Letter to Representative DeLauro

Posted by: Christine McSherry on February 13, 2014

February 10, 2014

Congresswoman Rosa L. DeLauro

2413 Rayburn House Office Building

Washington DC  20515-0703


Re:   Compassionate Use – February 6th letter to Dr. Margaret Hamburg (FDA Commissioner)


Dear Congresswoman DeLauro:

As a healthcare professional with 33 years experience in drug development, I wish to clarify a critical point in your recent letter to the FDA Commissioner (Dr. Margaret Hamburg).

Compassionate Use (CU) is an ineffective pathway in providing access to promising new drugs for patients with rare and/or life threatening diseases that have no other existing treatment.  Insurance companies will not reimburse for CU drugs (even when FDA authorizes the CU) and pharmaceutical companies rarely supply product due to undermining of clinical trial enrollments and perceived negative impact on eventual FDA market licensing.

Congress and President Obama addressed this nicely in 2012 with passage of the Food and Drug

Administration Safety and Innovation Act (“FDASIA”), Pub. L. No. 112-144, 126 Stat. 993 (2012).   FDASIA defines a mechanism and pathway for Accelerated Approval (AA) of new drugs that demonstrate excellent safety and surrogate evidence for efficacy  (Section 506(c) of the FD&C Act, as amended by section 901 of FDASIA ).  With this prerequisite, the vital medicine is commercially available to dying patients and a confirmatory study proceeds to establish permanent approval.

One of the most prominent examples where AA is warranted is for a new drug (eteplersen) to treat  Duchenne muscular dystrophy (DMD).   This drug has demonstrated excellent safety (not a single serious adverse event to date) and efficacy on both its biochemical marker (dystrophin) and clinical endpoints (walking ability and lung function stabilization) in over 2 years (120 weeks) of administration to DMD boys.  Last Friday, 07 Feb 2014, a group of DMD advocates (Mothers of DMD patients and 3 world expert physicians in DMD research) testified to Congressional staff – perhaps your office attended – on the urgent need for Accelerated Approval of eteplersen.  See below copied testimony from this hearing.

Congresswoman DeLauro, the medical & patient community urgently needs your understanding and support to make FDASIA work better with respect to the AA pathway.   It is heartbreaking to see a child lose faith in God because nothing is being done (in WDC) to give him hope for getting a safe & apparently effective drug.  Who can explain to another Mother (Jenn McNary) that 1 of her 2 sons who got eteplersen –and and is now walking- but the other son must wait for FDA even as he withers away?

It would by my pleasure to discuss this further and provide additional documentation.





Robert J. Fuentes, MSc, PharmD.

719 Summer Walk Drive

Gaithersburg, MD 20878

Bookmark the permalink | Tagged

comments powered by Disqus