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.
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.
One of the sole national issues that the government can agree on is bringing down the astronomical costs of drug prices in the United States. The Senate Finance Committee and House Oversight Committee began to take on drug prices by holding two hearings this year on the issue with leading pharmaceutical companies, and a third planned for early April to examine pharmacy benefit managers’ role in the issue after they were called out as major contributors in previous hearings.
During the February meeting, the biggest players in the industry testified before the committee and it became clear that big pharma and the government are having trouble coming to a solution.
There has been much discussion around this critical issue of drug pricing in America but now a few innovators are shifting the conversation to how precision medicine provides hope for a possible solution. Therapeutics come with the risk of not working effectively for some patients and causing adverse side effects. However, applying precision medicine stratifies patients based on their disease biology and matches that patient group to the drugs that target their specific disease.
The drug prescribed will be more likely to effectively work and the cost of treating the patient group will decrease. While the House Committee on Oversight and Reform’s investigation can eventually lead to legislation to mitigate price hikes, precision medicine technology currently exists to be a key driver in deflating drug spending for patients and payers and, thus, reducing drug prices. Two ways this type of technology can help to lower drug costs are: cutting spending on ineffective treatments and motivating the industry to develop and market personalized treatments.
The healthcare industry currently wastes roughly $2.5 billion in ineffective treatments annually. Take for example, the 1.3 million Americans living with rheumatoid arthritis (RA) – because autoimmune diseases like RA vary significantly from patient to patient, blockbuster therapies, which are “targeted” therapies and therefore only treat one pathway of the disease, are not effective for 66 percent of patients taking them.
This means that patients who see little or no benefit from these treatments are still paying up to $38,000 annually, proving to be a significant financial and emotional burden to patients and their loved ones. As medicine advances, it is becoming more apparent that giving these drugs to everyone diagnosed with the respective diseases no longer works, they need to be prescribed to sub-groups of patients based on their specific disease biology to truly be effective.
Through RNA data analysis, precision medicine can both help patients with autoimmune diseases like RA find targeted treatments that they are more likely to respond to before treatment is prescribed and direct pharmaceutical companies to develop targeted drugs specifically for those who do not respond to currently available therapies.
