Biopharma's Portfolio Drives New Technology

The portfolio sold by today’s global biopharmaceutical industry is fundamentally different than it was even a decade ago. This shift is a reflection of today’s global market, which features greater competition, more treatments for orphan diseases, an increase in large-molecule drugs, and personalized or targeted medicines. The result has been genotype-specific biopharmaceutical products produced in extremely limited production runs under tightly controlled manufacturing specifications. This new product mix, combined with the industry’s drive to improve production efficiency, is stimulating the development of new technologies and processes that are helping to improving economic outcomes, flexibility, and quality in biopharmaceutical manufacturing—all while benefitting patients.

What are some of these new technologies?

• Gene therapy replaces defective genes by inserting new, functional genes into patients’ cells. The field has flourished since the first clinical trials in the 1990s.
• Stem cells are unspecialized cells that can be guided to develop as multiple types of tissue- or function-specific cells. Stem cells offer great potential for chronic diseases like heart disease and diabetes, but much work remains to be done before they are fully understood.
• Nanomedicine works at the atomic level using microscopic particles called nanoshells; some are being studied for their ability to convert infrared light into heat energy that will destroy cancer cells.
• New drug delivery systems include biodegradable microspheres that dispense targeted drugs as the sphere degrades. These are being studied as possible treatment mechanisms for cancers and other diseases.

While regulators seek assurance of technically sound, risk-based, reliable, and predictive processing that is relevant to product quality, today’s regulatory environment also provides traction for the ongoing advancement of innovation. Authorities in the three ICH regions and beyond are encouraging industry to adopt new technology as supported by ICH Q8(R2), Q9, Q10 and Q11, along with the introduction of quality by design concepts.1 This may lead manufacturers to adopt cleaner, more flexible, and more efficient closed systems.

Many biopharmaceutical manufacturers are investing in:

• Continuous manufacturing, which can improve scalability, shorten time to market, and enhance quality, while reducing capital and operating costs
• Process analytical tools that streamline and fortify processes, accelerate production scale-up, and ensure resources are used efficiently
• Single-use systems that improve flexibility and reduce production lead times, yet reduce capital investment and energy consumption
• Alternative downstream processing techniques that increase yields and reduce costs
• Adopting green chemistry to diminish waste
• Improving capacity, scalability, and flexibility with new vaccine and therapy production methods
• Products that increase patient compliance and increase the effectiveness of medicines, such as drug-device combinations or improvements in drug-delivery systems.

These products require new manufacturing techniques both at the facility and throughout the supply network. When combined with changes in biopharmaceutical portfolios, these new technologies affect biopharmaceutical companies in several ways:

• They look for increasingly specialized employees. Some organizations are working with university biomanufacturing centers to design training programs that teach relevant skills.
• They collaborate on manufacturing innovation with academic institutions and diagnostics developers as well as production equipment and medical device manufacturers.
• They consider location and ecosystem advantages in strategic manufacturing decisions as a result of the new portfolios and technologies required to produce them.

Biopharmaceutical drugs have become standard therapy for multiple diseases, a trend that has spurred both increasing demand for biotechnology and the emergence of small biopharmaceutical manufacturing companies.

Companies hoping to ride the wave created by these trends will face new challenges, however. The world’s regulatory agencies are expected to further tighten their guidelines and will continue to call for the serialization of drug production.2

But as the global market continues to expand, the future looks bright. Indeed, market research firm IMS Health estimates the global sales of biological products will reach $390 billion by 2020, as much as 28% of the worldwide pharmaceutical market.3

• 1Allison, Gretchen, et al. “Regulatory and Quality Considerations for Continuous Manufacturing.” Presented at International Symposium on Continuous Manufacturing of Pharmaceuticals, 20–21 May 2014, MIT, Cambridge, Massachusetts. https://iscmp2014.mit.edu/sites/default/files/documents/ISCMP%202014%20White%20Paper%203%20-%20Regulatory%20and%20Quality%20Considerations%20of%20Continuous%20Manufacturing.pdf
• 2US Food and Drug Administration. “Unique Device Identification System; Final Rule.” Docket No. FDA-2011-N-0090. https://www.fda.gov/downloads/aboutfda/reportsmanualsforms/reports/economicanalyses/ucm36891.pdf
• 3IMS Institute for Healthcare Informatics. “Delivering on the Potential of Biosimilar Medicines.” March 2016. http://www.imshealth.com/files/web/IMSH%20Institute/Healthcare%20Briefs/Documents/IMS_Institute_Biosimilar_Brief_March_2016.pdf