Does Cost-Effectiveness Analysis Overvalue Potential Cures? Exploring Alternative Methods for Applying a "Shared Savings" Approach to Cost Offsets.

OBJECTIVES: To evaluate alternative methods to calculate and/or attribute economic surplus in the cost-effectiveness analysis of single or short-term therapies. METHODS: We performed a systematic literature review of articles describing alternative methods for cost-effectiveness analysis of potentially curative therapies whose assessment using traditional methods may suggest unaffordable valuations owing to the magnitude of estimated long-term quality-adjusted life-year (QALY) gains or cost offsets. Through internal deliberation and discussion with staff at the Health Technology Assessment bodies in England and Canada, we developed the following 3 alternative methods for further evaluation: (1) capping annual costs in the comparator arm at $150 000 per year; (2) "sharing" the economic surplus with the health sector by apportioning only 50% of cost offsets or 50% of cost offsets and QALY gains to the value of the therapy; and (3) crediting the therapy with only 12 years of the average annual cost offsets or cost offsets and QALY gains over the lifetime horizon. The impact of each alternative method was evaluated by applying it in an economic model of 3 hypothetical condition-treatment scenarios meant to reflect a diversity of chronicity and background healthcare costs. RESULTS: The alternative with greatest impact on threshold price for the fatal pediatric condition spinal muscular atrophy type 1 was the 12-year cutoff scenario. For a hypothetical one-time treatment for hemophilia A, capping cost offsets at $150 000 per year had the greatest impact. For chimeric antigen receptor T-cell treatment of non-Hodgkin's lymphoma, capping cost offsets or using 12-year threshold had little impact, whereas 50% sharing of surplus including QALY gains and cost offsets greatly reduced threshold pricing. CONCLUSIONS: Health Technology Assessment bodies and policy makers will wrestle with how to evaluate single or short-term potentially curative therapies and establish pricing and payment mechanisms to ensure sustainability. Scenario analyses using alternative methods for calculating and apportioning economic surplus can provide starkly different assessment results. These methods may stimulate important societal dialogue on fair pricing for these novel treatments.

[Systematic economic assessment and quality evaluation for traditional Chinese medicines].

To learn about the economic studies on traditional Chinese medicines in domestic literatures, in order to analyze the current economic assessment of traditional Chinese medicines and explore the existing problems. Efforts were made to search CNKI, VIP, Wanfang database and CBM by computer and include all literatures about economic assessment of traditional Chinese medicines published on professional domestic journals in the systematic assessment and quality evaluation. Finally, 50 articles were included in the study, and the systematic assessment and quality evaluation were made for them in terms of titles, year, authors' identity, expense source, disease type, study perspective, study design type, study target, study target source, time limit, cost calculation, effect indicator, analytical technique and sensitivity analysis. The finally quality score was 0.74, which is very low. The results of the study showed insufficient studies on economics of traditional Chinese medicines, short study duration and simple evaluation methods, which will be solved through unremitting efforts in the future.

[Economic perspectives of the research on advanced therapies]. / Perspectivas económicas de la investigación en terapia avanzada.

Since a new advanced therapy medicinal product is discovered until finally allowed its sale in the domestic market, it has to overcome a series of stages. Biomedical research is the first phase, currently its situation is encouraging to the increase in the number of clinical trials in Spain and in the rest of the world, despite the economic situation and the various difficulties that have faced the pharmaceutical laboratories. The next phase consists in obtaining the authorization of marketing of the European Medicines Agency. After authorization, will attempt to set a fair and moderate price for inclusion in the list of health provision of Social Security. A price for a drug that provides added value to health and society, a price that is generated profits for the pharmaceutical companies that hope to make up for the years of work and investment. Commitment to advanced therapy must be clear and forceful, to fund ongoing research projects and encouraging their creation with economic aid.

Desde que un nuevo medicamento de terapia avanzada sale a la luz hasta que finalmente se permite su venta en el mercado nacional, tiene que superar una serie de fases. La investigación biomédica es la primera fase, actualmente su situación resulta alentadora por el aumento del número de ensayos clínicos en España y en el resto del mundo a pesar de la coyuntura económica y de las diversas dificultades a las que tienen que enfrentarse los laboratorios farmacéuticos. La siguiente fase consiste en la obtención de la autorización de comercialización por parte de Agencia Europea del Medicamento. Tras dicha autorización, se intentará fijar un precio justo y moderado para la inclusión en el listado de prestación sanitaria por parte de la Seguridad Social. Un precio por un medicamento que aporta valor añadido a la salud y la sociedad, con el se generán ganancias para las empresas farmacéuticas que esperan compensar los años de trabajo e inversión. La apuesta por la terapia avanzada debe ser clara y contundente, financiando los proyectos de investigación en curso y animando a su creación con ayudas económicas.

