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BOSTON, September 14, 2023 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics) for metachromatic leukodystrophy (MLD).
“MLD is a devastating disease for children and their caregivers,” said ICER’s Chief Medical Officer, David Rind, MD. “It is extremely rare, and most doctors have never seen a case. Parent testimony shared throughout this report has provided a window into the hope and frustration that families are feeling as new treatments become available. With arsa-cel, we see a gene therapy again filling the promise of possible treatments for conditions that previously were untreatable.”
This Evidence Report will be reviewed at a virtual public meeting of the CTAF (CTAF) on September 29, 2023. The CTAF 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
All childhood forms of MLD are rapidly progressive and eventually fatal. We reviewed the clinical effectiveness of arsa-cel when compared to usual care for the treatment of three subpopulations of patients with MLD:
- Presymptomatic late infantile subtype (LI-MLD)
- Presymptomatic early juvenile form (EJ-MLD)
The evidence demonstrates that:
- The majority of presymptomatic LI and EJ-MLD patients who underwent arsa-cel therapy remained either asymptomatic or experienced mild symptoms. We therefore conclude that for children with these forms of MLD we have high certainty of a substantial net health benefit (“A”).
- For children with early symptomatic EJ-MLD evidence suggests that the magnitude and certainty of deriving benefit from treatment are lower, but there is high certainty that the treatment is better on average than usual care (“B+”).
Key Cost-Effectiveness Findings
Arsa-cel has not yet been approved by the FDA, and the manufacturer has not announced a US price if approved. ICER has calculated a health-benefit price benchmark (HBPB) for arsa-cel to be between $2.3M - $3.9M
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.
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