The Jett Foundation Fighting Duchenne Muscular Dystrophy

Template Medicaid Letter of Appeal and Request for Fair Review for Non-Ambulatory Patients

March 17, 2017

INSURANCE PROVIDER NAME

Subscriber Assistance Program

Address

Agency Contact’s Email

 

Re: Request for expedited outside independent hearing

Name of Patient SS#

Name of Insurance Provider

ID #

Dear AHCA – SAP: Name of Agency Contact:

After consulting with my physician, (Name of Physician), I am formally submitting a request for an expedited independent hearing of (Insert Insurance Provider) denial of the treatment plan with EXONDYS 51 for my son, (Name of Patient), who has Duchenne muscular dystrophy (DMD). I respectfully request, beg you actually, to assign this to a physician who has expertise in treatment of patients with neuromuscular diseases, specifically Duchenne muscular dystrophy.

The (insert initial denial) denial by (Insert Insurance Provider) reads, “The drug your doctor asked for EXONDYS 51 SOL 100/2 ML (Infuse 1500 Mg IV Over 1 Hr Every Week) is denied.  The health plan’s criterion was not met because (insert reasons why the drug was denied.) Your doctor has been given this information.”

Please refer to the attached memorandum from Janet Woodcock, MD the Director of the FDA’s Center for Drug Evaluation and Research (CDER). *(please see attachment A) This memorandum explains CDER’s final decision to approve Sarepta’s New Drug Application (NDA 206488) for EXONDYS 51 (eteplirsen) for marketing in the United States.  Dr. Woodcock writes, “I find that the data contained in NDA 206488 meet the standard for accelerated approval under 21 CFR 314.510 based on the surrogate endpoint of increased dystrophin protein production, a surrogate endpoint that I conclude is reasonable likely to predict clinical benefit.”

The FDA’s Accelerated Approval Program *(please see attachment B) was instituted in 1992 to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.  A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit (for example, dystrophin in DMD). The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. Drug companies are still required to conduct “post marketing” studies to confirm the anticipated clinical benefit.  If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug.”                  *(Please link at the end of this document “Accelerated Approval Program” and “FDA’s Drug Review Process).

The FDA defines a serious condition as, “…. a disease or condition associated with morbidity that has substantial impact on day-to-day functioning…. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.”

When you look at the natural history of the disease (please see attachment  C)Dr. McDonald’s publication Natural History of DMD/BMD, showing the known clinical course of Duchenne muscular dystrophy.  Patient’s disease clearly meets the criteria as a “serious condition” as defined by the FDA.  He is maintained on glucocorticoid steroids to slow disease progression and on ACE inhibitors to protect cardiac function; he is no longer ambulatory; he has limited movement of his arms; stability of the trunk is decreased and he occasionally has to use a “cough assist” machine.  This is needed because of progressing weakness in the diaphragm caused by lack of dystrophin.

EXONDYS 51 (eteplirsen) Injection, 50 mg per mL, was approved by the FDA for US marketing and is labeled for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. (please see attachment D) Name of Patient has a deletion of exons 48 through 50 in the gene making his disease amenable to exon 51 skipping therapy.  Without treatment with EXONDYS 51, the ONLY available treatment, not only will Patient succumb to this disease, the quality of his remaining life will be adversely affected.

The original request for coverage was submitted by Physician on (insert date of original prior authorization form).  The denial letter dated (insert date of initial denial letter) stated the reason for denial was that treatment was

“Not medically necessary… This medication is considered investigational. The FDA has not been given final approval for this medication”.

We appealed this decision in writing on (insert initial appeal letter date) and received their notice to uphold their decision to deny coverage on (insert secondary denial date).  Physician then had a peer to peer discussion with (insert name of physician who participated in peer to peer review) for (Insert Insurance Provider), on (insert date of peer to peer review).  We received notice on (insert date of third denial) that the end result of that discussion is that he, too, upholds the decision to deny coverage of Exondys51. (All documents included in Attachment E)

I believe NO medical director with Duchenne experience would ever classify Exondys 51 as “not medically necessary.”

