Evaluating high-cost technologies - no need to throw the baby out with the bathwater.

INTRODUCTION: Evidence generation for the health technology assessment (HTA) of a new technology is a long and expensive process with no guarantees that the health technology will be adopted and implemented into a health-care system. This would suggest that there is a greater risk of failure for a company developing a high-cost technology and therefore incentives (such as increasing the funding available for research or additional market exclusivity) may be needed to encourage development of such technologies as has been seen with many high-cost orphan drugs. AREAS COVERED: This paper discusses some of the key issues relating to the evaluation of high-cost technologies through the use of existing HTA processes and what the challenges will be going forward. EXPERT OPINION: We propose that while the current HTA process is robust, its evolution into accommodating the incorporation of real-world data and evidence alongside a life-cycle HTA approach should better enable developers to produce the evidence required on effectiveness and cost-effectiveness. This should lead to reduced decision uncertainty for HTA agencies to make adoption decisions in a more timely and efficient manner. Furthermore, budget impact analysis remains important in understanding the actual financial impact on health-care systems and budgets outside of the cost-effectiveness framework used to aid decision-making.

Atuação do enfermeiro auditor nos processos de órteses e próteses e materiais especiais / The work of the nursing auditor in orthetic processes and prostheses and special materials / El trabajo del auditor de enfermería en procesos y prótesis ortéticos y materiales especiales

Objetivou-se descrever o papel do enfermeiro auditor nos processos que envolvem Órtese, Prótese e Material Especial (OPME) em ambiente hospitalar público ou privado. Trata-se de um estudo teórico-reflexivo que propõe uma discussão sobre os processos de OPME em território brasileiro, assim como as legislações vigentes, tanto na saúde suplementar quanto no atendimento gratuito e universal praticado pelo Sistema Único de Saúde. Há uma necessidade de profissionais capacitados, com competência técnica e zelo pela segurança, para otimização no controle destes materiais, além de inibir práticas duvidosas e fraudulentas. Contribuindo com o controle e rastreabilidade dos materiais utilizados, garantindo o serviço prestado ao usuário e o monitoramento de qualidade do mesmo. Deste modo, conclui-se que a atuação do Enfermeiro Auditor nos processos de uso de OPME é primordial para que sejam cumpridas e respeitadas, conforme as normatizações e legislações vigentes, todas as etapas do processo, tanto nas instituições públicas ou privadas. Constatou-se também que poucas são as publicações científicas em âmbito nacional a respeito da temática.(AU)

The aim was to describe the role of the nurse auditor in the processes involving OPME in a public or private hospital environment. It is a theoretical-reflective study that proposes a discussion on the OPME processes in Brazilian territory, as well as the current legislation, both in supplementary health and in free and universal care practiced by the Unified Health System. There is a need for professionals trained, with technical competence and zeal for safety, to optimize the control of these materials, in addition to inhibiting doubtful and fraudulent practices. Contributing to the strict control and traceability of these inputs used, guaranteeing the service provided to the user and monitoring its quality. Therefore, it can be concluded that the role of the Nurse Auditor in the processes of use of OPME is essential for all stages of the process to be complied with and respected, in accordance with current regulations and legislation, both in public or private institutions. It was also found that there are few scientific publications nationwide on the subject.(AU)

El objetivo era describir el papel del auditor de enfermería en los procesos que involucran a OPME en un entorno hospitalario público o privado. Es un estudio teórico-reflexivo que propone una discusión sobre los procesos de OPME en el territorio brasileño, así como la legislación actual, tanto en salud complementaria como en atención gratuita y universal practicada por el Sistema Único de Salud. Se necesitan profesionales. capacitados, con competencia técnica y celo por la seguridad, para optimizar el control de estos materiales, además de inhibir prácticas dudosas y fraudulentas. Contribuyendo al estricto control y trazabilidad de estas entradas utilizadas, garantizando el servicio prestado al usuario y monitoreando su calidad. Por lo tanto, se puede concluir que el papel de la Enfermera Auditora en los procesos de uso de OPME es esencial para que se cumplan y respeten todas las etapas del proceso, de acuerdo con las regulaciones y la legislación vigente, tanto en instituciones públicas como privadas. También se descubrió que hay pocas publicaciones científicas en todo el país sobre el tema.(AU)

The different clinical guideline standards in Brazil: High cost treatment diseases versus poverty-related diseases.

