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

Table. 4.

Table. 4.

Patient safety plan

Country Patient safety plan
United States Expanded Access
(1) All expanded access uses (CFR21 §312.305(c))
• Investigators
- Reporting adverse drug events to the sponsor
- Maintaining accurate case histories and drug disposition records and retaining records
• Sponsors
- Submitting IND safety reports and annual reports
- Ensuring that licensed physicians are qualified to administer the investigational drug for the expanded access use
- Providing licensed physicians with the information needed to minimize the risk and maximize the potential benefits of the investigational drug
- Maintaining an effective IND for the expanded access use
- Maintaining adequate drug disposition records and retaining records
(2) Individual patients, including for emergency use (§312.310(c))a
• At the conclusion of treatment, the licensed physician or sponsor must provide FDA with a written summary of the results of the expanded access use, including adverse effects
• FDA may require sponsors to monitor an individual patient expanded access use if the use is for an extended duration.
• When a significant number of similar individual patient expanded access requests have been submitted, FDA may ask the sponsor to submit an IND or protocol for the use under §312.315 or §312.320
(3) Intermediate-size patient populations (§312.315(d))
• Upon review of the IND annual report, FDA will determine whether it is appropriate for the expanded access to continue under this section.
• The sponsor is responsible for monitoring the expanded access protocol to ensure that licensed physicians comply with the protocol and the regulations applicable to investigators
(4) Treatment IND or Treatment protocol (§312.320(c))
• The sponsor is responsible for monitoring the treatment protocol to ensure that licensed physicians comply with the protocol and the regulations applicable to investigators
Right to Try (§561B(d))
• The manufacturer or sponsor of an eligible investigational drug shall submit to the Secretary an annual summary of any use of such drugb
• The Secretary shall post an annual summary report of the use of this section on the internet website of the Food and Drug Administration
Australia SAS
• The prescribing health practitioner must report the details of any adverse events or defects to TGA and the sponsor within 15 calendar days of becoming aware of them
• The sponsor is required to provide us with six-monthly reports detailing each kind of therapeutic good supplied, the number of times supplied to a health practitioner and the quantity supplied by the sponsor during the period to which the report relates
• The sponsors are responsible for continually monitoring the use of the therapeutic good you supply under the SAS, reporting the types of adverse reactions, effects and any defects, and recording the balance of its benefit and risk
AP
• Authorized Prescribers must monitor the patient during and after use of the ‘unapproved’ good, provide the TGA with a six monthly report, inform TGA of adverse events associated with use of the good
• HRECs and specialist colleges monitor the medical practitioner’s use of the ‘unapproved’ goods to ensure continued endorsement is appropriate
• Sponsors of ‘unapproved’ therapeutic goods inform TGA of emerging safety concerns associated with the use of ‘unapproved’ goods that they supply
United Kingdom • Periodic report/renewal of Scientific Opinionc
• To notify regulatory authorities of
- Any regulatory action taken worldwide during the reporting period
- Adverse events that have been received from the Registry within a pre-specified time period
- To flag any safety concerns which may require further characterisation or risk minimisation
- To flag any new data which may alter the benefit risk opinion
- To confirm maintenance of the EAMS criteria
Republic of Korea 「Regulation on Safety of Pharmaceuticals, etc.」 Article 28
• A person who has used an investigational product after obtaining the approval of use for treatment purpose shall provide the supplier of the investigational product with information that have been gathered after the use, such as adverse events observed in relevant patients, effectiveness, follow-up results on efficacy and safety, etc. without any delay after completion of its use
• The supplier of the investigational product shall prepare to submit the results of the investigational product used to the Minister of Ministry of Food and Drug Safety within 20 days after receipt of the information

aTreatment is generally limited to a single course of therapy for a specified duration unless FDA expressly authorizes multiple courses or chronic therapy

bThe number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events

cThe exact frequency of these updates will be agreed before the scientific opinion is issued but is likely to be every 3 months

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