Concerns about the rising costs of prescription drugs and other areas of health care have stimulated many initiatives in the United States and other advanced economies to measure and inform the decision-making value of pharmaceuticals and other technologies. Consequently, HEOR research has grown in importance to examine the economics of health care, including the assessment of treatment costs, the effectiveness of medical technologies, and the evaluation of treatment outcomes.
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Cost estimation is a fundamental element in HEOR because it assesses the cost-effectiveness of health care. Here, cost means the balance between the costs of a treatment or medical technology and the results achieved in the form of improved patient health. Value estimation allows us to determine how efficiently healthcare resources are used and what measures can be taken to optimize outcomes with limited budgetary resources.
Value assessment systems are tools and methodologies that help to standardize the evaluation of costs and outcomes in HEOR. They provide a framework and criteria for comparing different medical technologies, procedures or medicines. Such systems enable informed decisions about resource allocation and healthcare management.
Key Value Assessment Frameworks
By 2023, several organizations in the United States and other countries had developed value-assessment frameworks that help patients, physicians and payers assess the relative value of care and health care. These frameworks vary in their missions, scope of work, and methodological approaches. For example:
- The National Institute for Health and Care Excellence (NICE) is an authoritative body that develops guidelines and assesses the value of medical technologies, drugs, and clinical procedures in the United Kingdom. They use economic evaluation, cost-benefit analysis, and other approaches to determine the value of medical interventions.
- The Institute for Clinical and Economic Review (ICER) in the US is an independent, not-for-profit organization dedicated to assessing the value of medical technologies. They develop guidelines to assist decision makers in determining the value and effectiveness of medical technologies.
- Pharmaceutical Benefits Advisory Committee (PBAC) in Australia. PBAC is the body that evaluates and recommends pharmaceuticals for funding.
- ASCO (American Society of Clinical Oncology), MSKCC (Memorial Sloan Kettering Cancer Center) and NCCN (National Comprehensive Cancer Network) are important oncology organizations that play a role in evaluating and determining the value of cancer treatment. ASCO is a professional society of oncologists that develops clinical guidelines and conducts research related to cancer treatment. MSKCC is one of the leading cancer centres in the world that treats cancer, conducts research and develops treatment recommendations. NCCN is a national organization of the leading cancer centres in the United States. They develop clinical practice guidelines for cancer treatment and set standards of care for different types of cancer. They also consider the value and effectiveness of technology and drugs in developing their recommendations. These organizations contribute significantly to assessing the value of cancer treatment and help ensure a high level of quality of care for patients with cancer.
- Health Technology Assessment International (HTAi) Recommendations is an international organization of experts in health technology assessment. They develop recommendations and methodologies for assessing the value and effectiveness of health technologies globally.
All of these organizations play an important role in determining the value of health technologies and influencing healthcare decision-making in their countries and beyond.
Components of Value Assessment Systems
Value assessment systems serve as tools for measuring the value of medical interventions and treatments, but the concept of “value” can have different meanings for payers, physicians, and patients. Each framework reflects the interests and expertise of its developing organization, takes into account different end-users, and different types of decisions. Because of this, evaluation systems may use different methods, data and inputs to evaluate treatment, medical devices or healthcare. Nevertheless, it is worth noting a few key components that go into different value assessment systems:
- Outcome evaluation. This component includes an assessment of the clinical and economic outcomes achieved with a particular medical technology. It may include analysis of patient health outcomes, treatment duration, quality of life, symptom reduction, or prevention of complications. Outcome evaluation helps determine whether the technology is effective and appropriate.
- Cost estimation. This component includes an estimate of the cost of the medical technology or medication. It may include the cost of production, the cost to patients or the health care system, and the cost of related services such as hospitalization or rehabilitation. The cost estimate helps determine how cost-effective the technology is.
- Comparative analysis. This component involves comparing different medical technologies or drugs to identify differences in cost and effectiveness. Benchmarking helps determine which technologies are more effective and feasible in terms of cost and outcomes achieved.
- Economic modelling. This component includes the use of mathematical models and statistical methods to predict and simulate the effects of medical technologies at the population level. Economic modelling estimates the long-term effects and impact of different strategies on healthcare costs and outcomes.
