The Art and Business of Clinical Translation

By Mr. Tong Sun —–

Q: What is clinical translation?

A: Clinical Translation is the process which turns research in the laboratory to usable clinical applications. Today, much of the research time and effort in the lab are spent on understanding the biological mechanisms, or causes, of diseases. Although these studies provide invaluable insights into basic biology – and measured with metrics such as good publications and grants – their usefulness to patients or physicians may take many years to reap, if ever.

Clinical translation plays a critical role in turning basic biological discoveries that occur every day in thousands of laboratories worldwide to usable drugs, medical devices, or clinical processes, for example, for the benefit of patients and to improve medicine and health care management. Improvements are manifested in cost reduction, better treatment efficacy, decreased toxicity or adverse effects, and more positive community engagement and patient advocacy.

Beyond the science and merit of research, clinical translation maintains an element of art – carefully designed, highly regulated, and capital-intensive – similar to an industrial process. An extensive investment of capital is typically needed, and the road to clinical application passes through intense scrutiny by agencies such as the Food and Drug Administration (FDA). After receiving regulatory approval, a drug or device candidate only becomes commercially viable through several steps:

1. The candidate drug or device advances beyond basic research to manufacturing in a quality-controlled environment under current Good Manufacturing Practices (cGMP), which ensures consistency and quality of the product (that is, the drug or device).
2. The product is then tested in animal studies under Good Laboratory Practices (GLP) which provide information on safety, toxicology, biodistribution, and other information that may merit progress into human trials.
3. After GLP animal studies, an investigational new drug (IND) or investigational device exemption (IDE) application must be filed with the FDA in order to proceed to Phase I human clinical trials that mainly test for safety.
4. This is followed by Phase II clinical trials where preliminary effectiveness and appropriate dosages are examined.
5. Phase III clinical trials involve testing, in large patient populations, the product’s efficacy for its intended diagnostic or therapeutic purpose.
6. If all of the above milestones are met successfully, then the FDA must approve the drug or device for clinical use.

Throughout this sequence of events, costs grow exponentially toward hundreds of thousands to millions of dollars, depending on the stage of product development or clinical investigation and various other factors. Studies have shown that a product can cost upwards of one billion dollars and over 10 years to reach the market, representing staggering investments in capital and human resources. The reasons for such costs are partly because many of these developments and product testing fail along the way, and pharmaceutical companies are raising the price tags in return for taking on higher risks.

Q: What is the Valley of Death?

A: The “Valleys of Death” are those points in the product development or clinical trials process that encounter the highest risks for failure or “death”, that is, where significant infusions of funds and effort are needed and the potential for failure in testing remains high. These “valleys” also represent gaps in funding. The typical university, medical school, or research institute does not heavily support risky research. Industry investors or those from other private sectors have become increasingly resistant to funding the development of a product unless it has successfully scaled the early-stage hurdles and risks of time and capital.

Q: How can a Master in Clinical Translation Management (MCTM) help in this process?

A: To be successful in bridging the “Valleys of Death”, one must possess more than a good scientific foundation or business acumen. Training in Clinical Translational Management endows students with a solid understanding of the biotechnology industry, as well as business and regulatory savviness needed to assess a product’s commercial potential and to navigate the pathway to clinical translation. This program also aims to promote a multidisciplinary approach to clinical translation and to foster individuals possessing an entrepreneurial spirit for technology commercialization.

In Texas, we do not have a robust biotechnology industry as seen on the east and west coasts. As one step toward changing this environment, the MCTM program is designed to provide selected students with real-life, practical knowledge and training in science, regulatory processes, and business development. We expect these bright individuals to help transform Houston’s Texas Medical Center into a biotechnology hub and to contribute significantly to the Texas economy.

Tong Sun, M.S., M.B.A.

Director, Central Operations
Houston Methodist Research Institute (HMRI)

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