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Biomedical researchers are increasingly turning to project-based online fundraising (i.e., crowdfunding) as a complementary source of research funding. To help inform the fundraising strategies adopted by researchers who take this approach, we conducted an online survey of prospective donors in North America. Respondents indicated not only an overwhelming preference for donating to projects conducted by nonprofit research organizations, but also an openness to donating to companies that have a 'for-benefit' corporate structure. They also showed a strong preference for projects that have alternate sources of funding, that have the potential to yield a curative therapy, and that focus on common and pediatric diseases., (Copyright © 2016 Elsevier Ltd. All rights reserved.)

This Article examines the potential stakeholder-related obstacles hindering the implementation of mechanisms to re-ignite the development of novel antibiotics. Proposed economic models and incentives to drive such development include: Public Funding of Research and Development ("R&D"), Tax Incentives, Milestone Prizes, End Payments, Intellectual Property ("IP") and Exclusivity Extensions, Pricing and Reimbursement Incentives, Product Development Partnerships ("PDPs"), and the Options Market for Antibiotics model. Drawing on personal experience and understanding of the antibiotic field, as well as stakeholder consultation and numerous expert meetings within the DRIVE-AB project and Uppsala Health Summit 2015, the Authors identify obstacles attributable to the following actors: Universities and Research Institutes, Small and Medium-sized Enterprises ("SMEs"), Large Pharmaceutical Companies, Marketing Approval Regulators, Payors, Healthcare Providers, National Healthcare Authorities, Patients, and Supranational Institutions. The analysis also proposes a characterization and ranking of the difficulty associated with implementing the reviewed mechanisms. Public Funding of R&D, Pricing and Reimbursement Incentives, and PDPs are mechanisms expected to meet highly systemic barriers (i.e., obstacles across the entire antibiotic value chain), imposing greater implementation challenges in that they require convincing and involving several motivationally diverse actors in order to have much effect.

The health and national security challenge of antibiotic resistance has led governments to adopt policies to stimulate new antibiotic R&D. Government programs that directly fund late-stage clinical development of antibiotics have emerged, including the Broad Spectrum Antimicrobial Program of the Biomedical Advanced Research and Development Authority in the United States, and the New Drugs for Bad Bugs program of the Innovative Medicines Initiative in the European Union. These efforts are collectively investing nearly $1 billion and are supporting nearly 20% of the global antibiotic pipeline. This article describes these programs, including the antibiotics and their targeted pathogens and clinical indications, as well as program mechanisms for project eligibility, selection, governance, funding, and IP management. Preliminary assessment of the impact of these mechanisms on the success of the programs is provided.

Given the number and diversity of risks in today's complex society, it is essential to focus on global risks that can be reduced through affordable, feasible approaches. Thus, the risks that should command the greatest focus are emergent infectious diseases. Fortunately, preparing responses to such threats can be entirely agnostic as to source or intent of the threat. This article considers the emergent infectious clinical threats, characterizes the steps that are essential to take to prepare for such threats, and discusses the roles that the biomedical industry should play in both the preparation for and response to such threats. The author assesses the readiness of the industry to play its role and suggests steps to consider to enable a more robust response.

Maintaining research and development (R&D) productivity at a sustainable level is one of the main challenges currently facing the pharmaceutical industry. In this article, we discuss the results of a comprehensive longitudinal review of AstraZeneca's small-molecule drug projects from 2005 to 2010. The analysis allowed us to establish a framework based on the five most important technical determinants of project success and pipeline quality, which we describe as the five 'R's: the right target, the right patient, the right tissue, the right safety and the right commercial potential. A sixth factor - the right culture - is also crucial in encouraging effective decision-making based on these technical determinants. AstraZeneca is currently applying this framework to guide its R&D teams, and although it is too early to demonstrate whether this has improved the company's R&D productivity, we present our data and analysis here in the hope that it may assist the industry overall in addressing this key challenge.

