REGENERATIVE MEDICINE
Over the Valley and Through the Hoods
Understanding the Fundamental Drivers Supporting the Growth of Translational Research
Translational research has experienced incredible growth within the past two decades. And although life science ventures are notorious for their challenging clinical timelines and high failure rates, various factors are accelerating the discovery and development of new tools and therapies.
With an industry as complicated and multifaceted as the life sciences, there are a lot of factors that affect the commercialization of promising technology. When mistakes mean serious injury or even death. It comes as no surprise that this field is known more for its evolutionary, rather than revolutionary growth. However, the demand for better treatments is growing daily, and maintaining an equilibrium between patience and ambition requires careful consideration. While this field remains one of the most volatile and heavily regulated industries, there are plenty of factors that have and will continue to promote the discovery of new tools and therapies.
While people are quick to look for a pivotal moment in the early 21st century marking an inflection point toward positive growth, we need to remember that disruptions in regulated businesses, where lives are at stake, require intentional evolution, rather than revolution [1]. Certain events, such as the Obama administration lifting the ban on federal funding for human embryonic stem cells, are momentous occasions. However, with so many positive developments occurring at the same time, it’s hard to believe that any one change deserves all the credit [2].
Regardless of initial hopes, biology is complicated, drug development is difficult, and failure is the most common result [3]. The majority of preclinical programs never advance to human studies, and less than 10% of drugs entering clinical trials eventually receive FDA approval [4]. Accounting for failures, the average cost of developing a new drug exceeds two billion dollars [5]. More importantly, FDA approval does not ensure clinical adoption. Doctors may choose not to prescribe an approved drug, and health insurers may not cover the costs [6].
Startup Support
Unlike software developers who can establish a proof of concept from a dorm room, a life science start-up requires lab space and a significant amount of funding. Within the past two decades, more universities are constructing what are known as Academic Innovation Centers (AICs). These facilities are designed to support young biotech entrepreneurs with the resources, expertise, and funding required to get their ideas off the ground.
There has also been an increase in the number of external incubators that serve a similar, almost progressive purpose. Incubators, sometimes referred to as accelerators, are designed to support the growth of young start-ups with facilities, equipment, and advisory services. Although these facilities appear similar to AICs, they tend to place a greater emphasis on accelerating the initial concept toward sustainable growth.
Funding, infrastructure, and resources aside, it’s worth highlighting the business expertise provided by both incubators and AICs. Young biotech entrepreneurs are often highly specialized scientists with limited prior exposure to business or entrepreneurship. Because of this, it’s crucial for these facilities to help with developing sustainable business models, financing, hiring staff, and ultimately scaling toward profitable growth.
Venture Capital (VC) firms have adopted a similar model for their long-term investment strategy. Not too long ago, most academic startups would spin out, assemble a pitch deck, and quickly look for funding. While this practice is still very much in effect today, more VCs working with a talented pool of entrepreneurs-in-residence (EIRs) will scan the waters for innovative new tech emerging out of academia and form a company around it [7]. These startups are often incubated in-house with VCs or EIRs temporarily taking on operating roles.
Enabling Technologies
Enabling technologies, such as advancements in AI and machine learning, dramatically lower startup costs, and increase efficiency. These technologies streamline computational drug design, high-throughput safety screening, DNA sequencing, bioinformatics, lab automation, biomarker identification, and a number of other previously cost-prohibitive factors. A systematic approach to developing and testing novel biopharmaceutical drug candidates will save billions of dollars and decrease the time and energy spent on determining the safety and efficacy of new therapies.
Contract Research Organizations
The growing trend of outsourcing clinical research and development to Contract Research Organizations (CROs) greatly decreases the developmental costs associated with identifying and manufacturing new tools and therapies. For those unfamiliar with CROs, they’re a relatively recent infrastructural advancement designed to support the pharmaceutical, biotechnology, and medical device industries with outsourced research services [8]. These services include preclinical and clinical research, pharmaceutical development and commercialization, clinical trial management, and pharmacovigilance. These services reduce the initial costs associated with product development and provide the required expertise to navigate scientific, commercial, political, and regulatory uncertainties.
Regulation and Standards
Over the years the FDA has made some notable contributions toward accelerating the regulatory process, including the creation of several programs to expedite the approval of new drugs. Five of these programs include designations for Fast Track, Breakthrough Therapy, Priority Review, Regenerative Medicine Advanced Therapies, and Accelerated Approval [9]. However, navigating the regulatory gauntlet is still one of the most daunting steps for entrepreneurs and investors.
Regulatory and reimbursement policies profoundly impact the amount of funding and types of projects that investors pursue. De-risking the regulatory process will more evenly distribute the skewed left allotment of capital currently concentrated around later stage ventures [10]. Whether you’re filing for an Investigational New Drug application (IND) or approaching Pre-Market Approval (PMA), the process is full of uncertainty and only a small percentage of products ever get approved. Because of this, over the years there has been a greater emphasis on better standards and regulatory transparency. Organizations such as the National Institute of Standards and Technology (NIST) and the American Society for Testing and Materials (ASTM) continue to broaden the availability of standards.
Various non-federal organizations emerged to support these efforts. One example is the Standards Coordinating Body (SCB), a non-profit organization focused on coordinating with key stakeholders and thought leaders from industry, academia, professional societies, and government entities to accelerate the translation of regenerative therapies by creating better standards and increasing transparency. Organizations like the SCB demonstrate the physical manifestation of public sentiment regarding the need for better communication throughout the approval process to minimize costly uncertainties that jeopardize billions of dollars of invested capital. Better standards will provide early-stage entrepreneurs with the necessary confidence to proceed while allowing organizations such as the FDA to better determine the safety and efficacy of the clinical pipeline.
Scalable Manufacturing
Similar public sentiment exists around the need for advances in large scale manufacturing. In a 2015 report entitled “Manufacturing Road Map for Tissue Engineering and Regenerative Medicine Technologies”, authors from the Wake Forest Institute for Regenerative Medicine (WFIRM) discussed the need for scaling up the manufacturing of regenerative therapies [11]. The authors reflect on the positive effects of automation on the automobile and semiconductor industries. “It was not until the concept of interchangeable parts, just-in-time production, and the moving assembly line reduced the cost of cars by half that automobiles became commonplace”. Although the human body is vastly different from cars and computers, automation and scalable manufacturing will decrease variability while increasing control over the final product.
Conclusion
With these incredible advancements supporting translational research in tandem, the industry is primed for continued growth. Non-dilutive funding will encourage new research. Supportive infrastructures, such as incubators and AICs will allow for a smooth transition out of academia. Computational advances will cut costs and increase efficiency. Improved regulation and standards will minimize uncertainty and skepticism. And scale-up manufacturing will improve product quality and consistency.
As a society, we need to shorten the time and lessen the effort it takes to turn science fiction into science fact. By continuing to bridge the chasm between business and science, while supporting the commercial translation of new ventures, we can make sure that promising therapeutics and devices make their way to a patient and are not lost to a publication.
Jack is results-driven with a refined aptitude for problem-solving and the communicative expertise to convey information in a way that is both professional and understandable. He is driven towards efficiency via algorithmic decision making to add value and maximize clarity when extracting insights. With extensive experience in healthcare, biotechnology, and the life sciences, he is committed to increasing the availability of new cures and treatments by supporting innovation from discovery to commercialization.
