From Aspiration to Application: 5 Years of Patient-Centricity

2012 WAS A LANDMARK YEAR FOR GREATER INVOLVEMENT of patients in research, development, and delivery of medical products and care. The U.S. Food and Drug Administration (FDA) Safety and Innovation Act was signed into law on July 9, 2012, with a requirement to foster patient-focused drug development through a series of 20 meetings. The Patient-Centered Outcomes Research Institute (PCORI), created two years earlier under the Affordable Care Act, dedicated 2012 to engaging hundreds of patients and caregivers in setting a national research agenda. And in 2012 digital strategist Leonard Kish declared, “If patient engagement were a drug, it would be the blockbuster drug of the century and malpractice not to use it,” a line seized by patient advocates and media alike to mark a new dawn in the patient-centricity movement.

Five years later, this handful of organized activities to foster patient engagement has precipitated a veritable “gold rush” of endeavors to construct a science of patient input. FasterCures has mapped nearly 100 multi-stakeholder initiatives producing frameworks, methods, toolkits, draft regulatory guidances, training programs, and case studies. These resources are evolving from broad outlines of the “possible,” to more detailed descriptions of what’s practical as experience is gained in a variety of clinical areas and contexts. 

Gold rush quote

The way in which patients are engaged has evolved, too. Early on, patients most often were invited to pinpoint problems with clinical trial recruitment or retention. Now, a wider array of activities are informed by patient perspectives, including setting priorities for early-stage research, selecting outcome measures, making benefit-risk and value assessments, and developing care guidelines. Patient organizations urge even earlier and more frequent opportunities to shape research plans, development programs, regulatory approaches, and care delivery practices.

As real-world evidence rises in value, patient perspective data collected from patient registries, smartphone apps, wearable devices, online communities, and social media provide new windows into the patient experience. Patient preference studies and patient journey maps help illustrate a more complete picture of the impact of disease and burden of therapies. This evidence can be used to align unmet medical need with targets, as well as identify barriers to participation in research and access to care.

While progress is swift by some measures, it has not been even. Leadership from FDA and PCORI has defined new expectations for parts of the system they affect, but much more work is needed to integrate patient perspectives at earlier stages of research and into decisions by payer organizations and health-care systems. Patient organizations representing certain rare diseases and cancers have pioneered much of the emerging practice at the grassroots level, with challenges still ahead to better engage individuals with highly prevalent conditions and those who have been underrepresented in traditional research and care settings.

Patients are the greatest force propelling the change seen over the past five years; incentives created through federal policy measures have accelerated adoption and shifted culture toward more patient-centered decision-making. As the vignettes presented in the next three pages illustrate, we are seeing early benefits as this practice takes hold. Over the next five years we hope to see the leap from application to actualization of the promise patient-centricity holds.

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