Understanding the Biologics Regulatory Pathway Is Key to Creating Market Solutions

Total Quality

biologics pathway

There are strict governmental regulations on biologic drugs, so life sciences firms must adopt research and development strategies that ensure compliance. Image Source: Flickr user Peter Schultz

Following the explosion of biomedical understanding over the last few decades, researchers have begun to develop biologic therapies for diseases that have stumped scientists for centuries. But because these therapies are derived from biological materials, they pose greater safety risks than traditional pharmaceuticals.

As a result, the government has imposed stricter regulations on biologics than on chemical drugs. Like chemical drugs, biologics are subject to the Federal Food, Drug, and Cosmetic (FDC) Act, but they must also comply with regulations under the Public Health Safety (PHS) Act.1

Some life sciences firms have unfortunately invested in the development of biologics that don’t meet both sets of standards and are either rejected at an intermediate step in the regulatory process or withdrawn from the market after approval. Both of these situations can be financially devastating for a company—but both are avoidable. Understanding the biologics regulatory pathway will help researchers utilize emerging technology while ensuring that their new therapies meet necessary standards at every step of the process.

Initial Steps: Candidate Identification and Early Testing

The first step on the regulatory pathway occurs when a life sciences firm submits an investigational new drug application (IND). This application consists of lab and animal test data that demonstrate the biologic drug is safe for human trials. Ensuring an IND will be accepted begins even earlier, though—when scientists first choose which biologics to pursue. Identifying the options most likely to provide effective treatment and meet regulatory standards is challenging, but modern biologics software can help researchers assess developability while still in the discovery stage.

Successfully moving a drug past its IND also requires carefully considered lab and animal testing. The tests need to be extensive enough to suggest the drug will be safe and effective in clinical trials, but the flip side, of course, is that life sciences firms don’t want to go too far into testing, as this can waste time and money. Researchers can use modern software solutions in several ways in the candidate identification and early testing stages:

  • Modern software can help researchers optimize testing strategies so that they focus specifically on potential compliance risks.
  • Using a computer-based quality strategy instead of a paper-based system can help improve the testing process by eliminating the risk of lost data points and transcription errors.
  • Software can streamline the overall process by eliminating the inefficiencies of cumbersome, paper-based systems.

By employing a software-based quality strategy, biologics companies can be sure that the IND will be accepted without a hitch.

Step Two: Clinical Trials

The candidate biologic is now ready to be tested in clinical trials, the results of which will be submitted to the FDA as part of a marketing application. For this application to be accepted, researchers must design and execute tests that verify the drug is safe for human use, thoroughly examining every aspect to ensure it really does work for its intended purpose.

It is important to note that not all unsafe or ineffective drugs will be caught by the FDA. If clinical studies are not well-designed, they can produce misleading results that suggest drugs are safe and effective—even when they aren’t. Your marketing application might be approved, but the drug itself can still be withdrawn at a later stage in the biologics regulatory pathway, or worse, after it hits the market. To avoid this scenario, life sciences firms can use software to aid in study design. The computer-based solution allows for the seamless integration of data at every stage of testing, ensuring that no critical piece of information is ever overlooked, even late in the process when a large amount of data has been accumulated. Because software ensures that clinical trials are designed based on an evaluation of all the available data, companies can be confident that only candidate biologics with true potential will be submitted to the FDA.

Where the Biologics Regulatory Pathway Diverges: the Biologics License Application

The first two steps in the regulatory pathway—the IND and the marketing application—apply to both biologic and chemical drugs, but there is an additional step for biologics: the Biologics License Application (BLA). Under the provisions of the Public Health Safety Act, FDA approval of biologics requires assurance of their safety, efficacy, and potency.2  On a practical level, this means that, unlike chemical pharmaceuticals, biologic drugs are analyzed by the FDA throughout the entire manufacturing process and not just at the end. The FDA holds that manufacturing can alter biologic drugs in ways that are undetectable by the standard chemical analysis techniques used to test final products, which is why biologics must be monitored at every stage of production.

This sets a high bar for life sciences firms. Since the manufacturing process is closely scrutinized, companies must develop high-quality, consistent procedures by the time they submit their BLAs. Not only do such processes guarantee the safety of future drug users, they reduce the odds that the FDA will flag a manufacturing flaw and force your firm to suspend testing or even terminate development. Withdrawing a candidate biologic this late in the development process can be extremely costly, so it makes sense to adopt a software-facilitated comprehensive quality strategy that ensures compliance at every step of the biologics regulatory pathway. That way, when it comes time to apply for a BLA, there is no doubt that your drug will meet the safety, efficacy, and potency requirements of the FDA.

Post-acceptance Regulatory Concerns

While FDA requirements for market approval are strict, there is always the chance an unsafe drug will slip through the cracks and get to market, as some chemical pharmaceuticals have done in the past.3  This is why the FDA reserves the right to immediately suspend the Biologics License for any drug that poses a threat to public health. It’s a nightmare scenario for biologics firms, as it opens them up to a wide range of negative consequences. The company could face costly litigation, since previous drug withdrawals have resulted in multimillion dollar settlements, which can cripple a life sciences firm and damage its public reputation. Moreover, depending on how soon after the approval of the license has been suspended, the company may not have earned enough to cover research and development costs. Life sciences firms can protect themselves from BLA suspension by following a comprehensive quality strategy, which helps lower the odds of future studies revealing that a biologic drug is unsafe and must be suspended.

BIOVIA offers several solutions that can help life sciences firms meet FDA standards. BIOVIA Biologics software makes it easier for researchers to discover the most promising candidate biologics, design comprehensive lab and clinical testing strategies, and optimize the manufacturing process. The BIOVIA Total Quality Solution employs a comprehensive approach to biologics development that ensures compliance and quality at every stage of the biologics regulatory pathway. Contact us today to learn more about how these solutions can revolutionize R&D processes at your firm. 

  1. “Frequently Asked Questions About Therapeutic Biological Products,” July 7, 2015, http://www.fda.gov/drugs/developmentapprovalprocess/



  2. “Key Regulatory Guidelines for the Development of Biologics in the United States and Europe,” 2013, https://www.cov.com/-/media/files/corporate/publications/



  3.  “Lessons From the Withdrawal of Rofecoxib,” October 16, 2004, http://www.ncbi.nlm.nih.gov/pmc/articles/PMC523096/