- Safety assessment. It helps determine the potential risks and adverse reactions associated with the use of a particular technology and the impact of these risks on the overall value and safety of use.
- Patient-reported outcomes and quality of life assessment. This component includes an assessment of patient perceptions and satisfaction with the outcomes of treatment or the use of medical technology. It may include assessments of quality of life, functional status, and pain or discomfort levels.
- Budget impact analysis and affordability considerations. This component includes an assessment of the economic impact and affordability of medical technologies or medications to patients, health systems, and society as a whole. It helps determine the cost of adopting and using the technology, as well as its availability to patients of different financial means and in different regions.
All of these components are important elements of various value systems, and help inform healthcare decisions. Their interaction and analysis allows the full value and effectiveness of medical technologies, drugs, or procedures to be assessed.
Methodologies within Value Assessment
A variety of methodologies are used in healthcare valuation to help scientists, healthcare organizations, and decision-makers assess the value and effectiveness of healthcare technologies and services. Here are some of them:
- Systematic literature reviews and evidence synthesis. These methods involve a systematic survey and review of available scientific research and literature to gather and synthesize evidence on the effectiveness, safety, and efficiency of medical technologies.
- Health technology assessment (HTA) methods for assessing the cost-effectiveness of medical technologies and services. These methods include cost-benefit analysis, cost-effectiveness analysis, cost-benefit analysis, and other economic evaluations.
- Analytic decision modelling methods. These methods are used to create mathematical models that estimate the cost and performance of medical technologies in various scenarios. Models can account for factors such as length of treatment, drug and technology costs, probabilities of outcomes, and other important variables.
- Quality-adjusted life years (QALY) is a measure that takes into account not only life expectancy, but also quality of life. It is used to assess the overall value of medical technology, taking into account both longevity and patients’ level of health. In addition to QALYs, other outcome measures such as DALYs (disability-adjusted life years lost), treatment effectiveness, and others are also used.
- Sensitivity analysis and uncertainty assessment. These methods are used to assess the impact of uncertainty and variability on value outcomes. Sensitivity analysis examines how changes in input parameters affect the final estimate, while uncertainty estimation estimates the range of possible values for the results.
Challenges and Opportunities of Value Estimation Systems
Value-assessment systems can be a critical tool that healthcare decision-makers use when making complex decisions related to healthcare services and treatments. To date, none of the valuation systems that have been developed are fully mature and ready to support healthcare decision-making. And because they have the potential to affect patients’ access to needed healthcare services, it is important to consider the strengths and limitations of these structures.
There are several challenges that value-based assessment systems face. These include:
- lack of patient-centeredness;
- lack of transparency;
- limited evidence base;
- unproven methods;
- confusing results and misinterpretation of value assessment results.
To ensure the validity of value assessments, framework methodologies and models must be fully transparent and reproducible. All high-quality evidence, including factual data, should be included in the estimates and should be regularly updated as new evidence becomes available. It is critical that the methodologies underlying the framework be robust and validated and that their potential impact on patients be understood.
Providing educational programs and information resources to end users (patients, physicians, payers) will help to prevent misinterpretation of value assessment results in medicine. Involving end users and other stakeholders in the value assessment process can help accommodate different needs, preferences, and perspectives. Consultation, public hearings, and feedback from users can help refine and improve the evaluation process.
Ensuring more informed and rational healthcare decision-making will help ensure that independent expertise is available to experts and organizations who can evaluate the results and methodology of the assessment. This will help verify the validity and reliability of the results and provide an independent perspective.
Also, the unique needs of patients and the health care system should be considered when interpreting the results of the value assessment. This allows the assessment results to be more accurately applied to specific situations and decisions.
Cost estimation plays an important role in health economics research (HEOR). It helps to measure the value of healthcare interventions and services, helps to make informed healthcare decisions and optimizes costs. Cost estimation also contributes to the development of policies and practices aimed at improving the quality of health care and ensuring access to care.
This article seeks to contribute to current discussions and advances in the field of value assessment of health care interventions in HEOR. It seeks to present a comprehensive analysis of existing evaluation frameworks and methods in order to improve decision-making, achieve cost-effectiveness, and improve patient outcomes in healthcare systems around the world.
The integration of robust and standardized platforms plays an important role in this process, contributing to the reliability of data and improving the quality of healthcare decision-making.