In the first and largest researcher-initiated blinded study comparing the effectiveness of a drug treatment for rheumatoid arthritis, researchers found a $1,000 treatment and a $25,000 treatment to have the same effectiveness. The costs are for one year of treatment before insurance.
In a two-year study, researchers compared the oral, “triple therapy” disease modifying anti-rheumatic drugs (DMARDS), methotrexate, sulfasalazine and hydroxychloroquine, to one DMARD (methotrexate) plus etanercept. Etanercept (brand name Enbrel) is part of a class of injectable drugs called TNF antagonist or anti-TNF therapy, also known as biologics.
Triple therapy was originally popularized in 1996 when researchers at UNMC published a landmark study in the New England Journal of Medicine.
Results were presented Oct. 20 at the American College of Rheumatology Annual Scientific Meeting in Philadelphia.
“Before the study, there was a general belief that biologics have significantly more potency, but this study has proven that not to be the case in this patient population,” said James O’Dell, M.D., co-author of the study and chief of the University of Nebraska Medical Center and Omaha VA sections of rheumatology and immunology. “The study shows when conventional therapy is used before biologics, there should be a significant cost-savings not only to patients, but to the health care system.
“Insurance doesn’t always cover treatments or if it does, it may not cover all of the drug costs, said Dr. O’Dell, an international expert in rheumatoid arthritis. “The more studies like this to compare things, the better off we’ll be. This is comparative effectiveness research at its best because it was done in a double-blind randomized trial where all patients received proven effective therapies. Unfortunately, many pharmaceutical sponsored rheumatoid arthritis studies are unethical – subjecting patients to placebos when effective therapies are available.”
Rheumatoid arthritis is a chronic disease that causes pain, stiffness, swelling and limitation in the motion and function of multiple joints. Though joints are the principal body parts affected, inflammation can develop in other organs as well. Of an estimated 2.5 million Americans with the disease, the disease typically affects women three times as often as men.
The studyevaluated 755 newly-diagnosed patients in 26 states over two years to determine reduction in pain, swollen joints, inflammation in the body and general improvement and function. The study was a double-blind study, in which neither the patients nor their physicians knew which regimen they were receiving.
Patients were divided into four treatment groups: two groups took the triple oral combination therapy, or methotrexate and etanercept, while two groups began with methotrexate then had sulfasalazine and hydroxychloroquine or etanercept added if their disease remained active.
“This study was the first to directly compare these two therapies, and data from this investigator-initiated study provides critical information for researchers,” said Larry Moreland, M.D., of the University of Pittsburgh Medical Center and lead author of the study. “However, much more work is needed in order for physicians to be able to better prescribe the most effective therapies for individual patients. Specifically, this study has not yet examined data from patient x-rays, which could provide crucial future data for determining how each of these drug treatment affects the body.”
One of the major reasons for the study was the significant difference in cost of the two drug regimens.
“We are looking for the safest and most effective medications. If we can do that with cheaper medications, then we’re all better off,” Dr. O’Dell said. “When I see my patients, I know I have a lot of different treatment options to offer them. Twenty years ago that wasn’t the case. Prognosis for patients newly diagnosed with rheumatoid arthritis is excellent if therapy is started early.”
Dr. O’Dell said the reason for the cost difference between DMARDs and biologics primarily is they’re made differently. DMARDs are made with synthetics versus biologics, which are much more complicated compounds that require more sophisticated production and monitoring techniques.
He said the considerations of which medication to prescribe include the patient’s disease state and potential side effects.
Biologic therapy is associated with risk of infections, including tuberculosis. They also have been associated with an increase risk of multiple sclerosis-like symptoms and leukemia and lymphoma. “Cancer is a concern but there’s accumulating evidence that the risk is small compared to the benefit,” Dr. O’Dell said.
He said patients on the triple therapy have minimal side effects that might include skin rashes and stomach upset. Patients on triple therapy are monitored for low white blood count through blood count and liver function tests.
Disclosure of interests statement: Dr. O’Dell has served as a consultant for Amgen, the maker of Enbrel.