Search

Information derived from routinely collected real-world data has for a long time been used to support regulatory decision making on the safety of drugs and has more recently been used to support marketing authorization submissions to regulators. There is a lack of detailed information on the use and types of this real-world evidence (RWE) as submitted to regulators. We used resources held by the European Medicines Agency (EMA) to describe the characteristics of RWE included in new marketing authorization applications (MAAs) and extensions of indication (EOIs) for already authorized products submitted to the EMA in 2018 and 2019. For MAAs, 63 of 158 products (39.9%) contained RWE with a total of 117 studies. For 31.7% of these products, the RWE submitted was derived from data collected before the planned authorization. The most common data sources were registries (60.3%) followed by hospital data (31.7%). RWE was mainly included to support safety (87.3%) and efficacy (49.2%) with cohort studies being the most frequently used study design (88.9%). For EOIs, 28 of 153 products (18.3%) contained RWE with a total of 36 studies. For 57.1% of these products, studies were conducted prior to the EOIs. RWE sources were mainly registries (35.6%) and hospital data (27.0%). RWE was typically used to support safety (82.1%) and efficacy (53.6%). Cohort studies were the most commonly used study design (87.6%). We conclude that there is widespread use of RWE to support evaluation of MAAs and EOIs submitted to the EMA and identify areas where further research is required., (© 2021 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.)

This narrative review describes implementation, current status and perspectives of a pharmacogenomic (PGx) program at the Brazilian National Cancer Institute (INCA), targeting the cancer chemotherapeutic drugs - fluoropyrimidines, irinotecan and thiopurines. This initiative, designed as a research project, was supported by a grant from the Brazilian Ministry of Health. A dedicated task force developed standard operational procedures from recruitment of patients to creating PGx reports with dosing recommendations, which were successfully applied to test 100 gastrointestinal cancer INCA outpatients and 162 acute lymphoblastic leukemia pediatric patients from INCA and seven other hospitals. The program has been subsequently expanded to include gastrointestinal cancer patients from three additional cancer treatment centers. We anticipate implementation of routine pre-emptive PGx testing at INCA but acknowledge challenges associated with this transition, such as continuous financing support, availability of trained personnel, adoption of the PGx-informed prescription by the clinical staff and, ultimately, evidence of cost-effectiveness.

Several expedited regulatory review projects for innovative drugs and regenerative medical products have been developed in the US, the EU, and Japan. Each regulatory agency has elaborated an original regulatory framework and adopted regulatory projects developed by the other regulatory agencies. For example, the Food and Drug Administration (FDA) first developed the breakthrough therapy designation, and then the Pharmaceuticals and Medical Devices Agency (PMDA) and European Medicines Agency (EMA) introduced the Sakigake designation and the priority medicines (PRIME) designation, respectively. In addition, the necessity of the product being first development in Japan is the original feature of the Sakigake designation, while actively supporting the development of advanced-therapy medicinal products (AMTPs) by academia or small/medium-sized sponsors is the original feature of the PRIME; these particular features are different from the breakthrough therapy designation in the US. In this review article, flexible and expedited review processes for new drugs, and cell and gene therapies in the US, the EU, and Japan are described. Moreover, all the drugs and regenerative medical products that were granted conditional approval or Sakigake designation in Japan are listed and analyzed herein.

Within the last decade, One Health has gained momentum in government agencies, academic institutions, intergovernmental organisations and civil society organisations within Thailand. One Health is an approach of multisectoral and multidisciplinary collaboration that connects human, animal and environmental health sectors to improve the health of all. In 2014, the Coordinating Unit for One Health (CUOH), based within the Thai Ministry of Public Health, was officially established to serve as a focal point within Thailand for One Health collaboration both domestically and internationally. The unit's main functions are to share data, information and resources, and to support One Health activities within the country. The CUOH Steering Committee meets regularly to provide technical and administrative guidance on the functions and directions of the CUOH. A memorandum of understanding on One Health, signed in 2016 by seven Ministries and the Thai Red Cross Society, has strengthened One Health collaboration and provides a formalised channel through which the CUOH can facilitate activities across Ministries and with non-governmental agencies in Thailand.

