The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agr... more The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agreements has emerged as a key element in the United States' TRIPS-Plus intellectual property (IP) negotiating agenda. However, the provisions establishing patent linkage mechanisms in several agreements appear to reflect a degree of ambiguity, potentially enabling some flexibility in their implementation. In this study, we reviewed the features of the prototypic patent linkage mechanism established by the Hatch-Waxman Act in the United States, and compared these with the implementation of systems in three countries whose agreements with the US include patent linkage obligations. From these analyses, we draw lessons for moderating the impact of these mechanisms on access to generic medicines. Methods: We reviewed the features of the patent linkage mechanism in the US, and undertook a detailed analysis of relevant treaty provisions and the manner of implementation in Canada, Australia, and South Korea. Results: A key difference between the US implementation of patent linkage and that of its trading partners is the disparate treatment afforded to biologics. Because of the significant differences in the regulatory frameworks applying to small molecule and biologic medicines in the US, the Hatch-Waxman provisions do not apply to biologics and they are not subject to patent linkage. By contrast, the regulatory frameworks in Canada, Australia and South Korea do not reflect similar distinctions and thus patent linkage mechanisms also capture biologics. Additional variations in implementation, mainly the result of constructive ambiguities in the respective treaty texts, offer potential opportunity for mitigating the adverse impact of patent linkage provisions on market entry of generic medicines. Practical measures include ensuring the availability of an accessible, transparent and easily searchable database of patent information; avoiding automatic stays of generic marketing approval where possible; and requiring certification by rights holders to prevent abuse of the system. Where countries accept treaty obligations to establish patent linkage mechanisms, the impact on access to generic medicines may be moderated to a degree by retaining and exploiting constructive ambiguities in the treaty text and addressing practical aspects of implementation.
Intellectual property (IP) protections proposed by the United States for the Trans-Pacific Part... more Intellectual property (IP) protections proposed by the United States for the Trans-Pacific Partnership Agreement (TPPA) have sparked widespread alarm about the potential negative impact on access to affordable medicines.
This paper provides an overview of the use of pharmacoeconomic analysis in the process that gover... more This paper provides an overview of the use of pharmacoeconomic analysis in the process that governs drug reimbursement decisions in Australia. It discusses the methods by which drugs are evaluated, both clinically and economically, and the means by which these 2 facets are amalgamated; the types of pharmacoeconomic data submitted in support of requests for reimbursement; the methods and standards used to assess these data; some of the more commonly encountered flaws in the data submitted; and how the different types of data influence reimbursement decisions.
We examined persistence in seven common preventive health practices for a nationally representati... more We examined persistence in seven common preventive health practices for a nationally representative sample of Medicare beneficiaries over 4-year observation periods. Six panels from the 1997-2005 Medicare Current Beneficiary Survey (MCBS) were used resulting in 13,913 unique individuals with ages ranging from below 65 (disabled) to over 80 years old. Persistence in behavior was defined as the proportion of the observation period beneficiaries participated in each activity. We estimated behavioral persistence as a function of baseline demographic, socioeconomic, and health characteristics using multivariate regression analysis. Beneficiaries were most persistent in smoking abstinence (81% reported not smoking) and least persistent with routine exercise (47% reporting none). From multivariate regression results, there was greater persistence among beneficiaries who were married when compared to those living alone (p < 0.01 except for weekly exercise, p < 0.05 and cholesterol screening, ns), with at least a high school education compared to no high school (p < 0.01 for weekly exercise, prostate cancer screening, pap smear, p < 0.05 for influenza vaccination and mammography, but ns for smoking cessation and cholesterol screening), and of higher income (>300% FPL compared to <100% FPL all p < 0.01). Increasing age (greater than 80 compared to 65-69) was associated with increased compliance in influenza vaccination and smoking cessation (p < 0.01) while negatively associated with weekly exercise and cancer screenings (p < 0.01). Medicare beneficiaries are inconsistently persistent with common preventive health practices.
