Regulatory bodies explained: Key checkpoints for drug efficacy and patient safety

Regulatory bodies, particularly the Food and Drug Administration (FDA), are often mentioned in healthcare settings and scientific publications, alongside phrases such as “ethical considerations” and “regulatory approval”. However, do patients truly understand the role of these processes in safeguarding their wellbeing?

While many other global and region-specific regulatory agencies exist, such as the European Medicines Regulatory Network (EMRN) and the Health Sciences Authority (HSA), this article will focus solely on the FDA for simplicity. (1) The FDA is a federal government agency based in the United States that regulates the use of a diverse range of products, including foods, medicines, medical devices, and cosmetics, with the primary aim of protecting public health. (2)

Purpose of medical product regulation 

Before individuals can access any medicinal product, whether a drug, biological material, or medical device, it must pass a series of regulatory checkpoints. But why is it critical for regulatory bodies like the FDA to conduct these checkpoints?

1. To ensure patient protection: The FDA undergoes a rigorous approval process, in which patient outcomes—both adverse side effects and beneficial therapeutic effects—are evaluated to guarantee that drugs and other medical products are well-tolerated and effective before reaching the market for distribution. (3)

2. To guide clinical trial design: The FDA oversees clinical trial protocols and data collection to establish methodologies that comply with the highest ethical standards, including the suitable collection of informed consent in the instance of human participation. (3)

3. To foster trust between patients and the industry: By disclosing pharma’s regulatory approaches, patients are more likely to gain confidence in medical treatments and products.

The key stages of regulatory approval

The medicinal product under investigation undergoes a step-by-step approach to final approval, providing multiple opportunities for ethical concerns to surface. The FDA first reviews a novel treatment after it has been preclinically tested in animals but prior to investigation in human participants. (3) At this stage, the agency reviews an investigational new drug application submitted by the drug sponsor, independently of a local Institutional Review Board (committees within specific institutions, such as hospitals or universities), for additional surveillance. (3,4) This application includes information on the medicine’s toxicity (for example, side effects observed among the animals tested), how the drug is manufactured, and a detailed overview of the proposed clinical trial design. (3)

The FDA also conducts review meetings after phase I and phase II trials—initial testing in a small group of healthy volunteers and subsequent testing in a moderate number of participants with the target condition, respectively. These meetings aim to optimise the protocol for phase III, the final stage of drug development involving a large cohort of the target population. The goal is to ensure that patients receive the dosage that delivers the maximum therapeutic effect at the most appropriate time. (3) 

Once phase III of the clinical trial is complete, a pre-new drug application is submitted to obtain final approval. This document provides an overall picture of the medicine, containing all animal and human data along with the associated statistical analysis results, as well as labelling and manufacturing information. If successful, the drug can proceed to market approval, where post-market monitoring is critical for the ongoing safety of individuals receiving treatment. (3)

Strengthening the FDA’s impact through collaboration

Notably, across all stages of FDA approval, the importance of effective collaboration must not be overlooked. While the FDA plays a crucial role in overseeing the development of medical products that make a meaningful difference in the lives of individuals in the United States, its partnerships with international governments and organisations help extend its impact to global standards and best practices for clinical trials and health regulations. (5) Global cooperation fosters an equitable approach to the availability of safe and effective healthcare interventions, ensuring that these products reach populations worldwide.

Arguably, the most important partnership is between the FDA and patient communities. Together with the Clinical Trials Transformation Initiative, the FDA established The Patient Engagement Collaborative to hear patients' lived experiences firsthand, guiding more patient-centric regulatory processes. (6) For example, understanding what clinical outcomes are valuable to patients can shape regulatory decision-making around protocol design. (7)

At Synthesis, we strive for a healthcare system that seamlessly integrates the patient voice into practice, whether patient-facing care or behind-the-scenes regulatory processes. To learn more about how we can help you foster effective, trusting patient partnerships, please contact info@synthesishealth.co.uk.

References

1. List of WHO Listed Authorities WLAs: https://cdn.who.int/media/docs/default-source/medicines/regulatory-systems/wla/list_of_wla_may24.pdf?sfvrsn=1f6c2140_37&download=true

2. What We Do: FDA Mission. FDA. Updated 11/21/2023. https://www.fda.gov/about-fda/what-we-do

3. Thind M, Kowey PR. The Role of the Food and Drug Administration in Drug Development: On the Subject of Proarrhythmia Risk. J Innov Card Rhythm Manag. 2020;11(1):3958-3967. Published 2020 Jan 15. doi:10.19102/icrm.2020.110103

4. Grady C. Institutional Review Boards: Purpose and Challenges. Chest. 2015;148(5):1148-1155. doi:10.1378/chest.15-0706

5. Partnerships and Collaboration. FDA. Updated 03/17/2025. https://www.fda.gov/international-programs/partnerships-and-collaboration

6. Patient Engagement Collaborative. FDA. Updated 12/11/2024. https://www.fda.gov/patients/learn-about-fda-patient-engagement/patient-engagement-collaborative

7. Bertelsen N, Dewulf L, Ferrè S, et al. Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: A Global Landscape Review. Ther Innov Regul Sci. 2024;58(1):63-78. doi:10.1007/s43441-023-00573-7