OrphanDrug
OrphanDrug
Pharma Knowledge Base
GB

United Kingdom

GB

Europe

Contents

  1. 1. Regulatory Overview
  2. 2. Regulatory Pathways
  3. 3. Available Drugs

Regulatory Overview

United Kingdom is regulated by the MHRA (Medicines and Healthcare products Regulatory Agency). The country has 3 documented regulatory pathways for accessing unapproved or unlicensed medicines, covering named patient programs, compassionate use schemes, and special access arrangements.

Regulatory Authority

MHRA (Medicines and Healthcare products Regulatory Agency)

Regulatory Pathways (3)

Early Access to Medicines Scheme (EAMS) – UK 90 days

EAMS provides patients with life-threatening or seriously debilitating conditions access to promising medicines before full marketing authorization. The scheme includes a scientific opinion from MHRA on the benefit-risk balance based on early clinical data.

Application Form: MHRA EAMS Application with Promising Innovative Medicine (PIM) designation
Legal Basis: Statutory
Eligibility: Patients with life-threatening or seriously debilitating conditions with significant unmet medical need, no alternative treatment available
Reference: Early Access to Medicines Scheme Regulations 2014
Named Patient Unlicensed Medicine – UK 7 days

UK physicians can prescribe unlicensed medicines for named patients under their personal responsibility. The prescriber takes responsibility for the decision and monitors the patient.

Application Form: Physician Declaration
Legal Basis: Legal under Human Medicines Regulations 2012 Reg 167
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: MHRA Unlicensed Medicines Guidance
UK Early Access to Medicines Scheme (EAMS) 90 days

EAMS gives patients with life-threatening or seriously debilitating conditions access to medicines that don't yet have a marketing authorisation.

Application Form: EAMS Application
Legal Basis: Regulated
Eligibility: Serious or life-threatening condition with unmet medical need.
Reference: MHRA EAMS Guidance

Drugs Available (230)

DrugAccess RouteStatusLead Time
alglucosidase alfa———
cytarabine———
Ataluren———
eliglustat———
furosemide———
eribulin mesylate———
Digoxin Immune Fab———
Sucralfate———
Dorzolamide———
Pravastatin———
Temsirolimus———
Memantine———
propranolol———
Berotralstat———
Everolimus———
Ursodeoxycholic acid———
tamoxifen citrate———
atropine sulfate———
mitapivat———
metoprolol———
carbamazepine———
ruxolitinib———
Corticosteroids———
Melphalan———
mycophenolate mofetil———
Epoprostenol———
Fludarabine———
Sodium Phenylbutyrate———
etanercept———
Denosumab———
dapsone———
Tofacitinib———
mavacamten———
mannose———
minoxidil———
Hydroxychloroquine———
cyclophosphamide———
Anakinra———
bosentan———
bimatoprost———
testosterone———
Artificial tears———
Rho(D) immune globulin———
Baclofen———
Dexrazoxane———
Vigabatrin———
Clobazam———
triheptanoin———
C1 esterase inhibitor (human)———
loncastuximab tesirine———
phacoemulsification surgery———
fosdenopterin———
melatonin———
amiodarone———
Total Parenteral Nutrition———
Growth hormone (somatropin)———
ascorbic acid———
hormone replacement therapy (estrogen/progesterone)———
Busulfan———
Digoxin———
Bimekizumab-bkzx———
Interferon-gamma———
Romidepsin———
Crizotinib———
physical therapy and orthotic management———
Retinoids (Tretinoin)———
Branched-Chain Amino Acids———
eculizumab———
miglustat———
lonafarnib———
Betaine anhydrous———
tizanidine———
Octreotide———
oxybutynin———
Valproic Acid———
tocilizumab———
sclerostin antibody———
Immunoglobulin———
Mesalamine———
alpha-L-iduronidase———
L-DOPA———
Intravenous Immunoglobulin (IVIG)———
Phenytoin———
Bisphosphonates (pamidronate)———
Metronidazole———
Nitisinone———
Human Chorionic Gonadotropin (hCG)———
Lomitapide———
treprostinil———
sirolimus———
temozolomide———
Carnitine———
brivaracetam———
Pembrolizumab———
Infliximab———
Pyridoxine———
Cibinqo———
vutrisiran———
Clomiphene Citrate———
Angiotensin-Converting Enzyme Inhibitors (Enalapril)———
idebenone———
Lorenzo's oil———
ivermectin———
botulinum toxin type A———
mexiletine———
Crizanlizumab———
azathioprine———
sunscreen (broad spectrum SPF 50+)———
Somatropin———
galantamine———
ambrisentan———
Deflazacort———
Brentuximab vedotin———
Dabrafenib———
L-carnitine———
luspatercept———
trabectedin———
eplontersen———
vamorolone———
vosoritide———
Arsenic trioxide———
cysteamine bitartrate———
rituximab———
Dapagliflozin———
allopurinol———
Carboplatin———
Topical corticosteroids———
Trametinib———
estradiol———
Erythromycin———
Erythropoietin———
coenzyme Q10———
Patisiran———
eladocagene exuparvovec———
Tolvaptan———
cerliponase alfa———
avalglucosidase alfa———
atidarsagene autotemcel———
Sodium Thiosulfate———
adalimumab———
Imiglucerase———
Amphotericin B liposomal———
ranibizumab———
Midostaurin———
Lumasiran———
Methotrexate———
carglumic acid———
Alendronate Sodium———
calcitriol———
doxorubicin———
Pyridostigmine———
Levetiracetam———
Cenegermin———
Acitretin———
prednisone———
efgartigimod alfa———
Hydroxyurea———
salicylic acid———
cochlear implant———
Pyridostigmine Bromide———
aflibercept———
ravulizumab———
laronidase———
Romiplostim———
Bleomycin———
hydroxycobalamin———
Metreleptin———
Laser photocoagulation therapy———
pregabalin———
riboflavin———
N-acetylcysteine———
NovoSeven———
Occupational and Physical Therapy Support———
Pamidronate———
nitroglycerin———
ezetimibe———
Belzutifan———
bevacizumab———
Ketoconazole———
mecasermin———
celecoxib———
Biotin———
Imiquimod———
cyclosporine ophthalmic———
Growth Hormone———
Gonadotropin———
deferasirox———
Diatrizoate———
Tafamidis———
urea———
Levodopa/Carbidopa———
Lanreotide———
cyanocobalamin———
Sodium Bicarbonate———
diazoxide———
baricitinib———
Azacitidine———
Cystagon———
Bezafibrate———
Zoledronic Acid———
Hypertonic saline———
copper histidinate———
Clonazepam———
Zidovudine———
emollients with keratolytics———
cornstarch———
disopyramide———
Vinblastine———
selumetinib———
Sunitinib———
mannose-1-phosphate———
Cisplatin———
Calcipotriol———
Imatinib———
imatinib mesylate———
nimodipine———
Colchicine———
Follitropin alfa———
surgical repair———
Tamoxifen———
Tacrolimus———
Artificial tears (Hypromellose)———
Ribavirin———
Sildenafil———
NusinersenEAMSAvailable via EAMS10 days
sotalol———
Hematopoietic stem cell transplantation———
Tretinoin———
Timolol maleate———
Amnioreduction———
GB
United Kingdom
ISO CodeGB
RegionEurope
Regulatory AuthorityMHRA (Medicines and Healthcare products Regulatory Agency)
Pathways3 documented
Drugs Available230 drug(s)