Table 3 Submission and consideration of PROs as part of the early benefit assessment process of rare diseases
Category | Number of PRO data sets (n) | Percentage of events in relation to overall number of processes (%) |
|---|---|---|
Industry – type of PRO data submitted (module 4) | ||
 Clinical PROs | 41 | 51% |
 HRQoL | 61 | 75% |
 Preferences | 2 | 2% |
 Satisfaction | 1 | 1% |
Industry - extent of PRO data submitted (module 4) | ||
 No PRO data submitted | 13 | 16% |
 Data on one PRO category | 35 | 43% |
 Data on two PRO categories | 31 | 38% |
 Data on three PRO categories | 2 | 2% |
Early benefit assessment – type of PRO data considered in synopsis | ||
 Clinical PRO | 39 | 48% |
 HRQoL | 58 | 72% |
 Preferences | 0 | 0% |
 Satisfaction | 0 | 0% |
Early benefit assessment – extent of PRO data submitted | ||
 No PRO data considered | 43 | 53% |
 One PRO category | 63 | 78% |
 Two PRO categories | 54 | 67% |
 Three PRO categories | 2 | 2% |
GBA decision – extent of PRO data considered in the decision | ||
 No PRO data considered | 31 | 38% |
 One PRO category | 25 | 31% |
 Two PRO categories | 25 | 31% |
 Three PRO categories | 0 | 0% |
GBA decision – type of PRO data considered in the decision | ||
 Clinical PROs | 39 | 48% |
 HRQoL | 59 | 73% |
 Preferences | 0 | 0% |
 Satisfaction | 0 | 0% |
Comparison between data submitted and data considered by the GBA | ||
 Identical number of PRO categoriesa | 42 | 50% |
 Diverging number of PRO categoriesa | 29 | 40% |
 Clinical PROs - not considered by GBA | 8 | 21% |
 Clinical PROs - added by GBA | 6 | 15% |
 HRQoL - not considered by GBA | 19 | 31% |
 HRQoL – added in GBA decision | 2 | 3% |
 Preferences – not considered by GBA | 2 | 100% |
 Satisfaction – not considered by GBA | 1 | 100% |