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Monday, November 17, 2008

How mature is the cell therapy industry? Do we know?

Survey
The week of November 17, 2008 I posed the following one-question survey:

How many phase III or pivotal industry-sponsored cell or gene therapy clinical trials do you believe are currently ongoing in the world?

to members of the following groups:
  • those who follow me on Twitter (currently a group 43 individuals sufficiently interested in cell therapy to follow my postings);
  • my connections on LinkedIn (a group of 188 professionals in or interested in the cell therapy sector);
  • the 90 or so members of the LinkedIn Cell Therapy Industry Group;
  • readers of this Blog; and
  • the roughly 55 members of ISCT's cell therapy commercialization committee.
There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-5 to 50+ in increments of 5 (e.g., 1-5, 5-10, etc).

Data, Assumptions, & Observations
There have been 18 responses to-date.

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

Based on these assumptions, the data leads to one simple, overarching observation:
There is significant divergence of perception (and perhaps misconception) about the maturity of the cell therapy industry even among those in and/or informed about the sector.
My database tells me there are 33 industry-sponsored cell or cell-mediated gene therapy trials currently underway around the world.

45% of survey participants answered fewer than 15 to the survey question; 33% indicated more than 45. In fact, only 1 participant (6%) do-date has chosen the category of 30-35.

Analysis
Admittedly this is an unscientific and informal survey. Furthermore there is likely some margin of error to account for different interpretations of what "industry-sponsored" means and/or what types of products should be included/excluded as "cell or gene therapies" since none of these terms were defined for participants.

Certainly in my day-to-day dialogue with people in the industry, the typical opinion is that there are but a handful of late-stage cell therapy trials (far fewer than what my data tells me actually exists). Having said that, when I asked a colleague in the industry yesterday to respond verbally to the survey question, they said "350" ( and then pulled back to say "maybe 200").

There is no doubt my data likely lacks a material number of industry-sponsored, phase III or pivotal gene therapy trials - given that the focus of my database is cell therapy and thus typically only includes cell-mediated gene therapies. This exposes a weakness in comparing the survey results to my data in that the survey related to "cell or gene therapy" while my data likely only reflects a small subset of gene therapy trials.

There is also without doubt more than 33 cell therapy or cell-mediated gene therapy clinical trials currently underway in the world with many of the indicia of a "late-stage" trial if one includes what is happening in markets where such trials or products are currently unregulated. I have not included these in my data because there is no mechanism for identifying whether such trials are, indeed, phase III or pivotal as there is no mechanism as yet in those markets for formal review or approval of these trials and/or products.

Nevertheless, what conclusions can/should be drawn?
  • There is much divergence of perception on the maturity of the cell/gene therapy industry even amongst those in the sector.
  • Even if my data is inaccurate, compared to whatever the real number is there is a significant % of industry professionals who are suffering from significant misconceptions about the true number of late-stage industry-sponsored trials in the sector.
  • Good data on the state of the cell/gene therapy industry is not readily accessible.
  • If those in the industry lack consensus on the state of the sector, it is no wonder investors and others outside the industry have mixed opinions on the matter.
  • We need to do a much better job as a sector in gathering and communicating solid data on the commercial side of the cell/gene therapy industry.
For those interested in the data, here's how it looks:


Industry-Sponsored Cell or Gene Therapy Phase III or Pivotal Clincial Trials (Nov 2008) - DocStoc

Coming soon...a similar discussion on the number of cell therapy products currently commercially available around the world.

In the interim, if any of you have data relevant to this discussion, please share...


6 comments:

Lee Buckler said...

Interesting that as I was finalizing this blog, two more people participated in the survey. One voted for under 15 and the other for over 50 thus reinforcing the conclusions. For those data-freaks out there the data now looks as follows:

15 or less - 45%
20 to 40 - 20%
45 or more - 35%

Alexey said...

if there is no information available online (where is your database?) about these trials, we can only guess how many of them going on right now.
There are a few books "Cell Therapy Market" issued annual at least 2-3 marketing companies. Each of them cost about 2500-4500$ USD. Even annual update sometimes not reflect the reality.
So I can conclude this information available only for business professionals.
I just wondering why this info should be classified?

Lee Buckler said...

Well, as they say, Alexey ..."nothing good's for free". My database is in part how I make my living so it won't be posted online anytime soon. I am, however, willing to put out there in the public domain some of my analysis from the data such as I did in this blog. I put my stake in the ground saying I think there are 33 corporate-owned cell therapy products (as I define them) currently in a registered phase III or pivotal trail around the world.

I've seen a number of the market reports on cell therapy, stem cells, and/or regenerative medicine and none that I have seen purport to be exhaustive in terms of number of companies, products, or trials. They all list what is a "representative" list or a list of the leading companies but none suggest it is "the" list. As you state, even then almost always have out-dated information in them by the time of publication. I spend time everyday updating my database and I still find companies I haven't heard of or information about companies that somehow I missed.

Nonetheless, together with the market research companies and other consultants in the field, I hope to build a common understanding, perception, and awareness of what the sector looks like so we can effectively communicate that to policy makers, investors, the larger biotech, pharma, and business communities, and the public in general.

JonRowley said...

Lee

i tend to see myself as somewhat informed of what is going on in the industry, and I was in the 15 or less catagory.... i would tend to think your database has a lot of value, and i would keep it close to the vest (as it sounds like you do).

cool results, i look forward to more surveys.

Jon

Alexey said...

Lee,
thanks for your comment, I voted "50 and more",
also, I think people have different definition of "cell therapy products", that's why variety of answers.
Can you tell us (one more time) how you define "cell therapy product" when you counted them in the trials?

We can discuss about definitions that should be clarified further (I have a list of them) on your and my blog, I think it's important to make a statistics and surveys.

thanks

Tim Leeder said...

Lee,

you make some very interesting observations and when I met with Anthony Hollander ( http://news.bbc.co.uk/1/hi/health/7735696.stm ) we touched on this topic.

My analogy: "Back in 1904, after watching the Wright brothers' maiden flight, who would propose creating a manufacturing plant for jet engines? Much less an automated production line!"

My point: that we are still at the very early stage of cell/regenerative medicines the broader strategic questions (ie autologous/allogeneic and the applicability of each to specific treatments or classes of disease) are not clear. Then there is an added complication in the form of the ethical considerations around particular cells/paradigms.

I guess that I agree - we don't know - and most importantly we need to rapidly scope the broad areas of likely success prior to the implementation of a (dogmatic) solution/investment based upon any one particular view. I don't believe that this needs to take decades, or that it needs to cost $100m's - in fact, I think, technology innovation will take an order of magnitude off both and create step-changes in understanding very shortly.