Canadian Dravet Syndrome Template Letter

Below is a letter on behalf of Canadian families who are seeking payment of Dravet syndrome therapies.  This letter was distributed to the National Health Plan on the patient’s behalf.

Michelle Welborn, PharmD

Founder

Intractable Childhood Epilepsy Alliance

PO Box 365

Lewisville, NC  27023

Date

Canadian xxxxxx

Xxxxxxxx

Xxxxxzxxz

zzzzzz

Dear Sir or Madam,

The purpose of this letter is to explain the nature of Dravet syndrome, a catastrophic and often fatal lifelong illness, and to encourage governmental plan coverage of the appropriate medications and therapies for children affected by Dravet Syndrome.  Doing so will improve the outcome and quality of life for these children, reduce the chances of mortality, and optimize healthcare spending by preventing the frequent emergency room, hospital, and ICU visits these children inevitably have without appropriate treatment.

Dravet syndrome is a rare and treatment resistant, refractory epilepsy syndrome presenting in the first year of life.  The incidence of this syndrome is 1:20,000-1:40,000.  About eighty percent of affected children have de-novo mutations of the SCN1A channels of the brain.  The severity is often not recognized at the time of diagnosis because the child appears to have normal or only slightly delayed development with no EEG abnormalities.  For the same reason,  appropriate diagnosis is often delayed, misdiagnosis occurs, and drugs that exacerbate seizures are administered.  Dravet syndrome over time will cause various degrees of developmental delay, mental retardation, ataxia, or loss of acquired skills. Frequent ambulance, emergency room, hospital and ICU visits are necessary to stop life-threatening episodes of status epilepticus that occur in some children several times per week.

Dravet syndrome often presents with a hemiclonic, prolonged, and fever related seizure. All seizure types present over time with status epilepticus necessitating hospitalizations  occurring frequently.  Polypharmacy is always necessary and few patients will obtain complete seizure control despite aggressive treatment.  The optimal treatment regimen is a combination of  stiripentol (Diacomit), valproic acid (Epival), and clobazam (Frisium).  Forty one children with SMEI were included in a randomized, placebo controlled, add-on trial. [i] Fifteen (71%) patients were responders on stiripentol (including nine (23%) free of clonic or tonic-clonic seizures), whereas there one patient on placebo had seizure reduction and none were seizure free..   Often patients with epilepsy of any origin are discouraged from addition of a third drug due to a very low (<10%) chance of any improvement in seizure control.  A 71% rate of reduction by 50% or more and a 23% rate of complete control of seizures is astonishing.  There are no comparable studies in the medical literature yielding these results in this seemingly hopeless and treatment resistant pediatric population with Dravet Syndrome.

In an abstract presented at the American Epilepsy Society in 2004[ii], a Canadian group reported a significant reduction in monthly healthcare cost in four children treated with the  combination of stiripentol, valproic acid, and clobazam.  Significant cost savings are still realized after adjusting  for the  current cost of stiripentol and inflated  medical costs from 2004 to present.  The price of improved seizure control, improved quality of life for the patient and the family, and ensuring optimal developmental potential for this child, and possible prevention of mortality is not measurable.

This combination is referred to by international experts as the “magic cocktail” and is first-line treatment in Europe, where each product is approved and sanctioned by the government, for patients with Dravet syndrome.   Stiripentol is currently available through Health Canada’s Special Access Programme from Biocodex, the French manufacturer.  Considering the prognosis of children affected by this rare disorder which includes progressive decline in mental function that has been directly correlated to number of prolonged seizures and a twenty percent mortality rate, availability of a drug combination that has shown astounding clinical efficacy in Dravet Syndrome, and the cost reduction the healthcare system will realize by offering the optimal treatment, I encourage the Canadian government to cover this medication for patients with Dravet syndrome under the publicly funded health plan.

Thank you for your consideration in optimizing the health and outcome of patients in Canada with Dravet Syndrome.  Please contact me with any questions you may have. The ICE Alliance website is www.ice-epilepsy.org for more information.  Enclosed are the cited references and the  Guide to the Treatment of Dravet Syndrome published by the ICE.

Sincerely,

Michelle Welborn, Pharm D

Intractable Childhood Epilepsy Alliance


[i] Lancet 2000; 356: 1638–42.

[ii] Epilepsia 2004: Vol 45, Supp 7:269.