A Sleeping Giant: Community Pharmacy's Potential Is Unrivaled.

DISCLOSURES: No funding was received for the writing of this commentary. The author has nothing to disclose.

What Was, Is No More: Community Pharmacy Economics.

DISCLOSURES: No funding supported the writing of this article. The author is an employee of AmerisourceBergen and leads its Good Neighbor Pharmacy program, which supports independent community pharmacy customers.

Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy.

In 2016, The Professional Society for Health Economics and Outcomes Research (ISPOR) formed a special task force (STF) to review approaches and methods to support the definition and use of high-quality U.S. value frameworks. As the leadership group of that initiative, we present our perspective, focusing on implications for the managed care pharmacy community. Our reflections are organized by 9 key observations and conclude with a summary recommendation. We begin by emphasizing the importance of distinguishing among "perspectives" and "decision contexts." Possible perspectives include patient, payer, provider, health care sector, and societal. Decision contexts range from formulary inclusion to guideline development to clinical shared decision making, and multiple perspectives can be taken on each of these decisions. The STF focused on value in the context of including a new medicine in a formulary and, thus, health plan, using a health economics approach that compares marginal benefit (gross value) and marginal (opportunity) cost, yielding the net value. Health care is unique compared with other markets. While economists often use market purchases as indicators of value, they also recognize that this does not work well in health care, since most patent-protected drugs are covered by insurance. To assess the likely health and economic impact, health economists often employ cost-effectiveness analysis, using the quality-adjusted life-year (QALY), a metric that combines mortality and morbidity into a single preference-based index. We strongly endorse the STF's recommendation that payers should use the cost-per-QALY metric as a starting point. However, like the STF, and many of those stakeholders who provided input, we recognize that this metric has some limitations in theory and in practice. Nonetheless, the cost-per-QALY metric is a pragmatic tool that can be augmented to address some of its limitations by integrating other elements of value, particularly those related to uncertainty, such as financial risk protection, health risk protection, the value of hope, real option value, and the value of knowing. The resulting adjusted ratio can be compared with a willingness-to-pay threshold or combined in a measure of net monetary benefit. Alternatively, the array of elements can be valued using multi-criteria decision analysis. We end with the key recommendation that further development and testing of these promising approaches is needed to improve the deliberative process of health technology assessment. DISCLOSURES: No outside funding supported the writing of this article. The authors are leaders of the ISPOR Special Task Force on U.S. Value Frameworks. Willke is employed by ISPOR. Garrison and Neumann have nothing to disclose. The opinions expressed in this article should be considered as belonging only to the authors.

Navigating joint HTA, procurement, and fair pricing: evidence-based insights and practical recommendations - A meeting report from ISPOR regional conference in Warsaw, 2019.

Introduction: The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) organized its first Central Eastern European regional meeting in 2019 in Warsaw, Poland. Area covered: The scientific program of the two-day conference covered a broad range of topics presented from the perspective of the region. Specifically, the focus was on cross-country collaboration within different steps of health technology assessment (HTA) and the need for local HTA adaptations in decision-making. Expert commentary: Attended by approximately 200 delegates from many countries and by several high level ISPOR leaders, the conference provided a valuable opportunity to exchange knowledge and strengthen the scientific network among experts from different stakeholder groups on issues specific to the region.

Incentives and disincentives to drug innovation: evidence from recent literature.

Aim: Drug innovation is strongly driven by economic incentives. How these incentives work in determining or changing the level of activity of innovators and the direction of their innovation remains understudied. We seek to address these issues in reviewing recent literature on drug innovation, which offers one major unifying theme of pharmacoeconomic scholarship presented at the 2019 AEA-ASSA annual convention. Methods: Drawn from three AEA-ASSA convention panel sessions, papers were reviewed for newly charted research terrains and new research trajectories, and their theoretical and practical implications on efficiency, effectiveness, and value in the production and utilization of pharmaceutical products. Results: While high and continuously rising drug prices are typically claimed as the price of scientific innovation, the reviewed literature finds that this link only partially accounts for the problem. High risk aversion owing to information asymmetries and vastly intractable uncertainties is prevalent among innovating firms. Predatory business models abound. Reverse predatory strategies also exist to maintain product exclusivity without much added clinical benefits, and to constrain generic competition. CEO compensation practices contribute to rising drug prices. Finally, the US government's hands-off policy on drug list prices leave the forces of supply and demand to allocate them and reward innovation (at times perversely), even as the government extensively regulates or over-regulates practically every other aspect of innovation. Conclusions: Price-elasticity of demand is critical in drug innovation. The drug value chain is price-sensitive to the balance of incentives and disincentives to innovation. American health policy should consider charting a middle course that introduces some form of regulatory price control, while stimulating and sustaining the benefits of market competition. That should incentivize stakeholders to take into account both resources and value for money in making decisions based on best-quality, clinical-economic evidence.

