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Drug Development Primer

10/13/2015

The drug development process is long and expensive. Discovering and bringing one new drug to the public typically costs over $1 B and takes an average of 10 to 15 years. Here are the big steps that need to occur to make it happen:



DRUG DISCOVERY AND DEVELOPMENT
Research for a new drug begins in a laboratory:
  • New drugs begin in the laboratory with scientists who identify cellular and genetic factors that play a role in specific diseases. Researchers search for chemical and biological substances that target these markers and are likely to have an effect.
  • At this stage, thousands of compounds may be potential candidates for development as a medical treatment. After early testing, only a small number of compounds are selected for further study.
  • Once a compound has been identified for development, researchers conduct experiments in the lab to gain information such as how it is absorbed, distributed, metabolized, and excreted.
  • Lead compounds that pass the initial screening are then “optimized” to make them more effective and safer.


PRECLINICAL RESEARCH
Drugs undergo laboratory and animal testing to answer basic questions about safety.
  • Researchers test the optimized compounds extensively to determine if they should move on to testing in humans. They carry out in vitro andin vivo tests to understand how the drug works and what its safety profile looks like.
  • In vitro tests are experiments in the lab, usually in test tubes and beakers.
  • In vivo tests are experiments in living cell cultures and animal models. Generally, testing is done to evaluate safety and efficacy in small animals first like mice and guinea pigs, and then in larger animals such as baboons and macaques.


INVESTIGATIONAL NEW DRUG (IND) APPLICATION AND SAFETY
File an IND with the FDA to ensure safety for clinical trial volunteers.
  • Researchers must file an IND application with the FDA. The application includes the results of the preclinical research, the drug’s chemical structure, how it works in the body, any side effects and manufacturing information. In addition, it includes a detailed clinical trial plan.

The FDA reviews the application extensively to ensure that the people participating in the clinical trials will not be exposed to unreasonable risks.


CLINICAL RESEARCH
Drugs are tested on people to make sure they are safe and effective.
  • Phase I – 20 to 100 patients
  • Phase II – 100 to 500 patients
  • Phase III – 1,000 to 5,000 patients


NEW DRUG APPLICATION (NDA) AND FDA REVIEW
Submit an application for approval to the FDA.
  • After all three phases of the clinical trials have been completed   , researchers analyze all of the data. If the data show that the treatment is safe and effective, the sponsoring company files a New Drug Application (NDA) with the FDA for approval to market the drug.
  • The FDA reviews all of the information in the NDA and determines whether the drug is safe and effective enough to be approved.