BackgroundSome 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.MethodsWe collected data from drug regulatory agencies as well as payers and drug evaluation authorities.ResultsWe 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.ConclusionPayers 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.

Background: In the US, a new approval pathway for biosimilars has been established as part of the Aff ordable Care Act. Biosimilars are anticipated to increase treatment options and lower the growth in spending on biologicals. How the commercial prospects for biosimilars will play out in the US is uncertain. From a regulatory, approvals, and market standpoint, Europe is ahead of the US with respect to biosimilars. Lessons may be drawn from European experience. Objective: To examine challenges and opportunities with respect to market uptake of biosimilars in the US. Methods: We reviewed Medline-indexed manuscripts and grey literature published in the past fi ve years on the topics of biosimilar development and market uptake. The data collected in this review informed the development of two stakeholder surveys for payers and physicians. Main Survey Results: Almost all physicians surveyed believe that if a biosimilar is approved by the US Food and Drug Administration (FDA), the product will perform similarly to the originator biological with regard to safety and effi cacy. Most physicians say they will likely prescribe biosimilars as soon as they are approved by FDA. Additionally, the majority of physicians feel comfortable switching an existing patient from the originator biological to a biosimilar. All payer respondents intend to promote biosimilar uptake by diff erentiating between the originator biological and biosimilar through the use of formulary tiering to steer patients and physicians towards biosimilars. Most payers said they would recommend therapeutic switching of biosimilars. Seventy-fi ve per cent of payer respondents expect biosimilars to have a 15-35% price discount. Discussion: Physicians will display caution when deciding on prescribing biosimilars to existing patients. Payers will look to regulatory authority guidance for further support. To maximize cost savings, payers will likely employ formulary management tools, such as higher cost sharing for originators and lower cost sharing for biosimilars. To ensure access to and monitoring of post-marketing safety and eff ectiveness of biosimilars, payers may establish patient registries through coverage with evidence development arrangements. The expected price discount of 15-35% for biosimilars is not large. Furthermore, higher rebates on originator biologicals may be used by manufacturers as a barrier to adopting biosimilars. Therefore, biosimilar manufacturers will likely have to treat biosimilars as any other branded product. Finally, biosimilars will be subject to competition from new biologicals in the same therapeutic class, including incremental improvements to existing originators.

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.

No abstract

Data from the National Health and Nutrition Examination Survey • One in five youths had high total cholesterol, low high-density lipoprotein (HDL) cholesterol, or high non-HDL cholesterol. Cardiovascular disease (CVD) is a leading cause of death among adults in the United States. To improve the cardiovascular health of the U.S. population, clinical practice guidelines (1,2) recommend screening children and adolescents for risk factors associated with CVD, including abnormal blood cholesterol levels. This report provides 2011-2014 estimates from the National Health and Nutrition Examination Survey (NHANES) on the prevalence of high total cholesterol, low high-density lipoprotein (HDL) cholesterol, and high non-HDL cholesterol among children and adolescents aged 6-19.