Vertex is focused on developing transformative medicines for people with serious and life-threatening diseases. 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. We believe that participating in clinical trials is the best way for patients to access investigational medicines prior to regulatory approval.
In some cases, when it is not possible for a patient to participate in a clinical trial, the patient is in critical medical need, 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 managed access, expanded access, early access, preapproval access, compassionate use and emergency use.
Making decisions about requests for expanded access to an investigational medicine is always complex. We endeavor to make these decisions in a fair and equitable manner, 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’s licensed physician submits an unsolicited request for expanded access.
- The patient has a serious or life-threatening illness and fulfills objective clinical criteria establishing that the patient is in critical need.
- There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition.
- The patient is ineligible for, or otherwise unable to, participate in a clinical trial for the product.
- Expanded access is permitted under applicable law.
- The investigational medicine has been evaluated in pivotal clinical trials and based on these pivotal clinical trials, there are sufficient and meaningful data to support a preliminary assessment that the potential benefits to the patient outweigh the potential risks.
- Staff and other required resources are available to provide the product, and there is a sufficient amount of product available.
- Providing the product will not jeopardize regulatory approval or clinical development of the product.
- Vertex expects to file for regulatory approval for the investigational medicine and there is a path to sustainable access for all patients in the patient’s country of residence.
All requests will be carefully evaluated in a fair and equitable manner by Vertex medical professionals with every effort made to provide a response promptly once we have all the necessary information. Decisions will be made based on scientific evidence available to the company at the time of the request and in consideration of applicable local laws and regulations.
Please note that enrollment of new patients in the Expanded Access Program will continue for a maximum of 12 months after marketing authorization is obtained in the patient’s country of residence, unless local regulations require that the program terminates earlier.
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.
Please contact Global Medical Information if you have questions about or requests for expanded access to Vertex medicines or investigational compounds. Please refer to the following website for the Global Medical Information contact details for your country: www.vertexmedinfo.com.
For further information on available expanded access programs, visit www.clinicaltrials.gov and search “expanded access programs."