Hello readers. It's been awhile. I'm pleased to be working with Reuters on some events including this upcoming webinar on how to utilize RWD
in cell and gene therapies with speakers from Novartis, BMS and Salesforce. Click here for more info.
The requirements of complex
and delicate value chains within cell and gene therapies (CGT) is something
pharma has traditionally never seen before. Leaders in CGT are innovating data
sharing approaches by utilizing RWE and RWD across the value chain, and
achieving multi-stakeholder alignment, to ensure commercial success.
Join C> delivery
trailblazers BMS, Novartis and Salesforce to learn how to turn data in
to true insights: that enhance the science, reduce the cost of deliver, improve
commercial decision-making and elevate the patient experience.
•Reimagine the
technology: digitally transform existing data integration models for cell and
gene therapies
•Reach for the future
together: develop a multi-stakeholder strategy collaboratively, that aligns on
needs and capabilities
•Cross-capture and
utilize data flow from R&D through to launch to win the marketplace with
science and data
•Prepare for and
address the major barriers to success: such as infrastructure non-readiness,
digitally siloed stakeholders, underdeveloped partnerships, and data illiteracy
•Turn data in to true
insights: that enhance the science, reduce the cost of deliver, improve
commercial decision making and elevate the patient experience
In anticipation of Phacilitate's 2014 Cell and Gene Therapy Forum to be held at the end of January in Washington, DC, I invited the Phacilitate team to provide blog readers with an interview of one of their upcoming faculty. The result is the very interesting Q&A thread you see below about GSK's call and gene therapy program combined with some of his thoughts on the sector.
Phacilitate: You head the Advanced Therapy Delivery group at GSK – please could you outline your specific goals and activities as an organisation for us? Which projects/technologies are you prioritising currently, and why?
Dr. Thirkettle: Our immediate priority is to deliver the Chemistry, Manufacturing and Controls (CMC) aspects of a collaboration we already have for an investigational ex vivo gene therapy currently in late stage development with TIGET, the San Raffaele Institute for Gene Therapy in Milan.
Through this collaboration we believe that we have a responsibility to complete the process for development of gene therapy, get it to file and make it available to more patients. We are really excited by the clinical data which TIGET have generated on the current programs thus far, but this is also laying really important groundwork for how we will work on other products in that collaboration and giving us real insight into the issues which we need to tackle in order to be able to progress following opportunities in this collaboration and make these autologous therapies more scalable.
This feeds into our second priority, which is to develop more efficient manufacturing technologies that can allow us to reduce the cost of goods, reduce the complexity, and increase the scalability of ex vivo gene therapy products. This is important within our existing collaboration but also it is going to be critical to allow us to engage with other opportunities which might have larger patient numbers.
Thirdly, whilst the immediate focus of our group is on the CMC aspects (process development, vectorology, cell characterisation, QC), we are also very focussed on working across the GSK platform organisation to leverage the expertise we have from our pharma and vaccine background and which is relevant to this area. This engagement and strategic planning is also critical to make sure that we build expertise in all of the other really key functions; for instance, this is an area where really strong understanding of the regulatory and the quality aspects is absolutely critical.
Phacilitate: This January, you will be joining a panel of big pharma representatives tasked with answering the questions of whether 2014 is the year pharma get serious about Cell & Gene Therapy, and whether it has been a lack of innovation or a lack of proven products that has held them back in the past - what’s your initial response to these questions?
Dr Thirkettle: I’m not sure it’s fair to say that big pharma has yet to get serious, if you look at the investments and deals of the past 3 years. I certainly don’t see any lack of innovation out there, it is almost the opposite. We talk about cell and gene therapy as a modality, but it is a huge, broad field; as broad as ‘biopharmaceuticals’ which is a field with many approaches and technologies.
Within the cell and gene therapy field we have a very large number of modalities, different cell types, different approaches, and a huge diversity of technologies. And they are all evolving at a really blistering pace. So one of the challenges for anybody who is outside of the groups who are really driving that core science is to know which is the right bit to grab hold of and it’s not the sort of area that you can just dabble in if you want to be really be effective and add value.
Rather than lack of innovation, then, the challenge is maybe more of knowing how to pick the winners at the right time and understand the potential utility of that technology/product at this early stage. I think the key thing is that it requires a number of elements to come together; you need the biology and the underpinning science to be strong; you need a therapy with good clinical data to really evidence that and discharge some of the risk; and then you need an understanding of how broadly applicable that technology might be - is there some sort of platform that sits behind it that spreads your risk? And finally, you need an understanding if whether you can actually make that product, can you scale it up?
Phacilitate: You are also kindly chairing a session which explores the long-standing but still critical challenges in developing cost effective and robust manufacturing models for cell & gene therapies at industrial scale. What progress do you see being made in this regard? To what extent will it be a case of these problems being solved relatively quickly and easily as big pharma come on board more and more, as some have suggested?
Dr Thirkettle: There are some massive challenges, but I suppose I would never underestimate the ability of really committed scientists, whether from academia or biotechs or big pharma, to solve those problems given enough focus and time.
I think the key is creating a focus because in a way it goes back to the previous point - this is a hugely broad field. When you stand back and look at this mass of really exciting science and opportunity, the challenge actually is to understand where to focus that investment in time and money. It becomes an awful lot easier to create that focus and energy to invest where you have got really compelling clinical data - you know you have got something that can potentially be a product. Equally if you can have confidence that a technology has multi product utility, your ability to invest is that much greater. If you can make a strong case on either of those points then you’re in a good position to create the focus needed to drive investment in the manufacturing and commericalisation aspects.
Phacilitate: Finally, what are you hoping to get out your participation in the meeting this January?
Dr Thirkettle: I’m really hoping first of all to meet lots of other people who are facing the same challenges and learning the same things. I think it is really exciting to see the journey over the past few year with increasing discussions around “How do we make these therapies? ” rather than “Will these therapies ever work?” This is a field which would potentially be greatly enabled if there players could find a way to share learnings as much as possible about what works and what doesn’t. There is a huge opportunity to work in a pre-competitive mode. It is in everybody’s interest because there are so many challenges, so many things to learn. I think this is a forum which makes it really easy to have those conversations, and start to build those relationships that, ultimately, should help everyone.
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:
