Challenge Awards

The PhRMA Foundation awarded $25,000 Challenge Awards to the authors of three manuscripts that explore crucial challenges facing the Centers for Medicare & Medicaid Services (CMS) in implementing Medicare drug price negotiation.

The Inflation Reduction Act authorizes Medicare to negotiate the prices of top-selling drugs with manufacturers. Although CMS released revised guidance in June on how it would implement this program, many questions remain around the execution of this long and complex process. The PhRMA Foundation partnered with the Journal of Managed Care & Specialty Pharmacy (JMCP) on a call for papers investigating unanswered questions about the process.

The three winning manuscripts are:

• Medicare Drug Price Negotiation: The Complexities of Selecting Therapeutic Alternatives for Estimating Comparative Effectiveness, Inmaculada Hernandez, PharmD, PhD; Emma Cousin, Pharm; Olivier J. Wouters, PhD; Nico Gabriel, MA; Teresa Cameron, MS; Sean D. Sullivan, BScPharm, PhD
• Identifying Therapeutic Alternatives in CMS Drug Negotiation: The Case of Etanercept, Helen Mooney, MPH; Matthew Martin, MA; Liam Bendicksen, BA; Aaron S. Kesselheim, MD, JD, MPH; Benjamin N. Rome, MD, MPH; Hussain S. Lalani, MD, MPH, MSc
• Evidence Inventory: A Patient-Centered Methodological Framework for CMS to Assess the Clinical Benefit of Drugs to Inform Maximum Fair Price Negotiation, Nabin Poudel, PhD; Salome Ricci, PharmD, MS; Julia F. Slejko, PhD

Read the press release to learn more.

Question: How can value assessment methods and processes better account for populations that are typically underrepresented in research and drivers of health disparities?

The challenges of COVID-19 brought the crucial work of our nation’s scientists to the forefront and highlighted the need to address racial inequity in health care. The PhRMA Foundation has established a this Challenge Award focused on finding solutions to racial health disparities. This Challenge Award aimed to inspire bold and vital research on how value assessment can better capture evidence regarding diverse populations and drivers of health disparities.

The winning papers were published in the Journal of Managed Care and Specialty Pharmacy.

First Place: Drivers of Health Disparities and Consequences for COVID-19 Vaccine Choices: Modelling Health Preference Heterogeneity among Underserved Populations Award Recipients

• Eline M. van den Broek-Altenburg, PhD, Larner College of Medicine at the University of Vermont
• Jamie S. Benson, BA, Larner College of Medicine at the University of Vermont
• Adam J. Atherly, PhD, Larner College of Medicine at the University of Vermont
• Stephane Hess, PhD, Choice Modelling Centre & Institute for Transport Studies, University of Leeds

New approaches to capture drivers of health disparities and account for underrepresented groups in value assessment methods are warranted. Dr. van den Broek-Altenburg and co-authors conducted an analysis of COVID-19 vaccine preferences among underrepresented populations and explored methodological approaches to incorporate differences in unobserved individual preferences in value assessment. The authors propose the use of discrete distributions and probabilistically segmenting a sample population into different segments, better reflecting the underlying values of individuals in underrepresented populations. The research concludes that health care interventions intended to reduce health disparities that do not reflect the underlying values of individuals in underrepresented populations are unlikely to be successful.

Second Place: Using Latent Class and Quantum Models to Value Equity in Healthcare: A Tale of Two Stories

• Surachat Ngorsuraches, PhD , Auburn University

To adequately capture the value of health equity in value assessment, systematic consideration of health equity in decision making is critical. In this research, Dr. Ngorsuraches describes two approaches to empirically address health equity in value assessment by valuing health care attributes with an equity lens. The first approach uses a discrete choice experiment to elicit preferences from individuals on preferences value attributes with a latent class model to derive the value of equity, and the second approach uses a flexible choice model to value health equity. These methodological approaches can be used by value assessors to determine how equity enters the preferences for decision makers.

