In 2016, the Clinical Research & Excellence (CARE) Awards were launched to recognize the people and teams driving drug development, which continually grows in its complexity, especially within times of uncertainty. Since the inaugural ceremony, R&D pipelines continued to grow in size, but at a reduced rate of expansion. In parallel, the number of drugs successfully launched during 2016 fell back from the highs seen in the prior two years. Despite the obstacles, the industry remains committed to bringing new medicines to market and overcoming the numerous unmet medical needs in healthcare. As such, Pharma Intelligence returned to the State Room in Boston, to once again pay tribute to those who embody the human element of clinical and research excellence during the second annual CARE Awards ceremony, which was hosted by the multi-media journalist, Janet Wu, former Anchor and Health Reporter for Boston’s NBC station and adjunct professor at Emerson College.
The awards honour the contributions of pharmaceutical, biotech and allied industries and organizations, highlighting key activities driving success in drug development. The categories selected for the 2017 CARE Awards, sponsored by ICON, celebrated a range of key R&D activities, and included two new categories to recognize notable achievements in rare disease drug development as well as patient centricity in trials. Throughout the evening, the spotlight clearly shined on industry activity within Oncology research. Although we are entering an era where more effective treatment options are available, this is only limited to specific patient populations and the need for ongoing oncology R&D remains for the many untreated, or refractory, cancer patients. The numerous CARE Award winners for various efforts in this area offers hope that additional options will continue to emerge and transform deadly cancers to manageable, or perhaps curable, diseases.
Transforming the clinical trial process
Best Patient Centric Clinical Trial went to Janssen Research & Development for their PRELUDE study, which evaluated the safety and clinical response of sub-retinal administration of CNTO 2476 (Palucorcel) in patients with geographic atrophy. A novel surgical procedure, surgical device and cell therapy for age-related macular degeneration are evaluated in this ongoing clinical trial, where patient centricity was central to the design and execution, aiding with recruitment and retention. Prior to the trial’s launch, Janssen directly engaged with patients and incorporated their insights into the planning process by conducting an in-depth trial simulation workshop with actual patients, which informed operational enhancements. In addition, an electronic consenting method, which included explanatory videos and a self-assessment quiz, was implemented to help patients better understand the trial during the recruitment process. Judges appreciated the practical application to patient needs, and felt that more work within this area would be very valuable.
Tracon Pharmaceuticals took home the prize for Most Innovative Clinical Trial Design with the TAPPAS study, “A Randomized Phase III Trial of TRC105 and Pazopanib Versus Pazopanib alone in Patients with Advanced Angiosarcoma”. The Phase III trial was developed with the support of Cytel and their expertise in biostatistics and innovative trial design and leverages the power of precision medicine, targeting a specific sub-population of patients with advanced angiosarcoma, a rare and deadly cancer. Tracon’s confirmatory Phase III trial, which has received Special Protocol Assessment approval from the FDA, impressed judges with its important application of a ground-breaking adaptive enrichment design.
Making waves in the R&D landscape
The Excellence in Rare Disease Drug Development award went to Tocagen for Toca 511 + Toca FC, their investigational therapeutic combination for patients with recurrent high grade glioma. The new approach developed by Tocagen includes a cancer-selective gene therapy platform that utilizes a modified retroviral replicating vector for delivery (Toca 511), as well as an investigational, orally administered, proprietary, extended-release version of the well-established anti-cancer agent, 5-fluorocytosine (Toca FC). The combination possesses both orphan drug and Fast Track designations, and has demonstrated positive data for this rare form of brain cancer, which judges felt would also likely have implications in a broader group of cancers. As is generally common within the orphan drug space, Tocagen has worked closely with various non-profit organizations and brain cancer advocacy groups to weave patient centricity into the development of Toca 511 + Toca FC, and facilitate rapid enrollment into their ongoing Phase II/III international trial.
Tocagen also took home a trophy for Most Successful Early Phase Trial (Preclinical & Phase I) with their Phase I trial of Toca 511 + Toca FC, which judges found to be “an exciting and creative approach” for this extremely difficult to treat cancer with a correspondingly high morbidity. The results for the Phase I trial was highlighted as the June 2016 cover for Science Translational Medicine, and demonstrated durable complete and partial responses, extended survival compared to an external control, and favorable safety.
