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Senators Ask FDA about Expanded Access Program in Response to Patient Concerns

(WASHINGTON, D.C.) – U.S. Senators Lamar Alexander (R-TN), Tom Coburn, M.D. (R-OK), and Richard Burr (R-NC) sent a letter to FDA Commissioner Hamburg asking for comprehensive data on the risks and benefits associated with the Expanded Access, or “compassionate use”, program.  Expanded Access allows patients to access non-FDA approved therapies which are outside of clinical trials to treat serious or immediately life threatening conditions.

The full text of the letter is below:

June 16, 2014

Margaret A. Hamburg, MD


U.S. Food and Drug Administration

10903 New Hampshire Avenue

Silver Spring, MD 20993

Dear Commissioner Hamburg:

We are interested in understanding how the Expanded Access, or “compassionate use,” program at the U.S. Food and Drug Administration (FDA) is serving patients who seek access to new, unapproved therapies outside of clinical trials to treat serious or life threatening conditions.  The work of our nation’s biomedical research infrastructure and potential for medical innovation on behalf of patients is astounding.  Yet, each day patients nationwide are burdened by diseases and conditions that no FDA-approved products can cure or treat.  Those patients are understandably desperate for any treatment that might improve their ailments, quality of life, or survival.  For some, there is hope in drugs or devices that have not yet completed the FDA review and approval process, but their hopes are often frustrated since clinical trials alone often take years to complete under current design requirements. 

As we perform oversight of the compassionate use program, we respectfully ask you to provide the following information with respect to investigational new drugs (INDs), specifically:

  1. The number of requests for expanded access INDs and protocols that FDA has received each year since 2009, including whether such requests were for an individual patient IND, an individual patient protocol, an emergency IND, an emergency protocol, an intermediate-size IND, an intermediate-size protocol, a broader treatment IND, or a broader treatment protocol.   
  2. The number of approvals for expanded access INDs and protocols each year since 2009, by category of access as listed in question 1. 
  3. The average time in calendar days it took to approve or deny such requests each year since 2009, by category of access as listed in question 1.
  4. Please list the conditions for which FDA has approved expanded access INDs and protocols each year since 2009, by category of access as listed in question 1.  Please also list the conditions by which expanded access INDs and protocols were rejected, if applicable, each year since 2009, by category of access as listed in question 1.
  5. Does FDA collect data about the clinical trial phase of the investigational new drug at the time it is approved for expanded access?  If so, please provide the number of expanded access approvals by clinical trial stage since 2009.
  6. How does FDA use data on safety and outcomes from expanded access uses in regulatory decisions regarding the safety and efficacy of investigational new drugs, including when such drugs have been submitted for review and approval by the agency?
  7. In May 2013, FDA released draft guidance that said, “FDA is currently considering whether other options might better facilitate individual patient expanded access while providing appropriate ethical oversight.”  What options in addition to the full Institutional Review Board process has FDA considered to better facilitate expanded access for individual patients?  What is FDA’s timeline for further consideration and/or implementation of such options?
  8. How have you worked with patients and healthcare providers to help them understand the compassionate use options and application processes?  Do you plan to finalize the May 2013 draft guidance, and if so when?  What feedback have you received on the educational expanded access training webinar?
  9. How does FDA interact with drug manufacturers in considering and implementing expanded access INDs and protocols?  Please outline any applicable steps FDA could take to minimize the risks and burdens for drug manufacturers when they supply product as part of expanded access INDs and protocols.

We look forward to working with you on this issue to ensure the FDA’s compassionate use program is operating well for patients, providers, and the agency.  We respectfully ask that you would provide a written response within 30 days of the date of this letter.