Mitotane
A relative of the insecticide DDT that is the only drug specific to adrenal cortex cancer; it needs blood-level monitoring and permanent steroid replacement.
Approved 1970. Adjuvant use for high-risk resected ACC (Terzolo 2007; ADIUVO 2023 negative for low risk), monotherapy for indolent advanced disease, and combined with EDP chemotherapy (FIRM-ACT). Therapeutic range 14-20 mg/L; toxicities include neurologic effects, GI intolerance, adrenal insufficiency (hydrocortisone replacement at higher doses), CYP3A4 induction affecting other drugs.
- Medicare
- Part D (self-administered)
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).
- Commercial insurance
- covered with prior authorisation
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. Adrenocortical carcinoma is rare enough that most plans cover on diagnosis.
- Assistance programmes
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Notes
- Only licensed drug for adrenocortical carcinoma; funded routinely without a TA.
- NHS England
- Routinely funded for the appraised indication (or via managed access)
Sources: NICE search: mitotane. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 1970 | Inoperable adrenocortical carcinoma (functional and non-functional) |
| EU | 2004 | Advanced adrenocortical carcinoma |
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Latest papers
topQuery for this drug: (TITLE:"Mitotane" OR ABSTRACT:"Mitotane" OR TITLE:"Lysodren" OR ABSTRACT:"Lysodren") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Mitotane, not a curated reading list.