Plant-Made Pharmaceuticals Background and Key Points

For centuries, plants have served as a natural source of treatments and therapies. New advances in biotechnology make it possible to turn plants into "factories" that produce therapeutic proteins for use in the manufacture of biotech drugs, medicines and therapies. Plant-made pharmaceuticals (PMPs) are the result of an innovative application of biotechnology to plants to enable them to produce therapeutic proteins that could ultimately be used by the medical community to combat life-threatening illnesses, such as heart disease, cancer, HIV, diabetes, Alzheimer's disease and cystic fibrosis. PMP technology to develop therapeutic proteins is a safe, efficient and cost-effective alternative to traditional methods using microbial fermentation or animal cell cultures. Plant-made pharmaceuticals have the potential to provide patients with the benefits of greater and faster access to medicines.

The Biotechnology Industry Organization (BIO) and its members are committed to protecting human health and the environment by ensuring the safety of PMPs during all stages of development and production through a close working relationship with the regulatory agencies on current and future regulations to assure they are both rigorous and enforceable.

Key Points

Plant-made pharmaceutical production is regulated under stringent requirements of the U.S. Department of Agriculture (USDA) and the Food and Drug Administration (FDA).

• USDA regulates PMPs during research and development, field trials and production. USDA oversees the process from seed through grain, including the transport and planned introduction of the seed in a greenhouse or field. FDA regulates the evaluation, production and distribution of pharmaceutical products. FDA regulates biologic products and devices, including plant-made pharmaceuticals used as human medicines. FDA also provides additional oversight as needed to ensure the safety of food and feed.
• The biotechnology industry supports a regulatory system that is grounded in science, and believes the agency's guidelines reflect established science and ensures plants grown for pharmaceutical protein production can be isolated for their intended use only. Manufactures of PMPs recognize their responsibility to guarantee the safety of the manufacturing process. They are ready to provide regulatory authorities with validated analytical methods and tools for detecting PMPs and fully cooperate with regulatory agencies in reviews and audits of confinement procedures.
• In addition to adhering to all regulations, PMP companies are committed to strict, self-imposed guidelines with a goal to create a uniform code of conduct throughout the industry. Thus, each manufacturer has developed standard operating procedures that cover every aspect of production and handling of PMPs, from pre-planting to the delivery of the plant material or the product derived from plant material to a processing facility.

Developing therapeutic proteins in plants can be a safer, more efficient and cost-effective method of protein production.

• Plants are the most efficient protein producers on the planet and can do so without propagation of human pathogens or other mammalian contaminants. Compared to existing production methods, plants have a superior ability to assimilate genetic information and produce complex proteins that can be used to make more effective therapeutics.
• Traditional cell culture methods require significant capital and labor investment. There are significantly lower facility and production costs associated with PMPs because the technology is being developed in natural, renewable resources such as plants. And because PMP growth is not limited to special manufacturing facilities, it will be relatively easier to scale production to meet increased and varied demand. For certain types of pharmaceuticals, plant-based protein production is the most cost-effective method to provide patients with the benefits of greater and faster access to new medicines.
• Traditional protein-manufacturing practices are facing a major global capacity shortage for the production of biotechnology medicines. Worldwide, there are fewer than two dozen facilities capable of large-scale biotech manufacturing; thus, the biotechnology industry faces a worldwide factory shortage. Traditional methods cannot produce sufficient quantities of therapeutic proteins to meet patient population needs. The addition of plants improved through biotechnology to become protein-producing facilities can help alleviate this shortage.
• In many cases, it will cost significantly less to grow plants with the ability to mass-produce pharmaceutical proteins because plant-based techniques do not require the same costly capital investments as other production methods. Production and cost advantages of plant-made pharmaceuticals can allow more capital to be invested in research and development of new therapeutics, and expanded manufacturing capacity of high-quality proteins will spur development of more medicines by removing a key hurdle to mass production.