The challenge faced by academic innovators
In the biotech community, there is a problem so common and so frustratingly difficult for many innovators to overcome that it is called the “Valley of Death”. The Valley of Death is where laboratory discoveries and cutting edge innovations die; running out of traction before they ever receive the opportunity to become potential life-saving treatments and provide value to our society.
Drug development is an extremely costly endeavor with a multitude of complex and specialized areas in which a potential drug can fail along its path to commercialization. Even when an innovation has received large-scale investment, the odds are still stacked against it ever coming to market. As a result, knowledgeable investors in the space do everything they can to ensure their dollars are placed in areas where there is the highest chance of success.
With this in mind, there are many steps that a discovery in a test tube or petri dish must go through before it will gain traction with potential partners who can bring it to market. Unfortunately, many of these steps lie outside the areas of expertise of the academic investigators who have made the initial discovery. This is the Valley of Death: a skills and expertise gap that includes many of the following aspects.
Business Case and Patent Strategy
A potential drug requires thorough market, intellectual property, pharmacoeconomic, and financial analysis. This analysis differs from the standard business case that can be analyzed by staff in a university commercialization accelerator due to the immensely complex regulatory frameworks and industry specific hurdles that drug candidates must overcome. It requires business strategies that are built into the scientific work and strategically managed in a holistic manner. It is seldom a straightforward route and requires expertise that can understand all aspects of drug development and is engaged as early as possible in the drug development process. A lead compound may demonstrate spectacular efficacy, but it will fail if there is not a strong market and patent strategy behind it.
The steps between a discovery in the lab and successful testing in humans lies within the disciplines of the pharmaceutical sciences. To develop a drug requires a team of experts in toxicology, pharmacokinetics and dynamics, formulation, pharmacology, and medicinal chemistry. While other disciplines touch on some of the concepts found within these areas, there are a myriad of subtleties that can be missed and cripple a development program.
Regulatory Compliant Science
A very common reason that potential drugs fail is that development and pre-clinical study results do not meet regulatory requirements. While an idea may show promise in a university lab, studies must be performed in accordance with a properly documented quality control system to ensure that the results are consistently reliable and reproducible. The pharmaceutical industry no longer accepts standard academic laboratory results even as proof of concept. In order to be of interest, research on a drug candidate must be done at a GLP/GMP/GCP level. This work also needs to be structured in a manner that will address as questions about a drug candidate’s efficacy and safety as early in the development process as possible.
A Proof of Concept with Time for Development
Once there is a patent on a drug candidate, a clock begins ticking. The longer it takes an innovator to develop a very solid proof of concept at a regulatory compliant level, the less likely it is that s/he will find investment. The period of market exclusivity diminishes with each passing day. Generally, a drug candidate needs to finish a regulatory compliant Phase II clinical trial before it will attract a licensing partner or investment, but if it gets there with not much time left on its patent, there won’t be a strong case for an investor to spend the hundreds of millions of dollars required to move through Phase III.
With all of these factors in mind, researchers are faced with a seemingly insurmountable challenge if they choose to move down the path of drug development. Current frameworks in Canada simply do not provide the support to move biotech innovation forward. Researchers faced by this Valley of Death are left looking for funding to hire a professional contract research organization (CRO) to meet some of these requirements—something that can cost millions of dollars simply for a proof of concept that could determine a drug candidate to be nonviable. Even if they are successful in achieving regulatory compliant proofs of concept, if they missed something in their business case and patent strategy, or have run out the clock on their market exclusivity, they will be faced with a dead end in their ability to gain investment required to complete Phase III. Life-saving discoveries are trapped, public investments made in post-secondary innovation are crippled, and world-class researchers spend years on a dead end.