http://stm.sciencemag.org/content/7/291/291fs25.full
Abstract
The
premier position of medical research on the U.S. national policy agenda
offers an unprecedented opportunity to advance the science of patient
input and marks a turning point in the evolution of patient engagement.
For
most of history, patients have been the passive recipients of medical
care with little or no role in research. Even as research subjects,
patients were not required to give informed consent prior to adoption of
the Nuremburg Code in 1947. Since then, patient participation has
expanded dramatically, and today, opportunities abound to serve as
active partners in defining and prioritizing research questions and
solutions. As digital strategist Leonard Kish declared in 2012, “If
patient engagement were a drug, it would be the blockbuster drug of the
century and malpractice not to use it” (1).
Patient
engagement offers the promise of advancing more personal and
efficacious medical products faster than the typical ~15-year
discovery-to-market timeline (2).
Here, we explore the early foundations of patient engagement (table
S1), where it occurs in the drug-development pipeline, the power of
recent policy initiatives, and prospects for success in improving health
outcomes.
FROM SIDELINES TO CENTER COURT
Early
in the last century, patients began to mobilize to accelerate research
for particular conditions. The March of Dimes, founded by President
Franklin D. Roosevelt in 1938 to expand polio research, is one of the
first examples of philanthropy directed at finding treatments and cures.
Research supported by individuals through the March of Dimes led to
development of the “iron lung” and a successful vaccine. Until recently,
this case was an outlier, considering that until the 1973 Patient Bill
of Rights was adopted by the American Hospital Association, patients did
not necessarily expect to be told their diagnosis, much less have a
voice in determining their care plan. Even in recent years, patients
didn’t always express their own preferences and expectations for care,
deferring to choices the doctor deemed best.
The HIV/AIDS
movement catapulted patient needs to the forefront of research and
created the force for change that dramatically altered regulatory
approval processes at the U.S. Food and Drug Administration (FDA),
funding formulas and emphasis at the U.S. National Institutes of Health
(NIH), and the path forward for disease organizations. People affected
by HIV rallied together and created a movement that demanded change and
got results (3):
from the creation of Gay Men’s Health Crisis in New York in 1982 and
the AIDS Coalition to Unleash Power in 1987, to the National Institute
of Allergy and Infectious Diseases’ (NIAID’s) formation of the largest
HIV clinical trials network in the world, to protests at both NIH and
FDA, to passage of the Ryan White Comprehensive AIDS Resources Emergency
Act in 1990.
The HIV/AIDS model continues to provide a
roadmap followed by other patient communities, demonstrating that it is
not enough to question the status quo; you have to do the hard work of
presenting well-founded alternatives. As Anthony Fauci, director of
NIAID, noted at a FasterCures event in 2011, “If you really
want to shake cages you have to be persistent. This is very different
than coming to a meeting once a year. We knew the HIV/AIDS activists
weren’t going away.”
Today, the role of patients as
partners permeates the R&D landscape, extending far beyond the
traditional model of funding basic science through donations. Spurred on
by the increase of entrepreneurial philanthropy and the proliferation
of technology that connects and empowers patient communities, patient
influence on decision-making is increasing. In particular, the venture
philanthropy drug-development model pioneered by the Cystic Fibrosis
Foundation—which led to the codevelopment, with Vertex Pharmaceuticals,
of Kalydeco, the first disease-modifying treatment aimed at the genetic
cause of cystic fibrosis—is gaining steam and altering the landscape of
disease research and cross-sector collaboration.
The U.S.
federal government recently initiated a series of efforts to more
formally incorporate patient input into its decision-making processes.
Efforts and entities have jumpstarted activities across the medical
products industry to elicit and include patient perspectives along the
full range of clinical development, such as the Patient-Centered
Outcomes Research Institute (PCORI), established through the Affordable
Care Act in 2010; the Patient-Focused Drug Development initiative at the
FDA, mandated under the fifth reauthorization of the Prescription Drug
User Fee Act (PDUFA) in 2012; and a Patient Preference Initiative
launched by the FDA’s Center for Devices and Radiologic Health (CDRH) in
2013.
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