Katya Tsaioun, Ph.D., CSO of Cyprotex, likens the pharmaceutical industry to the auto industry, given the unravelling of vertical integration and the movement toward specialized function each has seen.
For the most part, Dr. Tsaioun believes that niche CROs make the most sense “because no single organization, not even the largest pharmas, can now actually buy that much expertise. To get a compound from concept to market requires an enormous amount of very diverse expertise,” from basic biology and chemistry to pharmacology, formulation, animal toxicology, human toxicology, regulatory affairs, marketing, commercialization, and manufacturing. “The pharma industry is thus moving away from vertical integration and disaggregating its functions.”
The resulting demand for specialized expertise has pharmas turning to CROs, allowing pharmas’ research teams to focus on their core strengths. Dr. Tsaioun noted numerous other benefits of working with CROs, including expertise setting up the purpose-designed platforms and/or the assays that are outsourced, in turn saving on hiring and training time; assay validation; expertise to rescue failed or failing projects; and enough specialized knowledge to predict what assays might be needed downstream.
“A main scientific challenge is to combat the growing trend of failure in late stages of development or once in the market, either of which is hugely expensive,” Dr. Tsaioun said. “While failures due to pharmacokinetics have fallen dramatically over the last decade, failures due to adverse events in humans and to lack of efficacy are increasing.”
This highlights the need for better animal and human tissue models, along with better translation to human disease, as well new toxicity-assessment tools that can be applied early to reduce late attrition.
For example, Cyprotex has invested “massively into our highly automated ADME screening platform and into our CellCiphr™ high-content toxicology assays. At the scale and level of refinement that we have pursued, it wouldn’t make sense for even the world’s biggest pharmaceutical companies to invest at this level. By aggregating the volumes of hundreds of pharmaceutical companies, as a CRO, we see economies of scale that not even the large pharmas can feasibly attain,” according to Dr. Tsaioun.
As for addressing pharma’s need for earlier predicted eventual clinical relevance, Dr. Tsaioun cited various dedicated Cyprotex screening panels expressly designed for preclinical animal toxicity (rat primary hepatocytes) versus human-responsiveness panels—since not all toxicity mechanisms translate between animal and human assays. This alone can save customer companies hundreds of thousands of dollars, she claimed, given that proper IND-enabling studies cost roughly $1 million per compound.
“For years, the CRO industry was scientifically dull; we just executed. Now we are deeply involved in process innovation, frequently acting as an extension of pharma companies’ R&D teams. In the future, there will be nimble, fast-on-their-feet drug discovery companies spun out from universities, led by entrepreneurial leaders.”
Big pharma will continue to apply its decades-long expertise in managing drug portfolios—identifying markets and patient populations, determining manufacturing economics, assessing development-capacity and marketability of drug candidates—but, according to Dr. Tsaioun, it will be the CROs that “will innovate on how to get drugs from idea to FDA-approval quickly and cost-effectively, with the lowest risk of late failure.”