CPAP Module 23, Section 2: Neurology Case: Step Therapy Appeal
MODULE 23: SPECIAL TOPICS & CASE SIMULATIONS

Section 2: Neurology Case: Step Therapy Appeal

Beyond the denial: Constructing an evidence-based appeal to overcome payer protocols and champion patient care.

SECTION 23.2

Neurology Case: Step Therapy Appeal

Mastering the art of the clinical argument to prove that “fail first” is not always “patient first.”

23.2.1 The “Why”: The Step Therapy Gauntlet

If the oncology prior authorization was a test of your ability to gather precise, positive evidence, the step therapy appeal is a test of your ability to prove a negative. You are no longer just proving that a patient qualifies for Drug C; you are proving, with objective evidence, that they have a clinically valid reason for not taking the payer’s preferred Drug A and Drug B. This represents one of the most common, frustrating, and critically important battles you will fight as a PA pharmacist, especially in chronic neurologic diseases like multiple sclerosis, epilepsy, and migraine.

Step therapy, often called a “fail first” policy, is a core cost-containment strategy for payers. It mandates that a patient must first try and fail one or more lower-cost, “preferred” medications before the insurer will consider covering a newer, more expensive alternative. From a population health perspective, the logic is simple: if 70% of patients do well on the cheaper generic, the health system saves an enormous amount of money. The problem, as you know intimately from your pharmacy practice, is for the 30% who don’t. For them, a step therapy protocol is not a cost-saving measure; it is a mandated period of inadequate treatment, a gauntlet of side effects, or a delay in accessing the most effective therapy for their specific condition.

In neurology, this is particularly perilous. For a patient with relapsing-remitting multiple sclerosis (RRMS), a “failure” on a preferred platform therapy isn’t just an inconvenient side effect; it can mean a new relapse, resulting in irreversible nerve damage, vision loss, or decreased mobility. For a patient with epilepsy, a “failure” on a preferred anti-epileptic can mean a breakthrough seizure, leading to injury, loss of a driver’s license, and profound disruption to their life. The clinical stakes of a forced failure are immense.

Your role in this process is to be the patient’s advocate and the clinical historian. The neurologist knows the patient should be on the new medication, but they are often too busy to meticulously document years of treatment history into a format an insurance reviewer can understand. You are the bridge. You must dive into the patient’s record, excavate the proof of past failures, and assemble it into a compelling, evidence-based narrative that satisfies the payer’s rigid criteria for an exception. This requires a different set of skills: not just finding data, but interpreting a long-term clinical story and framing it in the language of the payer’s policy. Successfully overturning a step therapy denial is a victory that prevents harm and ensures the patient receives the care their specialist has determined is medically necessary.

Retail Pharmacist Analogy: The Antihistamine Override

A regular patient, a long-haul truck driver, comes to your counter with a prescription for fexofenadine (Allegra). You run the claim, and it’s rejected with a message: “STEP THERAPY REQUIRED. PREFERRED AGENTS: LORATADINE, DIPHENHYDRAMINE.” You know the patient can’t take diphenhydramine; it’s highly sedating and would endanger his career and his safety. Loratadine is non-sedating, but will it work?

You call the patient over. “Hi Mr. Jones, it looks like your insurance wants you to try a different allergy pill first. They suggest loratadine.” Mr. Jones scoffs. “I tried that for a month last year. It was like taking a sugar pill. My allergies were just as bad. That’s why Dr. Smith switched me to Allegra in the first place.”

You now have the basis for an appeal. The patient has already “failed” one of the preferred agents. But you can’t just write “patient tried it” on a claim. You need to make the case. You pull up his profile. Sure enough, you see a cash-pay fill for a 30-day supply of loratadine eight months ago. This is your proof of trial.

You then call Dr. Smith’s office. This is your clinical clarification call. “Hi, this is the pharmacist calling about Mr. Jones’s fexofenadine. His insurance is requiring a trial of loratadine and diphenhydramine first. Mr. Jones states he already failed loratadine due to lack of efficacy, which I can see he purchased here. Can you please confirm this lack of efficacy and also confirm that diphenhydramine is clinically inappropriate due to his occupation as a truck driver?”

The nurse confirms both points. Dr. Smith documented the loratadine failure in the chart. They agree diphenhydramine is contraindicated. You now have your complete argument. You call the insurance plan’s pharmacy help desk for a “prior authorization override.” This is your appeal submission.

You state your case clearly and logically: “Patient has a prescription for fexofenadine. Per your step therapy policy, he is required to try loratadine and diphenhydramine. Point 1: He has completed a trial of loratadine in [Month, Year] with documented lack of efficacy per the prescriber’s chart note. Point 2: A trial of diphenhydramine is contraindicated due to its sedative effects, which would be dangerous given the patient’s occupation as a commercial truck driver.”

