Introduction

What is HEOR?

Health Economics and Outcomes Research (HEOR) is a growing field of interdisciplinary research that provides key information for policymakers and payers to determine access to care services and treatments. Health Economics examines the population unmet needs, economic value of interventions, and efficiency of resource use in the current society, whilst Outcomes Research represents a wide range of observational studies and patient surveys following an intervention. In the era of resource scarcity, HEOR studies complement clinical development data to help decision makers balance both costs and benefits and choose the best available interventions.

What is Health Technology Assessment (HTA)?

HTA involves multiple disciplinaries’ effort to determine the value of a health technology with the goal of informing decision-making in a high-quality healthcare system. It composes of systematic evaluation of properties, effects, or other impacts of healthcare technology with explicit analytical frameworks and a variety of methods, with an interdisciplinary approach.

Is HEOR the same as HTA?

While the two fields share many similarities and common purposes in health policymaking, they are slightly different in terms of definition:

  • HEOR is a multidisciplinary field of research that measures and evaluates both the clinical and economic benefits of interventions.
  • HTA, however, refers to the process or activity to systematically assess the clinical efficacy, safety, and cost-effectiveness of interventions, which is typically performed by regulatory authorities or trusted third parties to determine the appropriateness of adopting and/or reimbursing the interventions.

 

Researchers and pharmaceutical companies often apply the principles and methods in HEOR to generate evidence to support decision makers in the HTA process within authorities. In this context, the two terms are often used interchangeably. However, HEOR can also be applied to understand prescribing behaviors and treatment outcomes in reality, which helps to improve clinical care.

What are the components in HEOR?

Several types of study are commonly seen in the field of HEOR:

  • Economic evaluation: Cost-effectiveness (CEA), cost-utility (CUA), and cost-benefit analyses (CBA) are the main techniques in Health Economics to examine whether the extra benefits of new interventions could be outweighed or justified by their extra costs when compared with, in most cases, the standard care or a reference intervention.
  • Budget impact analysis (BIA): A BIA is a technique similar to conventional economic evaluations, but it focuses on the financial consequences or total budgets required if a new intervention is adopted, instead of its value-for-money.
  • Cost-of-illness (COI) analysis: A COI analysis measures the tangible (e.g., medical, treatment and social care) and intangible costs (e.g., productivity losses) resulting from a medical condition, which usually indicate the size of burden of a disease in a society.
  • Health-related quality-of-life (HRQoL) study: An HRQoL study assesses the impact of a medical condition, an intervention or general health state on an individual’s ability to live a fulfilling life. It summarises the physical, mental health and social functioning of a patient usually with the help of surveys. The findings also provide good reference figures to set up economic models, cost-utility analysis, and cost-benefit analysis.
  • Projection modelling: Health economists also make projections on the future disease burden, such as incidence, prevalence, healthcare budgets, mortality etc., in order to provide reference values for policymakers to plan healthcare resources ahead.
  • Comparative effectiveness study: This is a direct comparison between interventions to assess the effectiveness or safety, which assists patients, clinicians, and policymakers to make informed decision on whether to adopt a technology. Methods mainly include clinical trials and observational epidemiological studies.
  • Systematic review and Meta-analysis: When there is abundant literature in the field, a systematic review gathers all available and eligible research by systematic methods to summarise an answer for a research question. A meta-analysis can then be conducted to pool the findings quantitatively from similar studies using statistical techniques.

Why are HEOR and HTA important in policymaking?

We live in a changing world of ever-expanding choices of healthcare products and services that meet the increasing health needs of the population. Given the scarcity of financial and human resources but an inelastic demand for effective treatments, there is a need to call for balance between cost and benefit – in other words – to decide whether the costs of new technologies can be justified by their incremental benefits to the population.

 

A formal HTA process that evaluates both the clinical and economic values of interventions, possibly with the help of HEOR methods, serves to guide evidence-based plans to prioritise resources and make formulary listing decisions, which ultimately maximise health equity. Here are some collective benefits of using HTA and HEOR:

  • Maximise the efficiency of resource allocation
  • Enhance the evidence basis and transparency of policymaking
  • Form the ground for reimbursement and pricing negotiation
  • Inform and facilitate regulatory approval and post-marketing surveillance
  • Understand clinical difficulties, patient outcomes and preferences
  • Identify burdensome therapeutic areas and unmet needs
  • Promote the financial sustainability of a healthcare system

Who could be the beneficiary of HEOR findings?

  • Clinical professionals
  • Policymakers and healthcare system payers
  • Regulatory authorities
  • Patients and advocacy groups
  • Pharmaceutical and life science companies
  • Researchers and academia

What are the roles of clinical trials and real-world evidence (RWE) in HEOR?

Clinical trial is a type of experimental study that actively and prospectively assigns human subjects into groups of interventions, which may include placebo, and assess the health effects of the tested interventions. Alternatively, RWE are observational studies using epidemiological methods generated from real-world data (RWD) or healthcare big data, which sources include electronic medical records, insurance claims, online and mobile data, surveys, registries and other routinely collected and maintained databases.

 

Over the years, randomised controlled trials have been considered as the gold standard in the evidence pyramid. However, they are typically expensive to conduct with stringent eligibility criteria when selecting participants. RWE has been a useful resource to work hand-in-hand with trial evidence, especially in post-marketing studies and rare disease areas. It also provides complementary evidence about patients excluded in a trial setting and drug use in the real-world and long-term setting. In recent years, regulatory authorities have also started to set up guidance about the use of RWE in HTA submissions.

What are the functions of HTA?

  • Informing policies or decisions made by advising payers, guiding clinicians and patients about technology use and acquiring/investing healthcare technology/interventions
  • Supporting decisions by public health authorities about conducting population health programs
  • Support decisions about technology development and marketing, investors in the healthcare sector, and research agencies about evidence gaps and unmet needs

How can HTA be used for informed decision-making?

  • To provide evidence-based information to support healthcare decisions
  • Systematically examines the short and long-term health and economic effects of healthcare technologies

What is Horizon Scanning?

Horizon scanning is an umbrella term that refers to the active and systematic identification and quantification of novel innovations, trends and factors that impact and bring about paradigm shifts in today’s medical treatment modalities on an ongoing basis.

 

Horizon scanning provides a foresight of the potential health technologies in the pipeline of innovation in the near future. Using together with other forecasting tools for healthcare burden, policymakers can easily identify unmet needs in the population and then proactively assess new treatment options with HEOR and HTA initiatives specific to chosen medical conditions.

Where can I find additional resources about HEOR, HTA and Horizon Scanning?

Check out our free HEOR101 mini lecture series! It provides fundamental yet comprehensive information about the key concepts, methods, and practical applications of HEOR and HTA for everyone who is interested.

 

Other useful resources: