Regulatory Research

Accelerating Progress

We find it helpful to take a broad systems approach rather than get bogged down in specific components or symptoms.  To be sure, there are examples (symptoms) to be used to enlighten and elaborate on the need for systemic improvements, but the system that allows those symptoms to develop must be addressed.  The end customer must be recognized as the patient.  For Accelerated Progress, the patient fighting cancer or another life-threatening disease is the primary consideration within the context of the public health at large.  Once the customer of the system is identified, the customer’s need is assessed and enumerated.  The need is for more, better, and less expensive options to help extend life in a meaningful way.  As Dr. Scott Gottlieb, former Deputy Commissioner of the FDA and current member of the Scientific Advisory Board for Accelerate Progress correctly noted, “We think that you can have an approval process that is rigorous . . . even though you have parallel mechanisms to allow broader access to a drug that has shown activity and promise.”

Scientific rigor is important, but remember who it is we’re trying to help is critical.  Discounting the needs and rights of current cancer patients to an effective level of zero in favor of a strict calculation for potential of benefit or harm to future generations of patients is inappropriate and inhumane.  A recognition must be made regarding the risk to a patient from their disease and then that risk must be balanced against the likelihood of benefit (a net benefit from efficacy judged against risk of catastrophic side effects) from an experimental therapy in order to judge whether to grant some type of ealier approvals for theses therapies in such patient populations. 

Approaches to Accelerated Approval (and Accelerating Progress)

Drs. Thomas Roberts and Bruce Chabner published a piece in the New England Journal of Medicine in 2004 entitled “Beyond Fast Track for Drug Approvals” and much of what is laid out in that piece could still be put to good use.  We’ll argue for some changes in their approach to include pieces of other articles and research found elsewhere on our site and in our advisors’ works, but the core is a reasonable and thought-provoking theme.  The piece is available here.

A very good interview with Dr. Gottlieb is found here and although short, Dr. Gottlieb touches on many of the key systemic improvements that need to be made to provide faster development and patient access to safe and effective new therapies. 

Accelerate Progress will work to design more efficient and effective systems to deliver more meaningful progress to patients in a timely manner.  This means more safe and effective therapies in less time with less average cost.  Our Policy Advisory Board will spearhead this effort, leaning on the multiple lifetimes of experience from our Scientific Advisors dealing with the many systemic barriers to progress and working to overcome them as efficiently as possible.  Transparency in the workings of the system and those in it, which allows greater coordination and less redundancy, will be the rule, not the exception.