Each year, evidence-based clinical guidelines gain more space in the health professionals' practice and in services organization. Due to the scarcity of scientific publications focused on diseases of poverty, the development of well-founded clinical guidelines becomes more and more important. In view of that, this paper aims to evaluate the quality of Brazilian guidelines for those diseases. The AGREE II method was used to evaluate 16 guidelines for poverty-related diseases (PRD) and 16 guidelines for global diseases whose treatment require high-cost technologies (HCD), with the ultimate aim of comparing the results. It was found that, in general, the guideline development quality standard is higher for the HCD guidelines than for the PRD guidelines, with emphasis on the "rigour of development" (48% and 7%) and "editorial independence" (43% and 1%) domains, respectively, which had the greatest discrepancies. The HCD guidelines showed results close to or above international averages, whereas the PRD guidelines showed lower results in the 6 domains evaluated. It can be concluded that clinical protocol development priorities need some redirecting in order to qualify the guidelines that define the healthcare organization and the care of vulnerable populations.

Surviving niche busters: Main strategies employed by Canadian private insurers facing the arrival of high cost specialty drugs.

The Canadian patchwork system of prescription drug coverage and the employer sponsored private health benefits group plans appear vulnerable to cost growth due to insufficient balance of power between fragmented public and private buyers, and pharmaceutical manufacturers. The emergence of "bad" insurance risks caused by new and very expensive treatments featuring high cost specialty medicines - also known as niche buster drugs - exposes this vulnerability. This study fills a gap in knowledge by seeking to better understanding how Canadian private insurers face the arrival of specialty pharmaceuticals. It completes an overview of a body of grey literature composed of publicly available online articles from the employment benefits and group insurance consulting and administration industry; online documents from group benefits sector conferences; and online or on demand materials from Canadian life and health insurers. Claims for high cost specialty drugs generate new bad insurance risks that Canadian health insurers attempt to mitigate through isolated corporate initiatives, industry-wide strategies and calls for universal, public catastrophic coverage. The outcomes of these strategies are limited cost-control measures as well as risk and cost transfers onto plan sponsors, patients and provincial public programs.

"Do not do" recommendations of the working groups of the Spanish Society of Intensive and Critical Care Medicine and Coronary Units (SEMICYUC) for the management of critically ill patients. / Recomendaciones de "no hacer" en el tratamiento de los pacientes críticos de los grupos de trabajo de la Sociedad Española de Medicina Intensiva, Crítica y Unidades Coronarias (SEMICYUC).

The project "Commitment to Quality of Scientific Societies", promoted since 2013 by the Spanish Ministry of Health, seeks to reduce unnecessary health interventions that have not proven effective, have little or doubtful effectiveness, or are not cost-effective. The objective is to establish the "do not do" recommendations for the management of critically ill patients. A panel of experts from the 13 working groups (WGs) of the Spanish Society of Intensive and Critical Care Medicine and Coronary Units (SEMICYUC) was selected and nominated by virtue of clinical expertise and/or scientific experience to carry out the recommendations. Available scientific literature in the management of adult critically ill patients from 2000 to 2017 was extracted. The clinical evidence was discussed and summarized by the experts in the course of consensus finding of each WG, and was finally approved by the WGs after an extensive internal review process carried out during the first semester of 2017. A total of 65 recommendations were developed, of which 5 corresponded to each of the 13 WGs. These recommendations are based on the opinion of experts and scientific knowledge, and aim to reduce those treatments or procedures that do not add value to the care process; avoid the exposure of critical patients to potential risks; and improve the adequacy of health resources.

Concise Review: The High Cost of High Tech Medicine: Planning Ahead for Market Access.

Cellular therapies and other regenerative medicines are emerging as potentially transformative additions to modern medicine, but likely at a staggering financial cost. Public health care systems' budgets are already strained by growing and aging populations, and many private insurer's budgets are equally stretched. The current systems that most payers employ to manage their cash flow are not structured to absorb a sudden onslaught of very expensive prescriptions for a large portion of their covered population. Despite this, developers of new regenerative medicines tend to focus on the demands of regulators, not payers, in order to be compliant throughout the clinical trials phases, and to develop a product that ultimately will be approvable. It is not advisable to assume that an approved product will automatically become a reimbursed product, as examples from current practice in hematopoietic stem cell transplantation in the U.S. demonstrate; similarly, in Europe numerous Advanced-therapy Medicinal Products achieved market authorization but failed to secure reimbursement (e.g., Glybera, Provenge, ChondroCelect, MACI). There are however strategies and approaches that developers can employ throughout clinical development, in order to generate clinical and health economic data which will be necessary to demonstrate the value proposition of the new product and help ensure market access for patients; furthermore, performance based managed entry agreements coupled with post-launch evidence generation can help overcome challenges around product uncertainty at launch and reduce market access delays. Stem Cells Translational Medicine 2017;6:1723-1729.

