With the New Year upon us, I thought it would be good to make a set of New Year’s Resolutions for the regenerative medicine industry. This set of resolutions is not company specific, but things that will be required to build long term value for this nascent industry.
- Get those marketing people working: Any way you look at it, cell-based therapies and combination products are going to be expensive to manufacture. It is therefore important to address unmet medical needs that have devastating consequences in order to justify reimbursement for the therapies. There are only a few multi-billion dollar markets out there (cardiac regeneration and diabetes are on the short list), and these are being addressed in clinical trials currently. As several regenerative medicine companies are moving forward with the “one product, multiple indications” model, it makes sense to take a page out of Genzyme’s orphan disease business model book and find some new indications where there is currently no therapy that works and that regenerative therapies can address. Of course, doing all of this and keeping reimbursement in mind will be a necessary challenge to address. Currently, most of the companies have very similar therapeutic pipelines for their cell products, and strategic marketing is where some real innovation could take place in regenerative medicine in 2008.
- Plan for success: There are a lot of cell-based therapies in clinical trials today, and the worst thing that can happen is if an approved product is needed to treat 100,000 patients a year and can’t be manufactured at that scale. Having too much demand is a good problem to have, but if you are not serving the market sufficiently it leaves time for competitors to also finish up their trials and get to market before you are seen as the leader. The cell therapy bioprocessing field is not ready for this type of demand today (see Chris Mason’s work for excellent commentary on this), and there are no clear cell manufacturing leaders that are taking us there (yet). We definitely need some innovative minds proactively addressing scale-up issues, and 2008 is the year to commit to success.
- Create a backup plan: I think this is what Stem Cell Inc. is doing if they actually purchase Progenitor Cell Therapeutics (PCT). Stem Cell Inc is several years away from marketing their cell product, let alone any profits coming from it. A purchase of PCT would diversify their business model by building an internal expertise around cell manufacturing, as well as add a contract manufacturing arm to the business that would generate revenue and potentially reduce their burn rate. Advanced Cell Technology has also diversified outside of their core business with the Mytogen purchase, but they have not created a near term revenue stream.
- Align senior management: It is critical within small companies (which almost all regenerative medicine companies are) to have senior management that is aligned and working towards the same goals. To use a Jim Collins’ analogy: everyone needs to be “rowing in the same direction” – it is the only way to get where you need to go quickly and efficiently.
- Collaborate on the next generation therapies: I think that today’s companies that are addressing the challenges of manufacturing clinical grade cells or engineered tissues have the opportunity to think really big and address some HUGE problems with true biological convergent technologies. Cell-based devices that include durable components (like a stent) as well molecular components (small molecules or proteins) will be a reality sooner than later. Cells, molecules, and biomaterial-based devices that are being used for today’s therapeutics will be the cornerstone of these technologies – but I don’t see them being incorporated into novel therapies yet. As long as we are planning for success in 2008, I would propose that we start thinking about what the next stage of research needs to be done in our industry to get some next generation technologies into our pipelines. Maybe this type of work needs to be done at a Google or Apple biotech equivalent – which I am not sure we have……. yet.
To sum it all up, the requirements for success in regenerative medicine are similar to other high tech industries: find some good markets that your product can address, figure out how you are going to scale your business, try to generate near-term revenues along the way (just in case), get everyone in the company working towards the same goal, and establish a strong pipeline of innovative products. It is difficult to take a step back from the day-to-day challenges to focus on the big picture. However, regenerative medicine has the opportunity to learn from other companies and industries, and this is the best way to create long term value. There is no need to make mistakes that have been made before.
I’m really enjoying the blog. You are certainly adding substance to the discussion about the regenerative medicine sector and what we need to do to accelerate its development.
I enjoyed your New Year’s resolution blog. Naturally I can’t comment about our potential acquisition but I can comment about the need for manufacturing scale-up. What I take from Chris’ article- and the point with which I certainly agree – is that the “science” of manufacturing and scale-up is being under-emphasized. While there are a few bioprocess programs like Chris’ and a few CMOs like PCT working hard on addressing scale-up technologies there is little funding and efforts are significantly distracted.
While it may be true that large-scale scale-up technologies and capacities do not yet exist, this is not a reason for therapeutic companies to seek its own solutions.
Of course this will sound self-serving but I believe that if and when companies attempt to tackle their own manufacturing and scale-up and treat it as proprietary, the field is not well served. Not only is this a costly experiment for companies with limited capital and senor management focus but it denies the opportunity to leverage scale-up technologies and resources.
We all certainly need to lobby for more funding of manufacturing and scale-up research, technologies, and projects.
Keep up the good work!
Lee Buckler, BEd, LLB
Progenitor Cell Therapy LLC
Business Development & Marketing Communications
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p.s. I would like to invite you and your readers to consider joining ISCT’s Cell Therapy Commercialization Committee comprised of representatives from around 40 companies in the sector. We are working on raising the profiles of regenerative medicine in the biotech community and leveraging existing organizations like ISCT, BIO, etc to engage in addressing issues common to stakeholders in the industry.
Lee Buckler, BEd, LLB
ISCT Cell Therapy Commercialization Committee Chair
Thanks for the insightful comments.
I agree with you that one-off scale up efforts will not serve the industry well. This may be difficult to avoid in the near term as individual companies will be attempting to build competitive advantages and we may end up with competing standards (just like BluRay and HD DVD are competing to become the DVD standard) or no standards at all. The industry is going to require big picture leaders that can look past short-term wins for long term benefit – and that is much easier said than done (and kind of hard to say with a straight face – to put it into perspective).
I will email you off-line regarding the ISCT Cell Commercialization Committee. I am interested to hear the discussions that are already going on in that circle related to these issues.
Best Regards from The Regeneration Station