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Expanded Access

About Expanded Access

Vertex is focused on developing innovative medicines so people with serious diseases can lead better lives. To do this, we conduct clinical trials to assess the safety and effectiveness of investigational medicines, which, if established, will help us obtain approvals from regulatory authorities. These approvals, in turn, are required before medicines can be made widely available to patients.

Consistent with our mission, we encourage awareness of and participation in clinical trials. Clinical trials are controlled research studies in humans designed to determine if an investigational medicine is safe and effective. Vertex believes that participating in clinical trials is the best way for patients to access investigational medicines prior to regulatory approval.   

In some extreme cases, when it is not possible for a patient to participate in a clinical trial and all other available medical options have been exhausted, the patient’s physician may seek special access to a Vertex investigational medicine outside of the clinical trial setting. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use. 

How Expanded Access Works

Making decisions about requests for expanded access to an investigational medicine is always complex. We endeavor to make these decisions as ethically and fairly as possible, taking into consideration the patient’s case, the available medical and scientific information about the investigational medicine, and the probability and timing of a regulatory approval. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.

To that end, Vertex will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:

  • The patient has a serious or life-threatening illness.
  • There are no comparable or satisfactory alternative therapies available.
  • The patient is ineligible for, or otherwise unable to, participate in a clinical trial.
  • The investigational medicine is currently being studied in clinical trials.
  • There is sufficient preliminary evidence to support an assessment that the potential benefits of the investigational medicine outweigh the potential risks.
  • The patient has a disease or condition that is similar in type and stage to the indication(s) for which the investigational medicine is currently being studied by Vertex and for which there is sufficient evidence to expect that the patient will experience a clinically meaningful benefit.
  • Providing the investigational medicine in an expanded access framework will not interfere with the initiation, conduct, or completion of clinical trials or otherwise jeopardize regulatory approval of the investigational medicine.
  • There is sufficient amount of drug available to support both ongoing clinical trials and any approved expanded access.
  • The expanded access will occur in a country where Vertex expects to file for regulatory approval and in accordance with the requirements of the national regulatory authority.

How to Request Expanded Access

We encourage patients to speak first with their physician about their eligibility to enroll in a clinical trial. If a treating physician believes expanded access may be the only option for a patient, the physician should contact Vertex to make a formal request on behalf of the patient. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements defined by Vertex. Requests for expanded access may only be made by licensed physicians through Vertex Global Medical Information. Vertex will acknowledge receipt of requests within five business days.  

Vertex is committed to evaluating all requests for expanded access in a fair and equitable manner.

All requests will be evaluated by medical professionals and decisions will be made based on scientific evidence available to the company at the time of the request.

Questions about or requests for expanded access to Vertex investigational medicines can be submitted to:

North America

Africa, Asia, Australia, Europe, and South America

For further information on available expanded access programs, visit and search “expanded access programs."