Reports

Expanding the Science of Patient Input: Building Smarter Patient Registries

Published: February 2016

Across the biomedical research and care enterprise is evidence of an accelerating shift toward patient-centeredness. The creation of the Patient-Centered Outcomes Research Institute (PCORI) by Congress in 2010, the Food and Drug Administration’s (FDA’s) Patient-Focused Drug Development initiative begun in 2012 and a strong commitment from the White House and the National Institutes of Health (NIH) to make patients full partners in the Precision Medicine Initiative that was announced in 2015 are just a few of the sign posts indicating that patient needs and expectations are shaping the national research agenda.

Decision-makers in research, industry, policy and health-care settings are actively seeking robust sources of patient data to inform patient-centered practices, policies and outputs. Scientific rigor throughout this process is of paramount importance to ensure solid outcomes. FasterCures is at the forefront of the burgeoning science of patient input, aimed at establishing rigorous methods and reliable practices for understanding and incorporating patient needs into the process of developing, regulating and delivering new therapies. One of the existing tools for leveraging patient input is the patient registry, “An organized system of collecting uniform data for a population defined by a particular disease or condition that serves one or more predetermined scientific, clinical or policy purposes.”

FasterCures examined the landscape of patient registries created or held in trust by patient-led foundations with the following three objectives:

  1. assess the state of patient-led patient registries as a robust source of patient insights and actionable data to meet emerging opportunities,
  2. evaluate use of patient registries by patient-led organizations as a surrogate measure of readiness for the expanding emphasis on patient-centricity and
  3. identify information and practices that would enhance existing patient registries and could inform the creation of new ones.

This report ties together research from interviews, FasterCures’ survey data, background research and data gathered at events, including a “Disruptors’ Academy” session focused on registries held at our 2015 Partnering for Cures conference. In this three-part report, we present key learnings, topline results from our survey and emerging best practices in the form of important considerations, checklists, resources and case studies. This guide is intended to be a starting point for learning more about patient registries. Through our Patients Count: The Science of Patient Input program, FasterCures will continue updating, enhancing and augmenting the tools in these pages.

 

TABLE OF CONTENTS

INTRODUCTION

PART ONE: Key Learnings

PART TWO: Survey Methodology and Topline Results

PART THREE: Emerging Best Practices for Registries

GETTING STARTED

SUSTAINING AND MAINTAINING A PATIENT REGISTRY

GENERATING MEANINGFUL OUTCOMES

CONCLUSION

APPENDICES