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April 10, 2020

Stephen Hahn, M.D.
Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD  20993

Dear Commissioner Hahn:

Thank you for your dedicated service to our country as we all work together to reduce and manage the impact of the COVID-19 crisis.  We write you as the Chair and the President of the Cure JM Foundation representing families across the country and the world with children who live with a debilitating rare autoimmune disease—juvenile myositis.

Many of these children depend on access to drugs that are being tested or stockpiled for COVID-19.  While we applaud the decision to test a wide array of approved drugs to combat COVID-19, we are deeply concerned that juvenile myositis children, as well as millions of other patients with autoimmune diseases, will be denied access to the drug therapies many of them need to survive.  Specifically, we must assure that our JM children have access to hydroxychloroquine (brand name: Plaquenil) through their local pharmacies.

Juvenile Myositis (JM) is a debilitating autoimmune disease resulting in severe muscle weakness and frequent immobility.  In JM, a child’s immune system is uncontrolled, attacking healthy tissue and often internal organs such as the heart and lungs.  Without question, JM is an underlying condition, and children or adults with JM would be at higher risk should they actually contract COVID-19.

JM is treated by suppressing an overactive immune system, and first-line treatments often include a mix of drugs such as prednisone, methotrexate, and hydroxychloroquine.  As JM is an idiopathic and heterogeneous disease that can present differently across each child, there is no single course of therapy.  Cure JM’s Medical Advisory Board (comprised of the leading JM medical experts in the world) estimates that 70% of JM patents will use hydroxychloroquine in the course of their disease treatment.

A survey taken this week of JM parents presents important information you may find helpful as you provide guidance to manufacturers and distributors of hydroxychloroquine.

  • 37% reported difficulty in having hydroxychloroquine prescriptions filled for their children.
  • 28% reported that their pharmacy was out of hydroxychloroquine supply.
  • 49% reported that their pharmacist cautioned that their local supply could run out in the immediate future.

Results were reported from 35 states, Puerto Rico, and the District of Columbia.

 

 

We are hopeful that hydroxychloroquine could prove to be helpful in the treatment of COVID-19, and support the clinical trials which will provide the needed scientific proof of its efficacy in such treatment.  We are also thankful to the pharmaceutical manufacturers who have committed to increasing production and donating millions of doses to hospitals and to the national stockpile.

However, at the same time we request the FDA makes every effort to require manufacturers and distributors of hydroxychloroquine to reserve adequate supply in pharmacies for JM children - and other autoimmune patients - who rely on this drug for maintenance and survival.  To fail these patients, where hydroxychloroquine is known to be efficacious and is often a first-line approved immunosuppressive treatment, would only add to the tragic outcomes of the COVID-19 pandemic.

Thank you for allowing us to share our concerns.  We are deeply appreciative of your and the FDA’s leadership to assure access to hydroxychloroquine for patients where it is a prescribed and proven therapy. 
We look forward to working with you and members of your staff on this critical access issue.

Sincerely,

Mitali

Mitali Dave Chairperson                                                                                                       Cure JM Foundation
(512) 638-1020  

Nikki

Nikki Hahn President                                                                                                      Cure JM Foundation
(914) 860-4671