Orphan drug policies and use in pediatric nephrology.

Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness. Processes related to drug pricing, reimbursement, and thereby availability, vary between countries, thus having implications on patient care. These processes are discussed, with specific focus on three drugs used in pediatric nephrology: agalsidase beta (for Fabry disease), eculizumab (for atypical hemolytic uremic syndrome), and cysteamine bitartrate (for cystinosis). Access to and costs of orphan drugs have most profound implications for patients, but also for their physicians, hospitals, insurance policies, and society at large, particularly from financial and ethical standpoints.

Critical and honest conversations: the evidence behind the "Choosing Wisely" campaign recommendations by the American Society of Nephrology.

Estimates suggest that one third of United States health care spending results from overuse or misuse of tests, procedures, and therapies. The American Board of Internal Medicine Foundation, in partnership with Consumer Reports, initiated the "Choosing Wisely" campaign to identify areas in patient care and resource use most open to improvement. Nine subspecialty organizations joined the campaign; each organization identified five tests, procedures, or therapies that are overused, are misused, or could potentially lead to harm or unnecessary health care spending. Each of the American Society of Nephrology's (ASN's) 10 advisory groups submitted recommendations for inclusion. The ASN Quality and Patient Safety Task Force selected five recommendations based on relevance and importance to individuals with kidney disease.Recommendations selected were: (1) Do not perform routine cancer screening for dialysis patients with limited life expectancies without signs or symptoms; (2) do not administer erythropoiesis-stimulating agents to CKD patients with hemoglobin levels ≥10 g/dl without symptoms of anemia; (3) avoid nonsteroidal anti-inflammatory drugs in individuals with hypertension, heart failure, or CKD of all causes, including diabetes; (4) do not place peripherally inserted central catheters in stage 3-5 CKD patients without consulting nephrology; (5) do not initiate chronic dialysis without ensuring a shared decision-making process between patients, their families, and their physicians.These five recommendations and supporting evidence give providers information to facilitate prudent care decisions and empower patients to actively participate in critical, honest conversations about their care, potentially reducing unnecessary health care spending and preventing harm.

Geriatric nephrology: a paradigm shift in the approach to renal replacement therapy.

The population of elderly individuals diagnosed with CKD continues to grow. Many have multiple comorbid conditions that will impact life expectancy as well as decisions about whether to pursue renal replacement therapy. Nephrologists are uniquely positioned to assist their patients and caregivers in this regard and spend considerable time counseling them about the benefits and risks associated with dialysis therapy. This article presents an overview of many of the issues facing nephrologists, and provides tools to assist busy clinicians in helping their elderly patients in deciding whether to consider dialysis or intensive, nondialysis care.

Tratamento da infecção pelo vírus da hepatite C em candidatos a transplante renal submetidos a diálise: impacto orçamentário no SUS / Hepatitis C treatment in patients on dialysis awaiting renal transplantation: a budget impact analysis

Trata-se de uma análise de impacto orçamentário derivada dos resultados do estudo "Custo-efetividade do tratamento da infecção pelo vírus da hepatite C em candidatos a transplante renal submetidos a diálise". Teve como objetivo estimar o impacto orçamentário da ampliação da oferta do tratamento da infecção pelo vírus da hepatite C (VHC) para candidatos a transplante renal. Para tal, foi construído um modelo de Markov, a fim de estimar o custo médio do tratamento de diferentes proporções da população-alvo. Foram estimados os casos prevalentes e incidentes da infecção na população em diálise, candidata a transplante renal, em um horizonte de tempo de dez anos. Com base nestas estimativas, foi calculado o valor a ser despendido pelo SUS para tratar a população-alvo em três cenários diferenciados, caracterizados pela proporção da população submetida ao tratamento. Os valores encontrados foram comparados com o gasto do sistema para garantia de terapias de substituição renal no período de um ano, identificando-se que o custo do tratamento de toda a população candidata a transplante, infectada pelo VHC, corresponde a 0,3 por cento do valor despendido com TRS pelo SUS.

This is an analyses of the budget impact derived from the cost-effectiveness study on the hepatitis C treatment in candidates for renal transplantation under dialysis. It aims to estimate the budget impact of an offer of hepatitis C treatment for all candidates for renal transplantation. A Markov model was developed to estimate the mean cost for treatment of distinct proportions of the target population. The prevalence and incidence of hepatitis C in the candidates for renal transplantation in the dialysis population was also estimated in a horizon of ten years. Based on these estimative, we calculate the amount needed for treatment of this population in three distinct scenarios characterized by a proportion of the population under treatment. The values were compared with the expense of the system to guarantee renal replacement therapies in one year, identifying the cost of treatment of all candidates for transplant, infected with HCV, corresponding to 0.3 percent of the amount spent with renal transplantation within the SUS.

Economic evaluation in clinical nephrology: part 2. Applying economic evaluations in clinical nephrology.

