Rare diseases drugs have always faced challenges when it comes to HTA approvals, even as governments bring in more regulatory policies that make their path through assessment easier. Factors causing challenges for HTA bodies to assess orphan drugs include a lack of robust trial data due to difficulties in finding patients, the absence of randomised controlled trials, the use of surrogate endpoints, and the lack of active drug comparators.
In this article, Steve Kelly, Ioanna Stefani, Nimisha Raj, and Charlotte Poon discuss the results of their analysis of various challenges faced in four European markets – England, France, Germany and Scotland. They also explore how manufacturers can increase the chances of successful appraisals for rare disease medicines. Their results show that HTAs for orphan drugs can vary widely across Europe, causing inconsistencies in evidence requirements and recommendations.
To read the article published in PharmaPhorum, click here.