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Most new medicines entering the market are high‐cost speciality drugs. These drugs can cost tens to hundreds of thousands of dollars per course of treatment and in some cases millions of dollars per dose. Approximately half of all spending on medicines is projected to target only 2–3% of patients, raising important questions about resource allocation. While there is no doubt that breakthrough innovations have transformed clinical care in some disciplines, it is also true that cost is becoming one of the primary barriers to treatment access and that many new medicines do not provide value commensurate with their prices. This article examines pricing trends, the reasons for high prices and their implications for access and clinical practice. [ABSTRACT FROM AUTHOR]

There is a paucity of public discussion of costs spent on drug trials during coronavirus disease 2019 (COVID‐19) and their value, and of large public outlay on research funding for vaccine and drug development that did not deliver medicines nor vaccines for Australians. This oversight left us at the behest of global supply chains, politics and commercial cost‐plus pricing for vaccines. It is possible that these outcomes were the result of some major cognitive biases and the failure of a clinical pharmacologist's voice in the leadership teams. Biases included unawareness of the complexities of taking interesting chemicals in vitro to development into therapeutic use that can be tolerated, show efficacy and have appropriate disposition in humans; lack of a systems approach to therapeutic development; and an understanding of the relevance and translatability of pharmacology, physiology and clinical drug development. We believe that reflecting on and addressing these biases will help Australia reposition itself better with a therapeutics and clinical trial strategy for future pandemics, built into the strategy of a Centre for Disease Control. [ABSTRACT FROM AUTHOR]

Background: In Australia, facilitated regulatory pathways (FRPs) became available with the introduction of priority review (PR) in 2017 and provisional approval (PA) in 2018, which aim to facilitate expedited review and approval for novel medicines. The pathways were developed in consultation with a wide range of stakeholders and have since been utilised by pharmaceutical companies for various therapeutic products. However, the perceptions of the firsthand users of these pathways have not been evaluated in Australia. Objectives: We have conducted a survey of Australian regulatory professionals aiming to solicit the perceived benefits, barriers to utilisation, shortcomings and proposed modifications to utilising these pathways. We have also solicited the users' perspective on key aspects of the pathways, including overall satisfaction, regulatory burden, availability and ease of use of guidelines, regulator support, impact on company strategy and recommendations for improvement. Methods: A survey was developed and distributed to Australian regulatory professionals from the pharmaceutical industry who had submission experience of new medicine applications via either PR, PA or the standard registration pathway to the Therapeutic Goods Administration (TGA). The questionnaire consisted of 44 questions with a skip logic and the option for free text comments. Results: We received responses from 16/42 companies that had utilised these new pathways. Nine respondents had experience with the PR pathway and ten with the PA pathway. The respondents were generally satisfied with the effectiveness of the PR process in expediting registration approvals, but they were ambivalent towards the PA pathway in terms of overall satisfaction and timelines. Respondents expressed a desire for further improvements in the speed of approval, earlier access for patients across various pathways and introduction of new Health Technology Assessment processes for medicines approved under PA. Conclusion: While the FRPs have been an important and positive development in the Australian regulatory landscape, there remain opportunities for further improvements, some of which have been highlighted by this study and may help inform future regulatory decisions. [ABSTRACT FROM AUTHOR]

Jack Jumper ant venom allergy is a uniquely Australian medical issue. The stinging ant is a leading cause of insect venom allergy in south‐eastern Australia. An effective venom immunotherapy‐based treatment was successfully developed by the Tasmanian Jack Jumper Allergy Research group. This paper provides a synopsis of our 25 years' research journey in developing this evidence‐based treatment modality. [ABSTRACT FROM AUTHOR]

Regulators and pharmaceutical companies across the world are intensifying efforts to get increasingly complex and innovative drugs to patients with high unmet medical need in the shortest possible time frame. This article reviews pathways to expedite drug development and approval available in member countries of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use and Australia. It is concluded that the increasing availability of expedited regulatory pathways and associated modernisation of regulatory systems changes the current regulatory paradigm and requires sponsors to rethink drug development and regulatory strategy. A transformation of the current sequence of regulatory submissions, favouring those countries/collaborations that are best regulatory equipped to make innovative medical need drugs available to patients in the shortest time frame is imminent. [ABSTRACT FROM AUTHOR]