Objective: National medicines policies (NMP) provide a means for governments to achieve their objectives in relation to pharmaceuticals and other medicines. This research aimed to identify challenges to implementing the objectives of the Australian NMP from the perspective of key stakeholders., Methods: In 2012 and 2103, we conducted 30 semistructured interviews with stakeholders involved in the discovery, clinical testing, regulation and funding of medicines in Australia. We asked participants to describe their careers and to give their opinions on specific issues surrounding drug development, clinical research, regulation and subsidisation in Australia. Data were analysed using Morse's outline of the cognitive basis of qualitative research and Charmaz's outline of data analysis in grounded theory. The initial phase of 'open coding' revealed findings that could be mapped to three of the four objectives of the NMP. We then conducted 'focussed coding' for themes relevant to these objectives., Results: Participants identified many issues relevant to the ongoing evolution of the NMP, relating primarily to ongoing tensions between the commercial objective of ensuring a viable medicines industry, and the non-commercial objectives of ensuring that medicines are safe, effective and affordable. There were also several other challenges identified to the achievement of both the commercial and non-commercial objectives of the NMP. These included limits to government funding, globalisation, consumer advocacy, changing scientific paradigms and new information technologies., Conclusions: There are many issues that need to be addressed if policymakers are to achieve the best outcomes from the NMP. Tensions between the commercial and non-commercial objectives of the NMP suggest the need to ensure that one stakeholder group's imperatives do not stifle those of other groups. At the same time, there are several emerging issues that are likely to concern all stakeholders equally, and these are both challenges and opportunities for new kinds of collaboration. What is known about the topic? We know that stakeholders have several concerns about medicines policy, but little is known about the specific challenges to implementing medicines policy from the perspective of those involved. What does this paper add? We demonstrate that stakeholders have many concerns that could impact the implementation of medicines policies. These relate primarily to ongoing tensions between the objective of ensuring a viable medicines industry, and the objectives of ensuring that medicines are safe, effective and affordable. There are also several issues that potentially pose a challenge to achieving both the commercial and non-commercial objectives of the NMP. These include limits to government funding, globalisation, consumer advocacy, changing scientific paradigms and new information technologies. What are the implications for practitioners? Policymakers need to systematically address the barriers to the ongoing implementation of the NMP. Policymakers should also ensure that one imperative (such as the commercial imperative) does not stifle other objectives. Other emerging issues are likely to concern all stakeholders, and these provide opportunities for new kinds of collaboration among stakeholders.

The development of disease-modifying treatments for Alzheimer's disease (AD) faces a number of barriers. Among these are the lack of surrogate biomarkers, the exceptional size and duration of clinical trials, difficulties in identifying appropriate populations for clinical trials, and the limitations of monotherapies in addressing such a complex multifactorial disease. This study sets out to first estimate the consequent impact on the expected cost of developing disease-modifying treatments for AD and then to estimate the potential benefits of bringing together industry, academic, and government stakeholders to co-invest in, for example, developing better biomarkers and cognitive assessment tools, building out advanced registries and clinical trial-readiness cohorts, and establishing clinical trial platforms to investigate combinations of candidate drugs and biomarkers from the portfolios of multiple companies. Estimates based on interviews with experts on AD research and development suggest that the cost of one new drug is now $5.7 billion (95% confidence interval (CI) $3.7-9.5 billion) and could be reduced to $2.0 billion (95% CI $1.5-2.9 billion). The associated acceleration in the arrival of disease-modifying treatments could reduce the number of case years of dementia by 7.0 million (95% CI 4.4-9.4 million) in the United States from 2025 through 2040., (© 2014 New York Academy of Sciences.)

Scholarly attention to pharmaceutical companies' ability to sustain research and development (R&D) productivity has increased as they increasingly handle business challenges. Furthermore, the deterioration of R&D productivity has long been considered a major cause of mergers and acquisitions (M&As). This study attempts to investigate quantitatively the possible causes of the deterioration and the relationship between the deterioration and M&As by examining the Japanese pharmaceutical industry. Japan from 1980 to 1997 is an ideal case because of the availability of official data, but more importantly the significant changes in its business environment at the time. Using the Malmquist Index and data envelopment analysis, we measured the deterioration of R&D productivity from 1980 to 1997 based on a sample of 15 Japanese companies. Two lessons can be learned from Japan's case. First, to sustain R&D productivity over the long term, companies should use licensing activities and focus on the dominant therapeutic franchises. Second, if a company fails significantly to catch up with the benchmark, it is likely to pursue an M&A or seek an alternative way to improve R&D productivity. These findings appear similar to the current situation of the global pharmaceutical industry, although Japan pursued more licensing activities than M&A to improve R&D productivity.

Pharmacogenomics knowledge and technologies, which couple genomics information with pharmaceutical drug response, have been promised to revolutionise both drug development and prescription. One notable promise of pharmacogenomics is the potential to contribute to some of the Millennium Development Goals (MDGs), namely to increase justice in global health by incentivising public research laboratories and pharmaceutical companies to develop drugs for populations (e.g., in low- and middle-income countries) that have been neglected by the traditional drug development model. To evaluate the credibility of this promise, we examined - both quantitatively and qualitatively - those scientific papers indexed in PubMed and published between 1997 and 2010, with a view to describing the major orientations and tendencies characterising the development of pharmacogenomics research. Our results demonstrate that pharmacogenomics research has focused on three major non-communicable categories of disease: cancer, depression and other psychological disorders and cardiovascular and coronary heart disease. Few publications - and thus, by extension, little scientific interest - concerned orphan diseases, infectious diseases or maternal health, indicating that pharmacogenomics research over the last decade has replicated the well-known 90/10 ratio in drug development. As such, we argue that research in the field of pharmacogenomics has failed in its promise to contribute to the MDGs by reducing global health inequalities.