The agent on the phone processes the override. You have successfully navigated the step therapy requirement by providing a documented history of failure on one agent and a clear clinical contraindication to the other. This process of identifying the payer’s required steps, finding the historical evidence of failure, and articulating a clinical rationale for avoiding other preferred agents is the exact skill set required to appeal a neurology step therapy denial.

23.2.2 The Case Presentation: Meet Mrs. Chen

A prior authorization request for a new multiple sclerosis therapy is routed to your work queue. The initial submission by the neurology clinic was automatically rejected at the point of prescribing.

Patient Demographics

  • Name: Emily Chen
  • Age: 45 years old
  • Insurance: MedSecure Advantage Gold PPO (Plan ID: MSA45B789)
  • Prescribing Physician: Dr. Marcus Thorne, MD (Neurology)

The Submitted Prescription

  • Drug: Ocrelizumab (Ocrevus)
  • Dose: 600 mg
  • Route: Intravenous (IV) Infusion
  • Frequency: Every 6 months (after initial loading doses)
  • Diagnosis Code Attached: G35 – Multiple Sclerosis

Attached to the PA case is the automated denial letter generated by MedSecure Advantage’s system.

Denial Notification: Ocrelizumab

Date: [Today’s Date]

Status: NOT APPROVED

Reason for Denial: The request for Ocrelizumab (Ocrevus) is not approved as the patient has not met the requirements of MedSecure Advantage’s Step Therapy Program (Policy #NEURO-017). Our policy requires a trial and failure, or a documented contraindication, to at least TWO preferred formulary agents prior to consideration of Ocrelizumab.

Preferred Formulary Agents Include:

  • Interferon beta-1a (e.g., Avonex, Rebif)
  • Glatiramer Acetate (e.g., Copaxone, generic)
  • Teriflunomide (Aubagio)
  • Dimethyl Fumarate (Tecfidera)

Action Required: To appeal this decision, please submit clinical documentation detailing the dates of trial and specific reasons for discontinuation (lack of efficacy, intolerance, or contraindication) for two preferred agents. You may submit your appeal via the provider portal.

23.2.3 The Mission: Deconstructing the Denial & Payer Policy

The denial is clear, direct, and completely expected. The payer isn’t questioning the diagnosis or the appropriateness of Ocrevus in a vacuum; they are enforcing a procedural rule. Your mission is to prove that Mrs. Chen has already completed this procedure. Your first step, as always, is to pull the full clinical policy to understand the exact definitions and documentation requirements.

MedSecure Advantage Policy #NEURO-017: Disease-Modifying Therapies for MS

MedSecure Advantage considers ocrelizumab medically necessary for relapsing forms of multiple sclerosis when the following criteria are met:

  1. Diagnosis Confirmation: Patient has a confirmed diagnosis of a relapsing form of MS (including RRMS, active SPMS) consistent with McDonald criteria.
  2. Step Therapy Requirement: The patient has a history of trial and failure of, or a documented contraindication to, at least TWO of the following preferred agents:
    • An interferon beta product (e.g., Avonex, Rebif, Plegridy, Betaseron)
    • Glatiramer acetate (Copaxone, generic)
    • Teriflunomide (Aubagio)
    • Dimethyl fumarate (Tecfidera)
  3. Definition of “Failure”: Failure is defined as ONE of the following occurring while on a stable dose of a preferred agent for at least 6 months:
    • A) Clinical Relapse: At least one documented clinical relapse (e.g., optic neuritis, transverse myelitis).
    • B) Radiographic Progression: Development of one or more new T2 or gadolinium-enhancing lesions on MRI compared to a previous scan.
    • C) Intolerable Adverse Effects: Discontinuation of the agent due to a documented adverse effect that could not be managed (e.g., persistent flu-like symptoms, significant LFT elevation, severe injection site reactions).
  4. Provider: The medication must be prescribed by or in consultation with a neurologist.
The Appeal Evidence Checklist

This policy gives you the precise, granular list of what you need to prove. You are no longer looking for one thing; you are looking for a complete story with two chapters.

For Preferred Drug #1 (e.g., Interferon beta-1a):

  • Proof of Trial: Find evidence of when she started and stopped the drug. Need at least a 6-month duration.
  • Proof of Failure: Find the clinical or radiographic evidence for WHY she stopped it. Was it a documented relapse in a progress note? New lesions on an MRI report? A note detailing intolerable side effects?

For Preferred Drug #2 (e.g., Glatiramer Acetate):

  • Proof of Trial: Find evidence of when she started and stopped the drug. Need at least a 6-month duration.
  • Proof of Failure: Find the clinical or radiographic evidence for WHY she stopped it. Was it a documented relapse in a progress note? New lesions on an MRI report? A note detailing intolerable side effects?

