Life Science Companies Research Articles

Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators.

Patients, life science industry and regulatory authorities are united in their goal to reduce the disease burden of patients by closing remaining unmet needs. Patients have, however, not always been systematically and consistently involved in the drug development process. Recognizing this gap, regulatory bodies worldwide have initiated patient-focused drug development (PFDD) initiatives to foster a more systematic involvement of patients in the drug development process and to ensure that outcomes measured in clinical trials are truly relevant to patients and represent significant improvements to their quality of life. As a source of real-world evidence (RWE), social media has been consistently shown to capture the first-hand, spontaneous and unfiltered disease and treatment experience of patients and is acknowledged as a valid method for generating patient experience data by the Food and Drug Administration (FDA). While social media listening (SML) methods are increasingly applied to many diseases and use cases, a significant piece of uncertainty remains on how evidence derived from social media can be used in the drug development process and how it can impact regulatory decision making, including legal and ethical aspects. In this policy paper, we review the perspectives of three key stakeholder groups on the role of SML in drug development, namely patients, life science companies and regulators. We also carry out a systematic review of current practices and use cases for SML and, in particular, highlight benefits and drawbacks for the use of SML as a way to identify unmet needs of patients. While we find that the stakeholders are strongly aligned regarding the potential of social media for PFDD, we identify key areas in which regulatory guidance is needed to reduce uncertainty regarding the impact of SML as a source of patient experience data that has impact on regulatory decision making.

Illegal interlocks among life science company boards of directors.

Competition between life science companies is critical to ensure innovative therapies are efficiently developed. Anticompetitive behavior may harm scientific progress and, ultimately, patients. One well-established category of anticompetitive behavior is the 'interlocking directorate'. It is illegal for companies' directors to 'interlock' by also serving on the boards of competitors. We evaluated overlaps in the board membership of 2,241 public life science companies since 2000. We show that a robust network of interlocking companies is present among these firms. At any given time, 10-20 percent of board members are interlocked; the number of interlocks has more than doubled in the last two decades. Over half of these interlocked firms report over $5 million in historical revenue, exceeding a legal threshold that makes an interlocking directorate a violation of antitrust law. Those interlocks are only illegal if the companies compete, even in part. Using the disease categories for which companies have sponsored clinical trials, we discover that a few markets are responsible for a large fraction of interlocks. We show that in dozens of cases, companies share directors with the very firms they identify as their biggest competitive threats. We provide a data-driven roadmap for policymakers, regulators, and companies to further investigate the contribution of anticompetitive behavior to increased healthcare costs and to enforce the law against illegal interlocks between firms.

Key Considerations When Designing Real World Evidence Research Involving The Secondary Use of U.S.-Based Electronic Health Record Data

The research use ofhealthcare records in the United States is protected by a wide variety ofregulations and ethical constructs. While healthcare providers are intimately familiar with these, lifescience companies and other researchers are often not.  This lack of awareness often results inprotocols and contracts that, although well intentioned, can cause increaseddelays and costs due to resistance from the curators of the electronic healthrecords. The better that protocols, contracts and budgets are written withthese considerations in mind, the better opportunity we have to generate realworld evidence using electronic health records with less cost, greater speedand most importantly in a manner that does not compromise individual privacy toachieve societal benefits.

227-LB: Kynurenine Pathway Is Associated with Preservation of Beta-Cell Function in Autoimmune Diabetes

