Kiera, Age 14
Living with CDKL5 Deficiency Disorder (CDD)
Our Promise
Since the company’s earliest days, people affected by rare and orphan diseases are at the heart of everything we do at Amicus Therapeutics. Our mission is to transform their lives by developing first-in-class or best-in-class medicines that improve their health and well-being. With the development of medicines that will be safe, effective and satisfy unmet medical need, comes the promise that these medicines will be broadly accessible. Our ultimate goal is to provide access through marketing authorization. However, before our product candidates might become available through these means, Amicus is committed to carefully designed and considered pathways to expanded access to our investigational drugs.
Paths to Access
The first of these paths is clinical research (also called clinical trials or clinical studies). As part of established processes for drug development, Amicus sponsors clinical research to best understand the safety and effectiveness, the risk and benefit of its investigational drugs. Parameters for these studies are determined together with regulatory authorities, including the criteria necessary for volunteer participants. Studies are generally multi-phased, closely monitored and can be spread out over many years. People wanting to help advance disease knowledge and who meet the appropriate criteria may participate in these clinical studies.
For those participants who complete an Amicus clinical study, it is the company’s general plan to provide an open-label, long-term extension trial, as appropriate to meet need. This gives study participants continued access to the investigational drug until it is approved and available in their respective geography or available through an Expanded Access Program (EAP). Long-term extensions also enable collection of additional data to support ongoing safety and, in some instances, potential efficacy of the still-experimental medicine.
Expanded Access
Sometimes, however, someone may not qualify for a clinical study or there may be no studies available. Depending upon where a patient resides and the local authorities, there may be other paths toward access. These approaches to expanded access may have slightly different names or approaches, such as Named Patient Program or Named Patient Use, Authorization for Temporary Use (ATU), Compassionate Use, Early Access or others, depending upon geography and what may be allowed by the respective regulatory authorities.
Amicus is dedicated to addressing the needs of those individuals who have a serious or potentially life-threatening disease for which Amicus has a potential therapy under investigation and who have exhausted all alternative treatment options, including enrolling in available clinical trials. For these patients, Amicus is committed to careful review of all physician-submitted requests for expanded access on an individual, case-by-case basis. Taken into consideration are factors such as a person’s current medical condition based upon information provided by their requesting physician, treatment options, the known safety and efficacy of the investigational medicine, available supply of medicine, applicable regulations, if any, among other pre-determined criteria for the patient and guidelines for the company. In collaboration with regulatory authorities, a patient may be given expanded access to Amicus investigational and pre-reimbursed products before such products have received regulatory approval, provided that the requirements listed below are met.
Criteria for Consideration of a Request for Expanded Access
All of the following conditions must be met by the individual patient’s situation, the requesting healthcare provider and Amicus in order for a request to be considered.
The individual patient’s situation must align with the following requirements:
The qualified physician or healthcare professional (HCP) providing medical care to the individual must:
The Amicus cross-functional internal review process must confirm that:
Making a Request for Early Access
Requests for expanded access to an Amicus investigational medicine must be made by the patient’s treating physician or qualified HCP. Physicians and HCPs may submit requests to ExpandedAccess@amicusrx.com or obtain additional information from the Amicus Medical Affairs representative in their area. As with any situation involving the care and concern for individuals living with rare and orphan diseases, all information submitted as part of a request will be maintained in the strictest of confidence and used solely for the purpose of evaluating a patient’s eligibility for expanded access. Confirmation of receipt of written requests will be made within three (3) working days. Decisions will be made as quickly as possible and the requesting physician notified.
Requests for early access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States and Canada at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.
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