Expanding the Science of Patient Input: Pain Points and Potential

Hell this should have been widely distributed back in 2013. This just proves once again what Amy Farber has to say.
For the past five years Farber has been battling not only her own disease but also the wall of resistance erected by those who believe that a patient can make about as much of a meaningful contribution to the process of scientific discovery as a laboratory rat.

Expanding the Science of Patient Input: Pain Points and Potential

Patient engagement has been called the “blockbuster drug of the 21^st century.” Indeed, studies find that the more engaged and involved patients are with their health and health care, the better the outcomes. There is now growing interest in engaging patients in another aspect of health care: drug and device development. Borrowing methods from the fields of health economics, outcomes research, epidemiology, social sciences and marketing sciences, a new science of patient input has emerged, embracing data as a means for measuring patient-centered outcomes and quantifying patient preferences.
ENGAGING PATIENTS IN RESEARCH ISN’T JUST A GOODWILL GESTURE: IT CAN MAKE RESEARCH BETTER. Patient engagement can influence plans and policies that shape how medical products move from microscope to marketplace, as well as deepen researchers’ understanding of the experience of living with a disease or condition. Together, this can inform research priorities and resource allocation. More importantly, patient engagement can lead to better, safer treatments that target what patients really need and want.
Several factors are driving this paradigm shift from patients as subjects in clinical research to patients as partners in research. They include patients themselves who are challenging the traditionally paternalistic health-care system; regulatory agencies such as the Food and Drug Administration (FDA); government policy initiatives, including the 21st Century Cures Act; the creation of the Patient-Centered Outcomes Research Institute (PCORI); and nonprofits like FasterCures, which are dedicated to integrating patient perspectives in medical product development to speed treatments of high value to patients.
We are at the beginning of this effort, however, with numerous unanswered questions. To explore current and future challenges, FasterCures hosted an all-day workshop on Feb. 17, 2016, as part of its Patient Count: The Science of Patient Input program.

More than 50 representatives from patient advocacy organizations and other nonprofits, biopharmaceutical and medical device companies, academia and government agencies participated. Reflecting the multi-disciplinary nature of the growing science of patient input, participants were invited to ensure representation from several functional areas and backgrounds, including health economics, regulatory science, patient advocacy, benefit-risk assessment, medical affairs, public policy, communications, public affairs, outcomes measurement and alliance development. Patient group representatives brought experience from diverse communities, including rare and prevalent conditions, diseases with multiple therapies and those with no FDA-approved medical products, and highly engaged patient populations and communities that are not well formed yet. Several participants have had professional experience working in one or more sectors, adding further dimension to their viewpoints and the discussion.

Table of Contents

Introduction
Key Takeaways
Signs of Success
Science of Patient Input: The Need for a Common Language
The Continuum of Patient Engagement
Building Alliances with Academic Researchers
The FDA: Where Are We? Where Are We Going?
The Industry Perspective
Measuring ROI on Engagement: An Update from PCORI
Priorities for Future Collaboration
Moving to Action
Participants
Endnotes