BEACON: A Summary Framework to Overcome Potential Reimbursement Hurdles.

OBJECTIVE: To provide a framework for addressing payers' criteria during the development of pharmaceuticals. METHODS: A conceptual framework was presented to an international health economic expert panel for discussion. A structured literature search (from 2010 to May 2015), using the following databases in Ovid: Medline(®) and Medline(®) In-Process (PubMed), Embase (Ovid), EconLit (EBSCOhost) and the National Health Service Economic Evaluation Database (NHS EED), and a 'grey literature' search, were conducted to identify existing criteria from the payer perspective. The criteria assessed by existing frameworks and guidelines were collated; the most commonly reported criteria were considered for inclusion in the framework. A mnemonic was conceived as a memory aide to summarise these criteria. RESULTS: Overall, 41 publications were identified as potentially relevant to the objective. Following further screening, 26 were excluded upon full-text review on the basis of no framework presented (n = 13), redundancy (n = 11) or abstract only (n = 2). Frameworks that captured criteria developed for or utilised by the pharmaceutical industry (n = 5) and reimbursement guidance (n = 10) were reviewed. The most commonly identified criteria-unmet need/patient burden, safety, efficacy, quality-of-life outcomes, environment, evidence quality, budget impact and comparator-were incorporated into the summary framework. For ease of communication, the following mnemonic was developed: BEACON (Burden/target population, Environment, Affordability/value, Comparator, Outcomes, Number of studies/quality of evidence). CONCLUSIONS: The BEACON framework aims to capture the 'essence' of payer requirements by addressing the most commonly described criteria requested by payers regarding the introduction of a new pharmaceutical.

The Suitability of End Point Designs for Health Technology Assessment in Chronic Pain Studies.

OBJECTIVES: To identify the pain instruments and study end points most commonly used in clinical trial settings and to provide insight into the extent to which outcome measures in clinical studies are meeting payer needs. METHODS: A literature review was conducted to identify published clinical studies and ongoing/recently completed registered trials in chronic pain. Inclusion criteria were interventional study, chronic pain in adults, and pain measured within the primary end point. RESULTS: Of 1256 PubMed citations and 3006 clinical trial registry entries, 356 reported large clinical studies in pain populations (e.g., malignant, neuropathic, functional, and musculoskeletal). Studies were designed for superiority in 28% of PubMed citations and 8% of registry entries. The primary end points of most studies were single-dimension pain instruments, such as the numerical rating scale (n = 131) and the visual analogue scale (n = 69). In cases in which multidimensional pain end points were used, this was most commonly the Brief Pain Inventory (n = 37). Payer-relevant end points were typically limited to secondary end points, and were limited and/or reported inconsistently in published studies and ongoing/recently completed studies: preference-weighted quality of life (36% and 42%), resource use (2% and 8%), physical function (28% and 39%), and psychological function (25% and 24%). CONCLUSIONS: Most pain trials were not designed to show superiority to an active comparator, and they used single-dimension pain scales as their primary end point in combination with a broader selection of secondary end points. The inclusion of payer-relevant end points among clinical trials was inconsistent.

The value of irbesartan in the management of hypertension.

Elevated blood pressure levels are highly prevalent and are a major reason for cardiovascular events and thus place a significant financial burden on healthcare systems worldwide. Guidelines recommend five first-line anti-hypertensive drug classes, but compelling indications may indicate favoring one drug class over another. Angiotensin receptor blockers (ARBs) have demonstrated a blood pressure lowering efficacy which is at least comparable with other drug classes, including ACE inhibitors (ACE-I), beta-blockers, calcium channel blockers and diuretics. They have, in addition, a lower side effect profile than other drug classes and patients on ARBs are more persistent with therapy. Compelling indications for the use of ARBs are heart failure, post-myocardial infarction, diabetic nephropathy, proteinuria/microalbuminuria, left ventricular hypertrophy, atrial fibrillation, metabolic syndrome and ACE-I induced cough. The ARB irbesartan has demonstrated a high efficacy in lowering blood pressure, which has been shown to be at least comparable with ACE-Is and superior to other ARBs such as losartan and valsartan. This translated into a better cost-effectiveness for irbesartan than for valsartan and losartan in the treatment of hypertension. In addition, irbesartan has been shown to be effective in both early and late stage diabetic nephropathy. It has further demonstrated considerable cost savings over standard therapy including beta-blockers, diuretics and non-dihydropyridine calcium channel blockers at all stages of kidney disease. Based on efficacy data from the Irbesartan Diabetic Nephropathy Trial and Reduction of Endpoints in NIDDM (non insulin dependant diabetes melitis) with the Angiotensin II Antagonist Losartan Study, it has also demonstrated cost savings over losartan in late stage renal disease. While both losartan and irbesartan are registered for the treatment of late stage diabetic nephropathy, irbesartan is also registered for early stage diabetic nephropathy in the EU. In summary, the data from randomized clinical trials on the efficacy of antihypertensive drugs provides an indication of their real value to patients. In addition observational data from clinical practice and proven end-organ protection in diabetic nephropathy provides further evidence of the true value of irbesartan compared to other ARBs in the treatment of hypertension.

Dementia care initiative in primary practice: study protocol of a cluster randomized trial on dementia management in a general practice setting.

