Orchard policy on pre-approval access to experimental medicines

Our mission at Orchard Therapeutics is to transform the lives of patients with rare disorders through innovative gene therapies. Our company utilizes the potential of autologous ex vivo gene therapy to address serious and life-threatening inherited disorders, including primary immune deficiencies, neurometabolic disorders and hemoglobinopathies. Because people living with these life-threatening, debilitating conditions have few or no treatment options, our work is marked by not only a commitment to the highest standard of preclinical and clinical research but also an acute sense of urgency. Patients are waiting.

Our commitment to clinical research

To ensure patients have the chance to benefit from these potentially transformative medicines, we focus our resources on conducting clinical trials which meet the exacting standards of regulatory authorities around the world and on obtaining their approval. We believe that regulatory approval, which reflects the robust demonstration of the safety and efficacy of our medicines through clinical studies, is the best way to ensure timely access to our medicines for patients.

Process for managing pre-approval access requests

We recognize, however, that there may be circumstances in which patients or caregivers seek access to our experimental medicines before they are approved by regulatory agencies. Therefore, we have developed the following process on pre-approval access requests:

  • Ahead of a medicine’s approval by independent regulatory authorities, all requests for pre-approval access received by Orchard will be evaluated to determine a patient’s eligibility for a company-sponsored clinical study.
  • Where a patient does not qualify for the ongoing clinical study, Orchard will endeavor to guide requests towards patient organizations, medical professionals and/or scientists who may be able to identify other clinical studies or approved treatment options which may be suitable.
  • In very exceptional circumstances, requests for pre-approval access will be carefully considered once enrollment in registrational studies is complete. Key considerations for potentially granting such requests include (but are not limited to):
    • The potential benefit/risk profile of the experimental medicine for individual patients based on existing safety and efficacy data
    • Lack of availability of treatment alternatives
    • Compliance with national legal and regulatory requirements and/or IRB approvals
    • Technical feasibility considerations, for example the availability of adequate infrastructure and clinical expertise to administer autologous ex vivo gene therapy
    • Other criteria, such as the adequacy of company resources or the adequacy of product supply to complete the development programs and, thus, help ensure all appropriate patients have the chance to benefit from treatment.


  • All requests for pre-approval access must come through a patient’s physician. Requests from patients will not be able to be progressed and we will refer them to their treating physician.
  • To initiate a request for pre-approval access or for all other questions, please contact medinfo@orchard-tx.com.
  • We anticipate acknowledging receipt of requests sent to this email within five business days.


Orchard Therapeutics reserves the right to revise this policy at any time.