New drugs are getting through the FDA approval process faster. According to a study published in January 2020, the review time for standard drug applications has decreased from more than 36 months in 1983 to approximately ten months in 2018. “It can be a good thing for drugs to reach the market faster, if those drugs offer large benefits. But often drugs offer only very small benefits over existing treatments,” says Dr. Jonathan Darrow, pharmaceutical policy expert and Assistant Professor at Harvard Medical School in Boston. “The system we have is a patchwork of various policies,” explains Dr. Joshua Sharfstein, former FDA Principal Deputy Commissioner, now Vice Dean and Professor at Johns Hopkins Bloomberg School of Public Health in Baltimore. “We should take a step back and say, ‘Are there ways to align incentives more so we have more money to spend for treatments that make a huge difference for people who need them?’”
Digital tools that revolutionize information and communication technology are increasingly employed to transform the quality and quantity of clinical trial data. “The ability for us to reach patients in geographic locations and in demographic groups that the current system is missing is arguably the most important role of these technologies,” suggests Lauren Oliva, Global Regulatory Policy Lead for New Technologies, Biogen. “With the increased quantity of data, you do increase the quality to some extent in terms of your reach and the types of things that you could measure now that you wouldn't be able to measure in the past,” explains Josh Cosman, Principal Engineer, Digital & Quantitative Medicine, Biogen. Lauren will Chair the Drug Development Tools in a Digital Era session at DIA Europe 2020.
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.”