Background: Under structural conditions of non-governability, most players in the policy arena in Israel turn to two main channels that have proven effective in promoting the policies they seek: the submission of petitions to the High Court of Justice and making legislative amendments through the Economic Arrangements Law initiated by the Ministry of Finance. Nevertheless, an analysis of the principal trends emerging from the High Court of Justice rulings and legislative amendments through the Economic Arrangements Law indicates that these channels are open to influence, primarily by forces that are essentially neo-liberal. Little is known about the effects of these trends on the right to healthcare services, which in Israel has not been legislated as an independent constitutional law in Basic Laws., Methods: We use four major legal cases decided by the Supreme Court of Israel in the past 10 years where the Court reviewed new legislative initiatives proposed by the Economic Arrangements Law in the area of healthcare. We utilize an institutional approach in our analysis., Results: A neo-institutional analysis of the legal cases demonstrates that petitions against the Economic Arrangements Law in the area of healthcare services have been denied, even though the Court uses strong rhetoric against that law and the government more generally in addressing issues that concern access to healthcare services and reforms in the healthcare system. This move strengthens the trend toward a neo-liberal public policy and significantly weakens the legal protection of the right to healthcare services., Conclusion: In deciding petitions against the Economic Arrangements Law in the area of healthcare, the Supreme Court allows the Ministry of Finance to be a dominant player in the formation of public policy. In doing so, it may be promoting a goal of strengthening its position as a political institution that aspires to increase the public's trust in the judiciary and especially in the Supreme Court itself, in addition to exercising judicial restraint and allowing more leeway to the executive and legislative branches more generally.

Objectives: To evaluate the current practice of companies and agencies to assess the changes made in aligning regulatory and health technology assessment (HTA) stakeholders; to identify areas of commonality of evidentiary requirements that could occur; and to identify strategic issues and trends of regulatory and HTA synergy., Methods: Two separate questionnaires were developed to assess stakeholders' perceptions on regulatory and HTA alignment, one for pharmaceutical companies and the other for regulatory and HTA agencies. The responses were analyzed using descriptive statistics., Results: Seven regulatory and 8 HTA agencies from Australia, Canada, and Europe and 19 international companies developing innovative medicine responded to the survey. This study provided a snapshot of the current regulatory and HTA landscape. Changes made over the past 5 years were reflected in three main areas: there is an increasing interaction between regulatory and HTA agencies; current conditional regulatory approvals are not always linked with flexible HTA approaches; and companies are more supportive of joint scientific advice. Four types of evidentiary requirements were identified as building blocks for better alignment: acceptable primary end points, inclusion of an active comparator, use of patient-reported outcomes, and choice and use of surrogate end point., Conclusions: The study showed that the gap between regulatory and HTA requirements has narrowed over the past 5 years. All respondents supported synergy between regulatory and HTA stakeholders, and the study provided several recommendations on how to further improve evidentiary alignment including the provision of joint scientific advice, which was rated as a key strategy by both agencies and companies., (Copyright © 2018 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Until the late 1990s, the Taiwan Tobacco and Liquor Corporation (TTL) focused almost exclusively on serving the domestic market as a highly protected monopoly. This paper describes how the company has adopted a more outward looking strategy since 2000, with ambitions to become a regional, and eventually global, business by 2021. Drawing on company documents and industry sources, the paper argues that this shift in strategy was a direct reaction to the decline in domestic market share following liberalisation of the Taiwanese tobacco market and adoption of tougher domestic tobacco control measures. Market opening occurred as a result of pressure from the U.S. Trade Representative in the 1980s, as well as World Trade Organization membership in 2002. It is argued that TTL's efforts to globalise operations have been limited by bureaucratic company management and structures, and ongoing political tension between Taiwan and China. However, the relative success of TTL's alcohol branch, and potential détente as the Taiwanese government reaches out to improve relations with China, may provide TTL with new opportunities to achieve its goal of becoming a regional player with global ambitions. This article is part of the special issue 'The Emergence of Asian Tobacco Companies: Implications for Global Health Governance.'

The Richmond County Mosquito Control program's mission statement is to incorporate strategies of integrated mosquito control management that are effective, practical, and environmentally safe and protect the health of Richmond County residents, as well as promote public education, in order to prevent large mosquito populations and the diseases that they transmit. To this end, the program coordinates efforts with other county agencies in order to provide better service. This is a small program with limited resources, so in an effort to provide better integrated mosquito management, the mosquito control program and the Phinizy Center for Water Sciences joined efforts to trap mosquitoes at sites across the county, identify the species, and send the mosquitoes off for viral testing. These data help determine locations of disease-carrying mosquitoes so the county can more efficiently control the mosquito populations and reduce the risk of West Nile virus transmission.

