Three Things Texas Families Can Do This Week
Educational only—talk to your clinician.
Editor’s Note
This issue is about leverage—new rules you can cite, data you can quote, and questions you can ask so you’re not walking into appointments or phone calls alone. We have more than enough barriers just with daily life. So don’t gas up the old truck until you let your fingers do the walking (never heard that before right?)
Every link below goes to an official or peer‑reviewed source verified in January 2026.
1. The 72-Hour Rule in Real Life
Sarah in Longview has CIDP. In December, her “urgent” IVIG prior auth sat for 19 days while her legs got weaker. No callbacks, no decision—just “it’s still pending.” As of January 1, 2026, that delay is no longer allowed for many plans.
What Changed January 1, 2026
For Medicare Advantage, Medicaid managed care, and CHIP managed care, new CMS rules now require:
Urgent prior auth: decision within 72 hours
Standard prior auth: decision within 7 calendar days
Denials: must include a specific reason in writing
These timelines are often shorter than what plans used before, especially for standard requests.
How Families Are Using It
When Sarah’s neurologist resubmitted her IVIG as urgent on January 3:
Her family wrote down the submission date, time, and request ID.
On day 3 with no answer, they called and said:
“This is an urgent prior authorization under CMS‑0057‑F. The 72‑hour window has passed. I need an immediate decision or a written reason for delay, and I will be filing a complaint with the Texas Department of Insurance.”
The plan approved IVIG that afternoon.
What to Do This Week
Ask your doctor’s office: “Is this being submitted as urgent or standard?”
Write down: submission date, time, and prior auth/reference number.
Set phone reminders:
Urgent: 72 hours
Standard: 7 days
If the deadline passes, say:
“Under the CMS Interoperability and Prior Authorization Final Rule (CMS‑0057‑F), this request is overdue. I need a decision today or a written reason for delay.”If they stall, call Texas Department of Insurance and log a complaint.
Key source (full URL):
CMS Fact Sheet – CMS‑0057‑F
https://www.cms.gov/newsroom/fact-sheets/cms-interoperability-and-prior-authorization-final-rule-cms-0057-f
2. Gene Therapy Denials: Appeal Language You Can Use
A September 2025 JAMA study from Tufts CEVR reviewed 541 coverage policies from 18 large commercial insurers (~70% of the commercial market). It found that 52% of policies added restrictions beyond the FDA label for cell and gene therapies.
Extra restrictions were more common in non‑cancer indications (63%) than cancer (45%).
Among restrictive policies: 78% narrowed to specific subgroups; 10% required step therapy; 12% did both.
Why This Matters in Rare Neuro
For a rare neurologic gene therapy, this can look like:
“Must have failed 3+ prior therapies” (not on label)
“Must meet extra lab criteria not required by FDA”
These additions mean you can meet the FDA indication and still be denied because the plan invented extra rules.
Step-by-Step If You’re Denied
Ask for the actual policy:
“Please send me the exact coverage policy and criteria used for this decision, including any step‑therapy or subgroup requirements.”Pull the official FDA label:
Go to https://www.fda.gov
In the search bar, type your drug’s name and choose the prescribing information (label).
Use this appeal language (copy‑paste):
“Your denial relies on coverage criteria that are not present in the FDA‑approved indication. A JAMA research letter (Chambers JD et al., Sept 29, 2025) reviewing 541 US commercial policies found 52% added restrictions beyond FDA labeling, especially for non‑cancer rare conditions. I am requesting coverage consistent with the FDA‑approved label and current standards of care for my condition.”
Ask your neurologist for a letter that states:
Your diagnosis and how you meet the FDA indication.
Why delay or step therapy is unsafe in your case.
Clear statement that the therapy is medically necessary.
Attach that letter and the FDA label to your appeal.
Key sources (full URLs):
JAMA article (Chambers JD et al.)
https://jamanetwork.com/journals/jama/fullarticle/2839339
PubMed record
https://pubmed.ncbi.nlm.nih.gov/41021246/
Tufts CEVR summary
https://cevr.tuftsmedicalcenter.org/news/spec-cgt-coverage
3. AI in Trials: One Smart Question
In late 2025, FDA qualified its first AI drug development tool to support liver disease (MASH) trials. The tool (often referred to as AIM‑MASH) helps pathologists score liver biopsy images more consistently and quickly than manual review alone.
