Ert Pompe denied for missing prior authorization by UnitedHealthcare?

UnitedHealthcare'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 UnitedHealthcare Requires Prior Authorization for ERT in Pompe Disease

Prior authorization (PA) for enzyme replacement therapy in Pompe disease is expected — virtually all commercial plans require it for ultra-rare, high-cost specialty biologics. A PA-required denial means either: (1) no PA request was submitted before the infusion or prescription was dispensed; (2) a PA was submitted but is pending or was denied on clinical criteria; or (3) a previously granted PA expired and was not renewed in time.

Importantly, a PA denial is different from a coverage denial: PA is an administrative gateway, and the path through it is well-defined.

## Why This Denial Is Appealable

If the PA was denied on clinical criteria, you have the full appeal rights described below. If the PA was simply never requested (for example, in an emergency infusion), you may be eligible for a retroactive PA — request this first, as it is faster than a full appeal. If the denial is for an expired PA on a therapy that is working and ongoing, document continuity of care as the basis for renewal.

## Federal Appeal Framework

• ERISA §503 (employer plans): A PA denial is a coverage adverse action subject to full-and-fair internal review.
• ACA §2719: External review by an accredited IRO is available after internal appeals are exhausted.
• The external-review window is generally within approximately four months of the denial notice — check your Explanation of Benefits.
• Expedited PA and expedited appeal: If delaying treatment would seriously jeopardize health — which is readily arguable for a progressive disease like Pompe — you are entitled to an expedited PA determination (typically 72 hours) and an expedited appeal. Request both in writing.

## Concrete Appeal Steps

1. Obtain UHC's prior-authorization criteria for this drug — typically published in their coverage policies or available by calling the PA line. 2. Have your specialist's office submit a new or corrected PA with complete documentation (see below) if the original was incomplete. 3. If the PA is formally denied, file an internal appeal within the deadline on the denial notice. 4. Simultaneously request expedited review given disease severity. 5. Proceed to external review if the internal appeal fails.

## Documentation to Gather

• Diagnosis confirmation: Genetic report confirming pathogenic GAA variants; enzyme assay confirming acid alpha-glucosidase deficiency; clinical phenotype characterization (infantile-onset vs. late-onset).
• Baseline functional assessments: Pulmonary function tests, motor assessments (six-minute walk or equivalent), and any respiratory support history — with dates.
• Disease progression documentation: Serial measurements showing decline or risk of imminent decline without treatment.
• Prescriber medical-necessity letter: Signed letter from the treating metabolic or neuromuscular specialist addressing each PA criterion by name, with the specific chart finding that satisfies it.
• Prior-treatment history: If transitioning from another ERT, document dates, clinical response, and reason for change.
• PA form: Completed PA request form with all required fields — incomplete forms are a leading cause of denial.

## Criteria-Mapping Structure

PA criteria for ERT in Pompe disease typically require confirmed diagnosis, documented functional impairment, and specialist oversight. Map each:

| PA Criterion | Documentation Submitted | |---|---| | Confirmed diagnosis (genetic + enzyme) | Genetic report [date]; enzyme assay [date] | | Functional impairment documented | Pulmonary function report [date]; motor assessment [date] | | Prescribing specialist identified | [Name, specialty, NPI] | | Ongoing monitoring plan | Specialist letter describes follow-up schedule |

A complete, criterion-matched submission resolves the majority of ERT prior-auth denials at the initial request stage.