Drug policy has been subjected to much scrutiny from different stakeholder groups who present sometimes very different opinions on solutions to address a problem. Reconciling such differences, that are underpinned by both anecdotal and empirical evidence, is a priority yet to be fully achieved. In this study, we examine: (i) whether differences exist among actor groups regarding their perceived importance in policymaking; (ii) whether those who dominate the decision process value the input of laypeople or minority actors; and (iii) whether differences exist, with regard to i and ii, between study sites (i.e. Australia and Hong Kong) that have markedly different drug strategies. Overall, differential preference weightings between dominant groups reveal a strong influence of political orientation. Results suggest that decisions made by health professionals were regarded as the most important, while most of the respondent groups gave a low preference toward policy decisions made by law enforcers. Dominant groups also tend to discount the inputs of laypeople and other minority groups. Our study highlights the importance for policy actor groups to consider such a differential weighting of inputs when synthesising knowledge and gathering opinions for the development of drug policy. [ABSTRACT FROM AUTHOR]

Introduction: Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers' costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method: An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results: A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer's costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers' costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer's costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion: Despite the attention given to manufacturers' costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs. [ABSTRACT FROM AUTHOR]

An innovative approach to patient management, evidence-based policy development, and clinical drug trials is required to provide personalized care and to improve the likelihood of finding an effective treatment for Motor Neurone Disease (MND). The MiNDAus Partnership builds on and extends existing national collaborations in a targeted approach to improve the standard and coordination of care for people living with MND in Australia, and to enhance the prospects of discovering a cure or treatment. Relationships have been developed between leading clinical and research groups as well as patient-centered organizations, care providers, and philanthropy with a shared vision. MiNDAus has established a corporate structure and meets at least biannually to decide on how best to progress research, drug development, and patient management. The key themes are; (i) empowering patients and their family carers to engage in self-management and ensure personalized service provision, treatment, and policy development, (ii) integration of data collection so as to better inform policy development, (iii) unifying patients and carers with advocacy groups, funding bodies, clinicians and academic institutions so as to inform policy development and research, (iv) coordination of research efforts and development of standardized national infrastructure for conducting innovative clinical MND trials that can be harmonized within Australia and with international trials consortia. Such a collaborative approach is required across stakeholders in order to develop innovative management guidelines, underpinned by necessary and evidence-based policy change recommendations, which, will ensure the best patient care until a cure is discovered. [ABSTRACT FROM AUTHOR]

Summary: Background. Previous research focused on the clinical evidence supporting new cancer drugs' initial US Food and Drug Administration (FDA) approval. However, targeted drugs are increasingly approved for supplementary indications of unknown evidence and benefit. Objectives. To examine the clinical trial evidence supporting new targeted cancer drugs' initial and supplementary indication approval in the US, EU, Canada, and Australia. Data and Methods. 25 cancer drugs across 100 indications were identified with FDA approval between 2009–2019. Data on regulatory approval and clinical trials were extracted from the FDA, European Medicines Agency (EMA), Health Canada (HC), Australian Therapeutic Goods Administration (TGA), and clinicaltrials.gov. Regional variations were compared with χ2-tests. Multivariate logistic regressions compared characteristics of initial and supplementary indication approvals, reporting adjusted odds ratios (AOR) with 95% confidence intervals (CI). Results. Out of 100 considered cancer indications, the FDA approved 96, the EMA 92, HC 86, and the TGA 83 (83%, p < 0.05). The FDA more frequently granted priority review, conditional approval, and orphan designations than other agencies. Initial approvals were more likely to receive conditional / accelerated approval (AOR: 2.69, 95%CI [1.07–6.77], p < 0.05), an orphan designation (AOR: 3.32, 95%CI [1.38–8.00], p < 0.01), be under priority review (AOR: 2.60, 95%CI [1.17–5.78], p < 0.05), and be monotherapies (AOR: 5.91, 95%CI [1.14–30.65], p < 0.05) than supplementary indications. Initial indications' pivotal trials tended to be shorter (AOR per month: 0.96, 95%CI [0.93–0.99], p < 0.05), of lower phase design (AOR per clinical phase: 0.28, 95%CI [0.09–0.85], p < 0.05), and enroll more patients (AOR per 100 patients: 1.19, 95%CI [1.01–1.39], p < 0.05). Conclusions. Targeted cancer drugs are increasingly approved for multiple indications of varying clinical benefit. Drugs are first approved as monotherapies in rare diseases with a high unmet need. Whilst expedited regulatory review incentivizes this prioritization, indication-specific safety, efficacy, and pricing policies are necessary to reflect each indication's differential clinical and economic value. [ABSTRACT FROM AUTHOR]

