Comparative Effectiveness Research in the Regulatory Setting

Abstract

Consumers today have access to more information regarding everyday decisions than ever before. Healthcare is no exception. The US health reform laws signed in March 2010, provided a vehicle for systematically facilitating the application of this trend to the US healthcare system through the creation of an independent, government-funded institute, whose purpose is to advance the understanding of which treatments and approaches to healthcare work best for which patients.

This article discusses the implications of comparative effectiveness research (CER) for governments, industry, healthcare systems, clinicians and patients. Properly developed and applied information derived from CER can help establish the value of medicines, procedures and services, and promote a more qualityfocused, cost-effective healthcare system. However, much uncertainty remains that centres on precisely how this information will be used. Recently, many patient groups, physicians and biopharmaceutical companies have raised concerns that CER might have the unintended consequences of restricting access to treatments.

These effects must be weighed carefully when considering whether comparative effectiveness data should become a prerequisite for US FDA submissions. The process of designing and implementing comparative clinical trials at such an early stage in product development poses numerous challenges including dose selection, use of marketed comparators (which may lead to consumer bias and increased patient dropout rates), physician comfort and experience (for medical devices), and endpoint selection, among others, and could delay introduction of new therapies. In addition, ‘over-interpretation’ of early comparative data could lead to an underappreciation of benefits in patient subgroups and an undervaluation of incremental innovation. Thus, a requirement for premarketing comparative studies could prove not only to create a costly disincentive to invest in developing therapies, but could also have a detrimental impact on their development.

Regardless of how information is provided to patients and healthcare decision-makers, it should be gathered in a manner that does not delay patient access to new technologies nor hamper innovation. The desire to provide consumers with more information regarding choice is well intentioned; however, patient care and satisfaction should always remain the foremost concern when discussing the implementation of new policies in the healthcare field.