In the intricate landscape of contemporary healthcare systems, the organized, systematic, and independent evaluation of the safety, efficacy, and quality of medicines and other healthcare technologies stands as a critical imperative. When coupled with considerations of costs, this evaluation transforms into the realm of Health Technology Assessment (HTA), often referred to as “cost-effectiveness analysis,” playing a pivotal role.
HTA involves comparing the health benefits and incremental costs of a studied treatment against its best alternative. It addresses a fundamental question: Are the additional health gains provided by the new technology worth the price we have to pay for them? A negative answer implies a misallocation of resources that could be better utilized for purposes capable of generating superior health outcomes, thereby adversely affecting the patients intended to benefit from these technologies. Here, efficiency is not merely about cost savings; it is a matter of healthcare.
Consider an example: A study in 2007[1] analyzed the health improvements provided by a therapy using the novel monoclonal antibody bevacizumab against metastatic colon cancer and its incremental cost. The cost of gaining one additional year of life compared to the alternative therapy was nearly €150,000, deemed well above the acceptable reference value (willingness to pay) of the Spanish National Health System (SNS), rendering it inefficient. The budgetary impact was also examined, reaching €20.5 million per year under conservative assumptions. These results are significantly influenced by the calculation of health gains, where uncertainties abound, and the price of the medication.
Systematically subjecting new medicines and healthcare technologies to the filter of HTA is imperative, given their position at the forefront of knowledge, with continuous groundbreaking innovations across various therapeutic fields. Assessing them individually and informally is illusory. We are currently witnessing a biorevolution driven by biotechnological drugs, genomics, chimeric antigen receptor T-cell therapies (CAR-T), and CRISPR tools for researching and treating genetic diseases. The latter was approved by HTA regulatory agencies for treating specific diseases just two months ago.
The rapid pace of innovation in complex fields is compounded by the fact that information about the risk/benefit balance obtained from clinical trials is challenging and costly to obtain, subject to high levels of uncertainty, and can be modified by new scientific evidence obtained through real-world usage. Moreover, evaluation studies cannot extend indefinitely due to the urgency of having potentially game-changing innovations for unmet therapeutic needs. Additionally, this information is not only imperfect but also asymmetric, as innovative companies are often better positioned to possess it.
This complexity is evident in a study that found that more than half of the drugs authorized in Germany between 2011 and 2017 could not demonstrate additional clinical benefits, with only 10% having significant benefits and 15% having considerable added benefits.
For these reasons, public administrations organize and develop legislation, bodies, and methods for the evaluation of healthcare technologies as a mechanism to safeguard consumer interests and the interests of public health systems.
HTA, apart from being necessary, is decisive because it directly impacts patient health. Depending on how it is organized, it can either stimulate or depress biomedical innovation, influence economically significant industries, and contribute to rationalizing public health expenditure by guiding decisions on prices to be paid and public funding allocation for the adoption of new medicines and technologies. In 2020, the pharmaceutical industry in Spain ranked ninth in value-added generated, employing 50,984 individuals (2.2% of the entire Spanish industry), and was the third sector in terms of innovation intensity (Ministry of Industry, Commerce, and Tourism 2023). In 2022, it also ranked third in exports (Farmaindustria 2023). On the other hand, the medical technology industry in 2022 generated a revenue of €10.15 billion, with 43% exported, and supported over 30,500 direct jobs (FENIN 2023). In 2022, public spending on medicines and medical products amounted to €28.26 billion (Ministry of Finance 2023).
Funcas has recently published three technical notes on HTA in Spain, authored by Juan Oliva, Professor of Economics at the University of Castilla-La Mancha; José Vida, Associate Professor of Administrative Law at the Carlos III University of Madrid, and the undersigned. The first examines the barriers to HTA, its regulation —which is deemed inadequate—, its degree of development in practice, and new trends in Spain. The second study describes the complex and flawed administrative organization serving HTA. The third contains a concrete and detailed proposal for the reorganization of responsible administrative entities. Furthermore, on December 12, 2023, these technical notes were presented and discussed in a webinar on Health Economics and Policies, which can be viewed on Funcas’s YouTube channel. Commentators included José Ramón Repullo, emeritus professor at the National School of Public Health, providing insights from the perspective of the National Health System, and José María Labeaga, Professor of Economics at UNED, contributing from the standpoint of public policy evaluation. In subsequent articles, we will summarize their content and conclusions.