Mobile Health Devices as Tools for Worldwide Cardiovascular Risk Reduction and Disease Management.

We examined evidence on whether mobile health (mHealth) tools, including interactive voice response calls, short message service, or text messaging, and smartphones, can improve lifestyle behaviors and management related to cardiovascular diseases throughout the world. We conducted a state-of-the-art review and literature synthesis of peer-reviewed and gray literature published since 2004. The review prioritized randomized trials and studies focused on cardiovascular diseases and risk factors, but included other reports when they represented the best available evidence. The search emphasized reports on the potential benefits of mHealth interventions implemented in low- and middle-income countries. Interactive voice response and short message service interventions can improve cardiovascular preventive care in developed countries by addressing risk factors including weight, smoking, and physical activity. Interactive voice response and short message service-based interventions for cardiovascular disease management also have shown benefits with respect to hypertension management, hospital readmissions, and diabetic glycemic control. Multimodal interventions including Web-based communication with clinicians and mHealth-enabled clinical monitoring with feedback also have shown benefits. The evidence regarding the potential benefits of interventions using smartphones and social media is still developing. Studies of mHealth interventions have been conducted in >30 low- and middle-income countries, and evidence to date suggests that programs are feasible and may improve medication adherence and disease outcomes. Emerging evidence suggests that mHealth interventions may improve cardiovascular-related lifestyle behaviors and disease management. Next-generation mHealth programs developed worldwide should be based on evidence-based behavioral theories and incorporate advances in artificial intelligence for adapting systems automatically to patients' unique and changing needs.

Teledermatology: from historical perspective to emerging techniques of the modern era: part II: Emerging technologies in teledermatology, limitations and future directions.

Telemedicine is the use of telecommunications technology to support health care at a distance. Dermatology relies on visual cues that are easily captured by imaging technologies, making it ideally suited for this care model. Advances in telecommunications technology have made it possible to deliver high-quality skin care when patient and provider are separated by both time and space. Most recently, mobile devices that connect users through cellular data networks have enabled teledermatologists to instantly communicate with primary care providers throughout the world. The availability of teledermoscopy provides an additional layer of visual information to enhance the quality of teleconsultations. Teledermatopathology has become increasingly feasible because of advances in digitization of entire microscopic slides and robot-assisted microscopy. Barriers to additional expansion of these services include underdeveloped infrastructure in remote regions, fragmented electronic medical records, and varying degrees of reimbursement. Teleconsultants also confront special legal and ethical challenges as they work toward building a global network of practicing physicians.

[Prostate cancer: what treatment techniques for which tumors? Ethical and methodological issues]. / Cancers de prostate : quelles techniques de radiothérapie pour quelles tumeurs ? Enjeux éthiques et méthodologiques.

The identification of the optimal radiation technique in prostate cancer is based on the results of dosimetric and clinical studies, although there are almost no randomized studies comparing different radiation techniques. The feasibility of the techniques depends also on the technical and human resources of the radiation department, on the cost of the treatment from the points of view of the society, the patient and the radiation oncologist, and finally on the choice of the patient. The slow evolution of prostate cancer leads to consider the biochemical failure as the main judgment criteria in the majority of the studies. A proper urinary radio-induced toxicity evaluation implies a long follow-up. Intensity-modulated radiotherapy (IMRT) combined with image-guided radiotherapy (IGRT) is recommended in case of high dose (≥76Gy) to the prostate, pelvic lymph nodes irradiation and hypofractionation schedules. For low-risk tumors, the aim of the treatment is to preserve quality of life, while limiting costs. Stereotactic body radiotherapy shows promising results, although the follow-up is still limited and phase III trials are ongoing. Focal radiation techniques are in the step of feasibility. For intermediate and high-risk tumors, the objective of the treatment is to increase the locoregional control, while limiting the toxicity. IMRT combined with IGRT leads to either a well-validated dose escalation strategy for intermediate risk tumors, or to a strategy of moderate hypofractionated schedules, which cannot be yet considered as a standard treatment. These combined radiation techniques allow finally large lymph node target volume irradiation and dose escalation potentially in the dominant intraprostatic lesion. The feasibility of simultaneous integrated boost approaches is demonstrated.