Background: Autoimmune diabetes (AD) results from a loss of immune tolerance leading to the destruction of β-cells in the pancreas. Heterogeneity in severity of β-cell damage among people with AD is well-recognized, with some patients retaining a certain amount of insulin-producing cells for years despite the presence of pancreatic autoimmunity. Cellular and molecular mechanisms driving the preservation of β-cell function in AD remain unclear. Aim & Methods: To identify metabolic pathways associated with preservation of β-cell function we conducted a metabolomic study using gas and liquid chromatography-mass spectrometry (GC- and LC-MS) based platform on plasma samples collected from 32 subjects with adult-onset AD (median [Q1-Q3] age: 57 [52-62]; disease duration: 4.5 [3.0-9.0] years) with different severity of β-cell dysfunction, compared with data from people with rheumatoid arthritis (RA, n=28). Metabolome profiles of pancreatic islets from healthy donors after in vitro treatment with proinflammatory cytokines was also evaluated. Results: Metabolomic analyses showed decreased plasma Kynurenine/Tryptophan ratio (KynTr), a marker of indoleamine 2,3 dioxygenase-1 (IDO1) activity, in people with AD compared to RA (0.023 vs 0.027, p=0.03). Among people with AD, KynTr was directly associated with fasting C-peptide levels (rho=0.365), independently of age, gender, body mass index, diabetes duration and Hba1c (p-value after adjustments= 0.003). Lower concentrations of Tryptophan was observed in pancreatic islets from healthy donors after treatment with proinflammatory cytokines. Conclusions: While confirming that IDO1 impairment is involved in the pathogenesis of AD and in the regulation of inflammatory response of pancreatic islets, our results show that KynTr is a marker of beta-cell damage in AD. Overall, this suggests that the kynurenine pathway may be studied as a possible target for therapies aimed at preserving beta-cell function also after a diagnosis of AD Disclosure E. Maddaloni: Consultant; Abbott, Merck KGaA, PIKDARE S. p. A., Speaker's Bureau; Medical Technology & Devices. F. Dotta: None. P. Marchetti: None. R. Buzzetti: None. A. Gastaldelli: Advisory Panel; Pfizer Inc., Novo Nordisk, Merck Sharp & Dohme Corp., Boehringer Ingelheim International GmbH, Consultant; Boehringer Ingelheim International GmbH, Eli Lilly and Company, Fractyl Health, Inc., Merck Sharp & Dohme Corp., Other Relationship; Pfizer Inc., Speaker's Bureau; Eli Lilly and Company. L. Navarini: Consultant; AbbVie Inc., Pfizer Inc., Bristol Myers, Celgene, MSD Life Science Foundation, Eli Lilly and Company, Novartis, Janssen Global Services, LLC. R. Amendolara: None. S. Fenizia: None. S. Pezzica: None. M. Tesi: None. L. D'onofrio: None. E. Bosi: None. Funding Italian Ministry of University and Research (PRIN 2017: 20175L9H7H)

Novartis Oncology Spain: Agile innovation and the RWE cell

Healthcare and life science companies are transforming their business models from product development toward precision medicine. Vasant Narasimhan was appointed in 2018 as the new CEO of Novartis—a global pharma company. He champions a multi-faceted strategic intent: 1) transform the company from “selling pills” to investing heavily in discovering of new treatments that combine life sciences and digital technologies. In Spain, José Marcilla, the Director General of Novartis Oncology, pioneered an organizational transformation by developing (1) a cell-based innovation organization and process and (2) a new startup accelerator. This case explores both initiatives and their far-reaching consequences. The “Spanish experiment” helped to develop of digital services that reach the patient in a highly regulated environment. This duo of cases—Novartis Oncology Spain: Agile Innovation and The RWE (real wold evidence) Cell—gives students the opportunity to examine the motivations behind the transformation at Novartis, explore the implementation of its strategic intent, and ask what is the appropriate pace for such a dramatic change. The case is taught as a single class for decision-making regarding which cells and startups are suitable to be continued given the innovation horizons. By walking with students through these innovation horizons, the case enables decisions to be made about whether this pioneering Spanish program should be continued or not.

Patient Quality-of-Life, Providers with Incentive Payments, Real-World Evidence, and Reducing Healthcare Costs through New Innovations

