General Policy Statement
Occasionally, patients with serious or life-threatening diseases or conditions seek access to investigational medicines outside of a clinical study setting. The associated provision for requesting access to an investigational medicine is called ‘Expanded Access’ but is also known by other terms such as ‘Compassionate Use’, ‘Early Access’, and ‘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 Epizyme 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. This Policy is applicable globally and across all therapeutic areas. Further information about Epizyme’s clinical trials is available at Clinicaltrials.gov.
Guiding Principles for Epizyme 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, Epizyme may be able to provide access under certain defined conditions through an established, company-planned Expanded Access Program, accessible by sending an email to ExpandedAccess@epizyme.com These conditions include:
- Sufficient clinical 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 serious or life-threatening diseases or conditions, or whose life is immediately threatened by their disease or condition;
- have no appropriate, comparable, or satisfactory alternative treatment available, or such alternatives have been tried without clinical success;
- be unable to participate in a clinical trial for the disease or condition; and
- be considered if 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
- 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
Guiding Principles for Individual Access: This Policy is designed to guide fair and equitable access decisions while maintaining Epizyme’s obligation to carry out the clinical development programs necessary to fully assess safety and efficacy of our investigational agents. The guiding principles for granting access to individuals outside the clinical development programs built on these Guiding Principles for Expanded Access Programs (see above) and include:
- Whether sufficient clinical data exist for a particular disease or condition to anticipate that the potential benefits outweigh the potential risks of the investigational medicine for that patient;
- Whether the granting of an individual request, given the current epidemiologic understanding of the number of patients who would have a similar clinical profile, would unduly burden the company with a magnitude of similar requests which should, by principles of justice and fairness, be provided to these other patients;
- Whether it is feasible for the Company to provide the required access for the duration of the patient’s requirements once any access is provided
The Company should evaluate, prior to the initiation of a program and periodically throughout a program, given the number of individual requests, whether a group or protocol-driven cohort program should be initiated.
Right to Try Requests: Epizyme is committed to helping patients with serious and life-threatening illnesses and their families request access to our investigational medicines. We may be able to support these requests by evaluating them through the processes and standards established by this Expanded Access Policy.
Closing of Expanded Access Program: The Company 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;
- Limited product supply or other manufacturing issues.