Understanding the Evolving Biopharma Regulatory Landscape
As the biopharmaceutical industry evolves, so do the regulations. In this article, we give an overview of some of the major regulatory bodies, emerging trends, and more in the current biopharma regulatory landscape.
Navigating the Regulatory Maze
Researchers in the biopharmaceutical industry are constantly innovating, creating new therapies and technologies with the goal of improving the quality of life for our population. With these new findings, it is imperative that a system of checks and regulations exists for each new product, ensuring it is safe to release to the general public.
Regulation can differ from country to country and depend on the ailment for which the new treatment is being developed. That is why it can sometimes be intimidating to understand the overarching regulatory landscape.
In the article below, we explore some of the major regulatory bodies, emerging regulatory trends, clinical trial regulation, best practices, and more.
Key Regulatory Bodies & Their Influence
FDA Regulations in Biopharma
Officially created in 1906 to enforce the Pure Food and Drug Act, signed into law by President Theodore Roosevelt, the FDA is a federal agency in the United States within the Department of Health and Human Services.
The main goal of the FDA is to protect public health. They do so by making sure human and veterinary drugs, medical devices, and biological products are safe, secure, and effective. Additionally, they work to ensure the safety of the United States food supply, cosmetics, and any products that emit radiation, among other things.
In the United States, the FDA is the regulatory body that evaluates and approves treatments and therapies throughout the clinical trial phases. On top of regular safety evaluations, the FDA also releases new broad regulations to align all biopharmaceutical manufacturing to the highest standards.
One new regulation the FDA has introduced is pushing for alternatives to animal testing. They are also working on new policies to introduce regarding the integration of artificial intelligence (AI) and other new technologies into common biopharma practices, which we will explore further in a section below.
EMA Regulatory Updates for Biotech
The EMA, or the European Medicines Agency, is a decentralized agency responsible for the safety monitoring of medicines in the European Union (EU). Its goal is to utilize a variety of regulatory mechanisms to achieve timely patient access to new medicines.
Similar to the FDA, the EMA is currently working on updating its regulatory framework to include new standards on emerging technologies like AI. In December 2020, the EMA, alongside the Heads of Medicines Agency (HMA), developed a strategy to foster the supply and development of new drugs in the European Union (EU).
After reviewing the major shifts in the biopharmaceutical landscape from 2020 to today, the EMA is now revising and renewing the strategy, previously titled EMA-HMA Strategy 2025, through 2028. Main focus areas of the updated EMA-HMA Strategy 2028 include accessibility to treatments, regulatory science and innovation, antimicrobial resistance, sustainability, digitization, availability, and more.
Global Regulatory Agencies
In addition to the FDA and the EMA, there are many regulatory bodies across the globe that are responsible for therapy and medicine technology regulations in different countries or areas of focus. Some of the most notable include the Therapeutic Goods Administration (TGA) in Australia, Pharmaceutical and Medical Devices Agency (PMDA) in Japan, and Health Canada.
Emerging Regulatory Trends
AI and Machine Learning in Biopharma Regulation
Artificial intelligence, digitization, and machine learning have become prominent and often debated topics in all industries, not just biopharma. However, as with the introduction of any new technology, regulatory bodies are now charged with the task of providing guidelines for these still-in-development advancements.
In the United States, the FDA introduced the draft guidance “Considerations for the Use of Artificial Intelligence (AI) To Support Regulatory Decision-Making for Drug and Biological Products” [FDA-2024-D-4689] on January 7, 2025. This draft guidance specifically provides guidance on acceptable and safe ways to use AI or similar technologies to generate data and other information to support regulatory decision-making.
In this guidance, the FDA urges organizations utilizing AI in their process to engage early with the agency, as this allows clear expectations for credibility assessment activities to be reviewed early. Different models of AI can be higher-risk, and those will require more in-depth information to be submitted. This also establishes that the FDA is expecting the use of AI to be transparent, detailed, and submitted ahead of treatment and therapy clearance.
It is important to note that regulations surrounding AI and similar technologies are likely to evolve significantly in the next few years, as the technology itself continues to change.
