Which Top HEOR Trends Will Have The Biggest Impact On Clinical Research

A conversation with ISPOR — The Professional Society for Health Economics and Outcomes Research (HEOR) CEO and Executive Director Rob Abbott

Earlier this year, ISPOR released its 2024-2025 Top 10 HEOR Trends report. The report marks the fifth publication of the Society’s biennial report, which conducts horizon scanning around the world to identify top trends in healthcare.

Unsurprisingly, real-world evidence (RWE), AI, accelerated drug approvals, and patient centricity made the list. In this interview, ISPOR CEO Rob Abbott discusses each trend and how it’s affecting the larger healthcare landscape as well as its direct impacts on clinical trials. 

ISPOR recently released its top 10 HEOR Trends report. Who are its target audiences, and how can they best use it in their healthcare roles? What about those working in clinical research?

At ISPOR, our audience has one common goal: to make healthcare more accessible, effective, efficient, and affordable. This includes healthcare decision makers across a broad spectrum: payers, governments, providers, doctors/pharmacists and hospitals, regulatory agencies, patients, researchers, and many more.

The biennial report captures healthcare trends by conducting horizon scanning around the world and hearing from ISPOR members in more than 100 countries. It shares and delves into the increasing pressures for healthcare decision makers, from each other and external audiences, that are drastically impacting the industry.

Clinical researchers are among those who can find value from delving into the key areas that are impacting HEOR across the globe. In doing so, they can gain insights into how to best research and assess how to make the best health choices for patients, which could include incorporating new components into clinical trials, such as real-world evidence, and making studies more applicable to real-world experiences and more accessible and understandable for patients, regulators, and payers.

Real-world evidence (RWE) came in as the top 10 HEOR trend in the most recent ISPOR report. Why is that? And what are some of the topics of interest from within RWE?

RWE is becoming ever more significant in healthcare decision-making by providing benefits that cannot be derived from randomized controlled trials (RCTs) alone.

RWE studies can present a more realistic view of patients and health outcomes in practice by offering insights into real people and their experiences with an intervention in the real world.

A clinical trial may produce a certain outcome based on the relatively small number of patients included in that trial. But outside of that highly controlled trial, RWE can help determine how a drug performs in the real world in large and various populations.

RWE analyzes real-world data (RWD) from wide and diverse groups of patients, through sources such as electronic health records (EHRs), patient registries that track specific diseases or treatments, and/or insurance claims and billing information.

Diverse data sources are of interest in RWE as data becomes more available through wearable devices (smart watches, heart monitors, etc.), in larger data sets (patient registries, EHRs), and through new analysis techniques (AI, ML, etc.).

So, as this data becomes more available and in larger data sets, a key element of RWE will be understanding data sources and ensuring the quality, accuracy, and transparency of the data.

What role does transparency play in the use of RWE, and how can those working in clinical research support it?

Transparency in RWE is a major factor in creating trust and use of data. For data to be trusted and used, the process of collecting, analyzing, and defining data needs to be transparent in RWE studies.

The Real-World Evidence Transparency Initiative — a joint initiative of ISPOR, the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council — is dedicated to establishing a culture of transparency for RWE studies.

Part of this initiative is ISPOR’s RWE Registry, which provides researchers with a fit-for-purpose platform to register their study designs before they begin work to facilitate the transparency needed to elevate the trust in the study results.

AI rose up the ranks, from #10 on the list to #3. What accounts for that increase in interest? And, specifically, how can AI/ML support HEOR in clinical research?

The rapid rise of AI and ML has taken off after OpenAI’s ChatGPT launch and AI’s increasing use by the public. AI and ML also have created the opportunity to revolutionize how researchers can gather, use, and interpret data.

Large language models (LLMs), such as ChatGPT, can deploy algorithms to look at data in various ways that can help researchers, scientists, payers, and others better understand the safety and effectiveness of treatment, analyze patient data, and help to determine the most effective treatment plans.

There is also particular interest in the potential of AI to assist in data mining to inform customized patient care — increasing both the efficiency and effectiveness/impact of patient care.  

After first appearing in the 2018 report, accelerated drug approvals are again a trending HEOR topic. What accounts for this return? What is the role of HEOR in supporting accelerated drug approvals?

It is not surprising to see increased interest in accelerating drug approvals, as there is tremendous pressure to expedite bringing innovative new therapies to the market, due in large part to significant unmet needs in rare diseases where often no viable treatments currently exist.

In 2022, 24 of 37 Center for Drug Evaluation and Research’s novel drug approvals used one or more expedited programs such as Fast Track, Breakthrough Therapy, Priority Review, or Accelerated Approval. And in the European Union in 2022, five medicines were approved under accelerated assessment, nine received conditional marketing authorizations, and five received approvals under extraordinary circumstances.

HEOR can play a critical role in filling evidence gaps for these accelerated-approval therapies in such ways as obtaining supporting evidence from RWD to supplement clinical trial data, which is often slow to conduct, if at all.

In some cases, HEOR can help validate markers with clinical outcomes data and bring greater confidence to the use of markers as indicators of efficacy. In others, HEOR can be applied to augment evidence that’s part of the regulatory process, particularly for medical devices and digital health apps.

Patient centricity is a term used as often as it’s lamented — some just don’t appreciate the vagueness it brings. From a HEOR perspective, what is patient centricity? And how can its principles be better applied in clinical research?

Patient centricity at its core is putting patients at the center of healthcare research, regardless of the specific use case.

HEOR harnesses the benefits of patient-centered research, which includes understanding the patients’ real-world journeys, preferences, and experiences, allowing researchers to gain insights into what patients consider to be a “success” in treatment and knowing what side effects they are willing to tolerate as part of treatment.

Beyond clinical research, patient centricity is now being introduced in regulatory processes and decision-making, including with the FDA, which is developing a series of patient-focused drug development guidance documents to address how patient experience data and other relevant information from patients and caregivers can be utilized for medical product development and regulatory decision-making.

For example, health equity, including the social determinants of health, is one patient-centered research concept that can be better applied, as treatments should be tested in populations where there is the greatest unmet need, often consisting of underrepresented minorities.

About The Expert:

Rob Abbott is the CEO and executive director of ISPOR — The Professional Society for Health Economics and Outcomes Research (HEOR), an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision-making for health globally. He is responsible for the Society’s global strategic direction and leadership.

Mr. Abbott has more than 20 years of experience providing executive-level strategic and operational leadership in businesses, nongovernmental organizations, government environments, as well as professional societies and associations. Most recently, he served as executive director and CEO of Health Technology Assessment International, a global nonprofit society focused on health technology assessment. He is recognized for his knowledge of HTA, strategy, and stakeholder relations. He is also a social responsibility thinker and advisor who has authored two books and numerous articles on the nature of business and society.

Mr. Abbott holds an undergraduate honors degree from the University of Victoria and graduate degrees from the University of Toronto.