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The fourth annual CARE Awards returned to Boston, Massachusetts 2 May to once again recognize and commend key achievements across the spectrum of drug development that took place in 2018.

ICON was the biggest winner overall of the 2019 Clinical and Research Excellence Awards, taking home two trophies for Best in Health Economics and Outcomes Research and Clinical Research Team of the Year.

Last night’s ceremony in Boston, Massachusetts was the fourth annual Clinical and Research Excellence Awards, hosted by Informa Pharma Intelligence. The CARE Awards recognize and commend key achievements across the spectrum of drug development in 2018, which was a standout year with a record-breaking 65 new molecular entities and novel biologics approved in the US.

The biggest awards of the night went to Lifetime Achievement Award recipient Charles DeLisi, also known as “the father of the Human Genome Project.” And the Marquee Award for R&D excellence went to Vertex Pharmaceuticals, whose key achievements demonstrated their focus on innovation and commitment to bringing drugs to patients faster. The winner of this special award was chosen by the Pharma Intelligence team based on various performance metrics that demonstrate cutting-edge research, prioritizing R&D, and operational excellence.

In 2018, Vertex initiated the highest proportion of clinical trials that incorporated precision medicine and patient-centric strategies. Additionally, the company’s active pipeline includes one of the largest percentages of new molecular entities, as well as drugs with various regulatory designations to streamline their drug development efforts, such as breakthrough therapy, fast track, and PRIME. Vertex also held one of the higher R&D intensity percentages, investing nearly half of their revenue into R&D spend in 2018 to support their keen focus on cystic fibrosis, as well as unmet needs in pain, sickle cell disease and beta-thalassemia, as well as alpha-1 antitrypsin deficiency.

Innovations

ICON kicked off the night with their first win for Best in Health Economics and Outcomes Research, sharing the award with Bristol-Myers Squibb and Latitude (Powered by AXON). Judges were impressed by the registry named “Studying Interventions for Managing Patients with Chronic Myeloid Leukemia in Chronic Phase (CP-CML): The 5-Year Prospective Cohort Study (SIMPLICITY),” deeming the trial as a “very important tool with real-world value for CML patients.” This ongoing longitudinal study recruited CP-CML patients receiving first-line tyrosine kinase inhibitor (TKI) treatment in the US and Europe, and provides much needed insights into managing CP-CML patients in routine real-world clinical practice, especially as no direct head-to-head studies of TKIs currently exist.

The award for Best Patient-Centric Clinical Trial went to Takeda for the Embolden Study, Shire’s Phase II open label trial of SHP611 in children with late infantile metachromatic leukodystrophy (MLD), an extremely rare and fatal condition with no approved treatments. To address the high unmet needs for this vulnerable population, Shire launched a novel patient-centric program that incorporated insights from critical patient partners to inform protocol development and study execution decisions. Judges found the program to be “exemplary of engaging the patient perspective using both direct (patient interviews) and indirect access to patient relevant issues and engaging with a range of stakeholders involved in developing a patient perspective.” By consulting caregivers of MLD patients, past and current SHP611 sites, and relevant patient advocacy groups, Shire designed a trial with the patient and their families at the forefront, developing creative approaches to personalize the clinical trial experience for sites and families. An example of the team’s creativity is the creation of “Warrior Kits,” which include an illustrated storybook to guide the conversation around study participation as well as a cape for the pediatric patients as a metaphor for their courage and bravery.

A high commendation for Best Patient-Centric Clinical Trial was given to the Alkaptonuria (AKU) Society for their final study of the DevelopAKUre program, SONIA 2 (Suitability of Nitisinone in Alkaptonuria 2). A consortium of 12 European partners ran this trial, which judges felt serve as “an example that should be broadly shared about how to design and conduct a patient-engaged program.” Due to the influence of patients and efforts by the AKU Society, the trial achieved a remarkable 92% retention rate after recruiting 50% of the known alkaptonuria patient population of Europe and Jordan.

