The history of the biopharmaceutical industry is one of continual invention and reinvention, of business models that have adapted to weather uncertain product futures and shifting economic fortunes. Some of us followed the up-and-down (and often financially painful) progress of monoclonal antibodies toward their eventual commercial success — a wealth of experience to draw from as other classes of products make their way from laboratories and onto the market.
The vast majority of regenerative medicines are still produced at laboratory scale, with suppliers targeting the current market: research hospitals and laboratories. These therapies could transform our approach to healthcare and have a profound effect on the biotechnology industry as a whole. They have the potential to reap tremendous commercial successes.
What has been lacking is a strategic bridge connecting current lab-scale cell therapies to commercialization. The core challenge faced by this market lies in securing funding — which in turn rests on the ability of companies to present solid data to ensure and support commercially viable processes. The days when business plans were approved and money secured based on exciting science and visionary “stories” (rather than solid projections) are over. The economic downturn and increased sophistication of potential investors have made it harder for start-up companies to secure funding.
Funding is indeed available. But a company must show a detailed business plan and corporate strategy that express confidence in market opportunities, early clinical successes, management transparency, and disease amelioration — as well as efficacy that compares favorably with existing treatment modalities.
That is the purpose of this issue: To serve as an educational bridge to help move cell therapies from laboratory to commercial scales. BPI will therefore continue to delve into the science, technology, and business realities of regenerative medicine, a major part of the biopharmaceutical industry that we support.
Hundreds of regenerative medicines are in development, and a number of tissue therapies (regulated for the most part as devices) are already doing well on the market. The hoped-for tipping point may have been reached in mid-2010 with the approval of Dendreon’s Provenge cell therapy. That company continues to pave the way by venturing into the reimbursement arena, helping to educate regulators and policymakers, and clearing the path for further innovative therapies. Companies poised on the brink of commercial launch are leading by example to show how business models for such therapies must diverge from well-known protein/antibody models.
For regenerative medicine, cells themselves are the product, and scale-out (rather than –up) is the task. Commercial success for most such therapies cannot rest on continuing to replicate the same manual cell “reprogramming processes” over and over again. Few if any of those processes are yet automated. One recent example suggested that, to produce enough cells to treat 1,000 patients, you might need some 20 cleanrooms with identical equipment and 100 staff. But to produce enough of the same product to treat 10,000 patients, you could need 200 cleanrooms with identical equipment and 850 staff. Add to that the logistics involved in rapid processing and shipment from and back to patients — of fragile and sometimes irreplaceable patient-specific cells — and an industry that initially looks familiar to what we already know about bioprocessing becomes, at commercial scale, something very very different.
So why is BPI publishing a cell therapy supplement? First, we remind you of our tagline: Covering the whole development process for the global biotechnology industry. BPI focuses on the myriad processes of moving products from discovery to production (not simply processing as a discrete step). From the start we’ve looked at the entire scope of the industry — both CDER- and CBER-regulated products — waiting for a point at which the regenerative industry is primed to go commercial.
Given the tremendous promise, the thought that cell therapy companies might struggle to replicate the hard-fought lessons of other industry segments is just unacceptable. One essential task is to help educate potential investors — be they venture capitalists, private investors, or pharmaceutical companies seeking to expand their product portfolios through strategic partnering. Our readers want to serve the needs of humanity; they are passionate about the promise of eliminating the physical and economic ravages of the most devastating diseases. Without successful commercialization, however, ground-breaking therapies cannot reach those who need them.
So we are delighted to introduce you to so many dedicated people and the good work they are doing to advance the future of healthcare. We are eager to help this segment of the industry communicate with and learn from the experiences of the others and thereby help speed these life-saving products to the market. And we are grateful to Bob Speziale and David James of Invetech; Lee Buckler from the Cell Therapy Group; and Jon Rowley of Lonza Walkersville — the “team” who encouraged us to develop this issue and advised us along the way. We also thank Jane Arthurs, who introduced us to the ISCT committees; the organizers of the Phaciliate Cell and Gene Therapy Forum; and all the authors and interviewees who assisted in putting this project together.