelines (produced by SIGN – Scottish Intercollegiate Guidelines Network) may only recommend medicines accepted by SMC, and local prescribing protocols and formularies are also limited to medicines with SMC approval, so the potential for “mixed messages” to prescribers is minimized. Monitoring of adherence to advice has been undertaken locally and generally with a “light touch,” increasingly using routinely collected prescription information. The collaborative processes have been evolutionary and took over 10 years to get from first steps to the present integrated system. They have been characterized by full involvement of clinicians throughout, with clinician leadership and ownership of the individual components, such as formularies, SMC, and SIGN. Other stakeholders, including the public, patient advocacy groups, and the pharmaceutical industry, are involved in parts of the process as appropriate. The expenditure required to develop and maintain the processes has been modest compared to the gains achieved and thus may represent a useful model for other small/medium-sized health care systems.
3.3 3.2 Rapid Health Technology Assessment Of New Medicines: Lessons From 15 Years Experience In Scotland K.R. Paterson University of Glasgow, Glasgow, United Kingdom Fifteen years ago, the challenges of new, and often expensive, medicines were recognized in Scotland and were addressed by the creation of a national health technology assessment organization (Scottish Medicines Consortium – SMC) to undertake rapid assessment of the clinical effectiveness and cost-effectiveness of all new medicines. SMC involves clinicians (doctors and pharmacists) from both primary and secondary care and from all across Scotland, ensuring short lines of communication to and from the prescribing “front line.” To achieve rapid assessment and minimize the health care system resources required to undertake assessment, the SMC process is based on review of a written submission prepared by the manufacturer of each new medicine to demonstrate the clinical value and costeffectiveness of the medicine. The “burden of proof” of both clinical value and cost-effectiveness lies with the medicine manufacturer. SMC has pharmacist and health economic reviewers with the necessary critical appraisal skills to assess submissions. The SMC process then involves 2 stages – its New Drugs Committee reviews the submission and makes its recommendation based on the clinical and
October 2016
A Model For Managed Introduction Of New Medicines In Sweden Y. Böttiger Linköping University, Linköping, Sweden Many of the new medicines that are now introduced into the market and approved by medical authorities are expensive and aimed at treating diseases confined to a rather narrow patient population, such as selected forms of cancer or autoimmune diseases. The era of blockbusting drugs for large patient groups, such as depression or hypertension, seems to have passed. These new and expensive drugs pose new challenges to the health care organization, public financing of medicines, and the equal distribution of new treatment possibilities throughout the country. In Sweden, the Medical Product Agency approves new medicines (within the EU collaboration), whereas subsidizing of prescription medicines is decided by the Dental and Pharmaceutical Benefits Agency. Furthermore, a national organisation for the managed introduction of new medicines, including those that are only used in the hospital setting, into health care has been created. The aim of this organization is to ensure the equal and cost-effective use of new medicines throughout the country. The procedure of horizon scanning, decision on what new products to focus on, protocols for the introduction and follow-up, economic evaluation, negotiating with manufacturers, recommendation for usage, and evaluation of outcome will be described in this presentation.
e13
Clinical Therapeutics 3.4 How To Assess The Value Of The Drug? An Overview On Producing Economic Evidence In The R&D Process Z. Kaló Health Economics Research Centre, Eötvös Loránd University and Syreon Research Institute, Budapest, Hungary Due to scarcity of public resources, assessment of the economic value is increasingly important in the pricing and reimbursement process of new pharmaceuticals. As a consequence, pharmaceutical R&D faces the need for a new type of development strategy. Innovators should prove – ideally in a real-world situation - that their new product is worth public financing against the best and most widely used alternative procedures. In addition to efficacy and safety end points, a new type of data must be collected alongside clinical trials, which increases the risks and the expected budget of the R&D program. The value proposition of new medicines can be strengthened by burden of disease and cost of illness studies, naturalistic health economic studies, and economic modelling. Health economists are involved in the pharmaceutical R&D process. They initiate and optimize pharmacoeconomic data collection during the clinical development program so that the cost-effectiveness of investigational medical products can be presented at the time of launch. Appropriate timing of their contribution is essential. During the early development phase, the health economic profile of target indications has to be established. Economic modelling could provide a reasonable estimate of the economic value of the target product profile, which is a key input into the expected net present value calculations at internal R&D decision points. Economic modelling may contribute to “go-no-go” decisions (including business development and licensing, selection of the order of target indications, and strategic pricing of new drugs). The health economic profile defines the major value drivers of the investigational product, based upon which health economic plan can be established. The health economic plan determines activities during the clinical development program between phases I and III. Before launch, the global health economic dossier is prepared by the global health economic team in parallel with the adaptation of results to most important countries or markets. Local health economists in affiliates prepare country-specific health economic dossiers and risk-sharing plans in order to obtain reimbursement and formulary listing in all countries. Health economists in the global teams and affiliates are also involved in the planning and pharmacoeconomic assessment of major phase IV and observational studies to extend the economic evidence base in a constantly changing clinical and economic environment. Finally, in the case of new indications, a new health economic dossier has to be prepared. In order to support the value proposition of new medicines, clinical teams in the pharmaceutical R&D process have to be familiar with the principles of pharmacoeconomics, including selection of health policy-relevant comparators, analytical techniques, and measurement of heath gain by QALYs.