Patient preferences and willingness to pay for tinnitus treatments.

PURPOSE: There will likely be several different tinnitus treatments necessary, and it is important to understand patient preferences and factors that might contribute to treatment acceptability. This study explores the acceptability of a wide range of different tinnitus treatments, from noninvasive wearable devices to surgically implanted devices in the brain. Understanding how tinnitus sufferers consider and rank such options and how they might be influenced by their own perception of the severity of their tinnitus could help clinicians, researchers, and companies plan future efforts for approaching new treatments. DATA COLLECTION AND ANALYSIS: 197 tinnitus self-help group attendees rated their acceptance of treatments on a scale from 0 (not acceptable) to 100 (fully acceptable). The treatments included external devices, medications, cochlear implants, an implant on the brain surface, and an implant in the brain. They were also asked how much they would pay for successful treatments. RESULTS: There was a significant correlation between loudness and annoyance (r = .78). To reduce tinnitus by half, an "acceptable" response between 91 and 100 was reported by 30% of the respondents for devices, by 52% for pills, by 25% for cochlear implants, by 13% for implants on the brain surface, and by 13% for implants in the brain. To reduce tinnitus completely, a 91-100 acceptable response was reported by 42% for devices, by 62% for pills, by 38% for cochlear implants, by 21% for implants on the brain surface, and by 19% for implants in the brain. To reduce tinnitus completely, participants most commonly selected to pay at least $5000, and 20.3% were willing to pay as much as $25,000. The ratings of tinnitus loudness and annoyance were positively correlated with the likelihood of using any treatment. Surprisingly, there was a weak relationship between annoyance and the amount they were willing to pay. CONCLUSIONS: Tinnitus patients are prepared to accept a wide variety of treatments. Medications are the most acceptable. Invasive procedures can also be acceptable to many, particularly if they provide complete relief.

Strategies for treating latent multiple-drug resistant tuberculosis: a decision analysis.

BACKGROUND: The optimal treatment for latent multiple-drug resistant tuberculosis infection remains unclear. In anticipation of future clinical trials, we modeled the expected performance of six potential regimens for treatment of latent multiple-drug resistant tuberculosis. METHODS: A computerized Markov model to analyze the total cost of treatment for six different regimens: Pyrazinamide/ethambutol, moxifloxacin monotherapy, moxifloxacin/pyrazinamide, moxifloxacin/ethambutol, moxifloxacin/ethionamide, and moxifloxacin/PA-824. Efficacy estimates were extrapolated from mouse models and examined over a wide range of assumptions. RESULTS: In the base-case, moxifloxacin monotherapy was the lowest cost strategy, but moxifloxacin/ethambutol was cost-effective at an incremental cost-effectiveness ratio of $21,252 per quality-adjusted life-year. Both pyrazinamide-containing regimens were dominated due to their toxicity. A hypothetical regimen of low toxicity and even modest efficacy was cost-effective compared to "no treatment." CONCLUSION: In our model, moxifloxacin/ethambutol was the preferred treatment strategy under a wide range of assumptions; pyrazinamide-containing regimens fared poorly because of high rates of toxicity. Although more data are needed on efficacy of treatments for latent MDR-TB infection, data on toxicity and treatment discontinuation, which are easier to obtain, could have a substantial impact on public health practice.

[Overview of pharmacoeconomic studies on traditional Chinese medicines and western medicines in treatment of stroke].

In recent years, pharmacoeconomic studies have drawn increasing attention from experts in clinical medicine, pharmacy, health economics and health management. Their findings play an important role in drug pricing, drafting of the List of Essential Medicines and rational selection of clinical drugs. The essay summarizes the literatures published in recent years concerning pharmacoeconomics of traditional Chinese medicines on stroke, in order to make clear the current development of the latest studies on traditional Chinese medicines on treating stroke and rehabilitation methods and provide basis for further studies on pharmacoeconomics of traditional Chinese medicines on stroke and reference for rational clinical drug use.

Retrospective evaluation of the impact of copayment increases for specialty medications on adherence and persistence in an integrated health maintenance organization system.

