Expanded Access Programs (also known as EAPs) for a drug provide access to an investigational clinical product outside of a clinical trial before the drug has been given marketing approval by the country’s regulatory agency. Expanded Access Program is sometimes referred as “Named Patient Program” or “Compassionate Use”.
The purpose of an Expanded Access Program is to provide patients who have a serious, chronic, or life-threatening disease or condition, or have other unmet medical needs, with a treatment option, which has 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 health/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 might benefit from it.
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 unsolicited individual named patient access through its program called: KEAP (Karyopharm Expanded Access Program).
- Patients with relapsed or refractory multiple myeloma
- Patients with relapsed or refractory diffuse large b-cell lymphoma (DLBCL)
- Patients with advanced sarcoma
- Patient with other diseases in which selinexor has been studied in clinical trials will be assessed on a case-by-case basis.
Karyopharm will consider access to an investigational medicine prior to its marketing authorization when the following minimum criteria are met:
- Sufficient clinical safety and efficacy data suggest a reasonably favorable benefit/risk profile for the proposed use/disease area.
- 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 to participate due 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 potentially 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
Caligor Rx will manage the KEAP on behalf of Karyopharm for selinexor. Caligor Rx is a specialty global pharmaceutical and services business dedicated to serving patients, the community, and healthcare industry. Caligor has compiled a team of experts in implementing global expanded access programs and will guide physicians through the request process.
The KEAP is available only to patients who do not qualify for any of Karyopharm’s ongoing clinical trials with selinexor, or for patients who do qualify for a trial, but do not have reasonable access to an existing clinical site. Patients are asked to speak with their physician about their eligibility to enroll in a clinical trial.
If a patient does not qualify for a clinical trial or does not have reasonable access to a trial for which he or she qualifies, the patient should speak to their physician about their eligibility to enroll in the KEAP. If the treating physician feels that the KEAP may be suitable for the patient, he/she should contact Caligor via email at KEAP_selinexor@caligorrx.com, and a member of the Caligor KEAP team will respond within 1 business day.
Caligor will then request a valid medical license and require that a Confidentiality Agreement be put in place with the treating physician’s institution before guiding the treating physician through the request process. If the request meets the eligibility criteria for the KEAP, the treating physician must apply for and receive approval from the applicable country regulatory authority. The treating physician must also agree to comply with all site, ethical and regulatory requirements, including safety reporting, governing expanded access programs for treatment with investigational medicines, before Caligor can ship the investigational medicine to the requesting physician.
Effective November 2018