ISPOR: The International Society for Pharmacoeconomics and Outcomes Research

A computer simulated cost-effectiveness model of fremanezumab for migraine found that it was cost effective versus no treatment. Researchers presented a poster about its cost-effectiveness for the prevention of chronic and episodic migraine at ISPOR 2019.

The upfront prices of potentially curative therapies are terrifying to commercial payers and government payers alike. A panel on the last day of ISPOR 2019 discussed these issues in a session called, “Is Affordability Driving a Need to Revolutionize Drug Pricing?”

Eleanor M. Perfetto, PhD, MS, senior vice president of Strategic Initiatives for the National Health Council, discusses criticisms of the quality-adjusted life year (QALY) and what factors the measure does not take into account.

Nicholas Robert, MD, medical director of Data, Evidence & Insights, McKesson Life Sciences, discusses the benefits of real-world evidence (RWE) for measuring end points, as well as the challenges posed by variability in clinical practice.

During ISPOR 2019 annual meeting, a session featured biosimilar experts who reviewed the current state of affairs and various challenges related to uptake, while also sounding some notes of optimism.

Devin Incerti, PhD, lead economist, Innovation and Value Initiative, discusses lessons learned and best practices that have emerged as a result of different approaches to value assessment.

At ISPOR 2019, the concept of real option value—paying for treatment that might keep a patient alive long enough to try a future improved therapy—was discussed.

Representatives from different parts of the healthcare system, as well as an outside observer, weighed in on whether the reports issued by the Institute for Clinical and Economic Review (ICER) are taking away patient choice.

It is incredibly important for these assessments to consider the context of the therapy, rather than just the clinical benefit that it brings, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.

Panelists at a session of the ISPOR 2019 annual meeting discussed the good and the bad about the quality-adjusted life-year (QALY) metric from their various perspectives.

Jonathan Campbell, PhD, associate professor, Department of Clinical Pharmacy, Pharmaceutical Outcomes Research, Skaggs School of Pharmacy and Pharmaceutical Sciences at the University of Colorado, discusses the need for more creative methods for value assessment, as well as how one-time, high-cost therapies make it harder to determine value.

Daniel Mullins, PhD, discusses what trends he expects to see in coming years regarding value in healthcare.

Medical futurist Daniel Kraft, MD, implored his audience of healthcare stakeholders attending the ISPOR 2019 annual conference to consider the different ways that technology disruption can improve care away from what he called “sick care,” while moderator Clifford Goodman, PhD, of The Lewin Group, moderated a panel that followed about the potential and pitfalls of disruption in healthcare.

The free flow of data needs to be an intrinsic part of the way we think about healthcare in this country, said Harlan Krumholz, MD, SM, the Harold H. Hines Jr professor, Medicine and Epidemiology and Public Health, Yale School of Medicine, and director, Center for Outcomes Research and Evaluation, Yale-New Haven Hospital.

If we’re truly serious about being outcomes oriented in healthcare, we can’t come near accomplishing what we’d like to accomplish without direct intervention with regard to social determinants, explained Clifford Goodman, PhD, senior vice president and director, Center for Comparative Effectiveness Research, The Lewin Group.

The most common question patients ask when they start a new treatment after a new diagnosis is "what will this do to me?" explained Sally Okun, RN, MMHS, vice president, Policy and Ethics, PatientsLikeMe.

Different value-based payment approaches are evolving for different sorts of conditions, different sorts of financial circumstances, and other factors, explained Clifford Goodman, PhD, senior vice president and director, Center for Comparative Effectiveness Research, The Lewin Group.

The purpose of Project Baseline is to map human health so we can convert from a reactive healthcare system to a proactive healthcare system, explained Adrian F. Hernandez, MD, MHS, vice dean, clinical research, Duke University School of Medicine

We need to collaborate and test these innovations in controlled circumstances so that we can evaluate their effect and understand what needs to be iterated to continue to improve them, explained Harlan Krumholz, MD, SM, the Harold H. Hines Jr professor, Medicine and Epidemiology and Public Health, Yale School of Medicine, and director, Center for Outcomes Research and Evaluation, Yale-New Haven Hospital.

Not all health data is collected in a similar manner, so analyzing such data can be challenging for researchers, explained Ejim E. Mark, MD, MPH, MBA, CEO and founder of Access Healthcare Foundation.

Sally Okun, RN, MMHS, vice president, Policy and Ethics, PatientsLikeMe, discusses findings from PatientsLikeMe's research on what patients define as good care.

Adrian F. Hernandez, MD, MHS, vice dean, clinical research, Duke University School of Medicine discusses how technology can be used to improve the way we gather and use real-world evidence, the promise that real-world evidence holds, and how he thinks real-world evidence will be used in the coming years.

If we’ve got data moving, and if we’re analyzing it in smart ways in real time, we should be able to detect problems within our healthcare system much more rapidly than we have been able to in the past, explained Harlan Krumholz, MD, SM, the Harold H. Hines Jr professor, Medicine and Epidemiology and Public Health, Yale School of Medicine, and director, Center for Outcomes Research and Evaluation, Yale-New Haven Hospital.

Sally Okun, RN, MMHS, vice president, Policy and Ethics, PatientsLikeMe, discusses how digital health improvements help include the patient voice in healthcare and how the United States' use of real-world evidence compares with how it's used in other countries.

As new treatments come to market that have a substantial impact on diseases, or even cure them, the healthcare system is facing the challenge of how to value these treatments. A panel of experts highlighted what evidence there needs to be, methods of valuing therapies, and the ethical implications of having cures.

On the closing day of the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, stakeholders gathered to grapple with the role of value assessments in a healthcare landscape that is increasingly focused on the use of precision medicine in treating disease.

Digital health can improve care around the world, but if used poorly could exacerbate existing disparities, said Ejim E. Mark, MD, MPH, MBA, CEO and founder of Access Healthcare Foundation.

In order to start evaluating the economics of new cures, the scope of the evaluation needs to be as broad as possible and be able to wrestle with uncertainty, said Steven Pearson, MD, MSc, founder and president of the Institute for Clinical and Economic Review.

Advances in medicine have produced breakthroughs in the treatment of a number of rare diseases, but these advances often come at a high cost. A multi-stakeholder panel at the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, addressed the question of how to define value in the always evolving and ever more expensive treatment landscape.

In some ways, the United States is progressive in its use of real-world evidence, but there are still areas where other countries do a better job of incorporating such evidence into the health system, explained Adrian F. Hernandez, MD, MHS, vice dean, clinical research, Duke University School of Medicine.