The PICOTS-ComTeC Framework for Defining Digital Health Interventions: An ISPOR Special Interest Group Report.

OBJECTIVES: Digital health definitions are abundant, but often lack clarity and precision. We aimed to develop a minimum information framework to define patient-facing digital health interventions (DHIs) for outcomes research. METHODS: Definitions of digital-health-related terms (DHTs) were systematically reviewed, followed by a content analysis using frameworks, including PICOTS (population, intervention, comparator, outcome, timing, and setting), Shannon-Weaver Model of Communication, Agency for Healthcare Research and Quality Measures, and the World Health Organization's Classification of Digital Health Interventions. Subsequently, we conducted an online Delphi study to establish a minimum information framework, which was pilot tested by 5 experts using hypothetical examples. RESULTS: After screening 2610 records and 545 full-text articles, we identified 101 unique definitions of 67 secondary DHTs in 76 articles, resulting in 95 different patterns of concepts among the definitions. World Health Organization system (84.5%), message (75.7%), intervention (58.3%), and technology (52.4%) were the most frequently covered concepts. For the Delphi survey, we invited 47 members of the ISPOR Digital Health Special Interest Group, 18 of whom became the Delphi panel. The first, second, and third survey rounds were completed by 18, 11, and 10 respondents, respectively. After consolidating results, the PICOTS-ComTeC acronym emerged, involving 9 domains (population, intervention, comparator, outcome, timing, setting, communication, technology, and context) and 32 optional subcategories. CONCLUSIONS: Patient-facing DHIs can be specified using PICOTS-ComTeC that facilitates identification of appropriate interventions and comparators for a given decision. PICOTS-ComTeC is a flexible and versatile tool, intended to assist authors in designing and reporting primary studies and evidence syntheses, yielding actionable results for clinicians and other decision makers.

Ethical and legal considerations in social media research for health technology assessment: conclusions from a scoping review.

OBJECTIVES: The objective was to identify and describe the published guidance and current academic discourse of ethical issues and standards related to the use of Social Media Research for generating patient insights for the use by health technology assessment (HTA) or health policy decisions. METHODS: A scoping review of the literature was conducted in PubMed and Embase and identified 935 potential references published between January 2017 and June 2021. After title and abstract screening by three reviewers, 40 publications were included, the relevant information was extracted and data were collected in a mind map, which was then used to structure the output of the review. RESULTS: Social Media Research may reveal new insights of relevance to HTA or health policies into patient needs, patient experiences, or patient behaviors. However, the research approaches, methods, data use, interpretation, and communication may expose those who post the data in social media channels to risks and potential harms relating to privacy, anonymity/confidentiality, authenticity, context, and rapidly changing technologies. CONCLUSIONS: An actively engaged approach to ensuring ethical innocuousness is recommended that carefully follows best practices throughout planning, conduct, and communication of the research. Throughout the process and as a follow-up, there should be a discourse with the ethical experts to maximally protect the current and future users of social media, to support their trust in the research, and to advance the knowledge in parallel to the advancement of the media themselves, the technologies, and the research tools.

Using social media research in health technology assessment: stakeholder perspectives and scoping review.

OBJECTIVES: The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA. METHODS: The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA. RESULTS: The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making. CONCLUSIONS: The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

How Useful Are Digital Health Terms for Outcomes Research? An ISPOR Special Interest Group Report.

