FDAAA Innovator's Award
Theresa Mullin, PhD. Associate Director for Strategic Initiatives, FDA Center for Drug Evaluation and Research
For reforming ICH to significantly increase domestic/international drug manufacturer participation and being a strong advocate for Patient Focused Drug Development in PDUFA 5 and 6.
Commander (CDR) Tracy MacGill
Commander (CDR) Tracy MacGill, representing the FDA’s U.S. Public Health Service in the Office of the Chief Scientist, Office of the Commissioner
The FDA Alumni Association is nominating Commander (CDR) Tracy MacGill for the FDA Innovator Award. She was the lead for the Development of Medical Countermeasures (MCMs) section under FDA’s Broad Agency Announcement (BAA) for Advanced Research and Development of Regulatory Science to include animal model alternative platforms through a partnership with the Wyss Institute at Harvard University. Harvard University’s Wyss Institute for Biologically Inspired Engineering is a pioneer in the development of micro-physiological systems (MPS or “organs-on-chips) that mimic the structure, function, and interactions between the living tissues within human organs – such as the lung or intestine – on chips the size of a thumb drive. Under one of the first MCM-focused contract, Wyss Institute scientists are developing and refining in silico models of radiation damage in their lung, gut, and bone marrow organs-on-chips and using these models to test candidate MCMs to treat such damage. This will provide a capability to evaluate candidate medical countermeasures for acute radiation syndrome (ARS) within the specific context of a target human organ which will yield valuable mechanistic information for facilitating product develop, that is difficult to study in in animal models of ARS. These models are potentially reducing the number of animal studies needed for product development by supporting the selection of the most promising MCMs to be advanced for consideration.
CDR MacGill has worked closely with the Defense Advanced Project Agency (DARPA), who provided early support, to advance the MPS platform through MCM applications and with the Center for Food Safety and Nutrition (CFSAN) to support establishment of CFSAN’s FDA MPS Core Facility, allowing FDA scientists the opportunity to gain experience with this innovative platform.
Through CMD MacGill’s efforts, this research is advancing more rapidly and with US government commitment and certainty with the expected outcome being a more rapid assessment of medical procedures and medical drugs by confirming there efficacy, effectiveness and impact on various human tissues.
We are confirming that CMD Tracy MacGill meets the criteria for the FDAAA Innovator Award and has received the complete support of the FDAAA Board of Directors.
CITATION: In recognition of the leadership and commitment which allowed the Wyss Institute at Harvard University to advanced medical testing platforms for assessment of new human disease treatments.
Drs. Brandon Gallas and Marios Gavrielides, along with Drs. Darren Treanor (NHS, England) and Stephen Hewitt (NIH)
Drs. Brandon Gallas and Marios Gavrielides, along with Drs. Darren Treanor (NHS, England) and Stephen Hewitt (NIH), established the Whole Slide Imaging (WSI) working group, a collaborative community with wide participation from industry, clinicians, academia and government to address the many challenges associated with the performance assessment of the emerging class of medical devices referred to as whole-slide imaging (WSI) digital pathology systems. The working group chairs recognized the need for collaboration with external experts and organizations to share data and expertise. Together the working group is striving to co- develop new, least burdensome methods for the evaluation of whole-slide scanning systems that would support premarket applications to the FDA, as well as the study of these systems for other purposes (in research settings as well as after they have been in long-time clinical use following FDA’s approval for marketing).
During the last 10 years, significant technological advances have allowed digital scanning systems to rapidly and automatically digitize large numbers of pathology slides at high spatial resolution. This in turn has facilitated the development of many unique applications making use of the digital pathology slide data in support of clinical decisions, especially through the use of machine learning and pattern recognition techniques. However, it wasn’t until this past year that FDA granted marketing approval for the first ever WSI system. The reason for this is that the evaluation of digital pathology systems is especially challenging because of the crucial role the microscope plays in medicine: pathology interpretations provide definitive diagnoses, drive treatment, and serve as the reference standard for evaluating treatments and diagnostics). Thus the risk associated with replacement of the optical microscope with a potentially lower-quality digital system is understood to be high. It must also be appreciated that there is a very wide range and diversity of pathology clinical tasks, as well as an expected large variety in the approaches vendors might take in the engineering design of their digital systems in terms of the image acquisition, processing, and display components.
The overarching goal of the WSI working group is to develop methods for properly characterizing WSI devices and components with systematic technical measurements and validation studies that will allow the clinical utility of digital pathology to be maximized. The group has been extremely well subscribed, with over 130 current members. More importantly, the group has been highly successful in facilitating collaboration across the digital pathology industry in a pre-competitive environment. The WSI working group has hosted multiple webinars and town halls at pathology conferences where ideas are shared, to the extent that vendors have been willing to discuss planned study designs in a public forum, much like a public grant review or study section, and obtain feedback from the entire community prior to any resource expenditure on their study. The WSI working group has become a widely recognized forum for the exchange of ideas, development of consensus strategies, and benchmarking of methods to assess the image quality of WSI systems. The efforts of Drs. Gallas and Gavrielides, as founders and leaders of the WSI working group have greatly benefited the research and industrial community associated with the design, development, and use of WSI systems. Their efforts benefit the working group members and nonmembers alike, as all the WSI working group discussions and materials are publicly available at this website: https://nciphub.org/groups/wsi_working_group. Most importantly, through the leadership of Drs. Gallas and Gavrielides, patients benefit by the development and dissemination of best practices for the evaluation of WSI systems, which naturally leads to the design and availability of improved devices.
