Expanded Access Programs (also known as EAP) for a drug provide access to a treatment option that is not yet an approved or marketed medicine. Expanded Access is sometimes referred as “Named Patient” or “Compassionate Use”.
The purpose of expanded access programs is to provide patients who have a serious, chronic, or life-threatening disease or condition, and who have an unmet medical need and have exhausted all approved treatment options, with access to certain promising medicines which have not yet been approved for marketing by government regulatory authorities.
Expanded Access at Karyopharm
Karyopharm is committed to bringing its investigational medicines to patients, under a marketing authorization, as quickly as possible.
Enrolling in a clinical trial is the primary way for patients to access Karyopharm’s investigational medicines prior to a marketing authorization by government regulatory authorities. To learn more about available clinical trials, visit clinicaltrials.gov and search by company, disease or investigational medicine. These clinical trials are needed to demonstrate that the investigational medicine meets the standards for safety and efficacy that government regulatory agencies, such as the U.S. Food & Drug Administration (FDA) or the European Medicines Agency (EMA), have established. Gaining regulatory approval and marketing authorization for a medicine is the only way to bring rapid access to the greatest number of patients who may benefit.
However, there may be patients who do not qualify for an ongoing clinical trial but who might benefit from the investigational medicine. For these patients, Karyopharm may be able to provide access to its investigational medicines through individual Named Patient access through our EAP.
Karyopharm will consider access to an investigational medicine prior to its marketing authorization only when the following minimum criteria are met:
- Sufficient clinical safety and efficacy data suggest a favorable benefit/risk profile for the proposed use.
- Adequate supply of the investigational medicine exists to perform necessary clinical trials as well as to support expanded access patients.
- The patient(s) for whom treatment with an investigational medicine is sought:
- Is/are suffering from a severely debilitating or life-threatening disease which cannot be treated satisfactorily by an authorized drug in the country where the request has originated.
- Has/have received appropriate standard treatment without success (or no standard treatment exists for the patient’s condition) and no satisfactory alternative drug is available.
- Is/are ineligible to participate in any ongoing clinical study with the investigational drug or unable due participate to geographical limitations.
Three core principles guide Karyopharm when determining whether and how to provide patients with access to its investigational medicine prior to regulatory approval and marketing authorization outside of clinical trials:
- Physicians, in consultation with their patients, are in the best position to determine whether their patients are appropriate candidates for investigational medicines.
- Patients who need a life-saving medicine should have access as soon as possible when the potential benefit to their health outweighs the risk of harm.
- Providing potentially life-saving medicines should be done on a fair and sustainable basis without undermining ongoing clinical trials.
Investigational medicines are provided to patients only through treating physicians who must follow all applicable safety-reporting regulations of the respective country.
Process for Applying to the Karyopharm Expanded Access Program – KEAP
Patients are asked to speak with their physician about their eligibility to enroll in any Karyopharm clinical trials or expanded access programs. If the physician feels that the expanded access program may be suitable for the patient, the physician should contact Karyopharm (using the email and form provided below) to make the request on behalf of the patient. This process will enable the physician to work with experts within the company to determine the best course of action. KEAP is available only to patients who do not qualify for any of Karyopharm’s ongoing clinical trials, or where the patient does not have reasonable means of reaching an existing clinical site to enroll in a Karyopharm clinical trial for which he or she qualifies.
Treating physicians should submit expanded access requests on behalf of patients or their caregivers using KEAP Request Form for Selinexor FRM-PS-0003 V2.0. The submission must contain a brief patient history, sufficient information on the patient’s current medical status to allow for an evaluation by Karyopharm medical staff, and contact information of the treating physician in case Karyopharm medical staff have additional questions. The submission must not contain any patient identifying information, such as names or ID numbers. Physicians must submit the request form to KEAP@karyopharm.com for consideration. Once Karyopharm has received the emailed request form, we anticipate responding to the patient or treating physician within five business days.
If the request is supported by Karyopharm, the treating physician must apply for and receive approval from the applicable country regulatory authority, and must comply with all site, ethical and regulatory requirements, including safety reporting, governing expanded access programs for treatment with investigational medicines, before Karyopharm can ship the investigational medicine to the requesting physician.
Effective June 13, 2017