Effects due to decision-making processes are unclear, although specific components or outcomes of... more Effects due to decision-making processes are unclear, although specific components or outcomes of these processes, such as payment, product categorization, and cost sharing, may have more discernable effects (described below). In theory, decisionmaking processes that are transparent and evidence-based are more likely to foster innovation that enhances consumer welfare by sending clearer signals to developers about the types of products that payers place more value on, and how they assess that value. Timeliness and consistency in decision-making processes may also help by reducing developers' and investors' level of uncertainty about the likelihood of payers reimbursing a new product. Product categorization or differentiation Empirical evidence is limited concerning the effects on ROI or innovation from the approaches to product categorization or differentiation used by payers. In theory, approaches that distinguish products based on value are more likely than administrative approaches to promote investment in and development of products that are clinically and cost-effective and address areas of unmet need. Method of payment With per-unit payments, effects on innovation largely depend on the method used to determine payment amounts, as discussed in the next panel of this table. With bundled payment, effects on innovation are uncertain. Radical innovations are unlikely to be affected by bundled payments because of the level of benefits they provide and the likelihood that they will be paid as an add-on to the bundle. At the same time, there may be significant disincentives for incremental innovations, unless they are cost-reducing. Effects of bundled payment on substantial innovations, which fall between radical and incremental innovations, are unclear. Method of defining payment amount External benchmarking, a process of defining a payment level based on the sales price in the market or markets in which the product is sold, or an estimate of the provider's acquisition cost, is likely to increase ROI and incentives to innovate, compared to other approaches used to define payment amounts. The effects of internal benchmarking, or defining a payment level based on what is paid for comparable covered products for which the payer has already established a payment amount, are largely unclear, but it is most likely that this approach will reduce ROI and incentives to innovate. Valuebased approaches are the most promising for yielding effects on ROI that reflect products' benefits relative to their costs (judged from consumer, payer, and/or societal perspective). Effects of lowest possible price strategies are unclear, but this approach may over-incentivize investment in incremental innovations and underincentivize investment in radical innovations. Patient cost sharing Although there is substantial evidence that cost sharing affects utilization, cost sharing seems unlikely to have substantial effects on ROI or incentives to develop or invest in innovative products. Patient demand for innovative products is likely to be relatively inelastic, especially for radical innovations. Coinsurance may lead to greater effects compared to fixed copayments, especially for high-cost products, but it still seems unlikely to have significant effects on innovation. Manufacturers' programs that help patients with their cost sharing further limit potential effects.
Responding to Smith et al. (Nature, 2014), this paper argues that for medical use, faecal microbi... more Responding to Smith et al. (Nature, 2014), this paper argues that for medical use, faecal microbiota transplantation (FMT) should be considered a sui generis biological drug, rather than a tissue. Smith and colleagues' thesis is based on possible undesirable economic consequences of this designation--not on its scientific and conceptual basis. The faecal transplant (including gut microbiota, metabolites, mucus, human cells, viruses, fungi, etc.) is not a tissue; it is of topographic--not cellular--human origin. We consider the donor a bioreactor, producing the faecal substrate of therapeutic interest. The debate is of singular importance as the FDA considers FMT a drug and released a new guidance for public consultation in February 2014, whereas to date the European Medicines Agency has not promulgated its position. The UK's National Institute for Heath and Care Excellence does not consider FMT to involve the transplantation of body tissue, and in March 2014 the French regul...
Analyzes British, Australian, and German health systems' policies on covering expensive end-o... more Analyzes British, Australian, and German health systems' policies on covering expensive end-of-life drugs while maintaining equity and efficiency, such as requiring evidence of comparative clinical and cost-effectiveness. Outlines challenges for the U.S
Australian health review : a publication of the Australian Hospital Association, Jan 9, 2017
Objectives The aim of the study was to estimate the potential savings to the Pharmaceutical Benef... more Objectives The aim of the study was to estimate the potential savings to the Pharmaceutical Benefits Scheme (PBS) and the Repatriation Pharmaceutical Benefits Scheme (RPBS) in 2015-16 if biosimilar versions of selected biologic medicines (biologics) had been available and listed on the PBS.Methods The research involved retrospective analysis of Australian Medicare expenditure data and PBS price data from 2015-16 for biologics, for which biosimilar competition may be available in future, listed on the PBS.Results Australian Government expenditure on biologics on the PBS and RPBS was estimated at A$2.29 billion dollars in 2015-16. If biosimilar versions of these medicines had been listed on the PBS in 2015-16, at least A$367million dollars would have been saved in PBS and RPBS subsidies. Modelling based on price decreases following listing of biosimilars on the PBS suggests that annual PBS outlays on biologics could be reduced by as much as 24% through the timely introduction of biosi...
The Journal of law, medicine & ethics : a journal of the American Society of Law, Medicine & Ethics, 2012
Misuse or misunderstanding of medication information is a common and costly problem in the U.S. T... more Misuse or misunderstanding of medication information is a common and costly problem in the U.S. The risks of misunderstanding medication information are compounded for the large and growing population of individuals with limited English proficiency that often lacks access to this information in their own language. This paper examines practices related to translation of medication information in the European Union that may serve as a model for future U.S. policy efforts to improve the quality and availability of medication information for individuals with limited English proficiency.
