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Phil Vanek is responsible for technical due diligence and evaluation of potential investments, as well as guiding operational, R&D and strategic initiatives carried out at portfolio companies. An entrepreneurial and strategic international business leader, Phil joins Gamma Biosciences from GE Healthcare’s Cell and Gene Therapy business unit where he directed strategy and portfolio growth. Phil received his Ph.D. in Biochemistry and Molecular Biology at Georgetown University Medical Center followed by an IRTA fellowship at the National Cancer Institute in Maryland, and at the Hollings Cancer Center in Charleston, SC. Phil was an instructor for Johns Hopkins University Advanced Academic Programs teaching Biotechnology Marketing in the Masters of Biotechnology/MBA program, and has held leadership positions in a number of life sciences companies including Life Technologies, Becton Dickinson, and Lonza. Phil is a Board Member of CCRM in Toronto, Canada, and a Board Member of the ARM Foundation.
A theme often raised in our field is that the industry is “building the plane while flying it.” And, yes, full confession, I’m often the instigator of the discussion. Having been in and around the industry for almost 20 years, it seems that our plane has been under construction for a long, long time.
It will come as no surprise to anyone in the field that living therapies are highly complex and dynamic biological materials that are difficult to design, manufacture, distribute and deliver. The complexity is compounded by the broad spectrum of therapeutic modalities considered as advanced therapies, the wide range of therapeutic targets, and the lack of streamlined manufacturing processes and technologies designed to produce these therapeutics efficiently.
But clearly, as demonstrated by the preponderance of clinical trials, the growing list of approved and reimbursed products available to patients, and the attention and enthusiasm of the investment community, there is a lot to celebrate. So, how can we continue to make the necessary strides to affordably deliver these innovative medicines to patients around the world, and what are the hurdles?
As a return contributor to Signals’ blog carnival, I am identifying what I believe to be the hurdles to overcome for cell and gene therapies to reach the marketplace.
I think there are three key areas where the industry can double-down to fully materialize the promise of advanced therapies: investment prioritization, process simplification and regulatory convergence.
Perhaps the biggest near-term impact is to prioritize investment into good science, defined here as science that is readily translatable into the clinic. This may sound obvious; however, there is often a disconnect – putting dollars to work on interesting scientific ideas that are ultimately difficult to manufacture, transport or administer.
Due to the current economic environment, much of the investment re-prioritization has already occurred, refocusing efforts onto the most promising therapies. This natural selection of clinical assets is ultimately good for the industry, and it is my hope that the best therapies continue to be funded and advance to commercialization.
Secondly, there is too much complexity in advanced therapy manufacturing, driven in part by the rush to get therapies to patients, but also due to the concept of process optimization rather than focusing more deliberately on process simplification. This is a tall order, but to paraphrase Elon Musk, “why spend time optimizing a step in a process that simply shouldn’t exist to begin with?”
There are two dynamics at work here. First is the “we’ve always done it that way” mindset, and the second is the concern that the product made will be changed using a different process.
This occurs both in therapeutic manufacturing workflows and the supply chains that support them. In manufacturing, process changes are expensive due to the regulatory concerns of product comparability between processes. Therefore, in the “building the plane” framework, it’s faster and easier to not change than to take a step back to review and refine manufacturing steps and drive process efficiencies.
In supply chains, the same issue arises with “buyers” demanding a fair amount of customization to fit their manufacturing workflows, and “suppliers” wanting to build more “off-the-shelf” platforms because these drive economies of scale. In a more perfect world, we’d have manufacturing processes with fewer steps (and shorter times), more standardized consumables, and better analytical technologies to assess process changes and control manufacturing through automation. Of course, there is a lot of nuance here that is worth debating, but we’ll save that for another blog.
Thirdly, regulatory convergence, defined here as the purposeful focus on clinical data portability between geographies, can help drive both development cost efficiencies and market access by giving developers incentives to reach into new markets.
For developers, this makes innovative therapies available to more patients through a streamlined regulatory process using clinical data from one geography to support approval in another, thereby simplifying clinical trials and reducing development costs.
For patients this means that therapies can be more broadly accessible because of geographic availability across global markets, fully supported by the trustworthiness of the collective regulatory authorities’ shared oversight alignment. The combination of streamlined clinical trials reducing the duplication of effort in place today to reach patients globally, together with the larger economic incentives of global distribution, will hopefully speed up approval and adoption of advanced therapies.
In summary, although the industry continues to build the plane while flying it, the combination of activities summarized above and the continual investment into innovative solutions by therapeutic developers, life science tools suppliers, and regulators will help to overcome the challenges faced by the industry today. I am very optimistic that, like the biologics industry has evolved over the past 40 years into the mature industry it is today, the advanced therapy industry will chart its own flightpath to an incredible future and deliver on the promise of regenerative medicine in support of patients in need around the world! The sky’s the limit!
Please click here to read other blogs on this topic as part of Signals’ seventh annual blog carnival.
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