We are also committed to a rigorous clinical development process, to evaluate the safety and efficacy of investigational treatments through clinical trials, while minimizing safety risks to patients. If clinical trials establish that an investigational medicine is safe and effective, marketing approvals will be sought from regulatory health authorities with the goal of making these treatments broadly available to the patient communities.
We recognize that there are situations when a patient with a serious or life-threatening condition will not qualify for a REGENXBIO-sponsored clinical trial and may seek access to an investigational medicine outside of a clinical trial setting, also known as expanded access, treatment use or compassionate use. While participation in clinical trials is the preferred way for patients to gain access to REGENXBIO investigational therapies, expanded access requests will be considered in limited situations according to available guidelines from regulatory authorities including, but not limited to, the following criteria:
Complete requests for expanded access will be evaluated in a fair, equitable manner; however, REGENXBIO cannot guarantee that the investigational gene therapy will be available to a specific patient. Any expanded access will also be subject to local laws or regulations, and the patient must provide informed consent. Approval of requests for expanded access must be obtained from the applicable regulatory health authority and Institutional Review Board or Ethics Committee.
Further information on available REGENXBIO clinical trials can be found at our website, www.regenxbio.com, or at the U.S. Government’s website for clinical trials, www.clinicaltrials.gov. Requests for information on expanded access to investigational products must be made by a licensed physician and may be submitted to firstname.lastname@example.org. It is anticipated that acknowledgement of any expanded access questions or requests will be made by REGENXBIO within five (5) business days of receipt. REGENXBIO reserves the right to revise this policy at any time, in accordance with the 21st Century Cures Act.