약학회지
YAKHAK HOEJI
ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)

Table. 1.

Table. 1.

Programs and regulations related to pre-approval access to investigational drugs for treatment use

Category United States Australia United Kingdom Republic of Korea
Regulatory authority Food and Drug Administration, FDA Therapeutic Goods Administration, TGA Medicines and Healthcare Products
Regulatory Agency, MHRA
Ministry of Food and Drug Safety, MFDS
Program • Expanded Access
- Individual patients
- Intermediate-size patient
- Treatment IND or Treatment protocol
• Right to Try (RTT) Access • Special Access Scheme (SAS) • Authorized Prescribers Scheme (AP) • Early Access to Medicines Scheme (EAMS) • Use of Investigational Products for Treatment Purpose
- Individual patients
- Two or more patients
Responsibility • Investigatora
• Sponsor
• An individual or entity that submits an expanded access IND or protocol
• Sponsor
• Manufacturer
• Prescribing practitioners • HRECs and specialist colleTGA
• Sponsors
• MHRA
• NICE
• NHSE
• EAMS applicants
• Individual patients: physicianb
• Expedited approval: Investigatorc
• Two of more patients: pharmaceutical company
Act/Regulation • 「Code of Federal Regulations (CFR)」Title 21, Part 312. INVESTIGATIONAL NEW DRUG APPLICATION, Subpart I. Expanded Access to Investigational Drugs for Treatment Use • 「Right to Try Act」 •「Therapeutic Goods Act 1989 (the Act)」 19(7A), 41HC(6), 32CM(7A)
• 「Therapeutic Goods Regulations 1990」12A
• Therapeutic Goods Rules
- Authorised Supply of Specified Medicines
- Authorised Supply of Specified Medical Devices
- Authorised Supply of Specified Biologicals
(None) • 「Pharmaceutical Affairs Act」
- Article 34 (Approval for Clinical Trial Protocols)
• 「Regulation on Safety of Pharmaceuticals, etc.」
- Article 28 (Application for Permission for Use of Investigational Products for Treatment Purpose)
- Article 29 (Billing for Approval for Use of Investigational Products for Treatment Purpose)

aA licensed physician under whose immediate direction an investigational drug is administered or dispensed for an expanded access use

bSpecialist with specialized knowledge and experience on the disease and investigational drug

cSpecialist with experience in using investigational drugs by participating in the clinical trial

Yakhak Hoeji 2021;65:441-52 https://doi.org/10.17480/psk.2021.65.6.441
© 2021 Yakhak Hoeji