When a new drug receives approval from the Food and Drug Administration, the pharmaceutical company that created the treatment receives a flood of press. But sometimes lost in that publicity is the crucial role that patient volunteers like Phyllis Smith play in the process of bringing a new drug to market.
In 2007, Smith was diagnosed with lupus, a disease in which the body’s immune system can’t differentiate between its own cells and foreign substances. The illness—which most commonly strikes the skin, joints, blood and kidneys—has no known cure and can be life-threatening.
“I was healthy all my life,” said Smith. “I loved to jog. I loved to garden and spend time in the sun. All of the sudden, I couldn’t do those things anymore.”
Smith began traveling from her home in Clinton, Okla. to receive treatment at OMRF. In the course of her visits to OMRF, she learned of a way she could help speed the development of new medications to treat lupus: by volunteering to participate in clinical trials.
New drugs must go through a multi-stage testing process, first in a test tube, later in animals, and finally in humans before it can be known if they are safe and effective enough for general use. OMRF offers patients the chance to take part in clinical trials for treatments that are in development for lupus, rheumatoid arthritis and multiple sclerosis. Some trials compare the safety and efficacy of new compounds with existing treatments. Others see whether adding the treatment to current therapy leads to improvement. Before any treatment can be considered for approval by the FDA, both safety and effectiveness must be demonstrated in large numbers of patients and this is compared to what can be accomplished with existing treatments.
The trials are often “blinded,” meaning patients do not know whether they are receiving the new drug or an inert substance, called a placebo. In Smith’s case, she came in about once a month to receive an injection. “I didn’t know if I was receiving the drug or not, but that’s part of the test—to see how I react either way,” she said. Even the researchers did not know whether she was assigned to treatment or placebo. They had to assume, while monitoring her safety and well being, that she might have been.
While individual patients may be enrolled in a clinical trial for a period of months, the process typically involves testing a drug in thousands of patients at hundreds of sites around the world. The vast majority of drugs fail to make it through clinical trials, and for those few that do, the process may cost hundreds of millions of dollars and last more than a decade.
“Bringing a drug to market takes years of testing, which means patients can wait a long time for new medications,” said Joan Merrill, M.D., who chairs OMRF’s Clinical Pharmacology Program and serves as an investigator in more than a dozen clinical trials. “Before the recent FDA approval of the lupus drug Benlysta”— for which OMRF was one of the study sites —“it had been more than 50 years since a drug to treat lupus had been approved.”
While the approval of Benlysta is an important step forward in treating lupus, it does not allay all of the disease’s symptoms, nor is it effective in all patients.
“Because lupus is caused by many different genes and does not behave the same way in all patients—there’s not going to be a silver bullet that will cure every patient,” Merrill said. “It’s going to take time, resources and knowledgeable and empowered patient volunteers like Phyllis Smith to help us find the next generation of lupus drugs and ensure better care for everyone.”
For Smith, the desire to help others drives her decision to volunteer for clinical trials. “I may not see a cure in my lifetime, but the information they get from my participation in the drug trials will help them find better medications for other patients I’ll never meet,” she said. “My reward is to make a difference.”