Health economics refers to the analysis of medical institutions considering their economic and social efficacy, but also the regularity and the relationships that govern the phenomena and the processes from the field of health with the final purpose of achieving better results with the minimum of resources; it represents the study of health price in its complexity. The economics of the population's health needs and in particular the health needs in case of the poor groups of the population, consider health to be the main component of global human vulnerability. Health economics tries to change the simple interpretation of health price and disease cost into a wider consideration of a system administration similar to educational and social economics and the study of health in the context of the multiple specializations of the macro economy of the national group, as it is an instrument in the country's great economics symphony.

Context: Public health practitioners, policy makers, and researchers alike have called for more data on individual worker's perceptions about workplace environment, job satisfaction, and training needs for a quarter of a century. The Public Health Workforce Interests and Needs Survey (PH WINS) was created to answer that call., Objective: Characterize key components of the public health workforce, including demographics, workplace environment, perceptions about national trends, and perceived training needs., Design: A nationally representative survey of central office employees at state health agencies (SHAs) was conducted in 2014. Approximately 25,000 e-mail invitations to a Web-based survey were sent out to public health staff in 37 states, based on a stratified sampling approach. Balanced repeated replication weights were used to account for the complex sampling design., Setting and Participants: A total of 10,246 permanently employed SHA central office employees participated in PH WINS (46% response rate)., Main Outcome Measures: Perceptions about training needs; workplace environment and job satisfaction; national initiatives and trends; and demographics., Results: Although the majority of staff said they were somewhat or very satisfied with their job (79%; 95% confidence interval [CI], 78-80), as well as their organization (65%; 95% CI, 64-66), more than 42% (95% CI, 41-43) were considering leaving their organization in the next year or retiring before 2020; 4% of those were considering leaving for another job elsewhere in governmental public health. The majority of public health staff at SHA central offices are female (72%; 95% CI, 71-73), non-Hispanic white (70%; 95% CI, 69-71), and older than 40 years (73%; 95% CI, 72-74). The greatest training needs include influencing policy development, preparing a budget, and training related to the social determinants of health., Conclusions: PH WINS represents the first nationally representative survey of SHA employees. It holds significant potential to help answer previously unaddressed questions in public health workforce research and provides actionable findings for SHA leaders.

Structured frameworks for benefit-risk analysis in drug licensing decisions are being implemented across a number of regulatory agencies worldwide. The aim of these frameworks is to aid the analysis and communication of the benefit-risk assessment throughout the development, evaluation, and supervision of medicines. In this review, authors from regulatory agencies, pharmaceutical companies, and academia share their views on the different frameworks and discuss future directions., (© 2015 American Society for Clinical Pharmacology and Therapeutics.)

Selection of a practice setting affects duty hours, practice autonomy, institutional relationships, administrative responsibilities, personal finances, and professional satisfaction. To identify national trends in practice settings reported by the College, we evaluated responses by Fellows (and Junior Fellows) in active practice from six College surveys on professional liability issues between 1992 and 2012. Although half of Fellows remained in an independent private practice, a decline was observed among solo health care practitioners (from 32% in 1992 to 19% in 2012). Direct employment increased either in hospital systems (from 5% to 15%) or as academic faculty (from 9% to 12%). The proportion of Fellows employed by either health maintenance organizations (from 4% to 2%) or the government (2%) remained low. We anticipate that practice settings will be increasingly influenced by health care reform, team-based care with use of nonphysician clinicians, physician age, and increasing subspecialization. Future surveys of Fellows about their practice settings, preferably required at the time of Maintenance of Certification, will aid in evaluating practice settings and their influence on quality of care, cost containment, and health care provider satisfaction.

