FDA’s Center for Biologics Evaluation and Research (CBER) is responsible for assuring the safety and efficacy of biological products including vaccines, allergenic products, blood and blood products, and cellular tissue and gene therapies. “This area of advanced therapy medicinal products (ATMPs) is just an incredibly exciting one to be working in right now,” explains Center Director Dr. Peter Marks. “We have our work cut out for us here, but to me it's wonderful work to have to do because it shows how fast the science is advancing, how fast hopefully the technology will advance, to bring these medical benefits to patients.”
The DIA Europe 2020 Keynote Address will focus on the intersections of science and business as explored by a scientist turned investor, Mr. Joep Muijrers, who currently serves as CFO of PureTech, a listed biopharmaceutical company that focuses on developing medicines for devastating diseases including cancer, CNS disease, and immunological disorders. “What is the value of a product that, quite often against the odds in this sector, makes it and gets to patients and then doesn't make a dent whatsoever – it's not being used. What's the relevance of that?” he asks. “Put it this way: In this industry, we tell the end user what's good for them. Isn't that crazy?”
Dr. Steven Murray, Group Vice President & Principal Engineer in the Electrical & Data Sciences Group for Exponent, assesses and analyzes potential risk and failure of products from underground lateral piping to surgical heart catheters. “In drugs and medical devices, developers really need to consider where their product lies on the scale of risk: Is this a product where it is impossible to disentangle the benefits from some other risk? Or is this a product that, if anything is even a little bit wrong, the benefit doesn't justify it?” he asks. “We're much more accepting to risks in things like cancer drugs or AIDS drugs, where the risk of death is highly present and you're averting it.”
Telematics has evolved since the first joint HMA/EMA Telematics strategy in 2014. “The vision for EU telematics is an IT collaboration that will deliver cost-effective, efficient, and interoperable services to the European regulators and to its stakeholders,” suggests Bernd Misselwitz, Director and Regional Head, Regulatory Submission Management, Bayer AG (Germany). “Maybe from an IT perspective, it was the technical problem first and business was second; now that you've seen how people want to get value out of it, it's starting to shift where the business problem comes first,” explains Vada A. Perkins, Executive Director, Regulatory Policy & Intelligence at Bayer Pharmaceuticals (Global). Vada and Bernd will help lead the DIA Europe 2020 session Telematics Strategy in the EU: Global & Regional Considerations for 2020-2025.
As drug development and market approval grow more connected and complex, effective regulatory information management (RIM) is more essential than ever. “The ideal state would be to leverage regulatory data as a corporate asset,” suggests Venkatraman “Bala” Balasubramanian, Senior Vice President of Life Sciences, Orion Business Innovation. “Regulatory teams will be able to better advise portfolio teams on probability of regulatory success in any given market, so you’re going to have higher success rates from a regulatory perspective going forward,” concurs Patterson Shaffer, Managing Director of Regulatory Compliance & Quality, Grant Thornton LLP. Bala and Pat serve in the Working Group developing the Regulatory Information Management reference model discussed at DIA's 2020 Regulatory Submissions Information & Document Management Forum.
Co-editors Drs. Gary Kelloff and David Parkinson discuss topics raised in the translational science series of articles in the 2019 Global Forum and their implications in 2020 and beyond. "Biological understanding is absolutely necessary for more efficient therapeutic development but also for more efficient clinical decision making," David suggests. "There are proven cases, like for rheumatoid arthritis, where you can look at the proteomic profiles and decide which patients are going to benefit from methotrexate from those that are that are not. That's an example of being able to predict ahead of time with proteomics and epigenetics which patients need to be enrolled to a given trial and which not," concurs Gary.
Risk-based monitoring (RBM) was codified into clinical research by the November 2016 Integrated Addendum to the ICH GCP Guideline (E6[R2]). Many organizations now face challenges in implementing RBM in an R&D environment which is simultaneously (and increasingly) impacted by machine learning and artificial intelligence. “Process automation has advanced to make large numbers of tasks ‘invisible,’ ‘behind the scenes’ in the computer and that leaves only the tasks that matter most available for us as humans to execute,” explains Gregg Larson, Vice President of Clinical Operations at Ultragenyx Pharmaceutical, Inc. “Technology has not yet evolved to the point of thinking for humans. We still have to do the thinking.”
In February 2019, heads of state and governments of African countries endorsed the creation of an African Medicines Agency. “The concept of reliance is becoming really, really important and many countries and many regions are recognizing that,” explains David Mukanga, Senior Program Officer Regulatory Affairs, Africa Systems, Bill and Melinda Gates Foundation. “Now companies see a region coming together, more simplified, more efficient processes, and it makes it attractive for industry to then file applications into these regions.”
“Though there's different perspectives, and different reasons why we're all in healthcare, it is all about the patient; and so, getting patient access to important therapeutics that change their lives is why we're all here,” suggests Sandra Milligan, Senior Vice President, Head of Global Regulatory Affairs and Clinical Safety, Merck. “It's amazing to see regulatory agencies stepping up, recognizing that the health of their population is so important. Look at the regulatory revolution in China. Incredible. Having a healthy population is key to economic success.”
Japan’s Clinical Research Act, which came into force in April 2018, codified the term “clinical research” for the first time in the nation’s history. “‘AI’ has become a big buzzword and it is making a huge impact on the way we conduct clinical trials, as well as on various areas of R&D activities,” explains Dr. Takashi Sato, Manager for Kyowa Kirin Co., Ltd., and Vice Chair of the 16th DIA Japan Annual Meeting 2019: To deliver rational medicine to all people in the world. “Another one is clinical trial renovation. In five years from now, clinical trials will look very different from what they are now.”