In 2005, CMS issued a national coverage determination for Positron Emission Tomography (PET) for ... more In 2005, CMS issued a national coverage determination for Positron Emission Tomography (PET) for oncologic indications not previously covered by Medicare, which provided payment for PET scans for these indications only in the context of an approved prospective clinical trial designed to assess clinical utility of PET. CMS instated this policy option, referred to as 'Coverage with Evidence Development' (CED), due to the lack of evidence demonstrating the clinical effectiveness of PET scans for patient management, which is necessary to determine whether PET scans are reasonable and necessary for the diagnosis, staging, re-staging, and monitoring of various cancer types. CED allowed CMS to guide evidence development while providing access to this potentially benefi cial technology. GOALS: To determine whether PET imaging is clinically effective for previously uncovered oncologic indications. OUTCOMES ITEMS USED IN THE DECISION: The main outcomes were change in intended management strategy and whether the PET scan allowed physicians to avoid other tests or procedures. Based on these, Medicare's Evidence Development and Coverage Advisory Committee (MEDCAC) members had to decide how confi dent they were that PET improves physician decision-making and clinical outcomes, and that the conclusions were generalizable to other cancers, to PET facilities in the general community, and to the Medicare population. IMPLEMENTATION STRATEGY: Since CMS did not have the capacity to design and fund a study, the agency partnered with the American College of Radiology and the Academy of Molecular Imaging. The study itself had to be implemented within a short time do to federal regulations. The registry began patient and physician registration in May of 2006. This design was chosen in part because it balanced the desire for access to this service with the goal of generating evidence of reasonable quality that could be used by CMS to make a fi nal coverage determination. RESULTS: After the fi rst year, most PET facilities in the United States had signed up to participate in the registry and a huge nationwide sample of data had been collected. The results from this data analysis demonstrate that physicians report a change in intended disease management strategy in about one out of every three cases and that when broken down by disease cancer type and indication, the fi gure remains fairly constant (Hillner et al. 2008). This evidence was reviewed in August 2008 at a meeting of MEDCAC. Based on these results and the results of a health technology assessment, MEDCAC members were asked to rate their confi dence in the clinical utility of PET and the generalizability of the conclusions. The ratings demonstrated that MEDCAC members have limited confi dence that PET improves clinical outcomes based on the evidence. Members pointed out that although the results show that physicians often change their intended management plan, there is no way to know if actual management changed and even if it did, it is also not known whether these changes actually lead to better patient outcomes. Still, on January 6, 2009, CMS issued a decision memo stating that there is now suffi cient evidence showing that PET improves health outcomes when used for the diagnosis and staging of all previously uncovered cancer types, warranting coverage for these indications. However, CMS does not believe that there is enough evidence demonstrating the clinical utility of PET for monitoring response to treatment and re-staging. Therefore, these indications will still only be covered through the CED policy, likely necessitating the development of a new prospective study. LESSONS LEARNED: The case presented above not only demonstrates that CED is operationally and technically possible while abiding to regulatory procedures, it also demonstrates CED can be used to help support research efforts designed to address questions of importance to health care decision-makers. In addition, as this is one of the fi rst examples of Medicare taking advantage of CED, it also offers some lessons for the future. For instance, in order to streamline the process, it is necessary to identify a stable source of funding for these projects, to identify promising technologies which lack evidence of clinical effectiveness earlier in the development process, to reach a shared understanding among stakeholders of standards of evidence that are feasible and suffi ciently robust for coverage decisions, and to reach a consensus as to the most effi cient methods for conducting real world trials. In the future, it would be best if decisions of study design did not take place while Medicare was making a coverage determination and if private payers became involved in CED so a broader range of patients could participate in the studies.
In Australia, most prescription drugs are subsidized through the Pharmaceutical Benefits Scheme (... more In Australia, most prescription drugs are subsidized through the Pharmaceutical Benefits Scheme (PBS), one of several government programs in which evidence-based decision making is applied to the funding of health technologies. PBS processes are intended to ensure "value for money" for the Australian taxpayer and to support affordable, equitable access to prescription medicines; they are not intended as a mechanism for cost containment. The inclusion of a drug on the national formulary depends on the recommendation of the Pharmaceutical Benefits Advisory Committee (PBAC), which considers not only the comparative effectiveness but also the comparative cost-effectiveness of drugs proposed for listing. While some decisions have been controversial, the PBS retains strong public support. Moreover, evidence does not suggest that the consideration of cost-effectiveness has created a negative environment for the drug industry: Australia has a high penetration of patented medicines...
