Table 1 Characteristics of orphan medicinal products approved between 2010 and 2022
From: Trends in orphan medicinal products approvals in the European Union between 2010–2022
Variable | No of OMPs (n = 192) | Percentage (%) |
|---|---|---|
Year of approval by the European Commission | ||
2010 | 5 | 2.6 |
2011 | 4 | 2.1 |
2012 | 10 | 5.2 |
2013 | 7 | 3.6 |
2014 | 14 | 7.3 |
2015 | 18 | 9.4 |
2016 | 18 | 9.4 |
2017 | 17 | 8.9 |
2018 | 24 | 12.5 |
2019 | 7 | 3.6 |
2020 | 22 | 11.5 |
2021 | 20 | 10.4 |
2022 | 26 | 13.5 |
Legal basis of the MA application | ||
Article 8.3 | 171 | 89.1 |
Article 10.3 | 12 | 6.3 |
Article 10(a) | 6 | 3.1 |
Type II variation | 2 | 1.0 |
Article 10(b) | 1 | 0.5 |
Registration status (signoff date: 31 dec 2023) | ||
Approved | 186 | 96.9 |
Withdrawn | 6 | 3.1 |
SME status | ||
Yes | 19 | 9.9 |
No | 173 | 90.1 |
Protocol assistance | ||
Yes | 165 | 85.9 |
No | 27 | 14.1 |
Type of MA | ||
Standard | 129 | 67.2 |
Conditional | 41 | 21.4 |
Exceptional circumstances | 22 | 11.5 |
Accelerated assessment | ||
Yes | 124 | 64.6 |
No | 68 | 35.4 |
Type of molecule | ||
Small molecule | 101 | 52.6 |
Biologic | 67 | 34.9 |
ATMP | 15 | 7.8 |
Oligonucleotide | 6 | 3.1 |
Herbal preparation | 2 | 1.0 |
Radiopharmaceutical | 1 | 0.5 |
Repurposing | ||
No | 159 | 82.8 |
Yes | 33 | 17.2 |
ATC Code | ||
L Antineoplastic and immunomodulating agents | 76 | 39.6 |
A Alimentary tract and metabolism | 33 | 17.2 |
B Blood and blood forming organs | 21 | 10.9 |
J Antiinfectives for systemic use | 12 | 6.3 |
N Nervous system | 11 | 5.7 |
H Systemic hormonal preparations, excluding sex hormones and insulins | 9 | 4.7 |
M Musculo-skeletal system | 6 | 3.1 |
C Cardiovascular system | 5 | 2.6 |
R Respiratory system | 5 | 2.6 |
S Sensory organs | 5 | 2.6 |
D Dermatologicals | 3 | 1.6 |
V Various | 2 | 1.0 |
G Genito-urinary system and sex hormones | 1 | 0.5 |
P Antiparasitic products, insecticides, and repellents | 1 | 0.5 |
Not assigned yet | 2 | 1.0 |
Therapeutic areas (TOP 5 oncological diseases) | ||
1 Multiple Myeloma | 12 | 6.3 |
2 Leukemia Myeloid | 8 | 4.2 |
3 Lymphoma’s | 8 | 4.2 |
4 Leukemia non-Myeloid | 7 | 3.6 |
5 Lymphoblastic Leukemia-Lymphoma | 4 | 2.1 |
Therapeutic areas (TOP 5 genetic diseases) | ||
1 Haemophilia (A and B) | 7 | 3.6 |
2 Cystic fibrosis | 6 | 3.1 |
3 Muscular Dystrophy or Atrophy | 5 | 2.6 |
4 Familial Amyloidosis | 4 | 2.1 |
5 Anemias | 4 | 2.1 |
Therapeutic areas (TOP 1 infections) | ||
1 Tuberculosis | 4 | 2.1 |
Target age | ||
Adults | 107 | 55.7 |
Adults and children | 40 | 20.8 |
Adults and adolescents | 21 | 10.9 |
All ages | 12 | 6.3 |
Children | 6 | 3.1 |
Adolescents and children | 4 | 2.1 |
Adults and elderly | 1 | 0.5 |
Elderly | 1 | 0.5 |
Disease prevalence (per 10,000) | ||
< 0.2 (ultra rare) | 41 | 19.9 |
0.2–0.5 | 27 | 13.1 |
0.6– ≤ 1 | 40 | 19.4 |
> 1– ≤ 2 | 36 | 17.5 |
> 2– ≤ 4 | 50 | 24.3 |
> 4–5 | 12 | 5.8 |
Additional monitoring | ||
Yes | 124 | 64.6 |
No | 68 | 35.4 |
Number of pivotal efficacy clinical trials | ||
No clinical studies performed | 10 | 5.2 |
1 | 136 | 70.8 |
2 | 39 | 20.3 |
3 | 4 | 2.1 |
4 | 2 | 1.0 |
5 | 1 | 0.5 |
Main clinical trial designs (n = 241) | ||
Randomized, double blind, controlled trial | 117 | 48.5 |
Randomized, single blind | 2 | 0.8 |
Randomized, open label | 42 | 17.4 |
Partially randomized, open label | 1 | 0.4 |
Non-randomized, open label, sequential | 1 | 0.4 |
Open label, single arm | 65 | 27.0 |
Open label, 2-arm | 3 | 1.2 |
Open label, 4-arm | 2 | 0.8 |
Retrospective studies | 5 | 2.1 |
Observational cohorts | 3 | 1.2 |
Type of primary efficacy endpoints (n = 181) | ||
Oncological OMPs (n = 66) | ||
Surrogate | 52 | 78.8 |
Clinical | 14 | 21.2 |
OMPs addressing genetic diseases (n = 109) | ||
Clinical | 44 | 40.4 |
Surrogate | 37 | 33.9 |
Functional | 28 | 25.7 |
Anti-infectives OMPs (n = 6) | ||
Clinical | 2 | 33.3 |
Surrogate | 4 | 66.7 |