Expanded Access Policy
Poxel’s mission is to develop innovative treatments for chronic serious diseases with metabolic pathophysiology. Consistent with its mission, Poxel is focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approval.
The best way to see if a new medicine is sufficiently safe and effective is to conduct clinical studies. As such Poxel prefers that, where possible, patients participate in clinical studies. Poxel recognizes however that not everyone has this opportunity. Poxel is committed to making investigational products available to seriously ill patients who have exhausted other treatment options and unable to participate in a clinical study. In accordance with FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND) (21 CFR part 312, subpart I), a treating physician in the United States (U.S.), who is able to comply with the requirements that are stated in this policy, may request information about how to apply for access to Poxel’s investigational products by contacting the Company.
This policy applies to provision of potential Expanded Access to a Poxel investigational product(s) for patients with serious disease who have exhausted other treatment options and are not eligible or able to join in a clinical trial and for product(s) that are not approved for any purpose in the U.S.
Any use of a Poxel investigational product outside a clinical trial in the U.S. must (i) comply with local laws and regulations, including Poxel policies and procedures, and (ii) meet certain conditions including the ones detailed below.
In general, the investigational product supplied via Expanded Access may no longer be provided by Poxel when it becomes commercially available.
Poxel may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the U.S.
This policy is subject to change. Poxel will revisit the policy periodically and amend it as appropriate. Individuals with questions regarding this policy can contact Poxel by email at: email@example.com
Patient Eligibility Criteria
To be eligible for access to an investigational product, a patient must meet the following criteria:
- Be an adult male with a confirmed diagnosis of X-linked adrenoleukodystrophy.
- Suffer from a serious or immediately life-threatening disease or condition.
- Have undergone standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
- Are not eligible or able to participate in any planned or ongoing clinical trial of the investigational product(s).
- Has manifestations of disease for which there is sufficient evidence of a projected benefit (e.g. based on prior positive clinical trial results) from the use of the investigational product and the benefit outweighs the known or anticipated risks.
- Any other pertinent medical criteria for access to the investigational product, as established by Poxel.
Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
- The product is in active clinical development.
- There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
- The provision of the investigational product will not interfere with or compromise the clinical development of the product, and that there is sufficient investigational product available to ensure that supply constraints will not impede continued use of the product(s) by patient(s) or the conduct of our clinical trials.
Treating Physician Criteria and Responsibilities
The physician(s) attending to the patient(s) who is/are receiving an investigational product through expanded use access is (are) properly licensed in the U.S. and fully qualified to administer the product. The physician must agree in writing to comply with:
- Any applicable legal and regulatory requirements related to providing an investigational product under Expanded Access.
- Any Poxel requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.
A treating physician may submit questions or requests regarding expanded access to firstname.lastname@example.org. Acknowledgement of a request will occur within 60 days.