Your investigation is now a two-part historical reconstruction.

23.2.4 The Investigation: Rebuilding the Treatment Timeline

You now enter the EMR with a clear objective: to find the story of Mrs. Chen’s MS treatment over the past several years. This requires a different search technique than the oncology case. You must use the EMR’s search functions and timeline views to piece together a chronological narrative.

Masterclass Table: EMR Forensics for a Step Therapy Appeal
Evidence Needed EMR Search Strategy Pharmacist’s Action & Interpretation
Drug #1: Identify the first DMT “Medications” Tab -> “Historical” or “Discontinued” view. Filter the medication list to show all medications, not just active ones. Sort by date. You are looking for the first MS-specific therapy ordered.
For Mrs. Chen: You find an order for Interferon beta-1a (Avonex) 30 mcg intramuscularly once weekly. Start Date: June 2018. Discontinued Date: October 2019. The duration was ~16 months, which is greater than the required 6 months. Trial Duration CHECK.
Drug #1: Find Reason for Discontinuation “Notes” -> “Neurology.” Search notes around the discontinuation date (Oct 2019). Open the progress notes from Dr. Thorne around October 2019. Use CTRL+F to search for keywords like “Avonex,” “interferon,” “relapse,” “MRI,” “lesions.”
For Mrs. Chen: You find a note from October 15, 2019. Dr. Thorne writes: “Patient presents for follow-up. Despite being adherent to Avonex for over a year, she reports a 3-week episode of numbness and weakness in her left leg last month, consistent with a clinical relapse. We will obtain a new brain MRI to assess for new radiographic activity. Plan to switch therapy.” This documents a clinical relapse. Failure Reason CHECK.
Drug #2: Identify the second DMT “Medications” Tab -> Follow the timeline from the last drug. Look for the next DMT that was started after Avonex was stopped.
For Mrs. Chen: You find an order for Glatiramer Acetate (Copaxone) 40 mg subcutaneously three times weekly. Start Date: November 2019. Discontinued Date: May 2021. The duration was 18 months, greater than the required 6 months. Trial Duration CHECK.
Drug #2: Find Reason for Discontinuation “Media” or “Radiology” Tab. Look for MRI reports. The previous note mentioned ordering an MRI. You need to find the results of MRIs performed while she was on Copaxone. Compare the report from just before she started Copaxone to the most recent one.
For Mrs. Chen: You find an MRI report from October 2019 (baseline before Copaxone) and another from April 2021. The radiologist’s impression on the April 2021 report reads: “Compared to the prior study of October 2019, there are TWO new T2 hyperintense, non-enhancing lesions within the periventricular white matter. These findings are indicative of disease progression.” This documents radiographic progression. Failure Reason CHECK.

23.2.5 Constructing the Appeal: The Letter of Medical Necessity

You have successfully unearthed the complete story. Mrs. Chen didn’t just try two preferred agents; she definitively failed both of them according to the payer’s own explicit definitions. Your task is now to write a formal appeal letter that presents this timeline and evidence so clearly that the denial cannot possibly be upheld.

This letter is your legal brief. It should be professional, factual, and relentlessly logical. You will cite your evidence like a scholar, guiding the reviewer through the history you have uncovered.

The Step Therapy Appeal Letter: A Master Template

SUBJECT: Appeal of Denial for Ocrelizumab for Emily Chen (ID: MSA45B789) – Failure to Meet Step Therapy

To Whom It May Concern,

I am writing to formally appeal your denial of coverage for ocrelizumab (Ocrevus) for the treatment of Relapsing-Remitting Multiple Sclerosis for the above-named patient. Your denial, dated [Today’s Date], cited a failure to meet the step therapy requirements outlined in MedSecure Advantage Policy #NEURO-017. This letter will provide clear clinical documentation demonstrating that the patient has, in fact, failed two preferred formulary agents as defined by your policy.

Summary of Documented Treatment Failures:

  1. Failure of Preferred Agent #1: Interferon beta-1a (Avonex)
    The patient was treated with Avonex 30 mcg weekly from June 2018 to October 2019 (16 months). This therapy was discontinued due to a lack of efficacy, evidenced by a clinical relapse.
    EVIDENCE: As documented in the attached neurology progress note from October 15, 2019, Dr. Thorne notes that the patient experienced a “3-week episode of numbness and weakness in her left leg…consistent with a clinical relapse” while adherent to Avonex therapy. This meets your policy’s definition of failure under section 3A.
  2. Failure of Preferred Agent #2: Glatiramer Acetate (Copaxone)
    Following the failure of Avonex, the patient was treated with Copaxone 40 mg 3x weekly from November 2019 to May 2021 (18 months). This therapy was discontinued due to a lack of efficacy, evidenced by radiographic disease progression.
    EVIDENCE: As documented in the attached brain MRI report from April 28, 2021, the interpreting radiologist states, “Compared to the prior study of October 2019, there are TWO new T2 hyperintense…lesions within the periventricular white matter. These findings are indicative of disease progression.” This meets your policy’s definition of failure under section 3B.