Dr. Jaview Revollo
Scientific breakthroughs have made genome engineering possible. Potential applications of these technologies include the editing of patients’ genetic material (DNA) and could lead to therapies against cancer and AIDS. The safety of genome engineering technologies, however, has yet to be determined, particularly because classic genetic toxicology assays are inadequate to determine the off-target mutations (i.e., unintended mutations) that results from genome engineering. Off-target mutations, even if rare, could have significant adverse health consequences if, for instance, they occur in carcinogenesis-associated genes.
To fill the gap in the FDA’s ability to assess the safety of genome engineering therapeutics, Dr. Javier Revollo developed a method that comprehensively identifies mutations genome-wide. The method works comparing whole genome sequencing datasets from single-cell-derived clones, and is an integral in two projects aimed at assessing the safety of genome engineering therapeutics. The first is an NCTR project titled “Methods to determine off-target effects of CRISPR-mediated genome engineering in mammals” and the second is a Chief Scientist’s Challenge Grant titled “In vitro and in vivo methods for functional evaluation of genomic alterations induced by genome editing.” Dr. Revollo’s newly developed methods have been fundamentally important to researchers trying to assess the safety of genome engineering to develop new therapeutics which do not unintentionally cause harm. We are confirming that Dr. Jaview Revollo meets the criteria for the FDAAA Innovator Award and has received the complete support of the FDAAA Board of Directors.
CITATION: In recognition of the development of a method that detects mutations genome-wide to enable the FDA to assess the safety of genome engineering therapeutics.
ShaAvhrée Buckman Garner, Director of CDER Office of Translational Sciences
The FDA Alumni Association is nominating CDER’s “DASH” Team for the FDA Innovator Award. The Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) is required to keep detailed records of the drug development review and approval process in order to monitor the agency’s performance and respond to internal and external queries, including congressional requests. The required data existed in a variety of electronic and paper formats, making it difficult and time-consuming to obtain and query accurate data for the required analyses. To begin to address this problem, CDER previously developed a small database for verified clinical development and regulatory information.
However, the current small database had performance, capacity, and security constraints which limit its utility. To better support the growing number of users reliant on these data, CDER established a team to work with the FDA National Center for Toxicological Research (NCTR) to develop an Oracle-supported large capacity database application compatible with FDA’s enterprise infrastructure and information technology standards. This application, called the “Data Analysis and Search Host (DASH)”, will provide a robust and secure platform for monitoring and querying the progress of Marketing Applications, and provide critical information needed to improve the application process and ultimately reduce the time between initial application and final approval. The CDER DASH Team have been addressing the planning as well as the details of converting the small existing database of information into the larger DASH database for access by many FDA and other outside, authorized, scientific bodies. We are confirming that CDER’s “DASH” Team meets the criteria for the FDAAA Innovator Award and has received the complete support of the FDAAA Board of Directors.
CITATION: For its work on the Data Analysis Search Host (DASH), an information-rich scientific database containing Marketing Application information on new molecular entities (NME) and biologics. DASH Team Members:
- CDER Leader and Members: ShaAvhrée Buckman Garner (Director of CDER Office of Translational Sciences), Salvatore Pepe,Ji Feng. Shahrukh Haider, David Guenther, Gregory Jackson, Nisha Bruce
- NCTR Leader and Members: Weida Tong (Director of NCTR Bioinformatics and Biostatistics), Joe Meehan, Jae Hyun Kim, Weizhong Zhao, Steve C. Harris, Suresh Subramani
Dr. Carlos Pena, head of CDRH’s Division of Physical Medicine and Neurological Devices (DNPMD)
Dr. Pena’s leadership, innovative thinking, and focus on patients and collaboration has significantly advanced the Mission of FDA. Under his leadership, DNPMD has transformed the manner in which they conduct their business in order to implement CDRH’s “Vision” of bringing high quality, safe and effective medical devices of public health importance, first to US patients in need of such devices. Dr. Pena has accomplished this first and foremost by focusing on patients. He recognizes that patients are at the heart of FDA’s Mission and it is critically important that FDA staff understand patient perspectives and engage with them to improve their understanding of the patient experience. He has established monthly disease-focused patient engagement seminars for his staff to ensure that they interact with patients, and consider patient perspectives in their work. Dr. Pena has fostered collaboration among regulators, patient organizations, health care providers, industry and other government agencies. He has transformed DNPMD’s approach to the review and oversight of clinical trials, including early feasibility studies, to help make the U.S. a more attractive environment for novel technologies and device development. These actions have started to have a noticeable impact as a number of important, novel neurological devices reaching U.S. patients has increased dramatically during his tenure. A few examples include:
- The first focused ultrasound device to treat essential tremor in medication-refractory patients;
- Clot retrieval devices as an initial therapy for strokes due to blood clots, in order to reduce paralysis, speech difficulties and other stroke-related disabilities;
- First-of-a-kind computerized tests to assess a patient’s cognitive function immediately after a suspected brain injury or concussion;
- A “halo” device that utilizes low-intensity electrical fields to treat patients with an aggressive form of brain cancer, extending the expected life of these patients by years, in some cases.
