Evicenter
P&T meetings

Tovorafenib

RAF-altered pediatric low-grade glioma requiring first-line systemic therapy

Also known as DAY101, TAK-580
Regulatory submission
Not announced
Launch
Not announced
155 sources

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.

Germany: G-BA orphan drug benefit assessment procedure status

England: NICE appraisal of dabrafenib plus trametinib (TA977) recommendation, model, and cost-effectiveness judgment

“Dabrafenib with trametinib is recommended, within its marketing authorisation, as an option for treating: • low-grade glioma (LGG) with a BRAF V600E mutation in children and young people aged 1 year and over who need systemic treatment” (opens the source at this quote in a new tab)

“The list price for dabrafenib is £2,800 per 420‑pack of 10 mg dispersible tablets (company submission). The list price for trametinib is £376 per 4.7 mg bottle of 0.05 mg per ml powder for oral solution (company submission).” (opens the source at this quote in a new tab)

“The company developed an individual-based state transition model which included analyses for both LGG and HGG cohorts. There were 3 health states common to both analyses: progression-free after first treatment, progressed and death.” (opens the source at this quote in a new tab)

“The committee concluded that severity weights of 1.2 (LGG) and 1.7 (HGG) applied to the QALYs were appropriate for this evaluation.” (opens the source at this quote in a new tab)

“So, the committee concluded that an acceptable ICER would be around £30,000 per QALY.” (opens the source at this quote in a new tab)

“In the LGG analysis, because of confidential commercial arrangements for other treatments in the model, the exact cost-effectiveness estimates are confidential and cannot be reported here.” (opens the source at this quote in a new tab)

“The committee concluded that treatment duration should reflect the marketing authorisations and a stopping rule should not be included for either LGG or HGG.” (opens the source at this quote in a new tab)

Cost-effectiveness analysis or budget impact model of tovorafenib

No evidence found.

CDA-AMC review: drug acquisition cost comparison and absence of a cost-effectiveness analysis for dabrafenib plus trametinib in pLGG

“For adult patients, based on public list prices, dabrafenib plus trametinib is expected to have a cost of $18,018 per patient per standardized 28-day cycle (Table 1), while vinblastine monotherapy and carboplatin plus vincristine are expected to have costs of $764 and up to $1,410 per patient per standardized 28-day cycle, respectively.” (opens the source at this quote in a new tab)

“For pediatric patients, no public prices were identified for dabrafenib 10 mg tablets for suspension or for trametinib 4.7 mg per bottle oral solution, both indicated for patients aged at least 1 year and weighing at least 8 kg.” (opens the source at this quote in a new tab)

“As such, the reimbursement of dabrafenib plus trametinib as first-line or later therapy for the treatment of LGGs in adult and pediatric patients with residual disease and with known BRAF V600 mutations is expected to increase overall drug acquisition costs.” (opens the source at this quote in a new tab)

“Given that dabrafenib plus trametinib is associated with increased drug acquisition costs and incremental benefit in terms of ORR and PFS compared to carboplatin and vincristine, a cost-effectiveness analysis would be required to determine the cost-effectiveness of dabrafenib plus trametinib relative to comparators.” (opens the source at this quote in a new tab)

“As this was not available, the cost-effectiveness of dabrafenib plus trametinib relative to carboplatin plus vincristine, vinblastine monotherapy, or dabrafenib monotherapy for the treatment of LGGs could not be determined.” (opens the source at this quote in a new tab)

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.