Pharmaceutical market access and the challenges of health technology assessment in the United Kingdom

Abstract

AbstractHealth Technology Assessment (HTA) has been formally used in the United Kingdom for more than 10 years to help determine evidence‐based decisions on the access to new drugs, based on evidence of clinical and cost‐effectiveness. Separate HTA systems have developed in England and Wales (NICE, AWMSG), and Scotland (SMC). The primary clinical evidence desired for technology appraisal by the HTA bodies is the comparative randomized controlled trial because of its good internal validity, but the role and value of observational study designs for providing data on real‐world effectiveness have been recognized in the methods guidance of these bodies. To date HTA has had a fairly limited impact on drug development and clinical trial design in the United Kingdom, coupled with limited use to date of non‐RCT data for assessing treatment effect and outcomes in NICE/SMC appraisals. Nonetheless, with new initiatives such as closer regulatory and HTA coordination in Europe, and “conditional market access” schemes such as the Innovation Pass in the United Kingdom, in the future pharmaceutical companies are likely to need to adopt a more flexible approach to drug development and clinical data collection involving RCT, observational data collection, and indirect comparisons to meet the needs to provide relevant clinical and cost‐effectiveness evidence at drug launch and post launch. Drug Dev Res 71: 478–484, 2010. © 2010 Wiley‐Liss, Inc.