For rare diseases, including neurologic, AI‑type tools could eventually mean:
Faster trial readouts: AI can speed scoring of images or tissue samples.
More consistent results: Less variation between different readers.
More potential trial sites: Community hospitals without ultra‑specialized pathologists might participate.
FDA is also building broader AI rules, including draft guidance on AI‑enabled device software functions that clarifies what information FDA expects in submissions.
What to Ask Right Now
AI is not reading most MG/CIDP MRIs or EMGs yet, but it’s moving into:
Imaging‑heavy trials
Digital gait/speech/movement measures
When you talk to your neurologist or a trial coordinator, you can ask:
“Are any AI tools being used to read scans, EMGs, or videos in this trial—or for my condition in general?”
And when you search ClinicalTrials.gov, try:
[your condition] AND Texas
[your condition] AND AI
[your condition] AND digital biomarker
Key sources (full URLs):
FDA info on first AI drug development tool for MASH trials
https://www.fda.gov/drugs/drug-safety-and-availability/fda-qualifies-first-ai-drug-development-tool-will-be-used-mash-clinical-trials
FDA draft guidance – Artificial Intelligence‑Enabled Device Software Functions
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/artificial-intelligence-enabled-device-software-functions-lifecycle-management-and-marketing
PDF of the guidance
https://www.fda.gov/media/184856/download
Trial of the Week: New MG Study (Adults, Generalized MG)
Condition: Generalized myasthenia gravis (gMG)
Trial ID: NCT06909214
Treatment: Efgartigimod PH20 SC (subcutaneous FcRn blocker) in adults with new‑onset gMG
What this study is asking:
Can early use of subcutaneous efgartigimod improve symptoms and outcomes in adults recently diagnosed with generalized MG?
Who might qualify (high level):
Age 18 or older
New‑onset generalized MG (details in protocol)
On relatively stable background MG treatment
No recent major infections or uncontrolled medical issues
Texas angle:
This is a multicenter study. Major neuromuscular centers (like UT Southwestern or Houston Methodist) often participate in FcRn‑blocker trials. Families can:
Search directly:
https://clinicaltrials.gov/study/NCT06909214Or ask their neurologist:
“Are you, or any center nearby in Texas, a site for the efgartigimod PH20 SC new‑onset MG study NCT06909214?”
Micro‑Glossary (Save This)
Prior Authorization: Insurance approval needed before a treatment; now timed at 72 hours (urgent) or 7 days (standard) for many plans in Medicare Advantage, Medicaid managed care, and CHIP managed care.
FDA Labeling: Official FDA‑approved uses and patient criteria for a drug; you can download labels at https://www.fda.gov.
Gene Therapy: Treatment that alters or replaces genes; often costs $500K–$3M per patient and is frequently restricted beyond FDA labeling in commercial coverage.
AI Drug Development Tool: AI software FDA has qualified for use in drug trials (like the AI tool used in MASH histology scoring).
How We Report
Texas NeuroRare translates peer‑reviewed research and federal regulatory changes into practical, Texas‑focused guidance for rare neuro families. Every statistic is traceable to CMS, FDA, NIH, or peer‑reviewed journals, and every issue is updated to reflect rules as of January 2026.
This newsletter is educational only and does not provide medical advice. Always consult your healthcare provider.
Texas Resources (Verified January 2026)
Insurance Appeals (Texas Department of Insurance):
1‑800‑252‑3439
https://tdi.texas.gov
Medicare/Medicaid Questions:
Medicare: 1‑800‑633‑4227
Texas Medicaid: 1‑800‑252‑8263
Patient Advocacy:
National Organization for Rare Disorders (NORD)
https://rarediseases.org | 1‑800‑999‑6673
Clinical Trials (all conditions):
https://clinicaltrials.gov (search by condition + “Texas”)
Contact: [email protected]
Bolivar 2011
