TRANSLATIONAL MEDICINE & ADAPTIVE TRIAL DESIGN
Advancing critical care by advancing R&D
Translational medicine and adaptive trial design have emerged as more efficient alternatives to traditional research and drug development (R&D). Translational medicine seeks to bring promising compounds from the lab to the patient with great confidence earlier in the development phase, while adaptive trial design involves the adaptation of clinical trial protocol based on data gathered before or during the trial with the goal of improving trial outcomes.1,2
- Translational medicine often is referred to as “bench-to-bedside” research. Rather than progressing a new drug or device from preclinical research through traditional Phase I, II, and III development, translational medicine focuses on much earlier patient-oriented research1
- Adaptive design incorporates interim data from a clinical trial and adjusts the trial design based on these data to ensure richer outcomes based on a deeper understanding of disease pathophysiology. Predefined changes to trial design ideally are stated prior to the initiation of the trial2
We're in good company
At Ikaria, we embrace both these concepts at every stage of research. We’re in good company. These strategies are strongly supported by both the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH).
- In its Critical Path Initiative, the US FDA seeks to modernize the process of developing and evaluating product development, including adaptive trial design3-5
- The NIH is advancing translational research with its Roadmap for Medical Research6
Looking for smart ways to lower costs
The development of a new drug is estimated to cost approximately $1.2 billion and take 10-15 years, on average. Only an estimated two out of every 10 marketed drugs match or exceed R&D costs.7
Strategies such as adaptive trial design and translational medicine have the potential to reduce these costs by facilitating more efficient clinical trials. Additionally, they provide the resources to determine at a significantly earlier date which formulations are likely to fail in later trials, potentially saving organizations substantial money and time.1,2
The need is great
This all means that Ikaria is focused on earlier patient research and accelerated drug delivery, because critically ill patients can’t wait for a traditional approach to R&D.
At Ikaria, when we say we care about critical care, we mean it.
- Littman BH, Di Mario L, Plebani M, Marincola FM. What’s next in translational medicine? Clin Sci. 2007;112:217-227.
- Chow SC, Chang M. Adaptive design methods in clinical trials -- a review. Orphanet J Rare Dis.2008;3:11.
- US Food and Drug Administration. FDA’s critical path initiative -- science enhancing the health and well-being of all Americans. January 2007. http://www.fda.gov/oc/initiatives/criticalpath/initiative.html. Accessed May 11, 2009.
- US Food and Drug Administration. Critical path opportunities list. March 2006. http://www.fda.gov/oc/initiatives/criticalpath/reports/opp_list.pdf. Accessed May 11, 2009.
- US Food and Drug Administration. Critical path opportunities initiated during 2006. http://www.fda.gov/oc/initiatives/criticalpath/opportunities06.html. Accessed May 11, 2009.
- National Institutes of Health. NIH roadmap for medical research: re-engineering the clinical research enterprise. Translational research. http://www.nihroadmap.nih.gov/clinicalresearch/overview-translational.asp. Accessed May 11, 2009.
- Pharmaceutical Research and Manufacturers of America. Pharmaceutical Industry Profile 2008. Washington, DC: PhRMA; March 2008. http://www.phrma.org/files/2008%20Profile.pdf. Accessed May 11, 2009.