Last Updated on
June 12, 2023
The US Food and Drug Administration (FDA) was formed on June 30, 1906 with the passage of the Pure Food and Drugs act, although its origins can be traced back to 1848 and the creation of the Agricultural Division in the US Patent and Trademark Office (USPTO).
The FDA was initially established to regulate food and drug safety and prevent the sale of adulterated or misbranded products. However, when the Federal Food, Drug, and Cosmetic Act was passed in 1938, it set up the modern framework for the regulation of drugs in the US, expanding the agency’s responsibilities.
Now the FDA had the authority to approve or reject new drug products based on their safety and efficacy and to establish requirements for pre-market review and approval of new drugs. This allowed the FDA to enforce standards for the manufacture and distribution of drugs.
The requirement for pre-market evidence of a drug’s safety marked the inception of the New Drug Application (NDA). The firsts applications were submitted in the early 1940s, including applications for drugs that treated bacterial infections, hormone therapies, and mental health conditions.
These early NDAs set the stage for drug development and the FDA’s review and approval process for new drugs. Over the years, the FDA has reviewed and approved thousands of NDAs for new drug products.
When a company wants to market a new drug in the US, it must submit a marketing application to the FDA for review and approval. There are multiple types of marketing applications, including:
An NDA is a submitted request for permission to market a new drug product, including new molecular entities (NMEs), small molecules, biologics, vaccines, new combinations, new indications, and more.
The NDA must be submitted in a standard format, which is organized using the common technical document (CDT) format, a technical standard for all agency submissions starting from the IND phase. This format provides a standardized way of organizing and submitting regulatory information, and it helps to ensure that the information is consistent and easily accessible.
It’s typical for sponsors to use the Electronic Common Technical Document (eCTD) format, as it is regarded as the fastest delivery method to move the NDA process along between the sponsor and FDA.
Over the years, the application process has evolved to include more stringent requirements and greater scrutiny of the data submitted by drug manufacturers due to advancements in scientific understanding of drugs and their effects as well as increased public attention to drug safety and efficacy.
An NDA application needs to tell the comprehensive story of the drug and include data the FDA can use to determine whether or not the drug is safe for humans to use as intended. Sponsors must include a wide range of required information, including:
This information is used together to demonstrate that the product is safe for its intended use and target population, that its benefits outweigh the known risks, and that it can be manufactured in a way that ensures a high-quality product, and can be safely marketed to and used by patients and consumers.
NDAs are essential to drug development because they serve as the primary mechanism for the FDA to evaluate the safety and efficacy of new drugs, providing the federal agency with the information it needs to make an informed decision about whether a new drug should be approved for marketing and what conditions should be attached to its use.
By requiring rigorous preclinical and clinical testing and a comprehensive evaluation of the data, NDAs help to identify potential safety concerns and ensure that drugs are only approved for marketing if they are safe and effective. This can ultimately lead to better patient outcomes and advances in public health.
The NDA process also requires ongoing monitoring of the drug’s safety and efficacy through periodic reports and updates, providing a mechanism for public health officials and healthcare providers to receive accurate information about the drug.
Planning, preparing, and completing an NDA is a complex and multi-disciplinary effort that involves many different job roles, from nonclinical scientists to chemists to data management teams, physicians, pharmacologists, and more:
Each of these positions often play a critical role in the NDA process, and their collective efforts are necessary to ensure that a new drug is safe and effective for human use. Submitting an NDA with all the necessary data and information requires a lot of collaboration between all the different individuals and departments to be successful. Assembling a team right away can help streamline the application process.
Beginning the planning process for an NDA should start as early in the drug development process as possible to ensure a successful outcome. Start as soon as you understand the drug’s mechanism of action, safety profile, and efficacy data from preclinical studies.
Doing so can help you stay ahead of regulatory requirements, avoiding potential roadblocks. It also provides you more time to gather your data and perform any additional studies that may be required to support the NDA submission, minimizing delays and ensuring you have enough time to format your application correctly.
Understanding the mechanism of action will help you determine the best approach for testing the drug in clinical trials, while the safety profile and efficacy data from preclinical studies will provide important information about the drug’s potential benefits and risks.
The process includes several stages, beginning with an initial review and decision by the FDA to file the NDA or not, which must be made within 60 days of the application’s submission. If the team decides the application is complete, they have 6 to 10 months to decide whether to approve the drug for the market.
Then, the FDA’s Center for Drug Evaluation and Research (CDER) conducts a scientific review of the NDA, evaluating the data and information provided to assess the safety and efficacy of the drug.
The FDA’s Office of Regulatory Affairs will also conduct a regulatory review to ensure the NDA meets all of the FDA’s regulatory requirements. If deemed necessary, an Advisory Committee meeting may be held to provide independent expert opinions on the drug’s safety and efficacy.
Based on the scientific and regulatory reviews’ results, the FDA decides on the NDA and, if approved, issues a marketing approval. The NDA review process is critical to the FDA’s goal of ensuring that only safe and effective drugs are approved for use.
After all of the necessary steps and meetings have taken place, a final decision can be made based on the review of the NDA. If the drug is approved, the FDA sets conditions for its use, such as labeling requirements, post-marketing studies, and other commitments from the sponsor.
This evaluation process can sometimes take several years, but it is designed to ensure that new drugs are safe and effective for the intended population before making them available to the public.
The pre-NDA phase is a period of drug development that involves a combination of research and development, testing, and evaluation. It is critical to the success of the NDA process.