Background: Value-Based Care is a program that was created by Professor Michael E. PORTER and Elizabeth O. TEISBERG which has been modified for implementation over the last seventeen years. This new program is paid by Commercial Health Insurers (Insurance Companies) and Centers for Medicare and Medicaid Services (Medicare). This program incorporates the patients, doctors, Life Sciences Companies, Insurance Companies, Holistic practice, traditional Chinese medicine, etc. with alternative traditional treatments and therapies for Real-World Evidence based and value-based treatment customized to patients’ needs and the patients benefit. Methods: The program was proposed to achieve value for patients and an improved healthcare system which is especially needed to provide treatment for patients in Serious or Life-threatening conditions or diseases. The program will accurately measure the outcomes of patients and provide a wider choice of treatments that matter to patients, also to measure the cost that successful alternative treatments provide which may lower the cost to Medicare and Insurance Companies. Conclusion: The proposed and now implemented program is to achieve value for patients. Golden Sunrise Nutraceutical (Golden Sunrise) provides a New Medical Innovation and treatment alternative to patients suffering from Serious or Life-threatening illnesses. By using Golden Sunrise Innovation and with the incorporation of Value-based Care patients will become the drivers or it could be said the patient Ambassadors of their own health. By incorporating Golden Sunrise Innovation, the doctors will have more diversity of treatments available for their patients. Payment should reflect value not volume, and the New Medical Innovation Golden Sunrise offers will help in reducing the costs to Insurance Companies accelerating the adoption of Value-Based Care.

Short- and longer-term goals for change – A report from the 2nd workshops of the EHC Think Tank Workstreams on Access Equity and Future Care Pathways

Abstract Introduction The second series of workshops for the EHC Think Tank Workstreams on Access Equity and Future Care Pathways involved working towards consensus on addressing challenges to progress around achieving equitable access to care and shaping rare disease care pathways that meet patient needs while remaining practicable and affordable to healthcare providers. This report summarises workshop outcomes from these two workstreams, in which stakeholder participants identified a ‘guiding star’ determining the direction of ongoing focus, defined achievable ‘near star’ milestones, and enablers and constraints to achieving these. Guiding Stars The Access Equity Workstream proposed focusing on developing a healthcare system that enables patients to benefit from care and treatment fairly and impartially. The Future Care Pathways Workstream agreed that their focus would be on developing care pathways that provide the right intervention at the right time by the right healthcare professional in the right formats with a variety of delivery methods to suit the person. Near Stars For the Access Equity Workstream, changes in narrative and behaviour were the achievable milestones that the group agreed to prioritise. Greater stakeholder collaboration and consistent data collection and use will be important enablers for change. Participants proposed greater emphasis on investment and value (instead of cost and return) and a change of mindset from ‘fixing the patient’ to achieving a healthy life through early intervention and preventing comorbidities. However, lack of clarity over what constitutes ‘value’ and around stakeholders' responsibilities, limited outcome measures and resistance to change may constrain progress. A near star for the Future Care Pathways workstream was the development of seamless, personalised care pathways with integrated digital and AI-based technologies to enable real-time measurement of pathway effectiveness. Participants felt that understanding and respecting patient behaviour and the nudges and incentives needed to promote pathway acceptance will be important. As in the Access Equity Workstream, they recognised the role of routine, standardised data collection for measuring outcomes, sharing information and informing decision-making. They predicted that building trust between stakeholder groups (including patients, healthcare providers, academic and life science companies) and using patient networks and advocates effectively would enable collaboration and ensure that patient needs and insights are acted upon. However, financial and legal aspects, inadequate implementation of technological infrastructure, limited systems integration, and lack of stakeholder time, effort and energy are all potential constraints that will need to be addressed.

Middlesex County, NJ: Where Life Sciences Companies Thrive

Middlesex County, NJ: Where Life Sciences Companies Thrive

A Journey Into the Unknown: PhD Students in a European Training Network on Age-related Changes in Hematopoiesis Conduct Their Project During a Global Pandemic

A Journey Into the Unknown: PhD Students in a European Training Network on Age-related Changes in Hematopoiesis Conduct Their Project During a Global Pandemic

Hiding in Plain Sight: Surprising Pharma and Biotech Connections to NIH’s National Cancer Institute

The National Institutes of Health (NIH), and especially its largest and oldest institute, the National Cancer Institute (NCI), have long been among the best - but perhaps not the most well-known supporters - for the life sciences and pharmaceutical industries, providing non-dilutive funding, scientific guidance, education for potential future employees, and importantly, collaboration opportunities including clinical trials and technology licensing partnerships. In this study, various databases were used, including the NCI Technology Transfer Center (TTC) internal agreement database, NIH technology licensing database, Global Data, Pitchbook, and NIH RePORTER, to explore and analyze collaborations and licensing partnerships between NCI TTC and life science and pharmaceutical companies. For early-stage companies receiving funding or taking part in collaborations and licensing, there is a significant relationship between these NIH agreements and successful financial exits. NCI support not only reaches these early-stage firms but also extends to the Top 20 pharmaceutical companies. These two findings highlight the importance of NCI collaborations for both early-stage and established life science and pharmaceutical companies.

