Access to Investigational or Unapproved Medicines

Jazz Pharmaceuticals is focused on improving patients’ lives by identifying, developing and commercializing meaningful products that address unmet medical needs. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, and the data generated from these trials allows us to obtain the necessary approvals from regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), which allow for broad access to medicines.

Jazz believes that the best way for patients to obtain access to investigational medicines before they are approved is by participating in clinical trials. However, we also recognize that in some extreme circumstances, when participating in a clinical trial is not possible, patients facing serious or life-threatening conditions may seek special access to investigational medicines outside of a clinical trial setting. These situations are typically referred to as expanded access programs, but can also be known as compassionate use, early access, pre-approval access and emergency use.

At Jazz, consistent with applicable laws and regulations, Jazz may provide patients with access to unapproved or investigational medicines through expanded access programs when there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.

Conducting clinical trials is extremely complex and challenging. The ultimate goal is the rigorous testing of the clinical product with the aim of securing regulatory approval and enabling the medicine to be available to as many patients as possible as quickly as possible. Jazz complies with good clinical practices and takes steps to help ensure the quality and integrity of our clinical trials and to minimize risks to current research participants and future patients.

For a list of clinical trials currently recruiting patients, please visit www.clinicaltrials.gov.

Expanded Access Programs

Jazz considers many factors when considering a request for expended access to an investigational medicine, such as the strength of the clinical data, the benefit-risk profile, the potential impact on the clinical development program, the phase of development, and probability and timing of regulatory approval.

At Jazz, expanded access can only be considered if all of the following conditions are met:

Jazz will consider the above criteria in determining whether to offer expanded access; however, Jazz cannot make a guarantee that an expanded access program will be available, and, even if an expanded access program is offered, Jazz cannot make a guarantee that the investigational medicine will be available to a particular patient.

How to Request Access

For patients that meet Jazz Pharmaceutical’s criteria above, treating physicians can make a request via Jazz Pharmaceuticals Medical Information center by email to jazzpharma@medcomsol.com or by telephone to 1-800-520-5568.

This call can be made by the patient, doctor or caregiver; however, a formal request must be made by the patient’s qualified treating physician.

How Decisions are Made

Jazz is committed to a fair and impartial evaluation of each request for access to our investigational medicines. Therefore, all decisions are based on clinical circumstances. Whenever possible, patients will be referred to ongoing clinical trials as the primary way to access investigational medicines. Decisions regarding potential expended access to investigational or unapproved medicines can only be made, in certain circumstances, after in-depth discussion between Jazz’s clinical teams and the patient’s qualified health care professional.