Have you heard the oft-repeated “fact” that it takes at least 10 years from initial discovery for a new drug to enter the marketplace? Take it with a grain of salt. The drug development journey is closer to 30 years.
I’ve experienced the lag time between discovery and commercial success as the co-founder of a biotech startup, and now I study it at the Center for Integration of Science and Industry at Bentley University.
In the early 1990s, I co-founded GeneMedicine Inc., one of the first gene therapy companies. It had a successful startup, raised hundreds of millions of dollars, pioneered new product and business opportunities, and completed an initial public offering, which gave our initial investors a substantial profit.
But the company never developed a product. Over time, the path proved to be too difficult, the company succumbed to adventitious mergers, and the technologies we had so much hope for were abandoned.
This experience was not atypical. During the 1990s, more than 50 gene therapy companies were founded, raised and spent billions of dollars, and disappeared without developing a product. It would be quarter of a century before Spark Therapeutics (ONCE) launched the first FDA-approved gene therapy in December 2017.
The long gap between scientific discoveries and successful drug development isn’t unique to gene therapy. It took a quarter of a century to translate the Nobel-Prize-winning discovery of monoclonal antibodies into products for treating cancer, infection, neurological disease, and cardiovascular disease. It took almost as long for the discovery of tyrosine kinases, a critical cell control mechanism, to be translated into products such as Gleevec for treating cancer and immune disorders. Both monoclonal antibodies and tyrosine kinases now provide the basis for many new medicines coming to market.
Why does it take so long?
Our work at the Center for Integration of Science and Industry focuses on understanding the reasons for this lag and how to shorten it. Applying theories of innovation alongside big data analytics, we have identified predictable patterns in the progression of biomedical science and the development of new medicines.
Studies of innovation in areas such as computing and aeronautics show that the maturity of a new technology is the single biggest predictor of product success. That makes sense. When a discovery is made, scientists often have only a rudimentary understanding of what they’ve discovered. If the discovery is important, their work is confirmed and refined through continued research until the new knowledge matures into a reliable technology.
We developed a mathematical model that applies this concept to biomedical science and drug development. It examines the thousands of research publications that characteristically follow an initial scientific discovery, recognizes patterns in the progress of this research, and measures the maturation of this research over time. Our work shows that biomedical research follows a predictable pattern of growth and maturation, and that few new medicines are successfully developed before this research passes a critical established point.
How long does it take for a new area of biomedical research to mature and produce new medicines? The typical time is 30 to 35 years.
We believe there are things that can be done to accelerate this process.
While most efforts to accelerate the emergence of new medicines focus on expediting the penultimate stages of testing in humans and passing FDA review, our work suggests that paying more attention to the longest stage — the time required for the underlying technologies to mature — will pay off. More strategic allocation of capital resources could accelerate the translational process.
In a recent study, we examined the National Institutes of Health’s contribution to the new medicines approved between 2010 and 2016. NIH funding supported basic science associated with every one of those products. Consistent support for basic research is essential for the efficient growth and maturation of the basic science underpinning new medicines.
Our group has also studied investments made in early gene therapy companies, and found that it is important to synchronize investment with technology maturation. Investments in companies with immature technologies may accelerate the growth and maturation of their technologies, but are unlikely to produce products. In addition, when products are prematurely tested in pivotal clinical trials and fail, companies often pivot to older, more established technologies and the advances that were made are dissipated or even abandoned. Enormous amounts of capital, time, and talent can be expended this way without benefiting patients desperate for new cures.
Gene therapy research in the 1990s generated headlines, excitement, expectations, and billions of dollars in investment — in an immature technology. A quarter of a century later, gene therapy has matured and companies are on the threshold of fulfilling the long-held expectations for it.
Waiting 30 years for new medicines is too long. If scientists, clinicians, investors, and businesses could coordinate their efforts, this time could be substantially shortened. The public is waiting.
Fred D. Ledley, M.D., is director of the Center for Integration of Science and Industry at Bentley University in Waltham, Mass.