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Although the prevalence of obesity and its related complications are increasing among pediatric patients, appropriate management can prevent chronic disease. This article will present an overview of metabolic syndrome, pediatric metabolic syndrome guidelines, pathophysiology, associated risk factors, and clinical practice implications.

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PURPOSE: Meeting marketplace demands for proving the value of new products requires more data than the industry has routinely produced. These data include evidence from comparative effectiveness research (CER), including randomized, controlled trials; pragmatic clinical trials; observational studies; and meta-analyses. METHODS: We designed and conducted a survey to examine the industry's perceptions on new data requirements regarding CER evidence, the acceptability of postapproval study types, payer-specific issues related to CER, communication of data being generated postapproval, and methods used for facilitating postapproval evidence generation. FINDINGS: CER is being used by payers for most types of postapproval decisions. Randomized, controlled trials were indicated as the most acceptable form of evidence. At the same time, there was support for the utility of other types of studies, such as pragmatic clinical trials and observational studies. Respondents indicated the use of multiple formats for communicating postapproval data with many different stakeholders including regulators, payers, providers, and patients. Risk-sharing agreements with payers were unanimously supported by respondents with regard to certain products with unclear clinical and economic outcomes at launch. In these instances, conditional reimbursement through coverage with evidence development was considered a constructive option. The Food and Drug Administration's initiative called Regulatory Science was considered by the respondents as having the most impact on streamlining the generation of postapproval research-related evidence. IMPLICATIONS: The biopharmaceutical industry is faced with a broad and complex set of challenges related to evidence generation for postapproval decisions by a variety of health care system stakeholders. Uncertainty remains as to how the industry and payers use postapproval studies to guide decision making with regard to pricing and reimbursement status. Correspondingly, there is uncertainty regarding whether the industry's investment in CER will have a positive return on investment in terms of reimbursement and market access.

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OBJECTIVES: To examine health plan compliance with essential drug benefit regulations in California and Massachusetts and at the federal level. STUDY DESIGN: Health plan formulary review and analysis. METHODS: We analyzed formularies from the 3 largest small group plans in California and Massachusetts, including each state's benchmark plan. With respect to both federal and state regulations, for each health plan, we examined whether the drug was covered, the designated patient cost sharing tier of the drug, and which conditions of reimbursement were applied to the drug. RESULTS: Most drugs included in state and federal mandates are covered by both benchmark and non-benchmark plans. However, health plans are not fully compliant with state and federal regulations. Significant differences among plans relate more to cost sharing and conditions of reimbursement, such as prior authorization, step edits, and quantity limits, than to drug coverage. CONCLUSIONS/POLICY IMPLICATIONS: Because health plans in California and Massachusetts are not fully compliant with state and federal mandates, they will have to adjust their formularies to meet minimum requirements. State policy makers need to balance competing aims of comprehensiveness of coverage and drug affordability. They must consider: (1) choice of benchmark plan -choice of a more generous benchmark plan implies less leverage for negotiating lower prices; and (2) breadth of state mandates which, if they exceed federal mandates, must be paid for by the states.

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BACKGROUND: Personalized medicine is gradually emerging as a transformative field. Thus far, seven co-developed drug-diagnostic combinations have been approved and several dozen post-hoc drug-diagnostic combinations (diagnostic approved after the drug). However, barriers remain, particularly with respect to reimbursement. Purpose, methods: This study analyzes barriers facing uptake of drug-diagnostic combinations. We examine Medicare reimbursement in the U.S. of 10 drug-diagnostic combinations on the basis of a formulary review and a survey. FINDINGS: We found that payers reimburse all 10 drugs, but with variable and relatively high patient co-insurance, as well as imposition of formulary restrictions. Payer reimbursement of companion diagnostics is limited and highly variable. In addition, we found that the body of evidence on the clinical- and cost-effectiveness of therapeutics is thin and even less robust for diagnostics. Conclusions, discussion: The high cost of personalized therapeutics and dearth of evidence concerning the comparative clinical effectiveness of drug-diagnostic combinations appear to contribute to high patient cost sharing, imposition of formulary restrictions, and limited and variable reimbursement of companion diagnostics. Our findings point to the need to increase the evidence base supportive of establishing linkage between diagnostic testing and positive health outcomes.

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BACKGROUND: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this article, we examine payer coverage in the United States, England and Wales, and the Netherlands of outpatient orphan drugs approved between 1983 and 2012, as well as the 11 most expensive orphan drugs. METHODS: We collected data from drug regulatory agencies as well as payers and drug evaluation authorities. RESULTS: We found that orphan drugs have more coverage restrictions than non-orphan drugs in all three jurisdictions. From an economic perspective, the fact that a drug is an orphan product or has a high per-unit price per se should not imply a special kind of evaluation by payers, or necessarily the imposition of more coverage restrictions. CONCLUSION: Payers should consider the same set of decision criteria that they do with respect to non-orphan drugs: disease severity, availability of treatment alternatives, level of unmet medical need, and cost-effectiveness, criteria that justifiably may be taken into account and traded off against one another in prescribing and reimbursement decisions for orphan drugs.