Section 5 of 6
Economic information and modeling report
31 evidence topics · 16 sources
Modeling overview
Summary
No published cost-effectiveness analysis or budget impact model of tovorafenib in any pLGG population was identified, and no health technology assessment has evaluated tovorafenib for first-line use. All tovorafenib HTA activity concerns relapsed or refractory pLGG. The EU Joint Clinical Assessment found no direct comparative data; the only included comparison, an unanchored matching-adjusted indirect comparison with dabrafenib plus trametinib in BRAF V600E-mutated disease, had effective sample sizes of 5.81 to 14.26. In Germany, the company-reported annual therapy cost of tovorafenib was €239,561.93 to €479,123.86 per patient, with 6 to 156 patients in the statutory health insurance target population; IQWiG judged the lower cost bound plausible and the upper bound overstated because tablets cost less than the suspension, and a G-BA decision is scheduled for early November 2026. The French HAS granted early access on July 2, 2026 for patients with BRAF-altered pLGG who progressed after at least 1 systemic treatment and were previously treated with, or ineligible for, dabrafenib plus trametinib, and the NICE appraisal (ID6557) was in scoping with no expected publication date.
For the comparator class, NICE (TA977, May 2024) recommended dabrafenib plus trametinib for BRAF V600E-mutated LGG in children and young people aged 1 year and over who need systemic treatment, with a confidential discount, a 1.2 severity weighting on QALYs, an acceptable ICER of around £30,000 per QALY, and confidential LGG ICERs judged to be within the cost-effective range. A company-sponsored analysis for England and Wales reported a probabilistic ICER of £26,606 per QALY (70.7% probability of cost-effectiveness at £30,000 per QALY). An independent Canadian microsimulation of first-line dabrafenib plus trametinib versus chemotherapy (TADPOLE efficacy; 2024 Canadian dollars; 1.5% discount rate) estimated 2.21 incremental QALYs at an incremental cost of CAD 554,769, a base case ICER of CAD 251,027 per QALY with lifetime treatment, and CAD 44,740 per QALY with 2 years of treatment; the authors concluded that a price reduction would be required. A CDA-AMC reimbursement review of dabrafenib plus trametinib for pLGG with BRAF V600 mutations (PX0375) was identified, but its pharmacoeconomic content could not be retrieved.
For sales, Day One reported U.S. OJEMDA net product revenue of $57.2 million in 2024 (launch in May 2024) and $155.4 million in 2025, with 4,635 prescriptions in 2025 and 2026 guidance of $225 million to $250 million. Management projections disclosed for the Servier tender offer assumed OJEMDA development as a front-line therapy and projected total company net revenue (including other products, royalties, and milestones) of $261 million in 2026, rising to a peak of $1,131 million in 2035. Day One estimated approximately 1,100 new U.S. BRAF-altered pLGG diagnoses per year in patients younger than 25 years and an addressable relapsed, progressive, or refractory pool of 2,000 to 3,000 patients per year. FIREFLY-2 enrollment was expected to complete in the first half of 2026, with data in mid-2027 and potential approval in 2028. Servier completed its acquisition of Day One on April 23, 2026 (equity value approximately $2.5 billion); no OJEMDA revenue figures for 2026 quarters were located.
OJEMDA U.S. net product revenue and prescriptions, 2024 and 2025
Revenue guidance for 2026 and first-line FIREFLY-2 timeline
Company statements on the U.S. patient population and market opportunity, including front-line use
Management long-range revenue projections disclosed in the Servier tender offer
Acquisition of Day One by Servier
Germany: annual therapy costs and target population for tovorafenib in relapsed or refractory pLGG (IQWiG assessment of the company dossier)
Germany: G-BA orphan drug benefit assessment procedure status
| Field | Quoted record |
|---|---|
| Start of procedure | “15.05.2026” |
| Publication of benefit assessment | “17.08.2026” |
| Decision | “Anfang November 2026” |
| Remarks | “Arzneimittel zur Behandlung eines seltenen Leidens (Orphan Drug)” |
EU Joint Clinical Assessment: absence of comparative evidence and indirect comparison uncertainty
France: HAS early access authorization
England: NICE appraisal of tovorafenib in development
England: NICE appraisal of dabrafenib plus trametinib (TA977) recommendation, model, and cost-effectiveness judgment
Cost-effectiveness of dabrafenib plus trametinib in BRAF V600E pLGG: England and Wales model
Cost-effectiveness of first-line dabrafenib plus trametinib versus chemotherapy in BRAF V600E pLGG: Canadian microsimulation
Cost-effectiveness analysis or budget impact model of tovorafenib
No evidence found.
Comparator, health states, and analytic conventions of the England and Wales model
Treatment duration and stopping assumptions in the England and Wales model
Drug acquisition and administration cost assumptions in the England and Wales model
Utility values derived from adult studies in the England and Wales model
Base-case incremental costs, quality-adjusted life-years, and severity weighting
Scenario and sensitivity analyses in the England and Wales model
Benefits of oral therapy not captured in the England and Wales model
Progression-free survival extrapolation in the England and Wales model
Post-progression survival by timing of first progression
Cost categories, monitoring, and malignant transformation costs in the England and Wales model
External validation and the evidence constraints acknowledged by the model authors
Modeled cohort characteristics used to cost chemotherapy
Line of therapy to which the cost-effectiveness result applies
Germany: evidence the G-BA orphan drug benefit assessment did not take into account
Earlier version of the Canadian microsimulation of first-line dabrafenib plus trametinib
CDA-AMC review: drug acquisition cost comparison and absence of a cost-effectiveness analysis for dabrafenib plus trametinib in pLGG
CDA-AMC review: trametinib patent expiry and dabrafenib monotherapy cost comparison
Budget impact model
Approach and framework
No evidence found.
Perspective and time frame
No evidence found.
Epidemiology and eligible population inputs
No evidence found.
Cost assumptions
No evidence found.
Model outcomes
No evidence found.
Results
Base case
No evidence found.
Scenario analyses
No evidence found.
Budget impact model discussion
No evidence found.