Objective: To determine maternal fetal medicine (MFM) referral trends in a Medicaid population over time., Study Design: Sixteen clinical guidelines and 23 clinical conditions were identified where co-management/consultation with MFM specialist is recommended. Linked Medicaid claims and birth certificate data for 2001-2006 were used to identify pregnancies with these conditions and whether they received co-management/consultation from a MFM specialist., Results: Between 2001 and 2006, there were 108,703 pregnancies with delivery of 110,890 neonates. Forty-five percent had one or more of the conditions identified for co-management/consultation. Overall pregnancies receiving MFM contact remained unchanged at 22.2% in 2001 and 22.1% in 2006. However, face to face contacts decreased from 14.6% (2001) to 8.7% (2006) while telemedicine consults increased from 7.6% (2001) to 13.3% (2006). Health departments were most likely and family practitioners least likely to refer to MFM (p<0.001). Pregnancy complications leading to MFM referrals include cardiac complications, renal disease, systemic disorders, PPROM, suspected fetal abnormalities, and cervical insufficiency., Conclusion: Referral of high-risk pregnancies to MFMs varies with the level of expertise at the primary prenatal site. Increased contact between MFMs and local providers increased MFM referrals.

Despite widespread enthusiasm for broader participation in health policy and programming, little is known about the ways in which multi-sector groups address the challenges that arise in pursuing this goal. Based on the experience of Peru's National Multi-sector Health Coordinating Body (CONAMUSA), this article characterizes these challenges and identifies organizational strategies the group has adopted to overcome them. Comprising nine government ministries, nongovernmental organizations, academia, religious institutions, and international cooperation agencies, CONAMUSA has faced three principal challenges: 1) selecting representatives, 2) balancing membership and leadership across sectors, and 3) negotiating role transition and conflict. In response, the group has instituted a rotation system for formal leadership responsibiliti es, and professionalized management functions; created electoral systems for civil society; and developed conflict of interest guidelines. This case study offers lessons for other countries trying to configure multi-sector groups, and for donors who mandate their creation, tempering unbridled idealism toward inclusive participation with a dose of healthy realism and practical adaptation.

The link between federal housing policy and public health has been understood since the nineteenth century, when housing activists first sought to abolish slums and create healthful environments. This article describes how the Obama administration-building on these efforts and those that followed, including the Great Society programs of President Lyndon Johnson-has adopted a cross-sector approach that takes health considerations into account when formulating housing and community development policy. The federal Department of Housing and Urban Development fully embraces this "health in all policies" approach. Nonetheless, the administration's strategy faces challenges, including fiscal and political ones. Some of these challenges may be overcome by conducting quality research on how housing and community development policies affect health outcomes, and by developing a federal budget strategy that takes into account how investments in one sector contribute to cost savings in another.

The German Agency for Quality in Medicine (ÄZQ) was established as a joint institution of the German Medical Association (BÄK) and the National Association of Statutory Health Insurance Physicians (KBV) in 1995. Starting as a small quality assurance co-ordination unit of the German physicians' national self-governmental bodies, ÄZQ has been developed during the last decade into a centre of excellence for clinical practice guidelines, patient information, patient safety, evidence-based medicine, and knowledge management. The article summarises the institution's acitivities over the period of 15 years with respect to its national projects and international collaboration, being a founding member of the Guidelines International Network. In the future ÄZQ's programme priorities will be in the fields of knowledge transfer and implementation of medical decision aids into the daily work of health-care providers., (© Georg Thieme Verlag KG Stuttgart · New York.)

September 11 and the subsequent anthrax attacks marked the beginning of significant investment by the federal government to develop a national public health emergency response capability. Recognizing the importance of the public health sector's contribution to the burgeoning homeland security enterprise, this investment was intended to convey a "dual benefit" by strengthening the overall public health infrastructure while building preparedness capabilities. In many instances, federal funds were used successfully for preparedness activities. For example, electronic health information networks, a Strategic National Stockpile, and increased interagency cooperation have all contributed to creating a more robust and prepared enterprise. Additionally, the knowledge of rarely seen or forgotten pathogens has been regenerated through newly established public health learning consortia, which, too, have strengthened relationships between the practice and academic communities. Balancing traditional public health roles with new preparedness responsibilities heightened public health's visibility, but it also presented significant complexities, including expanded lines of reporting and unremitting inflows of new guidance documents. Currently, a rapidly diminishing public health infrastructure at the state and local levels as a result of federal budget cuts and a poor economy serve as significant barriers to sustaining these nascent federal public health preparedness efforts. Sustaining these improvements will require enhanced coordination, collaboration, and planning across the homeland security enterprise; an infusion of innovation and leadership; and sustained transformative investment for governmental public health.

