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QbD represents a breakthrough in thinking, but does it go far enough to address today's business challenges? NeoStem has expanded it to include business and market issues, in "Development by Design." Could this be (or is it becoming) a new model for pharma?
Since FDA introduced the concept of Quality by Design (QbD) to the pharma industry, pharma's reaction has been interesting to watch. I was a keen observer, boring many people to tears over the years with detailed reports and commentaries.
There were, and are, so many good reasons to embrace QbD, as articulated by former OPS director Helen Winkle in this 2007 presentation, one of many we have seen from FDA leaders and industry zealots over the years. Years after the QbD guidance was published, the same questions remain: Will pharma modernize the way it handles drug development and manufacturing? Will it take a more holistic and modern approach, as other industries do, to improve R&D success and profitability? Can it take this approach, given the sensitive and patient-variable safety issues surrounding drugs?
Pharma's response to QbD has been mainly positive, moving in phases, from the initial stage of confusion when QbD followed so closely after the guidance on process analytical technology (PAT), the methods and technologies underlying QbD, and necessary for monitoring and control. Then came the “Pollyanna” stage, where everyone seemed to agree that QbD was the greatest thing, but many interpreted its underlying concepts differently. For example, some said they were doing QbD without PAT. Others had their own definitions of design space and critical quality parameters. Then there was that minority that insisted they’d always used these principles and there was no need for labels, or new guidances.
Pharma's response to QbD seemed to reach a “grounded,” more realistic phase when FDA made core QbD concepts part of Question-based Review. More companies, including those in developing markets, seemed to take QbD concepts to heart. FDA’s Office of Pharmaceutical Quality continues to advance these efforts.
But QbD has been around a while. Not to add any more confusion or introduce another three-letter acronym, but shouldn't the concept be broadened to address, holistically, the quality, scientific and business challenges of drug development? Could this approach help companies address, from the earliest stages, the reasons why so many drugs fail during clinical trials, regulatory review, or commercial stages?
Robert Preti, Chief Scientific Officer and President of NeoStem, a cell therapy specialist with a contract development and manufacturing organization (CDMO) division, Progenitor Cell Therapy (PCT), explained his company’s approach recently in an interview in <i>Pharmaceutical Technology’s</i> February outsourcing supplement. The company has expanded QbD to “Development by Design” so that some of the factors critical to market success are also considered from the earliest conceptual stages, such as:
· Cost of goods
· Sustainability of the process
· Regulatory compliance costs (e.g., the potential savings from examining comparability requirements to optimize timing for process changes, or by using new technologies, for instance, installing new IT system for batch records that would enable “release by exception” and reduce validation and documentation requirements)
With "DbD," Brian Hampson, VP of manufacturing has said, the product profile becomes an essential, and evolving document, from the start of product planning. Contrast that approach with current practices at many companies when the document is started late in the process, once people "have all the answers."
Cell therapy is a challenging area that magnifies some of the issues that developers of any pharmaceutical face. But, cell therapies have the same needs as any area of pharma: product quality, reasonable cost, and processes that will meet demand.
Could Development by Design be the way to go beyond QbD to ensure product value, quality and profitability? Are any of you out there already formally taking this approach, even if you call it something else? Please write to me at email@example.com and let our editorial team know.
This general topic, and others concerning cell therapies, will be discussed in a special session at Interphex 2015 in New York City on April 22 that Pharmaceutical Technology and Biopharm International are sponsoring, on the topic of Moving Cell Therapies Beyond R&D and into Manufacturing.
On the program will be Eytan Abraham, who heads up Lonza’s cell therapy R&D efforts, and had worked in this area for years at Pluristem, who will discuss commercialization roadblocks and the way ahead. (An interview with him also appears in February’s <i>Pharm Tech</i> supplement). Robert Preti will share insights into the DbD approach, while Richard Grant, VP at Invetech will talk about equipment design, Harvey Brandwein, with Pall Corp. will discuss allogeneic therapy issues, examining recent research from University College, London, and Phil Vanek, general manager of cell therapy technologies, at GE Healthcare, will also share insights into the topic.
I hope to see you there!