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.”
Through FDA’s Patient Focused Drug Development and similar initiatives, the value of the patient voice in drug development is clear. “It’s not just a question of science but also one of social justice,” suggests independent filmmaker Jennifer Brea, Director for Shella Films and founder of the rare disease community MEAction. “When no one’s paying attention to your community, and no one thinks that what you’re going through is particularly urgent or important, how do you put your disease on the agenda?”
“Patient engagement is a subset of participatory medicine. Participatory medicine is about partnership between the patient and the clinical or scientific expert,” explains “ePatient Dave” deBronkart. “The whole purpose, the context, for this entire initiative is not to have physicians feel threatened, but to help healthcare achieve its potential with the patient contributing what they're able to.” Author of Let Patients Help: A Patient Engagement Handbook, Dave is a cancer survivor, patient advocate, and activist working to revolutionize the relationship between patients and healthcare providers.
“Safety case processing is probably the largest bundle of effort in a PV department, and some of these robotic and cognitive automation tools today are allowing us to reduce the effort it takes to process a case while also improving both quality and compliance,” explains Dinesh Kasthuril, Director of Client Services, Covance Patient Safety. “When something like cognitive automation can be combined with robotic process automation, we’ll see significant savings and efficiencies.” This podcast is sponsored by Covance Patient Safety as part of DIA’s White Paper Library. Download and listen to this podcast.
In March 2019, the Health Ministry of India released new guidelines for clinical research. What is the state of pre-market and post-market safety for patients and drugs in India, six months after these new guidelines were announced? Dr. Srikanth Krishnamurthy, a consulting pulmonologist at Sri Bala Medical Centre and Hospital, discusses the pre-market clinical research safety perspective; and Dr. J. Vijay Venkatraman, Managing Director and CEO at Oviya MedSafe, contributes the perspective of post-market drug safety in India.
Where is real world data providing evidence that impacts therapeutic product development? “We're learning that, right now, the near-term uses are probably around label expansion and around providing information on new populations that's valuable but wasn't captured in randomized clinical trials or could not be captured in those trials,” explains Nicole Mahoney, Senior Director, Regulatory Policy, for Flatiron Health. “The real thing that you have to ask is: What decisions are you driving at? What is the regulatory question that you're trying to support with real world data?”
“We understand we need to educate people wherever they are about the power of personalized medicine,” says Edward Abrahams, President of the Personalized Medicine Coalition, which promotes the understanding and adoption of personalized medicine concepts. “We look forward to the day when patients ask, ‘Is this going to work for me?’ And we look forward to the day when providers can answer that question. It's a great question.”