The Patient's Perspective on Why Methods Matter

The U.S. spends nearly $150 billion on health research every year. Despite this investment, patients do not have, in many cases, the information they need to make significant, sometimes life-altering choices.

Even with the help of physicians the options can be daunting, and patients frequently struggle to understand how to choose the right treatment for them.

For cancer patients, it is critical to know how effectively and to what degree a treatment can stop, shrink or eliminate a tumor, but it can be equally important to know a treatment’s impact on their daily life. People want to know which treatment is right for a person their age with their background and makeup, balancing aspects of quality of life.

Recently, I was faced with a difficult personal medical decision regarding a major surgery. I received four different opinions from four different physicians. Why the conflicting opinions? Because too often the answers to our challenging health questions are that “we just do not know.”

Research in most cases has not provided adequate answers to our questions as patients. We and our clinicians are left to decide with incomplete information. And even with more information, if research does not study populations with our background and makeup, then the result isn’t likely to be relevant to us.

The Patient-Centered Outcomes Research Institute is working to improve decision-making by changing the way research is conducted. By designing a new model for research, we can ensure that the patient’s voice and perspective is reflected throughout the research process. The end result will be research results that are relevant, reliable, trusted and understood by patients and those who care for them.

One tool that can help in promoting this new approach is the draft report issued by PCORI’s Methodology Committee, which identifies 60 standards for patient-centered outcomes research. An excerpt from the report gets at the essence of good patient-centered research: “Every step of the design, conduct, analysis, and dissemination of PCOR should be directed towards informing health decisions that affect outcomes that are meaningful to a specific group of patients.”

The standards include patient-centered principles we can all embrace. They say research should:

• Be clear about the patients and decisions affected by the research.
• Measure the outcomes that patients care about.
• Include patients and their representatives at all stages of the research.
• Value patient-reported outcomes – what the patients are saying about their condition and experience – as the best source of information.

The report also calls for dissemination planning to be an integral part of research. Even the best research information will not help patients and clinicians if it is not widely available or not easily understood.

One needn’t be an expert in methodology to appreciate that, if we’re to be sure that research addresses patient and caregiver concerns and gives them answers to questions most important to them, the methods matter.

That’s why I hope patients and caregivers might take a moment to provide input and feedback on PCORI’s first draft Methodology Report. Your input and feedback are critical if we’re to be sure that our work is truly patient-centered. You can learn more about the Methodology Report, provide comments, and find other ways to engage with PCORI on our website.