On Wednesday, the Institute for Clinical and Economic Review (ICER)
published its
Draft Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (arsa-cel) for the treatment of metachromatic leukodystrophy (MLD). ICER concluded there is high certainty of net health benefit, awarding the rarely seen clinical evidence rating of “A” for the use of arsa-cel in children with
presymptomatic late infantile-MLD and
presymptomatic early juvenile-MLD compared to usual care. When assessing arsa-cel in children with
early symptomatic early juvenile-MLD, ICER issued a clinical evidence rating of “C++,” indicating high certainty of at least comparable benefits compared with usual care.
Using a placeholder price of $2.8M, ICER’s preliminary cost-effectiveness analyses estimated that the cost per quality-adjusted life-year (QALY) gained for arsa-cel compared to usual care was $370,000 using a healthcare sector perspective and $381,000 using a modified societal perspective, exceeding commonly cited thresholds of cost-effectiveness. ICER is accepting stakeholder feedback on these preliminary findings through August 22, 2023.
On Tuesday, ICER
announced plans to assess the comparative clinical effectiveness and value of iptacopan and danicopan for the treatment of paroxysmal nocturnal hemoglobinuria. Stakeholder comments are being accepted on the Draft Scoping Document through August 14, and the topic will be reviewed by the California Technology Assessment Forum (CTAF) in February 2024.
Last week, the
Orphanet Journal of Rare Diseases published a
new study that explores the feasibility of using ICER’s
Evidence Rating Matrix when assessing the clinical evidence for orphan therapies for pediatric indications. Researchers identified 11 products with European Medicines Agency (EMA) orphan product designations and applied ICER’s Evidence Rating Matrix to compare and grade the degree of evidence uncertainty and the associated benefits and risks for each therapy. The authors found that using ICER’s Evidence Rating Matrix to grade orphan therapies according to their treatment benefit and evidence certainty was feasible but noted that such assessments involve subjective judgments based on heterogeneous evidence.
The authors also acknowledged that their resultant evidence ratings focused only on clinical benefit, excluding other factors of relevance when assessing the value of orphan therapies, including impact on families and carers, ethical aspects, the lack of alternative therapies, and a broader societal perspective. The authors concluded that their findings “could inform requests from regulatory and health technology assessment (HTA) bodies for evidence generation (in conjunction with conditional approvals) by helping to identify evidence gaps and uncertainties.” However, it is also important to consider the recognized limitations of the Evidence Rating Matrix, including subjectivity and the exclusion of comprehensive value elements, when using it to inform decision-making.
If you need assistance with all things value assessment or ICER-related, please contact
Kimberly Westrich.
