PCORI Board awards $18 million for two new studies on sickle cell disease

The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved about $18 million to fund two studies comparing different ways to manage the transition of people with sickle cell disease from pediatric to adult care.

Although children with sickle cell disease generally receive the care they need to manage their condition, the transition to adult care can be hazardous. Many primary care clinicians who treat adults are unfamiliar with caring for this rare condition, and patients can find it challenging to access care across specialists. Losing a source of care, such as pediatric specialty care, decreases the likelihood that young adults with sickle cell disease will receive preventive care and screening visits, and it increases the chance they will seek emergency department care when painful symptoms flare.

"These newly funded studies will address important gaps in our understanding of how to improve care for older adolescents and young adults with sickle cell disease," said PCORI Executive Director Joe Selby, MD, MPH. "The good news is that young people with this serious disease are more likely than ever to live well into adulthood. The comparative clinical effectiveness research that PCORI funds can give these patients, their families and clinicians the information they need to make better-informed decisions about their care options."

The two awards the Board approved today are:

• A $9.8 million study based at Carolinas Medical Center comparing two interventions that have proven effective in facilitating care transitions for people with other conditions. One intervention involves peer support in addition to an education program designed to help young people learn to manage their condition. The other entails the education program alone. The study will determine how effective each approach is in reducing emergency department visits and hospital admissions and in improving quality of life among young adults with sickle cell disease.
• An $8.5 million project led by the Children's Hospital of Philadelphia that will compare three approaches: community health workers engaging with patients, use of mobile apps to help individuals manage their condition, and enhanced usual care. The study will test which approach is most effective at improving patients' quality of life during their transition to adulthood and decreasing their dependence on the emergency department.

These two projects join five others previously awarded that focus on sickle cell disease. Together they are a part of PCORI's $80 million investment in research on rare diseases.

In addition to these awards, the Board today approved more than $80 million to support nine studies on other conditions and problems that impose high burdens on patients, caregivers and the healthcare system. These other studies include five testing strategies to treat multiple sclerosis.

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.