Ert Pompe denied for failing step therapy by Humana?

Humana's specific coverage criteria for ert pompe are defined in its own published medical/coverage policy and the FDA-approved prescribing label. A successful appeal documents that your medical records satisfy each criterion those sources list — confirmed diagnosis, any required prior treatments (with dates and outcomes), and clinical severity. If the exact criteria weren't included with your denial, request them in writing; your appeal then maps each requirement to the matching fact in your chart.

## Why Humana Applies Step Therapy to Pompe Disease ERT — and Why This Denial Is Unusually Vulnerable

Step-therapy denials require a patient to try and fail a preferred alternative before the requested therapy is approved. For enzyme replacement therapy in Pompe disease, a step-therapy requirement is particularly difficult for Humana to defend because the FDA-approved treatment options for Pompe disease are limited in number and are not interchangeable in the way that competing agents in large drug classes are. If Humana's step-therapy protocol requires a different ERT product first, the appeal can argue that the patient has already tried or has a clinical reason to avoid that product, or that the products are not therapeutically equivalent for this patient's specific circumstances.

Many states have enacted step-therapy exception laws applicable to state-regulated plans, and federal non-interference provisions create appeal pathways even in ERISA plans when the clinical exception criteria are met.

## Federal Appeal Rights

ACA Section 2719 requires internal and external review for adverse benefit determinations on non-grandfathered plans. ERISA Section 503 guarantees a full-and-fair review with access to the specific criteria used. External review must generally be requested within four months of the final internal adverse determination. Expedited review is available when the standard timeline would seriously jeopardize health — Pompe disease's progressive and potentially irreversible respiratory and motor decline is a strong basis for expedited treatment.

## What to Gather

• Diagnosis confirmation — enzyme-activity assay, genetic testing, or biopsy establishing Pompe disease and specifying the disease variant (infantile-onset vs. late-onset, if relevant to the prescriber's product choice).
• Prior ERT history — if the patient has been on any prior ERT product, document the product name, dates of treatment, clinical response, and reason for transition. If the patient has not tried the step-therapy required product, document the prescriber's clinical rationale for bypassing it.
• Prescriber step-therapy exception letter — the physician should address Humana's specific step-therapy criteria and invoke the applicable exception basis: prior trial and failure, clinical contraindication, or clinical inappropriateness.
• Disease severity and progression documentation — pulmonary function tests, motor assessments, and functional measures showing the current disease burden and why delay poses a clinical risk.
• Humana's step-therapy and exception policy — obtain the current version and identify each step-therapy requirement and exception pathway.

## Criteria-Mapping Structure

Build a table: left column lists each step-therapy requirement and each exception criterion from Humana's policy; right column answers each with the specific chart fact, prescriber attestation, or clinical rationale. For any step that is claimed as "already tried," provide dates, product name, and documented outcome. For any step bypassed on clinical grounds, provide the physician's specific documented reasoning.

## Process and Timeline

1. File the written internal appeal within Humana's stated deadline, with the complete criteria-mapping package. 2. Request the specific clinical-review rationale and the policy version Humana used — you are entitled to these under ERISA. 3. If denied internally, file for external review within four months of the final adverse determination. 4. Assess the expedited track given the progressive nature of Pompe disease and the clinical risk of an extended treatment delay.