Expanded Access Policy Introduction
- 1. An investigational product should be used as part of a clinical trial. However, if patient enrollment is not possible for reasons, such as patient ineligibility, lack of ongoing clinical trials or if enrollment in a clinical trial is not feasible (e.g. distance to a trial precludes access), expanded access offers a possible route for gaining access to an investigational product, subject to FDA’s review.
- 2. The best way for patients to be treated with Investigational Products is through participation in a clinical trial, please visit www.clinicaltrials.gov for more information about the trial and trial sites.
- 1. Contact Information. At this time, Helixmith does not have any investigational gene therapy product candidates available for expanded access. If you have questions about our investigational product candidates or expanded access, please submit them to firstname.lastname@example.org.
Request Procedures. Helixmith is not currently making its investigational gene therapy product candidates available on an expanded access basis anywhere in the world. Requests for expanded access to Helixmith’s investigational gene therapy product candidates must come from the patient’s treating physician because only physicians can enroll patients in a clinical trial or expanded access program (EAP).
General Criteria. Helixmith is not currently making its investigational gene therapy product candidates available on an expanded access basis anywhere in the world. In the event Helixmith Spark Therapeutics decides to consider making one or more of its investigational gene therapy product candidates available through an EAP, Helixmith will evaluate and respond to each expanded access request that it receives on a case-by-case basis using criteria that ensures such requests are considered in a fair and consistent manner.
- 3. Anticipated Timing. Helixmith anticipates that it will acknowledge receipt of any expanded access questions or requests within seven (7) business days of receipt.
Clinicaltrials.gov. As stated above, Helixmith is not currently making its investigational gene therapy product candidates available for expanded access use anywhere in the world. In the event that Helximith decides to make its investigational product candidates available on an expanded access basis, this policy will be updated with a hyperlink to the expanded access record on clinicaltrials.gov after such record becomes active.
The posting of this policy by Helixmith shall not serve as a guarantee of access to any specific investigational drug by any individual patient. As authorized by and in accordance with the 21st Century Cures Act, Heixmith may revise this posted expanded access policy at any time without prior notice.
Helixmith Expanded Access Policy
Helixmith focuses on discovering and developing novel gene therapy products. The following is Helixmith’s global expanded access policy for investigational therapies that are intended to treat serious diseases: