Purpose and Scope
Clene is focused on the discovery and development of innovative, life-changing medicines for people with serious and life-threatening diseases. To do this, Clene conducts clinical studies to assess the safety and effectiveness of its investigational medicines. Clinical studies are controlled research studies in people designed to understand if an investigational medicine is safe and effective. Data generated by these studies provide health regulatory authorities with essential information needed to evaluate an investigational medicine. Only upon review of this information will regulators approve a new medicine, allowing it to become widely available.
Importantly, Clene believes that participating in clinical trials is the best way for patients to access investigational medicines prior to regulatory approval. In certain cases, when it is not possible for patients to participate in a clinical trial, a physician, hospital, or university may seek special access to a Clene investigational medicine outside of a clinical study. These requests are typically called ‘expanded access’, ‘early access’, ‘compassionate use’, ‘named patient use’, or ‘temporary authorization for use’. These requests are different from a clinical trial, where more comprehensive safety and efficacy data are collected.
Types of Expanded/Early Access Programs Offered
On a limited basis, Clene offers two different types of expanded/early access programs (collectively, hereinafter “EAP”) based on specific criteria described below:
(1) Multiple Patient EAP
If Clene believes it has sufficient scientific information to identify a population of patients for whom Expanded Access may be appropriate, Clene may establish an EAP program for a group of patients who meet specific eligibility criteria. An EAP granted on this basis will follow a specific protocol, which will typically be developed in consultation with a university or hospital physician investigator and are submitted to a health regulatory agency for review. These multiple-patient Expanded Access programs are managed either by Clene or the specific hospital, institution, or university.
(2) Individual Named Patient EAP
In rare cases, Clene may provide investigational medicine access to an individual patient for treatment, where it is allowed by local regulations. An individual patient EAP is managed by the patient’s physician.
Eligibility Criteria for an EAP Request
Making decisions about requests for an EAP is complex. Clene is committed to a thorough, individualized review of each request for an EAP on a case-by-case basis. Clene strives to make these decisions as ethically and fairly as possible, taking into consideration the patient’s need, the available medical and scientific information about the investigational medicine, the seriousness of the disease, and the potential risk and benefit to the patient. Foremost, Clene is committed to patient safety and well-being: Our top priority when evaluating requests for an EAP is to ensure that we are not putting patients at unreasonable risk of harm given the context of the disease or condition being treated.
Clene will consider an EAP for an investigational medicine only if all of the following criteria are met:
- The disease must be serious or life-threatening;
- There are no comparable or satisfactory alternatives available to treat the disease or condition;
- The patient is ineligible or unable to participate in a clinical trial for the investigational medicine;
- The EAP is permitted under applicable law;
- The investigational medicine has established safety data from a controlled clinical trial;
- In the judgment of Clene and the requesting physician, there are sufficient scientific evidence to assess that the potential benefit of the investigational medicine would likely outweigh the potential risks to the patient;
- There are available clinical data to identify an appropriate dose;
- There is adequate manufacturing capacity to support both the EAP as well as ongoing and planned clinical trials without compromising compliance with applicable manufacturing requirements;
- Granting the EAP is unlikely interfere with or delay current clinical trials or regulatory filings;
- A licensed physician, hospital, or university has communicated an unsolicited request for an EAP for the investigational medicine to Clene;
- The treating physician(s) has expertise with the specific disease and ability to monitor the patient’s safety, and will obtain appropriate institutional approval(s) and informed consent or assent from the individual patient for treatment use of the investigational medicine, as necessary;
- An EAP will only be granted in a country where Clene expects to file for regulatory approval for the investigational medicine;
- The treating physician(s) will collect patient data, and maintain and release to regulatory authorities, and to Clene, all treatment records and data, as applicable;
- The patient, or the patient’s legal representative, is able to sign an informed consent document.
Evaluation of Requests
Clene’s willingness to consider requests for an EAP for an investigational medicine is based on its review of the patient information submitted by the requesting physician to support the request as well as satisfactory evidence having being provided to Clene that the physician responsible for treating the patient is appropriately qualified in accordance with local laws and regulations.
To ensure a fair review of the EAP requests, decisions will be based on the above criteria. Requests will be considered on a first come first served basis for all patients but also the ability to supply drug will be considered.
In considering and administering requests for an EAP, our patient-focused principles are based on a commitment to be fair, impartial, and transparent. Our decisions are guided by the best clinical and scientific evidence currently available for any given investigational medicine.
Of note, Clene cannot guarantee that the investigational medicine will be available for a particular patient under an EAP. This policy does not provide a guarantee of access to any specific investigational medicine for any patient. If an EAP program is granted, access will be granted only for a designated period of time and then a request must be submitted and reviewed to continue the program. An EAP will be discontinued as soon as feasible once the investigational medicine is approved by the health regulatory authority for the relevant use. In accordance with the 21st Century Cures Act, Clene Nanomedicine, Inc. may revise this policy at any time. This policy will be updated with a hyperlink or other reference to the EAP on clinicaltrials.gov once posted.
Treating Physician Criteria and Responsibilities
Any physician treating a patient receiving an investigational medicine through EAP must be properly licensed and fully qualified to administer the product. Such physician will be responsible for collecting data throughout the treatment and for submitting those data to Clene, where applicable, and in accordance with local laws and regulations. In addition, the physician will be responsible for ensuring all local legal, regulatory and Health Authority requirements are met, including Investigational Review Board or Ethics Committee approval for the EAP, and reporting of Adverse Events.
The physician must agree beforehand in writing to comply with the following terms:
- To use the product only for the patient(s) specified.
- To follow any applicable country-specific legal and regulatory requirements related to providing an investigational medicine under Expanded Access, Early Access, or comparable program.
- To comply with any Clene requirements applicable to patient confidentiality and data privacy, medical criteria, safety reporting, drug supply handling and use, and protection of intellectual property and confidential information.
- To serve as the Sponsor-Investigator for the EAP with respect to the FDA and file all necessary and required forms, reports and other submissions with FDA.
- To obtain an informed consent form (ICF) from the patient(s), which has been approved by an Institutional Review Board, Human Ethics Research Committee, or comparable regulatory review body. The ICF must include a statement that the investigational medicine maybe provided under the EAP only until such time as regulatory approval has been received for the investigational medicine. Clene will provide the investigational medicine to be administered under the EAP. However, any associated costs of treatment, including but not limited to drug administration costs, costs associated with laboratory or radiological monitoring, travel, physician or facility fees will be the responsibility of the insurer, health care system, and/or patient.
Request and Approval Process
Requests for an EAP for a Clene investigational medicine must be made by the patient’s treating physician. Physicians should submit requests to: EAP@clene.com.
If an Individual Named Patient EAP is approved, Clene will send to the treating physician:
- a Letter of Acceptance (including a request for confirmation of applicable institutional approvals and the patient’s informed consent);
- a Letter of Authorization, if applicable, authorizing the physician to reference Clene’s existing IND in submissions to the FDA;
- Instructions for Use for the investigational medicine; and
- Adverse Event Report Form.
By signing a Letter of Acceptance, the treating physician agrees to fulfill all the responsibilities outlined in such letter.
If a Multiple Patient EAP is approved, Clene will partner with the requesting physician and/or institution to develop an EAP protocol, a formal contract to support or supply the EAP, and will file the protocol with the appropriate Institutional Review Board, Human Ethics Research Committee, or comparable regulatory review body, as applicable.