Circular Economic Surplus Asset Management: A Game Changer in Life Sciences

The integration of Surplus Asset Management (SAM) with the circular economy (CE) provides a solution to optimize the use of resources, assets, and equipment throughout their life cycle, enabling new business models, increasing profitability, achieving sustainability goals, and plus reducing obsolescence and waste. We introduce the CE-SAM as a business approach to create value by extending the life cycles of discarded useful equipment. We distinguish four types of value: 1) sourcing value, 2) customer value, 3) information value, and 4) environmental value. Carbon footprints can be reduced by up to 80% by redeploying equipment depending on residual value and customer needs. CE-SAM can be a business game changer if it is integrated as part of the business strategy. The implementation challenges can be best tackled through the collaboration of different actors along the value chain, depending on their capabilities and capacity. REUZEit, a case study company, provides a feasible case study that services life science and pharmaceutical companies to optimize the use of their resources, assets, and equipment, to reduce obsolescence, waste, and increase profit at the same time.

Aligning Corporate Strategy With Supplier Selection

This article aims to demonstrate if corporate strategy is deployed to purchasing function practices, specifically to select suppliers, considering purchasing categories. The article has a qualitative empirical approach, and it is characterized as descriptive in the specific context of a multinational life science company. Our findings indicate an alignment between corporate strategy and purchasing strategy regarding the elements of corporate strategy, such as innovation, quality, sustainability, delivery, flexibility, and costs. However, the hierarchical cascading of these elements for supplier selection practices does not always occur as in the literature. The results suggest that purchasing categories are determining factors in the strategy alignment. The main contribution of this article is to point out that there is an alignment between corporate strategy and purchasing strategy regarding the main elements of corporate strategy. However, the hierarchical cascading of such criteria for supplier selection practices does not always occur as previously described in the literature, as there are variables that influence this process. This article indicates that purchasing categories are key determinants of strategy alignment, as in each category a combination of criteria has been more relevant and not necessarily aligned with corporate or purchasing strategy.

Model of integrated reporting “concept in practice” in the light of pragmatic constructivist paradigm: case studies of life science companies

PurposeThis longitudinal research paper is based on a case study analysis of two Danish life science companies. The general purpose of the paper is to uncover changes in the reporting practices of experienced integrated reporting (IR) reporters. In order to meet that objective, a pragmatic constructivist paradigm was applied to make a better understanding of factors affecting disclosure decisions in the integrated reports.Design/methodology/approachThe research uses a qualitative methodological approach. It is based on content and discourse analyses of the written documents, including the integrated reports, auditors' statements and independent assurance reports.FindingsThe model developed in this study reflects a real phenomenon related to the development of IR practices. The pragmatic constructivist paradigm explains how practitioners perceive business reality, act in the face of changing facts and values and make decisions regarding material disclosures.Research limitations/implicationsThe investigation of only two companies may be perceived as a limitation of this study. However, a small number of life science companies have prepared integrated reports for a long time. The selected organisations are the pioneers in that field and have drawn up integrated reports since 2002 or 2004.Originality/valueThis paper develops an original model of IR “concept in practice”. It considers the regulatory framework regarding materiality in IR through the prism of facts that form a basis for practical work. It also emphasises an impact of a value system and social context on disclosure decisions in integrated reports. In that way, a link between the constructivist paradigm and IR is created.