Assessing Data Sources for Medicine Price Studies.

OBJECTIVES: There is no established methodology to assess the feasibility of medicine price data sources. Against this backdrop, a framework to guide the selection of most appropriate price data sources for pharmacoeconomic research has been developed. METHODS: A targeted literature review was carried out. Dimensions discussed in literature as relevant for medicine price comparisons and practical experience of the authors in medicine price studies informed the conceptional work of the framework development. A draft version of the framework was reviewed by peer pricing experts. The feasibility of the framework was tested in case studies. RESULTS: According to the developed framework (called Re-ADAPT), a medicine price data source should meet the following criteria: reliability and sustainability; accessibility at a cost that users can afford; provision of medicine price information at the date(s) required; information for the defined geographic area, or at least in a representative way; coverage of the pharmaceuticals and at the price type(s) required. Easy handling and provision of additional information were defined as supportive assets of candidate data sources (secondary criteria). The case studies confirmed the feasibility of the Re-ADAPT framework. In some cases, however, it can be difficult to disentangle assessment criteria (particularly geographic area, scope of pharmaceuticals and price types) for separate consideration, given their interlinkage. CONCLUSIONS: While selection of the most appropriate data sources will remain a challenge, the Re-ADAPT framework aims to provide practical guidance and thus contribute to a more careful, balanced, and evidence-based selection of data sources for medicine price studies.

Crowdfunding pharmacy- and medication-related products: How successful is it?

OBJECTIVE: To determine whether crowdfunding of pharmacy-related products through popular online platforms is a viable means to attain funding and what factors influence success. METHODS: Kickstarter and Indiegogo were searched for projects related to pharmacy using select key words. Projects were included for analysis if they were a device or system relevant to pharmacy care and excluded if found to be nonrelevant to medication management purposes or were of an artistic nature. Projects were assessed for their success in reaching their primary funding goals and also whether they were still in business following completion of their crowdfunding phase. RESULTS: Subsequent to the application of the inclusion and exclusion criteria to the dataset, 40 projects were identified, of which 13 reached their desired crowdfunding funding amounts. The most commonly created crowdfunded projects were those involving medication adherence or storage tools. Anecdotal evidence points to media attention leading to continued success beyond the initial crowdfunding phase of the business. The presence of a medical professional on the project team or the inclusion of a product demonstration did not lead to a different rate of success. CONCLUSION: The crowdfunding of pharmacy care-related products appear to have a low success rate, although Indiegogo might offer a higher success rate compared with Kickstarter in this niche product area. The products' ability to garner media attention seems to be a primary driver in the business surviving past the crowdfunding stage and becoming a lasting success.

[Consensus document: a model of integrated management of patients with psycomotor agitation]. / Documento di consenso: modello di gestione integrata del paziente con agitazione psicomotoria.

Psychomotor agitation (PMA) is a heterogeneous clinical syndrome associated with a widenumber of pathological conditions. The currently available recommendations and guidelines on PMA correct assessment and management are significantly dishomogeneous and suffer from a lack of standardization, especially regarding pharmacological interventions. Based on this deficiency, and on multidisciplinary nature of PMA, that includes factors shared by different health professionals other than pharmacoeconomic and risk management aspects, we started a project aimed to elaborate a shared model of integrated management for PMA patients. The model, developed by a scientific board and a multidisciplinary panel using the consensus Delphi-RAND method, aims to give indications of good clinical practice for the management of these patients. The present document reports the results of this consensus process, whose main principles are the centrality of the patient, as an active and collaborating subject, the importance of prompt and not coercive interventions able to block the escalation to violence and to allow a correct diagnostic and therapeutic workup, the appropriate use of pharmacological interventions based on the severity of symptoms and the importance of an integrated and harmonized approach by the different professionals involved in PMA management.

