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Appendix E: The Pathway from Idea to Regulatory Approval: Examples for Drug Development
Pages 375-383

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From page 375...
... Although these failures are disappointing and costly, they still contribute to the body of knowledge on disease processes. Academic health centers and research institutions play major roles in defining the targets applicable for small molecules and carrying out the clinical trials that are needed.
From page 376...
... (A more thorough review of the research and development process for small molecules, therapeutic proteins, vaccines, medical devices, and diagnostics can be found at www.rdguide.org.) BASIC RESEARCH: THE IDEA Long before a new drug can even be imagined, scientists are working to gain a basic understanding of a disease or of specific normal chemical pathways that are subverted in an abnormal cell.
From page 377...
... IND POC NDA Therapeutic Preclinical Exploratory Advanced Approval and Basic Research Discovery Development Development Development Postmarket Initial - Robust "The Idea" "The Compound" "The Medicine " "Proof of Concept" "Regulatory Proof" "Medical Marketing" Biotechs and Universities Emerging Institutes Pharma Governments Institutes Foundations Companies Associations Universities Academic Medical Centers, Hospitals, Clinics 10 to 14 years Pharmaceutical Companies and Large Biotech Companies FIGURE E-1 Defining biomedical research from idea to market. IND = Investigational New Drug application; POC = proof of concept; NDA = New Drug Application; Pharma = pharmaceutical companies.
From page 378...
... Box E-1 summarizes an example of successful, extended, and complex collaboration that involved scientists from the National Institutes of Health as well as academic and industry scientists. Chapter 4 of the committee report cites additional examples.
From page 379...
... Testing of the expanding number of available biological targets against millions of chemical entities requires some highly sophisticated screening methods. Researchers use robotics, for example, to simultaneously test thousands of distinct chemical compounds in functional and binding assays.
From page 380...
... Preclinical studies also help researchers design proposed Phase I studies to be conducted with human. For example, preclinical studies with animals help determine the initial dose to be evaluated in the clinical trial and help identify safety evaluation criteria.
From page 381...
... They help determine the correct dosage, common short-term side effects and the best regimen to be used in larger clinical trials. This usually begins with Phase IIa clinical trials, in which the goal is to obtain an initial proof of concept (POC)
From page 382...
... These trials confirm efficacy, monitor side effects, and sometimes compare the drug candidate to commonly used treatments. Researchers also use these clinical trials to collect additional information on the overall risk-benefit relationship of the drug and to provide an adequate basis for labeling after successful approval of the drug.
From page 383...
... 2002. The protein kinase complement of the human genome.


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