“Digitization is something that is happening to everything that we do. It’s being called the fourth industrial revolution,” explains Dr. Robert Califf, former FDA Commissioner and Vice Chancellor for Health Data Science at Duke Health. “And if we do it right, it will actually free up clinicians to spend more time directly with people and, when they become patients, with sick people, which is what we really want.”
Do changes in clinical research, driven by emerging technology, change what we mean by “evidence”? “It’s not necessarily ‘trials versus real world evidence’–that is not particularly helpful because either type of evidence, either type of data, has its place and is complementary,” explains Mr. Michael Seewald, WorldWide Head of the Center of Excellence for Real World Evidence, Novartis Pharma, and member of DIA’s Future of Evidence Workshop program committee. “The exciting development is in developing data sources over a timeframe of decades that at some point will revolutionize healthcare in a respective therapy area.”
“One critical area when you look at healthcare systems is the issue of optimizing clinical and operational effectiveness,” suggests Professor John Lim, founding Executive Director of the Duke-National University of Singapore Centre of Regulatory Excellence (CoRE). “This is an area where research in AI is developing to really inform and aid decision making by not only physicians but also then operational decisions by healthcare providers.” Professor Lim is also Chairman of the Singapore Clinical Research Institute and National Health Innovation Center.
“China joining ICH is a magnificent milestone for the pharmaceutical industry in China,” explains Dr. Jingsong Wang, Chairman and Chief Executive Officer of Harbour Biomed. “We can see clearly more and more local pharmaceutical companies adopting the international standards to have a more innovative portfolio to face the competition from their global partners or counterparts.” Dr. Wang serves on the Steering Committee for our DIA China 2019 Annual Meeting in Beijing.
Should prescription drug or device advertisements include their price? “I don’t know how patients will perceive that because they won’t really know the difference between what they may or may not end up paying,” suggests Micheline Awad, Director, Regulatory Affairs, Advertising & Promotion, Neurocrine Biosciences, Inc. “We really haven’t studied in any systematic way the impact on the patient or on drug use or on being more transparent about drug prices,” explains Wayne Pines, President, Regulatory Services & Healthcare, APCO Worldwide, Inc. Micheline and Wayne served on DIA's Advertising & Promotion Regulatory Affairs 2019 program committee.
Dr. Yoshiaki Uyama, Director of the Office of Medical Informatics & Epidemiology for the Pharmaceuticals and Medical Devices Agency (PMDA), Japan, and DIA Global Forum Regulatory Science co-editor, shares his vision for utilizing real world evidence in regulatory science. “The challenge is, we still don’t understand completely the characteristics and the limitations of those data. We are very familiar with assessing or evaluating the data of a clinical trial. But real world data is not completely of the same character as that of a clinical trial because those real world data are not primarily collected for such purposes.”
In 2018, the US FDA approved a record high number–59–of new, novel drugs. “We are at a point now where there is so much innovation in this space and the regulatory aspects are catching up, so we now have three approved gene therapy products in the US and over seven to eight hundred INDs for cell and gene therapy filed with FDA,” explains Dr. Adora Ndu, Executive Director of Global Regulatory Policy, Research & Engagement, BioMarin Pharmaceutical, and Regulatory Science Co-Editor for DIA Global Forum. “With each interaction with each product that’s filed and with each product that’s approved, the regulatory experience is broadened.”
“Genetics is about everybody, and this information is really fundamental to you and your health,” explains Dr. Emily Drabant Conley, Vice President of Business Development for 23andMe, the first company to receive US regulatory approval for their over the counter genetic ancestry and health tests. “If individuals are empowered with information about their genetic predisposition, it puts them in a position to take action.”
“One of the best questions we can ask is, why?” explains Deborah Collyar, Founder and President of Patient Advocates in Research. “People in industry are beginning to realize that we have reached a tipping point,” suggests Dr. Lode Dewulf, Chief Patient Officer at Servier Group. “The risk of not engaging patients has for the first time become bigger than the risk of engaging patients.” Deborah and Lode serve as Patient Engagement Editors for DIA's Global Forum.
Niklas Hedberg, Chief Pharmacist for the Dental and Pharmaceuticals Benefits Agency in Sweden, chairs the executive board for the European Network for Health Technology Assessment known as EUnetHTA. “We typically see that there are a lot of comparative questions that are not or even cannot be answered by traditional randomized control trials,” he explains. “Anything that is outside the typical randomized controlled trial scope is probably going to be of increasing importance for the relative effectiveness and relative cost evaluation.” Mr. Hedberg hosted the EUnetHTA Town Hall at DIA Europe 2019.