For more than a decade, the United States has faced an escalating opioid abuse crisis. The number of deaths associated with abuse of these prescription painkillers more than tripled between 1999 and 2012 and now outstrips the number associated with any other drug, according to the US Centers for Disease Control and Prevention. The problem is estimated to approach $56 billion in costs to society.
Often, opioid abuse or overuse is associated with patients seeking effective relief from the common and often devastating problem of chronic pain, which has been reported as affecting up to one in three US adults. People in pain often request opioid medications, not understanding that these drugs don’t effectively treat certain types of pain, don’t work well for many people, and can lead to a range of additional health problems.
Researchers, policy makers, clinicians, and their professional organizations are working hard to stem the opioid abuse epidemic, seeking ways to reduce overprescribing of these drugs by expanding the use of alternative and supporting therapies, as well as identifying more effective treatments for addiction.
These are important steps, but we clearly need more evidence-based information about how to better treat chronic pain. We at the Patient-Centered Outcomes Research Institute (PCORI) believe that when it comes to this issue, we can help by funding research that compares what works best, for whom, under which circumstances. In the last three years, we’ve funded at least 16 such projects that, as in all of our work, focus on the questions and outcomes most important to patients.
Focusing on What Patients Want
People suffering pain, or caring for someone in pain, want information that is relevant, trustworthy, and clear to inform their healthcare choices. That’s what PCORI-funded comparative clinical effectiveness research (CER) seeks to do – provide patients, their caregivers and clinicians with more and better information on the pain care options available to them and which therapies are most likely to help them achieve the outcomes they desire.
Patients may come to their clinicians with a range of questions about pain control. Which medications work best for the type of pain I am suffering? How will these drugs affect my quality of life and daily functioning? Will my age, gender, or other personal traits affect how the medication works? Can meditation, support groups, or physical therapy make a real difference in my ability to manage pain?
Answering these questions for patients and those who care for them can help change the course of pain treatment in America. It will enable patients and clinicians to confidently choose safer and more effective options that reflect their preferences and concerns.
PCORI was established to address just such as challenge—to fund studies to answer patients’ questions about our nation’s most common, burdensome, and expensive health problems.
Looking for Better Answers
Since 2012, PCORI has built a sizeable portfolio of studies on chronic pain, two of which specifically address opioid treatment. Here are some of the questions we’re tackling:
How can we improve patients’ ability to communication confidently with doctors about pain and pain medication?
What approaches to care delivery can minimize health and safety risks when long-term opioid use is considered?
What collaborative-care approaches to pain can improve quality of life, patient satisfaction, and adherence to other treatments?
How can we encourage patients to join and stay enrolled in integrative treatment plans that show promise for reducing chronic pain while lowering risk for opioid abuse?
Also, in our list of priority topics for large pragmatic studies (see below), we include strategies for preventing the progression of episodic back pain into chronic back pain.
We also plan future research relevant to pain treatment. For example, the topics in our research prioritization process pipeline include long-term use of opioids in treating chronic pain generally as well as treatment of chronic low-back pain and musculoskeletal pain specifically. In June, we are hosting two multi-stakeholder workshops to discuss whether CER studies can help to address these issues; the discussion may lead to the creation of a targeted PCORI funding announcement or the listing of other topics related to pain as high-priority areas in funding announcements for pragmatic clinical studies.
Among the questions that the workshop may consider:
In patients with chronic pain, what is the comparative effectiveness of opioid versus non-opioid versus combination interventions on outcomes related to pain and function?
In patients on long‐term opioid therapy, how does decreasing opioid doses compare to continuation of opioid dose on outcomes related to pain, function, quality of life, and withdrawal?
How do the harms of opioid treatment vary depending on the type or cause of pain, patient characteristics, or dose of opioids used?
I spoke about PCORI’s pain research portfolio—studies designed to answer patients’ questions about pain treatments and how information from CER can help reduce opioid overuse and abuse—in a keynote address at the American Pain Society’s (APS) annual scientific meeting in Palm Springs, Calif. I was particularly pleased to speak to this group because last December, the APS published a “Pain Research Agenda for the 21st Century,” identifying treatment of chronic pain as a national priority. Among its recommendations, the agenda calls for large-scale, real-world observational studies of therapies as used in practice to provide valuable information regarding long-term outcomes.
PCORI has two initiatives to support studies just like this:
Our Pragmatic Clinical Studies Initiative is our path to fund pragmatic clinical trials, large simple trials, and large-scale observational studies comparing two or more options for addressing major health problems. Calls for applications are made multiple times per year, and applicants may request up to $10 million in total direct costs for a project period not to exceed five years.
PCORnet, the National Patient-Centered Clinical Research Network, is our large, highly representative, national network for conducting observational and experimental CER. PCORnet is comprised of 29 health data networks that will use real-time data drawn from real-world settings to enable studies of clinical questions that can’t be addressed by traditional randomized controlled clinical trials.
Improving care for chronic pain is a priority for us at PCORI. We look forward to working with the community of patients, clinicians, and other healthcare stakeholders interested in using patient-centered CER toward that goal. Together, we can generate useful and trustworthy evidence that is relevant for patients, caregivers, and clinicians and the real-world clinical challenges they face every day.
Joe Selby MD is Director of the Patient Centered Outcomes Research Institute (PCORI).