VP81 Health Technology Assessment And Rare Disease Decision Making: Focus On Orphan Drugs

Abstract

INTRODUCTION:

Health Technology Assessment (HTA) is applied to determine the value of innovative technologies. It usually relies on robust assessment of the clinical cost-effectiveness of the technology, while clinical and economic evidence required for this purpose are often not available for orphan drugs (OD) (1,2). The objective of the study is to undertake a systematic comparison between HTA agencies worldwide in order to identify similarities and differences in the methods and processes in HTA of OD.

METHODS:

A cross-sectional web-based survey was conducted between September 2013 and May 2015. The data were obtained from a semi-structured questionnaire. We received responces from 161 HTA organizations based in 39 countries.

RESULTS:

HTA of OD is performed by agencies in South America (38.5 percent), followed by agencies in Australia (37.5 percent) and Europe (36.1 percent). The agencies in high income countries produce more assessments of OD (36.8 percent), which in 31.2 percent they determine as innovative technologies compared with 11.8 percent of the units based in low income countries and active in OD assessment (11.1 percent). We prove association (p< .05) between (i) the type of HTA and income per capita; the level at which the organization operates; its main activity; and the level of recommendation dissemination; (ii) the main target group and consumers of the final HTA product; the stage of evolution of the technology, on which it is likely to be assessed; and approaches to identify innovative technologies. The most active in the preparation of HTA reports are biomedical companies or other organizations in the private sector (50.0 percent) and organizations in the pharmaceutical and/or medical industry (66.7 percent). HTA bodies that assess OD develop (36.0 percent) and distribute recommendations (35.9 percent) nationally; their main activity is to produce guidelines for good clinical practice (46.9 percent). Agencies that perform OD assessment are active in evaluation of innovative (37.2 percent) and emerging (35.9 percent) technologies, which are able to be identified by developing early warning systems (32.0 percent).

CONCLUSIONS:

Making coverage decisions based on HTA recommendations control the technologies introduction into the healthcare system, that is why it's very important that this tool is properly adjusted to the specific needs of OD assessment (3).