This study examines the key features of government agencies created in Australia, France, Germany... more This study examines the key features of government agencies created in Australia, France, Germany, and the United Kingdom to lead research into the comparative effectiveness of medical treatments, procedures, and technologies. In each of the four countries studied, the agencies have a clear mandate to produce information that will inform clinical and health policy decisions. By contrast, comparative effectiveness legislation under consideration in the U.S. Senate explicitly separates the generation of knowledge from health care decision-making.
... London Due for review: 2005 Council Report CR102 April 2002 Page 2. 2 Membership of the Worki... more ... London Due for review: 2005 Council Report CR102 April 2002 Page 2. 2 Membership of the Working Group Dr Gillian Mezey (Chairman) South-West London and St George's Mental Health NHS Trust, London Dr Madelyn Hicks South London and Maudsley NHS Trust, London ...
Background: The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and in... more Background: The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agreements has emerged as a key element in the United States' TRIPS-Plus intellectual property (IP) negotiating agenda. However, the provisions establishing patent linkage mechanisms in several agreements appear to reflect a degree of ambiguity, potentially enabling some flexibility in their implementation. In this study, we reviewed the features of the prototypic patent linkage mechanism established by the Hatch-Waxman Act in the United States, and compared these with the implementation of systems in three countries whose agreements with the US include patent linkage obligations. From these analyses, we draw lessons for moderating the impact of these mechanisms on access to generic medicines. Methods: We reviewed the features of the patent linkage mechanism in the US, and undertook a detailed analysis of relevant treaty provisions and the manner of implementation in Canada, Australia, and South Korea. Results: A key difference between the US implementation of patent linkage and that of its trading partners is the disparate treatment afforded to biologics. Because of the significant differences in the regulatory frameworks applying to small molecule and biologic medicines in the US, the Hatch-Waxman provisions do not apply to biologics and they are not subject to patent linkage. By contrast, the regulatory frameworks in Canada, Australia and South Korea do not reflect similar distinctions and thus patent linkage mechanisms also capture biologics. Additional variations in implementation, mainly the result of constructive ambiguities in the respective treaty texts, offer potential opportunity for mitigating the adverse impact of patent linkage provisions on market entry of generic medicines. Practical measures include ensuring the availability of an accessible, transparent and easily searchable database of patent information; avoiding automatic stays of generic marketing approval where possible; and requiring certification by rights holders to prevent abuse of the system. Conclusions: Where countries accept treaty obligations to establish patent linkage mechanisms, the impact on access to generic medicines may be moderated to a degree by retaining and exploiting constructive ambiguities in the treaty text and addressing practical aspects of implementation.
The final text of the Trans Pacific Partnership Agreement (TPP), agreed between the 12 negotiatin... more The final text of the Trans Pacific Partnership Agreement (TPP), agreed between the 12 negotiating countries in 2016, included a suite of intellectual property provisions intended to expand and extend pharmaceutical company exclusivities on medicines. It drew wide criticism for including such provisions in an agreement that involved developing countries (Vietnam, Peru, Malaysia, Mexico, Chile and Brunei Darussalam) because of the effect on delaying the introduction of low-cost generics. While developing nations negotiated transition periods for implementing some obligations, all parties would have eventually been expected to meet the same standards had the TPP come into force. While the TPP has stalled following US withdrawal, there are moves by some of the remaining countries to reinvigorate the agreement without the United States. The proponents may seek to retain as much as possible of the original text in the hope that the United States will re-join the accord in future. This ar...
Medication use may be a target for quality improvement, cost containment, and research. We aimed ... more Medication use may be a target for quality improvement, cost containment, and research. We aimed to identify medication classes associated with the highest expenditures among pediatric Medicaid enrollees and to characterize the demographic, clinical, and health service use of children prescribed these medications. Retrospective, cross-sectional study of 3 271 081 Medicaid-enrolled children. Outpatient medication spending among high-expenditure medication classes, defined as the 10 most expensive among 261 mutually exclusive medication classes, was determined by using transaction prices paid to pharmacies by Medicaid agencies and managed care plans among prescriptions filled and dispensed in 2013. Outpatient medications accounted for 16.6% of all Medicaid expenditures. The 10 most expensive medication classes accounted for 63.9% of all medication expenditures. Stimulants (amphetamine-type) accounted for both the highest proportion of expenditures (20.6%) and days of medication use (1...
