Prescription drugs and vaccines revolutionized health care, dramatically decreasing death from disease and improving quality of life across the globe. But how do researchers, universities and hospitals, and the pharmaceutical industry decide which diseases to pursue developing drugs for?
In my work as director of the Health Outcomes, Policy, and Evidence Synthesis group at the University of Connecticut School of Pharmacy, I assess the effectiveness and safety of different treatment options to help clinicians and patients make informed decisions. My colleagues and I study ways to create new drug molecules, deliver them into the body and improve their effectiveness while reducing their potential harms. Several factors determine which avenues of drug discovery that people in research and pharmaceutical companies focus on.
Funding drives research decisions
Research funding amplifies the pace of scientific discovery needed to create new treatments. Historically, major supporters of research like the National Institutes of Health, pharmaceutical industry and private foundations funded studies on the most common conditions, like heart disease, diabetes and mental health disorders. A breakthrough therapy would help millions of people, and a small markup per dose would generate hefty profits.
As a consequence, research on rare diseases was not well-funded for decades because it would help fewer people and the costs of each dose had to be very high to turn a profit. Of the more than 7,000 known rare diseases, defined as fewer than 200,000 people affected in the U.S., only 34 had a therapy approved by the Food and Drug Administration before 1983.
The passage of the Orphan Drug Act changed this trend by offering tax credits, research incentives and prolonged patent lives for companies actively developing drugs for rare diseases. From 1983 to 2019, 724 drugs were approved for rare diseases.
The viral ALS ice bucket challenge in 2014 was a fundraising success.
Elise Amendola/AP Photo
Emerging social issues or opportunities can significantly affect funding available to develop drugs for certain diseases. When COVID-19 raged across the world, funding from Operation Warp Speed led to vaccine development in record time. Public awareness campaigns such as the ALS ice bucket challenge can also directly raise money for research. This viral social media campaign provided 237 scientists nearly US$90 million in research funding from 2014 to 2018, which led to the discovery of five genes connected to amyotrophic lateral sclerosis, commonly called Lou Gehrig’s disease, and new clinical trials.
How science approaches drug development
To create breakthrough treatments, researchers need a basic understanding of what disease processes they need to enhance or block. This requires developing cell and animal models that can simulate human biology.
It can take many years to vet potential treatments and develop the…
