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 or the drug is commercially available in the country. An EAP is sometimes referred to as a “Named Patient Program” or a “Compassionate Use Program.” EAP is applicable for patients who have a serious, chronic, or life-threatening disease or condition, or have other unmet medical needs and have exhausted available treatment options.
Expanded Access Policy
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 via its program called: KEAP (Karyopharm Expanded Access Program).
Criteria for Eligibility
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 obtain the relevant approval on behalf of their patient from the relevant regulatory agency and follow all applicable safety-reporting regulations of the respective country.
Healthcare professionals who feel that their patient may meet the criteria above, and are interested in obtaining more details about the KEAP can contact KEAP.email@example.com. You can expect to receive a response within two business days of receipt.
Effective July 2022