The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agr... more The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agreements has emerged as a key element in the United States' TRIPS-Plus intellectual property (IP) negotiating agenda. However, the provisions establishing patent linkage mechanisms in several agreements appear to reflect a degree of ambiguity, potentially enabling some flexibility in their implementation. In this study, we reviewed the features of the prototypic patent linkage mechanism established by the Hatch-Waxman Act in the United States, and compared these with the implementation of systems in three countries whose agreements with the US include patent linkage obligations. From these analyses, we draw lessons for moderating the impact of these mechanisms on access to generic medicines. Methods: We reviewed the features of the patent linkage mechanism in the US, and undertook a detailed analysis of relevant treaty provisions and the manner of implementation in Canada, Australia, and South Korea. Results: A key difference between the US implementation of patent linkage and that of its trading partners is the disparate treatment afforded to biologics. Because of the significant differences in the regulatory frameworks applying to small molecule and biologic medicines in the US, the Hatch-Waxman provisions do not apply to biologics and they are not subject to patent linkage. By contrast, the regulatory frameworks in Canada, Australia and South Korea do not reflect similar distinctions and thus patent linkage mechanisms also capture biologics. Additional variations in implementation, mainly the result of constructive ambiguities in the respective treaty texts, offer potential opportunity for mitigating the adverse impact of patent linkage provisions on market entry of generic medicines. Practical measures include ensuring the availability of an accessible, transparent and easily searchable database of patent information; avoiding automatic stays of generic marketing approval where possible; and requiring certification by rights holders to prevent abuse of the system. Where countries accept treaty obligations to establish patent linkage mechanisms, the impact on access to generic medicines may be moderated to a degree by retaining and exploiting constructive ambiguities in the treaty text and addressing practical aspects of implementation.
Intellectual property (IP) protections proposed by the United States for the Trans-Pacific Part... more Intellectual property (IP) protections proposed by the United States for the Trans-Pacific Partnership Agreement (TPPA) have sparked widespread alarm about the potential negative impact on access to affordable medicines.
This paper provides an overview of the use of pharmacoeconomic analysis in the process that gover... more This paper provides an overview of the use of pharmacoeconomic analysis in the process that governs drug reimbursement decisions in Australia. It discusses the methods by which drugs are evaluated, both clinically and economically, and the means by which these 2 facets are amalgamated; the types of pharmacoeconomic data submitted in support of requests for reimbursement; the methods and standards used to assess these data; some of the more commonly encountered flaws in the data submitted; and how the different types of data influence reimbursement decisions.
We examined persistence in seven common preventive health practices for a nationally representati... more We examined persistence in seven common preventive health practices for a nationally representative sample of Medicare beneficiaries over 4-year observation periods. Six panels from the 1997-2005 Medicare Current Beneficiary Survey (MCBS) were used resulting in 13,913 unique individuals with ages ranging from below 65 (disabled) to over 80 years old. Persistence in behavior was defined as the proportion of the observation period beneficiaries participated in each activity. We estimated behavioral persistence as a function of baseline demographic, socioeconomic, and health characteristics using multivariate regression analysis. Beneficiaries were most persistent in smoking abstinence (81% reported not smoking) and least persistent with routine exercise (47% reporting none). From multivariate regression results, there was greater persistence among beneficiaries who were married when compared to those living alone (p < 0.01 except for weekly exercise, p < 0.05 and cholesterol screening, ns), with at least a high school education compared to no high school (p < 0.01 for weekly exercise, prostate cancer screening, pap smear, p < 0.05 for influenza vaccination and mammography, but ns for smoking cessation and cholesterol screening), and of higher income (>300% FPL compared to <100% FPL all p < 0.01). Increasing age (greater than 80 compared to 65-69) was associated with increased compliance in influenza vaccination and smoking cessation (p < 0.01) while negatively associated with weekly exercise and cancer screenings (p < 0.01). Medicare beneficiaries are inconsistently persistent with common preventive health practices.
