FDA - Viewing Through a Different Lens

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Applied Clinical Trials

As I was trying out a series of corrective lenses at an optometrist last week to choose the one that finally provided me the clarity that I was looking for, it made me pose a profound question to myself about perception and reality.

As I was trying out a series of corrective lenses at an optometrist last week to choose the one that finally provided me the clarity that I was looking for, it made me pose a profound question to myself about perception and reality. This made me cherish the quote “Reality is merely an illusion, albeit a very persistent one” by Albert Einstein, that I think fits well in our current day to day lives, be it personal or professional.

Being a clinical trialist engaged in drug development in the pharmaceutical industry, my inherent wave of thoughts then proceeded to my profession and our industry as whole. That is when I realized, that as an industry, how we have over the years become religiously skeptic in our perception and views about the Food and Drug Administration (FDA). To my dismay, this has been the general discourse both in the media and the industry, although there have been one too many occasions especially in the last few years where FDA through its promotion of innovation has evolved to become one of the most revered premier scientific institutions that it was set to be in the 1800s.  

Perhaps it is time for us to take a moment to view the accomplishments of the FDA with a different set of lenses and also celebrate our colleagues at the FDA who have contributed to its success. Under the stewardship of Dr. Margaret Hamburg, the agency appears to be flourishing scientifically mainly because some of her path-breaking, innovative initiatives and therefore, I believe it is time to recognize and give credit when and where it is due and applaud from the sidelines.

Strained System:

With the drug discovery and development timelines strained more than ever before, a call to action was placed by a panel at a Conference on Clinical Cancer Research in 2011, towards devising innovative pathways for expedited approval of drugs.¹

Courtesy of the American Association of Cancer Research 2011 Cancer Progress Report

Break Through Designation:

Subsequently, FDA in order to expedite the drug approval process, collaborating with several external stake holders came up with a novel pathway called the “Breakthrough Therapy Designation” (BTD).  This is specifically tailored towards Sponsors seeking approval for a drug that treats a serious or life-threatening condition where the preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. Note, this in contrast to a fast track program for a drug that treats a serious or life-threatening condition, wherein nonclinical or clinical data demonstrate the potential to address unmet medical need.2 With collective support from the industry, researchers, and academia, Dr. Hamburg successfully led the collaborative effort on the Hill which eventually saw this novel ideal and her efforts behind the scenes, bear fruit.

In the spring of 2012, the bipartisan Advancing Breakthrough Therapies for Patients Act was introduced by Senators Michael Bennet (D-CO), Orrin Hatch (R-UT) and Richard Burr (R-NC) and Representatives Diana DeGette (D-CO) and Brian Bilbray (R-CA). President Obama signed this into a law as part of the FDASIA on July 9, 2012.  This was no mean feat to achieve, as Dr. Hamburg using her good offices and working with our elected representatives from both sides of the aisle was able to successfully have this bill enacted which led to Senators Tom Harkin (D-IA) and Michael Enzi (R-WY) releasing the following statement: “While people like to talk about polarization and gridlock in Washington, this bill is a victory for both bipartisanship and for the millions of American who rely on medicines and medical devices.”


Current Status:

The number of drugs to date that has been granted BTD status is a clear indicator that this novel program has indeed met its objectives since it successful launch.  Of course, the entire industry along with Main Street and Wall Street are closely watching this unfold, as we are yet to reap the economic benefits. Nevertheless, this phenomena has spurred innovation all around the globe including at the Department of Health, UK which is now implementing a similar program that is designed to offer patients access to novel medicines.4 With the initial response from all quarters very positive, my hope and optimism is that this novel program continues to expand, so millions of patients around the world receive access to medicines that otherwise would be unavailable simply because of lack of spirited innovation.

This was made possible by the sheer determination, devotion and the visionary leadership of Dr. Hamburg and her colleagues at the FDA and so for that, I salute them.




4. https://www.gov.uk/government/news/cutting-edge-drugs-to-be-fast-tracked-to-patients