What is an Investigational New Drug?

The development of new, safe, and efficacious medications is subject to strict guidelines set out by international bodies and governments. This article will explore the subject of investigational new drugs.

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The Importance of Drug Trials

Clinical trials play a key role in the drug development process. Strict protocols govern the safety of trials and the gathering of trial data. If a trial is not carried out following clear and defined protocols and seeks to avoid bias, the efficacy of data and results can become severely impacted.

Drug trials are typically randomized and anonymized (these types of trials are termed “blind” trials and can be single or double-blinded) but some types of trials are termed open-label trials, where the drug or treatment is known to both participants and researchers. Bias is avoided to ensure the viability of results, as incomplete and unreliable trial results can lead to the introduction of faulty or dangerous drugs onto the market or delay the introduction of effective ones.

Numerous clinical trials are ongoing at the same time, with some in more advanced phases than others. To ensure the reliability of results, the data and trial findings must be reproducible and replicable. There have been several well-publicized cases over the years which demonstrate what can go wrong if strict protocols and regulations are not adhered to in clinical trials.

Investigational New Drugs

Investigational new drugs are also known as experimental drugs. These are new drugs or biological drugs which are used in clinical trials. Investigational new drugs are also known as investigational drugs. The term also covers diagnostic biological products that are used in vitro. Approximately two-thirds of investigational new drugs are small-molecule drugs, with the rest consisting of biopharmaceuticals. Around half of these drugs fail during preclinical and trial phases.

Investigational new drugs are medications that have not been approved for general use by bodies such as the U.S Food and Drug Administration but are in the course of being tested on human patients. They are used to see if the drug improves medical conditions or diseases. In clinical trials, researchers try to see if a drug is safe and effective, how it might be used to treat a disease, how much is needed, and elucidate information about potential side effects and benefits.

Each patient’s reaction to an approved drug can differ. Approved drugs may be ineffective for treating their disease or condition. In these cases, clinical researchers and healthcare professionals may suggest the use of an investigational new drug. Factors that can influence this decision include severe side effects to approved drugs, limited treatment options, awareness of promising early study results, or the lack of approved drugs for the patient’s condition or disease.

If an investigational new drug is to be used in treating a patient’s disease, clearly defined and fully transparent informed consent forms must be used so the patient fully understands the risks as investigational new drugs may have unknown side effects or maybe completely ineffective for improving the patient’s medical outcome. Approved drugs already have well-known side effects and efficaciousness and, unless they are not available for the patient’s disease or condition, are much safer.

Even if an investigational new drug has demonstrated promising results in previous studies, patients must be aware that it is still experimental. Additionally, strict inclusion and exclusion criteria means that not everyone who wishes to participate in a trial with an investigational drug may be approved to take part. Criteria can be based on the type or stage of disease, previous therapies, being in a certain age group, the patient’s medical history, and their current health status.

Due to the uncertainty about investigational new drugs and the proven efficacy and safety of approved drugs, it is important that patients discuss ongoing concerns such as side effects they may be experiencing when taking these types of medication.

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The FDA’s Investigational New Drug Program

Unless exempted, clinical sponsors must obtain the Food and Drug Administration’s authorization in the U.S. before performing a clinical trial and administering an investigational drug or biological product to patients. All Centers for Biologic Evaluation and Research (CBER)-regulated products are covered by the FDA’s Investigational New Drug (IND) program.

All clinical studies must follow specific and strict laws and regulations to ensure the safety and well-being of human subjects who participate in them. Authorization from the FDA must be secured before interstate shipment and administration of investigational new drugs.

Access to investigational new drugs can be granted on a compassionate basis. If the patient has a serious disease or condition which has no clinical trials designed to develop drugs or treatments for it, approval can be granted by the FDA. Licensed physicians can apply if they feel that the benefits of an investigational new drug outweigh the risks of using an unproven treatment.

In Summary

Investigational new drugs are unapproved by bodies such as the FDA and carry potential risks to patients. However, in certain cases, they can be approved for use in clinical trials dependent on certain criteria, especially if there are no alternative, effective treatment options for the patient’s disease or condition.

Clear and transparent informed consent must be used when recruiting trial participants. Gathering data on how an investigational new drug works is a critical part of the drug approval process.

References:

• US Food and Drug Administration (2019) Understanding Investigational Drugs [online] Accessed online 17^th March 2022. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-investigational-drugs
• US Food and Drug Administration (2021) Investigational New Drug Applications (INDs) for CBER-Regulated Products FDA.gov [online] Accessed online 17^th March 2022 https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/investigational-new-drug-applications-inds-cber-regulated-products
• NHS Health Research Authority (2021) Clinical Trials of Investigational Medicinal Products (CTIMPS) [online] Accessed online 17^th March 2022 https://www.hra.nhs.uk/planning-and-improving-research/policies-standards-legislation/clinical-trials-investigational-medicinal-products-ctimps/

Further Reading

• All Clinical Trial Content
• What is a Phase 0 Clinical Trial?
• What is a Phase 1 Clinical Trial?
• What is a Phase 2 Clinical Trial?
• What is a Phase 3 Clinical Trial?

Written by

Reginald Davey

Reg Davey is a freelance copywriter and editor based in Nottingham in the United Kingdom. Writing for News Medical represents the coming together of various interests and fields he has been interested and involved in over the years, including Microbiology, Biomedical Sciences, and Environmental Science.