OBJECTIVES: This study aimed to review definitions of digital health and understand their relevance for health outcomes research. Four umbrella terms (digital health, electronic health, mobile health, and telehealth/telemedicine) were summarized in this article. METHODS: PubMed/MEDLINE, Embase, Cochrane Library, and EconLit were searched from January 2015 to May 2020 for systematic reviews containing key Medical Subject Headings terms for digital health (n = 38) and synonyms of "definition." Independent pairs of reviewers performed each stage of the review, with reconciliation by a third reviewer if required. A single reviewer consolidated each definition for consistency. We performed text analysis via word clouds and computed document frequency-and inverse corpus frequency scores. RESULTS: The search retrieved 2610 records with 545 articles (20.9%) taken forward for full-text review. Of these, 39.3% (214 of 545) were eligible for data extraction, of which 134 full-text articles were retained for this analysis containing 142 unique definitions of umbrella terms (digital health [n = 4], electronic health [n = 36], mobile health [n = 50], and telehealth/telemedicine [n = 52]). Seminal definitions exist but have increasingly been adapted over time and new definitions were created. Nevertheless, the most characteristic words extracted from the definitions via the text analyses still showed considerable overlap between the 4 umbrella terms. CONCLUSIONS: To focus evidence summaries for outcomes research purposes, umbrella terms should be accompanied by Medical Subject Headings terms reflecting population, intervention, comparator, outcome, timing, and setting. Ultimately a functional classification system is needed to create standardized terminology for digital health interventions denoting the domains of patient-level effects and outcomes.

Making patient blood management the new norm(al) as experienced by implementors in diverse countries.

BACKGROUND: Patient blood management (PBM) describes a set of evidence-based practices to optimize medical and surgical patient outcomes by clinically managing and preserving a patient's own blood. This concepts aims to detect and treat anemia, minimize the risk for blood loss and the need for blood replacement for each patient through a coordinated multidisciplinary care process. In combination with blood loss, anemia is the main driver for transfusion and all three are independent risk factors for adverse outcomes including morbidity and mortality. Evidence demonstrates that PBM significantly improves outcomes and safety while reducing cost by macroeconomic magnitudes. Despite its huge potential to improve healthcare systems, PBM is not yet adopted broadly. The aim of this study is to analyze the collective experiences of a diverse group of PBM implementors across countries reflecting different healthcare contexts and to use these experiences to develop a guidance for initiating and orchestrating PBM implementation for stakeholders from diverse professional backgrounds. METHODS: Semi-structured interviews were conducted with 1-4 PBM implementors from 12 countries in Asia, Latin America, Australia, Central and Eastern Europe, the Middle East, and Africa. Responses reflecting the drivers, barriers, measures, and stakeholders regarding the implementation of PBM were summarized per country and underwent qualitative content analysis. Clustering the resulting implementation measures by levels of intervention for PBM implementation informed a PBM implementation framework. RESULTS: A set of PBM implementation measures were extracted from the interviews with the implementors. Most of these measures relate to one of six levels of implementation including government, healthcare providers, funding, research, training/education, and patients/public. Essential cross-level measures are multi-stakeholder communication and collaboration. CONCLUSION: The implementation matrix resulting from this research helps to decompose the complexity of PBM implementation into concrete measures on each implementation level. It provides guidance for diverse stakeholders to design, initiate and develop strategies and plans to make PBM a national standard of care, thus closing current practice gaps and matching this unmet public health need.

Case studies for implementing MCDA for tender and purchasing decisions in hospitals in Indonesia and Thailand.