Third Place (Tie): It’s Time to Represent: Shifting the paradigm to improve the quality of inputs into value assessment frameworks

• Leticia R. Moczygemba, PharmD, PhD, TxCORE, The University of Texas at Austin
• Carolyn M. Brown, PhD, TxCORE, The University of Texas at Austin
• Michael Johnsrud, PhD, RPh, TxCORE, The University of Texas at Austin

To better understand the importance of equity in value frameworks, advancing the racial/ethnicity representation inputs in value assessment is critical. In this work, Dr. Moczygemba and colleagues propose a two-pronged strategy to increase the diversity of populations that participate in research and address drivers of health disparities to better inform value assessment with the following objectives: 1) Launch a comprehensive national campaign using a community-engaged approach to inform, create buy-in, and generate excitement for participation in research, and 2) Enhance information used in value assessment frameworks by expediting current methodological initiatives to require a minimum set of patient-reported social determinants of health elements to be collected and reported in research, including clinical trials and observational (real world) studies. Through this work, scalable efforts to increase representation of diverse racial/ethnic groups and social determinants of health in value assessment can be attained.

Third Place (Tie): Incorporating Health Equity into Value Assessment: Frameworks, Promising Alternatives, and Future directions

• Vakaramoko Diaby, PhD, University of Florida
• Askal Ali, PhD, Florida A&M University
• Aram Babcock, PharmD, MS, MBA, University of Florida
• Joseph Fuhr, PhD, University of Florida
• Dejana Braithwaite, PhD, University of Florida

To better understand the value of health equity in value assessment frameworks, exploring methods that value diverse perspectives is critical. In this study, Dr. Diaby and colleagues examine emerging value assessment frameworks in the United States and present examples, where evidence on outcomes and preferences for value do not take into consideration diverse perspectives. The authors identify possible solutions to improve existing value assessment methods and illustrate – using a hypothetical shared decision-making case study – an alternative to current value-assessment frameworks, “equitable multi-criteria decision analysis”, that could be implemented in the context of the value-based assessment of prevention choices for women at high risk of developing breast cancer. These proposed alternatives and solutions can be used by researchers and decision makers to incorporate health equity into value assessment.

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?

The winning papers were published in ISPOR’s Value & Outcomes Spotlight.

First Place: Generalized Risk Adjusted Cost Effectiveness (GRACE): Ensuring Patient-Centered Outcomes in Healthcare Decision Making

• Charles E. Phelps, PhD, University of Rochester
• Darius N. Lakdawalla, PhD, University of Southern California School of Pharmacy and Schaeffer Center for Health Policy & Economics

Drs. Phelps and Lakdawalla propose using a novel approach – Generalized Risk-Adjusted Cost-Effectiveness (GRACE) – that aligns the economics of cost-effectiveness analysis with the human circumstances of patients and consider, in particular, how in standard models a given gain in life expectancy is worth less to sicker or more disabled people, because of the approach to weighting. The GRACE model reveals how traditional methods fail to account for disease severity, patient risk-aversion, and other issues and assures that patient preferences, particularly the quintessential measure of untreated health status, reliably and consistently enter value measures used at both population and individual decision-making.

Second Place: Nudging Health Economists: A Process for Systematic Identification of Patient-Centered Outcomes for Inclusion in Value Assessment

• Joseph Mattingly II, PharmD, MBA, PhD, The University of Maryland School of Pharmacy
• Julia F. Slejko, PhD, The University of Maryland School of Pharmacy
• Elisabeth M. Oehrlein, PhD, MS, The National Health Council
• Eleanor M. Perfetto, PhD, MS, The National Health Council, The University of Maryland School of Pharmacy

The authors aim to provide practical guidance on a process for identifying and presenting patient-centered outcomes in a way that makes it easier to include them in value assessment, thereby “nudging” more economists to choose to include these outcomes in their models and not dismiss them simply as limitations. The process incudes forming a multi-stakeholder, patient-centered advisory board, engaging the board in the research agenda, conducting evidence synthesis and qualitative research to ensure viewpoints are not missed, and disseminating findings to multi-stakeholder audiences. A publicly available, centralized database of identified patient-centered value elements should be created to increase the likelihood of their uptake in value assessment.