Our previous article described the principles of conducting an economic evaluation for evidence-based medical decision making. This article provides some tips for reading, critically appraising and applying the findings of an economic evaluation in clinical practice.

[DRG's and cost/efficacy indicators in nephrology and dialysis. Results at a hospital division]. / DRG e indicatori di costo/efficacia in nefrologia e dialisi. Risultati in una Divisione ospedaliera.

The Diagnosis Related Groups (DRGs) classification system correlates hospital performance with their relative costs and encourages more efficient productive processes. We report the following parameters: a) the distribution of hospital discharges according to the Major Diagnostic Categories (MDCs) and DRGs; b) the relationship between mean length of stay and threshold values; c) economic analysis of the cost-reimbursement pay-off. The results showed that 71.3% of DRGs belonged to nephro-urological MDC 11 and 28.7% in other internal MDCs (mainly involving cardiac and respiratory system). Of the latter, 67.7% were utilized for dialysis and transplant patients and kidney donors. In MDC 11 the most common DRGs were: the surgical DRG 315, produced by the vascular accesses for hemodialysis and by insertion of Tenckoff catheter for peritoneal dialysis, DRG 316 by cases of acute and chronic renal failure, DRG 332 by biopsy-proven glomerulonephritides. The length of stay was most commonly within range of one-third of threshold value for specific DRG; there was a low percentage of one-day stays and outlier cases. The economic analysis demonstrated that mean daily reimbursement sum was 590,714 ITL. Analysis of the overall costs yielded a mean daily cost of 455,838 ITL. In conclusion, quality indicators show that, appropriately, our specialist activity is largely devoted to the diagnosis and treatment of acute and chronic nephropathies and complications following dialysis and renal transplant.

Nephrologists as primary care providers: a review of the issues.

Considering the role of nephrologists as primary care providers for their chronic dialysis patients requires exploration of a number of factors. These factors include the definition of a primary care provider, the time and expertise needed to provide primary care, the expectations of nephrologists and dialysis patients who give and receive primary care, the appropriate preventive care for end-stage renal disease (ESRD) patients, and the current and future roles of nephrologists within a changing health care environment. Unfortunately, few studies have addressed these issues, and there is little objective information on which to base guidelines and recommendations about nephrologist-directed primary care of ESRD patients. Most nephrologists spend a significant portion (30% to 35%) of their time caring for dialysis patients, and 90% report providing primary care to dialysis patients. Most dialysis patients view their nephrologist as their primary care provider. The increasingly aged and ill ESRD population will undoubtedly necessitate additional time and expertise for care from an understaffed nephrology work force. The increased use of advanced practice nurses and alliances with health care delivery systems under global capitation programs may develop into effective strategies to provide care for an increasing population of dialysis patients. The nonnephrologic health care needs, including specific and appropriate cancer screening and preventive health care protocols for ESRD patients whose life expectancies are significantly less than the general population, are unclear. The issues involved in considering nephrologists as primary caregivers for ESRD patients include these and other related factors, and will be discussed in this review.

Avaliaçäo de procedimentos de alto custo em nefrologia e oncologia: em busca de resultados / High-costs procedures assessment in nephrology and oncology: search for results

Projeto que examina os procedimentos ambulatoriais de alto custo nas áreas de Nefrologia e Oncologia. Apresenta o perfil de produçäo das duas áreas, seguido por análise dos seus custos. Estabelece um cardápio para orientar a supervisäo das unidades ambulatoriais de Nefrologia e Oncologia, acompanhado por um "critério de boa prática" que possa conduzir processos avaliativos futuros. Tendo Minas Gerais como estudo de caso, delineia uma proposta de estudo avaliativo para as áreas de nefrologia e oncologia

Current Procedural Terminology (CPT) coded services provided by nurse specialists.

In this exploratory study, a random sample of nurses from nine nursing specialties was surveyed to identify which Current Procedural Terminology (CPT) coded procedures they perform and how frequently they perform them. CPT codes are used universally to file claims for physician payment. The sample included 74 school nurses, 67 enterostomal nurses, 53 family nurse practitioners, 43 critical care nurses, 43 oncology nurses, 40 rehabilitation nurses, 39 orthopaedic nurses, 34 nephrology nurses and 25 nurse-midwives. Specific questionnaires were developed for each specialty with codes identified by expert panels. The number of CPT codes ranged from 233 for family nurse practitioners to 58 for school nurses. The mean number of coded services performed by individual respondents ranged from 79 (FNP) to 18 (school nurses); individual respondents performed 0-162 codes. Supervision by physicians was very infrequent. Charges to Medicare in 1988 for the coded services included in the survey were $22,793,427.34 (aggregate allowable charges). The study provides some documentation of the degree to which nurses perform the same services and procedures for which physicians are being paid. If policy makers are serious about reaching innovative solutions to the problems of quality, access and cost, everything must be "on the table," including the contributions of nurses.