I have also attached a copy of a letter from the Office of Drug Evaluation that is signed by 36 (thirty-six) Scientists and Physicians in support of approval of Exondys 51 treatment for Duchenne muscular dystrophy. (please see Attachment F)

In (insert month and year of diagnosis), my son, Patient was diagnosed with Duchenne muscular dystrophy.  Duchenne muscular dystrophy is caused by mutations in the dystrophin gene. (please see Attachment G)  This gene is an x-linked genetic disorder characterized by the progressive loss of skeletal muscle and degeneration leading to premature death.  Individuals with DMD lose the ability to walk and over time experience serious respiratory, orthopedic, and cardiac complications due to the lack of dystrophin in muscles. (Please see the attached webinar by Craig McDonald, MD)

EXONDYS 51 has been granted accelerated approved by the FDA based on an increase of dystrophin that was shown to be statistically significant in clinical studies. The determination by FDA is that this increase in dystrophin is reasonably likely to predict clinical benefit in patients.

Since the diagnosis, the only medication primarily used by patients like my son has been corticosteroids which do not treat the underlying cause of the disease, a lack of dystrophin.

I am greatly encouraged that my doctor believes my child is a good candidate for EXONDYS 51. This is the first FDA approved treatment for the disease and is intended to allow for production of an internally truncated but functional dystrophin protein.

My son is currently non-ambulatory, having lost the ability to walk independently at age (insert age of lost of ambulation.)  Studies in ambulatory patients with Duchenne have demonstrated that treatment with EXONDYS 51 (also known as eteplirsen) yielded stabilization of measures of pulmonary function including percentage maximum inspiratory pressure (% MIP) and percentage maximum expiratory pressure (% MEP). In addition, treatment with EXONDYS 51 yielded a slowing of the disease progression as related to pulmonary decline. Trial participants experienced a mean 2.5% reduction in percent forced vital capacity (%FVC) per year, as compared to the natural history rate of decline of 5% per year. Significant levels of FVC impairment are associated with an increased risk of respiratory infections, complications, and mortality in patients with Duchenne.1

The importance of maintaining effective cough and reducing the risk of airway infections was methodologically established through a quantitative patient preference study conducted by Parent Project Muscular Dystrophy (PPMD)4. Participants rated maintaining effective cough strength and reduced lung infections as important treatment priorities for Duchenne.

Physician has intimate knowledge of Patient’s medical history, diagnosis and the rationale used in determining that he should have access to EXONDYS 51. Delay in treatment means the loss of critical function and a delay of the ability to produce dystrophin for my child’s muscles, and therefore valuable life skills and independence. In Duchenne, every day represents the loss of precious muscle.

Please contact Physician or me if you need more information about the efficacy, safety and effectiveness of EXONDYS 51.  For your information, I have attached peer review studies, and briefing documents submitted to FDA.

I look forward to hearing from you. I feel confident that you will approve treatment of my son with Exondys 51 so he can enjoy a greater quality of life.  My contact information is listed below.

Sincerely,

 

(Caregiver)

(patient)

Address:

cell:

fax:

 

cc: Physician, email

Pediatric Neurologist, phone number, city and state

RN, email

 

Enclosures:

1 Eteplirsen Pulmonary Function Data 

2 AHCA Request for Review Application (insert patient’s request for review)

3 AHCA Appointment of Representation (insert patient’s chosen representative(s))

Parent Project Muscular Dystrophy’s Patient Preference Study on Pulmonary Outcomes

 

Attachments:

A: Center Director Decisional Memo (Janet Woodcock)

B: Accelerated Approval

C: Natural History of DMD/BMD

D: FDA Open Label

E: Prior Authorization  pg 1-3 (insert patient’s prior authorization form)

  • Initial Denial letter  (insert patients initial denial letter)
  • Appeal letter from Physician  (insert physician’s appeal letter)
  • Denial letter pg 21-22 (insert additional denial letters)
  • Notation of peer to peer call (insert notification of peer to peer review)
  • Upholding denial from peer to peer call (insert post peer to peer denial letter)
  • Patient’s genetic testing diagnosis

Additional Resources to Include: 

  1. Letter signed by 36 Duchenne Scientists and Physicians in support of approval of Exondys 51
  2. Jett Foundation’s Patient Reported Outcome Report

 

 

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