Typically, researchers discover new drugs through:
- Existing therapies have unintended consequences.
- New insights into a disease process enable researchers to develop a product that must contain the medicine’s recommended information.
- Many molecular compound studies may provide additional knowledge about possible beneficial effects against a wide variety of illnesses.
- New technologies, such as those that allow therapeutic products to be targeted to specific body regions or alter genetic material, are being developed.
Hundreds of compounds may be potential candidates for development as a medical treatment at this stage in the process. However, based on early testing, just a few compounds seem promising and merit further study.
Once researchers have identified a potential molecule for development, they run trials to acquire information on:
- The most effective dose
- The most effective method of administering the medication
- Its possible advantages and methods of action.
- Its efficacy in comparison to comparable medicines.
- How does it mix with other medications and treatments?
- How it impacts certain groups of people in different ways
- How is it absorbed, distributed, metabolized, and eliminated?
- Toxicity is a term used to describe side effects or undesirable occurrences.
Before testing medication on people, researchers must assess whether it can cause significant damage, a concept called toxicity. Preclinical research is divided into two categories:
- In Vitro
- In Vivo
The FDA requires researchers to observe good laboratory practices (GLP) for preclinical laboratory studies, outlined in medical product development regulations. The GLP rules are contained in Good Laboratory Practice for Nonclinical Laboratory Studies. These rules provide the basic minimum requirements for:
- study conduct
- study reports
- written protocols
- operating procedures
- and the quality assurance monitoring for each trial to guarantee the safety of FDA-regulated goods
Preclinical studies are often small in scope. These studies, on the other hand, must include detailed information on dosage and toxicity levels. Following preclinical testing, researchers analyze their findings to decide if they should test the medication in people.
While preclinical research offers answers to basic questions about a medicine’s safety, it is not a substitute for understanding how the drug will interact with the human body. Human studies or trials are referred to as “clinical research.” As the clinical trial is being built, the developers will evaluate what they want to accomplish for each clinical research phase and begin the Investigational New Drug Process (IND), which must complete before clinical research can begin.
This page contains information on:
- FDA IND Review Team
- Designing Clinical Trials
- Asking for FDA Assistance
- Clinical Research Phase Studies
- The Investigational New Drug Process
Designing Clinical Trials
Researchers design clinical trials to answer specific research questions regarding a medical product. These trials follow a predetermined study approach, a protocol developed by the researcher or manufacturer. Researchers analyze prior information about medicine to create study questions and goals before starting a clinical trial. Then they make a choice:
- How long will the study last?
- Who is eligible to participate?
- How many individuals will participate in the study?
- How will the data be evaluated and analyzed?
- How will the medication be administered to patients, and at what dosage?
- What assessments will be performed, when will they be conducted, and what will collect data?
- Whether or whether a control group will be used, as well as other methods for limiting study bias
Clinical trials are usually conducted in stages ranging from early-stage, small-scale Phase 1 studies through late-stage, large-scale Phase 3 investigations.
- Phase Studies in Clinical Research
- The Investigational New Drug (IND) Procedure
- Drug inventors or sponsors must submit an Investigational New Drug (IND) application to the FDA before beginning clinical trials.
Developers must provide the following information in their IND application:
- Data on manufacturing
- Details about the investigator
- Data from previous human studies
- Data from animal studies and toxicity tests
- Clinical procedures for upcoming trials
FDA Drug Review
Assume a medication developer has evidence that a treatment is safe and effective for its intended purpose based on early testing, preclinical and clinical trials. In such a scenario, the company may seek to have the medication commercialized. Before determining whether or not to approve a drug, the FDA review committee thoroughly examines all available data.
New Drug Application
A New Medicine Application (NDA) outlines a drug’s whole life cycle. It aims to demonstrate that a medication is safe and effective for the intended use in the population under study.
An NDA must include all medical information, from preclinical research through Phase 3 trial findings. They must include reports on all investigations, data, and analysis in developers. Developers must incorporate, in addition to clinical results, the following:
- Updates on safety
- Labeling Proposal
- Useful facts about patents
- Information about drug misuse
- Information about institutional review board compliance
- Any information obtained from investigations conducted outside the United States
When the FDA receives an NDA, the review team determines if it is complete. If it is done, the review team has 6 to 10 months to decide whether or not to approve the medicine. The review team may refuse to file the NDA if it is not complete. The following steps are included in the procedure:
Each member of the review team completes a thorough examination of their area of application. For example, the medical officer and statistician evaluate clinical data, but a pharmacologist evaluates animal trials. There is also a supervisory review for each technical field represented on the team.
