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April 26, 2018

Doro Shin

Despite the backdrop of political uncertainty in 2017, the pharma industry continued to persevere and push boundaries. The Clinical & Research Excellence (CARE) Awards, held April 25, honored a spectrum of key R&D activities that took place throughout last year, ranging from disruptive technology that support both sponsors and patients, to noteworthy clinical trial design and results, as well as showcasing the power of teamwork.

Now in its third year, the CARE Awards continues to acknowledge and celebrate the achievements of the pharma industry across the spectrum of drug development activities, from successes in early and late stage research, to advances in technological tools, and innovations in trial design and execution.

A New Leader in R&D Excellence

The night’s top prize went to AstraZeneca, which took home the crown for the Marquee Award for R&D excellence – an honor that went to Novartis for the first two years of the CARE Awards. This winner of this key award was selected by the Pharma Intelligence team, based on outstanding achievement across various performance metrics that indicate commitment, innovation, operational excellence, and success in bringing new medicines to patients.

Pharma Intelligence was impressed by the launch of multiple innovative medicines for patients, including Calquence for mantle cell lymphoma, Fasenra for severe eosinophilic asthma in patients aged 12 years and older, and Imfinzi for advanced bladder cancer in previously treated patients. Among the myriad of clinical advancements, regulatory successes, and collaborative activity, AstraZeneca also led the pack with the volume of partnership deals as well as the number of clinical trials initiated in 2017.

Transforming Clinical Trials

Pricing pressures and reimbursement considerations are increasingly driving the need for outcomes and value based evidence, and the industry must be able to demonstrate both clinical and economic evidence to providers, as well as to healthcare decision makers and payors. Merck KGaA’s Merkel Cell Carcinoma (MCC) Observational Study for Historic Comparison took home the trophy for Best in Health Economics and Outcomes Research, due to its impact in an area with limited literature on historical outcomes with chemotherapy. Through the robust real world evidence gathered through this study, decision makers were armed with compelling data to support the efficacy and use of Avelumab for MCC, which was validated by the FDA in their Drug Information Soundcast in Clinical Oncology (DISCO).

The award for Most Innovative Clinical Trial Design (sponsored by Syneos Health) went to the Autosomal Dominant Polycystic Kidney Disease (ADPKD) Tolvaptan Team at Otsuka Pharmaceutical Development & Commercialization. In an area where no treatments are available to date, judges recognized the value of the therapy for the patient population, and acknowledged “these efforts and dedication to seeing this innovation through to commercial patient use (particularly in an orphan condition) are remarkable in scope, depth of research and breadth of collaboration.” Otsuka’s approach incorporated a number of innovations, including the ongoing collaboration with multiple key stakeholders to develop and inform the trial design, as well as the qualification of a predictive clinical biomarker with the FDA and EMA.

R&D Progress

AveXis, newly acquired by Novartis, impressed judges within the Most Successful Early Phase Research (Preclinical & Phase I) with their Phase I Clinical Trial of AVXS-101, a gene replacement therapy for Spinal Muscular Atrophy (SMA) Type 1. SMA Type 1 is a devastating disease, which leads to progressive muscle weakness, and in the vast majority of patients, an ultimate death by the age of two years. With AVXS-101, all 15 patients were alive and free of permanent ventilator support, and 11 of these patients were able to sit unassisted, which is typically a feat that the majority of SMA Type 1 patients are unable to achieve. The CARE Awards judges recognized the major impact AVXS-101 could have on a debilitating disease, and how instrumental the study design for the Phase 1 trial was in delivering strong results.

Within the growing area of rare diseases, Alnylam Pharmaceuticals demonstrated Excellence in Rare Disease Drug Development through their program in hereditary ATTR (hATTR) amyloidosis. As a result of their efforts, Alnylam was able to report the first-ever positive Phase III results for an RNA interference (RNAi) therapeutic and mark the potential arrival of a new class of medicines. Judges deemed the program as “ground breaking R&D coupled with great trial design leading to successful data in an untreated patient population with a first in class technology” with a “well designed trial and strategy involving in depth teaching of the clinical sites and patient interaction through advocacy, KOLs and families.”

Despite various uncertainties clouding 2017, the year saw a number of exciting breakthroughs, including the entrance of CAR-T therapies to the market. As such, it’s no surprise that Novartis took home the trophy for Clinical Trial Result of the Year (sponsored by Cytel) with KYMRIAH (tisangenlecleucel), the first chimeric antigen receptor T-cell therapy to be approved anywhere in the world. Although its initial indication for pediatric precursor B-cell acute lymphoblastic leukemia is limited to a small population of patients, the judges noted it marks a “significant advance for pediatric/juvenile subjects with historically few options and poor outcomes.”

The judges also recognized it is “a major step forward in treatment technology in the cell therapy area” and that Novartis demonstrated “a great example of industry-academia collaboration resulting in breakthrough science and life-saving results in the cure category.” Kite Pharma, now part of Gilead Sciences, took home the same prize at the 2016 CARE Awards for the ZUMA-1 trial of axicabtagene-ciloleucel in aggressive non-Hodgkin lymphoma, the trial that earned Yescarta an FDA approval less than two months after Kymriah. 