BACKGROUND: A multi-criteria decision analysis (MCDA) approach has been suggested for helping purchasers in low- and middle-income countries in an evidence-based assessment of multi-source pharmaceuticals to mitigate potential adverse consequences of price-based decisions on patient access to effective medicines. Six workshops for developing MCDA-instruments for purchasing were conducted in Indonesia, Kazakhstan, Thailand, and Kuwait in 2017-2020. In Indonesia and Thailand, two pilot-initiatives aimed to implement the instruments for hospital drug purchasing decisions. OBJECTIVE: By analysing and comparing the experiences and progress from the MCDA-workshops and the two case-examples for hospital implementation in Indonesia and Thailand, we aim to gain insights, which will support future implementation. METHODS: The selection of criteria and their average weight were compared quantitatively across the MCDA-instruments developed in all four countries and settings. Implementation experiences from two case-examples were studied, which included (1) testing the instrument across a variety of drugs in seven hospitals in Thailand and (2) implementation in one specialty hospital in Indonesia. Semi-structured interviews were conducted via web-conferences with four diverse stakeholders in the pilot implementation projects in Thailand and Indonesia. The open responses were evaluated through qualitative content analysis and synthesis using grounded theory coding. RESULTS: Drivers for implementation were making 'better' decisions, achieving transparency and a rational selection process, reducing drug shortages, and assuring consistent quality. Challenges were seen on the technical level (definition or of criteria, scoring methods, access to data) or change-related challenges (resistance, perception of increased workload, lack of competencies or capabilities, lack of resources). The comparison of the MCDA instruments revealed high similarity, but also clear need for local adaptations in each specific case. CONCLUSION: A set a of measures targeting challenges related to utility, methodology, data requirements, capacity building and training as well as the broader societal impact can help to overcome challenges in the implementation. Careful planning of implementation and organizational change is recommended for ensuring commitment and fit to local context and culture. Designing a collaborative change program for each application of MCDA-based purchasing will enable healthcare stakeholders to maximally benefit in terms of quality and effectiveness of care and access for patients.

Pilot approach to analyzing patient and citizen involvement in health technology assessment in four diverse low- and middle-income countries.

BACKGROUND: In low- and middle-income countries (LMICs) striving to achieve universal health coverage, the involvement of different stakeholders in formal or informal ways in health technology assessment (HTA) must be culturally and socially relevant and acceptable. Challenges may be different from those seen in high-income countries. In this article, we aimed to pilot a questionnaire for uncovering the context-related aspects of patient and citizen involvement (PCI) in LMICs, collecting experiences encountered with PCI, and identifying opportunities for patients and citizens toward contributing to local decision- and policy-making processes related to health technologies. METHODS: Through a collaborative, international multi-stakeholder initiative, a questionnaire was developed for describing each LMIC's healthcare system context and the emergence of opportunities for PCI relating to HTA. The questionnaire was piloted in the first set of countries (Brazil, Indonesia, Nigeria, and South Africa). RESULTS: The questionnaire was successfully applied across four diverse LMICs, which are at different stages of using HTA to inform decision making. Only in Brazil, formal ways of PCI have been defined. In the other countries, there is informal influence that is contingent upon the engagement level of patient and citizen advocacy groups (PCAGs), usually strongest in areas such as HIV/AIDS, TB, oncology, or rare diseases. CONCLUSIONS: The questionnaire can be used to analyze the options for patients and citizens to participate in HTA or healthcare decision making. It will be rolled out to more LMICs to describe the requirements and opportunities for PCI in the context of LMICs and to identify possible routes and methodologies for devising a more systematic and formalized PCI in LMICs.

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group.

Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective. For an efficient, timely and practical precision medicine value assessment framework, it will be important to address these multiple perspectives through building consensus among the stakeholders for robust procedures and measures of value aspects, including performance of precision mechanism; aligned reimbursement processes of precision mechanism and subsequent treatment; transparent expectations for evidence requirements and study designs adequately matched to the intended use of the precision mechanism and to the smaller target patient populations; recognizing the potential range of value-generation such as ruling-in and ruling-out decisions.

Barriers and facilitators of exploiting the potential of value-added medicines.

INTRODUCTION: Pharmaceutical research and development (R&D) is costly and only a minority of patients can access innovative medicines due to affordability constraints. Value-added medicines (VAMs) can offer potential benefits at significantly lower R&D costs. AREAS COVERED: VAMs may address different health care needs and problems, including off-label use of medicines, poor patient adherence, problems related to polypharmacy, need for home and/or personalized health care services. However, several barriers prevent societies from maximizing the benefits of incremental innovation related to VAMs. Generic manufacturers have limited budget and experience to demonstrate the value of new VAMs. Current market exclusivity options do not efficiently exclude freeridership and do not guarantee a return on investment for VAM innovators. Value propositions of VAMs are limitedly consistent with current HTA frameworks, consequently, incremental innovation is not acknowledged, nor rewarded with differential pricing by payers. Moreover, VAMs are often perceived solely as generic medicines by prescribers. EXPERT OPINION: Current practices may need to be reconsidered to exploit the full societal benefit of VAMs, including more efficient policies to guarantee market exclusivity for incremental innovation, acknowledgment of a fair price premium based on a specific value framework and the acceptance of low-cost evidence generation methods.

