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

Table. 2.

Table. 2.

Requirements and eligible patients

Category United States Australia United Kingdom Republic of Korea
Requirements/Criteria Expanded Access
• There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition
• Patient enrollment in a clinical trial is not possible
• Potential patient benefit justifies the potential risks of treatment
• Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication

Right to Try
• For which a Phase 1 clinical trial has been completed
• That has not been approved or licensed by the FDA for any use
• For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA
• Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA
• Health practitioner must consider PRIOR to attempting to access any unapproved good via the SAS:
- All ‘approved’ therapeutic goods (those registered, listed or included on the ARTG) have been considered and found clinically unsuitable
- The specific unapproved good you intend to access is NOT substantially similar to any good on the ARTG or if substantially similar, the approved therapeutic good is NOT currently marketed ('availablé) in Australia

• Category A: notification for a patient defined as seriously ill - Patient is at risk of imminent or early death without treatment
• Category B: an application pathway that can be accessed by health practitioners if the patient does not fit the Category A definition and if the therapeutic good is not authorised for supply under the SAS Category C notification pathway.
• Category C: notification of use of specified therapeutic goods that are deemed to have an established history of use
1. PIM designation criteria
1) a. The condition should be:
• Life-threatening or
• Seriously debilitating
b. With high unmet need:
• There is no method of treatment, diagnosis or prevention available or
• Existing methods have serious limitations
2) The medicinal product is likely to offer major advantage over methods currently used in the UK
3) The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance

2. Positive scientific opinion criteria (same as above criteria 1-3)
4) The Applicant is able to supply the product and to manufacture it to a consistent quality standard (GMP)
• A person who intends to apply for use of investigational products to permission of use for treatment purpose shall submit to the Minister of MFDS the application for permission based on each size of the number of concerned patients
• Investigational drugs shall be those that have been reviewed by the MFDS for quality, toxic materials, etc. to the extent available for clinical trials

Treatment use for two or more patients
• A person who intends to apply for permission of use for treatment purpose for two or more patients shall submit the application including ‘plan for use’
• A person who intends to use investigational products provided to the clinical trial institution shall obtain consent from the concerned patients and approval from the clinical trial IRB
Eligible patients Expanded Access (CFR 21 §312.300)
• Immediately life-threatening disease or condition
• Serious disease or condition patients with serious diseases or conditions when there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the patient’s disease or condition.

Right to Try
• Been diagnosed with a life-threatening disease or condition
• Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug a
• And has provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician
• Patients with life threatening or seriously debilitating conditions
• Medicines that do not yet have a marketing authorisation when there is a clear unmet medical need
「Pharmaceutical Affairs Act」 Article 34 (4)
• Patient with a serious life-threatening disease, such as terminal cancer or AIDS
• An emergency patient prescribed by Ordinance of the Prime Minister, such as a patient whose life is threatened and a patient without alternative means of treatment

Treatment use for two or more patients
• Patients specified in patient inclusion criteria of ‘plan for use’

aThis must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying

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