Abstract

Background

We have seen significant advancement in a range of health technologies, some with transformative or curative potential. Nevertheless, it is often unclear how global health systems recognize or reward innovation.

Objectives

To consider what is transformative, challenges for transformative therapies, and downstream health ecosystem effects.

Methods

A systematic review of publications in English between 2012 and 2018 was conducted with a focus on value assessment processes and health system effects of a range of breakthrough health technology categories. After screening 9012 records, 222 unique studies were identified. The study also included an analysis of 100 health technology assessments (HTAs) from 5 markets to consider how and in what ways global HTA bodies evaluate transformative therapies. Global sales and technology/procedure utilization data were also evaluated to gain insights into patient access and commercial impact.

Results

This article evaluated uncertainties around evidence of efficacy, safety, and duration of effect, as well as underlying study quality and methodological considerations in the target categories. Although many HTA evaluations had similar approaches to assessing parameters such as safety, there were significant differences across technology categories. Technology-driven trends also surfaced where global HTA and payer systems may not yet be prepared to recognize and reward emerging technology impacts, including use of next-generation diagnostic results to guide care, considering novel impacts on therapy sequencing and clinical pathway management, and changes in payment and health delivery models.

Conclusions

Some trends stemming from rapid evolution of breakthrough therapies will prompt reconsideration of our conventional value assessment and reward models, because health system measurement and management processes have not fully anticipated their effects.