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 OLPRUVA® (sodium phenylbutyrate) for oral suspension. 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 OLPRUVA.

Please note–this letter template should only be used as a guide. Each patient will have their own unique and specific reasons for needing OLPRUVA. 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 OLPRUVA.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 OLPRUVA® (sodium phenylbutyrate) capsules, for oral suspension

To Whom It May Concern,

I am writing on behalf of my patient, [Patient Name], to document medical necessity for treatment with OLPRUVA® (sodium phenylbutyrate) for oral suspension to obtain coverage and subsequent payment for the drug. OLPRUVA is a nitrogen-binding agent indicated as adjunctive therapy to the standard of care, which includes dietary management, in the chronic management of adult and pediatric patients, weighing 20 kg or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). OLPRUVA is not indicated for the treatment of acute hyperammonemia.1

I am a [specialty – eg, geneticist] who has been treating urea cycle disorders (UCDs) for [number] year[s] and am confident in the medical necessity of OLPRUVA for this patient for urea cycle disorder [sub type].

I have [enclosed/attached] the full Prescribing Information, which supports the use of OLPRUVA for this patient. This letter serves to document that [Patient Name] needs OLPRUVA and that OLPRUVA is medically necessary for [him/her].

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 [Diagnosis and Subtype].

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

After thorough clinical evaluation and a positive [Blood, Urine, Liver Biopsy and/or Genetic Test] on [Date] [he/she] was diagnosed with [Diagnosis and Subtype]. Since diagnosis the treatment plan has included [Medications, PT, OT, Other Treatment Modalities].

Since instituting this treatment plan, I have observed the following [Symptom exacerbation and/or decrease in quality of life].

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 OLPRUVA.

Based on the above facts, I am confident that you will agree that [specific dose based on patient BSA] of OLPRUVA 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://olpruva.com/wp-content/uploads/OLPRUVA-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:

OLPRUVA® (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.