Knowing me, knowing you: the evolution of HTA practice and approaches from the perspectives of HTA Agencies and pharmaceutical industry

Health Technology Assessment (HTA) has emerged as an important tool to support healthcare decision-makers to make rational reimbursement decisions, with the ultimate purpose of promoting an efficient healthcare system. HTA agencies continue to review their methodology and refine processes and procedures to improve their practice. For pharmaceutical companies, HTA needs to be embedded from development to jurisdictional submission to HTA agencies. Interactions between HTA agencies and companies through the form of early scientific advice have been increasingly used to support evidence generation during development, in order to improve market access. Multi-stakeholder interactions between regulator, HTA agencies and companies need to be further mapped out and evaluated to assess the current experience, uptake, and value of such activities. This research is aimed to evaluate the HTA practice of pharmaceutical companies to enable better decision-making during development and at launch, examine the processes and performance of HTA agencies, and promote good practice across both stakeholders through self-improvement and interactions. The finding in this thesis has demonstrated a continuous evolution of HTA agencies throughout product lifecycles to support drug development, improve their methodology and processes, and engage in interaction with regulators and peer HTA agencies. By establishing a systematic framework to benchmark the organization and milestone performance of HTA agencies, we provided a baseline and tool to assess the evolvement of HTA. We also observed a mindset change within companies to embed HTA considerations during drug development, in order to improve the jurisdictional submission and proactively promote good HTA practice. Future opportunities for research can be built on this thesis in the context of lifecycle HTA. For example, indicators can be established to measure the interactions between agencies through development to review and assessment of new medicine. The agency benchmarking framework can be utilised as a foundation for capacity building for emerging HTA agencies, tracking and improve performance metrics and ensuring a good practice of HTA

Toward effective health technology regulation

New health technologies offer both challenges and opportunities. Regulation is one mechanism to help balance the benefits and risks of new health technologies. This thesis examines the extent to which ‘good’ health technology regulation is achieved and the effectiveness of the policy measures regulators (and others) employ to meet such aims. To accomplish these objectives, a conceptual framework of ‘good regulation’ based on the academic and practitioner literatures was developed and its various dimensions considered and explored across eight different studies. Taken together, the studies provide an analysis of the roles, processes, policies, and performance of the regulators responsible for the market authorisation and coverage and reimbursement of pharmaceuticals and medical devices in Europe and the US; the role and use of technology assessment in health technology regulation and its impact on attaining good regulation; and, the factors that impact regulatory performance. The thesis demonstrates that attaining good health technology regulation is complex and challenging, because of the inherent uncertainty regarding the benefits and risks of new technologies, their growing diversity and complexity, the limitations of existing study designs and assessment methods, the increased demands placed on regulators to meet sometimes conflicting objectives, and the underlying political nature of making decisions about public access to and financing of new health technologies. Regulators have made progress on addressing these challenges. However, additional improvements are needed to improve health technology regulatory performance. Like much of health care policy, movement toward achieving the various criteria of good regulation will be incremental, especially considering the often step-wise nature of technological innovation

Clinical trials in children: Equity, quality and relevance

This thesis investigates the equity, quality and relevance of clinical trials in children to inform better evidence-based child healthcare and outcomes worldwide. A comprehensive review of the literature revealed that despite current initiatives to encourage more trials in children, there is still a paucity of safety and efficacy data of many medicines prescribed in this population. An analysis of trials registered in children showed that disease burden was moderately correlated to trials and this scarcity was particularly prevalent in low-and middle-income countries. We explored the contributory factors to this inequity by conducting a systematic review of stakeholders’ views of trials in children in low-and middle-income countries. In the study evaluating the completeness of protocols of trials in children submitted to ethics committees, we found that protocols are generally comprehensive, but many key domains in trial design and conduct are not reported. Key-informant trial stakeholders who were interviewed proposed strategies to improve trials such as addressing the unique needs of children, embedding trials as part of routine clinical care and streamlining regulatory approvals. Increasing international collaboration, establishing sustainable centralised trials infrastructure, and aligning research to child health priorities were proposed to encourage more high-quality trials that address global child healthcare needs