Another anti-cancer gene therapy was recognized for the Clinical Trial Result of the Year award (sponsored by Medidata), which was received by Kite Pharma for their pivotal ZUMA-1 trial. This noteworthy trial evaluates axicabtagene ciloleucel, Kite’s chimeric antigen receptor T cell (CAR-T) therapy and is the first multicenter pivotal CAR-T trial for aggressive non-Hodgkin lymphoma (NHL). Judges referred to the ZUMA-1 results as “remarkable”, particularly in light of historical benchmarks established by the SCHOLAR-1 meta-analysis in chemorefractory diffuse large B-cell (DLBCL) cancer patients, the same NHL sub-population included in ZUMA-1. Interim ZUMA-1 data demonstrated an almost six-fold higher complete remission (CR) rate compared to the best CR rate from SCHOLAR-1. Judges also acknowledged that Kite has developed a “unique new treatment paradigm”, which could be the first CAR-T to market this year and transform the immunotherapy landscape.
Innovations through technology
In a similar vein to last year, the technological development categories garnered the largest volume of entries, confirming the industry’s ongoing commitment in advancing technology to optimize and facilitate best practices in clinical research. Efforts focused on facilitating better management and oversight of clinical trials, as well as technological innovations to improving patient data collection and/or the patient experience in clinical trials were recognized in the following two awards.
First, the award for Best Sponsor-Focused Technological Development honored the partnership between man and machine, and went to IBM Watson Health & Novartis & Highlands Oncology, deemed by the judges as a “powerful, forward-looking approach anticipating the convergence of clinical care and clinical research driven by electronic health and medical information.” Watson is a technology platform that applies natural language processing and machine learning to generate insights from large, sometimes unstructured, datasets, which was leveraged by the Watson Clinical Trial Matching Team to increase efficiencies in recruiting cancer patients into trials and dramatically reduced the patient screening workload. Patient recruitment in general can be one of the most challenging aspects of the clinical trial process, particularly for cancer research. The complexity is further impacted by the growing movement toward personalized medicine that requires identification of highly specific patient populations. The team integrated Watson into the electronic medical record (EMR) system, containing both structured and unstructured patient data, to quickly check eligibility across relevant clinical trials, increasing the productivity and accuracy of patient screening.
Next, NorthWest EHealth (NWEH) took the prize for Best Patient-Focused Technological Development with their Linked Database System (LDS). NWEH, a limited company developed from a partnership between the University of Manchester, Salford Royal Foundation Trust, and Salford Clinical Commissioning Group, developed the LDS to link consenting patients’ electronic health records across all healthcare related interactions to monitor patient safety in near real-time. This near real-time safety monitoring capability enables the collection of real-world evidence into clinical research, creating an opportunity for study designs that could not be delivered using standard randomized controlled trials. No other technology with the capabilities of LDS exists on the market, with the judges stating that they “have not ever seen anything quite this novel and unique.” and this revolutionary tool was first used in the pioneering Salford Lung Study (sponsored by GlaxoSmithKline).
Shining stars in collaboration
Collaborations, both between companies or within an individual company, are an intrinsic component of pharma R&D, and were celebrated by the CARE Awards in the following two categories.
The power of teamwork was first recognized in the Pharma/Biotech Product Development Team of the Year (sponsored by Cytel), which went to TESARO for the Niraparib Product Development Team. Due to this team’s diligence and rapid execution, niraparib, a potential new treatment for ovarian cancer, may soon be available to thousands of patients worldwide. While most patients are responsive to standard therapies, up to 85% will experience incurable recurrence of their disease after first-line treatment. Through the team’s efforts and commitment to the possibility of getting a new treatment option to ovarian cancer patients as soon as possible, nirapirab was successfully brought from being in-licensed to filing NDA and MAA submissions within five years. Judges acknowledged the efforts of the “well oiled team that would need to be in place to have such an extraordinary series of milestones.” As a result of the team’s efforts and the overwhelmingly positive results, the standard of care for ovarian cancer could potentially be redefined now that the drug has received FDA approval as of March 27, 2017, three months ahead of its scheduled PDUFA date.
Successful partnerships between companies were also celebrated in the Clinical Partnership of the Year award, which was won by Medidata Solutions and TESARO. In an age of rising R&D costs and complexities in clinical development, adaptive clinical trials are a key strategy to improve study productivity while conserving trial resources. Medidata Balance facilitates the execution of these types of trials, as a unified Randomization and Trial Supply Management (RTSM) solution that enables users to pool clinical supply inventory across multiple trials at the same site. With key input from TESARO, Medidata updated the software to design a customized solution that met TESARO’s needs in supporting the implementation of multiple immuno-oncology trials, deriving efficiencies to decrease costs, shorten timelines, and, ultimately, bring better treatments, and hope, to patients sooner.