Having clearly documented failures of two preferred agents from two different medication classes (an interferon and a synthetic polypeptide), Mrs. Chen has fulfilled the requirements of Policy #NEURO-017. Her neurologist, Dr. Thorne, has now determined that escalating to a high-efficacy, B-cell depleting therapy like ocrelizumab is the medically necessary next step to control her active disease.

We have attached the relevant progress notes and MRI reports for your review. We respectfully request that you overturn this denial and issue an approval for ocrelizumab so that this necessary treatment is not delayed. Thank you for your time and reconsideration.

Sincerely,
[Your Name], PharmD
Prior Authorization Pharmacist

23.2.6 The Final Step: The Peer-to-Peer Review

In many cases, a well-documented written appeal like the one above will be sufficient to overturn a denial. However, for very high-cost therapies, some payers may uphold the denial, forcing a final escalation: the peer-to-peer (P2P) review. This is a scheduled telephone call between the prescribing physician (Dr. Thorne) and a physician employed by the insurance company (a medical director).

The P2P is the ultimate test of the case you have built. The neurologist is the one making the call, but your work as the PA pharmacist is what will determine its success. A physician juggling a full clinic schedule does not have time to re-read two years of chart notes right before the call. Your final and most crucial role is to prepare them for this conversation. You must create a “P2P Briefing Sheet” that distills your entire investigation into a one-page, high-yield summary that the doctor can have in front of them during the call.

The Purpose of the Peer-to-Peer

It’s crucial to understand what a P2P is and isn’t. It is not an academic debate about the merits of different MS drugs. The insurance company’s medical director is rarely a neurologist. Their job is to be a policy expert. They are on the call with a checklist, looking to see if the case meets the documented criteria for an exception. They are not there to be convinced that Ocrevus is a good drug. They are there to be shown that the patient failed Avonex and Copaxone according to the policy’s definitions.

Your briefing sheet, therefore, should not be a clinical treatise. It should be a concise guide to the evidence that proves the policy criteria have been met.

The Pharmacist’s P2P Briefing Sheet for the Neurologist

To: Dr. Thorne

From: [Your Name], PA Pharmacist

RE: Peer-to-Peer Call for Emily Chen – Ocrelizumab Appeal

Date of Call: [Date], Time: [Time]

Dr. Thorne, here is a summary of the key evidence to support the ocrelizumab appeal for Mrs. Chen. The MedSecure medical director will need confirmation that she has failed two preferred agents per their policy.

Key Talking Points:

1. Failure of Avonex (Interferon beta-1a):

  • Treatment Dates: June 2018 – October 2019.
  • Reason for Discontinuation: Clinical Relapse.
  • Key Evidence to Cite: Your progress note from 10/15/2019 documents a clinical relapse (left leg weakness/numbness) while the patient was on therapy.

2. Failure of Copaxone (Glatiramer Acetate):

  • Treatment Dates: November 2019 – May 2021.
  • Reason for Discontinuation: Radiographic Disease Progression.
  • Key Evidence to Cite: The brain MRI from 04/28/2021, when compared to the baseline from 10/2019, showed two new T2 lesions, confirming breakthrough disease activity.

Anticipated Questions & Suggested Responses:

  • “Did you try another preferred agent, like Tecfidera?” -> “No, after failing two first-line agents from different classes with two different mechanisms of failure—a clinical relapse on an interferon and radiographic progression on glatiramer—the standard of care and my clinical judgment indicated that escalating to a high-efficacy B-cell depleting therapy was the necessary next step to prevent further irreversible disability.”
  • “Were the side effects of Avonex managed?” -> “The discontinuation was not due to side effects but due to a definitive lack of efficacy as evidenced by a clinical relapse.”

By presenting these two clear, evidence-backed failures, you will have definitively met their policy requirements. Please let me know if you need the source documents sent to you for reference during the call. Thank you.

By providing this level of support, you transform the P2P from a potential burden on the physician into a quick, efficient, and successful checkmate. You have done the legal and historical research, and simply handed the lawyer the two key precedents they need to win the case. This is the highest level of PA pharmacist practice: not just processing requests, but strategically managing the entire appeals process to ensure patient access to critical therapies.