This document sets forth the policy of Eloxx Pharmaceuticals, Inc. (“Eloxx” or “Company”) with respect to the consideration of requests for use of the Company’s investigational drugs outside of Company-sponsored clinical trials (“Expanded Access”).
It is the goal of Eloxx to provide potentially life-saving therapies to patients who might benefit from them while not exposing such patients to undue risk. Eloxx believes that, prior to approval of a therapeutic product, a patient and the Company would be best served if the patient sought access to the product as part of a clinical trial, because clinical trials can generate data that may lead to the approval of new medicines and, consequently, to wider availability for patients. Under certain circumstances, however, Eloxx will assess requests for access to unapproved products based on considerations set forth in this policy.
This policy applies to those employees of Eloxx charged with evaluation of Expanded Access requests for the Company’s investigational drugs.
This policy is not a guarantee of access to any specific investigational drug by any individual patient. The decision to approve an Expanded Access request is at the sole discretion of Eloxx, which shall have no liability in approving or denying such requests. This policy may be revised by Eloxx at any time.
- Eloxx Expanded Access Committee – Eloxx will establish a committee to review all Expanded Access requests. Eloxx, in its sole discretion, shall determine the appropriate personnel to serve on such committee, which may include, but not be limited to, representatives from Medical Affairs, Clinical Operations, Legal and Regulatory Affairs.
- Patient – Consults with licensed physician to explore and decide about alternative options.
- Licensed Physician – Agrees to oversee the patient’s treatment and works with industry (e.g., medical product developer), files paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting.
- Investigational Review Board (IRB)Reviews expanded access protocol and informed consent to ensure that the patient understands the nature of the investigational drug and the proposed treatment.
5. Key Principles and Considerations
Consistent with guidance provided by the US Food and Drug Administration (FDA), Expanded Access to an unapproved, investigational, therapeutic product may be appropriate when all the following apply:
- The patient must have a serious or immediately life-threatening disease or condition.
- There must be an unmet medical need, alternative therapies are not available or such alternative therapies have been exhausted.
- The patient is not eligible or cannot participate in a clinical trial. In assessing the eligibility of a patient for potential pre-approval access, preference will be given to providing the investigational medicine for use in clinical trials, then pre-approval access programs, and then single patient access.
- Sufficient scientific evidence demonstrating that the possible benefits of the investigational medicine outweigh the possible risks to the patient and that such risks are not unreasonable given the disease or condition to be treated.
- Providing pre-approval access will not jeopardize the initiation, conduct or completion of clinical investigations and the overall development program to support registration of the product or otherwise compromise its potential development.
- Requisite submissions are made to FDA and FDA determines that the Expanded Access request may proceed.
At Eloxx, the following principles are adhered to when and if the Company provides pre-approval access to investigational medicines through an Expanded Access program:
- All requests for pre-approval access are considered in a fair and just manner;
- At whatever stage of clinical development, a sufficient understanding of the potential benefits and risks of the investigational medicine has been established to support the consideration of the request;
- Patients are not put at risk of unnecessary harm;
- Fulfillment of pre-approval access will not jeopardize the development program that may lead to broader public access through marketing approval;
- Fulfillment of pre-approval access fully complies with applicable laws and regulations; and
- The treating physician making the request is licensed and qualified to administer the investigational medicine and agrees to comply with Company requirements, including providing regular reports to the Company, and comply with local regulations governing pre-approval access and all applicable laws and regulations.
In determining whether access is appropriate outside of a clinical trial, Eloxx will evaluate each request based on the following general criteria:
- The investigational therapy must be part of an ongoing clinical development program approved by a regulatory or health authority. Once a therapy is approved to treat a particular disease, it will no longer be available for pre-approval access to treat that disease;
- Providing access to the investigational therapy must not interfere with the completion of a clinical trial supportive of regulatory approval or compromise the potential development of the therapy or otherwise negatively impact the Company or any of its research and development activities (as determined by the Company);
- There are no beneficial alternative treatments available (including participation in ongoing relevant clinical trials);
- The efficacy and safety data available at the time are sufficient to determine whether the potential clinical benefit to the patient would likely outweigh the possible risks, including the outcome of the disease;
- The intent for use is limited to providing the patient with a clinical benefit in the treatment of the underlying disease, not a symptom or comorbidity of the disease, unless such symptom or comorbidity is itself serious, debilitating or life-threatening;
- A clinical study is not available, or the patient is not eligible (i.e. does not qualify);
- Patients with underlying medical conditions that may pose safety risks that have not been sufficiently characterized/studied would not be eligible to participate;
- An appropriate dose and/or treatment regimen for the investigational therapy has been determined, or proposed, for the disease for which treatment is sought;
- There is manufacturing capacity for supply of the investigational therapy;
- Relevant safety and other data (as determined by the Company) from a Phase 2 clinical study have been reviewed by the appropriate safety review or similar committee;
- Relevant patient translational data or other evidence showing potential positive activity or outcome (as determined by the Company) has been provided;
- Access is compliant with all applicable laws and regulations;
- The treating physician and the patient (and the patient’s family or custodians) agree to waive claims for damages against the Company;
- Health Authority and/or Institutional Review Board/Ethic Committee approval is obtained (when required); and
- Additional criteria regarding the therapy, its development program, the patient, or other circumstances may impact eligibility.
6. Process for Requesting Access
- The request for access to an investigational drug must be initiated by the patient’s treating physician. The physician may contact Eloxx to determine whether an Expanded Access program has already been established; if not, the physician may make a proposal for such a program, including the scientific rationale for treatment of the patient.
- The physician may request a Letter of Authorization (LOA) to allow the physician to reference the Eloxx IND in support of an Expanded Access IND.
- The physician must complete FDA Form 3926, Individual Patient Expanded Access Investigational New Drug Application (IND), which may be found at https://navigator.reaganudall.org/sites/default/files/newForm%20FDA%203926_1.pdfThe completed Form should be provided to Eloxx for review prior to submission to FDA.
When the form has been satisfactorily completed and submitted to Eloxx, the Company will seek to provide acknowledgement of receipt within three business days.
- Eloxx will review each request on a case by case basis and will typically make a decision to grant or deny the request or ask for more information within ten business days. The decision to grant access is solely at Eloxx’s discretion.
In carrying out this policy, Eloxx will comply with all applicable laws and regulations, as well as ethical and safety industry guidelines.
8. For Further Information
For further information or to submit an Expanded Access request, please contact:
By Mail: Eloxx Expanded Access Committee
c/o Eloxx Pharmaceuticals, Inc.
950 Winter Street
Waltham, MA 02451
By E-mail: EAC@eloxxpharma.com (please note that requestor must verify receipt; do not send personally identifiable or confidential information to this e-mail address)
Additional information about Eloxx’s clinical trials is available at www.Eloxxpharma.com and on the NIH’s ClinicalTrials.gov website.
Effective: October 3, 2019