Background: The Pharmaceutical Medicine program at the University of New South Wales provides postgraduate education for students seeking employment relating to development of new medicines and medical technology. The objective of this study was to determine which changes to the program were required to meet future educational needs of students. Methods: Responses to questions from 76 students, tutors, lecturers, and stakeholders were obtained via Qualtrics surveys. Results: Most respondents would recommend the program because of scope and program emphasis, content, quality of teaching, flexibility, and availability of distance and online content. Students valued links to industry and the national regulatory authority. Respondents recommended program content be current, expanded to meet needs of the Asia-Pacific region, and structured to ensure opportunities for networking and collaboration. Conclusions: Recommended changes to the program are being implemented to optimize skills and knowledge of graduates for roles in global pharmaceutical, medical technology, and biotechnology industries. [ABSTRACT FROM AUTHOR]

The Asia Pacific (AP) region is diverse and dynamic. It comprises over 40 economies, with about 24 markets that are relatively more active in terms of promulgating and implementing regulations that impact the biopharmaceutical industry and advance drug development. Although many country- and territory-specific regulations, often driven by the local economy and market dynamics, exist, the overall trend in the AP region is toward international harmonization and global convergence. More markets are now participating in the International Council on Harmonization (ICH), and there is growing interest in identifying and incorporating best practices from competent regulatory agencies. These include expedited pathways to speed access to new medicines, and incentives and other means to encourage innovation. In this paper, we provide a summary of recent activities in select AP markets. [ABSTRACT FROM AUTHOR]

Background Under-reporting of adverse drug reactions (ADRs) by healthcare professionals is prevalent worldwide. Community pharmacists are the most frequently visited healthcare professional and are well placed to document ADRs as a part of their routine practice. Objective To measure community pharmacists' knowledge and perspectives towards ADR reporting and their reporting practices. Setting Community pharmacists in the New South Wales, Queensland, Victoria and Tasmania, Australia. Method A survey tool consisting of 28 items was developed, piloted and validated by a panel of expert reviewers. The final anonymised survey was distributed online to community pharmacists. Exploratory factor analysis and Cronbach's alpha were used to measure the validity and reliability of the tool, respectively. Non-parametric statistical tests were used to analyse knowledge, perspectives and ADR reporting practices.Main Outcome Measures: Knowledge, perceived importance, enablers and barriers to reporting ADRs. Results The survey tool showed acceptable validity and reliability. A total of 232 respondents completed the survey. The median knowledge score was 5 out of 10 (interquartile range, 2). Less than a third of respondents (31.0%) reported sufficient knowledge and training on ADR reporting. Only 35.3% of pharmacists reported at least one ADR in the previous 12 months. Non-reporting pharmacists were more likely to report lack of time as a barrier (P < 0.001), conversely they were more likely to report if the practice was remunerated (P = 0.007). Conclusion Under-reporting of ADRs by community pharmacists is highly prevalent. Initiatives to educate and train them on ADR reporting and simplifying the reporting process may improve reporting practices. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Abstract: Aim: Precision oncology involves molecularly matching patients to targeted agents usually in early drug development (EDD) programs. Molecular profiling (MP) identifies actionable targets. Comprehensive commercial MP platforms are costly and in resource limited environments, a more practical approach to MP is necessary to support EDD and precision oncology. We adopted a clinician‐directed, tailored approach to MP to enrol patients onto molecularly targeted trials. We report the feasibility of this approach. Methods: All patients referred to the Royal Melbourne Hospital (RMH) EDD between September 2013 and September 2015 were identified in a prospective database. Key captured data included clinicopathological data, MP platform ordered (if any), molecular targets identified and subsequent enrolment onto clinical trials. EDD‐clinician decisions to order MP and the platform utilized was guided by patient consultation, tumor type, trial availability and requirement for molecular information. Results: We identified 377 patients referred to RMH EDD. A total of 216 (57%) had MP ordered. The remainder had known actionable targets (19%), or were inappropriate for clinical trials (24%). In those undergoing MP, 187 genetic aberrations were found in 113 patients with 98 considered actionable targets in 86 patients. Ninety‐eight (25%) patients were enrolled onto a clinical trial, including 40 (11%) receiving molecularly matched treatments. Median progression‐free survival was improved in patients enrolled onto molecularly matched trials compared to those on unmatched trials (3.6 months vs 1.9 months, HR 0.58 [0.38–0.89], P  =  0.013). Conclusion: A clinician‐directed, tailored approach to the use of MP is feasible, resulting in 11% of patients enrolled onto molecularly matched trials. [ABSTRACT FROM AUTHOR]

