The webinar, which starts at 1:00 pm (EST), will address such issues as the treatment’s high cost, insurance coverage regarding types of SMA, and community resources and support programs.
Registration is required, and patients, family members and other stakeholders are also asked to submit questions in advance. An opportunity to ask questions during an extended Q&A segment will be available at end of webinar presentation.
“We look forward to hosting an open, candid, and informal discussion of where our community stands on issues surrounding access to this first-ever approved therapy, and what our next steps will be,” CureSMA states on the webinar’s discussion page.
Spinraza was approved by the U.S. Food and Drug Administration (FDA) to treat SMA in both infants and adults in December 2016, the first medication to address the underlying genetic causes of the disease (SMA types 1–3 were included in clinical trials) and to show remarkable results in those studies.
One such trial, known as ENDEAR, tested the efficacy of Spinraza in 121 infants with SMA type 1, the disease’s most severe form. Data showed that 40% of treated infants achieved marked improvement in developmental milestones — in head control, crawling or walking ability, and other measures — compared to placebo.
Although the drug’s approval is welcome news, major concerns are being voiced regarding its price and insurance coverage in the U.S. Biogen is reported to have put the list price of Spinraza at $125,000 per vial, meaning that the first year of treatment could cost between $625,000 to $750,000 (five or six injections). Subsequent costs for this possibly lifelong therapy are expected to be about $375,000 annually, with about three injections required each year.
United Healthcare has said it will cover patients with SMA type 1, 2 and 3, but with specific limitations concerning initial and continued treatment use.
“We are working to help ensure no patient will forgo treatment because of financial limitations or insurance status,” Ligia Del Bianco, a spokeswoman for Biogen, told The New York Times in an interview.
CureSMA, a patient advocacy group, invested about $500,000 early research that led to the development of Spinraza.