External Reference Pricing for Pharmaceuticals-A Survey and Literature Review to Describe Best Practices for Countries With Expanding Healthcare Coverage.

BACKGROUND: Countries with expanding healthcare coverage (CEHCs) increasingly use external reference pricing (ERP) for pharmaceuticals. The ERP policies must aim to optimize efficiency, minimize disturbances, and maximize access to effective therapies for all patients. OBJECTIVE: This research aims to deduce best practices for prudent ERP regulations from past experiences and currently applied policies and to guide policymakers in CEHCs in implementing robust ERP policies. METHODS: The literature was reviewed for methods and effects of ERP for pharmaceuticals. Pharmaceutical pricing experts from Asia, the Middle East, Russia, and South Africa were surveyed for current approaches to ERP in their respective countries. RESULTS: Key determinants of ERP relate to scope, number, and choice of reference countries; price definitions; computation rules; frequency; and stringency of applying ERP. The scarce evidence shows that ERP seems to lead to narrower price windows with the risk of reducing prices in high-price countries and raising prices in low-price countries. Moreover, launch delays and indirect price effects are often observed. The ERP policies in CEHCs are often applied in isolation, not always in a consistent and transparent manner, neglecting its indirect effects. CONCLUSION: Policymakers should consider a set of requirements when introducing ERP, including clear definitions and decision criteria in full transparency. External reference pricing should inform and serve as a benchmark for pricing decisions, rather than being used as the sole pricing mechanism. External reference pricing is primarily a tool to support decisions regarding on-patent pharmaceuticals, and for off-patent products, competition may prove more effective in reducing prices than ERP.

Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs.

Although patient preferences have been studied broadly for marketed products or around the time of submission to authorities and launch, patient preference studies have rarely been used during the early drug development phases. In this paper, we formulate three hypotheses supporting the use of patient preference studies in early product development: (1) integration of the patient perspective into the development process from phase 1 onwards will result in healthcare solutions with outcomes that best address patients' needs; (2) a structured process to build patient-based evidence involving partnerships between patients and other key stakeholders will improve alignment of development activities with the needs of patients; (3) quantitative patient preference research built on robust qualitative insights is necessary to strengthen development decisions in the interests of patients. To illustrate such a structured process, we describe qualitative insights research (social media analysis and online bulletin boards) and quantitative patient preference studies in dry eye disease and non-alcoholic steatohepatitis conducted during early product development by a pharmaceutical company to generate patient-based evidence. The outputs from such early patient preference studies are being used to inform patient reported outcome strategies, clinical development strategies, product design and delivery features, and form the basis for early dialog with regulators, health technology assessment (HTA) bodies and payers to ensure focus and alignment on patient-relevant endpoints. Furthermore, to discuss and theoretically substantiate our hypotheses, we review how different groups and organizations are working to embrace fully the patient perspective in product development and healthcare decision-making. The hypotheses are commensurate with the general trend toward patient-centered healthcare and the activities initiated by regulators, HTA agencies, and patient organizations. We advocate that all healthcare players should actively contribute to aligning on best practices concerning choice of methodologies and engage in multi-stakeholder dialog along the entire product development chain, to realize health technologies that best meet the needs of patients.

Stakeholder driven development of a multi-criteria decision analysis tool for purchasing off-patent pharmaceuticals in Kuwait.