Australian and US military medical services have collaborated since World War II to minimize vector-borne diseases such as malaria, dengue, and scrub typhus. In this review, collaboration over the last 30 years is discussed. The collaborative projects and exchange scientist programs have resulted in mutually beneficial outcomes in the fields of drug development and personal protection measures against vector-borne diseases. [ABSTRACT FROM AUTHOR]

The author discusses updates on issues related to the marketing of drugs to society in clinical trials. Topics mentioned include the drug acquisition component of the cost of pharmaceutical clinical trials, a concept of penetration pricing developed by economist Joel Dean, and the rising costs of anti-cancer drugs in Australia.

Concepts of addiction differ across time and place. This article is based on an international research project currently exploring this variation and change in concepts of addiction, in particular in the field of alcohol and other drug (AOD) use. Taking AOD policy in Australia and Canada as its empirical focus, and in-depth interviews with policy makers, service providers and advocates in each country as its key method (N=60), the article compares the addiction concepts articulated by professionals working in each setting. Drawing on Bruno Latour's theoretical work on the body and his proposal for a better science based on the 'articulation of differences', it explores the accounts of addiction offered across the Australian and Canadian project sites, identifying a shared dynamic in all: the juggling of difference and unity in discussions of the nature of addiction, its composite parts and how best to respond to it. The article maps two simultaneous trajectories in the data - one moving towards difference in participants' insistence on the multitude and diversity of factors that make up addiction problems and solutions, and the other towards unity in their tendency to return to narrow disease models of addiction in uncomfortable, sometimes dissonant, strategic choices. As I will argue, the AOD professionals interviewed for my project operate in two modes treated as distinct in Latour's proposal: in turning to reifying disease labels of addiction they take for granted, and work within, a 'universe of essences', but in articulating the multiplicity and diversity of addiction, they grope towards a vision of a 'multiverse of habits'. The article concludes by addressing this tension directly, scrutinising its practical implications for the development of policy and delivery of services in the future, asking how new thinking, and therefore new opportunities, might be allowed to emerge. [ABSTRACT FROM AUTHOR]

Funding rare disease therapies presents a challenge in Australia where there is a legislative requirement to consider cost-effectiveness. Currently the Life Saving Drugs Programme (LSDP) provides subsidised access to high-cost therapies for rare, life-threatening conditions. However the LSDP is currently under review by the Minsiter for Health and future access to rare disease therapies in uncertain. Internationally there is no gold standard model to evaluate and fund rare disease therapies, and considerable variation exists. However, common features of international systems include the opportunity for early stakeholder engagement, flexibility with evidence requirements, cost-effectiveness criteria and transparency in relation to the decision making framework and outcomes. Australians value equality and equal opportunity in relation to health care. To meet these expectations there is a clear need to maintain a separate fit-for-purpose framework to evaluate and fund rare disease therapies drawing on overseas best practice. This will provide certainty for industry to continue to invest in such treatments, as well as ensuring funding recommendations are reflective of Australian values balanced against the need for financial sustainability. [ABSTRACT FROM AUTHOR]