[Intensity-modulated radiotherapy in head and neck cancer: ethics and methodology]. / Radiothérapie conformationnelle avec modulation d'intensité des cancers des voies aérodigestives supérieures : enjeux éthiques et méthodologiques.

Numerous studies have shown that intensity-modulated radiation therapy is the standard technique for the radiation treatment of head and neck cancers. Intensity-modulated radiation therapy reduces side effects (xerostomia, dysphagia, fibrosis, etc.) and improves the results for cancer localizations with highly complex shapes such as the cavum or nasal cavity. Intensity-modulated radiation therapy is also a costly technique that necessitates a numerous staff, highly trained, with regular practice. If this technique cannot be available (understaffing, overwork, etc.) the choice between entrusting the patient to a colleague and treating the patient with a less sophisticated technique such as 3-dimensional conformal radiation therapy depends on different objective and ethical criteria.

[Brachytherapy in France: current situation and economic outlook due to the unavailability of iridium wires]. / Curiethérapie en France : état des lieux et perspectives économiques après l'arrêt de la commercialisation des fils d'iridium.

In 2013, about 6000 patients were treated with brachytherapy, the number diminishing by 2.6% per year since 2008. Prostate, breast and gynecological cancers are the most common types of cancers. Since 2008, the number of brachytherapy facilities has decreased by 18%. In medicoeconomic terms, brachytherapy faces many problems: the coding system is outdated; brachytherapy treatments cost as much as internal radiation; fees do not cover costs; since iridium wire has disappeared from the market, the technique will be transferred to more expensive high-speed or pulse dose rates. The French financing grid based on the national study of costs lags behind changes in such treatments and in the best of cases, hospitals resorting to alternatives such as in-hospital brachytherapy are funded at 46% of their additional costs. Brachytherapy is a reference technique. With intense pressure on hospital pricing, financing brachytherapy facilities will become even more problematic as a consequence of the disappearance of iridium 192 wires. The case of brachytherapy illustrates the limits of the French financing system and raises serious doubts as to its responsiveness.

[CT scanners in the Brazilian Unified National Health System: installed capacity and utilization]. / Capacidade de produção e grau de utilização de tomógrafo computadorizado no Sistema Único de Saúde.

This study analyzes the supply and use of computed tomography scanners (CT) in the Brazilian Unified National Health System (SUS) according to State and administrative levels in the year 2009. Secondary data were used to estimate the installed CT capacity in public healthcare facilities and in private services outsourced by the SUS and calculated the rate of utilization. Average national CT utilization was less than 10%. The public sector showed lower CT use than the private sector outsourced by the SUS. The number of CT tests in the SUS was less than half the number produced in developed countries. The results thus suggest the need for further studies on management practices with high-technology equipment in order to improve allocation of current and future public resources in supplying CT tests.

Capacidade de produção e grau de utilização de tomógrafo computadorizado no Sistema Único de Saúde / CT scanners in the Brazilian Unified National Health System: installed capacity and utilization / Capacidad de producción y grado de utilización del tomógrafo computarizado en el Sistema Único de Salud brasileño

O objetivo deste estudo foi analisar a distribuição da oferta da tomografia computadorizada e o grau de utilização do tomógrafo computadorizado (TC) no Sistema Único de Saúde (SUS), por estado e esfera administrativa, no ano de 2009. Utilizando dados secundários, estimou-se a capacidade de produção dos tomógrafos públicos e privados disponíveis ao SUS e calculou-se o grau de utilização dos mesmos. Os resultados mostraram que o grau de utilização dos TCs dos prestadores SUS possui uma média nacional abaixo de 10% e que o setor público tem o menor grau de utilização do TC, em comparação com o setor privado conveniado ao SUS. Foi observado que a produção de exames por TC no SUS é menor do que a metade da produção obtida em países desenvolvidos. Assim, os resultados sugerem a necessidade de um aprofundamento nos estudos das práticas de gestão tecnológica desses equipamentos, uma vez que é possível uma melhor alocação dos recursos públicos necessários à produção de tomografias computadorizadas.

