Expanded Access Policy

ARIAD is focused on discovering, developing and commercializing precision therapies for patients with rare cancers. ARIAD is working on new medicines to advance the treatment of rare forms of chronic and acute leukemia, lung cancer and other rare cancers which includes conducting clinical trials.

Clinical trials are conducted to determine the safety and efficacy of an investigational medicine. The safety and efficacy data collected from clinical trials is used to expand our knowledge about how investigational medicines work as well as support approval from the regulatory authorities. Approval from a local regulatory authority is usually required before a medicine can be made available to patients.

There are some cases where a patient is not able to participate in a clinical trial and other treatment options have been exhausted, are not appropriate or have not been beneficial. In those cases, the patient’s physician may choose to request access to an investigational medicine without participation in a clinical trial. Depending on the setting, this type of access may be referred to as expanded access, early access, compassionate use, named patient use, pre-approval access or emergency use. If you are a patient with questions concerning access to an investigational medicine - either through a clinical trial or expanded access - please discuss your situation with your treating physician.

How Expanded Access Works

ARIAD considers many factors when considering a physician’s request for Expanded Access with an emphasis on making a decision that is both ethical and fair while not exposing the patient concerned to undue risk.

Factors that will be considered in making a decision include the type of request, the health of the patient, the scientific and/or medical rationale for use based on the specific request, applicable regulatory requirements, and the potential impact on ARIAD’s development program for the investigational product. Please note that this Expanded Access Policy does not serve as a guarantee of access to any ARIAD investigational product by any individual patient. This policy is subject to change from time to time, and ARIAD reserves the right to terminate or modify this policy at any time.

In alignment with the above guidelines, ARIAD will consider a request for expanded access where, in ARIAD’s sole estimation, the following criteria are met:

  • The request is coming from a physician licensed in the jurisdiction where the Expanded Access is sought, who will take primary responsibility for supervising use of the investigational ARIAD product and will comply with applicable regulatory requirements
  • The request is for the treatment of a serious or life-threatening disease
  • There are no other appropriate therapies available
  • The patient is unable to enroll in a clinical trial
  • Data collected on the investigational drug in ARIAD clinical trials indicates that there is a scientific and/or medical rationale as well as a positive risk/benefit ratio
  • There is enough supply of the investigational ARIAD drug to support expanded access alongside other business commitments, such as those made for ongoing or planned clinical trials
  • The request for expanded access will not, in ARIAD’s sole judgment, impact any of ARIAD’s clinical trial plans or regulatory submissions for the investigational drug
  • The request is made from a country or region where ARIAD intends to file for regulatory approval
  • The arrangement would comply with applicable regulatory and legal requirements

 

How to Request Expanded Access

The treating physician should contact ARIAD to make a formal request with supporting medical and/or scientific rationale for expanded access on behalf of a specific patient, consistent with applicable regulatory requirements.

ARIAD strives to confirm receipt of requests submitted with complete medical documentation as quickly as possible, usually within five (5) business days. Patient eligibility will be determined by ARIAD in accordance with established policies and procedures. ARIAD’s acceptance and processing of an expanded access request does not guarantee that access to investigational product will be provided.

All questions about or requests for expanded access to ARIAD investigational medicines can be submitted to: medinfo@ariad.com or by calling +1 (855) 552-7423 to speak with our medical information department.

For further information on the US brigatinib expanded access protocol that has been established for a cohort, or group, of patients who meet specific eligibility criteria, click here or visit www.clinicaltrials.gov and search “brigatinib” or “NCT02784158”.

 

02/09/2017v1