December 19, 2018 | December was full of exciting news in the clinical trial and healthcare community, including partnerships, products, and promotions from The National Science Foundation, Cytel, Rho, and more.

The National Science Foundation announced a grant to VMT for developing technology focused on pragmatic clinical trials allowing clinical outcomes to be extracted from real world data. The approach uses artificial intelligence to dive deep into the electronic health record (EHR) for outcome data to enable EHR-based pragmatic clinical trials. Upon completion of the grant, VMT intends to spin out its technology to Verantos, the market leader in regulatory-grade EHR-based studies. As regulatory bodies increasingly look to real world evidence (RWE) to augment the standard of care, pharmaceutical companies are exploring how to run advanced studies to understand real world impact of therapies on important clinical outcomes. Outcomes, such as symptoms, signs, and disease-free survival, are unavailable or inaccurate in claims and EHR structured data. This award will deploy advanced semantic technologies to extract these hidden outcomes and enable valid and relevant RWE. The Small Business Innovation Research (SBIR) / Small Business Technology Transfer (STTR) programs are intended to provide early stage, high-tech small businesses with grants for proof-of-concept / feasibility research that could potentially be followed by grants for cutting-edge, high-quality scientific research and development to de-risk their technologies. Press release

Cytel announced the launch of East version 6.5, further enhancing the most trusted technology platform for designing, monitoring, and simulating adaptive trials. The upgraded version of East unveils new modules MCPMod (for dose-finding trials using MCPMod methodology); ENRICH (for simulating a population enrichment design with a survival endpoint); and PROGRAM for optimizing a clinical development program using frequentist or Bayesian go/no-go rules. The upgrades also incorporate a range of enhancements to the platform’s overall user interface and various new features within established modules including ESCALATE, MAMS, and PREDICT. Yannis Jemiai, Senior Vice President of Software and Consulting at Cytel said in a press release, “The enhancements in East 6.5 have been developed in collaboration with our strategic consulting customers to address a number of their most pressing drug development concerns.  The new ENRICH and PROGRAM modules particularly reflect the concerns of drug developers in the oncology and rare disease space where it is critical to make the best possible use of scarce data, at a program as well as at a trial level.  Selection of the right dose for confirmatory phase 3 trials continues to be one of the biggest headaches in drug development and the MCPMod module will help our customers more easily put the EMA- qualified dose-finding methodology into practice, and use available data in the most effective manner.” Press release

Rho announced a new Regulatory Strategy and Submissions business segment. The segment will support Rho’s continued growth and excellence in helping sponsors plan, develop and submit marketing applications, including new drug applications (NDAs), biological license applications (BLAs) and marketing authorization applications (MAAs). Rob Woolson, an applied statistician with an extensive background in statistical and project leadership working on US and ex-US regulatory submissions, will lead the new segment for Rho as the Chief Strategist. It will be supported by 35 highly experienced staff including new hire Kevin Barber, Vice President, Regulatory Strategy and Submissions, who brings more than 20 years of experience in regulatory affairs and product development across all stages of development. The Regulatory Strategy and Submissions business segment provides an integrated approach, allowing regulatory, clinical, nonclinical, CMC and statistical experts to work side-by-side. Teams responsible for managing, integrating, analyzing, assessing and summarizing the data to support the approval of therapeutic products will work closely together to help clients at all stages of development, from pre-IND through post-approval. Press release