As a result of the many positive contributions made by Dr. Pena, the FDAAA is nominating him to be receive the FDAAA Innovator Award.
CITATION: In recognition of innovative thinking and management which resulted in increased acceptance of new medical device technology, improving the lives of countless US patients
Transfusion Transmitted Infection Monitoring System (TTIMS) Team led by Steven Anderson and Alan Williams US Food & Drug Administration’s Center for Drug Evaluation and Research (CDER)
Mr. Anderson and Mr. Williams representing a group at FDA’s Center for Drug Evaluation and Research (CDER) that established the revolutionary “Transfusion Transmitted Infection Monitoring System (TTIMS)”. The development of this system was led by CBER, in collaboration with the National Heart, Lung, and Blood Institute (NHLBI), the Center for Disease Control (CDC) and the Office of the Assistant Secretary for Health (OASH) and industry partners. The TTIMS as developed, is the first nation-wide real-time monitoring system of the blood supply to be established in the United States. It has the ability to collect information on more than half of the blood collected voluntarily in the United States and will both facilitate protection of the blood supply against infectious diseases and help FDA understand whether its blood donation policies can be further refined.
As a result of the landmark efforts by Mr. Anderson and Mr. Williams, the FDAAA is nominating both since they led the Team that developed the TTIMS. TTIMS will make significant advancement in public health and significantly reduce the risk of contracting blood-borne disease transmitted through the US blood supply to individuals in need of blood transfusions. Both together meet the criteria for the FDAAA Innovator Award and have received the complete support of the FDAAA Board of Directors.
CITATION: In recognition of the leadership demonstrate to successfully build the TTIMS by integrating the views of various government and industry partners.
FDA’s Center for Tobacco Product’s (CTP’s) Human Capital Team led by Executive Officer Janelle Barth.
The FDA Center for Tobacco Product’s (CTP’s) has over the past year developed new and innovative approaches to create in-house automated solutions to build workforce hiring plans and staffing plans. The use of these innovative human resource approaches also allows the tracking of metrics in real-time by monitoring actual program which motivates CTP supervisors and management to keep the hiring plans moving forward appropriately. As a result, CTP has the fastest hiring metrics at FDA (68 days on average in FY16) while the Agency model is slower (80 days on average), with some parts of FDA even slower ( over 100 days on average in FY 16). In addition, they are leading corporate recruitment efforts for the agency by leveraging public platforms not traditionally used by the US government and establishing partnerships with universities and interest groups to quickly identify qualified candidates. Lastly, they found a way to mine resumes in USAJobs to proactively identify candidates that may be eligible for future CTP vacancies and then sharing those potential opportunities with these qualified individuals.
The CTP Human Capital Team, led and directed by Janelle Barth has through initiative, energy and use of new and innovative human resources tools and partnerships, managed to identify qualified employment candidates, assist CTP managers and supervisors in moving rapidly through the necessary paperwork to qualify and employ qualified individuals for open positions which makes a significant contribution to the work capacity and efficiency of operation of the CTP. This human resources model is an example of an internally developed and successful program that should migrate to other FDA Centers to improve the hiring metrics and directly contribute to the overall ability of these Centers to deliver on their mission(s). The complete Team along with Ms. Janelle Barth meet the criteria for the FDAAA Innovator Award and have received the complete support of the FDAAA Board of Directors.
CITATION: In recognition of the development and implementation of innovative human resource techniques and tools to increase Agency hiring metrics leading to improve CTP operations.
FDA Alumni Advisor Design Group. This was a group award for the following FDA employees who successfully developed a process to re-hire FDA retirees to mentor current employees in leadership skills and career development, having an immediate and positive impact on FDA.
- Patricia Alcock - Director, Division of Human Resource Development, ORA
- Loney Nunemaker - Deputy Director, Division of Human Resource Development, ORA
- Brooke Mullican - Manager, Division of Human Resource Development, ORA
- Denise Collins - Program Lead/Training Officer, Division of Human Resource Development, ORA
- Rosemary Warneke - Consultant, Office of Resource Management, ORA
These employees worked collaboratively to design a process to re-hire twelve former senior FDA employees who had retired with many years of management and leadership experience, and to return them on a part time basis as rehired annuitants. Their areas of strength were identified and they were matched with all level managers from the Office of Regulatory Affairs (ORA) who had identified the same leadership competencies as developmental needs. The resulting advisor/learner relationships were nurtured in a pilot program called the FDA Alumni Advisor Program that began in June 2015 and concluded in March 2016.