|
BOSTON, March 26, 2025 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of sonpiretigene isteparvovec (Nanoscope Therapeutics) for the treatment of advanced retinitis pigmentosa.
“Many individuals with retinitis pigmentosa develop severe vision loss as the disease progresses and photoreceptor cells are lost,” said ICER’s Chief Medical Officer David Rind, MD. “Researchers have examined the idea of inserting proteins into other cells that remain in the back of the eye to allow those cells to react to light. Sonpiretigene isteparvovec is a one-time gene therapy that codes for such proteins in remaining retinal bipolar cells. We have a number of uncertainties about the efficacy and durability of this therapy given the limited evidence to date, but the underlying approach is remarkable.”
This Evidence Report will be reviewed at a virtual public meeting of the New England CEPAC on April 11, 2025. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
Register here to watch the live webcast of the virtual meeting.
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Clinical Findings
For adults with advanced retinitis pigementosa and severe vision loss, ICER rated the current evidence on sonpiretigene isteparvovec as promising but inconclusive (“P/I”) due to concerns about durability of benefits and unknown short-term and long-term harms.
Key Cost-Effectiveness Findings
Sonpiretigene isteparvovec has not yet been approved by the FDA for retinitis pigmentosa, and the manufacturers have not yet announced a US price for the therapy if approved.
ICER has calculated a health benefit price benchmark (HBPB) to be between $67,400 and $101,300 for treatment in one eye.
ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.
| 
