Proceedings: DIA 2018 Global Annual Meeting

Analyzing Innovations Progress in the Gottlieb Era

Chris M. Slawecki
DIA Senior Digital Copy Editor


hrough his writing on the FDA Voice blog and numerous public remarks and speeches, FDA Commissioner Dr. Scott Gottlieb has made his ambitions clear: more modernization and innovation in clinical research, drug development, and regulatory review and approval, to significantly reduce the clinical impact and cost burden of disease for healthcare systems and patients. In the DIAmond session “Analyzing Innovations Progress in the Gottlieb Era,” experts from the realms of regulatory affairs, venture capital and industry discussed the Commissioner’s impact on, and new opportunities for, industry-agency collaboration in the development and regulation of innovative new therapies.

Key Takeaways

Key focus areas of Commissioner Gottlieb’s modernization efforts:

  1. Improving FDA’s efficiency by reorganizing the Office of New Drugs (OND) to centralize project management functions and processes.
  2. Pushing boundaries of FDA’s oversight scope into cell therapies, opioids, and drug costs (the latter “indirectly” by driving more rapid FDA reviews and approvals of generic drugs).
  3. Ensuring sufficient FDA resources through Congressional appropriations and improving employee recruitment and retention.
  4. Facilitating and streamlining medical product development by promoting innovative clinical trial approaches and designs, and by expanding partner and pilot programs such as the Center for Devices & Radiological Health’s (CDRH’s) Digital Health Innovation Action Plan in anticipation of emerging technologies.

DIA Global Chief Executive Barbara Lopez Kunz discusses “Analyzing Innovations Progress in the Gottlieb Era” with Session Chair Nancy Bradish Myers.

Because drug development is a data-driven enterprise, Myers presented metrics from Gottlieb’s first (approximately) 400 days. “Most Commissioners are judged by the numbers while they’re there, and these are quite impressive,” she suggested.

  • New draft and final guidances released: 286
  • Breakthrough therapy designations granted: 53
  • Regenerative medicine and tissue (RMAT) designations granted: 16
  • Generic drug approvals: 859
  • Biosimilars approved: 7

Perspective: Prescription Biotechnology and Pharmaceutical Industry

The amount of intelligence and communication emanating from this FDA is remarkable. FDA has quite obviously (and strongly) embraced:

  • a new understanding that the Agency is now an environment of rapid learning, and that regulatory science continues to advance in Agencies both in the US and in other countries,
  • the concept of accelerated regulatory approvals – nearly 60 percent of 2017 approvals utilized some type of accelerated pathway, and
  • the patient-centric approach to benefit-risk analysis by asking: What truly matters to patients with this disease?

We’ve seen FDA’s Breakthrough Therapy Designation adapted and customized locally by the EMA in the EU and by PMDA in Japan, but it’s also interesting to observe the current regulatory reforms in China, which seems to be adopting the best and brightest ideas from other agencies around the world.

Perspective: Generics, Devices, and OTC Industry

As supplier of over the counter (OTC) tests with many different potential outputs, 23andMe faced numerous regulatory challenges when it planned its market entry. The first challenge: What was FDA regulating: the test, its results, the device, or all three? For example, FDA might not be interested in what genetic percentage of you came from Scotland, but might be interested if you’re genetically predisposed to cystic fibrosis. To answer these and related questions, collaboration with the Agency was essential. Throughout this process, FDA demonstrated its willingness to work with the sponsor and consider its data. In 2017, 23andMe received FDA authorization for a genetic test and ten specific genetic health risk reports, and is now cited as an example of how the FDA pre-authorization process can work effectively.

Session Chair: Nancy Bradish Myers, Catalyst Healthcare Consulting, Inc.

Panelists: Sandra A. Milligan, DIA Fellow, Merck; Kathy Hibbs, 23andMe; Doug Cole, Flagship Pioneering

Perspective: Venture Capital

Progress in therapeutics is usually incremental; but the landscape can change into something unrecognizable over ten to fifteen years, even if each step along the way seemed unremarkable in its time. As modern biological and related sciences can inform questions in remarkable new ways, we collectively stand at a pivotal point in what Cole called “the biological century,” a special moment worth cultivating.

The “tone from the top” of the Agency remains so positive that capital continues to flow into what remains a relatively risky drug and device investment market. More certainty in the regulatory process leads to less uncertainty in the requirements for regulatory review and approval. At the same time, regulatory science must continue to evolve as science and technology continue to advance.

Are there areas of regulatory science where FDA could do more to move the ball forward?

An unintended impact of increased regulatory transparency is that it’s increasingly difficult to keep up with all the regulatory communications coming from not only the FDA but other agencies as well. Some companies are exploring artificial intelligence tools to more effectively manage this wave of information.

That said, several recent guidance documents for device developers not only reflect FDA thinking in this area but prepare the way for incorporating artificial intelligence, machine learning, and real-world data into their development, and prepare regulators for these innovations. The recent Patient-Focused Drug Development: Collecting Comprehensive and Representative Input guidance is a good example; it is a useful tool for pharmaceutical companies, device companies, and patient engagement groups in their collaborative work.

Cole suggested that society receives the drugs that its regulatory systems are set up to deliver. For example, why is there so much current clinical research in cancer? Is it because the FDA has implemented a relatively distinct regulatory pathway for these products?