This study analyzes the supply and use of computed tomography scanners (CT) in the Brazilian Unified National Health System (SUS) according to State and administrative levels in the year 2009. Secondary data were used to estimate the installed CT capacity in public healthcare facilities and in private services outsourced by the SUS and calculated the rate of utilization. Average national CT utilization was less than 10%. The public sector showed lower CT use than the private sector outsourced by the SUS. The number of CT tests in the SUS was less than half the number produced in developed countries. The results thus suggest the need for further studies on management practices with high-technology equipment in order to improve allocation of current and future public resources in supplying CT tests.

El objetivo de este estudio fue analizar la distribución de la oferta de tomografía computarizada y el grado de utilización del tomógrafo computarizado (TC) en el Sistema Único de Salud (SUS) brasileño, en los diferentes estados y niveles administrativos, durante 2009. Se utilizaron datos secundarios, se evaluó la capacidad de producción de tomógrafos públicos y privados, disponibles para el SUS, y se calculó el grado de utilización de los mismos. Los resultados mostraron que la utilización de los tomógrafos contratados por el SUS tienen un promedio nacional menor al 10% y que el sector público tiene un menor grado de utilización del TC, en comparación con el sector privado contratado por el SUS. Se observó que la producción de exámenes por TC en el SUS es menor que la mitad de la producción observada en los países desarrollados. De esta forma, los resultados sugieren la necesidad de profundizar en las investigaciones sobre prácticas de gestión de esos equipos, ya que puede ser posible una mejor utilización de los recursos públicos necesarios para la producción de tomografías computarizadas.

[Clinical and economical comparison between in-house (Make) and outsourcing (Buy) management of the cardiac catheterization laboratory from two high-volume diagnostic and interventional centers: immediate and 6-month results]. / Confronto clinico ed economico tra gestione aziendale (Make) ed esternalizzata (Buy) del laboratorio di emodinamica di due centri ad elevato volume di procedure diagnostiche ed interventistiche coronariche: risultati immediati e a 6 mesi.

BACKGROUND: Percutaneous coronary interventions (PCI) are widespread procedures in the Italian Healthcare System, but concerns are raised about their economic sustainability. In the last decade, public hospitals have outsourced the PCI services (building and maintaining the technological instruments and the personnel) "buying" them from private companies (Buy) rather than building and maintaining them through public expenditure (Make). The aim of this study was to compare the economic and clinical impact of these two management solutions (Buy and Make) in two community hospitals located in the Turin metropolitan area (Italy). METHODS: We conducted: 1) a quantitative assessment in order to compare differences in the economic impact between Buy and Make for providing PCI; 2) a qualitative assessment comparing the clinical characteristics of two inpatient populations undergoing PCI and then analyzing the efficacy of the procedure in-hospital and at 6-month follow-up. RESULTS: Between January and June 2010, a total of 332 patients underwent PCI at the "degli Infermi" Hospital in Rivoli and 340 at the "Maria Vittoria" Hospital in Turin (Italy). There were no significant differences between the two populations neither about the clinical characteristics nor in procedural efficacy (either immediate or at follow-up). For 600 units of diagnostic-therapeutic pathway, the net present value at a discount rate of 3.5% of the Make project is higher than that of the Buy by €278.402,25, and is therefore the less convenient of the two solutions. The Buy solution is still the more convenient of the two at volumes <700 units. CONCLUSIONS: Our findings show that the Buy solution, if tailored to the specific local needs, provides access to sophisticated technology without making worse quality of services and may save capital expenditure below 700 PCI/years.

[Hospital-based health technology assessment in France: how to proceed to evaluate innovative medical devices?]. / Évaluation des technologies de santé en milieu hospitalier : quelle organisation pour évaluer et acquérir des dispositifs médicaux innovants ?

Innovative medical devices offer solutions to medical problems and greatly improve patients' outcomes. Like National Health Technology Assessment (HTA) agencies, hospitals face numerous requests for innovative and costly medical devices. To help local decision-makers, different approaches of hospital-based HTA (HB-HTA) have been adopted worldwide. The objective of the present paper is to explore HB-HTA models for adopting innovative medical devices in France and elsewhere. Four different models have been conceptualized: "ambassador" model, "mini-HTA" model, "HTA unit" model and "internal committee". Apparently, "HTA unit" and "internal committee" (or a mixture of both models) are the prevailing HB-HTA models in France. Nevertheless, some weaknesses of these models have been pointed out in previous works. Only few examples involving hospital pharmacists have been found abroad, except in France and in Italy. Finally, the harmonization of the assessment of innovative medical devices in France needs a better understanding of HB-HTA practices.