Basic biomedical research: Drug development model
Basic biomedical research refers to activities intended to find means of detecting, preventing or treating human disease. Such research covers the discovery and exploratory studies that precede the regulated phases 1 of drug development or programmes to develop other methods of disease control. These later stages of biomedical research are time consuming and require vast financial, human and technical resources; this is true whether new basic principles are being established or new drug candidates developed. The candidates for further development come from the first R&D stage in basic biomedical research, often called ‘discovery’. This discovery stage can be further subdivided into three: discovery per se, transitional research, and non-regulated non-clinical research the contents of each stage are illustrated by reference to three examples.
During this stage, the researcher identifies a possible new drug candidate. To begin with, the researcher notices signs that a compound may have therapeutic potential and finds ways to establish whether or not it is a fruitful lead to follow. The idea for the compound may come from direct observation, scientific literature, knowledge of traditional practices, or systematic screening. It is unlikely that the research progresses smoothly; the researcher will probably meet many dead ends and collect many inconclusive results. The researcher may not be able to formulate a testable hypothesis or make a firm plan. Even if the research does not concern drug discovery and development, the stages are analogous, as illustrated in figure 3. In the discovery stage, ideas are formulated and tested by observation or experimentation. The contents of each stage of basic biomedical research for products other than drugs are illustrated by three examples.
A researcher suspects that one of the body’s own macromolecules might be exploited to alleviate a progressive, crippling condition, common throughout the world. In the discovery stage the researcher makes efforts to establish whether this is a reasonable idea, and how the molecule might function. He/she finds out how to obtain a supply of the molecule for the next stages of research. A researcher knows that a population traditionally uses a local herb to alleviate an affective disorder. But the herb contains dozens of interesting compounds, of which several might be the active principle. The researcher needs to establish whether only the herb is used, or whether other methods or drugs are used simultaneously. The herb is identified, and its habitat mapped. It is very difficult to establish at this stage whether the herb is effective or whether the population is enjoying a placebo effect. Although an observational study might be relevant at this stage, it is important to emphasize that this is not the right stage for a clinical study. A large company with expertise in psychiatric disorders routinely screens many thousands of potential compounds from a ‘drug library’ shared with a neighbouring company which has expertise in another field. The screening programme is designed to detect molecules with a good ‘fit’ to the target receptors, and dozens are found each week.
Journal of Biochemistry & Biotechnology