PCORI to Offer Up to $41 Million for New Patient-Centered Comparative Clinical Effectiveness Research on Opioids, Advanced Illnesses

WASHINGTON, DC — The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved the development of two new PCORI Funding Announcements (PFAs) that together will offer up to $41 million in support of comparative clinical effectiveness research (CER).

The PFAs will focus on two important topics. One will provide up to $16 million for research on the best ways to deliver medication-assisted treatment for opioid use disorders to pregnant women. The other will offer up to $25 million for studies on how to most effectively manage symptoms in patients with advanced illness. Each responds to key concerns voiced by healthcare decision makers.

The prevalence of opioid use disorders among pregnant women and new mothers has increased dramatically alongside the well-documented opioid crisis in America. Medication-assisted treatment involves counseling and medications such as methadone and buprenorphine. These medications reduce withdrawal symptoms and block the effects of heroin and prescription opioids.

It is not known which approaches to delivering this therapy achieve the best results, particularly among low-income women, making this a priority concern for the Medicaid Medical Directors Network. Medicaid covers about 44 percent of births in the United States annually. PCORI consulted with midwives, physicians, researchers and other specialists to determine how the CER it funds could provide the most useful evidence.

PCORI aims to fund up to four new studies through this announcement. Specifically, it seeks to support studies focusing on how best to provide this treatment to pregnant women with differing levels of addiction severity. Studies will compare patient-centered outcomes for treatment models that differ in resource intensity and that use different levels of integration of prenatal and addiction care, including remotely supported integrated care.

“The nation’s opioid crisis has devastating consequences for families,” said PCORI Executive Director Joe V. Selby, MD, MPH. “Pregnancy may motivate women to seek treatment, and PCORI wants to fund research that addresses some of the many barriers to care that they face.”

The need for CER to help patients and families manage the symptoms associated with advanced illness arose during a multi-stakeholder workshop on palliative care that PCORI convened in March 2016. Patients with illnesses such as cancer, advanced heart disease and kidney failure may face a variety of challenges, including symptoms, caregiver burden and repeated hospitalizations, which can lower their quality of life. Unfortunately, there is a lack of evidence on the most effective ways to manage patients’ symptoms as disease advances.

The new funding announcement will expand PCORI’s previous investments in palliative care CER by funding up to 10 studies that address evidence gaps in symptom management. In particular, it seeks to fund studies that compare two or more treatments, including at least one medication-based treatment, to relieve symptoms such as pain, fatigue, insomnia, depression and anxiety.

“Having received much feedback from many healthcare stakeholders highlighting the burdens of ill-managed symptoms among those with advanced diseases, we are pleased to move forward in issuing a funding announcement to tackle this high-priority topic,” Selby said. “We intend to fund research that will make meaningful improvements in patients’ and their families’ quality of life.”

Preannouncements offering preliminary details to prospective applicants are available on PCORI's website. PCORI will issue the two new PFAs and begin accepting letters of intent from applicants in June. As with all PCORI-funded studies, applicants must show meaningful involvement of patients, advocates, or other healthcare stakeholders in their applications.

These PFAs continue PCORI’s efforts to support patient-centered CER. To date PCORI has awarded nearly $1.2 billion for 365 patient-centered CER studies and $119 million to fund 142 projects to improve the methods for conducting CER.

In other business, the Methodology Committee presented an updated version of the PCORI Methodology Standards, and the Board of Governors adopted these revised Standards for public posting and dissemination. The Methodology Standards help to ensure that all patient-centered outcomes research studies are designed and conducted in ways that generate trustworthy findings that patients and clinicians can rely on. The current updates include new standards on analysis of clustered data, inclusion of a formal study protocol in public data and approaches to selection and usage of data networks.

Presentation materials and an archive of the webinar from today’s Board meeting are available on PCORI’s website.

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About PCORI

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.