Why would an agency charged with promoting the public health need to be told to consider patients’ perspectives? Further, why would the Food and Drug Administration need be given two years to come up with a “structured risk benefit framework” for the meaningful incorporation of patient experience data into the regulatory decision-making process when the agency has been saying since February 2013 that it is doing just this?
That’s the mandate of the 21st Century Cures bill passed last week by the House Energy & Commerce Committee by a vote of 51 to zero. Included in the bill is $550 million in additional funding to the FDA.
To me, the 21st Century Cures bill begs a critical question — why? Why is this bill needed? The FDA has been given an incredibly daunting responsibility, and equally broad authority, to fulfill its duty “to promote health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of regulated products in a timely fashion.” This includes “ensuring that ... human and veterinary drugs are safe and effective” and “there is reasonable assurance of the safety and effectiveness of devices intended for human use.” Notice the words promptly, efficiently and timely in the statute.
Why does Congress need to tell the FDA to do it, again, after instructing them to do so as part of the 2012 Food and Drug Administration Safety and Innovation Act Prescription Drug User Fee Act (PDUFA) re-authorization? Clearly, the FDA does not want to obey.
Why would the FDA need to be told to issue new guidance documents for the use of development tools like biomarkers and surrogate endpoints when the FDA has issued three guidance documents over the past two years on the use of biomarkers?
Why would the agency need to be told to do better with respect to its compassionate use program, which allows for experimental drugs to be made available to terminal stage patients when Right to Try Laws have now been passed in 20 states and there has been intense discussion of the issue with FDA in the media for the last two years?
Why did the FDA do nothing substantive in this regard? When a patient is dying, the benefit/risk ratio moves to infinite because there is no risk, only potential gain. Still the FDA resists this grassroots, patient-initiated movement.
But why? Why does the FDA not want to do these very rational things, which it has the full authority to do and for which it has spent precious time and resources preparing policies and guidance documents that do not satisfy Americans?
The answer is philosophy. The philosophy at the FDA is off; instead of embracing its congressional directive to promote health, it states its mandate as “protecting” health. Instead of embracing technology, the knowledge-based economy, and the empowered patient, the FDA assumes that patients and doctors are stupid and industry has no incentive to market safe and effective products apart from FDA hurdles and sanctions. Instead of looking for reasons to approve (and label) drugs, biologics and devices for uses that will maximize benefits and minimize risks, the FDA seeks the utopian ideal of magic bullets with no risk, thereby making approval standards untenably high, except for niche products for small populations. Instead of boldly advancing health, the FDA is driven by fear. And this philosophy has led to the creation of massive structure that impedes medical innovation and further entrenches the medical status quo.
The good news is that 21st Century Cures boldly exclaims there is a problem. The bad news is that Congress keeps throwing money at the problem rather than doing the hard work of fixing the ideology, the leadership and the structure of the FDA for the 21st Century. What is particularly bothersome to me is that even more money is on the way in 2017 with PDUFA re-authorization. The FDA continually cites underfunding as the reason approval times are in violation of the law. And Congress keeps falling for it, repeatedly giving the agency more money even when specific directives are wantonly flouted, for example, the Sunscreen Innovation Act.
I am beginning to think that the Steve Miller Band works at the FDA: “With nothin’ better to do ... he makes his livin’ off of the people’s taxes ... singin’, go on, take the money and run.”
Seriously, there is a recurring theme characterizing the interactions between Congress, the FDA and the industry: More money is given to an agency that continues to demonstrate a lack of ability to adapt and make good on its daunting responsibility. And patients continue to suffer.
Gulfo is the executive director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University.