Clinical trials in children: Equity, quality and relevance

This thesis investigates the equity, quality and relevance of clinical trials in children to inform better evidence-based child healthcare and outcomes worldwide. A comprehensive review of the literature revealed that despite current initiatives to encourage more trials in children, there is still a paucity of safety and efficacy data of many medicines prescribed in this population. An analysis of trials registered in children showed that disease burden was moderately correlated to trials and this scarcity was particularly prevalent in low-and middle-income countries. We explored the contributory factors to this inequity by conducting a systematic review of stakeholders’ views of trials in children in low-and middle-income countries. In the study evaluating the completeness of protocols of trials in children submitted to ethics committees, we found that protocols are generally comprehensive, but many key domains in trial design and conduct are not reported. Key-informant trial stakeholders who were interviewed proposed strategies to improve trials such as addressing the unique needs of children, embedding trials as part of routine clinical care and streamlining regulatory approvals. Increasing international collaboration, establishing sustainable centralised trials infrastructure, and aligning research to child health priorities were proposed to encourage more high-quality trials that address global child healthcare needs

Health Care Costs and the Arc of Innovation

Health care costs continue their inexorable rise, threatening America’s long-term fiscal stability, competitiveness, and standard of living. Over the past half-century, efforts to rein in spending have uniformly failed. In this Article, we explain why, breaking with standard accounts of regulatory and market dysfunction. We point instead to the nexus of economics, mutual empathy, and social expectations that drives medical innovation and locks in low-value technologies. We show how law reflects and reinforces this nexus and how and why health-policy-makers avert their gaze. Next, we propose to circumvent these barriers instead of surmounting them. Rather than targeting today’s excessive spending, we seek to leverage available legal tools to bend the arc of innovation, away from marginally-beneficial technology and toward high-value advances. To this end, we set forth a novel, value-based approach to pricing and patent protection—one that departs sharply from current practice by rewarding innovators in proportion to the therapeutic benefits new tests and treatments yield. Using cancer therapy as an example, we explain how emerging information technology and large troves of electronic clinical data are opening the way to near-real-time assessment of efficacy. We then show how such assessment can power ongoing adjustment of pricing and patent terms. Finally, we offer a blueprint for how laws governing health care payment and intellectual property can be tailored to realize this value-focused vision. For the reasons we lay out, the transformation of incentives we urge will both slow clinical spending growth and greatly enhance the social value that this spending yields

Advisory Committee on Immunization Practices (ACIP) summary report : June 20-21, 2018, Atlanta, Georgia

Publication date from document properties.min-2018-06.pdf2018704

The future of early stage and growth finance in Northern Ireland, Report to the Department for Enterprise, Trade and Investment (DETI)

SQW Ltd (SQW), working alongside academics from Glasgow University and Middlesex University were appointed by the Department for Enterprise, Trade and Investment (DETI) in April 2014 to undertake research into early stage and growth finance in Northern Ireland. The purpose of the study was to provide guidance on the development of early stage and growth finance, focused particularly on angel investment and venture capital, and drawing on the experience of other ‘successful’ locations internationally. This Synthesis Report sets out the key findings and messages from the research, and the guidance – covering a set of principles, strategic recommendations, and specific actions – that should inform the development of early stage and growth finance going forward in Northern Ireland

An expanded role for clinical coordinators in investigator initiated clinical trial research