An honorable mention for Clinical Partnership of the Year was given to the Clinical Pharmacology Capability Building in Sub-Saharan Africa project that involved Novartis Pharma, Kenya Medical Research Institute Center for Clinical Research/Strathmore University, Kintampo Health Research Center, Ifakara Health Institute, Division of Clinical Pharmacology at the University of Cape Town, Addis Ababa University/Regional Bioequivalence Center, and African Institute of Biomedical Science and Technology. Since 2012, this partnership between Novartis and Africa-based research institutions has enabled the management and execution of regulated clinical pharmacology trials in Africa. This type of research is much needed to better understand local intrinsic and extrinsic factors that could influence drug response in African populations that comprise about 15% of the world’s population and more than 20% of the global burden of disease. Activities were tailored to each individual clinical research unit, and were not linked with ongoing Novartis drug discovery or development projects.
The reigning leader of R&D excellence
As the ceremony neared its end, Novartis was once again crowned as the Marquee Award 'R&D Excellence' winner. Novartis was selected by the Pharma Intelligence teams in recognition of their outstanding achievement across the full spectrum of drug development in 2016, determined by performance across key R&D metrics, spanning a range of business activities, which are included in the CARE Index. In particular, Novartis’ commitment in bringing drugs to market faster for unmet medical needs by proactively seeking expedited designations for numerous assets from their vast portfolio stood out. Other notable accomplishments include the volume of Phase I to IV clinical trials that were initiated 2016, as well as their pipeline size. In fact, Ian Lloyd’s 2017 R&D review found that Novartis had moved up to the top position among companies with the largest number of active drugs in their pipeline.
The evening culminated with the presentation of the Lifetime Achievement Award to Dr. Michael Murphy, Chief Medical & Scientific Officer at Worldwide Clinical Trials, whose 25-year long career within the pharmaceutical industry has consistently integrated medical and scientific acumen with operational excellence. Dr. Murphy is considered to be a true visionary within the clinical trial industry and is widely recognized for his dedication and passion in driving innovation. He has been instrumental in building Worldwide Clinical Trials into one of the industry’s leading CROs since its establishment in 1995, working tirelessly to develop the company’s consulting services for protocol and program design. According to his colleagues, “his passion, hard work and unwavering commitment inspires those around him to do the same.”
Pharma Intelligence is honored to extend hearty congratulations to all the 2017 CARE Award winners, as well as to all the nominees who, in their own unique ways, have made important contributions to advancing human health.
The 2017 CARE Awards Winners
|Best Patient Centric Clinical Trial
||Janssen Research & Development, A Study to Evaluate the Safety and Clinical Response of Subretinal Administration of CNTO 2476 (Palucorcel) in Participants with Geographic Atrophy (PRELUDE)
|Clinical Trial Result of the Year
Kite Pharma, ZUMA-1 Pivotal CAR-T Therapy Trial of Axicabtagene Ciloleucel in Patients with Aggressive Non-Hodgkin Lymphoma (NHL)
|Most Innovative Clinical Trial Design
|Tracon Pharmaceuticals (in collaboration with Cytel), A Randomized Phase 3 Trial of TRC105 and Pazopanib Versus Pazopanib alone in Patients with Advanced Angiosarcoma (TAPPAS)
|Excellence in Rare Disease Drug Development
Tocagen, Toca 511 + Toca FC for recurrent high grade glioma
|Best Sponsor-Focused Technological Development
IBM Watson Health & Novartis & Highlands Oncology, Watson Clinical Trial Matching Team
|Best Patient-Focused Technological Development
||NorthWest EHealth, Linked Database System
|Most Successful Early Phase Trial (Preclinical & Phase I)
||Tocagen, Study of a Retroviral Replicating Vector to Treat Patients Undergoing Surgery for a Recurrent Malignant Brain Tumor
|Pharma/Biotech Product Development Team of the Year
||TESARO, Niraparib NOVA Trial
|Clinical Partnership of the Year
||Medidata Solutions & TESARO
| Marquee Award 'R&D Excellence'
|Lifetime Achievement Award
||Dr. Michael Murphy, M.D., Ph.D. Chief Medical & Scientific Officer, Worldwide Clinical Trials
Ian Lloyd’s “Pharma R&D Review 2017” is available at https://goo.gl/T3LLQR