Expanded Access Policy
General Policy Statement
The US Food and Drug Administration (FDA) has established criteria for the provision of investigational medicines to patients outside of clinical trials, called “Expanded Access”, “Compassionate Use”, “Early Access”, or “Emergency Use”. Patients who seek access to investigational medicines outside of an established clinical study and prior to health authority marketing authorization may wish to do so because standard treatments have failed, they cannot tolerate already approved medicines, because they are unable to participate in a clinical study, or because there are no comparable or satisfactory therapy options available outside of clinical trials.
The policy below sets forth the general guidelines that Cartesian Therapeutics (Cartesian) will apply to its Expanded Access Program to ensure that requests for access to our investigational medicines are considered and processed in accordance with applicable legal and regulatory requirements, as well as in alignment with principles consistent with the Company’s policies and procedures. Further information about Cartesian’s clinical trials is available at Clinicaltrials.gov.
Cartesian Expanded Access Programs: The best route to enable the broadest number of patients to have access to a medicine for a particular condition is by making it commercially available through an established health authority approval process. Prior to health authority approval, clinical trials are the preferred mechanism by which patients would be able to obtain access to an investigational medicine due to the controlled environment and associated safeguards. However, there may be circumstances in which a patient may not be eligible to participate in a clinical trial. In these cases, Cartesian may be able to provide access under certain defined conditions through an established, company-planned Expanded Access Program, accessible by sending an email to EAP@cartesiantx.com. These conditions include:
- Sufficient data must exist for particular serious diseases or conditions to anticipate that the potential benefits outweigh the potential risks of the investigational medicine.
- Patients who may possibly obtain access must:
- have a serious or life-threatening disease or condition;
- have no appropriate, comparable, or satisfactory alternative treatment available, or have tried such alternatives without clinical success;
- be unable to participate in a clinical trial for the disease or condition; or
- be in a situation where the potential benefit justifies the potential risks of treatment.
- Providing access must not significantly jeopardize the clinical development program intended to support regulatory approval, registration, or commercialization of the investigational medicine. Development may be jeopardized if access could:
- interfere with the initiation, conduct, or completion of clinical trials, including participant availability and enrollment; or
- result in insufficient drug supply or the requisite medical and other internal expertise needed to support the clinical trials necessary for regulatory approval.
- The treating physician must be licensed and otherwise qualified to:
- safely administer the investigational medicine;
- comply with local regulations for expanded access; and
- monitor and report adverse events, as required by law and by company requirements.
Cartesian will acknowledge receipt of Expanded Access inquiries within five (5) business days of receipt.
Closing of Expanded Access Program: Cartesian may consider closing a program for various reasons, including but not limited to:
- Commercial availability of the medicine for a particular need or condition, at which point it would be more broadly available to these patients in need;
- A negative regulatory decision, or a Company decision not to proceed with and/or the discontinuation of clinical trials, or commercialization for a particular disease or condition. In this instance, patients on expanded access treatment at the time of such a decision may be allowed to continue to access therapy until disease progression;
- New information becomes available about the activity or safety of a medicine that could substantially change its benefit/risk profile; or
- Limited product supply or other manufacturing issues.
This policy is subject to change and is not intended to create or imply any contract. The posting of this policy by Cartesian shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.