Clinical Trial Process Innovation was introduced this year as a new category for CARE to recognize the importance of improving the methods involved in trial planning and conduct, and went to Fortress Medical Systems and Accelerate Diagnostics for their CLINDEX XML solution. This customizable system, recognized as a “notable innovation” by judges, addresses issues with managing the vast amounts of electronic data generated by medical devices necessary to a clinical trial. By automating the uploading of device data in close to real time, the XML tool saves sponsors hours of manual data entry time and improves both quality and monitoring of collected data.

R&D Progress
Medicines360 took home the prize for Clinical Trial Result of the Year (sponsored by Cytel) for ACCESS IUS (A Comprehensive Contraceptive Efficacy and Safety Study of an IUS). This Phase III open-label evaluation of Liletta, a levonorgestrel-releasing intrauterine system for long-term, reversible contraception, was the largest study of a hormonal IUD conducted solely in the US, enrolling a total of 1,751 women with no limitations on race, weight, or BMI. Judges were “impressed by the size of the cohort studied” and the “impact of patient-centered research on improving contraception and women’s health.” Thanks to the unique findings of ACCESS IUS, clinicians are armed with previously unavailable data and are able to better counsel their patients about the long-term safety and efficacy of IUSs.

Eidos Therapeutics demonstrated Excellence in Rare Disease Drug Development for their efforts in treating transthyretin amyloidosis (ATTR) with AG10, which “jumped off the page for [judges] as a stand out among the entries.” Eidos aims to target all forms of ATTR with AG10, a drug that mimics a naturally-occurring variant of the TTR gene (T119M) that has been shown to prevent ATTR in those carrying the disease-causing mutation, earning it the reputation as a “rescue mutation.” In 2018, AG10 received orphan drug designation from the FDA and EMA, and was found to be well tolerated with clear evidence of drug activity in all treated subjects in Eidos’ Phase II trial, providing compelling evidence to pave the way to begin Phase III development.

A high commendation for Excellence in Rare Disease Drug Development went to Amicus Therapeutics for Galafold, an oral precision medicine for the rare genetic lysosomal storage disorder Fabry disease. As a results of Amicus’ commitment to Fabry disease, Galafold is the first ever oral medication for the disease, as well as the first new treatment option in 15 years, and is now available in eight geographies.

Altasciences’ innovative approach to identify and recruit the complex population of non-alcoholic steatohepatitis (NASH) patients was awarded the Most Successful Early Phase Research (Preclinical & Phase I). Recruiting NASH patients can be a challenging task since NASH doesn’t present with distinct symptoms, leaving the majority of NASH patients unaware of their condition until severe liver damage has already occurred, prompting the diagnosis. To mitigate this obstacle and facilitate rapid study start-up and recruitment, Altasciences proactively built a database of NASH patients by running a four-month campaign targeting potential patients with relevant, known conditions strongly associated with NASH, such as obesity and diabetes. Altasciences ultimately identified 50 previously undiagnosed NASH patients, and leveraged this database for a 2018 trial at one of 10 sites, becoming the top enrolling site and recruiting more than 35% of the total study population.

The Impact Of Technology
Oracle Health Sciences won Best Sponsor-Focused Technological Development for their Data Management Workbench (DMW), which underwent one of its most significant updates last year. By enabling the creation of standardized datasets, DMW provides a single source of truth for clinical trial data, which is “the Holy Grail we’ve been seeking all these years,” according to the judges. Last year DMW, including an enhanced user interface and improved user experience. DMW provides greater efficiencies in data management, tackling the time-consuming and error prone process of cleaning and reconciling copious volumes of data from disparate sources, and empowering Oracle Health Sciences customers to make critical decisions, quickly. 

The Best Sponsor-Focused Technological Development category also highly commended IQVIA for their Mobile CRA platform, which streamlines the monitoring process for Clinical Research Associates (CRAs). The platform is comprised of three mobile applications and enables efficiencies in the way CRAs manage their sites, clinical trial participants, trial data, and Trial Master File documentation, resulting in 20%-30% faster site documentation and follow-up.

