Health Technology Performance Assessment: Real-World Evidence for Public Healthcare Sustainability

Abstract

Objective: Health technology financing is often based on randomized controlled trials (RCTs), which are often the same ones used for licensing. Since they are designed to show the best possible results with typically Phase III studies conducted under ideal and highly controlled conditions to seek high internal validity and maximize the chance of demonstrating clinical benefit, they often do not reflect likely effectiveness in routine clinical care. Consequently, it is not surprising that technologies do not always perform in real life in the same way as controlled conditions. Since financing (and price paid) decisions can be made with overestimated results, health authorities need to ask whether health systems achieve the results they expect when they choose to pay for a technology. The optimal way to answer this question is to assess the performance of financed technologies in real world settings. Health technology performance assessment (HTpA) refers to the systematic evaluation of the properties, effects, and/or impact of a health intervention or health technology in the real world to provide information for investment/ disinvestment decisions and clinical guideline updates. The objective is to describe the development and principal aspects of the Guideline for HTpA commissioned by the Brazilian Ministry of Health. Method: Extensive literature review, refinement with experts across countries and public consultation. Results: A comprehensive guideline was developed, which has been adopted by the Brazilian government. Conclusion: We believe the guideline, with its particular focus on disinvestment, along with the creation of a specific program for HTpA, will allow the institutionalization and continuous improvement of the scientific methods to use real world evidence to optimize available resources not only in Brazil but across countries.Keywords: Technology Assessment, Biomedical; Observational studies; Health policy; Health Information Systems Policy implications: Real-world evidence is emerging as an important tool to aid decision-making regarding health technology financing. Over the last decade, countries have developed health and/ or administrative registries capable of providing quality data for assessing health results in real life. At the same time, budgetary constraints and the pressure to finance emerging high-priced technologies are making it inevitable for health authorities to start assessing whether patients and society are getting the results from financed technologies agreed upon during investment decisions. This paper presents the concept of Health Technology performance Assessment and the development of a guideline, commissioned by the Brazilian Ministry of Health, to implement such activity. We believe it is the first guideline to formally incorporate the use of real-world evidence for updating clinical guidelines, price renegotiation and disinvestment decisions.

Assessing the efficacy‐effectiveness gap for cancer therapies: A comparison of overall survival and toxicity between clinical trial and population‐based, real‐world data for contemporary parenteral cancer therapeutics

“…Interest in the use of RWE to support health policy decisions, such as for drug reimbursement decisions, also has been generated . In particular, there is interest in the use of this evidence to provide ongoing re‐evaluation of technologies to ensure that effectiveness and value are preserved after regulatory approval and adoption …”

“…64 In particular, there is interest in the use of this evidence to provide ongoing re-evaluation of technologies to ensure that effectiveness and value are preserved after regulatory approval and adoption. 65 However, RWE is not without its limitations. Comparative population-based studies may have limited internal validity and population-level databases may not collect all relevant information, including data concerning patient performance status, comorbidities, and sociodemographic variables (eg, socioeconomic status, rural residence, and/or ethnicity), which have been demonstrated to have an impact on health care disparities and outcomes.…”

Policy Change and the National Essential Medicines List Development Process in Brazil between 2000 and 2014: Has the Essential Medicine Concept been Abandoned?

“…Delisting or moving to second-generation DAAs is now taking place across countries, including Brazil. The country, faced with growing litigation, has also advanced in forging a disinvestment protocol, fuelled by Conitec [15,37,38].…”

Role of Health Technology Assessment in Pharmaceutical Market Access in Developed Countries

“…HTA might highlight the drugs that are expensive compared to their benefits, or might outline the indications and patient groups that would have additional value. Unsafe and ineffective drugs will also be discovered during these processes, leading to active dissemination [18][19][20].…”

“…The proposed models include the role of HTA. In addition, HTA activities are increasingly used to guide disinvestment activities, with monies transferred to fund more effective and/or more efficient medicines [18][19][20]. Discussion of disinvestment activities is outside the scope of this chapter.…”