Effects due to decision-making processes are unclear, although specific components or outcomes of... more Effects due to decision-making processes are unclear, although specific components or outcomes of these processes, such as payment, product categorization, and cost sharing, may have more discernable effects (described below). In theory, decisionmaking processes that are transparent and evidence-based are more likely to foster innovation that enhances consumer welfare by sending clearer signals to developers about the types of products that payers place more value on, and how they assess that value. Timeliness and consistency in decision-making processes may also help by reducing developers' and investors' level of uncertainty about the likelihood of payers reimbursing a new product. Product categorization or differentiation Empirical evidence is limited concerning the effects on ROI or innovation from the approaches to product categorization or differentiation used by payers. In theory, approaches that distinguish products based on value are more likely than administrative approaches to promote investment in and development of products that are clinically and cost-effective and address areas of unmet need. Method of payment With per-unit payments, effects on innovation largely depend on the method used to determine payment amounts, as discussed in the next panel of this table. With bundled payment, effects on innovation are uncertain. Radical innovations are unlikely to be affected by bundled payments because of the level of benefits they provide and the likelihood that they will be paid as an add-on to the bundle. At the same time, there may be significant disincentives for incremental innovations, unless they are cost-reducing. Effects of bundled payment on substantial innovations, which fall between radical and incremental innovations, are unclear. Method of defining payment amount External benchmarking, a process of defining a payment level based on the sales price in the market or markets in which the product is sold, or an estimate of the provider's acquisition cost, is likely to increase ROI and incentives to innovate, compared to other approaches used to define payment amounts. The effects of internal benchmarking, or defining a payment level based on what is paid for comparable covered products for which the payer has already established a payment amount, are largely unclear, but it is most likely that this approach will reduce ROI and incentives to innovate. Valuebased approaches are the most promising for yielding effects on ROI that reflect products' benefits relative to their costs (judged from consumer, payer, and/or societal perspective). Effects of lowest possible price strategies are unclear, but this approach may over-incentivize investment in incremental innovations and underincentivize investment in radical innovations. Patient cost sharing Although there is substantial evidence that cost sharing affects utilization, cost sharing seems unlikely to have substantial effects on ROI or incentives to develop or invest in innovative products. Patient demand for innovative products is likely to be relatively inelastic, especially for radical innovations. Coinsurance may lead to greater effects compared to fixed copayments, especially for high-cost products, but it still seems unlikely to have significant effects on innovation. Manufacturers' programs that help patients with their cost sharing further limit potential effects.
Responding to Smith et al. (Nature, 2014), this paper argues that for medical use, faecal microbi... more Responding to Smith et al. (Nature, 2014), this paper argues that for medical use, faecal microbiota transplantation (FMT) should be considered a sui generis biological drug, rather than a tissue. Smith and colleagues' thesis is based on possible undesirable economic consequences of this designation--not on its scientific and conceptual basis. The faecal transplant (including gut microbiota, metabolites, mucus, human cells, viruses, fungi, etc.) is not a tissue; it is of topographic--not cellular--human origin. We consider the donor a bioreactor, producing the faecal substrate of therapeutic interest. The debate is of singular importance as the FDA considers FMT a drug and released a new guidance for public consultation in February 2014, whereas to date the European Medicines Agency has not promulgated its position. The UK's National Institute for Heath and Care Excellence does not consider FMT to involve the transplantation of body tissue, and in March 2014 the French regul...
Analyzes British, Australian, and German health systems' policies on covering expensive end-o... more Analyzes British, Australian, and German health systems' policies on covering expensive end-of-life drugs while maintaining equity and efficiency, such as requiring evidence of comparative clinical and cost-effectiveness. Outlines challenges for the U.S
Australian health review : a publication of the Australian Hospital Association, Jan 9, 2017
Objectives The aim of the study was to estimate the potential savings to the Pharmaceutical Benef... more Objectives The aim of the study was to estimate the potential savings to the Pharmaceutical Benefits Scheme (PBS) and the Repatriation Pharmaceutical Benefits Scheme (RPBS) in 2015-16 if biosimilar versions of selected biologic medicines (biologics) had been available and listed on the PBS.Methods The research involved retrospective analysis of Australian Medicare expenditure data and PBS price data from 2015-16 for biologics, for which biosimilar competition may be available in future, listed on the PBS.Results Australian Government expenditure on biologics on the PBS and RPBS was estimated at A$2.29 billion dollars in 2015-16. If biosimilar versions of these medicines had been listed on the PBS in 2015-16, at least A$367million dollars would have been saved in PBS and RPBS subsidies. Modelling based on price decreases following listing of biosimilars on the PBS suggests that annual PBS outlays on biologics could be reduced by as much as 24% through the timely introduction of biosi...
The Journal of law, medicine & ethics : a journal of the American Society of Law, Medicine & Ethics, 2012
Misuse or misunderstanding of medication information is a common and costly problem in the U.S. T... more Misuse or misunderstanding of medication information is a common and costly problem in the U.S. The risks of misunderstanding medication information are compounded for the large and growing population of individuals with limited English proficiency that often lacks access to this information in their own language. This paper examines practices related to translation of medication information in the European Union that may serve as a model for future U.S. policy efforts to improve the quality and availability of medication information for individuals with limited English proficiency.
