Heterogeneity in treatment effects across diverse populations

Differences in patient characteristics, including age, sex, and race influence the safety and effectiveness of drugs, biologic products, and medical devices. Here we provide a summary of the topics discussed during the opening panel at the 2018 Johns Hopkins Center for Excellence in Regulatory Science and Innovation symposium on Assessing and Communicating Heterogeneity of Treatment Effects for Patient Subpopulations: Challenges and Opportunities. The goal of this session was to provide a brief overview of FDA-regulated therapeutics, including drugs, biologics and medical devices, and some of the major sources of heterogeneity of treatment effects (HTE) related to patient demographics, such as age, sex and race. The panel discussed the US Food and Drug Administration's role in reviewing and regulating drugs, devices, and biologic products and the challenges associated with ensuring that diverse patient populations benefit from these therapeutics. Ultimately, ensuring diverse demographic inclusion in clinical trials, and designing basic and clinical research studies to account for the intended patient population's age, sex, race, and genetic factors among other characteristics, will lead to better, safer therapies for diverse patient populations.     

The Patient Transit Assistance Scheme: A case for social work advocacy!

Abstract

The Patient Transit Assistance Scheme is one of Queensland Health's initiatives designed to facilitate equity of access for Queensland residents to essential health care services. The purpose of the scheme is to help all Queenslanders, irrespective of where they live, to have access to specialist medical services. It provides direct financial assistance to patients and in some cases their carers, to facilitate access to specialist medical services irrespective of geographic location.

The findings of recent research conducted on social worker involvement with PTAS for patients diagnosed with leukaemia and associated haematological disorders will be used to argue that the problems in relation to the administration of this scheme require serious attention at the level of patient care and health policy.     

Spinal Cord Injury

ABSTRACT

This article provides an overview of spinal cord injury (SCI) that is useful and informative for social workers and other health care professionals who work with this population. Social workers new to the specialty of spinal cord injury must expand their knowledge base of this chronic injury. Social workers contribute to the rehabilitation process through assessment, education, and discharge planning. This article also may be used to inform persons with spinal cord injury and their families and to encourage them to engage in dialogue about SCI in the earliest stages of treatment and rehabilitation.     

“互联网+医疗健康”背景下考虑患者反馈机制的药品质量监管策略研究

药品质量关乎公众生命健康、国民经济发展、社会稳定和国家安全,一直以来都是政府和全社会关注的热点问题之一。"互联网+医疗健康"背景下药品流通速度加快使得药品安全问题影响范围扩大,因此,建立患者反馈机制是促进互联网药品质量监管的有效方式。考虑患者反馈机制下如何设计互联网药品质量监管策略的问题,基于演化博弈理论,构建了政府部门、药品生产商、医药电商和患者参与的药品质量监管四方演化博弈模型,通过分析各博弈主体策略选择的稳定性,并基于Lyapunov第一法则对复制动态系统十六种均衡点进行稳定性分析,研究了促进药品生产商提供高质量药品的政府监管策略和患者反馈机制,并利用Matlab 2017仿真分析了各关键要素对策略演化的影响。研究结果表明:1)患者倾向于失真评价或投诉率较低时,医药电商更倾向于选择药品质量不检测,不利于互联网药品质量安全;2)患者失真评价下,不存在药品生产商提供高质量药品的稳定策略组合,而真实评价下有效的反馈机制和监管机制可使互联网药品质量安全具有稳定性;3)提高患者评价(正面评价或负面评价)带给医药电商和药品生产商的声誉价值增加或声誉损失,将会使药品生产商选择提供高质量药品做为...     

Human Dose–Response Data for Francisella tularensis and a Dose‐ and Time‐Dependent Mathematical Model of Early‐Phase Fever Associated with Tularemia After Inhalation Exposure

Military health risk assessors, medical planners, operational planners, and defense system developers require knowledge of human responses to doses of biothreat agents to support force health protection and chemical, biological, radiological, nuclear (CBRN) defense missions. This article reviews extensive data from 118 human volunteers administered aerosols of the bacterial agent Francisella tularensis , strain Schu S4, which causes tularemia. The data set includes incidence of early‐phase febrile illness following administration of well‐characterized inhaled doses of F. tularensis . Supplemental data on human body temperature profiles over time available from de‐identified case reports is also presented. A unified, logically consistent model of early‐phase febrile illness is described as a lognormal dose–response function for febrile illness linked with a stochastic time profile of fever. Three parameters are estimated from the human data to describe the time profile: incubation period or onset time for fever; rise time of fever; and near‐maximum body temperature. Inhaled dose‐dependence and variability are characterized for each of the three parameters. These parameters enable a stochastic model for the response of an exposed population through incorporation of individual‐by‐individual variability by drawing random samples from the statistical distributions of these three parameters for each individual. This model provides risk assessors and medical decisionmakers reliable representations of the predicted health impacts of early‐phase febrile illness for as long as one week after aerosol exposures of human populations to F. tularensis .     

