Expanded Access Policy Statement
Clinical development is a vital part of our company mission to provide innovative medicines, particularly those that treat rare diseases, to patients with unmet treatment needs. We are committed to bringing safe and effective medicines to patients as quickly as possible and view that participating in clinical trials is the best way for patients to access medicines that are not yet approved by regulatory authorities such as the U.S. Food and Drug Administration. In circumstances when participating in a clinical trial is not possible, patients may seek special access to investigational medicines outside of a clinical trial setting and prior to regulatory approval. In the United States, these situations are typically referred to as expanded access.
We are and will be evaluating medicines in clinical trials for a range of potential conditions. Additional information on a majority of these trials is posted at www.clinicaltrials.gov.
As part of our commitment to patients, we may provide unapproved medicines via expanded access programs in the United States. At this time, we are unable to provide unapproved medicines via expanded access programs or compassionate use outside of the United States. In general, we are not able to provide expanded access to an approved or unapproved medicine if it is for a disease or condition that we have not studied or to an approved marketed medicine for an indication that has not been approved in the medicine’s label. As the trials progress and we learn more about these medicines and how they perform in patients, we will periodically re-evaluate the potential for expanded access programs and update the physician, patient and advocacy communities.
When providing an expanded access program for an unapproved medicine, we will consider many factors, including:
- Seriousness of the Disease: The underlying disease or condition that the unapproved medicine treats is seriously debilitating or life-threatening, and the disease or condition is one where a treating physician would reasonably determine that a comparable or satisfactory alternative does not exist for the patient.
- Safety and Effectiveness of the Medicine: The unapproved medicine must be safe and effective for its intended use and the treatment of the patient. Specifically, there must be sufficient safety and efficacy data for the physician to determine that the benefits outweigh the risks of using an unapproved medicine.
- Available Clinical Data: Clinical data, preferably from Phase 3 clinical trials, must be available for the proposed use of the unapproved medicine.
- Regulatory Pathway and Supply: We will evaluate whether the program will interfere with the initiation, conduct or completion of clinical studies, including our ability to maintain supply for the clinical trials, as such interference could impact our ability to ultimately obtain approval for the medicine and provide it to a broader patient community in need. Further, we will make reasonable assessments of potential risk versus benefit for patients outside the clinical trial setting.
In the event we decide to consider expanded access use in the future, we will evaluate and respond to each expanded access request on a case-by-case basis in a fair, impartial and equitable manner relying on the general criteria set forth below. Please refer to www.clinicaltrials.gov for more information about our ongoing clinical trials and expanded access availability for an unapproved medicine.
- Treating Physician’s Request: A licensed treating physician must submit the request for expanded access to the unapproved medicine on behalf of the patient. Such physician must be qualified to treat the underlying disease or condition. Further, such physician must agree to follow all applicable legal and regulatory requirements when treating such patient with the unapproved medicine. If you are a licensed treating physician requesting expanded access to an unapproved medicine for your patient, please contact us directly at email@example.com with the basis for the request, ensuring that the request addresses all of the criteria set forth herein and any specific criteria for the applicable expanded access program. We anticipate we will acknowledge receipt within twelve (12) business days or less.
- Patient’s Health and Safety: A licensed treating physician must determine, based on the safety and effectiveness data from clinical studies and the patient’s medical history, that the benefit of the unapproved medicine to the patient outweighs the risk the patient faces from the disease or condition to be treated by the unapproved medicine. Further, the patient’s underlying medical conditions do not present additional or increased safety risks.
- No Comparable or Satisfactory Alternative: The licensed treating physician submitting the request must have made a determination that the patient has no comparable or satisfactory alternative therapy to diagnose, monitor or treat the underlying disease or condition.
While we endeavor to help patients, please know that we cannot guarantee access to our unapproved medicines, and even if we do decide to provide an early access program, certain circumstances may arise that will prevent us from providing access to an unapproved medicine. For additional information about this policy please contact us at firstname.lastname@example.org.