I was contacted by the Senate Committee on Homeland Security and Governmental Affairs following the release of my paper, “The Role of the FDA for the 21st Century” to testify at the Feb. 25 hearing entitled “Connecting Patients to New and Potential Life Saving Treatments.”
Thank you for inviting me to participate in this hearing. As Senator [Ron] Johnson [R-Wis.] and my fellow witnesses eloquently explained, Right to Try is an especially important concern for patients and their families, particularly those for whom no approved options exist and are terminally ill.
The Right to Try debate also vividly illustrates several foundational principles contained in my recent research about the proper role of the FDA [Food and Drug Administration] in the 21st-century medical ecosystem, which apply to patients at all stages of their disease process, not just the terminally ill with no other good choices.
I have six brief points to make:
1. First, the FDA has substituted its statutory mission to promote health with a new, misperceived duty to protect health, virtually at any cost. When terminal patients have no other options, what is FDA protecting when it prohibits access to an experimental treatment? The potential harms of FDA’s approach to protecting health is not limited to terminal patients — it has serious implications for all who suffer disease and it is having a chilling effect on innovation.
2. Second, benefit-risk is a private health decision, requiring consideration of the individual’s needs. FDA’s reliance on the benefit-risk of the “average patient” is not an acceptable substitute. Right to Try patients seeking experimental therapies have a right to make that decision; and patients and their doctors know far more about their specific circumstances than the FDA ever could. Of course, the same is true for ALL patients with all stages of disease.
3. Third, the FDA’s restated mission to protect rather than promote health has necessarily made it judge new drugs and devices not on SAFETY and EFFECTIVENESS, as specified in the law, but rather on clinical utility, benefit-risk and long-term outcomes, including survival. This truly stifles medical innovation by necessitating larger and larger and longer and longer trials that are extremely expensive. The net result is that many compounds that could possibly help patients aren’t even developed, and many that are obviously safe and effective and could be helpful to patients actually fail in these kinds of trials because the studies are improperly structured — it is simply impossible to control for all of the variables that modulate long-term outcomes.
4. Fourth, increasingly, the drugs that are developed, even by large companies, are geared toward narrow, niche and orphan populations, where benefit-risk is a low bar because there are no other products available. In 2014, 40 percent of all approved new drugs were orphans; in 2015, 48 percent were orphan drugs.
5. Fifth, the very fact that we are here today to discuss this magnificent movement is testimony to the sophistication of today’s medical ecosystem and marketplace. That which we may have needed the FDA to do just a few years ago is unnecessary today. With the Internet and rapid communications among patients and among physicians, knowledge is shared at a lightning pace. Patients have easy access to timely, high-quality information that allows them to make excellent treatment decisions with their doctors. Today’s reality could not have been anticipated 20-plus years ago when the FDA began to move away from safety and effectiveness as the rightful bases of approval.
My final point is that medical innovation is fragile because most of it occurs in small, start-up companies. I have been responsible for the development and approval of products for prostate cancer, bladder cancer and melanoma. As such, I have worked extensively with all three FDA centers — drugs, biologics and devices — in the development of novel products. The two things that start-up companies need most are investment and regulatory certainty. Two companies that I ran no longer exist because the FDA changed the criteria for approval after we performed large studies that achieved all of the endpoints FDA had originally required. In both circumstances, the FDA told us that safety and effectiveness were not enough; rather, clinical utility and long-term outcomes were required. This made investment disappear and rendered it impossible for us to launch the products and bring them to doctors and patients effectively after ultimately obtaining approval, with no new data in either case.
In summary, the need for the Right to Try movement is emblematic of FDA’s “protect health at all costs” ideology. This has made the FDA assume a role that it was never intended to have — being arbiters of benefit-risk, clinical utility and long-term outcomes. And, it has brought the FDA into private health decision-making.
We need the FDA to return to its public health mission of promoting health by making approval decisions on the basis of safety and effectiveness.
At the conclusion of the hearing, Sen. Thomas Carper (D-Del.), the ranking member, asked all witnesses to propose solutions that he and his colleagues could pursue to address the problem. I offered a three-part plan, which was well-received. First, it is imperative that all of us show the FDA love so that it does not fear public reprisals when approved products do not work out as well as all of us hope; unfortunate things do happen, occasionally, and the FDA cannot be publicly humiliated when this occurs. Second, Congress needs to encourage the FDA to get back to its lawful mission to promote health, with the understanding that Americans do not expect the FDA to guarantee that all new products will be helpful to all patients all the time. Finally, Congress needs to encourage the FDA to use safety and effectiveness as the bases of approval. I alluded to my written testimony in which I outlined four categories of evidence that could be used to demonstrate effectiveness (biomarkers, clinical signs and symptoms, disease modification and long-term outcomes including survival).
I hope that I am invited back to discuss these solutions in greater depth and that we are successful in our goals.
Gulfo is the executive director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University and author of “Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances” (Post Hill Press). He has more than 25 years of experience in the biopharmaceutical and medical-device industries and is the former CEO of Mela Sciences. Follow him on Twitter @josephgulfo.
This article originally appeared on www.thehill.com on March 4, 2016.