BACKGROUND: In Kuwait, the government is planning to improve the specifications for purchase of medicine and to improve the tendering system intending to slow the growth of the expenditure for medicine and to improve the sustainability of the healthcare system. Multiple Criteria Decision Analysis (MCDA) is a method which can help to assess multiple and sometimes conflicting criteria in the evaluation of the available alternatives. The objective of this initiative was to develop collaboratively a MCDA tool which is locally relevant, and which could be used to improve the use of performance indicators in the purchasing of off-patent pharmaceuticals. METHODS: Nineteen leading experts representing a broad range of pharmaceutical policy stakeholders elaborated a locally adapted MCDA format by following a 7-step process for criteria selection, scoring, ranking and weighting. RESULTS: The most important criterion was the price measured as savings versus the originator product with a weight of 35% in the final decision and a full score with a 60% price reduction. In addition, eight criteria were considered important for assessing the product performance in the Kuwaiti healthcare system: 'equivalence with the reference product' (weight of 16.2%), 'stability and drug formulation' (13.5%), 'quality assurance' (11.2%), 'reliability of drug supply' (8.8%), 'macroeconomic benefit' (5.5%), 'real world outcomes (clinical and economic)' (4.2%), 'pharmacovigilance' (3.3%), and 'added value services related to the product' (2.3%). CONCLUSIONS: A MCDA model was successfully adapted to the Kuwait decision context by a group of Kuwaiti pharmacists from a broad range of institutions. The participants agreed with the approach and considered it suitable to improve the transparency and consistency of decision making for off-patent pharmaceuticals in Kuwait. A pilot implementation project was proposed.

Shaping Pharmaceutical Tenders for Effectiveness and Sustainability in Countries with Expanding Healthcare Coverage.

Policy makers in countries, aiming to build and expand their healthcare systems and coverage, need effective procedures to support the most efficient use of limited financial resources. Tendering is commonly deployed to minimize and fix the purchasing price for the contract duration, especially for off-patent pharmaceuticals. While tenders can reduce acquisition costs, they may also expose the healthcare systems to risks including drug shortages, quality trade-offs, and ultimately, compromised patient health outcomes. Careful planning is therefore required. The effectiveness and impact of tendering were examined in different healthcare settings to establish good tender practices and to develop guidance for tender stakeholders in countries with expanding healthcare coverage for the effective conduct. The literature was reviewed for tender practices and outcomes in all countries, and tender experts from one multi-national pharmaceutical company in 17 countries with expanding healthcare coverage were surveyed on current tender practices. Tendering is a common practice for multisource pharmaceuticals in most countries worldwide. However, countries with expanding healthcare coverage specifically are vulnerable to the risks of defective tendering practices. Risk factors include non-transparent tender practices, a lack of consistency, unclear tender award criteria, a focus on lowest price only, single-winner tendering, and generally, a lack of impact monitoring. If well planned, managed, and conducted, tenders can be advantageous. Countries with expanding healthcare coverage should approach tenders strategically to achieve the desired improvements in healthcare. The good tender practices derived from this study may guide policy makers and purchasers in countries with expanding healthcare coverage on how to expand access to healthcare at an affordable cost. These include the use of multiple selection criteria and performance monitoring. Plain Language Summary Decision makers in countries aiming to expand their healthcare systems must best use the limited money available for healthcare. Tendering is commonly deployed for pharmaceuticals produced by multiple manufacturers (so-called multisource pharmaceuticals), to choose the product with the lowest price. Through tenders, purchasers request offers from suppliers for the needed products.The ultimate purpose of our research was to develop a guidance on robust tender processes. Therefore, we reviewed the literature to examine the effectiveness and impact of current tendering practices. In addition, we conducted a survey among tender experts from one pharmaceutical company in 17 countries with expanding healthcare coverage.In both the survey and the literature review, we confirmed that worldwide, tendering is a common practice for multisource pharmaceuticals. However, defective tendering practices may increase the vulnerability for some risks including abuse due to intransparent processes, lack of consistency, unclear tender award criteria, a focus on lowest price only, single winner tendering, and generally, a lack of impact monitoring after the end of the tender process.Hence, tenders must be well planned, managed, and conducted to be advantageous. Countries with defined and transparent tender frameworks and processes will be better equipped to achieve the desired improvements in the healthcare systems. 'Good tender practices' include the clear definition of requirements to be used as selection criteria in addition to acquisition costs, and for monitoring of the tender success. 'Good tender practices' may help to manage cost and improve healthcare at the same time.

