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BOSTON, October 30, 2023 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics) for metachromatic leukodystrophy (MLD). ICER’s report on these therapies was the subject of the September 2023 public meeting of the of the CTAF (CTAF), one of ICER’s three independent evidence appraisal committees.
Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations
“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. We again see a gene therapy potentially filling the promise of treating a disease that was previously untreatable, delivering remarkable clinical benefits and extending life. If the manufacturer prices arsa-cel in line with its price in Europe, despite being very expensive, arsa-cel would be cost-effective.”
ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations
ICER assessed, and the independent appraisal committee voted on, the evidence of arsa-cel for three types of MLD:
1. For children with presymptomatic late infantile MLD:
- All panelists (13-0) found that current evidence is adequate to demonstrate a net health benefit for atidarsagene autotemcel (arsa-cel) when compared to usual care.
2. For children with presymptomatic early juvenile MLD:
- All panelists (13-0) found that current evidence is adequate to demonstrate a net health benefit for atidarsagene autotemcel (arsa-cel) when compared to usual care.
3. For children with symptomatic early juvenile MLD:
- A majority of panelists (12-1) found that current evidence is adequate to demonstrate a net health benefit for atidarsagene autotemcel (arsa-cel) when compared to usual care.
For children with presymptomatic late infantile MLD or presymptomatic early juvenile MLD, panel members also weighed potential benefits and disadvantages beyond the direct health effects and broader contextual considerations. Voting highlighted the following as particularly important for payers and other policymakers to note:
- The acuity of need for treatment of individual patients based on short-term risk of death or progression to permanent disability;
- The magnitude of the lifetime impact on individual patients of MLD;
- The likelihood that these new treatments will improve patients’ broader ability to achieve major life goals related to education, work, or family life;
- The likelihood that these new treatments will improve caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life.
ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money
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), meaning a price range that matches the clinical benefit patients are expected to receive, for arsa-cel to be between $2.3M to $3.9M
Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for the treatment, the panel did not take separate votes on the treatment’s long-term value for money.
Key Policy Recommendations:
ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including the manufacturer, clinical experts, patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include:
- In the context of a rapidly progressive disease such as MLD, when a treatment has a high likelihood of being approved by the FDA, payers should be evaluating evidence and preparing policies in advance to avoid a new-to-market block on insurance coverage.
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Payers should cover fertility preservation in concert with coverage of gene therapies.
- Manufacturers should actively engage with independent value assessment efforts to allow public dialogue on access and fair pricing with broad input from patients and other stakeholders. Orchard Therapeutics has set a good example for other developers of transformative gene therapies.
ICER’s detailed set of policy recommendations, including comprehensive considerations for establishing evidence-based prior authorization criteria, is available in the Final Evidence Report and in the standalone Policy Recommendations document.
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