PAREXEL announced a new collaboration with Eli Lilly and Company to launch a clinical research learning and development program in China. The program will bring high-value training opportunities to China’s clinical trial sites and investigators, enhancing the execution of local clinical trials and driving innovation in China’s biopharmaceutical industry. To encourage innovation in drug development, the Chinese government recently released a series of new policies for clinical research, resulting in a sharp increase in the number of clinical trials in China. According to China’s clinical trial registration platform, 1,258 drug clinical trials were registered and publicized for the first time in China in 2017, an increase of 62.66% compared to 2016. This increase in clinical research demands has put a strain on the clinical research capacity and resources in China. A recent report indicates that the total number of class 1.1 chemical drugs and class 1 biologic drugs approved for clinical trials from January to October 2017 was three times that of 2014. Still, the number of GCP-qualified clinical trial sites did not significantly increase between 2014 and 2016. PAREXEL and Lilly’s collaboration is based on a shared vision to address this unmet need and promote globalization and innovation of the market’s drug development. The joint program will use curriculum developed by PAREXEL Academy for clinical trial investigators, and issue certifications to professionals who complete the training. Designed by clinical research practitioners, the curriculum combines the latest policies, regulations and clinical research technologies. It also uses behavior-oriented curriculum design, real-world cases, as well as advanced knowledge and industry trends, to provide world-class expertise and practical experience for clinical research investigators. The curriculum has been reviewed and certified by TransCelerate. Press release

uniQure has announced the incorporation of its promoter, developed by Synpromics, in a new preclinical research target. This follows recent positive data, presented at the ESGCT world congress, demonstrating that this highly selective liver promoter is at least eight times more potent than the current industry standard. uniQure’s new gene therapy aims to treat all hemophilia A patients, one of the two main types of hemophilia. Hemophilia A is caused by missing or defective Factor VIII, a protein essential for clotting. Approximately 30 percent of patients with severe hemophilia A will develop an inhibitor that neutralises the infused Factor VIII (FVIII) activity. This patient population has in the past been excluded from gene therapy approaches in clinical development, however, uniQure's new candidate has been demonstrated in preclinical studies to circumvent inhibitors to FVIII. This data shows that the candidate may lead to durable expression in hemophilia A patients and may provide long-term prevention of bleeds. Press release

The Santa Casa da Misericordia hospital of Porto Alegre, Brazil, will expand Clinerion’s network of partner hospitals in Brazil. Patients from the hospital’s seven clinics will gain access to leading-edge, innovative medical treatment via Clinerion’s Patient Network Explorer platform. On the Patient Network Explorer, patients of Santa Casa da Misericordia will have the opportunity to be matched to international clinical trials being run by pharmaceutical companies and clinical investigators around the world. Participation in these trials enhances their access to leading-edge, innovative medical treatments and care. Users of Patient Network Explorer will gain an expanded insight into the patient population in Brazil and be able to make strategic drug development decisions based on real-time, real-world data. “Together with our partner, iHealth, Clinerion is in the process of a significant push to encompass coverage of the majority of patients in Brazil,” Clinerion CEO Ian Rentsch said in a press release. “Santa Casa da Misericordia represents an important addition to Clinerion’s network of partner hospitals in the country, the region, and the world.” Press release

Certara announced the launch of its BaseCase Toolkits, an add-on to BaseCase Interactive which helps pharmaceutical and medical device field representatives better manage and utilize their resources for use during customer presentations. BaseCase Toolkits allows interactive tools – such as mobile apps – to be bundled together with other assets – such as product safety and efficacy data, and marketing assets – into user-friendly, highly-searchable, branded packages that can be easily maintained and updated from a central location. BaseCase mobile apps allow field representatives to connect live to mathematical models and algorithms, and provide answers to complex questions on site. Toolkits can contain PDFs, slide decks, spreadsheets, videos, and other assets and can be used for a broad range of purposes. For example, they can serve as an evidence repository, allowing all of the clinical evidence for a specific product to be bundled together. They make excellent market access portfolios, allowing value communication tools, such as interactive visual aids, eDetailing aids, and interactive value story presentations, economic models, and objection handlers, including pre-launch engagement tools and marketing feedback tools, to be neatly packaged together in one place. They can be employed to create global value dossiers, enabling staff at global companies to search content by value message and accelerate local dossier submissions. In addition, they are tailor made for use as product toolkits, providing a single, corporate branded, access point to all of a product’s marketing assets. “It is more important than ever that pharmaceutical and medical device field staff are able to convey in a clear and compelling fashion the value that their products deliver to patients and payers. BaseCase Toolkits allows those field representatives to optimize every customer engagement by ensuring that they always have the latest data, graphics and messaging in their presentations and are fully compliant with local health authority regulations,” said Thomas Kerbusch, president of Certara Strategic Consulting Services, in a press release. “Toolkits can be easily shared with customers in a single action, enabling comprehensive and efficient follow up after the meeting.” Press release

