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Biotech Industry Overview - Government Regulatory Requirements


"This document is not an official pronouncement of the law or the position of the Service and cannot be used, or cited, or relied upon as such."

8. Government Regulatory Requirements

A. Federal Requirements

The Food & Drug Administration (FDA) regulates the introduction of new drugs. It is also responsible for approving the safety of all foods with new food ingredients.  It requires that manufacturers perform extensive testing to prove that products are safe and effective before it will sanction commercial sale.  The U.S. Department of Agriculture (USDA) and the Environmental Protection Agency (EPA) impose safety and/or performance standards on the development of pesticides, herbicides, and genetically altered crops.  Biotechnology companies must comply with the standards of all three agencies as they develop their biopharmaceutical, agricultural and hazardous waste type treatments.  The U.S. rigorous approval process is respected around the world.  In many foreign countries, approval by FDA is enough to guarantee approval.

The biopharmaceutical approval process (which accounts for approximately 90% of industry revenue) is both lengthy and costly (12 to 15 years to get from preclinical to market at a cost of between $300 to $600 million dollars).  The effort to discover and develop new therapeutics generally consists of several distinct steps.

Step 1 – Early discovery and preclinical development (including target identification, target validation, assay development, primary screening, lead optimization, and preclinical studies).  This first step allows the researchers to identify a potential therapeutic or vaccine candidate.  It takes several years to study how a protein works and whether it is a candidate for development as a biopharmaceutical.  The researchers must create these proteins, which the body produces in minute amounts, in larger quantities, which large-scale production involves genetic engineering.  Scientists isolate the genetic sequences or genes that instruct cells how to make specific proteins.  The isolated gene is spliced into the genetic information of the microorganism or cell. Once this information is inserted, providing simple nutrients (i.e. sugar) causes the cell or bacterium to produce the desired protein in larger quantities.  The added genetic information results in the production of human insulin or a new interferon.  The manufacturing process is then carefully controlled so that these new biopharmaceuticals are not contaminated.

Before testing the protein in humans (pre clinical trials), laboratory and animal studies are carried out to determine safety (in terms of carcinogenicity and other toxic consequences) and biological activity.  After these studies are complete, the company files an investigational new drug application (IND) with the FDA for approval to test the product in humans.  The IND will contain all the data collected during discovery and preclinical trials.

Step 2 – Clinical Trials designed to show safety and efficacy in humans.  While the FDA does not mandate a particular methodology, the scientific community has established a four Phase methodology:

Phase I – Drug given to a relatively small number of healthy people to test its safety.

Phase II – Drug administered to patients suffering from the disease or condition the drug is intended to treat.  These trials usually involve a few hundred patients and are designed to evaluate the biopharmaceuticals effectiveness and safety in treating the targeted disease or medical condition at the dosage level.

Phase III – Large-scale trials are conducted involving thousands of patients to prove the product is effective against a targeted disease.  Human trial subjects must provide consent to be part of the experimental group.  The results must be statistically valid and include the determination of side effects that may exist in the general population.  

When clinical testing and research on a biopharmaceutical have been completed, the manufacturer analyzes all the data and, if the data successfully demonstrates safety and efficacy, submits a Biologics License Application (BLA) or a New Drug Application (NDA) to the FDA for product marketing approval.  The BLA or NDA must contain all the data (formula, production, labeling, and intended use) the company has collected on the drug.

Most biopharmaceuticals, blood products, and all vaccines derived from biotechnology are reviewed by the FDA's Center for Biologics Evaluation and Research (CBER).  Some biotech drugs are reviewed by the Center for Drug Evaluation and Research (CDER).

Once a BLA or an NDA is approved, the FDA continues to monitor the biopharmaceutical product.  Sometimes, side effects or other unexpected developments come to light after the drug is widely used. If so, the FDA may require additional studies (Phase IV) to evaluate long-term effects.

Phase IV – Post approval marketing testing.  These types of trials are conducted to generate marketing data that will be used in the competitive market.  The trials usually involve one or more competitor products.

Chapter 5, 6,  & 7 | Table of Contents | Chapter 9

Page Last Reviewed or Updated: 15-Apr-2015