Table 4.

Summary of key similarities and differences between DURDs and DORDs identified in the present study

Similarities between DORDs and DURDs
Number of studies	Submissions for DURDs and DORDs are similar in terms of the number of studies considered in the CDR review of clinical data
Overall reasons for negative recommendations	Insufficient clinical evidence was the most common reason for a negative recommendation for both DURDs and DORDs, followed by cost-related issues
Differences between DORDs and DURDs
Growth in annual submission number	The steady growth in the total number of annual DRD submissions is predominantly attributable to growth in the number of annual submissions for DORDs, whereas the annual number of DURD submissions has risen only slightly
Molecular basis	DURDs are distinct in by being almost exclusively biologic molecules, whereas DORDs include a substantial proportion of small molecule-based therapies
Study size	Sample sizes for studies that support submissions for DURDs are generally smaller than those for DORDs
Study design	The majority of submissions for DURDs contained clinical data from non-randomized uncontrolled trials without comparator, whereas most DORD submissions included data from high-quality trial designs with active and/or placebo control arms and double-blinding
Cost	The average treatment costs of DURDS are generally substantially higher than those of DORDs
Recommendation type	Relatively more negative than positive reimbursement recommendations were issued for DURDs compared to DORDs
Reasons for negative recommendation	The rate of negative recommendations relating clinical reasons only was greater for DURDs than for DORDs