Importance of Non-Clinical Science

Research is critical for diseases like Parkinson’s disease (PD), where the exact cause is still unknown and there are no current treatments that can cure or slow the progression of the disease. Through research, scientists systematically test hypotheses (educated guesses) to learn more about how PD develops, the risk factors involved, and potential new therapies that could alleviate the symptoms or slow the progression of PD. Prior to testing new approaches, drugs, or therapies in humans, researchers engage in non-clinical science.

Non-clinical science is the stage of research that is conducted prior to a new therapy being tested in humans. Also called preclinical studies, the goals of this stage of research include understanding the safety and effectiveness of the potential therapeutic product.1

The regulatory process

The search for successful therapeutic interventions to treat PD and improve the quality of life for people living with PD involves a long, difficult, and expensive process. Across all research for all diseases, it is estimated that only 1 out of 5,000 screened compounds is approved as a new medicine.2

Regulatory agencies like the U.S. Food and Drug Administration (FDA) have established procedures for ensuring that new treatments are effective and safe before they are approved to be available to patients in the market. The stages of the development process are:

  • Discovery and development – research begins in the laboratory, where insights into the way PD develops and progresses allow researchers to design or find products that may impact the disease
  • Preclinical (or non-clinical) research – testing of a new drug or therapy to determine its safety and effectiveness prior to testing in humans
  • Clinical research – testing the new treatment in humans
  • FDA review – analysis of all the data from research trials by the FDA review team
  • Post-market safety monitoring – additional studies done after the drug is approved and is available in the market to gather information on long-term use1

The development process has several “no-go” decision gates: points at which the therapy must meet predetermined criteria in able to move forward. The outcomes of the therapeutic treatment are evaluated at each stage to reduce risk to human subjects. The entire process aims to bring to market only those therapies that are effective and safe for patients.2

Types of preclinical research

There are two types of preclinical research that are performed on potential therapies:

  • In vitro – studies performed in a test tube, culture dish, or other mediums outside of a living organism
  • In vivo – studies performed in a living organism, usually done in animal models1

In vivo studies are critical for toxicology, researching the potential side effects of a new drug or therapy. Studies are conducted in animals to help determine the safe dosage, any negative side effects, and the reversibility of those side effects. These studies depend on having a model of PD that most closely resembles the features of the disease. This way the effectiveness of a new therapy can be tested.2

Next step: clinical research

Drugs or therapies that pass preclinical research are then moved to the clinical research phase, in which the treatment is tested on humans. Volunteers to clinical trials play an important role to help move potential new treatments forward.3

Written by: Emily Downward | Last reviewed: March 2017
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