Achievement of optimal therapeutics requires individuals with analytic skills appropriate to the balancing of enterprise, innovation and the need for rigorous scientific validation. A synergistic convergence of discovery research, clinical investigation, evaluative, regulatory and implementation sciences will be essential. None of the needed research capacities are likely to prove obtainable on demand. On the contrary, they require accurate projection of future needs and careful planning of post-secondary training programs. A survey conducted for Health Canada in 2010 revealed significant shortfalls in research skills available outside government and industry. This commentary argues that such an environment represents an outstanding opportunity for the academic community to demonstrate that it is eager to meet the needs of the Canadian public. University leaders should be assertive about their commitment to the ideals of patient oriented research and all governments should be clear about deliverables anticipated in return for consistent post-secondary funding.

The phase between research and successful innovation is known as the valley of death. Increasingly, researchers from the pharmaceutical industry and academia are working together, often encouraged by governments, to cross this 'valley' as they seek to bring basic research to the market. This is consistent with newer models of innovation policy that stress interaction between the different agents across the innovation process. Here, we examine this interaction in the UK, the EU and the USA using several specific examples, suggesting that cooperation is still far from perfect and that the return for academia on its research investment is relatively small. Countries are also beginning to use research as a tool of industrial economic policy., (Copyright © 2013 Elsevier Ltd. All rights reserved.)

Most research prizes in biomedicine, from the Nobels to the Laskers, are restricted to three recipients. But in an age of big science, when much larger teams are generally needed to make important research discoveries, all the people who provide seminal contributions deserve to be awarded.

By quickly clearing the development pipeline of failing or marginal products, fast-fail strategies release resources to focus on more promising molecules. The Quick-Kill model of drug development demonstrates that fast-fail strategies will: (1) reduce the expected time to market; (2) reduce expected R&D costs; and (3) increase R&D productivity. This paper outlines the model and demonstrates the impact of fast-fail strategies. The model is illustrated with costs and risks data from pharmaceutical and biopharmaceutical companies., (Copyright © 2012 Elsevier Ltd. All rights reserved.)

Between 1970 and 2009, US public institutions participated in the research and development of 153 drugs, including 15 vaccines. Between 1990 and 2007, nearly 10% of all the new drugs and vaccines marketed in the US and about 20% of those representing a therapeutic advance originated in public institutions. The proportion of these drugs that represented a substantial therapeutic advance was over twice that of those produced exclusively by the private sector.

Orphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of orphan diseases is seen by industry as an attractive strategy, there are limited economic data available to support its use. In this paper we show that the revenue-generating potential of orphan drugs is as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success. The data support the targeting of rare diseases as an important component of a successful biopharma R&D strategy., (Copyright © 2012 Elsevier Ltd. All rights reserved.)

Diseases that cause comparatively few problems in developed countries kill millions of people in the Third World each year. In many cases, people die because they cannot afford the medication needed to save their lives. In others, there are simply no drugs available because there are no wealthy western patients to justify pharmaceutical companies investing in a cure. This reveals a deep-seated problem within the patent system and the pharmaceutical industry that emphasises markets and profits at the expense of health and global welfare. Global efforts have seen substantial improvements in access to medicines in isolated areas, but with international agreements driving towards stronger patent protection and the expiry date for the TRIPS grace period fast approaching, it is time to consider alternatives which will allow the patent system to work for the humanitarian cause rather than against it. This paper considers two such problems in the patent system and pharmaceutical industry - prohibitive pricing and misdirected incentives - to offer a mode of regulation and enforcement that will support both a viable pharmaceutical industry and the human right to health and medication.

Recent reports suggest that R&D productivity in the pharmaceutical industry can be strengthened by reducing costs both in the early phase and, mostly, in the clinical phase of drug discovery. However, a cost-benefit probabilistic analysis reveals that despite high costs, the clinical phase shows healthy productivity, whereas the early phase, particularly lead optimization, exhibits very low productivity., (Copyright © 2011 Elsevier Ltd. All rights reserved.)

The Hatch-Waxman Act provides 180 days of market exclusivity to encourage generic companies to challenge the validity of pharmaceutical patents issued to innovator pharmaceutical companies. The consequent patent losses have been exacerbated owing to the application of holdings of the 2007 Supreme Court KSR decision to questions of pharmaceutical patentability by the judiciary and the US Patent Office. The resulting negative effect on support for new drug and formulation discovery by pharmaceutical scientists is discussed. To counteract the societal detriment of this negative effect, the adoption of a 12-year US Food and Drug Administration (FDA) market exclusivity paradigm for all approved new chemical entities including prodrugs is proposed. Such market exclusivities have already been enacted in the United States for follow-on biologicals and are in substantial harmony with those of the European Union, Japan, and Canada. An extension of the existing 3-year FDA market exclusivity for new formulations under 21 U.S.C. (United States Code) §505(b)(2) to 5 years should also be considered., (Copyright © 2011 Wiley-Liss, Inc.)