Health economic assessment of Gd-EOB-DTPA MRI versus ECCM-MRI and multi-detector CT for diagnosis of hepatocellular carcinoma in China.

Limited data exists in China on the comparative cost of gadolinium ethoxybenzyl diethylenetriamine magnetic resonance imaging (Gd-EOB-DTPA-MRI) with other imaging techniques. This study compared the total cost of Gd-EOB-DTPA-MRI with multidetector computed tomography (MDCT) and extracellular contrast media-enhanced MRI (ECCM-MRI) as initial imaging procedures in patients with suspected hepatocellular carcinoma (HCC). We developed a decision-tree model on the basis of the Chinese clinical guidelines for HCC, which was validated by clinical experts from China. The model compared the diagnostic accuracy and costs of alternative initial imaging procedures. Compared with MDCT and ECCM-MRI, Gd-EOB-DTPA-MRI imaging was associated with higher rates of diagnostic accuracy, i.e. higher proportions of true positives (TP) and true negatives (TN) with lower false positives (FP). Total diagnosis and treatment cost per patient after the initial Gd-EOB-DTPA-MRI evaluation was similar to MDCT (¥30,360 vs. ¥30,803) and lower than that reported with ECCM-MRI (¥30,360 vs. ¥31,465). Lower treatment cost after initial Gd-EOB-DTPA-MRI was driven by reduced utilization of confirmatory diagnostic procedures and unnecessary treatments. The findings reported that Gd-EOB-DTPA-MRI offered higher diagnostic accuracy compared with MDCT and ECCM-MRI at a comparable cost, which indicates Gd-EOB-DTPA-MRI could be the preferred initial imaging procedure for the diagnosis of HCC in China.

EVALUATION OF PATIENT AND PUBLIC INVOLVEMENT INITIATIVES IN HEALTH TECHNOLOGY ASSESSMENT: A SURVEY OF INTERNATIONAL AGENCIES.

OBJECTIVES: Although there is increased awareness of patient and public involvement (PPI) among health technology assessment (HTA) organizations, evaluations of PPI initiatives are relatively scarce. Our objective as members of Health Technology Assessment International's (HTAi's) Patient and Citizen Involvement Group (PCIG) was to advance understanding of the range of evaluation strategies adopted by HTA organizations and their potential usefulness. METHODS: In March 2016, a survey was sent to fifty-four HTA organizations through the International Network of Agencies for Health Technology Assessment (INAHTA) and contacts of members of HTAi's PCIG. Respondents were asked about their organizational structure; how patients and members of the public are involved; whether and how PPI initiatives have been evaluated, and, if so, which facilitators and challenges to evaluation were found and how results were used and disseminated. RESULTS: Fifteen (n = 15) programs from twelve countries responded (response rate 27.8 percent) that involved patients (14/15) and members of the public (10/15) in HTA activities. Seven programs evaluated their PPI activities, including participant satisfaction (5/7), process (5/7) and impact evaluations (4/7). Evaluation results were used to improve PPI activities, identify education and training needs, and direct strategic priorities. Facilitators and challenges revolved around the need for stakeholder buy-in, sufficient resources, senior leadership, and including patients in evaluations. CONCLUSIONS: A small but diverse set of HTA organizations evaluate their PPI activities using a range of strategies that reflect the range of rationales and approaches to PPI in HTA. It will be important for HTA organizations to draw on evaluation theories and methods.

Health economic evaluation of Gd-EOB-DTPA MRI vs ECCM-MRI and multi-detector computed tomography in patients with suspected hepatocellular carcinoma in Thailand and South Korea.