FDA inspectors visit clinical trial sites to perform routine inspections. The Agency searches for evidence of data falsification, manipulation, or withholding.
All individual evaluations and other papers, such as the inspection report, are compiled into an “action package” by the project manager. This document serves as the official record for FDA review. The review team makes a recommendation, and a senior FDA official makes a decision.
When the FDA decides that a medication is safe and effective for its intended use, it must work with the applicant to develop and revise prescription information. This is known as “labeling.” Correct and objective labeling communicates the reasons for approval and the optimal method to use the medication.
However, many times, before the medication may be approved for commercialization. The FDA may require that the developer reply to questions based on current data from time to time. In other cases, the FDA requests further research. At this point, the developer has the option of continuing to work on the project. If a developer does not agree with an FDA decision, they may file a formal appeal.
FDA Advisory Committees
The NDA often provides adequate information for the FDA to evaluate a drug’s safety and effectiveness. However, now and again, a problem emerges that needs additional consideration. In certain cases, the FDA may call a meeting of one of its Advisory Committees to get independent, expert advice and enable the public to comment. These Advisory Committees include a Patient Representative who provides input from the perspective of the patient. Find out more about the FDA’s Advisory Committees.
There are limitations in the drug creation process, despite the rigorous processes. Even though clinical trials provide critical information regarding a drug’s effectiveness and safety, it is difficult to have complete knowledge about a drug’s safety at the time of licensure. When the FDA receives complaints about prescription and over-the-counter medicines, it may choose to add warnings to the dosage or use instructions and take further measures in the event of more serious issues.
On this page, you will find information on:
- Generic Drugs
- Drug Advertising
- Reporting Problems
- Active Surveillance
- INDs for Marketed Drugs
- Manufacturer Inspections
- Supplemental Applications
Developers must file a supplementary application if they wish to make substantial modifications to the original NDA. In general, any changes to formulation, labeling, or dosage strength must be approved by the FDA before being implemented.
INDs for Marketed Drugs
Sponsors may use an IND to develop an approved medication for a new purpose, dosage strength, new form, or different form (such as an injectable or oral liquid instead of tablet form) or conduct post-market safety research.
If approved medicines are manufactured abroad, FDA inspectors conduct regular inspections of drug manufacturing facilities in the United States and worldwide. Manufacturers may be notified in advance of evaluations or may conduct the assessments unannounced. May perform checks regularly or in response to a particular issue or concern. The purpose of these inspections is to verify that developers follow optimal manufacturing standards. The FDA has the power to shut a facility if basic requirements are not met.
The FDA is in charge of policing prescription drug advertisements and promotional labeling.
All marketing, such as product claims or reminder advertising, must be accurate and not misleading. They must also give precise information about a drug’s effectiveness, side effects, and prescription directions. These advertisements may appear in medical journals, newspapers, magazines, the Internet, television, and radio.
How promotional labeling is presented differentiates it from pharmaceutical advertisements. Pamphlets and other promotional materials are sent to physicians and patients by pharmaceutical companies. The promotional labeling must include the prescription information for the medication.
When a novel medication is approved for commercialization, it is granted a patent. It indicates that the sponsor is the exclusive owner of the drug’s marketing rights. When the patent expires, competing pharmaceutical companies will manufacture a generic version of the drug. Generic medicines must have the following qualities to be considered comparable to brand-name medications:
- Dosage form
- Intended use
- Performance characteristics
Manufacturers of generic drugs are excused from performing clinical trials to verify that their product is safe and effective since generic medicines are interchangeable with existing therapies. They instead perform bioequivalence studies and submit an Abbreviated New Drug Application.
The FDA has various systems to enable manufacturers, health professionals, and consumers to report problems with approved medicines.
MedWatch is a website where you may report medical products and learn about new safety information. You may also sign up for MedWatch safety alerts regularly.
The Medicinal Product Safety Network monitors the safety and effectiveness of medical goods. The FDA employs 350 healthcare practitioners throughout the nation to report any medical device issues that result in severe harm or death. Once a month, the FDA publishes the MedSun newsletter. Customers may get important information about medical device safety from the newsletter.
As part of the Sentinel Initiative, the FDA is constructing a new national system to identify possible safety risks quickly. The system will monitor the safety of approved pharmaceutical products in real-time by using large existing electronic health datasets such as electronic health records systems, administrative and insurance claims databases, and registries. This tool will complement, rather than replace, the FDA’s current postmarket safety assessment tools. Learn more about the Sentinel Initiative and its major projects.