Regulatory Pathways — Named Patient, Compassionate Use & Early Access

FDA Emergency IND – USA

Emergency Investigational New Drug application for life-threatening situations requiring immediate treatment where there is no time to submit a formal IND. FDA may authorize treatment by telephone.

Form: Emergency IND Request (telephone/fax)

<15d fast1d21 CFR 312.310

FDA Expanded Access – United States

The FDA expanded access pathway allows patients with serious or immediately life-threatening diseases to receive investigational drugs outside clinical trials when no comparable or satisfactory alternative therapy exists. Use requires that potential patient benefit justify the potential risks and that providing the drug will not interfere with clinical development. [4][5]

Form: FDA Form 3926 or sponsor/physician expanded access request materials

Statutory/Administrative

FDA Expanded Access (Compassionate Use) – USA

FDA program allowing patients with serious or immediately life-threatening conditions to access investigational drugs, biologics, or medical devices outside of clinical trials.

Form: FDA Form 3926 (Individual Patient IND)

15–45d medium30d21 CFR Part 312 Subpart I

FDA Ghana Special Access Program

Ghana Food and Drugs Authority special access program for unregistered medicines for patients with serious or life-threatening conditions.

Form: FDA Ghana Special Access Form

15–45d medium21dPublic Health Act, 2012 (Act 851)

FDA Orphan Drug Designation

US designation for drugs treating rare diseases affecting fewer than 200,000 people, granting 7-year market exclusivity, tax credits, and fee waivers.

Statutory

FDA Philippines Compassionate Special Permit (CSP)

FDA Philippines issues Compassionate Special Permits for unregistered drugs needed by individual patients with serious or life-threatening diseases who have no other treatment options.

Form: CSP Application Form

15–45d medium21dRA 9711 FDA Act 2009

FDA Thailand Special Import License

Thai Food and Drug Administration permits import of unapproved drugs under a special license for individual patients with serious or life-threatening conditions for whom no registered medicine is suitable.

Form: FDA Thailand Special Import Form

15–45d medium30dDrug Act B.E. 2510

Fiji Pharmacy and Poisons Board Special Import Authorization

Fiji Pharmacy and Poisons Board special import authorization for unregistered medicines for patients with serious medical conditions.

Form: Fiji PPB Special Import Form

15–45d medium21dPharmacy and Poisons Act (Fiji)

Finland Compassionate Use (Article 83 EMA)

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 Finland.

Form: National compassionate use form

15–45d medium30dRegulated under EU Directive 2001/83/EC

France Compassionate Use (Article 83 EMA)

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 France.

Form: National compassionate use form

15–45d medium30dRegulated under EU Directive 2001/83/EC

Gabon MOH Special Import Authorization

Gabon Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Gabon Special Import Form

15–45d medium45dRegulated

Gambia MOH Special Import Authorization

Gambia Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Gambia Special Import Form

15–45d medium45dRegulated

Georgia MOH Special Import Authorization

Georgia Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Georgia Special Import Form

15–45d medium30dRegulated

Germany Compassionate Use (Article 83 EMA)

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 Germany.

Form: National compassionate use form

15–45d medium30dRegulated under EU Directive 2001/83/EC

Greece Compassionate Use (Article 83 EMA)

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 Greece.

Form: National compassionate use form

15–45d medium30dRegulated under EU Directive 2001/83/EC

Grenada MOH Special Import Authorization

Grenada Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Grenada Special Import Form

15–45d medium45dRegulated

Guatemala MOH Named Patient Authorization

Guatemala Ministry of Health named patient authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Guatemala Named Patient Form

15–45d medium30dRegulated

Guinea MOH Special Import Authorization

Guinea Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Guinea Special Import Form

15–45d medium45dRegulated

Guinea-Bissau MOH Special Import Authorization

Guinea-Bissau Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Guinea-Bissau Special Import Form

15–45d medium45dRegulated

Guyana MOH Special Import Authorization

Guyana Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Guyana Special Import Form

15–45d medium45dRegulated

Haiti MOH Special Import Authorization

Haiti Ministry of Health special import authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Haiti Special Import Form

15–45d medium45dRegulated

Health Canada Special Access Program (SAP)

Health Canada's SAP allows manufacturers to sell drugs not approved in Canada to practitioners treating patients with serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable.

Form: HC/SC 3011 SAP Request Form

<15d fast5dSection C.08.010 Food and Drug Regulations

Honduras MOH Named Patient Authorization

Honduras Ministry of Health named patient authorization for unregistered medicines for patients with serious or life-threatening conditions.

Form: MOH Honduras Named Patient Form

15–45d medium45dRegulated

HSA Special Access Route (SAR) – Singapore

Singapore's Health Sciences Authority allows access to unregistered therapeutic products via the Special Access Route for individual patients with serious medical conditions, subject to physician application.

Form: HSA SAR Application Form

<15d fast7dHealth Products Act

Hungary Compassionate Use (Article 83 EMA)

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 Hungary.

Form: National compassionate use form

15–45d medium30dRegulated under EU Directive 2001/83/EC