OrphanDrug
OrphanDrug
Pharma Knowledge Base
IT

Italy

IT

Europe

Contents

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

Regulatory Overview

Italy is regulated by the AIFA (Agenzia Italiana del Farmaco). 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

AIFA (Agenzia Italiana del Farmaco)

Regulatory Pathways (3)

Italy Compassionate Use (Article 83 EMA) 30 days

EMA Article 83 compassionate use framework allowing access to unauthorised medicinal products for groups of patients with a chronically or seriously debilitating or life-threatening disease in Italy.

Application Form: National compassionate use form
Legal Basis: Regulated under EU Directive 2001/83/EC
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: EMA Article 83 Compassionate Use
Uso Compassionevole – Italy (DM 7 settembre 2017) 45 days

Italian compassionate use under Ministerial Decree of 7 September 2017 for individual patients with serious, rare or life-threatening diseases.

Application Form: AIFA Compassionate Use Application
Legal Basis: Decreto Ministeriale 7 settembre 2017
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: AIFA DM 2017 Compassionate Use
Legge 648/96 – Italy Off-Label Reimbursement 45 days

Italian Law 648/96 allows off-label reimbursement of medicines not yet authorised in Italy for rare diseases or conditions where no valid alternative exists.

Application Form: AIFA Legge 648 Application
Legal Basis: Legge 648/96
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: AIFA Legge 648/96 Guidance

Drugs Available (106)

DrugAccess RouteStatusLead Time
cytarabine———
Ataluren———
eribulin mesylate———
Sucralfate———
Memantine———
propranolol———
Ursodeoxycholic acid———
tamoxifen citrate———
mitapivat———
carbamazepine———
ruxolitinib———
mycophenolate mofetil———
Epoprostenol———
Fludarabine———
etanercept———
dapsone———
mavacamten———
cyclophosphamide———
bosentan———
testosterone———
Rho(D) immune globulin———
triheptanoin———
melatonin———
amiodarone———
Total Parenteral Nutrition———
ascorbic acid———
hormone replacement therapy (estrogen/progesterone)———
Interferon-gamma———
physical therapy and orthotic management———
Retinoids (Tretinoin)———
eculizumab———
miglustat———
lonafarnib———
Betaine anhydrous———
Octreotide———
oxybutynin———
Valproic Acid———
tocilizumab———
Immunoglobulin———
alpha-L-iduronidase———
sirolimus———
temozolomide———
Carnitine———
brivaracetam———
Angiotensin-Converting Enzyme Inhibitors (Enalapril)———
idebenone———
botulinum toxin type A———
azathioprine———
L-carnitine———
luspatercept———
trabectedin———
vosoritide———
cysteamine bitartrate———
rituximab———
Dapagliflozin———
allopurinol———
Carboplatin———
Topical corticosteroids———
estradiol———
eladocagene exuparvovec———
Tolvaptan———
cerliponase alfa———
avalglucosidase alfa———
atidarsagene autotemcel———
adalimumab———
tacrolimus ophthalmic———
Amphotericin B liposomal———
ranibizumab———
diacerein———
carglumic acid———
calcitriol———
doxorubicin———
Pyridostigmine———
Levetiracetam———
Acitretin———
prednisone———
aflibercept———
laronidase———
Bleomycin———
hydroxycobalamin———
Metreleptin———
pregabalin———
N-acetylcysteine———
Pamidronate———
Diosmin———
ezetimibe———
bevacizumab———
mecasermin———
celecoxib———
Imiquimod———
Growth Hormone———
deferasirox———
Tafamidis———
Levodopa/Carbidopa———
Lanreotide———
baricitinib———
Azacitidine———
Zoledronic Acid———
cornstarch———
disopyramide———
Cisplatin———
imatinib mesylate———
nimodipine———
Colchicine———
surgical repair———
Hematopoietic stem cell transplantation———
IT
Italy
ISO CodeIT
RegionEurope
Regulatory AuthorityAIFA (Agenzia Italiana del Farmaco)
Pathways3 documented
Drugs Available106 drug(s)