During the pre-NDA phase, drug makers conduct research to identify new drug candidates and evaluate their potential as treatments for various medical conditions, and it typically involves laboratory and animal testing.
Once the research has been completed, the drug maker moves on to pre-clinical testing, conducting more comprehensive safety and efficacy studies in animal models.
The primary goal here is to determine whether the drug can be safe and effective in humans and identify any potential toxicities or other issues that may arise during the clinical testing phase. The results of pre-clinical testing are then used to support the submission of an IND application.
The post-NDA phase occurs after a new drug product has received FDA approval for marketing in the US and has been made available for prescription and use by patients.
It is an essential part of the drug development process, as it provides ongoing monitoring of the drug’s use and safety and can help identify any potential issues or concerns that arise. It can also include additional regulatory requirements.
Some key activities during the post-NDA phase include post-marketing surveillance, adverse event reporting, label updates, and risk management plans.
Post-marketing surveillance involves continuously monitoring the drug after it has been approved through post-marketing surveillance programs, such as spontaneous reporting systems and ongoing clinical trials. Adverse event reporting encourages healthcare providers and patients to report any adverse events associated with drug use to the FDA or the sponsor.
Label updates occur when new information about a drug’s safety or efficacy comes to light, and post-approval studies are sometimes conducted upon the FDA’s request to gather more information about the drug’s safety and effectiveness.
Risk management plans and strategies can be implemented to minimize the risk of harm to patients in the future.
By monitoring the drug’s performance and collecting additional data, the FDA and the sponsor can make informed decisions about its use and identify any necessary changes to its labeling or risk management strategies.
Here are several tips for successfully submitting an NDA:
Submitting a successful NDA requires careful planning, a commitment to quality, and the help of regulatory affairs experts and legal counsel. These tips can help ensure that your NDA is complete, accurate, and meets all of the FDA’s requirements, increasing the chances of a successful outcome.
Developing a drug to submit for an NDA generally involves several steps, all of which need to happen in subsequent order. These steps include discovery and development, preclinical research, clinical research, FDA review, and FDA post-market safety monitoring.
Discovery and development involves identifying a potential target for a drug, finding and optimizing compounds that have potential activity against the target.
Preclinical testing involves conducting laboratory and animal testing to determine the safety and efficacy of the drug. If preclinical studies go well, the company can submit an IND application to the FDA, providing information about the drug’s composition, manufacturing processes, testing results, and the plans for clinical trials.
If the IND application is approved, the company can then conduct clinical trials to evaluate the drug’s safety and efficacy in humans. Trials are categorized into three phases, each involving an increasing number of patients.
If all three phases are completed, and the clinical trials are a success, the company can submit an NDA for approval, including detailed and updated information about the drug, its composition, the clinical trial results, and proposed labeling.
The FDA reviews the NDA and evaluates the data submitted by the drug maker, and conducts inspections of the facilities to ensure the product is being produced following good manufacturing practices.
After a thorough review, the FDA can approve or deny the drug for marketing in the US. The agency may determine that additional information is needed or request additional testing or data.
This is a high-level way to look at the steps to getting a drug approved for marketing in the US, which can take several years and require significant time and resources. The NDA submission and approval process alone is quite time-consuming, but it aims to ensure that new drugs that reach the market are both safe and effective and that the public can access accurate and reliable information about the medicines they take.
Commercialization can begin after FDA drug approval. Once the product has been approved to sell and market, the sponsor can start producing and selling the drug to healthcare providers and patients. This process involves a variety of manufacturing activities, including:
This is a critical stage in the drug development process, as it represents the transition from an experimental product to a marketed product available to patients.
Successful commercialization depends on many factors, including the size of the target patient population, the drug’s pricing and reimbursement status, and the competition in the market.
A New Drug Application (NDA) is a request submitted to the FDA seeking permission to bring a new drug product to market. This includes new molecular entities (NMEs), small molecules, biologics, vaccines, new combinations, new indications, and other similar products.
The submission process is organized using the common technical document (CTD) format, which is a standardized format used for all submissions to regulatory agencies, starting from the IND phase. The electronic CTD (eCTD) is considered the quickest and most efficient method for exchanging information between the submitting party and the FDA during the NDA process.
The accuracy and quality of your preclinical and clinical research, and your overall NDA submission, will play critical roles in receiving approval for a new drug product, and will depend on various factors, including the experience and expertise of the research team, the design and conduct of the preclinical and clinical trials, and the quality of the regulatory documentation.
That said, having access to the latest and most advanced equipment can help improve the efficiency and accuracy of your testing. Sadly, acquiring scientific instrumentation can be challenging.
When using your capital expenditure (CapEx) budget to procure new equipment doesn’t make financial sense, lease with Excedr. Our leasing program is an excellent cost-effective alternative to purchasing, which is often especially important for small or emerging companies working with limited resources.
With the proper equipment, you can ensure that the data generated during preclinical testing is high quality and supports the overall objectives of the NDA. It’s worth noting, however, that simply having new equipment is not enough to guarantee a successful NDA.
Lab equipment must be used appropriately, and your research team must analyze and document the generated data properly. The equipment must also be used following good laboratory practices (GLP) to ensure the reliability and validity of the data generated.
New equipment alone won’t guarantee successful studies and an NDA submission, however, it can help you generate more accurate results, streamline workflows, increase throughput, and meet higher standards and regulations.