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Pre-Clinical Studies

Pre-Clinical Studies involve research that is conducted prior to any testing in humans. During the IND/IDE review process, the FDA will evaluate these studies for evidence that the new treatment or product has the potential to be safe and effective in humans. Only when these requirements have been satisfied can your trial begin.

Depending on whether the compound or product has been studied or marketed previously, the scientists may have several options in fulfilling these requirements:

  • Existing nonclinical data from past in vitro laboratory or in vivo animal studies
  • Previous clinical testing or marketing of the compound or product
  • New Pre-Clinical Studies designed to support the safety of administrating the compound or product to humans

Questions that Pre-Clinical testing may answer:

  • Is there data to support that the potential target is involved in the disease and does the new treatment/product act on this target?
  • Does the preclinical testing add to the understanding of how the treatment works as well as provides information about its safety profile?
  • Does the proposed treatment have the potential to be more effective that the current standard of care?
  • What is the maximum tolerated dose (MTD)?
  • Why should the FDA believe that data from a presented animal model is comparable to a human?

The following links represent regulations that Investigators should consider when developing a Pre-Clinical Study.

International Conference on Harmonization (ICH)

The ICH regulations are the international standards that bring greater harmonization of regulatory requirements throughout the world, including the US, EU, and Japan. The ICH produced this comprehensive set of guidance documents regarding the required standards for safety (including risks such as carcinogenicity, genotoxicity, and reprotoxicity) on drugs and biologics under development. 

International Conference on Harmonization (ICH)

Pediatric Research

Testing drugs in children presents considerable scientific, clinical, ethical, technical, and logistical challenges. Over the years, several practical issues have discouraged the testing of drugs in pediatric populations.  These include:

  • Lack of incentives for companies to study drugs in neonates, infants, and children.
  • Lack of necessary technology to monitor patients and assay very small amounts of blood and other biological samples.
  • Lack of suitable infrastructure for conducting pediatric pharmacology drug trials.

As a result, the majority of drugs used in children today, are used off label, without adequate understanding of appropriate dose, safety, or efficacy.  

The following Acts were created to address these issues and encourage more clinical research and drug development in the pediatric population.

Best Pharmaceuticals for Children Act

Pediatric Research Equity Act                                             

Other Important Documents

Pre-IND Meeting with FDA

A Pre-IND meeting is a formal meeting with the FDA (at the request of the Investigator) to discuss preclinical and manufacturing issues.  Background information is provided by a sponsor to the FDA and is included in the Pre-IND meeting package.  This information must be received at the FDA at least four weeks prior to the scheduled meeting date.  These links also explain how to request a formal meeting and the information you are required to submit for review.

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