3.5 Evaluation Of Medicines For Reimbursement By The National Social Insurance Institutions In Austria T. Griesbacher1,2; and R. Sauermann3 Medical University of Graz, Graz, Austria; 2Medicines Evaluation Commission, Vienna, Austria; and 3Main Association of Austrian Social Security Institutions, Vienna, Austria
1
e14
Hundreds of new medicinal products enter the market each year and apply for reimbursement by national public health services. In order to keep public health systems affordable, it is of paramount importance that the amount of money spent on medicinal products is kept in proportion to the therapeutic benefit of these products for the patients. In Austria, all medicinal products for which the responsible company applies for inclusion in the Code of Reimbursement (EKO), a positive list of drugs reimbursed by the public social insurance institutions, undergo a three-step evaluation performed by the Main Association of Austrian Social Security Institutions. The first step (“pharmacological evaluation”) classifies the product according to its degree of innovation in eight categories, from product with the same active ingredient to product that allows for treatment of a disease for the first time; in addition, based on the drug’s mechanism of action and the indication, the “therapeutic alternatives,” which are already reimbursed and are pharmacologically most similar to the drug, are determined. The second step, the “therapeutic evaluation,” determines the additional therapeutic benefit of the drug in comparison to the therapeutic alternatives, categorized in six categories from no additional benefit to significant additional benefit for the majority of patients. As third step, the “economic evaluation” determines acceptable prices based on the outcome of the therapeutic evaluation: among other criteria that have to be fulfilled (external reference pricing), acceptable prices are determined based on the therapeutic benefit of the new drug compared to that of the therapeutic alternatives. If a significant additional benefit has been determined, a pharmacoeconomic study must demonstrate costeffectiveness for the defined group of patients; for all other benefit categories, strict rules set the acceptable prices in relation to the costs of already reimbursed therapeutic alternatives.
3.6 Current Situation And Future Directions For Health Technology Assessment In Europe F. Meyer Haute Autorité de Santé, Saint Denis, France The first bodies performing Health Technology Assessment (HTA) were set up in the 1980s-1990s in Western Europe. Since then, HTA has been expanding all over Europe and has taken a growing importance. Decision makers for the organization of health care systems face increasingly difficult issues and need to be adequately informed by a sound, solid, well-accepted assessment and appraisal of health technologies. To achieve this, HTA has evolved over time with regard to its scope, organization, objectives, processes, and procedures. HTA is organized at the national (and/or regional) level, and criteria and processes vary from one country to another. Despite these differences, HTA systems have a lot in common, notably a shared priority on the assessment of relative effectiveness, i.e., the benefit over existing therapies. Whether cost-effectiveness is applied or not, and for what purpose, is, in contrast, more country-specific. Since the early 2000s, cooperation between HTA bodies in Europe has been developing, with the support of EU-funded projects. The European Network for HTA (EUnetHTA) is currently starting a new Joint Action that will last 4 years. This operational and scientific level of cooperation has been, since 2013, reinforced by the set-up of a governance body, the HTA Network, as a consequence of the entry in force of the European Directive on cross-border health care. Objectives are to reduce duplication of work, increase quality and consistency of European assessments, and interact with industry to improve the quality of data produced during technology development. At a time when all health care systems are challenged by the arrival of personalized
Volume 38 Number 10S