Expanded Access: Requesting Access to Investigational Medicines and Devices Prior to Regulatory Approval
B. Braun Medical Inc.’s (BBMI) mission to develop innovative medical products to the meet the needs of patients is apparent in our clinical programs which incorporate only the highest standards of quality and safety.
Patients are the center of BBMI’s clinical programs, as we develop new health enhancing drugs, devices and healthcare solutions, recognizing that well-designed and carefully executed clinical trials are crucial to determining drug or device safety and efficacy. For more information on BBMI clinical trials visit www.clinicaltrials.gov.
We ensure that patients taking part in our clinical trials meet specific criteria. Not every patient who wishes to participate in a clinical trial will meet that eligibility criteria. For those patients not meeting ongoing clinical trial criteria, or for whom no clinical trial is available, Expanded Access may offer access to our investigational medicines or devices. Under Expanded Access physicians may request BBMI’s consideration for access to the Braun Family of Company’s medicines or devices which are not available for use in the United States.
Criteria
BBMI may support Expanded Access requests when there is adequate preliminary scientific evidence to support efficacy and safety. BBMI will review and consider all requests for Expanded Access. The following include factors are considered before making a decision:
- Does the patient have a serious or life-threatening illness with no comparable or satisfactory alternative therapies, including the inability to tolerate available or standard treatments?
- Is the patient unable to participate in a clinical trial for failing to meet eligibility criteria or because no appropriate clinical trial is available, among other possible reasons?
- Is there sufficient clinical and non-clinical data available about the respective investigational drug or device and the disease condition for which the application is sought, to anticipate potential benefits from treatment, i.e. is the benefit of access likely to outweigh any potential risks to the patient?
- Will Expanded Access impede or compromise any clinical trials or regulatory approval of the drug or device?
- Is there adequate supply of the requested investigational drug or device to support ongoing or planned clinical trials and the Expanded Access request?
Availability
Expanded Access is generally not available when an investigational medicine or device is in the early stages of testing. Additionally, Expanded Access may not be available for every investigational medicine or device.
Making a Request
Information for the Patient: Requests for Expanded Access will be accepted only if made by a qualified physician. Patients are encouraged to engage in candid conversations with their physicians about available treatment options and reasons for considering an investigational medicine or device. If the patient and physician mutually agree that Expanded Access is a treatment option, the physician makes the request on behalf of the patient.
If a request is granted the treating physician is required to supervise the treatment with the investigational medicine and comply with safety reporting responsibilities. As with any drug or device, investigational drugs or devices may have risks associated with their use. With that understanding the patient must be informed of all potential risks and once understanding, will be required to sign an Informed Consent Form confirming that understanding and consent and that Investigational Drugs and Devices have not been approved by the FDA.
BBMI maintains the privacy of all personal information and protected health information. All personal health records will be de-identified (anonymized) by the requesting physician and used solely to review the request within the context of the disease and the patient’s treatment history.
For additional information on Expanded Access, patients, caregivers and patient advocates may visit the U.S. Food and Drug Administration website Expanded Access: Information for Patients.
Physicians and Healthcare Professionals: Requests on behalf of a patient may be made only by appropriately licensed physicians, qualified to administer and oversee the investigational treatment and comply with safety requirements including safety reporting responsibilities. The requesting physician must provide an Expanded Access (request?) with a protocol which describes the patient’s intended treatment plan, incorporating the requested Expanded Access product. This protocol must be consistent with BBMI policies and in compliance with all applicable laws, including FDA’s regulations regarding investigational new drug (IND) or investigational device exemption (IDE) applications. The physician must be willing to meet and follow all applicable legal and regulatory requirements.
The physician will be required to obtain the patient’s informed consent, or in the case of pediatric patients, the assent and informed consent of the parent or legal guardian. The requesting physician will provide an anonymized medical history and treatment information of the patient. Institutional Review Board (IRB) approval for use of the investigational medicine will be required.
Physicians may request Expanded Access by contacting BBMI Medical Information at 800-854-6851 or MedInfo.US@bbraunusa.com
Decision
Making an Expanded Access request does not guarantee that Expanded Access will be available to a patient. All requests are evaluated in a fair and unbiased manner using objective and scientifically sound criteria. After careful consideration BBMI will respond to the request within two weeks. This response may be to request additional information to assist with determining patient eligibility.
If approved, the patient’s Expanded Access supply of drug or device may be continued until the drug or device is approved for general use through the local healthcare system, or until, in the opinion of the treating physician, there is no longer any treatment benefit. BBMI cannot guarantee a continuing drug or device supply and reserves the right to restrict access or discontinue the Expanded Access investigational drug or device supply at any time. In cases of medical drug or device shortage, product could become unavailable until the local supply situation is resolved.
For additional information on Expanded Access, physicians may visit the U.S. Food and Drug Administration website Expanded Access: Information for Physicians.