Next, the Best Patient-Focused Technological Development was awarded to Aseptika Limited (Activ8rlives) for Asthma+me, a self-care solution to support children and young people with asthma that bridges the transition from hospital outpatient care to primary care. The CE-marked Asthma+me is the first remote monitoring self-management tool for pediatric clinical trials, and was specifically designed to meet the needs of children, with separate views for the child and the parent/caregiver within the same app. Through a novel motivating rewards system, a smart tracker to provide reminders for the next dose and built-in educational tools, Asthma+me offers key strategies to address the global and costly problem of adherence.

The Power Of Collaboration
ICON received its second trophy of the night for Clinical Research Team of the Year, honoring ICON’s Eli Lilly Cardiovascular Outcomes Trial (CVOT) team who supported a Phase III trial of 9,901 patients with Type 2 Diabetes Mellitus (T2DM). The high performing team coupled their expertise in CVOTs and Lilly study management with close collaboration and engagement to deliver “outstanding operational result[s]” by hitting milestones significantly ahead of planned timelines. The team met an ambitious target for first patient in (FPI) and continually exceeded enrollment projections, ultimately achieving enrollment of the large trial one year early and the longest follow-up period for a CVOT in this drug class. Robust retention strategies led to >99% vital status (VS) ascertainment at study closeout, followed by a mere 38-day period between last patient visit (LPV) and database lock (DBL) and a DBL nine months early, far exceeding expectations.

Medidata Clinical Partnership of the Year
(sponsored by Medidata) was won by TransCelerate BioPharma, a non-profit comprised of 19 biopharma organizations. Through TransCelerate’s framework, these 19 member companies pool their resources to address a myriad of R&D challenges, supporting a mission of simplifying and accelerating R&D for new therapies to improve the health of people across the globe. The CARE Awards judges recognized the “great impact that the organization has made to harmonize, facilitate, and where possible simplify the conduct of clinical trials.” TransCelerate’s network spans 30 countries, enabling them to create global solutions by tapping into the diverse experiences and expertise and developing innovative ideas that tackle issues around placebo and standard of care data sharing, comparator network, site qualification, risk-based monitoring, and more.

Lifetime Achievement
The evening culminated with the Lifetime Achievement Award, which was presented to George DeLisi, PhD, for his pioneering contributions, particularly to mathematical and computational immunology, cell biophysics, and genomics.

During his extraordinary career spanning decades of academic and public service, DeLisi and his advisors proposed and defended the largest and most controversial biomedical endeavour at the time: the Human Genome Project. Through DeLisi’s advocacy and vision, lawmakers approved the revolutionary project that aimed to determine the DNA sequence of the entire human genome within 15 years. The Human Genome Project is considered to be one of the greatest feats of exploration, and has enabled new strategies to treat, cure, or prevent disease.

For his seminal role in initiating this project, DeLisi received the Presidential Citizens Medal in 2001, and earned the unofficial title of “the father of the Human Genome Project.”

In addition to the Human Genome Project, DeLisi championed the application of mathematical concepts to advance immunology and cell biology, forming the foundation of the now widely used bioinformatic approaches to guide development of peptide vaccines.

Pharma Intelligence is honored to recognize and congratulate all the 2019 CARE Award winners and entrants, whose vision and dedication continues to propel the industry forward in the shared mission of advancing human health through clinical research.

The 2019 CARE Award Winners

Category 
 
 Winner
 
Best in Health Economics and Outcomes Research
 Bristol-Myers Squibb, ICON and Latitude (Powered by AXON)
 Best Patient-Centric Clinical Trial
 Takeda
 Best Patient-Focused Technological Development  Aseptika (Activ8rlives)
 Best Sponsor-Focused Technological Development
 Oracle Health Sciences
 Clinical Research Team of the Year 
 ICON, Eli Lilly and Company
 Clinical Trial Process Innovation
 Fortress Medical Systems, Accelerate Diagnostics
 Clinical Trial Result of the Year  Medicines360
 Excellence in Rare Disease Drug Development
 Eidos Therapeutics
 Medidata Clinical Partnership of the Year  TransCelerate BioPharma
 Most Successful Early Phase Research (Preclinical & Phase I)  Altasciences
 Marquee R&D Award
 Vertex Pharmaceuticals
 Lifetime Achievement  Charles DeLisi, PhD

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