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Voices in Value: 2020 Year in Review

March 11, 2021

The PhRMA Foundation continued to set new milestones in value assessment last year with the announcement of the winners of the 2020 Research and Challenge Awards.

Although 2020 ushered in unprecedented challenges that tested the American health care system, it also presented a unique opportunity to chart a new course for a world forever changed by a global pandemic. The vast health disparities prevalent in the United States pushed us to reevaluate and refocus our efforts to shift to a health care system that rewards value for all. The field of value assessment – and the decision-making frameworks that allow one to weigh health care outcomes relative to costs – will continue to play an important role in helping policymakers tackle these challenges.

The Foundation continued to set new milestones last year with the announcement of the winners of the 2020 Research and Challenge Awards. A total of $300,000 in research award funding was awarded to three teams of researchers whose proposals put forward new, innovative strategies for assessing the value of medicines and health care services. This year’s awardees offered ideas such as a new tool to evaluate the quality of published cost-effective analyses, a novel method for measuring economic endpoints in clinical trials of pediatric health care interventions and an evaluation framework to incorporate the benefits and costs of precision diagnostics to patients’ family members into economic evaluations of those treatments.

The Foundation also awarded $85,000 in Challenge Award funding to four teams of promising researchers who submitted a project proposal responding to the question: What approaches are needed to consistently and reliably incorporate patient-centered outcomes in value assessment for both population- and individual-level health care decision making?

Winning proposals from this year include a novel approach to measuring disease severity in economic models, a set of practical guidelines for identifying and including patient centered outcomes value assessment, a framework to empower care teams to ensure treatment decisions are tailored toward patient needs and a demonstration for how the co-production can improve the health care system for people with epilepsy.

In recognition of the need to address racial inequity in health care – which has only been exacerbated by the COVID-19 pandemic – the Foundation has established a new Challenge Award that aims to inspire bold and vital research on how value assessment can better capture evidence regarding diverse populations and drivers of health disparities. Awardees are expected to be announced this spring.

Throughout the year, Voices in Value featured guest contributors who used our blog to discuss how their research is making the field more transparent and inclusive and better representative of the individuals for whom this work is most important – namely, the patients.

In March, Eleanor M. Perfetto and Elisabeth M. Oehrlein of the National Health Council discussed how the value assessment community could do more to incorporate the patient perspective by considering the impact of a disease and treatment on a family caregivers, a patient’s ability to engage socially, remain active in their community and plan for the future. Recognizing that every patient is different, accurate research depends on an approach that ensures the patient voice remains at the forefront of the conversation.

But the patient voice is not the only element where researchers should direct their focus. A holistic assessment of health care interventions – from pharmaceuticals to public health decisions – should include a consideration of the societal consequences of a particular treatment. David D. Kim and Peter Neumann of the Center for Enhanced Value Assessment (CEVA) at Tufts University School of Medicine highlighted some of their recent research that explores this topic and aims to better understand how cost-effectiveness analyses have historically sought to measure this aspect of value.

Though the pandemic has caused significant upheaval in many aspects of American health care, it has also brought on an unanticipated opportunity to readjust today’s misaligned health care system towards one that is more equitable, sustainable and valuable to patients. Beth Beaudin-SeilerA. Mark FendrickGeorge Miller, and Paul Hughes-Cromwick argued that reducing the prevalence of low-value care within the American health care system – which has long been the focus of the Research Consortium for Health Care Value Assessment – is not only necessary to advance our health system towards one that prioritizes value, it is crucial to ensuring it is sufficiently prepared for the next major crisis.

The Value Consortium has been working for the last several years to develop approaches to identifying, measuring and reducing low-value care which, Dr. Beaudin-Seiler argued in a follow up blog, is also good for employers and can help firms that offer employer-sponsored insurance more efficiently manage resources in the face of rising health care costs.

The historic events that took place this year – from the COVID-19 pandemic to a nation-wide reckoning with systemic racism – have also reinforced the urgent need to improve health equity and explore ways to make value assessment more inclusive of diverse populations. As the Foundation’s President Eileen Cannon outlined in a blog post introducing the Foundation’s new health equity challenge award, the value assessment community will have to rethink our conceptions of value, more acutely identify and address everyday drivers of health inequities and increase representation of diverse populations in research.

Such an example of this can already be seen by the work of researchers at the Patient-Driven Values in Healthcare Evaluation Center (PAVE) at the University of Maryland School of Pharmacy. In a guest blog post, Dr. Wendy Camelo Castillo at PAVE highlighted the center’s experience engaging the Hispanic patient community in Baltimore as part of a broader research initiative to study how diversity affects the value placed on treatment decisions and the tradeoffs patients are willing to make in health care decisions.

The blog was not only a platform through which the Foundation’s partners and supported researchers highlighted their work. Throughout 2020, our partners at the Foundation’s Centers of Excellence had articles featured in numerous publications, ranging from peer-reviewed research journals to media outlets read by the payer, provider and policymaker communities. The work of three of the Centers was highlighted in an article by Sachin Kamal-Bahl and Bryan Luce in the American Journal of Managed Care discussing the need for a better understanding of the health outcomes that are most important to patients through the establishment of a national patient-centered outcomes repository. Additionally, Lori Frank and Thomas Concannon, who were the first place winners of the Foundation’s 2019 Challenge Award, published an article in Stat discussing how the research and advocacy communities can overcome barriers to including the patient voice in value assessment models and outline a path forward for stakeholders to follow.

Moving into the new year, the Foundation will continue to support research initiatives that advance the field of value assessment and develop transformative solutions that meet the needs of all stakeholders.

Other featured work of the Centers includes:

The Center for Pharmaceutical Value (pValue)

Results of a new study, titled “Criteria and Scoring Functions Used in the Value Assessment of Rare Disease Therapies: A Systematic Literature Review of Multi-Criteria Decision Analysis Tools and Value Frameworks,” are under peer review and expected to be published by pValue soon.