Across 261 Phase III rare disease trials submitted through the European Union Clinical Trials Information System (CTIS), the median end to end interval from initial CTIS submission to first authorization was 98 days, with a standard deviation (SD) of 43.2 days. Only 112 of 261 trials (42.9%) reached first authorization within 60 days, while 212 (81.2%) did so within 120 days. Country specific CTIS Part II spans were more dispersed, with a median of 159.5 days and SD of 243.9 days across 1,394 country records. Submission month, site footprint, digital recruitment, adaptive design and modality showed the clearest descriptive associations with speed or delay.
The trial level end to end CTIS clock had a 98 day median, SD 43.2 days and interquartile range of 36 to 116 days. The country specific Part II clock had a 159.5 day median, SD 243.9 days and interquartile range of 28 to 453.8 days across 1,394 country records.
End to end is the correct trial level EU submission metric. It is not assigned to individual countries. Country comparisons in this report therefore use CTIS Part II only.
End to end, 45 of 261 trials (17.2%) were authorized within 30 days, 112 (42.9%) within 60 days and 126 (48.3%) within 90 days. At the substantial delay thresholds, 150 of 261 (57.5%) took at least 60 days and 135 (51.7%) took at least 90 days. The cumulative share reached 212 of 261 (81.2%) by 120 days. For country Part II, 381 of 1,394 records (27.3%) closed within 30 days, 563 (40.4%) within 60 days and 615 (44.1%) within 90 days.
A 60 day planning assumption covered only 42.9% of first EU authorizations. A 120 day end to end planning window covered 81.2%, making it a materially more realistic operational base case for this cohort.
Among countries with at least 15 Part II records, Finland had the shortest median at 22 days (SD 119.7), followed by Denmark at 30.5 days (SD 206.2), Norway at 39.5 days (SD 265.4) and Sweden at 48.5 days (SD 258.1). The longest high volume medians were Spain and Belgium at 228 days, Italy at 226 days and Germany at 193.5 days.
| Country | n | Median d | SD d |
|---|---|---|---|
| Cyprus | 3 | 14 | 186.5 |
| Finland | 15 | 22 | 119.7 |
| Estonia | 5 | 29 | 247.7 |
| Denmark | 40 | 30.5 | 206.2 |
| Norway | 24 | 39.5 | 265.4 |
| Sweden | 42 | 48.5 | 258.1 |
| Ireland | 21 | 57 | 261.9 |
| Croatia | 9 | 68 | 255.3 |
| Portugal | 42 | 69.5 | 243.1 |
| Romania | 28 | 69.5 | 165.0 |
| Slovenia | 9 | 91 | 317.5 |
| Hungary | 39 | 134 | 240.7 |
| Greece | 40 | 134.5 | 211.7 |
| Latvia | 4 | 140 | 169.1 |
| Austria | 52 | 149.5 | 229.6 |
| Netherlands | 83 | 153 | 255.5 |
| France | 163 | 170 | 254.4 |
| Poland | 115 | 174 | 234.6 |
| Czechia | 53 | 176 | 235.1 |
| Germany | 156 | 193.5 | 245.4 |
| Bulgaria | 26 | 216 | 203.3 |
| Italy | 160 | 226 | 250.9 |
| Belgium | 83 | 228 | 247.4 |
| Spain | 163 | 228 | 250.5 |
| Slovakia | 13 | 248 | 309.5 |
| Lithuania | 6 | 392 | 264.2 |
Country medians were operationally meaningful, but SDs were large in nearly every market. Country selection therefore changes the expected Part II midpoint, while individual trial and country sequence still drive substantial variation.
July EU CTIS submissions had the shortest median at 31 days, with 16 of 25 trials (64.0%) below the 98 day cohort median. February submissions had a 41 day median and May submissions 50 days. November was slowest at 121 days, followed by December at 118 days and October at 116 days; 15 of 21 November submissions (71.4%) took at least 90 days.
The late year cluster was materially slower: October through December medians were 116 to 121 days, compared with 31 to 50 days for February, April, May and July. Submission timing should therefore be treated as a planning variable, not only a calendar detail.
Trials using digital or remote recruitment had a 112 day median: 56 of 79 (70.9%) took at least 60 days and 53 (67.1%) took at least 90 days, versus 94 of 182 (51.6%) and 82 (45.1%) without digital recruitment. Adaptive trials had a 109 day median, with 18 of 25 (72.0%) at least 60 days and 16 (64.0%) at least 90 days. Trials with more than 32 sites had a 112 day median, with 41 of 63 (65.1%) at least 60 days and 39 (61.9%) at least 90 days, versus 76.5 days, 109 of 198 (55.1%) and 96 (48.5%) among smaller site footprints.
Digital recruitment, adaptive design, randomisation and a large site footprint were all associated with longer EU CTIS review times. Paediatric trials moved faster in this cohort, with a 65 day median versus 105 days for non paediatric trials.
Cell therapy records had a 35 day median and gene therapy 41 days, compared with 106.5 days for peptide, protein or enzyme modalities. At disease level among groups with at least five trials, sickle cell disease was shortest at 42 days, while myasthenia gravis was longest at 118 days and 8 of 9 trials (88.9%) took at least 90 days.
| Modality | n | Median | ≥90 d |
|---|---|---|---|
| Cell therapy | 17 | 35 | 29.4% |
| Gene therapy | 17 | 41 | 35.3% |
| Oligonucleotide | 20 | 57.5 | 45.0% |
| Small molecule | 130 | 79.5 | 49.2% |
| Monoclonal antibody | 60 | 88.5 | 50.0% |
| Peptide/protein/enzyme | 56 | 106.5 | 60.7% |
| Disease | n | Median | ≥90 d |
|---|---|---|---|
| Sickle cell disease | 5 | 42 | 40.0% |
| Fabry disease | 6 | 75.5 | 50.0% |
| Phenylketonuria | 5 | 103 | 60.0% |
| IgA nephropathy | 5 | 108 | 80.0% |
| CIDP | 5 | 115 | 80.0% |
| Hereditary angioedema | 11 | 117 | 72.7% |
| Myasthenia gravis | 9 | 118 | 88.9% |
Modality did not produce a uniform complexity penalty. In this cohort, gene and cell therapy trials were faster than protein or enzyme trials. Disease specific results were more variable and should be treated as directional where denominators were small.