Procedure for applying for Viltolarsen Expanded Access
For United States Only
Physician must conduct assessment of patient eligibility. All eligibility must be fulfilled. No patient is guaranteed entry into the program by applying as other criteria are taken into consideration.
The physician must do the following:
- Register in the Early Access Portal and submit CV and Medical License to establish physician qualifications to oversee the investigational drug and to treat the patient according to the treatment plan. Pre-registration can be done at any time.
- Requests for viltolarsen can be made as of March 16, 2020. Submit the completed and signed eligibility questionnaire and accompanying laboratory documentation (per the eligibility questionnaire) to the Early Access System. Eligibility questionnaires submitted by email will not be accepted.
- Patients meeting all inclusion criteria and none of the exclusion criteria are eligible. Enrollment will be on a rolling basis.
- Notification of approved patients will be made by email to the applying Sponsor-Investigator.
- Documentation supporting inclusion and exclusion criteria must be submitted to EAC within 10 business days of approval. Failure to submit documentation or submission of incomplete documentation within this time period will result in delay of review.
- Physicians will be notified of patient selection or non-selection and the reason within 3 business days of EAS™ time-stamped receipt of the all required documentation.
- Physicians notified of patient non-selection will have the opportunity to appeal the decision.
Once notified of selection, the physician must agree to fulfill responsibilities associated with the administration, monitoring and reporting of viltolarsen. In addition, the physician must agree to the following:
- Obtaining informed consent, including fully informing the patient/parent/caregiver of the investigational nature of viltolarsen, known and unknown risks, and obtain agreement by parent/caregiver to comply with the expected visit schedule.
- Complete Form FDA 3926
- Obtain Letter of Authorization (LOA) from NS Pharma
- Submit the completed form, CV, LOA and treatment plan to the FDA.
- Obtain Individual Sponsor IND number from FDA
- Obtain IRB review and approval
Arrangements for drug shipment will be made upon furnishing Early Access Care with the FDA Letter of Approval with the IND number in the Early Access System.
Contact Information: A treating physician may submit questions or requests regarding Expanded Access by contacting the NS Pharma Expanded Access Call Center 1-877-539-8515 or by emailing firstname.lastname@example.org