Clinical research is conducted to advance human medicine by developing efficacious treatments and improving patient outcomes when new therapies are developed and implemented. Clinical trials are a subset of the types of clinical research conducted on human volunteers in the development of new drugs, devices and other therapies. Prior to the start of a trial, a country’s regulatory authority must review the trial to ensure it is scientifically and ethically sound. In Canada, the regulatory authority is Health Canada. The International Conference on Harmonization (ICH) of technical requirements for the registration of pharmaceutics for humans aims to provide ethical and scientific quality standards for design, conduct, data collection and reporting in clinical trials. The Good Clinical Practice (GCP) Guidelines were created by the ICH Steering Committee to assure the public that rights, safety and well being of subjects are protected according to the Declaration of Helsinki, and the clinical data obtained in a ICH/GCP compliant clinical trial will meet regulatory requirements. Health Canada has adopted the ICH/GCP Guidelines and therefore, in Canada, all clinical trials involving humans must comply with these Guidelines. The clinical trial coordinator is an important and central position on the research team executing many trial duties and communications. Regulatory authorities, Research Ethics Boards and the sponsor, overlook the role and responsibilities of a highly trained clinical coordinator, despite their vital and central position. The GCP Guidelines also fail to address the role and responsibilities of a clinical coordinator. Disconnect between guidelines, regulatory expectations and actual trial conduct provides an apparent need to formalize and clearly define the role and scope of a clinical coordinator. The Registered Nurse (RN) brings professionalism, knowledge, skill and a holistic perspective to the expanded role of a clinical coordinator and to the clinical trial. Highly trained health professionals are capable of assuming more responsibilities and executing clinical trial design, setup and management as compared to the traditional administrative roles of the clinical coordinator. The expanded role of the clinical coordinator is especially beneficial for Principal Investigator initiated trials due to limited research personnel and resources. Postoperative adhesions are a common complication following pelvic surgery, therefore, this clinical trial is relevant and a response to a healthcare need. My graduate studies focused on the development and set up of the clinical trial Protocol ADE002-2013 Phase I Trial of L-Alanyl-L-Glutamine for the Reduction of Peritoneal Adhesions in Adult Females Undergoing Myomectomy. My thesis is a discussion of general Canadian clinical trial research information followed by an explanation of how we executed the information to design and set up our PI initiated clinical trial. The value of the expanded role of the clinical coordinator as a member of the research team will also be discussed

Public infrastructure: inquiry report

There is an urgent need to comprehensively overhaul processes for assessing and developing public infrastructure projects, argues this report. Volume 1 contains the overview, recommendations and findings, chapters 1 to 8 and volume 1 references . It covers infrastructure provision, funding and financing. Volume 2 contains chapters 9 to 16, appendices A to J and volume 2 references. It considers the scope for reducing the cost of public infrastructure and the necessary steps to implement the reforms recommended in both volumes of the report. The series of appendices provide case studies and further detail on some issues. Key points: There is an urgent need to comprehensively overhaul processes for assessing and developing public infrastructure projects. There are numerous examples of poor value for money arising from inadequate project selection, potentially costing Australia billions of dollars. Additional spending under the status quo will simply increase the cost to users, taxpayers, the community generally, and lead to more wasteful infrastructure. Reliance on the notion of an infrastructure deficit, too, could encourage poor investment choices. It is essential to reform governance and institutional arrangements for public infrastructure to promote better decision making in project selection, funding, financing and the delivery of services from new and existing infrastructure. Well-designed user charges should be used to the fullest extent that can be economically justified. However, governments will have to continue to fully or partly fund some infrastructure projects and address equity issues. Significant institutional and longer-term road pricing arrangements will create more direct links to road users, taking advantage of advances in vehicle technology. Private sector involvement in infrastructure provision and/or financing delivers efficiency gains only if well designed and well implemented. Private financing is not a \u27magic pudding\u27 — ultimately users and/or taxpayers must foot the bill. Government guarantees and tax concessions are not costless and often involve poorly understood risks. Governments will have some capacity to fund more projects than under current fiscal and debt management practices, provided the reform package in this report is implemented to ensure the selection of projects with strong net benefits. Data problems limit analysis and benchmarking. A coordinated and coherent data collection process will address this and improve future project selection decisions. Nevertheless, there is evidence of recent significant increases in the costs of constructing major public infrastructure in Australia. Elevated labour costs due to the mining construction boom has been one factor, but no single input has played a decisive role in cost increases. Until recently, labour productivity growth in the construction sector generally has been sluggish. There is no conclusive evidence that Australian levels of productivity in construction are significantly different from other developed countries. The industrial relations environment in the construction industry remains problematic, mainly in general rather than civil construction, with the problems much greater for some sites, unions and states. Governments can use their procurement policies to drive reform, and penalties for unlawful conduct should rise. Despite significant concentration in the market for large public infrastructure projects, the market appears to be workably competitive today, though a few simple measures would make it more so and would reduce the cost pressures facing procurers. There is significant scope to improve public sector procurement practices and lower bid costs for tenderers, with potentially large benefits for project costs and timin