How the FDA Views Access

FDA position on access to investigational drugs outside of a clinical trial (Expanded Access).

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Lexicon Pharmaceuticals, Inc., is focused on developing breakthrough treatments for human disease. To do this, Lexicon conducts clinical trials to assess the safety and efficacy of investigational medicines, which if proven safe and effective, 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 life-threatening diseases or conditions may seek special access to investigational medicines outside of a clinical trial setting. These situations are typically referred to as compassionate use, but also are known as expanded access, early access, pre-approval access, and emergency use.

The clinical development process (the process by which a drug is developed and tested for safety and efficacy, and if proven safe and effective, is submitted to regulatory authorities for approval for use) involves controlled testing in humans to ensure both safety and efficacy. Because it is not known during clinical development whether an investigational medicine is safe or effective, compassionate use may present risks for both the patient and the clinical development program. For patients, compassionate use may bring potential safety risks or a false sense that the medicine will provide benefit; for the clinical development program, it can delay or jeopardize the approval of a new medicine being sought by many.

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. Lexicon has ethical responsibilities to ensure the quality and integrity of clinical trials and to minimize risks to current research participants and future patients. These ethical responsibilities require that strict criteria are applied for the compassionate use of its investigational medicines.

Lexicon considers many factors when considering a request for compassionate use of an investigational medicine, such as the strength of the clinical data, the known benefits and risks to the patient, the impact on the clinical development program, the phase of development, and the probability/ timing of regulatory approval.

At Lexicon, a compassionate use program, or a single request for compassionate use of an investigational medicine, will only be considered if all of the following conditions are met:

  • The disease or condition being studied is serious or life-threatening.
  • There are no adequate alternative therapies or clinical trials available.
  • Sufficient preliminary efficacy and safety data exist about the investigational medicine in order for Lexicon to make a benefit-risk analysis consistent with the establishment of a compassionate use program. This would not occur earlier than the time that results from Phase 2b studies are known, and depending on the clinical program, potentially even later.
  • Sufficient clinical data are available to identify an appropriate dosage.
  • The patient’s treating physician and Lexicon’s Chief Medical Officer (or equivalent position) both have evidence to believe there is the potential for the specific patient under consideration to reasonably expect benefit from the treatment, and there is ample evidence to support the possibility that the benefit will outweigh the potential risk of treatment.
  • An adequate supply of the investigational medicine exists to support both the ongoing clinical trials and approved compassionate use, until (and if) the product becomes commercially available.
  • The patient is not eligible for a Lexicon-sponsored clinical trial of the investigational medicine. Geographic limitations to participation in a trial would generally not fulfill this criterion.
  • Granting access to the investigational medicine must not interfere with the completion of clinical trials that could support regulatory approval of the medicine or otherwise compromise the potential development of the investigational medicine.
  • The request must be made by the patient’s treating physician, unsolicited by Lexicon or any other individual or organization.
  • The patient is a resident of a country in which Lexicon retains development and commercialization rights to the investigational medicine. Lexicon cannot make a guarantee that a compassionate use program will be available, and, even if a compassionate use program is offered, Lexicon cannot guarantee that the investigational medicine will be available to a particular patient.

If all these conditions are met, Lexicon will consider compassionate use requests from treating physicians, subject to applicable laws and regulations. All requests will be evaluated in a fair, unbiased manner. Patients with any underlying medical condition(s), that may pose safety risks (or those that have not been sufficiently characterized/studied), would not be granted compassionate use. Any pre-approval access to investigational product must always comply with all applicable laws and regulations, and approvals must be secured as required from applicable regulatory bodies and Institutional Review Boards or Ethics Committees. If approved, the patient (or his or her guardian) must provide informed consent and consent to comply with the safety and monitoring requirements defined by Lexicon. The treating physician must also agree to comply with all applicable safety and monitoring requirements. Compassionate use will cease being made available once the investigational medicine becomes commercially available or if, as demonstrated by clinical trials, the investigational medicine does not demonstrate a positive benefit-risk to patients.

Patients who believe they have a medical need and meet Lexicon’s criteria for compassionate use should speak to their treating physicians, who can make a request via the following: This email address is being protected from spambots. You need JavaScript enabled to view it.. Lexicon expects that it will acknowledge receipt of any such requests within 10 business days.

When clinical trial records containing information about expanded access to Lexicon’s investigational products become 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 drug by any individual patient.