Tim Cote, M.D., M.P.H. and Richard Rodgers, M.D.

Office of Orphan Products Development
Office of the Commissioner
Rockville, MD

Proposed Research Project for FDA Fellow:

With personalized medicine and advances in the understanding of the genetic basis of disease, pharmaceutical products are likely to be increasingly developed for smaller patient populations. We are taking an epidemiologic approach to studying the 25-year experience of the orphan products program that currently has ~2000 orphan product designations. We hope to glean evidence that will increase the number and development speed of new products that will help patients with the ~6000 rare diseases and conditions. Investigational New Drugs (INDs), New Drug Applications and Biologics License Applications, as well as orphan product designation requests and annual reports are the substrate for our research. Analyses of confidential data can be presented in summary form that allow their use in reports that can be published in the peer-reviewed literature—a feasible goal for this fellowship position. Currently our research is descriptive, but we expect to embark on analytic studies (e.g., survival analysis, case-control, cohort studies). We also are initiating an international collaboration.  Given the vast area of our work, there is potential to incorporate the fellows' experiences and training into the proposed research project.

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John W. Gardner, M.D., Dr.P.H. 

Office of Information Management, OC
Rockville, MD

Background:

M.S., Physical Chemistry & Biochemistry, Brigham Young University
M.P.H., Epidemiology & Biostatistics, Harvard University
M.D., University of Utah
Dr.P.H., Epidemiology & Biostatistics, Harvard University
FDA Experience - 4 years

Research Interests:

Data systems for epidemiologic research and regulatory purposes to enhance the public health protection of the public.

Proposed Research Project for FDA Fellow:

This project would be for a medical informatics fellow to work on integrative scientific computing issues for FDA data systems. There are many aspects of FDA system integration and scientific computing efforts, which are all open to the fellow for project development. A key FDA integrative data concept is called JANUS, which is intended to house all structured data (according to data standards, eg, Health Level Seven) received by FDA. This would include clinical and non-clinical study data, adverse event reports, facility registrations, and product listings, among others. The concept of the JANUS system is to manage structured data at multiple levels – Exchange (receive HL7 messages), Persistence (load, validate and store data), Database (user-friendly databases), Data Mart and Special Purposes (data organized for analytic use), analysis (tools for analysis), and results (outputs of analyses). The fellow would work with FDA business and IT professionals in integration efforts on projects such as developing, testing, and implementing the JANUS model, defining its benefits and risks, and exploring and evaluating its utilization for various specific purposes.

Selected Recent Publications:

1. Gardner JW: Fatal Pneumococcal Meningitis: Failure of Acute Care Focus of Medical Practice. Military Medicine, Aug 2007; 172(8): 902-905.
2. (Contributing Editor): Force Health Protection. Vision Document, Offices of the Joint Chiefs of Staff and Secretary of Defense, 2003, 53 pages.
3. Scoville SL, Gardner JW, Magill AJ, Potter RN, Kark JA: Nontraumatic Deaths during U.S. Armed Forces Basic Training, 1977-2001. Am J Prev Med, 2004; 26(3): 205-212.
4. Scoville SL, Gardner JW, Potter RN: Traumatic Deaths during U. S. Armed Forces Basic Training, 1977-2001. Am J Prev Med, 2004; 26(3): 194-204.
5. Gardner JW, Kark JA: Clinical Diagnosis, Management and Surveillance of Exertional Heat Illness. In: Pandolf KB, Burr RE, eds. Medical Aspects of Harsh Environments, Volume 1… 2001: ch 7, pp 231-280.
6. Gardner JW, Gibbons RV, Hooper TI, Cunnion SO, Kroenke K, Gackstetter GD: Identifying new diseases and their causes: The dilemma of illnesses in Gulf War veterans. Military Medicine, Mar 2003; 168: 186-193.
7. Potter RN, Gardner JW, Deuster PA, Jenkins P, McKee K Jr, Jones BH: Musculoskeletal Injuries in an Army Airborne Population. Military Medicine, Dec 2002; 167: 1033-1040.
8. Gutmann FD, Gardner JW, Potter RN, Kark JA: Nontraumatic Exercise-related Deaths in the U. S. Military, 1996-1999. Military Medicine, Dec 2002; 167: 964-970.
9. Gardner JW: Death by Water Intoxication. Military Medicine, May 2002; 167: 432-434.
10. Gardner JW, Cozzini CB, Kelley PW, Kark JA, Peterson MR, Gackstetter GD, Spencer JD: The Department of Defense Medical Mortality Registry. Military Medicine, 2000; 165, Suppl. 2: 57-61.