Reimbursement Decisions for Pharmaceuticals in Sweden: The Impact of Disease Severity and Cost Effectiveness.

OBJECTIVE: The Swedish Dental and Pharmaceutical Benefits Agency (TLV) is the government body responsible for deciding whether outpatient drugs are to be included in the pharmaceutical benefits scheme. This paper analyzes the impact of cost effectiveness and severity of disease on reimbursement decisions for new pharmaceuticals. METHODS: Data has been extracted from all decisions made by the TLV between 2005 and 2011. Cost effectiveness is measured as the cost per quality-adjusted life-year (QALY) gained, whereas disease severity is a binary variable (severe-not severe). In total, the dataset consists of 102 decisions, with 86 approved and 16 declined reimbursements. RESULTS: The lowest cost per QALY of declined reimbursements is Swedish kronor (SEK) 700,000 (€ 79,100), while the highest cost per QALY of approved reimbursements is SEK1,220,000 (€ 135,600). At a cost per QALY of SEK702,000 Swedish kronor (non-severe diseases) and SEK988,000 (severe diseases), the likelihood of approval is estimated to be 50/50 (€ 79,400 and € 111,700). CONCLUSIONS: The TLV places substantial weight on both the cost effectiveness and the severity of disease in reimbursement decisions, and the implied willingness to pay for a QALY is higher than the often cited 'rule of thumb' in Swedish policy debates.

Investigation of comparative effectiveness research in Asia, Europe, and North America.

Comparative effectiveness research (CER) is an important branch of pharmacoeconomics that systematically studies and evaluates the cost-effectiveness of medical interventions. CER plays instrumental roles in guiding government public health policy programs and insurance. Countries throughout the world use different methods of CER to help make medical decisions based on providing optimal therapy at a reduced cost. Expenses to the healthcare system continue to rise, and CER is one-way in which expenses could be curbed in the future by applying cost-effectiveness evidence to clinical decisions. China, India, South Korea, and the United Kingdom are of essential focus because these country's economies and health care expenses continue to expand. The structures and use of CER are diverse throughout these countries, and each is of prime importance. By conducting this thorough comparison of CER in different nations, strategies and organizational setups from different countries can be applied to help guide public health and medical decision-making in order to continue to expand the establishment and role of CER programs. The patient-centered medical home has been created to help reduce costs in the primary care sector and to help improve the effectiveness of therapy. Barriers to CER are also important as many stakeholders need to be able to work together to provide the best CER evidence. The advancement of CER in multiple countries throughout the world provides a possible way of reducing costs to the healthcare system in an age of expanding expenses.

Clinical pharmacologists in pharmacoeconomics: what is their role?

Clinical pharmacologists have three distinct contributions to make in the economic evaluation of new and existing pharmaceutical products: they should play a significant role in promoting the principles of "opportunity costs" in healthcare; they need to have a broad understanding of the methodology of economic evaluation in healthcare; they have a critical role in bringing their specialist knowledge, skills and experience in decision-making. In fulfilling these essential roles clinical pharmacologists may find themselves outside their conventional "comfort zones". Nevertheless, clinical pharmacologists need to rise to the occasion if they are to meet their obligations to patients and to society as a whole.

How should we deal with patient heterogeneity in economic evaluation: a systematic review of national pharmacoeconomic guidelines.

OBJECTIVE: To review and analyze recommendations from national pharmacoeconomic guidelines with regard to acknowledging patient heterogeneity in economic evaluations. METHODS: National pharmacoeconomic guidelines were obtained through the ISPOR Web site. Guidance was extracted by using a developed data extraction sheet. Extracted data were divided into subcategories on the basis of consensus meetings. RESULTS: Of the 26 included guidelines, 20 (77%) advised to identify patient heterogeneity. Most guidelines (77%) provided general methodological advice to acknowledge patient heterogeneity, including justifications for distinguishing subgroups (65%), prespecification of subgroups (42%), or methodology to acknowledge patient heterogeneity (77%). Stratified analysis of cost-effectiveness was most commonly advised (20 guidelines; 77%); however, guidance on the specific application of methods was scarce (9 guidelines; 34%) and generally limited if provided. Guidance to present patient heterogeneity was provided by 15 guidelines (58%), most prominently to describe the definition (31%) and justification (31%) of subgroups. CONCLUSIONS: The majority of national pharmacoeconomic guidelines provide guidance on acknowledging patient heterogeneity in economic evaluations. However, because guidance is mostly not specific, its usefulness is limited. This may reflect that the importance of acknowledging patient heterogeneity is usually recognized while there is a lack of consensus on specific methods to acknowledge patient heterogeneity. We advise the further development of national pharmacoeconomic guidelines to provide specific guidance on the identification of patient heterogeneity, methods to acknowledge it, and presenting the results. We present a checklist that can assist in formulating these recommendations. This could facilitate the systematic and transparent handling of patient heterogeneity in economic evaluations worldwide.

