ISPOR’s Top 10 HEOR Trends

These trends are summarized on their webpage and in a larger report. Here they are with some key excerpts from their report:

Real world Evidence. “As RWE continues to gain significant traction in healthcare, agencies including the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), The National Institute for Health and Care Excellence (NICE), and Canada’s Drug and Health Technology Agency (CADTH) have recently released guidance on different aspects of RWE generation and use. Key topics have been data quality, fit-for-purpose use, registries, and external control arms”Drug Pricing. “Discussion has been driven by policies such as the Inflation Reduction Act (IRA) in the United States, under which the US Medicare program will be entering negotiations with manufacturers over the prices of 10 high-expenditure drugs, as well as the European Union pharmaceutical strategy, which is proposing several ways to improve access and affordability” Artificial Intelligence. “In health economics, these machine learning (ML) algorithms can be employed to look at data in various ways that can help researchers and payers better understand the safety and effectiveness of treatment. ML algorithms could also be used to build physician diagnostic tools, act as a quality check on human work (such as physicians’ assessments on radiology exams), or build apps that can be used to self-manage conditions (such as for low back pain or schizophrenia).”Fostering Innovation. “…some areas of medicine desperately require innovation, but are not getting the necessary attention due to lack of financial incentives for drug development. A prime example of this is the search for new treatments for drug-resistant bacteria, fungi, and other pathogens.”Health Equity. “Application of social determinants of health (SDoH) to HEOR is increasing… The US Department of Health and Human Services’ (HHS) Office of Disease Prevention and Health Promotion looks at SDoH as part of its Healthy People 2030 initiative. HHS sorts SDoH into 5 domains: economic stability, education access and quality, healthcare access and quality, neighborhood and built environment, and social and community context.”Accelerated drug approvals. ” In 2022, 24 of the 37 Center for Drug Evaluation and Research’s novel drug approvals, or 65%, used one or more expedited programs, such as Fast Track, Breakthrough Therapy, Priority Review, or Accelerated Approval… In the European Union, of the 89 medicines approved by the European Medicines Agency in 2022, 5 were under accelerated assessment, 9 received conditional marketing authorizations, and 5 received approvals under extraordinary circumstances.”Value measurement. “There have been several efforts to address these issues with the QALY. The ISPOR Value Flower includes other important aspects of value such as severity of disease, equity, and value of hope, as well as other individual and societal considerations.40 The Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach incorporates risk aversion in people’s preferences, uncertainty in treatment outcomes, and many of the Value Flower elements, as well as some others, in a single economic measure.”Patient Centricity. “…[FDA] has engaged in a series of patient engagement initiatives, including developing a series of 4 methodological patient-focused drug development guidance documents to address, in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making. EUnetHTA, in its “Guidance on Patient & Healthcare Professional Involvement,” recommends that the Joint Clinical Assessment planned to facilitate cross-country collaboration on health technology assessment in Europe includes meaningful participation by patients to reflect their views on coverage and reimbursement decisions.”Precision Medicine. “…in 2022, the United States Food and Drug Administration (FDA) approved 12 personalized medicines, representing approximately 34 percent of all newly approved therapeutic molecular entities. Personalized medicines accounted for at least a quarter of new drug approvals for each of the last 8 years.”Public health. Factors such as contagious disease, social unrest, and economic recessions all can impact health and were mentioned in the ISPOR report.