United States Expanded Access Policy
At NS Pharma, Inc. ("NS Pharma"), in coordination with our parent company Nippon Shinyaku Co., Ltd., we are committed to developing pharmaceuticals for patients with rare diseases that have unmet medical needs.
Investigational medications are not approved for commercial sale by the United States Food and Drug Administration ("FDA"). A pharmaceutical company must perform clinical trials to establish the safety and efficacy of investigational medications. Approval by the FDA is the only way to make these treatments commercially available to the medically appropriate indicated patient population.
NS Pharma maintains that, prior to approval, participating in our clinical trials is the best way for patients to access our investigational drug in order to generate efficacy and safety data, in accordance with the FDA. Therefore, when our investigational drug is in clinical development, we encourage patients to speak with their treating physician about their eligibility to enroll in our clinical trials. Information about NS Pharma’s clinical trials of its investigational medication is available at clinicaltrials.gov.
However, for those patients who are unable to participate in a clinical trial or in whom standard treatments have failed or who have been unable to tolerate already approved medications, they may seek access to investigational medications through what is often referred to as an expanded access program ("EAP").
NS Pharma will, in its sole discretion, evaluate requests for expanded access in the United States. NS Pharma may consider, without limitation, some or all of the following criteria:
- Whether the patient suffers from a serious or immediately life-threatening disease or condition
- Whether there are no comparable or satisfactory alternative therapies or clinical trials available
- Whether sufficient preliminary efficacy and safety data exist to support an assessment that the benefit for the patient outweighs the potential risks and that the potential risks are not unreasonable in the context of the patient’s diagnosis or condition being treated
- Whether sufficient clinical data is available to identify an appropriate dose of the investigational drug in the context of the patient’s diagnosis or condition being treated
- Whether there is adequate drug supply to support the ongoing and necessary clinical trials as well as to support expanded access in a sustainable and equitable manner
- Whether the patient is not eligible to participate in any ongoing clinical trials of the investigational drug
- Whether expanded access will not adversely affect the clinical development program, including the initiation, conduct, or completion of the clinical trials that are required for regulatory approval
- Whether the unsolicited request is made by a qualified and licensed physician
- Whether the requesting physician has the expertise and facilities appropriate for the administration of the investigational drug and for monitoring, managing, and reporting any side effects and the patient’s overall experience
- Whether all required regulatory and institutional approvals have been obtained; and/or
- Whether the patient has provided written informed consent
Request Procedures and Contact Information
NS Pharma encourages patients to consult with their treating physician about their eligibility to enroll in a clinical trial.
Patients, Caregivers and Patient Advocates: Requests for Expanded Access must be made by a qualified physician. NS Pharma encourages patients and caregivers to have a candid conversation about reasons for considering an investigational medicine. Your physician will be required to supervise the treatment with the investigational medicine and comply with safety and reporting responsibilities. Not all physicians are able or willing to assume the responsibilities required for an investigational medicine. If you have mutually agreed that expanded access is a treatment option, your physician must make the written request on your behalf.
Patients, caregivers and patient advocates may find additional information on Expanded Access by visiting the U.S. Food and Drug Administration website Expanded Access: Information for Patients.
Physicians and Healthcare Professionals: Only appropriately licensed physicians may make a written request on behalf of a patient. The physician must be qualified to administer and oversee the investigational treatment and comply with NS Pharma safety reporting responsibilities. Investigational Review Board (IRB) approval for the use of the investigational medication is required. The physician must be willing to meet and follow all applicable legal and regulatory requirements. The physician will be required to provide adequate medical history and treatment information for a specific patient, obtain informed consent, and may be required to provide additional data on the progress and safety throughout the expanded access treatment interval. Physicians may find additional information on Expanded Access by visiting the U.S. Food and Drug Administration website Expanded Access: Information for Physicians.
If the treating physician believes that expanded access may be the only option for a patient, the physician should contact firstname.lastname@example.org to make a formal request on behalf of a patient. NS Pharma will acknowledge the request for expanded access within 7 business days. We may request additional information to assist with patient eligibility for an investigational medication.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each of our investigational medicines is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests where circumstances may be different. Expanded access requests for our investigational medication will be individually evaluated by NS Pharma. NS Pharma is committed to evaluating all requests for expanded access in a fair and equitable manner. All requests will be evaluated by medical professionals and decisions will be based on available scientific evidence at the time of the request and any decision will be at NS Pharma’s sole discretion. NS Pharma cannot make a guarantee that Expanded Access will be available to a patient. An expanded access program may be closed at any time including, among other reasons, once the investigational therapy has been approved and is commercially available in the patient’s country.
Contact Information: A treating physician may submit questions or requests regarding Expanded Access by contacting the NS Pharma Expanded Access Call Center 1-877-539-8515 or by emailing email@example.com.