Electronic Health Reporter

By Alif Saleh, chief executive office, Scipher Medicine

With hundreds of thousands of clinical trials currently underway worldwide, we are continuously seeing innovation applied to how we treat and cure diseases, but the traditional four-phase method of getting there has not been updated since 1963. What makes this antiquated process for pharmaceutical companies and long wait time for patients in need worth it is the promise of a safe and effective therapy for the vast majority of patients.

This promise, however, is negated before the clinical trial begins when pharmaceutical companies opt to perpetuate the creation of blockbuster treatments for the “average” patient that disregards individual patient disease biology.

Today, many of the biggest selling drugs are still ineffective for the patients they are supposed to be treating. Approved drugs have shown to be largely ineffective for more than 225,000,000 patients worldwide. For example, anti-TNF (tumor necrosis factor) drugs are the largest selling drug class in the world but 65 percent of patients prescribed these drugs today do not respond adequately.

The cost of ineffective treatment for both patients and the healthcare industry is high in many ways, and there is a clear need to change the process to bring more effective treatments to market. The current system was developed to provide blanket treatments for a particular disease without considering the disease biology of individuals. Moving forward, pharmaceutical companies first need to study the individual’s disease and then create a personalized treatment for patient subgroups within each therapeutic area.

Precision medicine technology holds the key to meet this need and could change the current clinical trial system that has been in place for years. Companies are working on technology such as this to enable more targeted trials that are smaller, nimbler, equally as effective and safe, and encourage the creation of personalized treatments to finally break the cycle of expensive, ineffective blanket treatments.

Enabling smaller, more effective and affordable trials

For years, patients and doctors have started to become exasperated with this traditional, slow-moving clinical trial model and are searching for a more personalized route as an alternative. Precision medicine offers the unique ability to deeply understand the genetic makeup of patients’ diseases, which in turn would enable the development of better drugs with clinical trials that consist of sample sizes based on genetic disease make-up rather than phenotypic expressions.

Backed by preliminary research into the patients being treated, these smaller and more targeted trials can hypothetically be conducted more rapidly and at lower costs, allowing for breakthrough therapies to come to market faster at potentially more affordable prices.

Reshaping the clinical trial format would have a huge impact for both patients and pharma. Every day that patients live without effective treatments, it takes a toll on the quality and length of their lives while draining their bank accounts. Amidst ongoing conversations about astronomical U.S. drug prices, pharmaceutical companies claim research and development (R&D) costs attribute greatly to their inability to lower prices – they reported spending a record $71.4 billion on R&D in 2017. Controlling these costs could have a residual effect on overall drug efficacy and prices, once approved, which might create a less expensive, better outcome for patients through lower production costs with a more saturated market for pharma.

Putting an end to generalized treatments

Putting several years and hundreds of thousands of dollars into research and development means long, costly trials for pharmaceutical companies and many years of waiting for often ineffective therapies for patients. To optimize value and ROI for both pharma and patients, personalized medicine is key to help first study a patient subgroups’ disease biology and then safely develop a treatment. Technologies like ours at Scipher Medicine are the first step towards this new frontier of clinical trials and personalized drug development – a breakthrough at the system-level is close on the horizon.