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Appeal outcomes · Biologics · Genetic Diseases

Biologics for Genetic Diseases: 92.9% of denials overturned

In 14 California IMR decisions from 2019 to 2025, reviewers overturned 13 (92.9%). In the last five years: 90.0% of 10. 10 were medical-necessity disputes, 4 experimental/investigational.

By year (last five)

YearDecisionsOverturned
202110.0%
20232100.0%
20243100.0%
20254100.0%

What the findings mention

What the findings mentionDecisionsOverturnedAll decisions here
Published evidence citedThe findings refer to peer-reviewed or published evidence.580.0%92.9%

Keyword matches on the reviewer’s findings, not causes. A tag that appears mostly in overturned decisions is worth documenting in an appeal; it is not a guarantee.

From recent overturned decisions

The physician reviewer found that Nature of Statutory Criteria/Case Summary: A patient has requested authorization and coverage for Strensiq. A review of the medical literature reveals that a low alkaline phosphatase level suggests hурοрhοѕрhatаsiа as the underlying etiology in the setting of a clinical presentation with low bone density and fragility fractures. In patients not taking a vitamin B6 (pyridoxine) …

Reviewer findings, overturned decision · Medical Necessity · 2025 · Source: California DMHC IMR determinations (CHHS Open Data), reference MN25-43588

Nature of Statutory Criteria/Case Summary: The parent of the patient has requested authorization and coverage for gene therapy with delandistrogene moxeparvovec-rokl (Elevidys). Based on the records, gene therapy with delandistrogene moxeparvovec-rokl (Elevidys) is likely to provide greater clinical benefit for this patient with Duchenne muscular dystrophy (DMD) compared to any available standard therapy. Elevidys is U.S Food and Drug Administration …

Reviewer findings, overturned decision · Experimental/Investigational · 2025 · Source: California DMHC IMR determinations (CHHS Open Data), reference EI25-44784

The physician reviewer found that Nature of Statutory Criteria/Case Summary: A patient has requested authorization and coverage for intravenous (IV) infusion Elevidys (delandistrogene moxeparvovec-rokl).The records indicate that this patient has been diagnosed with DMD with a confirmed mutation in the DMD gene. The patient’s provider has recommended treatment with Elevidys. As noted in the U.S. Food and Drug Administration (FDA) …

Reviewer findings, overturned decision · Experimental/Investigational · 2025 · Source: California DMHC IMR determinations (CHHS Open Data), reference EI25-43994

And one the reviewer upheld

Nature of Statutory Criteria/Case Summary: An enrollee has requested authorization and coverage for Prolastin-C infusion augmentation therapy.Findings: The physician reviewer found that the submitted medical records and laboratory data do not support that the requested intravenous augmentation therapy is medically necessity for the patient. The current peer reviewed literature supports that augmentation therapy is not recommended for patients who are …

Reviewer findings, overturned decision · Medical Necessity · 2021 · Source: California DMHC IMR determinations (CHHS Open Data), reference MN21-34777
Read these numbers carefully
  • California only: these are decisions by independent reviewers under California's IMR program (plans regulated by the Department of Managed Health Care). Other states and self-funded employer plans use different reviewers and rules.
  • Selection: only denials that a member took all the way to IMR appear. Most denials are never appealed, and the ones that reach an external reviewer are not a random sample.
  • Overturned means the reviewer disagreed with the plan; it does not mean the treatment worked or that a similar request will be approved.
  • Argument tags are keyword matches on the reviewer's findings. They describe what the findings mention, not why the case was decided.
  • Categories are DMHC's own labels (treatment sub-category and diagnosis category); 'Other' is a catch-all and is excluded from rankings.

Source: California Department of Managed Health Care, Independent Medical Review (IMR) Determinations, Trend (CHHS Open Data). Public California government data; Apellica's aggregates and tags are CC BY 4.0.

Questions

How often were Biologics denials for Genetic Diseases overturned?

In 14 California IMR decisions from 2019 to 2025, reviewers overturned 13 (92.9%). In the last five years: 90.0% of 10. 10 were medical-necessity disputes, 4 experimental/investigational.

What should an appeal document, based on these findings?

Whatever the plan's criteria ask for, shown in the record: the treatments already tried and their results, contraindications to the plan's preferred option, the guideline or evidence that supports the request, and the treating clinician's reasoning written to the criteria. The tag table shows which of these the reviewers mentioned most in overturned cases.

Does a California IMR result apply to my plan?

Only California plans regulated by the Department of Managed Health Care go to IMR. Other states and self-funded employer plans use different reviewers, but they weigh the same things.

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Independent reviewers reverse plans when the record answers the criteria. Upload the denial letter; a senior reviewer reads it within 24 hours and tells you in writing whether it can be appealed and how. $0 upfront, 10% of what is recovered, nothing if we do not recover. Not a law firm.