What Is An Expanded Access Program (EAP)?

An Expanded Access Program (EAP) allows patients to access investigational medication before it has been approved for use for for a specific indication by the FDA. EAPs typically accompany a clinical trial, and offer access to the investigational drug because there are no other treatments available. FDA regulations enable manufacturers to provide those patients investigational drugs that are being studied, through an “Expanded Access” or “Compassionate Use” program, when they have been determined to be beneficial to the patient.

It is important to remember that investigational drugs have not been approved by the FDA and, therefore, have not been deemed safe and effective. Only a licensed physician is able to apply for expanded access. To be part of an EAP, a patient will need to find a licensed physician who is willing to:

  • Oversee your treatment
  • Work with the manufacturing company and the FDA
  • Obtain the drugs or biologics
  • Monitor you during the course of treatment
  • File the necessary paperwork

What is the Amifampridine Phosphate EAP 

Amifampridine phosphate is a new investigational drug not currently available in the United States. Catalyst’s EAP was introduced to ensure that patients who are seriously ill with certain rare, debilitating diseases gain access to a therapy when no other FDA-approved therapy is available.  Eligible patients must meet all the inclusion criteria and have a confirmed diagnosis and/or documentation of:

  • Lambert-Eaton myasthenic syndrome (LEMS)
  • Congenital myasthenic syndromes (CMS)
  • Downbeat nystagmus

Amifampridine phosphate will be supplied at no cost to eligible patients through the EAP. All diagnostic tests and/or routine medical treatment considered standard of care will be covered by the patient’s health insurance plan or at the patient’s expense and those that are not will be covered by the EAP.

Patients who wish to enroll in the amifampridine phosphate EAP should speak with their physicians. For more information about the amifampridine phosphate clinical trial for patients diagnosed with LEMS and CMS, go to clinicaltrials.gov.

Learn more about the apmifampridine phosphate Expanded Access Program.

Physicians who wish to participate in the amifampridine phosphate EAP should call 1-844-347-3277 or email us with any questions regarding the EAP and/or participation in a clinical study.

Catalyst’s Compassionate Use Policy

As a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, Catalyst is committed to developing safe and effective therapies and providing them to rare disease patients as quickly as possible. In line with that mission, Catalyst supports the Expanded Access and Compassionate Use Programs when substantial scientific evidence exists to support both the safety and the efficacy of an investigational product for an indication, and when it is logistically practicable.

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Compassionate Use Programs, which may also be known as Expanded Access, Named Patient or Emergency Access Programs, have been created to allow, under certain circumstances, for patients with no available therapies for their condition or disease, to obtain access to a drug or therapy prior to its regulatory approval. These are provided through clinical trial-like mechanisms but are not the same as traditional clinical trials. The circumstances under which Compassionate Use access for patients may be allowed are controlled by the regulatory agencies that have jurisdiction for a specific territory and are also subject to the decision of the entity that owns the product.

Catalyst’s decisions on whether it can support requests to supply products in development is based on guidance provided by regulatory agencies such as the United States Food and Drug Administration and focuses on a two-step analysis for evaluating those requests.

In the first step, Catalyst assesses whether a requested product can be considered appropriate for Compassionate Use Programs by reviewing the extent to which:

  • There is substantial scientific evidence to suggest both the safety and the efficacy of the product for the indication(s) sought, this is usually after positive Phase 3 results are available
  • Providing access on a compassionate use basis has the potential to negatively affect clinical trials or delay/prevent regulatory approval
  • There will be sufficient product supply to address the experimental therapy
  • The requested product is feasible to provide to patients and administer outside of a formal clinical trial setting

If Catalyst is satisfied that all parts of the first step of the analysis are adequately addressed and that the product itself is appropriate for Compassionate Use access then Catalyst initiates further review under the second step of the process to determine whether individual requests otherwise meet the criteria for compassion use for the patient(s) in question.

In the second step, Catalyst weighs the following criteria under the specific request(s):

  • Whether a patient who is the subject of a compassionate use request has any conditions or issues that may be outside of safety data available from completed studies
  • Whether the requesting physician has appropriate expertise and facilities for treating the patient in a manner consistent with available product data
  • Whether the patient’s condition is consistent with the specific indication (including specific stage or type) for which the investigational therapy has been studied and approval is sought
  • Whether the requested patient’s participation in the compassionate use program would be at the expense of participation in existing or pending clinical trial(s)
  • Whether all necessary regulatory/institutional approvals have been obtained to allow the administration

While information about the process for Compassionate Use program eligibility can be addressed to Catalyst by anyone, formal requests for Compassionate Use access to Catalyst products may only come from licensed prescribers. It is expected, though not guaranteed, that all such requests will be reviewed and a decision made within 30 days.

While information about the process for Compassionate Use program eligibility can be addressed to Catalyst by anyone, formal requests for Compassionate Use access to Catalyst products may only come from licensed prescribers. It is expected, though not guaranteed, that all such requests will be reviewed and a decision made within 30 days.

For more information about or to make a request for Compassionate Use access to Catalyst products, please contact Gary Ingenito at medical@catalystpharma.com, or call 305-420-3223.

To review Catalyst’s Compassionate Use Policy, Click Here