Insight Genetics announced the launch of its proprietary Insight TNBCtype, a new test for categorizing triple negative breast cancer (TNBC) tumors into distinct molecular subtypes through its wholly owned CLIA- and CAP-accredited lab, Insight Molecular Labs.  Insight TNBCtype, the first and only CLIA-validated assay for TNBC molecular subtyping, will enable prospective clinical trials with pharmaceutical, biotechnology, and academic medical institutions and advance targeted therapies for TNBC patients, addressing a significant unmet need in oncology today. The Insight TNBCtype test is based on a proprietary algorithm that uses gene expression data from next-generation sequencing to generate five molecular subtypes (BL1, BL2, LAR, MSL and M), as well as a complementary immunomodulatory (IM) classifier that may help predict response to immuno-oncology therapies. Insight Genetics has worked extensively to refine the TNBC classification algorithm to maximize its potential for clinical use by reducing the number of genes from over 2,000 down to 101, allowing heterogeneous tumors to be classified by more than one subtype, and optimizing the test for use on single patient samples. Press release

Trialbee announced the official release of its digital patient engagement solutions. With Trialbee, sponsors, CROs and investigator sites can increase patient retention, protocol compliance, and adherence to treatment plans through customized messaging and support. Trialbee’s digital patient engagement solutions allow sponsors, CROs, and sites to improve protocol adherence and reduce data variability, leading to fewer drop-outs and better patient retention. Improving patient retention leads to a more complete set of data points and higher quality data, boosting statistical power. “Our integrated solutions improve our clients’ opportunities for success with marketing approval and go-to-market timeline,” said Hanne Van de Beek, customer project manager, Trialbee, and trained nurse with many years of experience in clinical drug development, in a press release. “Keeping study patients enrolled and actively engaged in a trial impacts data quality and boosts the statistical power of a study – enabling improved ability to detect differences between treatment groups and speed the process to market.” Trialbee puts clinical trials in the hands of patients by integrating studies into their daily lives through a bring-your-own-device (BYOD) application. The app is iOS and Android compatible and includes patient-centric features that can be customized to empower patients with the necessary tools and information, and optimize engagement throughout the entire trial. The integrated site personnel administration tool and “Participant Manager” simplify site workflow and decrease work burden on research sites. Built-in interactive response technology (IRT) connects patients to sites, driving better adherence to treatment, higher quality data, and improved patient retention. Press release

BD (Becton, Dickinson and Company) announced the launch of a new software application designed to help hospitals and health systems identify drug diversion. Addiction to prescription narcotics in the United States has reached epidemic proportions, contributing to the opioid crisis and becoming a major driver of drug diversion within healthcare settings. Diversion of drugs, for personal use or illegal distribution, can cause significant financial loss and potentially impact care to patients and staff safety.

As part of the BD HealthSight platform that is designed to support enterprise-wide medication management, the new BD HealthSight diversion management application is the next step in the company's efforts to address drug diversion through integrated solutions and analytics. The solution leverages data from existing BD products, including the BD Pyxis ES system, and the Electronic Medical Record (EMR) to provide actionable insights to assist with a hospital or health system's diversion investigations. "Medication diversion is a growing and complex challenge for hospitals and health systems. We believe that to best address this challenge, a holistic approach to medication management that includes a combination of connected technologies and robust analytics is required," said Ranjeet Banerjee, worldwide president of Medication Management Solutions for BD, in a press release. "Specific cases of diversion can be difficult to detect, and the impact can be devastating from a patient and healthcare worker safety standpoint. The new BD HealthSight diversion management application is designed to address the unique challenges associated with diversion by tracking patterns and risky behavior to help identify diverters as early as possible." BD HealthSight diversion management is a hosted, cloud-based application that assists with drug diversion investigations by creating an investigation workflow to monitor, triage and assign potential diversion cases to specific investigators. Compared to traditional, statistically-based analytical tools that only look at amounts dispensed to identify potential diversion, BD utilizes machine learning algorithms and multiple dispensing behaviors —such as overrides, canceled transactions, delays in dispensing, administering and wasting medications — to surface clinicians whose behavior indicates higher risk for diversion. BD has partnered with Microsoft, who brings industry leading expertise in artificial intelligence (AI) and data science methodologies, to support development of these machine-learning based algorithms. Importantly, the application also aggregates EMR and dispensing cabinet data to automate a normally time-consuming and tedious manual review process to reconcile and automatically flag anomalous dispense, administration and waste transactions. Press release

