Letter of Medical Necessity and Intent to Treat Template

Dear Healthcare Provider,

The purpose of this template letter is to assist your office in developing a customized Letter of Medical Necessity which outlines the medical justification for MIPLYFFA® (arimoclomol) capsules, for oral use. Often, by submitting a Letter of Medical Necessity tailored around the history and current treatment needs of your patient, insurance plans may better understand the reasoning for MIPLYFFA.

Please note–this letter template should only be used as a guide. Each patient will have their own unique and specific reasons for needing MIPLYFFA. In addition, each insurance plan may have its own rules and guidelines for approving medications.

This sample letter and related information are provided for informational purposes only. It is the responsibility of the HCP and/or their office staff, as appropriate, to determine the correct diagnosis, treatment protocol, and content of all such letters and related forms for each individual patient. Zevra Therapeutics does not guarantee coverage or reimbursement for the product. There is no requirement that any patient or healthcare provider use any Zevra product in exchange for this information, and this template is not meant to substitute for a prescriber’s independent medical decision-making.

For full Prescribing Information and Instructions for Use, please go to miplyffa.com.

Letter of Medical Necessity and Intent to Treat

[Date]

[Insurance Contact Name]

[Insurance Contact Title]

[Name of Health Insurance Company]

[Insurance Address]

Patient Name: [Patient Name]

Policy Number: [Policy Number]

Group Number: [Group Number]

Diagnosis: [ICD Code and Description]

Subject: Intent to Treat with MIPLYFFA™ (arimoclomol) capsules, for oral use

To Whom It May Concern,

I am writing on behalf of my patient, [Patient Name], to document medical necessity for treatment with MIPLYFFA® (arimoclomol) capsules for oral use, to obtain coverage and subsequent payment for the drug. MIPLYFFA is the first approved drug for the treatment of Niemann-Pick disease and is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.1

NPC is a progressive disease that affects the brain and visceral organs, including the liver and spleen. It is a serious neurodegenerative and life-limiting disease often leading to early fatality, so treatment is imperative.2,3

I am a [specialty – eg, geneticist] who has been treating lysosomal storage disorders (LSDs) for [number] year[s] and am confident in the medical necessity of MIPLYFFA for this patient.

The patient will be treated with MIPLYFFA for NPC.

I have [enclosed/attached] the full Prescribing Information, which supports the use of MIPLYFFA in combination with miglustat for this patient.

In the event of a denial of coverage, all levels of appeal will be pursued, up to and including peer-to-peer review with like-specialty and third-party review, if applicable.

Summary of Patient’s Medical History and Diagnosis:

[Patient Name] has been in my care since [Date]. [He/She] is a[n] [age]-year-old [male/female] suffering from NPC.

Prior to [his/her] diagnosis [he/she] presented with [NPC clinical features: Neurological, Psychological, and/or Psychiatric Manifestations and Systemic Signs and Symptoms].

After thorough clinical evaluation and a positive [Genetic Test] on [Date] [he/she] was diagnosed with [Diagnosis and Subtype]. Since diagnosis, the treatment plan to stop the progression of the disease has included [Medications, PT, OT, Other Treatment Modalities].

Based on my clinical judgement and [his/her] [Current Medical State/Further Medical Decline] it is my clinical opinion that the [he/she] will experience [Poor Prognosis] without treatment with MIPLYFFA.

Based on the above facts, I am confident that you will agree that [specific dose based on patient weight] of MIPLYFFA is medically necessary and the appropriate therapeutic choice for [Patient Name].

To assist with your review attached you will find the following:

Medical records to include:

[Office chart notes]

[Laboratory test results]

[Genetic testing results]

Publicly available medical literature/publications supporting the use of MIPLYFFA for NPC

[Supporting literature/publications available at https://zevra.com/medical-literature]

Additional information on MIPLYFFA

https://zevra.com/documents/MIPLYFFA-Prescribing-Information.pdf

I would appreciate your evaluation of this request and ask that you approve MIPLYFFA. If you have any questions or wish to conduct a Peer-to-Peer discussion, feel free to contact me at [phone number].

Sincerely,

[HCP Name, credentials, and/or participating provider number]

[Hospital, Office, or Clinic Name]

[Office phone number]

[Office fax number]

[Office email address]

[Office Staff/Contact Name]

Enclosures: [List of documentation described in above letter]

References:

MIPLYFFA Full Prescribing Information. Celebration FL, US. Zevra Therapeutics Inc.; 09/2024

Vanier MT. Niemann-Pick disease type C. Oprhanet J Rare Dis. 2010, 5(16):1-18. doi:10.1186/1750-1172-5-16

Mengel, E., Patterson, M.C., Chladek, M. et al. Impacts and Burden of Niemann pick Type-C: a patient and caregiver perspective. Orphanet J Rare Dis.2021;16(1).493. doi:10.1186/s13023-021-02105-8