Therapeutic Effectiveness Assessment: Approaches in Contemporary Research

Abstract

Today, the way in which therapeutic effectiveness is assessed is once again subject to criticism. Burdensome bureaucratic regulatory procedures in traditional clinical research, the costs of health care systems, and the uncertainness as to the most effective treatment options available have raised significant questions about whether traditional research methods, such as randomized controlled trials (RCTs), can adequately address the contemporary issues posed by the health care systems. In particular, the recent development of health economics-related research demonstrated the limitations of RCTs as research methods ( Kumar, 2013 ), widely criticized for its incapacity to reduce uncertainty about relative effects of treatments in individual patients. In order to better inform decision-makers clinicians as well as patients on the best practices available, the health economic studies pointed out the need for developing new research methods and designs more likely to provide evidence-based information on the effectiveness, benefits, harms, and costs of different treatments. These changes in clinical research designs and methods of evaluation of therapeutic effectiveness raise new ethical concerns and regulatory questions that need to be addressed properly. Recent initiatives to revise the legislative and regulatory framework for biomedical research (USA, EU, and France) directly tackle some of these issues. After describing contemporary advances in types of research on therapeutic effectiveness, this article will give an overview of the ongoing revision of the regulatory and legislative framework in the USA, EU, and France with a special focus on the way these initiatives address the legal and regulatory issues raised by the therapeutic effectiveness evaluation.