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.
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.
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.
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...