Analysis of Over 2,200 Life Science Companies Reveals a Network of Potentially Illegal Interlocked Boards

Analysis of Over 2,200 Life Science Companies Reveals a Network of Potentially Illegal Interlocked Boards

РАЗВИТИЕ LIFE SCIENCES ИНДУСТРИИ В ПЕРИОД ПАНДЕМИИ COVID-19

The authors study the trends of the Life Sciences industry, the dynamics of investment in the industry with correlation to external processes, review investments in a new promising industry around the world, update the information technologies used in Life Sciences companies based on digitalization and innovation.

Molly McCarthy, MBA, BSN, RN, RN-BC

Molly McCarthy, MBA, BSN, RN, RN-BC

Leveraging Informatics and Technology to Support Public Health Response: Framework and Illustrations using COVID-19.

To develop a conceptual model and novel, comprehensive framework that encompass the myriad ways informatics and technology can support public health response to a pandemic. The conceptual model and framework categorize informatics solutions that could be used by stakeholders (e.g., government, academic institutions, healthcare providers and payers, life science companies, employers, citizens) to address public health challenges across the prepare, respond, and recover phases of a pandemic, building on existing models for public health operations and response. Mapping existing solutions, technology assets, and ideas to the framework helped identify public health informatics solution requirements and gaps in responding to COVID-19 in areas such as applied science, epidemiology, communications, and business continuity. Two examples of technologies used in COVID-19 illustrate novel applications of informatics encompassed by the framework. First, we examine a hub from The Weather Channel, which provides COVID-19 data via interactive maps, trend graphs, and details on case data to individuals and businesses. Second, we examine IBM Watson Assistant for Citizens, an AI-powered virtual agent implemented by healthcare providers and payers, government agencies, and employers to provide information about COVID-19 via digital and telephone-based interaction. Early results from these novel informatics solutions have been positive, showing high levels of engagement and added value across stakeholders. The framework supports development, application, and evaluation of informatics approaches and technologies in support of public health preparedness, response, and recovery during a pandemic. Effective solutions are critical to success in recovery from COVID-19 and future pandemics.

Postmortems on diagnostic testing start-ups: reports of commercial successes and failures and the case of the Zombie life science company

BackgroundFrom 2014 to 2017, more than 1000 diagnostic companies were launched, securing more than US$10 billion in investment.MethodsWe performed an in-depth exploration of 28 diagnostic companies to differentiate successful and...

PMU84 IMPACT of COVID-19 on Physician Engagement and Life Science Companies' Digital and Operational Strategy

COVID-19 has forced life science companies to rapidly adopt a more digital-centric engagement model to support customer engagement and sustain value creation. This has accelerated the need to invest in and optimize digital infrastructure and operating models to support innovative ways of working and evolve traditional sales and marketing strategies in the “new normal.” This study assessed how companies have been impacted by COVID-19 and how they are investing in and implementing digital capabilities and infrastructure in response.

The nature of individual experiential knowledge in internationalizing SMEs: pitfalls of superstitious learning and the need for wisdom

PurposeThis study addresses the research question: How does the experiential knowledge, superstitious knowledge and the wisdom of CEOs influence the internationalization behaviour of SMEs?Design/methodology/approachAn exploratory qualitative study is used. Longitudinal case studies of two Swedish life science companies are analysed.FindingsAn individual's prior experiential knowledge influence the newly started SME's market commitments and internationalization behaviour. Such prior experiences can enable early and rapid resource commitments in the newly started SMEs. Relying upon such prior experiential knowledge in deciding upon the company's market commitments however heightens the risk of superstitious learning. The findings illustrate how wisdom can work as an antidote to superstitious learning. Wisdom lures even experienced CEOs away from believing they know more than they actually know.Research limitations/implicationsThe study contributes to extend the Uppsala model by incorporating the role of individual-level experiential knowledge. The study also adds value to the literature on small firm internationalization by providing propositions for how the prior knowledge of individual key decision makers influences SMEs' internationalization behaviour. The propositions provide new input to the ongoing discussion in the literature and help to guide future research.Originality/valueGiven the fact that the Uppsala model is centred upon a firm-level view on experiential knowledge, our theoretical understanding is still limited regarding how individual-level experiential knowledge influences the internationalization behaviour of SMEs. This study addresses calls for research on how individuals' prior knowledge influences small-firm internationalization.