Translational Medicine Case Studies - InnovationWell 2015

“Translational medicine”, “personalized medicine”, “precision medicine” and “Big Data” are all terms used to describe research efforts to improve healthcare, lower its cost and improve quality of life.

Case studies of pediatric ARDS and breast cancer risk, diagnosis and treatment will be presented through the perspectives of the patient, physician, pharma, payer, regulatory agency and provider to show the value of collaboration across traditional boundaries.

This session includes an introduction to these issues and two case studies that exemplify how collaboration across the entire healthcare ecosystem is necessary to create clinical and commercial solutions.


Redefining Translational Medicine: Addressing Unmet Clinical Need, M. Liebman (IPQ Analytics, LLC, Chair, PhRMA Advisory Board on Translational Medicine and Therapeutics)

An expanded view of translational medicine, as adopted by PhRMA, will be presented along with fundamental issues that can impact the translation of research results into clinical use.  This will include the presentation of disease process modeling its use in identifying critical issues of unmet clinical need and unstated, unmet clinical need.  The impact on Comparative Effectiveness Research will also be presented.

Case Study 1:

Breast Cancer: From Risk to Treatment-Understanding the Personal Issues of Risk and Management, Sabrina Molinaro, Institute for Clinical Physiology, National Research Council of Italy (Pisa)

Personalized risk and disease analysis requires understanding the real world patient and their clinical and personal history as well as their genomic makeup.  This study focuses on the risk and disease of an individual rather than statistical analysis of large databases, to enable the physician to address the patient in a more precise manner.  Results of studies done with the regional healthcare system and Online Data Integration Network (ODINet) will be presented along with the physiological model for breast cancer development.  Current extension to US populations, through community health systems, will be described.

Case Study 2:

Pediatric ARDS: The Challenges and Opportunities in Rare Disease Research and Treatment, Iris Melendez (President, Nathaniel Adamczyk Foundation (NAF) and Michael Liebman (IPQ Analytics, LLC)

Pediatric ARDS (Acute Respiratory Distress Syndrome) is a devastating (~50% mortality) disease without known risk factors, and diagnosed only after presentation in the pediatric ICU.  NAF’s interest in identifying risk factors is tempered against the intensivists focus on patient survival.  This effort has evolved towards creation of a national tissue and data resource, in collaboration with IPQ, CNR, PALISI (Pediatric Acute Lung and Sepsis Investigators) and the Virtual PICU (CHLA) with an analytical interface to support clinical research and clinical decision support.  Initial results highlight the need to recognize unmet clinical need rather than the allure of applying new technologies and suggest a new model for patient advocacy groups to impact their patients and families.  This effort is being further expanded to include centers in China.  The overall effort, collaborations and approaches to this complex clinical problem will be presented.