BACKGROUND: Current guidelines for dementia care recommend the combination of drug therapy with non-pharmaceutical measures like counselling and social support. However, the scientific evidence concerning non-pharmaceutical interventions for dementia patients and their informal caregivers remains inconclusive. Targets of modern comprehensive dementia care are to enable patients to live at home as long and as independent as possible and to reduce the burden of caregivers. The objective of the study is to compare a complex intervention including caregiver support groups and counselling against usual care in terms of time to nursing home placement. In this paper the study protocol is described. METHODS/DESIGN: The IDA (Initiative Demenzversorgung in der Allgemeinmedizin) project is designed as a three armed cluster-randomized trial where dementia patients and their informal caregivers are recruited by general practitioners. Patients in the study region of Middle Franconia, Germany, are included if they have mild or moderate dementia, are at least 65 years old, and are members of the German AOK (Allgemeine Ortskrankenkasse) sickness fund. In the control group patients receive regular treatment, whereas in the two intervention groups general practitioners participate in a training course in evidence based dementia treatment, recommend support groups and offer counseling to the family caregivers either beginning at baseline or after the 1-year follow-up. The study recruitment and follow-up took place from July 2005 to January 2009. 303 general practitioners were randomized of which 129 recruited a total of 390 patients. Time to nursing home admission within the two year intervention and follow-up period is the primary endpoint. Secondary endpoints are cognitive status, activities of daily living, burden of care giving as well as healthcare costs. For an economic analysis from the societal perspective, data are collected from caregivers as well as by the use of routine data from statutory health insurance and long-term care insurance. DISCUSSION: From a public health perspective, the IDA trial is expected to lead to evidence based results on the community effectiveness of non-pharmaceutical support measures for dementia patients and their caregivers in the primary care sector. For health policy makers it is necessary to make their decisions about financing new services based on strong knowledge about the acceptance of measures in the population and their cost-effectiveness. TRIAL REGISTRATION: ISRCTN68329593.

Irritable bowel syndrome in Germany. A cost of illness study.

OBJECTIVES: When asked, between 10% and 15% of people in the Western world report symptoms compatible with irritable bowel syndrome, and around 5% seek medical advice for these complaints. This should incur considerable costs. The present study was designed to give a cost estimate for the treatment of patients with irritable bowel syndrome paid for by German statutory health insurance. METHODS: Fifty doctors working in private practice were randomly selected to each carry out personal interviews on four irritable bowel syndrome patients chosen from their own records (total 200 patients). Using a structured questionnaire, information regarding diagnostic procedures, drugs and other therapies, hospitalization and days off work were obtained from the case records. To calculate the total direct costs of the illness, all single cost elements such as physician services, medication and hospitalization were included. RESULTS: The number of office visits was nine per patient per year; nearly one-third of employed patients missed work for irritable bowel syndrome, and one out of 15 patients was hospitalized for this condition. Several technical procedures were ordered, mostly laboratory tests. Nearly all patients had at least one drug prescription for irritable bowel syndrome during the year of the survey, with a mean of 3.5 prescriptions per patient. The largest pharmacological groups were antispasmodics (29.2%) and prokinetics (8.9%). Total direct costs for one irritable bowel syndrome patient per year amounted to 1548 DEM (791.48 [OV0556]), comprising roughly 25% for physician visits and tests, 50% for drugs and 25% for hospitalization. Including indirect costs for sick leave, total costs were 1946 DEM (994.97 [OV0556]) per patient per year. CONCLUSIONS: The costs incurred by irritable bowel syndrome are considerable.

Diffusion of innovations: treatment of Alzheimer's disease in Germany.

Systematic barriers seem to slow down the market penetration of innovative acethylcholinesterase (AChE) inhibitors in Alzheimer's disease. The goal of our study was to examine the diffusion of AChE inhibitors into the German market in more detail. On the basis of using the ongoing surveillance panel of the Institute of Medical Statistic (IMS) Health, the prescription patterns of 100 physicians (72 general practitioners, 28 neurologists) were examined. In addition, structured telephone interviews with the same 100 physicians were conducted. The interview included the assessment of a hypothetical treatment situation (i.e. physicians were asked what they would prescribe if a close relative of theirs had Alzheimer's disease) as well as qualitative items examining the physicians' attitudes towards AChE inhibitors and the perceived impact on drug budgets. As a major result, the analysis revealed that neurologists prescribed AChE inhibitors to 44.6% of their patients, while general practitioners only treated 9.0% of their patients with AChE inhibitors. The analysis of the qualitative items revealed positive attitudes regarding the safety and efficacy of AChE inhibitors, but negative attitudes regarding the budgetary limitations to prescribing these drugs. A correlation of r=0.21 (P<0.05) was found between the perceived impact on drug budgets and the adoption of AChE inhibitors and a correlation of r=0.32 (P<0.002) was seen between the physician's specialty and the adoption of AChE inhibitors. These data show that, while the AChE inhibitor adoption process has passed the early stages, various barriers slow down the final stages of AChE inhibitor adoption. The drug budget in particular seems to inhibit the adoption of the innovation by the majority of general practitioners. This leads to a more short-term cost control strategy instead of long-term disease management and cost saving approaches.