Affiliations: Health Economics and HTA, Institute of Health and
Wellbeing, University of Glasgow, Glasgow, UK. Tel.: +44 1360 660316/+44 789
414 3931; Fax: +44 1360 660316; E-mail: [email protected]
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.