OBJECTIVE: The effectiveness of treatment decisions and economic outcomes of using gadolinium ethoxybenzyl diethylenetriamine pentaacetic acid-enhanced magnetic resonance imaging (Gd-EOB-DTPA-MRI) were compared with extracellular contrast media-enhanced MRI (ECCM-MRI) and multi-detector computed tomography (MDCT) as initial procedures in patients with suspected hepatocellular carcinoma (HCC) in South Korea and Thailand. METHODS: A decision-tree model simulated the clinical pathway for patients with suspected HCC from the first imaging procedure to a confirmed treatment decision. Input data (probabilities and resource consumptions) were estimated and validated by clinical experts. Costs for diagnostic alternatives and related treatment options were derived from published sources, taking into account both payer's and hospital's perspectives. RESULTS: All experts from Korea and Thailand agreed that Gd-EOB-DTPA-MRI yields the highest diagnostic certainty and minimizes the need for additional confirmatory diagnostic procedures in HCC. In Korea, from the payer's perspective, total cost was USD $3087/patient to reach a confirmed treatment decision using Gd-EOB-DTPA-MRI (vs $3205/patient for MDCT and $3403/patient for ECCM-MRI). From the hospital's perspective, Gd-EOB-DTPA-MRI incurred the lowest cost ($2289/patient vs $2320/patient and $2528/patient, respectively). In Thailand, Gd-EOB-DTPA-MRI was the least costly alternative for the payer ($702/patient vs $931/patient for MDCT and $873/patient for ECCM-MRI). From the hospital's perspective, costs were $1106/patient, $1178/patient, and $1087/patient for Gd-EOB-DTPA-MRI, MDCT, and ECCM-MRI, respectively. CONCLUSIONS: Gd-EOB-DTPA-MRI as an initial imaging procedure in patients with suspected HCC provides better diagnostic certainty and relevant statutory health insurance cost savings in Thailand and Korea, compared with ECCM-MRI and MDCT.

Need for multicriteria evaluation of generic drug policies.

Policymakers tend to focus on improving patented drug policies because they are under pressure from patients, physicians, and manufacturers to increase access to novel therapies. The success of pharmaceutical innovation over the last few decades has led to the availability of many off-patent drugs to treat disease areas with the greatest public health need. Therefore, the success of public health programs in improving the health status of the total population is highly dependent on the efficiency of generic drug policies. The objective of this article was to explore factors influencing the true efficiency of generic prescription drug policies in supporting public health initiatives in the developed world. Health care decision makers often assess the efficiency of generic drug policies by the level of price erosion and market share of generics. Drug quality, bioequivalence, in some cases drug formulations, supply reliability, medical adherence and persistence, health outcomes, and nondrug costs, however, are also attributes of success for generic drug policies. Further methodological research is needed to measure and improve the efficiency of generic drug policies. This also requires extension of the evidence base of the impact of generic drugs, partly based on real-world evidence. Multicriteria decision analysis may assist policymakers and researchers to evaluate the true value of generic drugs.

Challenges in the development and reimbursement of personalized medicine-payer and manufacturer perspectives and implications for health economics and outcomes research: a report of the ISPOR personalized medicine special interest group.

BACKGROUND: Personalized medicine technologies can improve individual health by delivering the right dose of the right drug to the right patient at the right time but create challenges in deciding which technologies offer sufficient value to justify widespread diffusion. Personalized medicine technologies, however, do not neatly fit into existing health technology assessment and reimbursement processes. OBJECTIVES: In this article, the Personalized Medicine Special Interest Group of the International Society for Pharmacoeconomics and Outcomes Research evaluated key development and reimbursement considerations from the payer and manufacturer perspectives. METHODS: Five key areas in which health economics and outcomes research best practices could be developed to improve value assessment, reimbursement, and patient access decisions for personalized medicine have been identified. RESULTS: These areas are as follows: 1 research prioritization and early value assessment, 2 best practices for clinical evidence development, 3 best practices for health economic assessment, 4 addressing health technology assessment challenges, and 5 new incentive and reimbursement approaches for personalized medicine. CONCLUSIONS: Key gaps in health economics and outcomes research best practices, decision standards, and value assessment processes are also discussed, along with next steps for evolving health economics and outcomes research practices in personalized medicine.

