What You Need to Know to Get Approved
Bringing a new drug therapy or medical device to market is a journey fraught with risk and complexity. For innovators and pharmaceutical companies, the path to regulatory approval can feel like an insurmountable challenge, filled with delays and legal hurdles. Yet, for a new therapy to reach the millions of people who need it, a methodical approach is essential.
This guide will demystify the complex, multi-stage FDA approval process and provide a clear, step-by-step overview of what it takes to bring a new therapy to market in the U.S.
What is the FDA and Why is its Role So Critical?
The Food and Drug Administration (FDA) is the regulatory arm of the United States Federal Government. Its mission is to promote public health by ensuring food and drug safety, and the effectiveness of drugs, medical devices, and a host of other products.
Their oversight helps consumers and health professionals have confidence that the drug is safe and ready for market. The FDA’s role is not to hinder innovation but to serve as a gatekeeper, ensuring that new products meet the highest standards for public use.
The FDA’s authority is derived from federal law, including the Federal Food, Drug, and Cosmetic Act. This foundational legislation gives the FDA the power to supervise a wide range of products, including:
- Human and veterinary drugs
- Biologics (e.g., vaccines, blood products)
- Medical devices
- Foods and dietary supplements
- Cosmetics and tobacco products
The review and approval process for a new drug is a meticulous journey that can be both long and costly. However, a deep understanding of its structure and requirements can significantly improve your chances of success.
The Four Stages of the FDA Approval Process
The path to FDA approval for a new drug is broken into four distinct stages. Each phase builds upon the last, requiring extensive data and documentation. The drug evaluation and research is a meticulous process.
Stage 1: Pre-Clinical Research & Development
Before a drug or therapy can ever be tested on a human, it must undergo a rigorous period of laboratory and animal testing. This is the pre-clinical stage.
- Discovery: The process begins with the discovery of a new compound or therapy that shows promise for a specific disease. This can involve decades of research.
- Initial Testing: The new drug is tested in a lab to assess its basic properties, pharmacology, and toxicology.
- Animal Testing: The drug is then tested on animals to gather safety data and determine if it’s safe enough for human trials. The goal is to identify potential risks and side effects.
If the results from pre-clinical testing are positive, the drug sponsor will prepare an Investigational New Drug (IND) application. This is a comprehensive document submitted to the FDA that outlines all the data gathered so far and proposes a plan for human testing, known as clinical trials.
Stage 2: Clinical Trials (Human Testing)
Once the FDA has approved the IND, the drug can move into clinical trials, which is a three-phase process involving human subjects. The primary purpose of this stage is to collect essential data on safety and efficacy for the drug developed.
- Phase 1: The drug is tested on a small group of healthy volunteers (typically 20-80 participants). The main objective is to evaluate the drug’s safety, determine a safe dosage range, and identify side effects. Approximately 70% of drugs proceed to the next phase.
- Phase 2: The drug is tested on a larger group of people (several hundred) who have the disease or condition the drug developed is intended to treat. The focus is on efficacy—how well the drug works—while continuing to monitor safety. Approximately 33% of drugs move on.
- Phase 3: The drug is tested on a much larger population (several thousand participants) to confirm its effectiveness, monitor side effects, and compare it to existing treatments. This is the most extensive and expensive phase, and about 25-30% of drugs approved successfully complete it.
Stage 3: New Drug Application (NDA) Review
After a drug has successfully completed clinical trials, the sponsor can submit a New Drug Application (NDA) to the FDA. This is the formal proposal for approval.
The drug application NDA is a massive document that must provide the drug’s complete history, including:
- All pre-clinical and clinical trial data.
- Detailed manufacturing information.
- The drug’s chemical composition and how it behaves in the body.
- Proposed labeling, including directions for use and a list of side effects.
The FDA reviews each application meticulously. This review of drugs can take anywhere from 6 to 10 months, depending on the review designation. The outcome is either an approval or a rejection, which comes in the form of a detailed letter from the Center for Drug Evaluation.
Stage 4: Post-Marketing Safety Monitoring
The approval of a drug is not the end of the process. The FDA and the drug’s sponsor are required to continue monitoring the drug’s safety once it is on the market.
- Ongoing Surveillance: The manufacturer must continue to submit safety reports to the FDA.
- MedWatch Program: The FDA’s MedWatch program allows healthcare professionals and consumers to voluntarily report adverse events.
- Post-Approval Studies: In some cases, the FDA may require a sponsor to conduct a new study after approval to address specific safety concerns.
This continuous monitoring helps identify rare or long-term side effects that may not have been apparent during clinical trials. This can result in label changes, new warnings, or, in rare cases, a product’s withdrawal from the market.
How the FDA Approval Process Can Be Completed Faster
The traditional pathway can be a long and difficult one. To help bring new drug therapies for life threatening conditions to market sooner, the FDA has created several special programs and designations that can expedite the review and approval process. An experienced attorney can advise on which of these may apply to your product.
Accelerated Approval
This program is used to streamline the approval of drugs developed that treat a serious disease or fill an unmet medical need. It allows the FDA to grant approval based on a “surrogate endpoint,” which is a marker that predicts a clinical benefit but is not a direct measure of it. For example, a drug’s ability to shrink a tumor can be used as a surrogate endpoint for its ability to extend a patient’s life.
Fast Track
The Fast Track Designation program is designed to facilitate the development and expedite the review of drugs intended to treat a life threatening condition and fill an unmet medical need. A drug that receives this designation benefits from more frequent meetings with the FDA, which can lead to a quicker approval.
Breakthrough Therapy
This breakthrough therapy designation is reserved for a drug whose preliminary clinical evidence demonstrates a substantial improvement over existing therapies for a serious condition. A drug designated as a Breakthrough Therapy also receives all the benefits of Fast Track, along with intensive guidance from senior FDA managers.
Priority Review
Under the Prescription Drug User Fee Act (PDUFA), the FDA has two review systems:
- Standard Review: The expected action time is 10 months.
- Priority Review: The expected action time is 6 months.
A drug that offers a clinically significant endpoint in the treatment, diagnosis, or prevention of a serious condition may be eligible for priority review designation.
How an Experienced Attorney Can Help You Through the FDA Process
Reviewing and approving new drug therapies is a highly complex undertaking. The legal and regulatory landscape is constantly evolving, and a single misstep can lead to significant delays and costs. A skilled and experienced attorney with a deep understanding of the FDA’s regulations can streamline the entire experience.
- Strategic Planning: An attorney can advise you on the most efficient and legally sound pathway for your drug or device.
- Application Preparation: They will ensure your IND and new drug application are meticulously prepared, complete, and compliant with all FDA requirements, saving you time and reducing the risk of a rejection.
- Proactive Communication: An attorney can manage communication with the FDA, ensuring all questions are answered promptly and effectively to build trust and avoid delays.
- Understanding Your Legal Rights: They can help you understand all of your legal rights and those rights remain protected, and also can help you explore the expedited review programs.
By partnering with a law firm that has expertise in FDA regulatory strategy, you can focus on the science, confident that your legal strategy is sound and your path to market is clear.
Crowley Law: Your Partner in Regulatory Success
The FDA approval process doesn’t have to be a roadblock to your innovation. At Crowley Law LLC, we’ve guided countless life sciences and technology companies through successful applications, helping them secure the regulatory approvals needed to bring their groundbreaking devices to market.
With decades of experience in FDA regulatory strategy and a deep understanding of the life sciences ecosystem, we provide the strategic counsel and meticulous preparation your application demands. From pre-submission strategy to final approval, we’re your trusted partner in turning regulatory complexity into a competitive advantage.