VeraSci announced multiple significant enhancements to Pathway, their proprietary eCOA platform. Pathway, which reinvented how raters, sponsors and site administrators conduct assessments and scales for clinical trials, now offers enhanced data collection and administration, and access to a larger number and type of scales and assessments. The Pathway team worked with raters, scale-developers, and sponsors to ensure that all Pathway users would derive optimal functionality while accommodating the needs of each study and site. The improved flexibility of Pathway allows for the use of a single device for multiple assessments and multiple protocols, permits high enrolling sites to use different devices for parallel assessment of multiple patients, and easily integrates all of the data. VeraSci's proprietary assessments, BAC and VRFCAT, and licensed gold standard endpoints and safety measures for multiple therapeutic areas such as the EDSS and C-SSRS, are now accessible through a single Pathway-enabled device. In addition, raters can administer assessments on Pathway and obtain consent from test subjects on the same device. Available in multiple languages, Pathway is able to accommodate a sponsor's specific needs for an integrated experience in a variety of countries, with negligible failure rates or device issues. VeraSci's multi-lingual tech support teams in Europe, Asia, and the United States provide support in sites' local languages, and VeraSci's Global Language Solutions team provides scientifically valid translations and cultural adaptations of required assessments. Press release

Shivom said that it has partnered with Lifebit to offer an artificial intelligence-powered platform for identifying the right patients for enrollment in clinical trials. Under the terms of the deal, the companies will combine their respective technologies to offer a DNA data platform that Shivom said will allow users to perform immediate genome-wide association study analyses, and access proprietary software and AI-powered tools for data analysis. Additional terms were not disclosed. "Through this partnership with Lifebit, we are providing enterprise users with the tools they need to find the right patients for their clinical trials more easily and more accurately than is possible through other solutions," Shivom Cofounder and CSO, Axel Schumacher, said in a statement. "Not only that, it brings AI into GWAS analysis in a way that hasn't been seen before." The deal marks the latest alliance forged by Shivom, which has partnerships with nonprofit eMQT to sequence the genomes of 1,000 sickle-cell disease patients in sub-Saharan Africa; SingularityNet on genomic and medical analytics; and Genetic Technologies to build a genomic data platform for diagnostic development. Press release

Medocity released a study detailing improved behavioral health outcomes for patients with depression and/or anxiety when using remote care technology as part of an outpatient treatment regimen. Carter Health, a Texas-based medical practice, selected Medocity’s Virtual Care Platform to conduct a real-world, observational study of remote symptom monitoring and telehealth utilization when treating psychiatric outpatients. Carter Health documented consistently higher medication adherence rates and a demonstrably positive impact on patient health, engagement, and satisfaction when using the Medocity Platform. The Medocity team worked closely with Carter Health to configure the Virtual Care Platform for multiple behavioral health protocols including remote symptom monitoring, medication reminders, clinician alerts and interventions, and patient support resources. Patients were enrolled and stratified into one of two cohorts with diagnoses of either depressive or anxiety disorders. “Digital technologies continue to simplify our daily tasks, but when used to stimulate a deeper patient commitment to personal health and continuing engagement with care providers, the value of these solutions is truly elevated,” said Raj Agarwal, President and CEO of Medocity, in a press release. “Our Virtual Care Platform can be configured for any health condition, making it the perfect digital companion for daily healthcare management. We are proud that Carter Health has validated our platform’s ability to improve patient engagement, outcomes, and satisfaction.” Press release