Science and Progress at the FDA

Realizing the Return on Investment from Biomedical Research
by Cecilia Arradaza, Communications Director, FasterCures


Speakers at a congressional briefing on March 24 said that in order to realize the full return on investment from biomedical research, the U.S. Food and Drug Administration (FDA) needs to be in a position to take the baton of innovation from the research community and turn it into medical products that are safe, effective, and accessible for patients who need it most. The briefing, Science and Progress at the FDA, was convened by Friends of Cancer Research and FasterCures/The Center for Accelerating Medical Solutions.


In her opening remarks, Rep. Rosa L. DeLauro (D-CT) said that the FDA must work in new ways to enhance science at the agency, recruit more top minds to formulate regulatory regimens to promote safety without stifling innovation, and adopt a multidisciplinary approach. She noted that for FDA to be the thriving center of scientific knowledge, it requires more than resources alone, its culture must change from within.


Regulatory Science Requires Resources and Collaboration


Jesse L. Goodman, MD, MPH, Chief Scientist and Deputy Commissioner for Science and Public Health (Acting) at FDA defined the premise of the FDA’s focus on regulatory science. He said that the science done by academia – often supported by NIH – gives us tremendous insight into basic mechanisms of disease while the work done by the biopharmaceutical industry focuses on getting products on the market. Regulatory science is applied science that aims to develop, assess and provide new, validated tools and approaches to better evaluate the utility of new medical products.


“At our very best, FDA can join with partners to develop tools that make the evaluation process sounder,” Goodman said. “The model of going it alone doesn’t work. The world of science and amount of information out there is just too huge. What we need to do is collaborate and engage with the scientific community.”


Echoing DeLauro’s point, he said that some of it is about resources, but some of it is about doing things in a more innovative way. He noted the need to reduce risk while maintaining patient benefit.


“Along with medical and scientific risks, we need to consider financial risk as well,” said Max Wallace, CEO of Accelerated Brain Cancer Cure. Wallace, who has also founded a number of biotechnology companies, said that capital is the rocket fuel that propels the process from discovery to product.


“Capital is rocket fuel without soul – it hates risk. If risk is doubled by time delays or failure to understand science or opaque non-collaborative environment, the capital will seek its level in other places,” he noted.


Regulatory Science to Benefit Patients


“So, how do these discussions about regulatory science translate into patient benefit?” asked briefing moderator Margaret Anderson, Executive Director of FasterCures.


John Marshall, M.D., Clinical Director of Oncology for Georgetown University Hospital and principal investigator of over 150 clinical trials provided real-world context. He said that the FDA sets the goal, the rules, the parameters that we are to aim for in clinical research: safety and efficacy.


Marshall said that by having the scientific capacity to determine the best models to measure safety and work and collaborate with researchers to design appropriate trials, the FDA can make the process more effective, and efficient. He noted that having the ability to tailor trials to a subset of patients that are known to respond to specific medical products paves the way to a better development system and realizes the promise of personalized medicine.


Ellen Sigal, Chair and Founder of Friends of Cancer Research said that patients have different risks and profiles. But consistent with all patients is the need for more information. Sigal said that “we can evaluate risk with the right data. We want to be able to make choices based on the data.”


Marshall later noted the importance of “building a system that allows buy-in from the patients.”


Regulatory science is the backbone that supports all other FDA activities. Improvements in regulatory science will support better assessment of drug and device safety, and create efficiencies in the development process.


Realizing Change


But, how do we make change happen? Goodman likened making the appropriate changes at FDA to building an airplane while it is flying. He noted that the FDA could be in a position to catalyze change.


Collaborative science is critical to make this happen. Efforts like the Critical Path Initiative and Reagan-Udall Foundation can help by bringing together large numbers of stakeholders to solve problems.


But the FDA needs resources to be able to engage early in the process and enable staff to make regulatory be a part of what they do. He said that the FDA is “very stressed and limited to even engage at the baseline level.”


According to Wallace, “those who are developing ideas are on the bleeding-edge of new technologies and they need dance partners at the FDA.” The agency should ramp up to attract the best scientific talent – that’s where change starts.


Goodman cautioned that it will take five to 10 years to build this new mindset at the FDA. He said that “in the long run, it’s a transformation of culture from just trying to keep up to one that’s a true outstanding scientific partner.”

Research's all abuzz about collaboration and communication

by Margaret Anderson, Executive Director, FasterCures

There have been a fair number of meetings in Washington of late, and a lot of activity related to appropriations. As I speak at, participate in, and attend meetings, I am always listening closely for themes. Here are a few I am picking up with my bionic ear, and it surprises me how often these themes take us back to basics:

1.) Talk to me. Communication is key. In this era of information overload, it’s always good to align (or re-align) priorities and stay focused. For example, at the recent IOM Drug Forum meeting on Regulatory Science, I spoke about how if people don’t understand the many steps it takes to get to treatments and cures, and what processes are in play inside FDA, it’s hard for them to understand how FDA does its job or why a term like "regulatory science" is now more important than ever. In speaking to a group at a prominent academic medical center today, we discussed how collaboration across sectors is critical, but it adds another important to-do to the list. With all the multi-tasking we have to do, unless it's someone's job to ensure we collaborate strategically, collaboration falls under the important but not urgent category. In Hill visits for the Alliance for a Stronger FDA, I am always reminded about how that face-to-face interaction about these issues is needed. Yup, communication.

2.) Work with me. Collaboration has to happen to create advances, but it’s not always a picnic. In the words of Bob Dylan, everybody’s gotta serve somebody. And we all come from different cultures and serve different missions. So we were really glad to see the recent FDA-NIH announcement about a new partnership designed to fast-track treatments to patients. We hope we’ll hear about what works well, what's at a stand-still, and what areas we should just call it quits and chalk it off to experience and move on. To collaborate well, you have to know what the pitfalls are, and where it’s failed before. At the IOM’s recent meeting on "The Public Health Emergency Medical Countermeasures Enterprise" it was great having global health expertise in the room to share lessons learned on process and incentives and apply that to development of vaccines, drugs, and diagnostics for responding to public health emergencies. That discussion was also relevant to the pursuit of therapies for rare and orphan diseases.

At FasterCures, we are looking at some of the thorny issues that stand in the way of collaboration. The list is long and we plan to dive into them deeper this year: biobanking, metrics for medical philanthropists, best practices in the conduct of clinical trials, the value of electronic health records in research, and how we’ll all traverse the valley of death.

3.) It's about me. We will all be patients at some point in our lives and this whole discourse, this enterprise, this system is all about us. We need to always remember to bring the patients into the mix - through effective communications and as a central driver of any collaborative effort. At the IOM Drug Forum meeting, Garrett Fitzgerald of the University of Pennsylvania cited the success of patient-driven foundations at modeling a more modular approach to drug development. Phil Pizzo of Stanford University School of Medicine mentioned that patient advocacy groups have played a critical role in mediating conflict of interest issues. Ellen Sigal of Friends of Cancer Research showed the power of patient-driven groups in partnership with practitioners, to drive change at the FDA. She said we need to increase the focus in patient groups on advocacy for FDA resources and to partake in discussions about their priority areas.

In search of a-ha moments, what I found were some reminders of just how important it is to get the basics right.

Investing in Innovative Medical Research

Each year, donors invest millions of dollars in organizations fighting diseases. Much of this money goes to support individual disease research and finding ways to help our loved ones and communities. But experts in medical research say not all of it is used effectively. This Issue Brief developed by Arabella Advisors and FasterCures explores strategies donors can employ to help improve the current medical research process and to support the search for breakthrough treatments and cures.