Here is the link to my article that appeared today in El Mundo's (translation below) supplement on innovation and technology (Innovadores). El Mundo is one of the most prestigious newspapers in Spain.
Much has been said about the major discoveries that will change our lives. These range from treatments for cancer to those that change the course of the increasing number of rare and new diseases. Developing cures or treatments aligns with the business goal of developing a product or intervention that will improve lives and generate revenue for the innovator company.
Given all the products that are in the discovery pipeline it is clear that companies want to develop new products that improve or enhance lives. And of course the product must be priced so that it is affordable; without sales there are no revenues. Unfortunately, too often in the discussion of the need to develop new treatments the arguments about price are at best myopic. One way to manage prices is to decrease the cost of drug development that according to industry estimates is about $1.2 to $1.3 billion. Advances in different fields like engineering, information technology, and telecommunications need to be applied to drug development.
Other opportunities lie in building new methodologies for clinical trials so that we know more precisely the characteristics of people who will benefit from the treatments that are in development. What we know is that the bulk of trials are based on models of drug and treatment development that ignore that health is very individualized.
There is much upbeat talk about the promise of precision medicine or personalized medicine but it will remain just talk as long as the clinical trials that underlie the development process continue to be structured in a way that is neither precise nor personalized. The innovator working with regulatory agencies has to rethink these critical aspects of development.
The use of animal studies has long been an essential part of basic research. Yet even in these often seminal efforts there have been huge omissions that effect everything from validity to reliability. Reardon pointed out that, “In 2014, the NIH [United States National Institutes of Health] began requiring researchers to include female animals in studies, and giving out supplementary grants to those who complained about the cost." The omission of female animals was not good science.
Likewise factors that also effect outcomes in animal studies are what the animal has been fed and its living conditions. Research indicates that variations in these conditions change the health and longevity of the animals, which impacts on the outcomes and the ability to replicate the research. Although leading experts indicate that animal studies are becoming less relevant we still often extrapolate from animals to humans.
When it comes to studies of people our failures to develop targeted treatments and interventions are compromised even further. To develop precision or personalized medicine clinical trials must at a minimum include and analyze information using race, ethnicity, and gender. These are characteristics that are essential to understanding the success or failure of treatments. The process will be enhanced as information about the individual’s genetic and microbiome become more available.
The solution is not to spend more money on larger clinical trials but to use the advances in all of the sciences and design trials that are better defined, that are inclusive, and carefully monitored. This will make it possible to develop medicines and treatments that meet the health needs of the individual, the revenue goals of the innovators, and most importantly, continue to support future discoveries to benefit us all.
 Reardon, S. "A mouse's house may ruin studies— Environmental factors lie behind many irreproducible rodent experiments." Nature. February 18, 2016 vol. 530 Pg. 254