Karyopharm Expanded Access Program
Expanded Access Programs (also known as EAPs) 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. An EAP is sometimes referred to as a “Named Patient Program” or a “Compassionate Use Program.”
The purpose of an EAP 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 and is not commercially available in the country.
Expanded Access Program Details
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, provided they have exhausted all other available treatment options, 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).
Criteria for Eligibility
Patients with the following conditions who meet certain inclusion/exclusion criteria may be provided access to selinexor depending on the country of residence:
Criteria for Eligibility:
- Patients with relapsed or refractory multiple myeloma
- Patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL)
- Patients with advanced sarcoma
- Patients with advanced glioblastoma
- Patient with other diseases in which selinexor has been studied in clinical trials will be assessed on a case-by-case basis
Additional Required Criteria
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.
The Process for Applying to the Karyopharm Expanded Access Program(s)
Clinigen Group currently manages a Paid Named Patient Program in the following countries for requests from physicians interested in prescribing selinexor to their patients with either Multiple Myeloma or Diffuse Large B-Cell Lymphoma:
- Europe – Austria, Bulgaria, Czech Republic, Denmark, Estonia, Finland, France, Hungary, Iceland, Ireland, Italy, Lithuania, Luxembourg, Netherlands, Norway, Poland, Portugal, Slovakia, Spain, Sweden, Ukraine, United Kingdom
- Asia, Africa & Middle East – India, Japan, Kazakhstan, Kenya, Nigeria, Russia, South Africa, Sri Lanka, Turkey and Yemen
- Latin America – Argentina, Brazil, Chile, Costa Rica, Dominican Republic, El Salvador, Guatemala, Mexico, Panama, Paraguay, Peru, Puerto Rico
Healthcare professionals can obtain details about obtaining selinexor for their patients by calling the Clinigen customer service team at +44 1932 824 123 (in all countries outside of the UK) or +44 1932 824 100 (within the UK) or emailing: firstname.lastname@example.org
Caligor Coghlan Pharma Services (CCPS) manages requests from physicians from countries other than those listed in section 1. For the countries which are listed in section 1, indications other than Multiple Myeloma and Diffuse Large B-Cell Lymphoma will be managed by CCPS following Karyopharm approval.
Healthcare professionals can obtain details about obtaining selinexor for their patients by contacting the CCPS team at: KEAP_selinexor@calcog.com.
Certain requirements and/or restrictions will apply for physicians and patients interested in obtaining selinexor as part of Karyopharm’s Expanded Access/Named Patient Programs. The teams at Clinigen and/or CCPS will help guide interested physicians through the process and explain all requirements needed before selinexor can be supplied. The Karyopharm team (KEAP.email@example.com) is available to answer any questions you may have relating to the two programs.
Effective October 2021