Third Place (Tie): Evolution of Precision Medicine: Applying a Population-based Evidence Assessment Repository (PEAR) to Achieve Patient-Centered Outcomes at the Point-of-Care

• Jonathan H. Watanabe, PharmD, PhD, University of California Irvine School of Pharmacy & Pharmaceutical Sciences
• Derjung M. Tarn, MD, PhD, University of California Los Angeles School of Medicine
• Jan D. Hirsch, PhD, University of California Irvine School of Pharmacy & Pharmaceutical Sciences

The authors propose a step-wise framework that shapes goals based on patient values and shared decision-making that is continuously refined by utilizing a population-based evidence assessment repository (PEAR) to achieve person-centered care. The PEAR aims to empower the care team to ensure measurable data-driven treatment decisions that are tailored towards patient needs, characteristics, and wishes.

Third Place (Tie): Coproduction in Learning Healthcare Systems is the Key to Unlocking True Healthcare Value

• Brandy Fureman, PhD, Epilepsy Foundation, Epilepsy Learning Healthcare System

The author shares her experience using co-production – a process where patients, care partners, and providers work together to design a health system that optimizes the health outcomes that matter most to patients. For example, evaluations of value in epilepsy must look beyond standard clinical endpoints of seizure control and address other outcomes that are important to epilepsy patients such as motor skills, communication, learning, attention and emotional wellbeing.

Question: What are innovative, patient-centered approaches to contribute to healthcare value assessment that move beyond the inherent limitations of analyses based on the quality-adjusted life year metric?

The winning papers were published in the American Journal of Managed Care.

First Place: The Opportunities and Challenges of Rethinking Our Approach to Value Assessment in Healthcare

• Lori Frank, PhD, RAND Corporation
• Thomas W. Concannon, PhD, RAND Corporation

Drs. Frank and Concannon propose the Strategy for Patient-Engaged Healthcare Valuation to address the challenges of incorporating patient preferences into value assessment by employing multi-criteria decision analysis methods using patient-informed criteria derived through Goal Attainment Scaling (GAS).

Second Place: Expanding Use of Multi-Criteria Decision Analysis for Health Technology Assessment

• Charles E. Phelps, PhD, University of Rochester

Dr. Phelps’ paper discusses the advantages of using MCDA in the evaluation of healthcare technologies, barriers to expanded use, and the specific steps needed to increase ease of use. MCDA facilitates a relative ranking of treatment candidates by identification of treatment attributes, their relative importance, and performance along the attributes. His paper notes that though cost effectiveness analysis (CEA) is the de facto method of choice for assessing the value of these technologies, MCDA models present a promising opportunity to incorporate multiple dimensions of value into these assessments.

Third Place (Tie): Using Patient Experience Data and Discrete Choice Experiment to Assess Values of Drugs

• Surachat Ngorsuraches, PhD, Auburn University

Dr. Ngorsuraches proposes a Patient Experience Value Method that facilitates the incorporation of patient-derived attributes of value based on FDA protocol and discrete choice experiments and addresses heterogeneity of patient preferences by capturing and reporting the full distributions of patient values.

Third Place (Tie): Evaluation of Medical Technologies with Uncertain Benefits

• Darius N. Lakdawalla, PhD, University of Southern California
• Charles E. Phelps, PhD, University of Rochester

Drs. Lakdawalla and Phelps propose a novel method of incorporating risk and uncertainty into assessments of healthcare technologies, including specifically measuring the value of reducing uncertainty (“the value of insurance”) and the value of increasing the likelihood of positive outcomes (“the value of hope”).

Question: What are potentially transformative strategies and methods to define and measure value at all levels of decision making that are aligned with personalized/precision medicine?

The winning papers were published in the Journal of Managed Care & Specialty Pharmacy.