[Risk-sharing schemes: a new paradigm in adopting innovative pharmaceuticals].

In recent years, spending on prescription drugs contributes substantially to the continuous growth in health expenditure in most Western countries. This increase in spending is influenced by both a rise in the use of existing drugs and by the adoption of new and expensive drugs. Risk-sharing agreements between pharmaceutical companies and health insurers have emerged as insurers began to deny reimbursement of expensive innovative treatments with unfavorable cost-effectiveness ratios. This occurred in cases where the expected budgetary impact was too high or when the long-term effectiveness was questionable. Risk sharing agreements serve the interests of both the insurers and the drug manufacturers. Pharmaceutical companies' interests are to dispel the uncertainties encountered by the health insurers white deciding on drug reimbursement. The insurers are interested in these agreements in order to reduce the budgetary risk while allowing their patients access to innovative drugs. Currently, only a few risk sharing agreements have been implemented, and the scientific literature on such schemes is still sparse. Since the health insurers' interest is to develop mechanisms that will contain health costs, without affecting the insured, it appears that this trend will continue to emerge. It is also likely that the adoption of similar mechanisms in the Israeli National List of Health Services updating process, would improve the accuracy of early estimates of the budgetary impact, and the actual use of the new technologies would be close to early estimates. This article reviews the principles of risk-sharing schemes and issues involved in the actual implementation of such agreements.

[Pharmacoeconomics in uro-oncology: the concept of QALYs]. / Pharmakoökonomie in der Uroonkologie : Das "QALY-Konzept"

The QALY (quality-adjusted life year) is often used in pharmacoeconomic evaluations. It combines the two dimensions 'quality of life' and 'life expectancy' into one index. Quality of life is expressed as a utility, corresponding to a value between 0 (death) and 1 (perfect health). Life expectancy is then multiplied by the utility corresponding to the quality of life of the respective life time. Accordingly, 1 QALY corresponds to 1 year in perfect health (1 year multiplied by utility 1) or 2 years with a quality of life reduced by 50% (2 years multiplied by utility 0.5). Results of pharmacoeconomic evaluations are often reported as additional costs in relation to the added value of a new treatment, expressed as cost per additional QALY gained for the patient with the new therapeutic intervention. The main advantage of the QALY concept is its validity for all patients and indications. And the use of benefit measures which are not restricted to a specific indication is most important for resource allocation, i.e. to avoid that varying amounts of money are paid for the same health benefit in different disease areas.

[Evaluation of cancer therapy from the perspective of a statutory health insurance]. / Bewertung onkologischer Therapien aus Sicht einer Kasse.

Increasing life expectancy, the introduction of costly new drugs and contributions from the health fund which do not cover overall costs, all contribute to financial problems for statutory health insurances (SHI) in oncology. Only an evidence-based approach can help to address these problems. In a first step patient-relevant benefits have to be substantiated as a necessary prerequisite for coverage of any treatment by SHIs. For products with no additional benefit compared to established forms of therapy the price will be limited by the established cost-benefit ratio. For products with additional benefits pricing is more difficult. For this situation the Institute for Quality and Efficiency in Healthcare (IQWiG) has developed general methods for the assessment of the relation of benefits to costs. Pricing based on this health economic evaluation is developed using efficiency frontier plots. However, this method is prone to manipulation and needs to be refined. Therapies without comparators, so-called soloists, cannot be priced in this way. New approaches to increase cost efficiency need to be developed in order to ensure the availability of high quality care in the future.