Cost Sharing of Disease-Modifying Treatments (DMTs) as Policy Lever to Improve DMTs' Access in Multiple Sclerosis.

OBJECTIVES: To understand the nonlinear relationship between out-of-pocket (OOP) payments and disease-modifying treatment (DMT) use and adherence, primarily to pinpoint the threshold at which the use of DMTs becomes price sensitive. METHODS: Individuals with more than two multiple sclerosis (MS) diagnoses (International Classification of Diseases, Ninth Revision code 340) were identified from the MarketScan database (2006-2009). Heterogeneity in treatment was normalized by calculating an annual OOP payment as the average OOP payment for purchasing a fixed basket of DMTs at the insurance plan level. A local linear regression with a model-based recursive partitioning algorithm was applied to explore the relationship between OOP and consequently lower DMT use and adherence as measured by days covered by DMT. RESULTS: We identified the inflection points in annual OOP payments as $442 for DMT use and $890 for DMT adherence. For patients with annual OOP payments of more than $442, a $100-increase in OOP payment was associated with a decline of 0.6% in DMT use; for annual OOP payments of more than $890, a $100-increase in OOP payment was associated with two fewer days of DMT treatments. CONCLUSIONS: Although the use of DMTs and DMT adherence appeared unassociated with OOP payment below $442 and $890, respectively, an excessive OOP payment was a barrier to DMT access. This information can inform maximum monthly and yearly payment caps when designing valued-based insurance plans.

Choosing Wisely BMT: American Society for Blood and Marrow Transplantation and Canadian Blood and Marrow Transplant Group's List of 5 Tests and Treatments to Question in Blood and Marrow Transplantation.

Choosing Wisely encourages dialogue about reducing unnecessary procedures, tests, or treatments in healthcare. The American Society for Blood and Marrow Transplantation (ASBMT) and Canadian Blood and Marrow Transplant Group (CBMTG) established a Choosing Wisely BMT Task Force whose objective was to create a list of top 5 practices in blood and marrow transplantation to be questioned. The Task Force consisted of representatives from ASBMT's Quality Outcomes, Education, and Practice Guidelines committees; ASBMT's Pharmacy Special Interest Group; CBMTG Program Directors; and Center for International Blood and Marrow Transplant Research (CIBMTR). Suggestions for current transplantation practices to question were elicited from the CBMTG Program Directors; members of ASBMT's Quality Outcomes, Practice Guidelines, and Education committees; and chairs of the CIBMTR scientific working committees. We received 119 unique suggestions that were ranked based on their potential impact on harm reduction, cost reduction, necessity of the test or practice, and the strength of available evidence. Through a modified Delphi process, suggestions were narrowed down to 6, which were then subjected to systematic reviews. The final 5 recommendations focus on graft source for patients with aplastic anemia, corticosteroid dose for initial treatment of graft-versus-host-disease, optimal number of umbilical cord blood units for transplantation, graft source in matched unrelated donor transplantation, and use of prophylactic intravenous immunoglobulin in transplant recipients. These Choosing Wisely BMT recommendations are relevant to the current clinical practice of blood and marrow transplantation and focus on tests, treatments, or procedures that may be harmful, wasteful, or for which there is no apparent clinical benefit.

Why Do Promising Therapies Stall in Development and How Can We Move Them Forward?

There are many reasons that molecules fail to progress to market and various principles of risk-benefit decisions that can help drive the molecule through development. This symposium included discussions on global strategies involved in pushing promising molecules to market, what to do when a molecule stalls in its progress to market, and options for rescuing the molecule and pushing it forward again. Innovative partnerships that bring stalled drugs back into clinical development were also addressed. A regulatory perspective on common reasons for a molecule to fail in its forward progress was presented. In addition, situations arise when a third-party advisory committee can provide input to help overcome issues identified by a regulatory agency. Using examples from the private and public domain, presentations centered on how to repurpose a molecule and when more science is needed.

The worldwide trend of using botanical drugs and strategies for developing global drugs.

Natural product drugs, or botanical drugs, are drugs composed of natural substances which have constituents with healthenhancing or medicinal activities. In Korea, government-led projects brought attention to botanical drugs invigorating domestic botanical drug industry. Foreign markets, as well, are growing bigger as the significance of botanical drugs stood out. To follow along with the tendency, Korea puts a lot of effort on developing botanical drugs suitable for global market. However, standards for approving drug sales vary by countries. And also, thorough standardization, certification, clinical studies and data of these will be required as well as data confirming safety and effectiveness. Meanwhile, as an international exchange in botanical drug market continues, the importance of plant resources was emphasized. Thus countries' ownership of domestic natural resources became vital. Not only establishing a systematic method to secure domestic plant resources, but also cooperation with other countries on sharing natural resources is essential to procure natural resources effectively. Korea started to show visible results with botanical drugs, and asthma/COPD treatment made out of speedwell is one example. Sufficient investment and government's active support for basic infrastructure for global botanical drugs will bring Korea to much higher level of botanical drug development. [BMB Reports 2017; 50(3): 111-116].

Optimal control for an age-structured model for the transmission of hepatitis B.

