Understand The R&D Process

Preparation for Clinical Trials

Testing a therapy candidate in preclinical studies is usually the next step. But if a therapy appears to be both safe and effective in the initial studies, there are actually several other elements that need to be in place prior to moving to clinical trials in humans. In addition to helping fund preclinical studies, your group can choose to be involved in these key areas as a therapy moves forward towards clinical trials.

Preclinical studies: In most cases, the FDA requires preclinical laboratory studies in disease cell lines and animal models prior to beginning any therapy studies in humans .
- When a potential therapy is a drug or biologic, it will be tested in unaffected and disease cell lines and animal models. Preclinical studies are needed to:
  - Initially demonstrate that the therapy will not cause serious harm.
  - Establish toxicity levels.
  - Determine dosage level to achieve treatment
- Medical devices may also have to complete preclinical studies to establish biocompatibility and safety depending on several factors. For example:
  - If the medical device will be inserted into the human body (an implant), potential toxicity and ability to cause mutations must be ruled out.
  - If the medical device is made of a new material, the new material must go through a series of tests to determine its safety.
- The FDA requires researchers to use good laboratory practices, defined in therapy development regulations, for preclinical laboratory studies.
  - The FDA Guidance for Industry: Good Laboratory Practices Questions and Answers (2007) provides more information.
Special FDA designations: The FDA has several designations and approaches to speed the development and approval of effective and safe therapy for rare and serious diseases that have unmet medical needs or no effective treatment. The FDA provides several resources to help you become familiar with the special statuses, including:
- Developing Products for Rare Diseases & Conditions
- Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review .
Approval to move to clinical trials: When preclinical studies support the safety and potential therapeutic potential of a new therapy, the Sponsor will submit an Investigational New Drug (IND) Application. The FDA will review the IND and if approved the therapy will move forward to human clinical trials.
- Approximately half of the candidate therapy that start preclinical studies will make it to clinical trials.
NCATS Toolkit provides more information on preclinical studies and the IND, including other key areas your group can support the process in Prepare for Clinical Trials. Other key elements that may occur during preparation for clinical trials include:
- Establishing a network of clinical centers.
- Using data from patient registries, natural histories, and patient preference studies, as well as your own knowledge of your patient community, to inform the design of the clinical trial protocol and the informed consent process.
- Engaging with the FDA.

Resources

Preparation for Clinical Trials

FDA Guidance for Industry: Good Laboratory Practices Questions and Answers U. S. Food and Drug Administration (FDA) (link)

Developing Products for Rare Diseases & Conditions U. S. Food and Drug Administration (FDA) (link)

Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review U. S. Food and Drug Administration (FDA) (link)

Tips to Learn More

How Does FDA Approve a Drug U. S. Food and Drug Administration (FDA) (link)

The Drug Development Process U. S. Food and Drug Administration (FDA) (link)

The Device Development Process U. S. Food and Drug Administration (FDA) (link)

CDER Learn Training and Education U. S. Food and Drug Administration (FDA) (link)

Drug Approval: Bringing a New Drug to the Market U. S. Food and Drug Administration (FDA) (link)

Drug Development Road Map Global Genes (link)

From Molecules To Medicine: How Are New Drugs & Therapies Developed? Global Genes (link)

The 10 Things You Need to Pursue Research For Your Disease: What Are the Options for Research and Why is it so Hard? Global Genes (link)

European Patients' Academy European Patients' Academy on Therapeutic Innovation (link)