Clinical review report (resubmission) : indication : treatment of patients with 5q spinal muscular atrophy. Nusinersen (Spinraza) (Biogen Canada Inc.) / Canadian Agency for Drugs and Technologies in Health.

Format:

Book

Author/Creator:

Canadian Agency for Drugs and Technologies in Health, author, issuing body.

Language:

English

Subjects (All):

Outcome assessment (Medical care).

Physical Description:

1 online resource (185 pages)

Edition:

Final (with redactions).

Place of Publication:

Ottawa (ON) : Canadian Agency for Drugs and Technologies in Health, 2019.

Summary:

On December 20, 2017, the CADTH Canadian Drug Expert Committee (CDEC) issued a final recommendation regarding Spinraza treatment for patients diagnosed with SMA. The recommendation was based on evidence presented in a CADTH Common Drug Review (CDR) report of Spinraza. CDEC identified the following areas as constituting an evidence gap: patients with symptom onset at birth or within one week of birth; patients with advanced SMA who require ventilation; patients older than seven months of age; patients with more than two copies of the SMN2 gene; patients diagnosed at later stages of disease; and patients who are pre-symptomatic. A copy of the CDEC original recommendation can be viewed in Appendix 4. The manufacturer provided this resubmission with additional data and information from ongoing and recently conducted studies that were not available during the original submission. In light of new the information, this resubmission has been conducted with the primary goal of attempting to provide and assess any clinical studies that can fill the evidence gaps identified by CDEC.

Notes:

Description based on publisher supplied metadata and other sources.