The Impact of Technology

In light of ongoing disruptions to the pharma industry, it’s no surprise that the technological development categories continue to grow and attract the largest number of entries year after year. The ever-evolving nature of the field and the need to develop and adapt new technologies to streamline the clinical research process are highlighted by the numerous candidates as well as the winners for the following two categories.

Medidata Solutions took home the prize for Best Sponsor-Focused Technological Development with their Strategic Monitoring Suite, an end-to-end solution that supports effective risk based monitoring (RBM) and workflow. This robust system impressed judges with the reduction in critical end of study cycle times by upwards of 40%, as well as a decrease in monitoring visits by 25% or more. Judges also noted the suite’s ability to address significant trial monitoring issues and the fact that this is the only technology that adheres both to ICH E6 guidelines and TransCelerate’s RBM Functional Requirements.

Through Bracket Mobile ePRO, winner of Best Patient-Focused Technological Development, patients are transformed into partners by engaging and empowering them throughout the entire clinical trial. A recent clinical study that evaluated drop-out rates found that patients who used the app were 80% more likely to complete the study than non-users, which judges deemed to be an impressive statistic. Judges also noted that the app provided a “good approach to bringing the patient into the heart of the trial with proven results that indicate increased subject retention and engagement which should result in higher quality data and improved data integrity for clinical trials.”

The Power of Teamwork

ICON took home the award for Clinical Research Team of the Year (sponsored by Oracle Health Sciences) with their AMAG Adult Iron Deficiency Trial. According to judges, the “processes and teamwork on display in this trial are both noteworthy and evidence of an evolving approach to a challenging program.” Despite the obstacles for this large Phase III trial in iron deficiency anemia, which included a compressed timeline and a black box warning for its drug, the team achieved key milestones earlier than expected, leading to the drug’s submission for regulatory approval nearly six months ahead of schedule.

Collaboration between companies was highlighted through Clinical Partnership of the Year (sponsored by Medidata), which went to Theravance Biopharma and Mylan for their global clinical development program for revefenacin. This partnership resulted in the development of the first once-daily, nebulized, long-acting muscarinic antagonist for the treatment of chronic obstructive pulmonary disease (COPD), which judges found to be an important therapeutic approach for an area with a large patient class and unmet need.

Lifetime Achievement

As the evening drew to a close, Pharma Intelligence honored Josef von Rickenbach, Chairman of the board, co-founder, and former CEO of PAREXEL, for his remarkable achievements and lifetime of service to the biopharmaceutical industry. Through this co-founder’s efforts over the past 35 years, PAREXEL grew from a small start-up in von Rickenbach’s basement in Watertown, Mass. to a global biopharmaceutical services organization, now with locations in 51 countries and a workforce exceeding 18,000.

Since its humble beginnings, PAREXEL underwent its IPO in late 1995 and more than 40 mergers and acquisitions, growing into its standing as a trail blazer in the biopharmaceutical services industry. Von Rickenbach’s vision and leadership transformed the clinical development process and created a now vital industry that brought a new type of partnership, providing standardization, efficiencies, and scalability as well as globalization.

As a testament to the essential partnerships clinical research organizations provide, the business von Rickenbach created has worked with all top 25 large biopharma companies, and with nearly 500 emerging companies, and has contributed to the development of 195 of the 200 top-selling biopharmaceuticals on the market today.

It is the distinct honor of Pharma Intelligence to celebrate and congratulate all the 2018 CARE Award winners, as well as to all the entrants who continue to inspire the industry through in their steadfast quest to create a new future through medical breakthrough and advances in the clinical trials process.

Pharma Intelligence would also like to thank the event sponsors for their generous support. In addition to ICON, the CARE awards are sponsored, in part, by Cytel, Medidata, Oracle Health Sciences, Syneos Health, and supported by MassBio. The ceremony, held at the Boston Harbor Hotel, was once again hosted by multi-media journalist Janet Wu, former anchor and health reporter for Boston’s NBC station and adjunct professor at Emerson College.

The 2018 CARE Awards Winners

Best in Health Economics and Outcomes Research

Merck KGaA, Merkel Cell Carcinoma (MCC) Observational Study for Historic Comparison

Most Successful Early Phase Research (Preclinical & Phase I)

AveXis, Phase 1 Clinical Trial of AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

Most Innovative Clinical Trial Design

Otsuka Pharmaceutical Development & Commercialization, Autosomal Dominant Polycystic Kidney Disease (ADPKD) Tolvaptan Team

Excellence in Rare Disease Drug Development

Alnylam Pharmaceuticals, Hereditary ATTR (hATTR) amyloidosis

Clinical Research Team of the Year

ICON, AMAG Adult Iron Deficiency Trial

Clinical Trial Result of the Year

Novartis, KYMRIAH™ (tisagenlecleucel) suspension for intravenous infusion

Best Sponsor-Focused Technological Development

Medidata Solutions, Medidata Strategic Monitoring

Best Patient-Focused Technological Development

Bracket, Bracket Mobile ePRO

Medidata Clinical Partnership of the Year

Theravance Biopharma and Mylan, Revefenacin

Marquee Award 'R&D Excellence'


Lifetime Achievement Award

Josef von Rickenbach, Chairman of the board, co-founder, and CEO of PAREXEL



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