Implementing CER: what will it take?

BACKGROUND: Comparative effectiveness research (CER) is undeniably changing how drugs are developed, launched, priced, and reimbursed in the United States. But most organizations are still evaluating what CER can do for them and how and when they can utilize the data. A roundtable of stakeholders, including formulary decision makers, evaluated CER's possible effects on managed care organizations (MCOs) and what it may take to fully integrate CER into decision making. OBJECTIVES: To examine the role of CER in current formulary decision making, compare CER to modeling, discuss ways CER may be used in the future, and describe CER funding sources. SUMMARY: While decision makers from different types of organizations, such as pharmacy benefit management (PBM) companies and MCOs, may have varying definitions and expectations of CER, most thought leaders from a roundtable of stakeholders, including formulary decision makers, see value in CER's ability to enhance their formulary decision making. Formulary decision makers may be able to use CER to better inform their coverage decisions in areas such as benefit design, contracting, conditional reimbursement, pay for performance, and other alternative pricing arrangements. Real-world CER will require improvement in the health information technology infrastructure to better capture value-related information. The federal government is viewed as a key driver and funding source behind CER, especially for infrastructure and methods development, while industry will adapt the clinical development and create increasing CER evidence. CER then needs to be applied to determining value (or cost efficacy). CONCLUSIONS: It is expected that CER will continue to grow as a valuable component of formulary decision making. Future integration of CER into formulary decision making will require federal government and academic leadership, improvements in the health information technology infrastructure, ongoing funding, and improved and more consistent methodologies.

Current and Future Use of HEOR Data in Healthcare Decision-Making in the United States and in Emerging Markets.

BACKGROUND: Health economics and outcomes research (HEOR) is a growing field that provides important information for making healthcare coverage and access decisions. However, there is no standard process for incorporating HEOR into the decision-making process, and the current use of HEOR by healthcare payers remains unknown. OBJECTIVES: To examine how HEOR data are being used by healthcare payers, including managed care organizations today, and how the use of such data is expected to change in the future in relation to access and reimbursement decision-making. METHODS: The Managed Care Survey (MCS) and the Pharmacy & Therapeutics (P&T) Committee Survey (PTS) were distributed to decision makers in the United States. A total of 72 managed care decision makers responded to the MCS and 30 P&T Committee members responded to the PTS from US healthcare organizations that cover from tens of thousands to millions of lives. The goal of these surveys was to understand the current use of HEOR data, perceived barriers and limitations in the use of HEOR, and the expectations for future use, and how these and other factors affect formulary decisions. An international perspective was gained by modifying the MCS based on feedback received at a European conference, and a pilot version was given to individuals in emerging markets across Asia, Latin America, and the Middle East and Africa. RESULTS: The majority of US respondents to the MCS (74%; N = 53) and to the PTS (77%; N = 23) indicate that HEOR is currently being used in their decision-making process; but the majority of respondents to the MCS (66%; N = 48) also state that quality assessment is limited (quality assessment was not addressed in the PTS). In addition, the majority of respondents to the MCS (82%; N = 59) expect the use of HEOR to increase in the future. Safety and efficacy were reported in the PTS to be the most important factors in the P&T Committee decision-making process, followed by head-to-head comparisons, and cost. The current use of HEOR in Asia, Latin America, and the Middle East and Africa varied widely across respondents. CONCLUSION: This study provides an important benchmark of HEOR use in the United States before the implementation of healthcare reform. Between the years 2010 and 2011, HEOR data were used to varying extents across global regions, but their use in the future is likely to increase in relation to access and reimbursement decisions.