Policy on Granting Expanded Access to Investigational Medicines
Lexicon Pharmaceuticals, Inc. is a biopharmaceutical company with a mission of pioneering medicines that transform patients' lives. To accomplish this, Lexicon conducts clinical trials to assess the safety and efficacy of investigational medicines over the span of several years to evaluate whether they are both safe and effective in the diseases being studied. These trials will allow Lexicon to seek the necessary approvals from regulatory authorities to provide patients with access to these medicines.
In general, Lexicon believes that participating in clinical trials is the best way for patients to access investigational medicines prior to approval. In some extreme circumstances, when this is not possible, patients with serious or immediately life-threatening diseases may seek special access to investigational medicines outside of a clinical trial setting. These situations are typically referred to as Expanded Access, but also are known as Compassionate Use, Early Access, Pre-Approval access, and Emergency Use.
While there may be reason to believe that an investigational medicine may represent an option for patients who have failed or could not tolerate approved treatments for a particular condition, it is not known before the completion of all clinical trials and review by the US Food and Drug Administration whether it meets the standard of being be safe and effective.
Therefore, use of the investigational medicine in an Expanded Access program, before safety and efficacy have been adequately characterized, may present risks for both the patient and the research trials needed to demonstrate safety and effectiveness. For patients, use of the investigational medicine in an Expanded Access program, particularly in a disease which has not been previously studied, may bring safety risks as well as undue optimism that that the medicine will provide benefit. With respect to the clinical research trials, by providing the investigational medicine to patients through the Expanded Access program rather than through clinical trials, Lexicon may not be able to complete the trials in a timely manner. This, in turn, may mean a delay in the review and possible approval of the medicine by FDA, which would delay the availability of the medicine and its potential benefits to many thousands of patients.
Therefore, in reviewing a request for Expanded Access to one of its investigational medicines, Lexicon considers many factors and must have adequate information to determine that the potential benefits outweigh the risks of treatment for a particular patient, and what impact granting the request would have on its clinical trials. These factors include, but are not limited to, whether the patient’s condition is serious or immediately life threatening, whether the patient qualifies for enrollment in an ongoing clinical trial studying the medication, whether there are alternative therapies that may be appropriate for the patient, the likely outcome of the disease itself, and what is known about the investigational medicine at the time of the request (including safety data, efficacy data, and the appropriate medication dose), and whether there is adequate supply of the medication for the patient and the ongoing trials.
Lexicon will review Expanded Access requests from treating physicians for patients who reside in the United States, subject to applicable laws and regulations. All requests will be evaluated in a fair, unbiased manner.
Patients who believe they have a medical need and meet Lexicon's criteria for Expanded Access should speak to their treating physicians, who can make a request via the following electronic mail address: CorpComm@lexpharma.com. Lexicon expects that it will acknowledge receipt of any such requests within 3 business days of receipt. In certain circumstances, it may be necessary for Lexicon’s physicians to speak directly with the patient’s physician to determine whether Expanded Access is appropriate for the patient.
If an Expanded Access Record containing information about expanded access to Lexicon's investigational products becomes publicly available pursuant to section 402(j)(2)(A)(ii)(II)(gg) of the Public Health Service Act, Lexicon will add a hyperlink or other appropriate reference to those records on this website page.
As authorized by the 21st Century Cures Act, Lexicon may revise this position statement at any time. Additionally, the posting of this position statement by Lexicon shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.
Information regarding Lexicon's clinical trials is available at www.ClinicalTrials.gov, by searching with the name Lexicon in the “Other terms” field.