Gene Therapy and Cell Therapy Regulations
In comparison with artificial intelligence, gene therapy and cell therapy have been around longer, and thus have more established regulations. In the United States, any cell or gene therapy requires the approval of a biologics license application (BLA), under Section 351 of the Public Health Service Act.
The specific sector of the FDA that is responsible for the regulation of cell and gene therapies, which are classified as biological products, is the FDA’s Center for Biologics Evaluation and Research (CBER).
Over 40 cell and gene therapies have been approved by the FDA to date; however, the FDA has predicted that by 2025, it could be approving as many as 10-20 cell and gene therapies annually. Due to the increase in demand and the increased workload of reviewing applications for these therapies, the CBER developed a new office in February 2023 called the Office of Therapeutic Products (OTP).
Even with these new offices and application processes, many professionals in cell and gene therapy development still cite regulatory hurdles as one of the greatest challenges they face when attempting to get their product to market.
Orphan Drug Designation & Rare Disease Regulations
The FDA’s regulations and incentives around orphan drugs and rare disease drugs make it possible for researchers and organizations to spend time on treatments and therapies that would only be effective for small portions of the population. These therapies would likely not be incredibly profitable, as there would be a much smaller market for them.
However, the FDA encourages the development of these types of treatments through incentives regulated by the FDA’s Office of Orphan Products Development (OOPD). Orphan drugs, as defined by the 1984 amendments to the United States Orphan Drug Act (ODA), are therapies intended “to treat a condition affecting fewer than 200,000 persons” in the U.S. or that “will not be profitable within 7 years following approval by the FDA.”
Ultimately, these regulatory bodies and regulations ensure that treatments for rare diseases are not ignored due to their low return on investment. Companies can apply for orphan product designation of drugs and biological products by submitting a request on the FDA’s CDER NextGen portal.
Clinical Trial Regulations
Overseen by the FDA in the United States, clinical trials are a multi-phase process in which a treatment or therapy is thoroughly evaluated for effectiveness, safety, potential side effects, and more. To ensure all clinical trials are as safe as possible for participants, the FDA ensures they are compliant with both good clinical practice (GCP) and federal law.
The National Library of Medicine provides a comprehensive database where you can look up specific clinical trials occurring around the world. The United States conducts a significant number of clinical trials annually, contributing a large portion to global research efforts.
We dive deeper into the specifics of clinical trials, specifically regarding their phases, in our article “What is Drug Discovery?”.
Manufacturing and Quality Control (CMC) Regulations
From drug development to market, Chemistry, Manufacturing, and Control (CMC) regulations detail the critical quality attribute expectations for a treatment through its entire life cycle. These quality expectations apply to both the product itself as well as production specifications.
By enforcing that a new treatment or therapy be CMC compliant from the start, regulatory bodies help to eliminate some bottlenecks that may have otherwise been present later in the approval process.
CMC regulations can vary by region. Key agencies and organizations that establish or contribute to these regulations include the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH), which develops harmonized guidelines adopted by regulatory bodies worldwide, as well as specific regional agencies like the FDA, EMA, and WHO.
HPNE, a Getinge Company’s Dedication to Quality
At High Purity New England (HPNE), a Getinge Company, we strive to make sure we reach the highest regulatory standards. Our HPConnexx™ Single-Use Assemblies are manufactured in an ISO Class 7 cleanroom. Additionally, our two manufacturing facilities are ISO 9001:2015 certified.
In order to offer better audit service to our customers, HPNE is also a proud member of the Rx-360 International Pharmaceutical Supply Chain Consortium and participates in the Joint Audit Program. To learn more about the benefits of the Rx-360 membership to our customers, such as minimizing on-site audits for suppliers, visit our Rx-360 page.
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About HPNE
As the industry needs grow, High Purity New England, Inc. continues to supply the biopharmaceutical industry with a range of innovative products, from drug discovery and development to fill-finish, including their flagship product, custom single-use assemblies, as well as pumps, sensors, bioreactor systems, storage and handling solutions and other single-use solutions. Along with their own manufactured products for the global market, they are also a distributor for more than 18 brands in North America.