One of the characteristics of HBV transmission is the age structure of the host population and the vertical transmission of the disease. That is the infection is transmitted directly from infected mother to an embryo, fetus, or baby during pregnancy or childbirth (the perinatal infection). We formulated an age-structured model for the transmission dynamics of HBV with differential infectivity: symptomatic and asymptomatic infections. The model without intervention strategies is completely analyzed. We compute the basic reproduction number which determines the outcome of the disease. We also compute equilibria and study their stability. The sensitivity analysis of the initial model parameters is performed (to determine the impact of control-related parameters on outbreak severity). Using optimal control theory, we determine the cost-effective balance of three interventions methods which minimizes HBV-related deaths as well as the costs associated with intervention.

The Role of Chronic Disease, Obesity, and Improved Treatment and Detection in Accounting for the Rise in Healthcare Spending Between 1987 and 2011.

BACKGROUND: To curb rising healthcare expenditures in the U.S.A., the factors underlying this growth must be well understood. OBJECTIVE: We aim to explore how chronic disease prevalence, obesity, and improved disease detection and treatment rates contributed to the growth in health spending in the U.S.A. between 1987 and 2011. METHODS: We use spending decomposition equations to estimate the portion of spending growth attributable to prevalence increases, rising treatment costs, and population growth, respectively. We use two-part models to estimate the portion of prevalence-related spending that is potentially due to obesity. We examine changing diagnosis and treatment rates to assess how much of the growth in spending might be desirable. RESULTS: We find that the share of total healthcare spending associated with the treatment of chronic disease has risen dramatically from 1987-2011. In particular, we estimate that 77.6% of healthcare spending growth is attributable to patients with four or more chronic conditions. We find that rising obesity levels may explain between 11.4 and 23.5% of the increase in healthcare expenditure for several specific chronic conditions. Diagnosis and treatment rates for chronic disease are improving. CONCLUSIONS: Individuals with multiple chronic conditions are disproportionately responsible for rising healthcare expenditure. Much of spending growth associated with rising rates of chronic disease can be linked to rising obesity rates. Though much of the growth in spending is generally considered undesirable, disease detection and treatment rates are also rising, suggesting that at least some of the recent growth in healthcare expenditure may be beneficial.

Newborn screening programmes in Europe; arguments and efforts regarding harmonization. Part 2. From screening laboratory results to treatment, follow-up and quality assurance.

In a survey conducted in 2010/2011 data from the 28 EU member states, four EU candidate states (Croatia, FYROM, Iceland, Turkey), three potential EU candidate states (Bosnia Herzegovina, Montenegro, Serbia), and two EFTA states (Norway and Switzerland) were collected. The status and function of newborn screening (NBS) programmes were investigated from the information to prospective parents and the public via confirmation of a positive screening result up to decisions on treatment. This article summarises the results from screening laboratory findings to start of treatment. In addition we asked about the existence of feedback loops reporting the conclusions of confirmation of screening results to the screening laboratory and communication of long-term outcome to diagnostic units and possibly existing central registries. Parallel to the description of actual practices of where, how and by whom the different steps of the programmes are executed, we also asked for the existence of guidelines or directives regulating the screening programmes, material to support information of parents about diagnoses and treatment and training facilities for professionals involved in the programmes. This survey gives a first comprehensive overview of the steps following a positive screening result in European NBS programmes. The 37 data sets reveal substantial variation of national screening panels, but also a lot of similarities. Analysis across all countries revealed that actual practice is often organised but not regulated by guidelines. Material to inform patients is available more often for explaining treatment (69 %) than explaining the necessity of confirmatory diagnostics (41 %). Training of professionals is rarely regulated by a guideline (2 %), but is offered for paediatricians (40 %) and dieticians (29 %) and only rarely for other professions (e.g. geneticists, clinical nurse specialists, psychologists). Registry-based evaluation of long-term outcome is as yet almost nonexistent (3 %).

Therapeutic erythrocytapheresis versus phlebotomy in the initial treatment of hereditary hemochromatosis - A pilot study.

Hereditary Hemochromatosis (HH) is a genetic disorder of iron metabolism, resulting in excessive iron overload. Currently, phlebotomy is the standard effective treatment that prevents progression of tissue damage. Aim of the therapy is to reach ferritin levels between 20 and 50mugl(-1). In patients with total iron stores of more than 30g, intensive treatment by means of weekly phlebotomies during 2-3 years is required to reach this aim. More recently mechanical removal of erythrocytes through therapeutic erythrocytapheresis (TE) has become a new therapeutic modality. By means of TE, up to 1000ml erythrocytes per session can be removed, depending on patient characteristics, compared to 250ml erythrocytes per phlebotomy. Thus, TE potentially offers a more efficient method of removing iron overload with less procedures in a shorter treatment period. In a pilot study between 2002 and 2005, results from a group of HH patients treated with TE (N=6) were compared to the results of a historical control group of HH patients (N=6) treated with phlebotomy. The results showed a reduction of almost 70% in both the total number and the duration of treatments in the TE group. Although, the procedure costs compared on the basis of a single TE session were higher, the total costs for the whole treatment were comparable or cheaper with the use of TE. Future prospective studies are needed to compare both therapies in a randomized setting.