WASHINGTON (Sept. 12, 2017) - The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved about $38 million to fund five studies comparing the effectiveness of different strategies for treating multiple sclerosis (MS). These projects will focus on topics such as strategies for treating MS-related fatigue and treatment approaches for people newly diagnosed with the relapsing-remitting form of MS.
MS is a progressive neurological disorder with an unpredictable course that typically unfolds over many years. Symptoms can range from mild to severely disabling among the estimated 400,000 Americans with the condition. To choose which treatment options might work best in a specific situation, patients and their clinicians need information about the comparative benefits and harms of available therapies, but such data are often in short supply.
"This research will help patients and clinicians address some of the difficult questions that arise with a disease that has a wide variety of symptoms, such as fatigue, pain, loss of vision and loss of coordination," said PCORI Executive Director Joe Selby, MD, MPH. "As is the case with all our work, input from patients, clinicians and other stakeholders was essential to the process of identifying the research questions and health outcomes that these studies will address."
The five awards approved today include:
- A $13.5 million study based at Johns Hopkins University comparing two treatment options for people newly diagnosed with the relapsing-remitting form of MS. One option is an escalation approach, in which patients start taking a less-powerful drug with the option of switching to a more-potent therapy if the initial drug is not adequate. The other approach involves starting with a stronger drug that is potentially more effective, but also carries greater risk for significant adverse effects. This study will evaluate if patients can prevent or delay a worsening of MS symptoms by using stronger medications early on.
- A $10.6 million project led by the Cleveland Clinic that will also compare the escalation strategy with the more-aggressive approach. This study will monitor patient-reported outcomes and use magnetic resonance imaging to measure changes in patients' brain volume, a predictor of long-term disability that people with MS and their families consider very important.
- A $5.7 million study based at the Shepherd Center in Atlanta that will evaluate the effectiveness of different types of exercise programs for people with MS. Decreased mobility is a common symptom of the disease, and it can lead to less physical activity. This project will compare exercise programs provided at gyms or rehabilitation facilities with a telerehabilitation program available to patients at home.
- A $4.9 million project based at Case Western Reserve University that will compare three modes for delivering a course on fatigue-management strategies. Around 90 percent of people with MS experience fatigue. One strategy involves in-person instruction in the management strategies course during medical visits, and the two others involve delivery of the course via telehealth formats--teleconference or internet--to patients in their homes.
- A $3.5 million study led by the University of Michigan that also focuses on treating fatigue. This study will compare the effectiveness of cognitive behavioral therapy versus use of the wakefulness-promoting drug modafinil. The study will also assess a combination of both therapies. Some of each patient's information will be collected through a wrist-worn device that measures activity.
With these latest awards, PCORI has invested $64 million to fund 12 comparative clinical effectiveness research (CER) studies related to MS. The previously funded studies include research comparing strategies for how best to use disease-modifying drugs and ways to treat MS-related fatigue. PCORI also has supported the development of iConquerMS, a patient-powered research network within PCORnet, the PCORI-funded initiative to harness the power of data and patient partnerships to make health research more efficient and less expensive. More information on PCORI's MS portfolio is available on the institute's website.
In addition to the MS awards, the Board today approved $59.9 million to support six studies on other conditions that impose high burdens on patients, caregivers and the healthcare system. These include $18 million for two studies on strategies to treat adolescents with sickle cell disease as they enter adulthood and $8.8 million for a study on reducing long-term opioid use though improved management of chronic pain.
With these latest awards, PCORI's Board has approved $1.9 billion in funding since 2012 for more than 600 patient-centered CER studies and related projects to enhance the methods and infrastructure that support rigorous, efficient CER. All awards are approved pending completion of a business and programmatic review by PCORI staff, as well as completion of a formal award contract.
The Patient-Centered Outcomes Research Institute (PCORI) is an independent nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work.