Clinical trials provide the safest opportunity for patients to access investigational medicine before it is approved by a regulatory agency. By volunteering to take part in a clinical trial, participants receive closely monitored care from health care providers managing the trial and help others by contributing to medical research.
Clinical trials allow Bexion Pharmaceuticals, Inc. to evaluate the safety and effectiveness of its investigational new treatments. These data are necessary to obtain regulatory approval of those treatments and then make them available to the broader patient population.
To participate in a trial, patients must meet certain criteria and provide written informed consent acknowledging their willingness to accept the potential risks and benefits of the investigational treatment. Information about Bexion’s clinical trials can be found at www.bexionpharma.com/clinical-trials or www.clinicaltrials.gov.
Expanded access (sometimes referred to as compassionate use) is a potential pathway for patients with a serious or immediately life-threatening disease or condition to gain access to an investigational medicine outside of a clinical trial when no other treatment options are available.
The FDA has prepared guidelines related to expanded access, including a “question and answers” guideline (FDA guideline titled: Expanded Access to Investigational Drugs for Treatment Use —Questions and Answers, Guidance for Industry, June 2016, Updated October 2017).
Access to Bexion’s investigational products outside of a clinical trial would be considered only under limited circumstances, and as permitted by applicable law, in the following situations:
Once a trial is completed, Bexion may provide research participants with continued access to the investigational product.
For patients with a serious or immediately life-threatening disease or condition who lack other treatment options, Bexion may provide physician-requested expanded access to its investigational products.
In addition, Bexion will consider granting expanded access to an investigational medicine only if all the following criteria are also met:
The patient has a serious or immediately life-threatening disease or condition for which there is no comparable or satisfactory alternative therapies.
The patient is ineligible for enrollment in a clinical trial.
There is sufficient clinical evidence of safety and effectiveness for the specific disease condition, and the potential benefit from the use of the investigational medicine outweighs the known or anticipated risks.
Providing the investigational medicine will not interfere with clinical trials that could support the development or marketing approval for the treatment indication.
There is adequate supply of the investigational medicine.
Bexion encourages patients to first speak to their treating physician about clinical trial eligibility and enrollment options. If the physician believes that expanded access may be the only option, the physician should contact Bexion to request information about applying for access on behalf of the patient. This will enable the physician to work directly with Bexion to determine the best course of action. It is important to note that the patient’s physician must oversee all aspects relating to the potential use of the drug under expanded access (e.g., regulatory compliance, IRB approval, patient treatment, adverse event management, and reporting).
Physicians may contact Bexion at 1-800-746-3915 or by email at [email protected]. Bexion will typically acknowledge receipt of a request for expanded access within 3-5 business days.
Bexion will carefully review all expanded access requests in consideration of the above criteria. If approved by Bexion, final approval of the expanded access by the regulatory authority in the country where the request originated is required before shipment of any investigational medicine will occur. All physicians who receive investigational medicine from Bexion through expanded access are required to comply with all applicable laws and regulations, and contractual conditions, including those relating to safety reporting.
More information can be found at https://www.fda.gov/news-events/public-health-focus/expanded-access