BACKGROUND: Specialty drugs are generally defined as high-cost injectable, infused, oral, or inhaled drugs that require close monitoring. Specialty drugs account for an increasing percentage of total drug expenditures, and management of specialty drugs has become a priority. A Central Texas-based integrated health maintenance organization system implemented a specialty drug benefit to manage expensive specialty drug costs. OBJECTIVES: Our objective was to measure and compare the change in adherence and persistence after implementation of copayment increases for select specialty medications used on a long-term basis (at least 2 years). METHODS: Patients who were long-term users of anti-inflammatory, immunosuppressant, cancer, and multiple sclerosis medications were selected. The intervention group consisted of those whose out-of-pocket payment for specialty medications increased, and the control group consisted of those whose out-of-pocket costs did not change. Adherence, defined by proportion of days covered, was measured every 3 months for 12 months before and after the change. Individual growth model analysis evaluated the changes in adherence. Cox regression analysis determined the difference in persistence between groups. RESULTS: There were 178 and 202 patients in the intervention and control groups, respectively. The growth model showed a small but statistically significant decrease in proportion of days covered of 0.040 after copay changes in the intervention versus control group (P < 0.001) for immunosuppressants. The Cox regression analysis indicated a higher probability of intervention patients on anti-inflammatory drugs (hazard ratio [HR] = 2.53; 95% CI, 1.38-4.62) and immunosuppressants (HR = 3.01; 95% CI, 1.20-7.56) would be nonpersistent compared with those in their control groups. CONCLUSIONS: The move to the specialty formulary allows for closer scrutiny of specialty utilization by pharmacists, who actively monitor utilization and access. Despite the minimal adherence decrease and significant persistence changes with certain drug types, the results indicated relatively more stability with specialty drug use than reported with traditional pharmaceuticals.

Animal products, diseases and drugs: a plea for better integration between agricultural sciences, human nutrition and human pharmacology.

Eicosanoids are major players in the pathogenesis of several common diseases, with either overproduction or imbalance (e.g. between thromboxanes and prostacyclins) often leading to worsening of disease symptoms. Both the total rate of eicosanoid production and the balance between eicosanoids with opposite effects are strongly dependent on dietary factors, such as the daily intakes of various eicosanoid precursor fatty acids, and also on the intakes of several antioxidant nutrients including selenium and sulphur amino acids. Even though the underlying biochemical mechanisms have been thoroughly studied for more than 30 years, neither the agricultural sector nor medical practitioners have shown much interest in making practical use of the abundant high-quality research data now available. In this article, we discuss some specific examples of the interactions between diet and drugs in the pathogenesis and therapy of various common diseases. We also discuss, using common pain conditions and cancer as specific examples, how a better integration between agricultural science, nutrition and pharmacology could lead to improved treatment for important diseases (with improved overall therapeutic effect at the same time as negative side effects and therapy costs can be strongly reduced). It is shown how an unnaturally high omega-6/omega-3 fatty acid concentration ratio in meat, offal and eggs (because the omega-6/omega-3 ratio of the animal diet is unnaturally high) directly leads to exacerbation of pain conditions, cardiovascular disease and probably most cancers. It should be technologically easy and fairly inexpensive to produce poultry and pork meat with much more long-chain omega-3 fatty acids and less arachidonic acid than now, at the same time as they could also have a similar selenium concentration as is common in marine fish. The health economic benefits of such products for society as a whole must be expected vastly to outweigh the direct costs for the farming sector.

Do interventions to prevent lifestyle-related diseases reduce healthcare expenditures? A randomized controlled clinical trial.

BACKGROUND: In 2008, the Japanese government implemented a program of health lifestyle interventions to reduce health care expenditure. This study evaluated whether these interventions decreased health care expenditures. METHODS: The study enrolled 99 participants insured by Japanese National Health Insurance, who, in our previous study conducted in 2004, were allocated by random sampling into an intervention group (50 participants) and a control group (49 participants). In the intervention group, we used a health support method that facilitated the attainment of goals established by each participant. The control group received instruction in exercise, as well as health support using publically available media. Although 3 participants in the intervention group and 9 participants in the control group did not participate in a follow-up health examination 1 year after the intervention, the health care expenditures of all initial participants were assessed. Expenditures before and after the intervention were compared within and between groups. Data on health care expenditures were obtained from inpatient, outpatient, pharmacy, and dental health insurance claims. RESULTS: After the intervention, the pharmacy and dental expenditures were significantly higher in the intervention group, while the pharmacy expenditure was significantly higher in the control group. However, there was no significant difference in any medical expenditure item between the intervention and control groups before or after the intervention. CONCLUSIONS: No significant differences were observed in short-term medical expenses for any medical expenditure item after a lifestyle intervention.

[Application of pharmacoeconomics in clinical management]. / Aplicación de la farmacoeconomía en la gestión clínica.

