Patient involvement in HTA: What added value?

Abstract

HTA is an interdisciplinary assessment of evidence and knowledge about the intended and unintended effects of using a health technology. Patients with rare diseases have valuable knowledge about the illness in the real-life setting, but too often their views are seen as anecdotal or biased. So, more needs to be done to elicit patients’ perspectives to add value to HTA through effective participation of patients throughout the HTA process and collection of evidence about patients’ perspectives through robust qualitative research.

Traditionally HTA has been a broad assessment to move evidence into practice, but in recent years a more limited view of clinical and cost effectiveness has been the focus. For HTAs in rare diseases, this is not enough. Consideration of ethical, organizational and social issues are vital and here patients’ perspectives could be particularly valuable to bring a real-life understanding of the potential impact of the health technology.

As countries around the world put more emphasis on creating clear plans to manage rare diseases, we need to ensure that all stakeholders work together to ensure that HTAs are being used flexibly to ensure that there is equity of access to therapies for rare diseases that provide real added value.