In 2005, CMS issued a national coverage determination for Positron Emission Tomography (PET) for ... more In 2005, CMS issued a national coverage determination for Positron Emission Tomography (PET) for oncologic indications not previously covered by Medicare, which provided payment for PET scans for these indications only in the context of an approved prospective clinical trial designed to assess clinical utility of PET. CMS instated this policy option, referred to as 'Coverage with Evidence Development' (CED), due to the lack of evidence demonstrating the clinical effectiveness of PET scans for patient management, which is necessary to determine whether PET scans are reasonable and necessary for the diagnosis, staging, re-staging, and monitoring of various cancer types. CED allowed CMS to guide evidence development while providing access to this potentially benefi cial technology. GOALS: To determine whether PET imaging is clinically effective for previously uncovered oncologic indications. OUTCOMES ITEMS USED IN THE DECISION: The main outcomes were change in intended management strategy and whether the PET scan allowed physicians to avoid other tests or procedures. Based on these, Medicare's Evidence Development and Coverage Advisory Committee (MEDCAC) members had to decide how confi dent they were that PET improves physician decision-making and clinical outcomes, and that the conclusions were generalizable to other cancers, to PET facilities in the general community, and to the Medicare population. IMPLEMENTATION STRATEGY: Since CMS did not have the capacity to design and fund a study, the agency partnered with the American College of Radiology and the Academy of Molecular Imaging. The study itself had to be implemented within a short time do to federal regulations. The registry began patient and physician registration in May of 2006. This design was chosen in part because it balanced the desire for access to this service with the goal of generating evidence of reasonable quality that could be used by CMS to make a fi nal coverage determination. RESULTS: After the fi rst year, most PET facilities in the United States had signed up to participate in the registry and a huge nationwide sample of data had been collected. The results from this data analysis demonstrate that physicians report a change in intended disease management strategy in about one out of every three cases and that when broken down by disease cancer type and indication, the fi gure remains fairly constant (Hillner et al. 2008). This evidence was reviewed in August 2008 at a meeting of MEDCAC. Based on these results and the results of a health technology assessment, MEDCAC members were asked to rate their confi dence in the clinical utility of PET and the generalizability of the conclusions. The ratings demonstrated that MEDCAC members have limited confi dence that PET improves clinical outcomes based on the evidence. Members pointed out that although the results show that physicians often change their intended management plan, there is no way to know if actual management changed and even if it did, it is also not known whether these changes actually lead to better patient outcomes. Still, on January 6, 2009, CMS issued a decision memo stating that there is now suffi cient evidence showing that PET improves health outcomes when used for the diagnosis and staging of all previously uncovered cancer types, warranting coverage for these indications. However, CMS does not believe that there is enough evidence demonstrating the clinical utility of PET for monitoring response to treatment and re-staging. Therefore, these indications will still only be covered through the CED policy, likely necessitating the development of a new prospective study. LESSONS LEARNED: The case presented above not only demonstrates that CED is operationally and technically possible while abiding to regulatory procedures, it also demonstrates CED can be used to help support research efforts designed to address questions of importance to health care decision-makers. In addition, as this is one of the fi rst examples of Medicare taking advantage of CED, it also offers some lessons for the future. For instance, in order to streamline the process, it is necessary to identify a stable source of funding for these projects, to identify promising technologies which lack evidence of clinical effectiveness earlier in the development process, to reach a shared understanding among stakeholders of standards of evidence that are feasible and suffi ciently robust for coverage decisions, and to reach a consensus as to the most effi cient methods for conducting real world trials. In the future, it would be best if decisions of study design did not take place while Medicare was making a coverage determination and if private payers became involved in CED so a broader range of patients could participate in the studies.
In Australia, most prescription drugs are subsidized through the Pharmaceutical Benefits Scheme (... more In Australia, most prescription drugs are subsidized through the Pharmaceutical Benefits Scheme (PBS), one of several government programs in which evidence-based decision making is applied to the funding of health technologies. PBS processes are intended to ensure "value for money" for the Australian taxpayer and to support affordable, equitable access to prescription medicines; they are not intended as a mechanism for cost containment. The inclusion of a drug on the national formulary depends on the recommendation of the Pharmaceutical Benefits Advisory Committee (PBAC), which considers not only the comparative effectiveness but also the comparative cost-effectiveness of drugs proposed for listing. While some decisions have been controversial, the PBS retains strong public support. Moreover, evidence does not suggest that the consideration of cost-effectiveness has created a negative environment for the drug industry: Australia has a high penetration of patented medicines...