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Norman S. Marks, M.D., M.H.A. 

Medical Director
MedWatch Program
Office of Scientific and Medical Programs
Office of the Commissioner
Rockville, MD

Background:

B.Med.Science, University of Wisconsin-Madison
M.D., University of Wisconsin-Madison
M.H.A., University of Colorado

Research Interests:

Since 2000, Dr. Marks has lead the team managing the FDA’s MedWatch Safety Information and Adverse Event Reporting Program, an outreach program to both healthcare professionals and patients that uses social marketing methods to support the agency's activities in post-market medical product safety surveillance for all human healthcare products regulated by FDA. Prior to coming to the MedWatch position, Dr. Marks was a medical officer in CDER's Division of Reproductive and Urological Drug Products. From 1977 to 1998, he practiced urological surgery in community-based private practice and integrated group practice settings in the Midwest. He also served in the US Public Health Service, Indian Health Service as a general medical officer in western South Dakota.

Dr. Marks' professional interests focus on risk communication and biomedical informatics, with a specific interest in studying the application of clinical medical informatics theory and methodologies to drug and device risk management. Research on enhancing medical product risk identification through access to clinical electronic health record data and the use of clinical decision support tools and applications to incorporate new safety information and best practices for use of medical products into point-of-care clinical decisions are of particular interest.

Since its inception in 1993, the MedWatch program has supported two specific agency risk management goals: To increasing the quality and timeliness of the reporting process from healthcare professionals to FDA for serious post-market safety adverse events (risk identification), and to develop and upgrade the quality and timeliness of dissemination of actionable and clinically important new safety information that results from post-market surveillance of all human medical products regulated by FDA (risk communication).

With the passage of the FDA Amendments Act of 2007 [FDAAA], FDA received both the authority and a mandate to develop new methods for identifying post-market safety events for drugs and devices and to develop new methods for, and evaluation of, the risk communication process for communicating safety information on regulated drug products.

MedWatch has an interest in research that contributes to a better understanding of the extent to which new risk information about regulated products that are disseminated to MedWatch Partners organizations for further dissemination to members and subscribers is received, understood, and acted on by recipients and end-users. FDA is beginning to develop a research capacity, based in the social or behavioral sciences, to assess this process of communicating safety information to point-of-care providers and patients. This qualitative and quantitative research would assess the effectiveness of the messages we communicate - how they are being received and understood, and how they could more optimally be disseminated. The findings will help FDA design communications to more effectively meet the decision-making needs of our provider audiences.

Proposed Research Project for FDA Fellow:

An FDA Fellow participating in this research program will be exposed to all aspects of FDA risk communication practices for human healthcare[medical] products regulated by the three human medical product centers [ drugs, biologics, and medical devices]. The Fellow will be offered the opportunity to learn the specifics of how FDA applies the theory and practice of human healthcare risk communication. The Fellow will be asked to select a more focused project on some aspect of risk communication and to develop and implement an original evaluation of current agency communication processes and/or design pilot interventions to study potential new ways to communicate safety information more effectively. Fellows interested in working in this research domain will be expected to use both qualitative and quantitative data collection methodologies to evaluate the knowledge, values, attitudes, beliefs and needs of individual physicians, other clinician providers, and professional specialty organizations as they relate to drug safety risk communication. Some previous experience in, or an interest in gaining a working knowledge of, social science research methodologies is preferred.

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