The present article discusses the importance of clinical management in the transformation of organizations and its role in the daily activities of health professionals and, in particular, of hospital pharmacists. Because of social changes, healthcare models must make the shift from more rigid management models toward new organizational models based on clinical management. From this perspective, pharmacoeconomics is viewed as a useful tool to introduce the criteria of efficiency in all decisions subject to clinical management, including those on pharmacotherapeutics. Subsequently, the application of this discipline is discussed in real decision-making scenarios and settings for its use within the context of the work of hospital pharmacy are proposed.

An antimicrobial stewardship program's impact with rapid polymerase chain reaction methicillin-resistant Staphylococcus aureus/S. aureus blood culture test in patients with S. aureus bacteremia.

Rapid organism detection of Staphylococcus aureus bacteremia and communication to clinicians expedites antibiotic optimization. We evaluated clinical and economic outcomes of a rapid polymerase chain reaction methicillin­resistant S. aureus/S. aureus blood culture test (rPCR). This single­center study compared inpatients with S. aureus bacteremia admitted from 1 September 2008 through 31 December 2008 (pre­rPCR) and those admitted from 10 March 2009 through 30 June 2009 (post­rPCR). An infectious diseases pharmacist was contacted with results of the rPCR; effective antibiotics and an infectious diseases consult were recommended. Multivariable regression assessed clinical and economic outcomes of the 156 patients. Mean time to switch from empiric vancomycin to cefazolin or nafcillin in patients with methicillin­susceptible S. aureus bacteremia was 1.7 days shorter post­rPCR (P = .002). In the post­rPCR methicillin­susceptible and methicillin­resistant S. aureus groups, the mean length of stay was 6.2 days shorter (P = .07) and the mean hospital costs were $21,387 less (P = .02). rPCR allows rapid differentiation of S. aureus bacteremia, enabling timely, effective therapy and is associated with decreased length of stay and health care costs.

The costs of drugs used to treat myelodysplastic syndromes following National Comprehensive Cancer Network Guidelines.

Guidelines for management of patients with myelodysplastic syndromes (MDS) have been generated by the National Comprehensive Cancer Network (NCCN) Myelodysplastic Syndromes Panel. Because MDS is a heterogeneous spectrum of disorders, these patients have been categorized into prognostic subgroups, predominantly using the International Prognostic Scoring System (IPSS). Several drugs have been used to treat these patients, and their selection and sequential recommended use by the panel depend on disease characteristics and responses to treatment. Recombinant erythropoietin alfa and darbepoetin alfa have been the mainstay of therapy for treating anemia associated with MDS. The FDA has recently approved several other drugs for treating MDS, including azacytidine and decitabine for all stages of disease, lenalidomide for low-risk anemic patients with del(5q) chromosomal abnormality, and deferasirox for treating iron overload. For iron chelation, deferoxamine is also used occasionally. Treatment with immunosuppressive therapy (antithymocyte globulin and cyclosporin) has been therapeutically beneficial for a subset of younger patients with MDS. Because the financial cost of these therapies are substantial and have received only limited attention, this article evaluates the costs of specific drugs and their sequential use in the lower-risk IPSS (low and intermediate-1) subgroups based on the NCCN guidelines. Results estimate an average annual cost for potentially anemia-altering drugs of $63,577 per patient, ranging from $26,000 to $95,000, depending on the specific therapies. In patients for whom the therapies fail, annual costs for iron chelation plus red blood cell transfusions are estimated to average $41,412. The economic impact of drug therapy should be weighed against the patient's potential for improvement in clinical outcomes, quality of life, and transfusion requirements.

Health economic consequences related to the diagnosis of fibromyalgia syndrome.

OBJECTIVE: To evaluate the use and costs of medical resources before and after a diagnosis of fibromyalgia syndrome (FMS) in a large primary care population in the UK. METHODS: We applied an existing data set for medical resource use among patients with a coded diagnosis of FMS. The observed quantities of 157 types of medical resource use before and after the diagnosis of FMS were multiplied by unit costs in order to calculate the cost of care (general practitioner [GP] visits, drugs, referrals, and diagnostics) within the National Health Service, excluding hospital costs. Costs before diagnosis were used in a trend analysis to predict later costs, assuming the diagnosis had never been made, and these predicted costs were compared with the observed costs after diagnosis. RESULTS: Following a diagnosis of FMS, a decrease in costs as compared with the predicted trend was observed. In the 4 years after diagnosis, the average difference between the predicted and observed cost was pound66.21 per 6 months per patient. This suggests that making the diagnosis leads to savings and a decrease in resource use. The main effect was observed for tests and imaging ( pound24.02 per 6 months), followed by pharmaceuticals ( pound22.27), referrals ( pound15.56), and GP visits ( pound4.36). CONCLUSION: Failure to diagnose a true case of FMS has its own costs, largely in excess GP visits, investigations, and prescriptions.