This study examines the key features of government agencies created in Australia, France, Germany... more This study examines the key features of government agencies created in Australia, France, Germany, and the United Kingdom to lead research into the comparative effectiveness of medical treatments, procedures, and technologies. In each of the four countries studied, the agencies have a clear mandate to produce information that will inform clinical and health policy decisions. By contrast, comparative effectiveness legislation under consideration in the U.S. Senate explicitly separates the generation of knowledge from health care decision-making.
... London Due for review: 2005 Council Report CR102 April 2002 Page 2. 2 Membership of the Worki... more ... London Due for review: 2005 Council Report CR102 April 2002 Page 2. 2 Membership of the Working Group Dr Gillian Mezey (Chairman) South-West London and St George's Mental Health NHS Trust, London Dr Madelyn Hicks South London and Maudsley NHS Trust, London ...
Background: The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and in... more Background: The inclusion of patent linkage mechanisms in bilateral and plurilateral trade and investment agreements has emerged as a key element in the United States' TRIPS-Plus intellectual property (IP) negotiating agenda. However, the provisions establishing patent linkage mechanisms in several agreements appear to reflect a degree of ambiguity, potentially enabling some flexibility in their implementation. In this study, we reviewed the features of the prototypic patent linkage mechanism established by the Hatch-Waxman Act in the United States, and compared these with the implementation of systems in three countries whose agreements with the US include patent linkage obligations. From these analyses, we draw lessons for moderating the impact of these mechanisms on access to generic medicines. Methods: We reviewed the features of the patent linkage mechanism in the US, and undertook a detailed analysis of relevant treaty provisions and the manner of implementation in Canada, Australia, and South Korea. Results: A key difference between the US implementation of patent linkage and that of its trading partners is the disparate treatment afforded to biologics. Because of the significant differences in the regulatory frameworks applying to small molecule and biologic medicines in the US, the Hatch-Waxman provisions do not apply to biologics and they are not subject to patent linkage. By contrast, the regulatory frameworks in Canada, Australia and South Korea do not reflect similar distinctions and thus patent linkage mechanisms also capture biologics. Additional variations in implementation, mainly the result of constructive ambiguities in the respective treaty texts, offer potential opportunity for mitigating the adverse impact of patent linkage provisions on market entry of generic medicines. Practical measures include ensuring the availability of an accessible, transparent and easily searchable database of patent information; avoiding automatic stays of generic marketing approval where possible; and requiring certification by rights holders to prevent abuse of the system. Conclusions: Where countries accept treaty obligations to establish patent linkage mechanisms, the impact on access to generic medicines may be moderated to a degree by retaining and exploiting constructive ambiguities in the treaty text and addressing practical aspects of implementation.
The final text of the Trans Pacific Partnership Agreement (TPP), agreed between the 12 negotiatin... more The final text of the Trans Pacific Partnership Agreement (TPP), agreed between the 12 negotiating countries in 2016, included a suite of intellectual property provisions intended to expand and extend pharmaceutical company exclusivities on medicines. It drew wide criticism for including such provisions in an agreement that involved developing countries (Vietnam, Peru, Malaysia, Mexico, Chile and Brunei Darussalam) because of the effect on delaying the introduction of low-cost generics. While developing nations negotiated transition periods for implementing some obligations, all parties would have eventually been expected to meet the same standards had the TPP come into force. While the TPP has stalled following US withdrawal, there are moves by some of the remaining countries to reinvigorate the agreement without the United States. The proponents may seek to retain as much as possible of the original text in the hope that the United States will re-join the accord in future. This ar...
Medication use may be a target for quality improvement, cost containment, and research. We aimed ... more Medication use may be a target for quality improvement, cost containment, and research. We aimed to identify medication classes associated with the highest expenditures among pediatric Medicaid enrollees and to characterize the demographic, clinical, and health service use of children prescribed these medications. Retrospective, cross-sectional study of 3 271 081 Medicaid-enrolled children. Outpatient medication spending among high-expenditure medication classes, defined as the 10 most expensive among 261 mutually exclusive medication classes, was determined by using transaction prices paid to pharmacies by Medicaid agencies and managed care plans among prescriptions filled and dispensed in 2013. Outpatient medications accounted for 16.6% of all Medicaid expenditures. The 10 most expensive medication classes accounted for 63.9% of all medication expenditures. Stimulants (amphetamine-type) accounted for both the highest proportion of expenditures (20.6%) and days of medication use (1...
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Papers by Ruth Lopert