Demystifying the FDA Meeting, September 10, 2013

For many years, patient representatives have been invited to participate in meetings around drug development as the patient voice is extremely important if reviews are to understand how patients weigh benefit and risk within the context of their disease. I was honored to present earlier this week at meetings for NINDS and the FDA. Below are some of the notes I took and some of the information I heard that I wanted to share with you all.


How does it work?  FDA issues guidance to explain reasoning for a particular approach.   Guidance is about the thoughts and process that leads FDA in a particular direction.  These decisions become FDA’s ‘case law’ and are made either in the context of established policy or establishing new policy.  Framework – laws and regulations establish ‘hard boundaries.’  When facts are clear, decisions are easier.  When facts are less clear, FDA weighs the big picture impact, effect on other decisions and makes a judgment, asking the question “how does this decision agree with established policies and legal interpretation?”

The role of patient input is: to establish what are acceptable tradeoffs between benefit and risk, to discuss what  the perceived benefits and liabilities of existing interventions are, to weigh the benefits/side effects of investigational therapies from the patient stand point, and how to weigh what is important for patients.

FDA has established two Centers of Excellence in Regulatory Science:  University of Maryland and Georgetown.  Another proposal is under view.

CDER also has a biomarker quantification program which provides a framework for scientific development and regulatory acceptance.  This is established to facilitate integration of qualified biomarkers in the regulatory review process.

Addressing challenges for rare disease – benefit/risk is a critical piece in drug development.   It is critical to utilize objective endpoints to minimize bias, which is complicated in rare disease because of clinical variability and small patient numbers. FDA requires rigorous natural history, which means there MUST be concordance across data sets.

Dr. Temple discussed FDASIA (referred to by the FDA as 1137)

Fast track – does not have to have clinical evidence.   This may be determined based on an animal study (replace enzyme) or in vitro evidence.   It must be a serious condition and the drug/biologic an advantage over current therapy.   These designations result in frequent and early meetings/advice, rolling review of NDA, and consideration for priority review. This designation can typically save 3-4 months, which is meaningful in the development process.

Breakthrough – in breakthrough designation, all advantages of fast track are present (basically builds on fast track). Requires early clinical evidence and preliminary clinical evidence that indicates the drug may demonstrate substantial improvement over available therapy.

What is markedly different is an organizational commitment – involvement of senior FDA staff is collaborative/cross-disciplinary effort (all hands on deck philosophy). People from the highest levels will be in the room and interested in decisions. 

Designation given before submission – Priority Review – this improvement could mean:

  • Greater effectiveness
  • Reduction of important adverse reaction
  • Better (documented) patient compliance
  • Safety and effectiveness in a new subpopulation

FDA does not want drugs that look good be held up by a chemistry problem. 


Accelerated approval (AA) – is unlike fast track or breakthrough.   AA is a different basis of approval.   Only for drugs with serious/life threatening diseases. Comes with a commitment that the drug be studied post approval.  AA is based on a surrogate – thought to predict clinical benefit.  

Subpart E – emphasizes efforts to implement the most effective study designs.  This is exactly why natural history is essential.


Nancy Roach discussed the Duke/FDA-clinical trials transformation initiative to improve the quality of clinical trials.  This initiative uses the frame of reference as ‘products’ and ‘customers’.  Product is the drug/biologic.  Customers include people connected with the clinical trial (sites/PI/Patients).  Learn more by clicking here

Lynn Yao discussed the development of agency-wide procedures and strategies to facilitate increased patient participation.  

  • Patient rep program in place
  • Increased patient recruitment in OHCA
  • Meets with centers, offices, and divisions
  • Webinar to patient reps
  • Implementation work group.


There are some challenges to include identifying patient representatives, timing of participation, conflict of interest, and most appropriate meeting to insure value and impact.

Finally, Andrea Tan discussed patient focused drug initiative which identified 20 specific diseases.  There were a number of factors that determined these conditions. FDA recognizes that there are many needs and is developing plans for expansion.

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