Updated version of National Institute of Health Sciences Computer Network System (NIHS-NET) is described. In order to reduce its electric power consumption, the main server system was newly built using the virtual machine technology. The service that each machine provided in the previous network system should be maintained as much as possible. Thus, the individual server was constructed for each service, because a virtual server often show decrement in its performance as compared with a physical server. As a result, though the number of virtual servers was increased and the network communication became complicated among the servers, the conventional service was able to be maintained, and security level was able to be rather improved, along with saving electrical powers. The updated NIHS-NET bears multiple security countermeasures. To maximal use of these measures, awareness for the network security by all users is expected.

The formation of the first Gerontopole in Toulouse was a response to a mission letter sent by French Ministers of Health on February 2007. The mission of the Toulouse Gerontopole is based around three major axes: 1) To facilitate the access of frail elderly people to innovative therapy and clinical research: the Gérontopôle set up the national network for clinical investigation into Alzheimer's disease (AD) funded through the CeNGEPS (National Centre For Management of Trials on Health Products) calls for proposals since July 2008. In addition, the Gérontopôle coordinates several national clinical trials with promising drugs with potential effect on the mechanisms and evolution of AD and actively participates in studies on biomarkers; 2) To develop health promotion actions and prevention trials for healthy elderly people, through the Institute of Aging: the Gérontopôle has implemented the GuidAge (Phase III trial concerning the efficiency of Ginkgo Biloba on the impact and delay of appearance of an Alzheimer type dementia) and MAPT (Multi-domain Alzheimer Preventive Trial) studies on prevention of AD and cognitive decline. It is curently working on the new generation of preventive trials based on biomarkers; 3) To develop clinical research for dependant elderly people, through the implementation of the REHPA research network including 240 nursing homes in France. In December 2009, additional grants were delivered by the French government to extend the three research axes for two more years, and establish a charter of quality for geriatric care in relation with the administration and relevant agencies.

The European Medicines Agency (EMA) is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union (EU). Since 2005, the agency has become responsible for the approval of all new oncology drugs in the EU. In this article we describe the mission, role, and responsibilities of the EMA, and provide a brief summary of recent initiatives related to cancer drug regulation. The EMA recently published its Road Map to 2015. Over the next 5 years, the agency aims to continue to stimulate drug development in areas of unmet medical needs. Concerning drug safety, one of the priorities over the next few years will be to establish a more proactive approach in ensuring patient safety. This is the result of new EU legislation coming into force in 2012 that will strengthen the way the safety of medicines for human use is monitored in the EU. In terms of its general operation, the agency is committed to increased openness and transparency, and to build on its interactions with stakeholders, including members of academia, health care professionals, patients, and health technology assessment bodies. The agency recently created an oncology working party to expand the current guideline for the development and evaluation of cancer drugs. The guideline focuses on both exploratory and confirmatory studies for different types of agents. The current revision will address a number of topics, including the use of biomarkers as an integrated part of drug development and the use of progression-free survival as a primary endpoint in registration trials., (©2011 AACR.)

Authors draw attention to their own experience about the occurrence and epidemic measures of tuberculosis in two micro-regions of Hungary, Fadd and Józsefváros situated far from each other. According to data from the National Statistical Office of Hungary, tuberculosis does not belong to the most significant causes of morbidity and mortality. However, it still represents a notable factor in national healthcare and public health. Currently, tuberculosis is not an important issue to be regulated by health care decision makers or at the level of legislation. Based on their own experience, authors suggest reconsidering the possibility of epidemic measures for the prevention of tuberculosis.

The CLP Regulation introduces the criteria of the UN Globally Harmonised System of Classification and Labelling (UN GHS) in the EU. The European Chemicals Agency (ECHA) manages the CLP related tasks - such as harmonised classification and labelling, handling requests for alternative names and maintaining the Classification & Labelling Inventory (C&L) - to ensure consistent implementation in the EU. The obligations for industry depend on their role in the supply chain. Manufacturers and importers have to notify to ECHA the identity and classification and labelling of substances within one month of placing them on the market either on their own or in a mixture, and regardless of the quantitity. As of 3 January 2011 ECHA has received some 3.1 million notifications of over 107 000 substances. This information is stored in the C&L Inventory and accessible to Member State Competent Authorities. The non-confidential information will be made publicly available on ECHA's website in 2011.