The effects of copayments on medication adherence during the first two years of prescription drug treatment.

OBJECTIVE: The aim of this study was to examine effects of initial prescription copayment size and observed increase on adherence by analyzing data from a managed care database. METHODS: Medical-pharmacy claims data were abstracted from the Integrated Health Care Information Services (IHCIS) National Managed Care Benchmark database for primary employer-sponsored subscribers. Incident fills and refills for the 10 most common medication groups between 2001 and 2003 were predicted by size and observed increase in copayment with the use of survival analysis. RESULTS: High copayments and observed copayment increases were associated with termination of medication use. Whereas effects of copayment level were limited to the first few fills, effects of observed increases in copayments were persistent. CONCLUSIONS: The strategy of increasing initially low copayments after the patient has made enough fills to become insensitive to copayment level is contraindicated by observed increases in copayment, predicting termination. However, other financial incentives might nonetheless help reduce early termination of medication use.

A cost-benefit analysis of music therapy in a home hospice.

Medicare's fixed daily rates create an absolute cost constraint on hospices; consequently, the growth in hospice brings financial pressures. The patient efficacy of music therapy has been demonstrated in the literature and includes improving pain, agitation, disruptive behaviors, communication, depression, and quality of life. Music therapy is well suited to hospice as it addresses the four domains of palliative care (physiological, emotional, social, and spiritual care). In this small study, the total cost of patients in music therapy was $10,659 and $13,643 for standard care patients, resulting in a cost savings of $2984. The music therapy program cost $3615, yielding a cost benefit ratio of 0.83. When using cost per patient day, the cost benefit ratio is 0.95.

A survey of herbal and alternative medication use among participants with eating disorder symptoms.

OBJECTIVE: The purpose of this survey was to collect data on herbal use in participants with eating disorder symptoms. METHOD: A survey was administered to 100 participants who had either sought treatment at the Eating Disorder Institute (EDI) or had been enrolled in previous research as the result of eating disorder symptoms. RESULTS: Of the 100 participants, 64% used an herbal product for weight loss. The mean monthly expenditure on herbs over the past year was 33.88 dollars +/- 41.10 dollars, with a range of 2 dollars-200 dollars. Dexatrim (Chattem, Chattanooga, TN; N = 27) and St. John's Wort (N = 19) had the highest reported use. Magazines were the most common source of product information (38.3%), with health care professionals being reported less frequently as the source of information. Knowledge of ephedra-related adverse effects was variable, and depended on a previous history of use. The majority (62.3%) of herb users reported an adverse effect. CONCLUSION: Herbal use is frequent among those with eating disorder symptoms, often resulting in substantial financial cost. Health professionals are rarely the source of herbal information. Therefore, there is ample room for educational interventions, which may result in the safer use of herbal products.

[Chemotherapy and DPC, prospective payment].

In 2003, the Japanese government introduced a prospective payment methodology into acute inpatient care services by developing a new, national patient grouping, called Diagnosis Procedure Combination(DPC). It raised issues relating to; 1) settling charges for combinations of treatment modalities in a single admission, 2) large practice variations in chemotherapeutic regimens and its pharmaceutical costs, 3) effects of shorter length of stays and outpatient chemotherapy, 4) payment adjustment for hospitals providing care to terminally ill, relapse and metastatic cases. In order to overcome these issues; a) oncologists need to develop treatment guidelines and standardize chemotherapeutic regimens, b) refine DPC to incorporate chemotherapy protocols, c) develop adjustment measures for different densities of care and casemix.

Shifting the focus from cost to value: a government perspective.

OBJECTIVE: To examine the federal government.s current health care initiatives in the Centers for Medicare and Medicaid Services (CMS) to improve quality of care and cost-effectiveness around the prescription drug benefit. SUMMARY: The government has a unique perspective on quality and outcome. Pharmacists need a working knowledge of 3 areas: (1) Medicare Part D, (2) medication therapy management efforts, and (3) demonstration projects. All of these will make demands on health care providers but should result in better outcomes data and patient care. All of these areas also have value components, and all have strategic implications as 2006 moves into 2007. CONCLUSION: CMS does not consider managing cost and providing value to be mutually exclusive and is attempting to move America.s health care toward a value-based program.