Welcome to the inaugural issue of the Journal of Medicines Development Sciences. This new open access journal will publish articles on the entire process of discovery and development of new medicinal products, with a particular emphasis on translational research. This inaugural issue is devoted to “Open Innovation”, a booming concept that is starting to deeply change the context of medicines development.

Asian countries are striving to transition into innovation-driven economies, and healthcare is a key sector on government agendas. Strong funding at academic institutions, high-impact publications, state-of-the-art clinical infra-structures, vast talent pools, increasing start-up activities and strengthening intellectual property regimes, all aligned with coherent government policies, is creating an evolving innovation ecosystem in countries such as China, South Ko-rea, Singapore, Taiwan and India. Such factors have fuelled a desire from global pharmaceutical companies to seek in-novation in Asia. In this article, we review the varied strategies of large multinationals using two key aspects—the capital investments and operational model—to understand such diverse approaches. Based on a qualitative and quantita-tive analysis, we have classified these strategies into four distinct approaches—“Captive”, “Partner”, “Service” and “Open” models—and discussed case studies that fit into each of these clusters. The model may provide global pharma-ceutical companies with a framework to evaluate their respective approaches for sourcing innovation and align them with the operational, financial, business and strategic needs of their organizations in Asia.

Disease-focused philanthropic organizations play an increasing role in the strategy and conduct of biomedical research, with many focusing on drug development for specific diseases and patient populations. More and more they
not only provide resources and expertise, but also take active part managing the strategy and objectives of targeted research programs, using approaches such as venture philanthropy. Many also lead and participate in public–private partnerships. One example is the partnership between the Polycystic Kidney Disease (PKD) Foundation and the Critical Path Institute (C-Path) which brings together several pharmaceutical companies and academic institutions to develop
new broadly-used biomarkers. Another case is the partnership between JDRF (formerly known as the Juvenile Diabetes Research Foundation) and the Innovative Medicines Initiative (IMI), involving financial support of the IMIDIA project (Innovative Medicines Initiative for Diabetes) which is focused on improving beta-cell function and identifying biomarkers for diabetes treatment monitoring. These examples show that in addition to providing financial support and expertise, philanthropic foundations are also in a unique position to coordinate the patient and research communities to enable and accelerate specific medicines development projects.

As the economic pressures increase on healthcare systems around the world due to aging populations, chronic diseases, expanding patient populations in emerging markets and advances in medical technology, it is crucial that we focus on developing and delivering innovative and quality medicines with true medical value to patients around the world in a more collaborative, quality-focused and cost-effective manner.
An important component to this mission across the biopharmaceutical industry is identifying and solving common issues that compromise the success of a clinical development program – the shared pathway to safer and more clinically meaningful medicines. However, subject recruitment challenges, data collection and follow-up issues, identification of high-quality trial sites, and lack of successfully achieving study timelines continue to stress clinical trial operations teams across companies. Although there has been progressing across this range of roadblocks by individual companies, the underlying economics continue to threaten the research and development (R&D) business model.
Failure to solve these key issues will affect all parties involved in the clinical trial enterprise: patients, clinical inves-tigators, health authorities, academia, tax-payers and the sponsor companies. The question remains whether a deep and broad collaborative effort that stretches across the clinical development arena—one that is charged with a common goal of improving quality, enhancing the investigator and patient experience, reducing costs and sharing data—can be a catalyst for success. With encouraging signs already realised, the operation of TransCelerate BioPharma Inc., a non-profit organisation created to improve the health of people around the world by accelerating and enhancing the R&D of innovative new therapies will test this premise.

The European Lead Factory (ELF) is a unique collaborative public–private partnership aiming to deliver innovative drug discovery starting points and improving the value generated by ultra-High Throughput Screening (uHTS) approaches. Composed of a unique compound collection derived from private pharmaceutical company collections and complemented with new chemistries from a unique public collection, it offers a unique uHTS platform accessible to both private companies and publicly funded researchers. One of the key challenges in setting up ELF has been to balance access to screening results with protecting the value of compounds in the collection. Through an ‘honest data broker’ data management platform and a royalty reward scheme based on achieved milestones, ELF has been able to overcome these challenges. Set up in 2013, it has already accepted 42 targets for screening, submitted by publicly funded researchers, and generated 12 Qualified Hit Lists. In addition, 55,000 new library compounds have been generated by the public partners and added to the 320,000 compounds made available by the companies. Although it faced many challenges in becoming operational, this unique experiment in collaboration is already generating exciting results that will hopefully, eventually lead to better medicines and tools to advance our biological knowledge, and should act as a template for future approaches in the area.

The Predictive Safety Testing Consortium (PSTC) is one of nine consortia comprising the Critical Path Institute (C-Path), a non-profit organisation launched in 2005 and dedicated to playing the role of a catalyst in the development of new approaches that advance medical innovation and regulatory science. C-Path achieves this by lead-ing teams that share data, knowledge and expertise resulting in sound, consensus-based science. PSTC is a unique, public-private partnership that brings pharmaceutical companies together to share and validate safety testing methods under the advisement of worldwide regulatory agencies, including the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). The eighteen corporate members of PSTC share a common goal: to find improved safety testing methods and approaches utilizing fluid-based safety biomarkers which accurately predict drug-induced tissue injury. Specifically, the primary goal of PSTC is the qualification of novel translational safety biomarkers for use in early clinical trials in order to ena-ble safer investigations and development of new drug candidates. This manuscript describes the critical importance of improved safety biomarkers to the drug development process and the present state of the biomarker qualification process with regulatory agencies. In addition, the work that the PSTC and its collaborative partners have done and con-tinue to do to identify and qualify more selective and specific safety biomarkers is highlighted. Finally, successes in-cluding the recently adopted regulatory Letter of Support and ongoing efforts to better define the regulatory qualifica-tion process and an integrated translational safety strategy are also discussed.

