Expanded Access Policy
Introduction
JCR Pharmaceuticals believes that access to non-approved, investigational drugs is best given
through a clinical trial. We understand that there may be circumstances under which a patientdoes not qualify for participation in a clinical trial. Under such circumstances JCR
Pharmaceuticals will carefully consider provision of access to investigational drugs to individual
patients on a case-by-case basis.
Patients need to understand that treatment with an investigational drug on an individual basis
outside of a clinical trial involves risks as the safety profile of investigational drugs is not fullyestablished.
This document outlines the criteria under which JCR Pharmaceuticals considers providing
access to investigational drugs via an Expanded Access (Compassionate Use) Program under
US FDA guidelines, the factors we take into consideration when evaluating the request and theprocedures to request Expanded Access.
General Criteria:
Single-patient Expanded Access should only be considered if a patient is seriously ill and has
exhausted approved treatment options and there is no option to participate in an ongoing clinical
trial or soon to open clinical trial. Geographic limitations to participate in such a trial may not bea criterion to grant Expanded Access to an investigational drug.
Sufficient medical and scientific rationale needs to exist to suggest that the mechanism of actionof the investigational drug may have a positive effect on the patient’s disease burden, signs andsymptoms, or prognosis.
The potential benefit to the patient seeking access to the investigational medicine must
outweigh the potential risks to the patient receiving the investigational drug. This includes
outcome of the disease itself.
Specific criteria related to the investigational drug
The investigational drug must have completed phase I clinical studies at a minimum to assure apreliminary basic understanding of and confidence in the investigational drug’s safety profile andpotential risks associated with treatment. The drug must be in active clinical development for its
intended indication and early signs of efficacy in humans should have been reported prior toconsidering Expanded Access.
There may be underlying safety risks specific to a particular patient that may preclude him/herfrom access to the investigational drug.
Sufficient data must be available to have a basic understanding of the dosing frequency andstrength for the patient requesting Expanded Access.
There may be only limited supply of an investigational product available. Access to an
investigational medicine must not interfere with the completion of ongoing or planned clinical
trials or otherwise compromise the development of the investigational drug.

JCR Pharmaceuticals Co., Ltd.

Expanded Access Policy
The treating physician must have access to a facility where the drug can be safely prepared foradministration (e.g. dilution into an infusion bag) and safely administered to the patient. JCR
Pharmaceuticals does not provide materials required to safely administer the drug to the patient.
JCR Pharmaceuticals does not provide support for additional clinical testing, routine analyses,
diagnostic or pathological testing to ensure the safety of the drug or to evaluate the benefit/risk
ratio under compassionate use.
What patients should consider before requesting Expanded Access to an investigationaldrug
The physician and the patient need to understand that Expanded Access may only bepossible/granted only for a certain time period, e.g. when there is only limited supply of aninvestigational drug available or additional trials open for which the patient would qualify. Oncethe investigational drug is approved by the regulatory authorities for the indication the patient isdiagnosed with, Expanded Access will no longer be available.
New evidence from clinical trials may arise that could affect the safety profile or efficacy of aninvestigational drug or challenge the benefit/risk ratio under certain conditions. Under suchcircumstances, JCR Pharmaceuticals reserves the right to withdraw its permission forcompassionate use even if it was granted before.
How JCR Pharmaceuticals evaluates Expanded Access requests
JCR Pharmaceuticals takes every request for Expanded Access seriously and will decide on an
individual basis and in close communication with the treating physician. Some guiding questionsin the decision process include:
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Is the patient suffering from a serious or life-threatening illness?
Is the patient eligible to enroll and able to participate in a clinical trial?
Has the patient exhausted approved therapeutic options?
Is the patient’s medical diagnosis/status appropriate for the investigational treatment?
Is the requested investigational treatment approved for any indication in the country
concerned?
Does the company have good confidence in the safety of the drug?
Are clinical data available to suggest that the potential benefits for this patient outweigh
the risks of the investigational treatment?
Is the physician appropriately licensed and qualified?
Is the company able to supply the investigational treatment?
Will providing access on a single-patient basis negatively affect the company’s ability tocomplete its clinical trials?

JCR Pharmaceuticals always strives to make decisions as transparently as possible and does
this in close communication with the treating physician and our internal team. The patient needs
to understand that the final decision is with the company providing the drug. There may becircumstances under which Expanded Access may not be possible despite a positive opinion on
all of the above criteria.

JCR Pharmaceuticals Co., Ltd.

Expanded Access Policy
Requesting access to investigational drugs via Expanded Access
General information
Requests for Expanded Access should be made by the patient’s treating physician. Treating
physicians are asked not to submit personal information (e.g. subject names) covered asprotected health information in the initial request for Expanded Access.
The following information should be provided at the initial request:
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Contact details for key point of contact.
The specific medical condition for which the physician is inquiring.
Existing diagnostic information in support of the medical condition.
The general medical status of the patient for whom Expanded Access is requested.
Additional questions may be asked to further clarify eligibility.
The place of residence of the patient and general ability to travel to the treatment center.
A brief explanation by the treating physician that he/she is able to administer aninvestigational drug in an environment that ensures the safety of the patient.

The process
The process and the conditions for Expanded Access may differ from country to country. Below
we describe the process for requesting Expanded Access under US FDA guidelines:
After JCR Pharmaceuticals has positively decided on granting compassionate use access, inthe United States, the FDA and an appropriate Institutional Review Board (IRB) must review
and approve the use of the investigational drug to ensure that the patient or legal representative
understand the potential risks associated with treatment using investigational drugs.
Once JCR Pharmaceuticals and the IRB agree to the treatment under Expanded Accessprotocol, JCR Pharmaceuticals will issue a Letter of Authorization to the treating physician,allowing the physician to cross-reference the IND for the investigational drug. The physician
then requests permission to proceed from the FDA by submitting form 3926. This can be done
online (Expanded Access eRequest). Further information is found at https://www.fda.gov/newsevents/expanded-access/expanded-access-how-submit-request-forms
Once the FDA grants permission, the physician informs JCR Pharmaceuticals and provides
appropriate documentation. JCR Pharmaceuticals will then initiate the shipment of drug to thetreatment center.
For additional information on compassionate use access or to submit requests, please contact:ir-info@jp.jcrpharm.com

JCR Pharmaceuticals Co., Ltd.