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Expanded Access

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.

At Jazz, we believe that wherever possible, use of an investigational medicine for a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. 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.

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.

Consistent with applicable laws and regulations, Jazz may provide patients with access to investigational medicines through Expanded/Early Access Programs (EAPs) when there is sufficient evidence of the safety and effectiveness of the investigational medicine to support its use in the particular circumstance.

It is important to note that terminology used can vary from country to country and dependent on the way that the medicine is accessed. Although often referred to as Expanded Access Programs or Early Access Programs, these types of access can also be known as Compassionate Use, Pre-approval Access, Named Patient Access and Emergency Use.

EAPs do not apply to the use of a currently marketed medicine for an indication outside of the approved label.

Types of Early Access Programs

In general, there are two ways that Jazz can grant Early Access to an investigational medicine. The type of program available may be dependent on local laws and regulations. It is important to note that terminology used can vary from country to country and in some instances different countries use identical terminology to describe different approaches.

  • Individual Patient Access – where a qualified healthcare professional requests an investigational medicine for use for a single patient because, in their judgement, currently available therapies are not satisfactory. This request may or may not require approval by the national health authority dependent on local regulation. Jazz may also make an investigational medicine available on a named, single patient basis through a third party provider in some territories.
  • Multiple Patient Access – This type of program is commonly referred to as Compassionate Use in EU or Expanded Access in the United States and describes the process by which an investigational medicine is made available to a defined group of patients with an unmet medical need under a specific treatment protocol. In most countries this type of program is initiated by Jazz and approved by the national health authority.

Eligibility Criteria

Jazz considers many factors when evaluating a request for individual patient access, also known as named patient access in Europe, 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, the country-specific regulations, and probability and timing of regulatory approval within the applicant’s country, though the medicine may be approved in another country.

In determining whether named patient access is appropriate outside of a clinical trial, the criteria Jazz evaluates include the following:

  • Jazz has sufficient evidence of the safety and effectiveness of the investigational medicine to support its use in the particular circumstance.
  • The disease is serious or life threatening.
  • There are no beneficial available alternative treatments or patients have exhausted all other therapies.
  • A clinical study is not available, either because patients are ineligible or because they have no access to the trial.
  • The efficacy and safety data available at the time are of sufficient strength to determine whether the benefit to patient would likely outweigh the potential risks.
  • Access is compliant with local rules and laws.
  • The request must be made by the patient’s treating physician, unsolicited by Jazz Pharmaceuticals or any other individual or organization.
  • The patient’s treating physician determined that there are sufficient safety and efficacy data, including dosing information, to support the use of the investigational medicine, that there is no comparable or satisfactory therapy available to treat the patient’s disease and that the probable risk to the patient from the investigational medicine is not greater than the probable risk from the disease itself.

 

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

Decisions regarding potential named patient access to investigational medicines can only be made, in certain circumstances, after in-depth discussion between Jazz’s clinical teams and the patient’s qualified healthcare professional and in line with required national health authority approval, if applicable.

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.

For further information

For individual patients in the U.S., treating physicians can submit requests through the EAP portal, which you may access HERE. If you have not previously submitted a U.S.-based individual EAP request, please begin by reading "How to Apply" and then create an account (requires a valid email address). If you already have an account, please sign-in using your username and password.

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

For patients outside the U.S., treating physicians can request information by contacting Jazz by email at EarlyAccess@jazzpharma.com.

Access for preapproved cohorts of patients is regulated at a national level and details vary from country to country. This type of program is commonly referred to as Compassionate Use in the EU or Expanded Access Program in the U.S. In general, once approval is granted by the national health authority, a patient’s qualified healthcare professional can apply to Jazz for access to the medicine, under a specific treatment protocol.

These programs are always conducted within the anticipated product label and are only commenced after sufficient data are available to support regulatory submission and support health authority approval.

The JZP458 Managed Access Program in the EU, Switzerland, Australia and New Zealand is managed by Bionical Emas on behalf of Jazz Pharmaceuticals. Inquiries and requests for JZP458 expanded access use in these territories should be directed to Bionical Emas at Patient.Access@Bionical-Emas.com.

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