(This article is based on remarks made in Paris at the June 16th conference, “Transforming Health Systems with Patient Outcomes, held at the Conseilé Economique Social et Environnemental under the high patronage of President Macron and the attendance of Dr. François Braun, French Minister of Health and Prevention.)
What is value-based health care? Why is it so important? And why is it so difficult to measure?
In a world of increasingly complex innovation and finite financial resources, these three questions are crucial to advancing 21st-century health care technology assessment. Such inquiries are devilishly contentious and complicated. In order to answer them, academics, government officials, practitioners and patients must agree on, among other things, a pallet of harmonized terminology: a lexicon of value.
We stand at a value-based inflection point. Public and private health systems and their constituent and over-lapping stakeholders are being held increasingly accountable for the value their decisions, products and services provide to individual patients and society at large, yet the emergence of value-based health care is consistently hindered by a lack of transparent and standardized outcome data.
The general consensus is that health care ought to be driven by a relentless focus on delivering outcomes that truly matter to patients. How can problems be addressed, and questions answered so that opportunities can be realized?
Value in health care is not a vague or theoretical concept. It is defined by a simple equation: outcomes/costs. Patient outcomes designate the end results of health care expressed in terms of quality of life and functional scores based on validated methodologies.
Over the years, health economists have developed sophisticated tools and techniques to measure the denominator — costs. However, the numerator — patient outcomes — remains ill-defined and unevenly measured even though it reflects the true meaning of “success.”
According to the American educator, Laurence Peter, “Bureaucracy defends the status quo long past the time when the quo has lost its status.” PROMs (Patient Reported Outcomes Measurements), aggressively, creatively, regularly, and comparatively used, are status quo busters.
But, if you can’t measure it, it doesn’t count. Benchmarking PROMs will advance healthcare by leapfrogging over the healthcare status quo.
Nowhere is the status quo more passe than when it comes to drug development and regulation and nowhere is that more evident than in clinical trial design, evidence generation, and data review methodologies.
To broadly generalize, to many dedicated long-time regulators in the USFDA, the EMA, the MHRA, ANSM, and other developed licensing agencies, if it’s not data from a double-blind, randomized clinical trial, it’s garbage. But that’s changing and it’s changing fast. From a regulatory perspective, it is PROMs’ time.
Rather than fighting change or insisting on yet another one-size-fits-all rigid framework du jour, PROMs force us to embrace differences. To learn from them. To accept collective, comparative learning -- because it’s these data-driven combinations and permutations that make us stronger and smarter – the same way Roger Federer made Rafael Nadal stronger, and Novak Djokovic smarter. PROMs are a wonderful virus that infects us and makes us more adaptable, more able to survive and thrive, providing healthcare policy herd immunity against not just misinformation and disinformation – but lack of information.
At the USFDA new treatments for Alzheimer’s Disease, Amyotrophic lateral sclerosis (ALS), and Duchenne’s Muscular Dystrophy are being developed, reviewed, and approved based on validated patient-reported outcomes data and measured in the post-marketing environment by real world evidence. A first-ever therapy for Friedreich’s ataxia was approved through a combination of a small clinical trial and 20 years’ worth of natural history studies. That’s not the promise of PROMs – it’s the reality of PROMs.
The goal of value-based health care is to facilitate making “outcomes” the defining variable in the multifaceted decision-making process, superseding both cost and “quality.” In that respect, VBHC becomes “21st-century tendering” for both payers and patients. VBHC plus quality becomes “3D quality.” It advances the concept of quality from a “soft” to a “hard” measurement tool.
PROMs have value because they define value in new and exciting, and threatening, and outcomes-based ways. PROMs force us to compare and judge. And that is its brilliance. But to be able to judge, we must be able to validate. And to validate we must benchmark best practices.
The future is becoming increasingly clear. Value-based health care turns concepts such as “value and quality” into “hard data.” It is time to adopt the same language to measure success in health care with indicators that truly matter to patients. Value-based health care isn’t about harmonization of decision-making; it’s about harmonization of design and process. This is crucial as other HTA “harmonization” efforts are heralding themselves as a quasi-nice “one-size-fits-all” approach. “Value” should be a constant, and political entities should make decisions based on constants — but decisions can be different based on different national needs, priorities, and biases.
Peter J. Pitts, a former USFDA Associate Commissioner, is a Visiting Professor at the University of Paris School of Medicine and President of the Center for Medicine in the Public Interest.