First Place: A Strategy to Support the Efficient Development and Use of innovations in Personalized and Precision Medicine

• Louis Garrison, PhD, University of Washington
• Adrian Towse, MPhil, MA, Office of Health Economics, London

In their research proposal, Dr. Garrison and Mr. Towse call for a broadening of the concepts of value in personalized/precision medicine, laying out six basic policy principles as pathways to help determine value. These range from the need for flexible, value-based pricing to real-world evidence generation in personalized/precision medicine and the challenging implications for assessing and rewarding value.

Second Place: Genome Diagnostics: Novel strategies for Measuring Value

• Robin Z. Hayeems, ScM, PhD, Hospital for Sick Children, Toronto

In their research proposal, Dr. Hayeems and colleagues discuss the substantial medical and economic benefits of genome wide sequencing (GWS) as a means to enhance personalized medicine across a broad range of therapeutic areas, noting that assessing the full value of these technologies requires a set of metrics that extend beyond laboratory-based performance parameters. The authors summarize their progress in developing a methodology for measuring the clinical and personal value of genome diagnostics, building on preliminary findings from the Hospital for Sick Children’s Genome Clinic, a translational genomics research platform that routinely generates genomic data on children with a range of clinical phenotypes.

Third Place: Patient-Level Modeling Approach Using Discrete-Event Simulation – A Cost-Effectiveness Study of Current Treatment Guidelines for Women with Postmenopausal Osteoporosis

• Quang A. Le, PharmD, PhD, Western University of Health Sciences, College of Pharmacy

In his research proposal, Dr. Le discussed discrete-event simulation (DES), an event-driven, continuous time, patient-level modeling method for health economic evaluations that addresses some limitations of other common modeling techniques. Flexibility, the ability to reflect patient heterogeneity, increased precision, and better characterization of modeling uncertainty are advantages in the DES model. Dr. Le’s proposal aims to describe and demonstrate an application of the DES model to evaluate the cost-effectiveness of the current treatment guidelines for women with postmenopausal osteoporosis.

Question: What are transformative strategies to measure or evaluate value of health care interventions that could be implemented to advance a value-driven health care system in the United States?

The winning papers were published in ISPOR’s Value in Health.

First Place (Tie): A Framework for Measuring Low Value Care

• George Miller, PhD, Altarum Institute

It has been estimated that more than 30 percent of health care spending in the U.S. is wasteful. Low-value care, which drives up costs unnecessarily while increasing patient risk, is a significant component of this wasteful spending. Altarum’s winning paper summarizes and critiques existing methods that measure low-value care and describes an integrated framework that combines multiple methods to comprehensively estimate and track the magnitude and principal sources of clinical waste.

First Place (Tie): Improving Value for Eczema Patients

• Julie Block, National Eczema Association

With the cost of chronic diseases now representing a majority of spending in the United States health care system, a reliable model to measure value is critical to identify replicable improvement methods. The National Eczema Association’s (NEA) plan to establish a Shared Decision-Making Resource Center is a transformative strategy that can evaluate value of health care interventions for eczema patients to advance a value-driven health care system in the U.S.

Second Place: Emerging Good Practices for Transforming Value Assessment: Patients’ Voices, Patients’ Values

• Eleanor M. Perfetto, PhD, MS, National Health Council

Most U.S. value frameworks and subsequent assessments are developed with little to no patient engagement. The National Health Council (NHC) started a value workgroup in 2016 to change this. In the past two years, patient advocacy group engagement with value framework developers and assessors has increased, with groups reporting both positive experiences and challenges in their interactions. The NHC will use these early experiences can help recommend emerging good practices that can eventually get the field to implement standards for patient engagement.

Third Place: Integration of Patient Reported Outcome Measures to Aid in the Assessment of Value Health Care

• Joel Gagnier, ND, Mc, PhD, University of Michigan

Patient reported outcome measures (PROMs) are tools that assess patient perspectives on their health without interpretation by any other parties, and they are key tools in clinical research. The integration of electronically delivered PROMs in clinical practices can dramatically change health outcomes by influencing decision-making. The authors of this paper propose that the implementation of generic and disease/condition specific PROMs across health care systems will advance a value-driven health care system.