Exondys 51 Letter of Medical Necessity Template
December 16, 2016
Name (if known)
RE: name of patient, patient’s insurer id number
Dear Sir or Madam:
I am writing to support an appeal of your denial of Exondys 51, an antisense oligonucleotide indicated for the treatment of patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, for my patient Mr./Ms. _________. I have reviewed your letter to my patient and continue to recommend Exondys 51 as the treatment of choice in this case based on my (XX years) of experience treating patients with Duchenne muscular dystrophy (DMD.)
Mr./Ms. _______ has been under my care for his/her DMD since (date). DMD is the most common form of congenital muscular dystrophy and the most severe form of dystrophinopathy. This condition is caused by variants in the dystrophin-encoding gene. Approximately 65% of the variants in patients with Duchenne are deletions of one or more exons in this gene that often disrupt the reading frame and disable the dystrophin protein from being produced. The absence of dystrophin in muscle cells leads to a progressive muscle weakness and atrophying that rapidly worsens with age and will cause a loss of ambulation, a decrease of arm and trunk strength, the inability to independently preform activities of daily living, and eventually, the complete absence of dystrophin distinctive to a diagnosis of Duchenne will lead to DMD-associated dilated cardiomyopathy and death. 
Exondys 51 is the preferred therapy for Mr./Ms._______ due to the amenability of his/her mutation (a) deletion (s) of (XX-XX) on the dystrophin encoding-gene to the skipping of exon 51. Exondys 51 received FDA approval for marketing on September 19th 2016 for the treatment of patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on a statistically significant increase in dystrophin as a surrogate endpoint in skeletal muscle observed in pre and post treatment muscle biopsies obtained from patients treated with Exondys 51 as part of a clinical trial. Though Exondys 51 was granted accelerated approval, marketing approval based off of a change in a surrogate endpoint or biomarker observed sooner than mortality or irreversible morbidity and is reasonably likely to predict a clinical benefit, in this case dystrophin data, the totality of Exondys 51’s clinical data shows a clear trend towards efficacy and a departure from what I know to be the natural history of DMD as a physician with (XX years) experience treating DMD. As with all drugs granted accelerated approval by the FDA, further confirmatory trials to confirm the clinical efficacy of Exondys 51 are required and are currently in progress. I fully expect and look forward to reviewing those results.
Mr./Ms._________ needs weekly infusion of Exondys 51 to treat his DMD. Without weekly infusions of Exondys 51 and the dystrophin the treatment has proven to produce based off of clinical trial data, I know that his/her muscles will continue to weaken and atrophy, resulting in morbidity and eventual mortality. As I stated before, DMD is a progressive disease whose effects on muscle loss are irreversible. DMD has already robbed Mr./Ms._________ of his/her ability to preform activities of daily living, for example, (insert lost activities of daily living, for example: walk, climb stairs, get off the floor, get into the car, feed him/herself, brush his/her teeth, open bags of food or bottles of water, maintain independence at school, write, turn him/herself in bed, dress, bathe, etc.) Therefore, it is my expert opinion that treatment of Exondys 51 must begin as soon as possible in order to maintain or slow the loss of Mr./Ms._________ ability to preform additional and important activities of daily living, for example, to (insert activities of daily living you hope to maintain or slow the loss of, for example: walk, climb stairs, get off the floor, get into the car, feed him/herself, brush his/her teeth, open bags of food or bottles of water, maintain independence at school, write, turn him/herself in bed, dress, bathe, etc.)
As a physician who specializes in treating neuromuscular disorders and who has an abundance of experience treating patients affected by DMD, I will continue to use my medical judgment in prescribing Exondys 51, and I strongly encourage payers to provide coverage to all of my amenable patients as soon as possible.
I may be reached at the number below should you require additional information on this patient. Otherwise, I look forward to your response directly to me as soon as possible.
Physician’s name and credentials
CC: (patient’s name)
1 Hoffman EP, Fischbeck KH, Brown RH, Johnson M, Medori R, Loike JD, Harris JB, Waterston R, Brooke M, Specht L. Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne’s or Becker’s muscular dystrophy. N. Engl. J. Med. 1988 May; 318(21):1363–1368. PubMed ID: 3285207.
2 Food and Drug Administration. FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. 19 Sept. 2016. < http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm > 30 Nov. 2016.
 Exondys 51, [package insert]. Sarepta Therapeutics, Inc., Cambridge, MA; September 2016
 Eteplirsen [briefing package]. Sarepta Therapeutics, Inc., Cambridge, MA; April 2016
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