Infections remain a significant cause of disease, disability, and death in developing countries. Unfortunately, many of these infections, including centuries-old neglected diseases such as malaria and newly emerging and re-emerging diseases such as Ebola virus disease (EVD), have limited products available to prevent, diagnose, and treat them. One barrier that hinders the development of these products is neglected and emerging disease experts’ limited access to the biopharmaceutical industry’s small molecules, technologies, and know-how. Conversely, the biopharmaceutical industry’s lack of attention to and expertise in these diseases impedes the development of much-needed products. Organisations are addressing these challenges by developing platforms through which disease experts can access industry’s knowledge and assets. Strategic partnerships are applying a synergistic approach to leverage respective strengths of academia and industry. The following article describes two open innovation platforms, the Pool for Open Innovation against Neglected Tropical Diseases (POINT) and WIPO Re:Search, and two strategic, cross-sector collaborative efforts to develop therapeutics for EVD.

The Critical Path to Tuberculosis Drug Regimens (CPTR) initiative aims to support the rational deployment of new tuberculosis (TB) therapies by speeding the development and impact of new and markedly improved drug regimens as well as rapid drug susceptibility tests. Co-founded by the Bill & Melinda Gates Foundation, the Critical Path Institute, and the TB Alliance in 2010, CPTR is a coalition comprising the world’s leading pharmaceutical companies, product development sponsors, diagnostic companies, regulatory agencies, and civil society organizations which support and catalyze advances in regulatory science, the development of infrastructure, and other progress needed to accelerate the pace of development and introduction of novel regimens and rapid drug susceptibility tests. This manuscript summarizes the work of two subgroups within CPTR, the Regulatory Sciences and Rapid Drug Susceptibility Test consortia, and their efforts to drive innovation. These consortia are supported by a robust TB clinical data platform, which continues to evolve through contributions of contemporary TB clinical trial data sets as well as whole genome sequence level data from isolates across the globe. Examples of innovation are described and include a recently-qualified drug development tool and emerging programs to support the development of clinical trial simulation tools.

The biopharmaceutical industry has traditionally been the key link between basic biomedical discovery and novel medicines. Today, the industry faces numerous challenges including the broad agreement that the current clinical trial system is inefficient and flawed. Most challenges are worsened by the inability of the stakeholders to work colla-boratively. Over the last decade, many cooperative efforts to transform clinical research have been launched, but a sys-temic solution has not been envisioned. A systems approach, including the application of systems engineering principles, has been used in other sectors and proposed for use in healthcare and medicines development. Clinical research, when looked at in systems terms, can be defined as an open system involving patients, investigators and associated staff, regulators, sponsors and stakeholders interconnected through a series of processes to bring effective and safe medicines into the market. ACRES is a global nonprofit organization with a mission of creating a multi-sector alliance of individuals and institutions collaborating on building a shared global system for clinical research excellence. A fundamental element of the ACRES system includes a global network of high-performing research sites interconnected through a shared information technology platform, with standardized policies and operational procedures and a robust, secure database to support performance, quality and safety. Five core initiatives address the larger mission and are currently ongoing. De-liverables will roll out over 2015–2018. Positive reception to the concept, vision and goals among critical stakeholders, and a steady influx of strategic allies willing to work collaboratively demonstrates the strong pull exerted by the vision of a global system. However, due to the undertakings’ scope and complexity, challenges remain. Recognition that effective shared collaboration is the best long-term option among stakeholders and the general public constitutes a powerful in-centive for ACRES contributors and strategic allies to keep working and make it happen.

In the last decade, the environment for medicines development has undergone unprecedented change, including: a shift from predominantly big Pharma to one of extensive industry–academia and SME partnerships and collaborations, greater emphasis on understanding the molecular basis of disease for stratified and personalised medicine, and a focus on societal/patients’ needs and priorities.
A variety of European initiatives have been established to support the training of scientists/professionals involved in medicines development in order to respond to this constantly changing, demanding, and chaotic landscape. Some of these initiatives are directed specifically at medicines development, some address scientific research including training relevant to medicines development, and some support general training needs which can include medicines development.
There are a number of European initiatives supporting better training in medicines development. They differ in their approach, ranging from IMI (specific to medicines development) to Erasmus+ (boosting skills and employability in all areas). The scope, budgets, and requirements differ considerably and they each have to be reviewed in detail to decide which might be the most appropriate for a particular target group. The common theme is to support the aims of the European Research Area (ERA), i.e., a significant improvement in Europe’s research performance to promote growth and job creation.

The value of collaborations and partnerships between different stakeholders to achieve optimum outcomes in the medicines research and development process is being recognised. Historically, there has been a lack of collaboration with patients and many research consortiums consisting mainly of academia and/or industry partners. Although patient experts are able to bring valuable first-hand experience and insights, they might not possess detailed knowledge about medicines research and development to actively participate in the collaboration process. The European Patients’ Academy on Therapeutic Innovation (EUPATI) was established to deliver training to patient experts, and education resources
to patient advocates and members of the health-interested public across Europe. EUPATI was launched in February 2012 and is a patient-led Innovative Medicines Initiative (IMI) project, with a multi-stakeholder consortium of patient advocates, academia, industry and not-for-profit organisations. Training and educational materials will be used for capacity building among patients, for educating patient advocates and for informing the health-interested public. The successful uptake of EUPATI’s materials will hopefully translate into a new paradigm of increased patient involvement across the entire medicines research and development process, bringing mutual benefits, including better medicines, to all stakeholders.