Clinical Research: FDA Approval, Regulations, and Compliance

Clinical research is a complicated and lengthy process. However, sometimes drugs or treatments show promise for life-threatening diseases and may qualify for a faster approval process with some conditions. Compliance with specific regulations is essential.

Clinical Research: FDA Approval, Regulations, and Compliance

Table of Content

  1. Navigating the FDA Approval Process
  2. Common FDA Inspection Findings
  3. Post-Market Surveillance

Clinical research is a complicated process with several steps and many regulations. FDA compliance for clinical trials is essential and can mean the difference between continuing research or total shutdown.

These research delays are costly for drug companies, but robust systems, quality checks, records, and reviews help you stay compliant. The key is understanding the approval process, common trial problems, and the post-market surveillance process.

The FDA approval process is lengthy, but for good reason. Research usually involves discovery, animal testing, several phases of clinical trials, applications, review, approval, and post-surveillance. However, sometimes drugs or treatments show promise for life-threatening diseases, so the FDA allows a faster process with some conditions.

Accelerated Approval

The FDA allows researchers to jump the approval line if they can show a drug is likely to predict a meaningful clinical benefit using markers like a lab value, imaging test, or effect on disease or death rate. Companies usually do single-arm studies to confirm the drug benefits after approval, and the FDA may remove the drug if the results don’t prove the effects.

However, the FDA suggested new changes to the program in 2022. Under this proposal, researchers will do randomized controlled trials before and after approval or one before approval with a long-term follow-up. The goal is to establish a higher bar for safety and drug benefit.


Some drug therapies can reach patients faster through the fast-track process if they can help treat serious diseases or meet an unmet need. A fast-track approval process gives you more access to the FDA with frequent meetings about trial design and data and faster problem-solving. You also become eligible for accelerated approval, priority review, and expedited new drug applications.

Breakthrough Therapy

Another accelerated pathway is the breakthrough therapy route, available for drugs that treat a serious condition and have animal studies showing better results than current drugs. You can submit via this route when animal studies and phase 1 or 2 trials show a better safety profile or produce markers that impact serious symptoms.

Priority Review

Every drug must go through either a standard review or a priority review process before approval. Priority review is available for products that significantly improve safety, drug reactions, disease diagnosis, treatment, or prevention.

Deciding on the right approval pathway depends on how well your new drug works and the evidence you have to support those claims. Following research best practices and regulations is critical and helps avoid common issues that may lead to rejection or delay.

Common FDA Inspection Findings

The FDA inspects trials to confirm that researchers and organizations follow best practices to protect patient safety, well-being, and privacy. Most clinical trials are according to the law, but missteps happen. The most common issues are problems observing FDA regulations for clinical trials, including protocols, informed consent, and drug handling.

Protocol Noncompliance

The FDA states under Section 801 that researchers must register applicable studies and report results with This website provides patients, healthcare professionals, caregivers, and the general public with information about the trial and its outcomes.

The most common inspection problems include issues with standard operating procedures, including registration and reporting requirements. An analysis and comparison of findings from the FDA and European Medicines Agency found that clinical trials in the U.S. had consistent problems with:

  • Safety reporting
  • Reporting in case report forms
  • Eligibility criteria
  • Efficacy testing

These issues often happen because of gaps in the quality check system. However, researchers can avoid these problems with training, audits, and good record-keeping practices.

Informed consent is more than just signing a form. According to the FDA, it also involves giving trial participants enough information to consider the risks, benefits, and potential outcomes of joining a trial.

Under this practice, researchers must give participants enough time to ask questions and discuss the study with a doctor or family. The goal is to confirm that participation is by choice and not under pressure or force.

However, FDA inspections commonly find issues with informed consent. Issues are often related to how researchers gain consent, the information on the consent forms, and proof of consent at the research site.

Drug Tracking and Handling Problems

Researchers are accountable for drug use, storage, handling, and documentation during a clinical trial. These drugs can only be dispensed and used by study participants under the supervision of a trained research associate and according to the protocol.

However, drug handling concerns are another common finding of FDA inspections, which usually involve how researchers access, destroy, or administer drugs. For example, missing shipping, receiving, or drug dispensing logs and unsecured drug storage all open the trial up to errors and tampering.

Post-Market Surveillance

Safety monitoring is a key part of the FDA regulations for clinical trials and new drugs. Health professionals, patients, and manufacturers can report problems through the MedWatch database.

However, post-market surveillance also includes marketing, advertising, and manufacturing inspections. Drug companies may not promote a drug because of unapproved uses or misleading information, and the FDA monitors these claims. The FDA also performs scheduled or random manufacturing inspections to confirm that companies follow guidelines.

Regulators also track safety data through electronic health records and insurance claims, which gives insight into real-time trends. If necessary, the regulator can then quickly update drug use information. The process encourages new medical developments and treatments while safeguarding patient health and well-being.

Accelerate Compliance With Studypages

Tracking documents and compliance through the many stages and phases of research is a messy and time-consuming process. Workflow management platforms, such as Studypages, automate communications and reduce manual tracking.

With a streamlined process, you can make recruiting, onboarding, and retaining patients easier. Bring your insights forward and drive compliance with Studypages. Learn more.