患者自主决定权与医疗干涉权的；中突与解决

患者的自主决定权和医疗干涉权在现实中确实存在着冲突,但是二者又有着伦理的一致性,都是为了实现患者的最佳利益。在正确认识二者冲突的基础上,通过加强立法完善对民事行为能力的界定,建立第三方裁决机构提前快速审查机制,谨慎对待关系患者生命的患者自主决定权,通过立法明确界定医疗干涉权的行使范围等对策来化解二者的冲突,兼顾医患双方的权利。     

让医学回归人性

医学是一门关于人的科学,以解除疾病给人们带来的痛苦为目的,其本质是人性的,即以人为本的。但后来随着科学主义、技术至善主义价值观的张扬,医学领域出现了技术化、市场化、医患关系物化的倾向,这与医学的人性本质相偏离。至今依然普遍存在,找回人性的医学,需要采取加快推进医药卫生制度的改革、加强医院管理和建立医务人员的医德评价、奖惩机制、强化医德教育等措施,但这些举措要建立在合理的价值选择的基础上,并体现和保证合理价值规范的实现。尊重病人自主,这一蕴含丰厚的人性底蕴的医学伦理原则是当今让医学回到人性的重要合理价值选择之一,全面、正确理解并认真贯彻、落实这一原则尤为必要。     

Design and Analysis of Hospital Admission Control for Operational Effectiveness

Variability in hospital occupancy negatively impacts the cost and quality of patient care delivery through increased emergency department (ED) congestion, emergency blockages and diversions, elective cancelations, backlogs in ancillary services, overstaffing, and understaffing. Controlling inpatient admissions can effectively reduce variability in hospital occupancy to mitigate these problems. Currently there are two major gateways for admission to a hospital: the ED and scheduled elective admission. Unfortunately, in highly utilized hospitals, excessive wait times make the scheduled gateway undesirable or infeasible for a subset of patients and doctors. As a result, this group often uses the ED gateway as a means to gain admission to the hospital. To better serve these patients and improve overall hospital functioning, we propose creating a third gateway: an expedited patient care queue. We first characterize an optimal admission threshold policy using controls on the scheduled and expedited gateways for a new Markov decision process model. We then present a practical policy based on insight from the analytical model that yields reduced emergency blockages, cancelations, and off‐unit census via simulation based on historical hospital data.     

The Effect of Integrated Scheduling and Capacity Policies on Clinical Efficiency

In outpatient healthcare clinics, capacity, patient flow, and scheduling are rarely managed in an integrated fashion, so a question of interest is whether clinic performance can be improved if the policies that guide these decisions are set jointly. Despite the potential importance of this issue, we find surprisingly few studies that look at how the allocation of capacity, paired with various appointment scheduling policies and different patient flow configurations, affects patient flow and clinical efficiency. In this paper, we develop an empirically based discrete‐event simulation to examine the interactions between patient appointment policies and capacity allocation policies (i.e., the number of available examination rooms) and how they jointly affect various performance measures, such as resource utilization and patient waiting time. Findings suggest that scheduling lower‐variance, shorter appointments earlier in the clinic (and, conversely, higher‐variance, longer appointments later) results in less overall patient waiting without reducing physician utilization or increasing clinic duration. Additionally, exam rooms exhibited classic bottleneck behavior: there was no effect on physician utilization by adding exam rooms beyond a certain threshold, but too few exam rooms were devastating to clinic throughput. Some significant interactions between these variables were observed, but were not influential to the level of managerial concern. Clinicians' intuition about managing capacity in healthcare settings may differ substantially from best policies.     

医患关系的变化与医院的可持续发展

医疗体制改革的深入和市场经济的发展使医患关系发生了巨大变化 ,医患关系变成了法律或合同关系、经营者与消费者关系 ,患者已从过去的求医变成了现在的选择医生。如何应对这些变化 ?作者认为 ,医务人员应通过转变观念、依法行医、给患者提供优质服务等来建立市场经济条件下的融洽、相互信赖与尊重的新型医患关系