Health systems have developed at different speeds, and with differing degrees of complexity throughout the twentieth century, reflecting the diverse political and social conditions in each country. Notwithstanding their diversity, all systems, however, share a common reason for their existence, namely the improvement of health for their entire populations. To attain this goal a health system undertakes a series of functions, most notably, the financing and delivering of health services.
Since available resources are limited, delivering health services involves making decisions. Decisions are required on what interventions should be offered, the way the health system is organized, and how the interventions should be provided in order to achieve an optimal health gain with available resources, while, at the same time, respecting people’s expectations. Decision-makers thus need information about the available options and their potential consequences. It is now clear that interventions once thought to be beneficial have, in the light of more careful evaluation, turned out to be at best of no benefit or, at worst, harmful to the individual and counterproductive to the system. This recognition has led to the emergence of a concept known as “evidence-based medicine” (EBM), which argues that the information used by policymakers should be based on rigorous research to the fullest extent possible.
Health technology assessment (HTA) increasingly plays an important role in informing reimbursement and pricing decisions and providing clinical guidance on the use of medical technologies across the world. In addition to safety and efficacy information, health economic and outcomes research (HEOR) data are also receiving expanded attention in these assessments in many countries, due to payers seeking better value for money spent on treatments. HTA is now commonly viewed as a tool to assist evidence-based health-care decisions.
EBM has been defined as “the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients”. The origin of this evidence-based approach can be seen in the application of clinical medicine delivered at an individual level. Pressure to base decisions on evidence has, however, been extended to other areas of health care, such as public health interventions and health care policy-making. In this context, evidence is understood as the product of systematic observation or experiment. It is inseparable from the notion of data collection. The evidence-based approach relies mainly on research, that is, on systematically collected and rigorously analyzed data following a pre-established plan.
There are exciting new developments in basic science that could lead to targeted, highly effective and curative treatments. Health systems are improving their electronic records and recording health outcomes, which can be analyzed using structured, sophisticated analyses in real-time. There are also new collaborative approaches between healthcare providers and technology developers to enable evaluation of technologies in the health system before adoption or early in adoption to optimize use. There is a need and an opportunity to harness these developments and improve the effectiveness and efficiency of evidence production for new health technologies to input to HTA and inform decision making. Clinicians, managers, patients, and technology developers need to be involved to ensure that the process to a coverage decision is not only efficient but that it is also effective. To be effective, health services need to be organized to enable rapid and appropriate introduction of effective technologies and disinvestment of ineffective technologies. This suggests an additional responsibility for HTA and it would involve helping technology developers understand clinical and patient needs, evidence generation requirements, and limitations and helping health systems understand the potential and implications of new technologies and possible challenges of implementation.
Therefore, to sum everything up, the evidence should be both efficient as well as effective in order to develop more agile and adaptive processes that help to broker alignment among technology developers and health systems (including healthcare professionals and patients). This suggests that HTA needs to innovate and be prepared to play a more active role to influence evidence production and help facilitate dialogue among stakeholders to optimize technology development and use.
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The growing cost of discovery and development of orphan drugs is having an overall impact on the patient access to orphan medicines. Orphan drugs, which are usually not covered by insurance systems are inaccessible to patients due to their high cost, and even when they are covered, patient cost-sharing (through co-payments or coinsurance) can still limit their access.