Where do things stand with the FDA in terms of key issues such as compassionate use, fast track, and such? Are you communicating with the FDA? If so, how’s that going? Are they receptive?
Ted: I have communicated with the FDA on numerous occasions. I had the chance to “testify” to them in 2013 where approximately 50 people from the ALS community had the opportunity to lay out their concerns to the FDA. It ranged from patients and caregivers to Neurologists and researchers and even one Bio-company CEO. I also went to training for what they term their Patient representative program. If the appropriate trial comes to review I will have the opportunity to provide the patient’s perspective on the clinical trial.
As far as their various programs I think they sound good, however from a patient perspective I do not see them as overly impactful. I want to be clear I do not want to place the entire onus on the FDA, they need partners from drug and bio industries, research institutions, etc. to participate in these programs. That being said I have heard over and over from patients and some Doctors that the application process for these programs can be cumbersome and time consuming. A read one estimate that said that the paperwork for a Doctor and patient can take up to 100 hours to complete. For someone that has a terminal illness and most likely a brief lifespan remaining this in my opinion is unacceptable.
As far as are they receptive? Well they say many of the right things and I have even heard from Doctors in the trial I am in at Emory University that after the ALS testimony have been trying to be more cooperative and have slightly reduced wait times for approvals. This is great however what I really want to see is changes in the process. Dr. Margret Hamburg, FDA commissioner, herself admitted to Congress that the FDA is utilizing 20th century regulatory processes for 21st century medicine. I think this is especially true with stem cells. I think it is great that they recognized they have “a problem” I would like to understand what actions they are taking to correct that and get their regulations caught up to the speed of medicine and science.