Access pathways
50 regulatory frameworks governing patient access to specialty and unregistered medicines.
Named Patient Program (NPP)
The foundational pathway for providing individual patients access to drugs not yet registered in their country.
Compassionate Use
A regulatory pathway allowing seriously ill patients access to investigational or unregistered drugs.
Expanded Access Program (EAP)
Structured programs offering access to investigational drugs outside of clinical trials.
Right to Try
US federal legislation (2018) providing an alternative pathway to investigational drugs.
Managed Access Program
Manufacturer-operated access programs with structured eligibility and oversight.
Early Access to Medicines Scheme
UK MHRA's pathway for early access to promising unlicensed medicines.
Personal Import Scheme
Country-specific personal-use import pathways for unregistered drugs.
Special Access Scheme
Australia's TGA-operated pathway for unapproved therapeutic goods.
Section 21 (South Africa)
South Africa's SAHPRA pathway for access to unregistered medicines.
Section 29 (New Zealand)
New Zealand's Medsafe pathway for unapproved medicines.
Cohort Named Patient Program
Structured NPP for groups of patients sharing a common indication.
Physician-Initiated Access Request
Provider-driven request for access to unregistered or investigational drugs.
Hospital Exemption (EU)
EU pathway for hospital-specific advanced-therapy medicinal products.
ATU (France — nominative)
France's Authorization for Temporary Use, nominative form.
ATU (France — cohort)
France's Authorization for Temporary Use, cohort form.
Sonderzulassung (Germany)
Germany's special-authorization pathway for unapproved drugs.
Uso Compassivo (Italy)
Italy's compassionate-use pathway.
Uso Compasivo (Spain)
Spain's compassionate-use pathway.
GVHO-Pathway (Netherlands)
Netherlands' pathway for group or individual compassionate access.
Compassionate Use Nordic
Denmark / Sweden / Norway / Finland compassionate-use frameworks.
Individual Patient Supply (UK MHRA)
UK's MHRA individual-patient unlicensed-medicine supply.
Personal Use Import (FDA)
US FDA personal-use import framework for drugs abroad.
Hardship Program
Manufacturer program for patients without alternatives.
Single Patient IND
US FDA single-patient Investigational New Drug authorization.
Intermediate-Size Expanded Access
US FDA framework for groups of patients.
Treatment IND
US FDA treatment Investigational New Drug category.
Rule 36 India Import
India's personal-use import pathway under Rule 36 of D&C Rules.
Hospital Ethics Committee Approval
Institutional pathway requiring IEC or IRB approval.
Humanitarian Use Device
US FDA pathway for humanitarian-use devices and combination products.
Priority Review Voucher Program
Incentive program producing PRVs that accelerate review timelines.
Breakthrough Therapy Designation
FDA designation that accelerates development and review.
Accelerated Approval Program
FDA pathway for serious conditions using surrogate endpoints.
Orphan Drug Designation
Regulatory designation for drugs treating rare diseases.
Rare Pediatric Disease Designation
FDA designation for serious/life-threatening pediatric rare diseases.
Conditional Marketing Authorization (EMA)
EMA pathway for drugs addressing unmet medical need.
Marketing Authorization Under Exceptional Circumstances (EMA)
EMA pathway for drugs where comprehensive data is unobtainable.
PRIME (EMA)
EMA's PRIority MEdicines scheme.
Sakigake Designation (Japan)
Japan PMDA's breakthrough-equivalent designation.
Notice of Compliance with Conditions (Canada)
Health Canada's conditional approval pathway.
Project Orbis (FDA international)
Cross-border oncology review program.
Article 83 (EMA cohort compassionate)
EU legal framework for cohort compassionate-use programs.
Pre-approval Access Hub
Industry-standard framework for pre-approval access programs.
Hospital-Based Specialty Pharmacy Supply
Institutional supply channel for high-cost specialty drugs.
Cross-Border Prescription Coordination
Physician-to-physician coordination across borders.
Registry-Linked Access
Access tied to patient registry enrollment for real-world evidence.
Diagnostic-Linked Access
Access predicated on companion-diagnostic confirmation.
Pay-for-Performance Access
Outcomes-based pricing / access arrangements.
Pharmacy Compounding Exception
Compounding exceptions for drugs not commercially available.
Medical Tourism Access
Patients travelling to countries where drug is registered.
Named Patient Import via IOR
Importer-of-Record structures for cross-border NPP.