Why Methods Matter for Research and Innovation

The Patient-Centered Outcomes Research Institute is working to develop a new model of research that will benefit innovation and provide all healthcare stakeholders with the reliable information they need to make good decisions that reflect patients’ preferences.

The foundation of this work is sound research methodology, and that’s why the first draft of PCORI’s Methodology Report is being met with optimism and enthusiasm from industry representatives, and the entire healthcare community. The report is a critical step in improving the nature and quality of health research.

As patients, caregivers and healthcare professionals, we all want to know which prevention strategies, diagnostics and therapies will work best for individuals or specific groups of patients in real-world settings. Our healthcare questions are personal: How will a treatment affect someone like me? What will be the impact on my quality of life?

Unfortunately, the ‘gold standard’ of research, the randomized controlled trial (RCT), is not well suited to address questions at this level. RCTs are the main source of evidence by which our medical treatments get approved for use by the FDA. They’re great at telling us if a therapy is safe and effective in a strictly defined, well-controlled setting. But the reality is that none of us live in such sterile environments, so there’s a limit to how well the results of RCTs can inform our healthcare decisions.

A lot of the information most important in decision making isn’t discovered in the pre-approval RCTs designed to test for safety and efficacy in a controlled setting. In addition, RCTs are incredibly expensive and increasingly cost prohibitive in an uncertain environment, where we have to collect evidence and demonstrate value for all stakeholders, from patients and clinicians to payers and regulators.

We need a new model for research and innovation that reduces uncertainty, embraces new types of evidence generated by sound methods, and provides information that is broadly accepted and utilized.

This is the mission of PCORI. PCORI was created to help people make informed healthcare decisions and improve healthcare delivery and outcomes by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers and the broader healthcare community.

PCORI’s work and patient-centered outcomes research (PCOR) can benefit research and innovation in two key ways:

• Developing consensus standards for research. We need standards for patient-centered outcomes research and comparative effectiveness research that are validated and broadly accepted by researchers, payers, regulators and the patients, caregivers and clinicians, who will rely on their results to make health decisions. The development of consensus standards will reduce the uncertainty researchers face. We need to know what data is acceptable. Agreed upon standards will allow us to produce evidence with greater certainty, knowing that the results of our studies will be reliable, verifiable and trusted.
• Identifying gaps in patients’ needs. A better understanding of what works and doesn’t work in real-world settings, focused on patient and caregiver needs, questions and concerns, will give the makers of pharmaceuticals and medical devices valuable information about the gaps in effective treatments for specific conditions. Innovators will be able to address unmet needs and create new treatment approaches to help patients whose options currently are limited or don’t address their particular needs or concerns.

PCORI’s Methodology Report is a critical step in creating a new innovation model. It outlines draft standards for selecting study designs, appropriate data sources and collection methods, and identifying which populations to study. It importantly includes standards for studies of diagnostic tests, addressing a major need in research methodology.

The standards address how to measure outcomes that demonstrate value and have relevance to patients, clinicians, regulators, policymakers and payers. They also include methods for creating the data networks needed to facilitate real-world outcomes research and the validation and use of electronic medical records.

We now seek your feedback on the standards. We need the input of all healthcare stakeholders to refine these draft standards and develop consensus standards that are widely accepted and enable all researchers to move forward and produce with greater certainty the evidence we all want and need to support better decision making and lead to enhanced healthcare quality.

With better information that is tailored to patient preferences, and better clinical options that address unmet needs, the medical community can achieve its fundamental goal of improved outcomes. This is the promise and potential of PCORI’s draft Methodology Committee report.

You can read the draft report, provide comments and become engaged in our efforts to transform research here on our website.