TEGSEDI - Akcea Therapeutics, Inc.

Akcea Therapeutics must ensure that healthcare providers, patients, pharmacies, and wholesalers-distributors comply with the following requirements:

Akcea Therapeutics must provide training to healthcare providers who prescribe TEGSEDI.

The training includes the following educational materials: Prescriber Training, Program Overview, Prescriber Knowledge Assessment. The training must be available online and by hard copy via fax or mail.

Akcea Therapeutics must provide training to pharmacies that dispense TEGSEDI.

The training includes the following educational material: Program Overview. The training must be available online and by hard copy via fax or mail.

To inform healthcare providers about the REMS and the risks and safe use of TEGSEDI, Akcea Therapeutics must disseminate REMS communication materials according to the table below:

To support REMS operations, Akcea Therapeutics must:

1. Authorize dispensing for each patient within every 90 calendar days but not later than every 115 calendar days from the date of first dispense of TEGSEDI documented by receipt of the Patient Status Form. If a completed Patient Status Form is not received to authorize the continuation of treatment within 115 days, the patient is not authorized to receive the drug until a completed form is received.
2. Establish and maintain a REMS website, www.TegsediREMS.com. The REMS website must include the capability to complete prescriber certification online, the capability to enroll and manage patients online including patient authorization status, report severe thrombocytopenia, serious bleeding with severe thrombocytopenia, and glomerulonephritis events online, and the option to print the Prescribing Information, and REMS materials. All product websites for consumers and healthcare providers must include prominent REMS-specific links to the REMS website. The REMS website must not link back to the promotional product website(s).
3. Make the REMS website fully operational and all REMS materials available through the website or call center by the date TEGSEDI is first commercially distributed.
4. Establish and maintain a REMS call center for REMS participants at 1-844-483-4736.
5. Establish and maintain a validated, secure database of all REMS participants who are enrolled and/or certified in the TEGSEDI REMS.
6. Ensure prescribers are able to become certified in the REMS by fax and online.
7. Ensure prescribers are able to enroll patients in the REMS by fax and online.
8. Ensure pharmacies are able to become certified in the REMS by fax.
9. Ensure pharmacies are able to obtain authorization to dispense by phone or online.
10. Ensure prescribers and pharmacies are able to report adverse events suggestive of severe thrombocytopenia, serious bleeding with severe thrombocytopenia, and glomerulonephritis by contacting the REMS by phone, online and fax, or using the Patient Status Form by online and fax.
11. Provide the Program Overview, Prescriber Training, Prescriber Enrollment Form, Patient Guide, Patient Enrollment Form, Wallet Card, and the Prescribing Information to prescribers who attempt to prescribe TEGSEDI and are not yet certified or inquire about how to become certified.
12. Provide the Pharmacy Enrollment Form and Program Overview and the Prescribing Information to pharmacies who are contracted with Akcea Therapeutics and attempt to dispense TEGSEDI and are not yet certified or inquire about how to become certified.
13. Notify prescribers and pharmacies within 48 hours after they become certified in the REMS.
14. Provide certified prescribers access to the database of their enrolled patients.
15. Provide certified pharmacies access to the database of certified prescribers and enrolled patients.
16. Provide public access to a database of certified pharmacies.
17. Establish and maintain a registry which includes a reporting and collection system for all patients to provide information on the incidence of 1) severe thrombocytopenia 2) serious bleeding with severe thrombocytopenia, and 3) glomerulonephritis.
18. Ensure that once a report suggestive of severe thrombocytopenia, serious bleeding with severe thrombocytopenia, or glomerulonephritis is received, Akcea Therapeutics follows-up with the healthcare provider to obtain all required data for the registry.

To ensure REMS participants’ compliance with the REMS, Akcea Therapeutics must:

1. Ensure the Patient Status Form is received for each patient. If the form is not received within every 95 calendar days of the date of the first dispense of TEGSEDI, Akcea Therapeutics must contact the prescriber to obtain the form. If the form is not received within every 115 calendar days, the patient is not authorized to receive the drug until the form is received.
2. Verify annually that the authorized representative’s name and contact information correspond to those of the current designated authorized representative for the pharmacy. If different, the pharmacy must be required to re-certify with a new authorized representative.
3. Maintain adequate records to demonstrate that REMS requirements have been met, including, but not limited to, records of: TEGSEDI distribution and dispensing; prescriber and pharmacy certification; patient enrollment; and audits of REMS participants. These records must be readily available for FDA inspections.
4. Establish and maintain a plan for addressing noncompliance with REMS requirements.
5. Monitor prescribers and pharmacies on an ongoing basis to ensure the requirements of the REMS are being met. Take corrective action if non-compliance is identified, including de-certification.
6. Audit pharmacies and wholesalers-distributors no later than 90 calendar days after they become certified/authorized to dispense and distribute TEGSEDI, respectively, and annually thereafter, to ensure that all REMS processes and procedures are in place, functioning, and support the REMS requirements.
7. Take reasonable steps to improve operations of and compliance with the requirements of the TEGSEDI REMS based on monitoring and evaluation of the TEGSEDI REMS.