In January 2021, 10 of the world’s leading biopharmaceutical companies announced the formation of Accumulus Synergy, a nonprofit corporation intended to enable global, real-time collaboration and data exchange and submission between industry and health authorities. “Our aim is to be at the intersection of where those who develop medicines, and those who approve them, can interact differently,” explains CEO Francisco Nogueira. “Our technology will provide the ability for a sponsor to work with...

Global Forum Co-Editors Gary Kelloff and David Parkinson discuss translational science milestones in 2021 against the backdrop of disruptive technologies advancing disease diagnostics and therapies. “The sensitivity of the diagnostic assays is getting better and better, and so is the analytical validation,” says Gary. “We have now the opportunity with liquid biopsies that allow from 20 cc of blood whole exome sequencing, whole transcriptome sequencing, and therefore deep DNA and RNA...

Innovations in therapeutic product development will only be effective when patients can access and use them. But establishing and explaining the value and price of these innovations among so much unmet need, and ensuring access by patients who need them, has proven difficult throughout Europe and elsewhere. “Policymakers more often or too often have a short-term view, short-term perspective, rather than a long-term perspective, and sometimes are driven more by political goals than promoting...

“There are good reasons to explore the use of artificial intelligence in pharmacovigilance. There are an increasing number of sources of drug safety information, and efficient screening, processing, or evaluation of them would benefit from accurate automated methods,” explains Gerald Dal Pan, Director, Office of Surveillance and Epidemiology (OSE), CDER. “First, there’s the potential application of AI to the processing of case reports. Second, there’s the potential application of AI for...

COVID has demonstrated the importance of regulatory systems that ensure the safety and efficacy of therapeutic products as well as patient access to these products, especially in the complex network of healthcare, scientific, pharmaceutical, regulatory, and patient communities in Europe. “Regulators and industry have worked day and night over the last two years to bring vaccines and therapies forward. We know very clearly that sustainability of the system is an issue. More regulatory...

How are clinical research and clinical care industries in Japan responding to the new challenges emerging in the post-pandemic world? “During 2020, it became a very difficult time to initiate a clinical trial, a difficult time to recruit patients to clinical trials which were ongoing, especially if those were clinical trials in patients who were elderly or in cancer therapies. Patients were a little bit reluctant to go to hospitals, even though we had very few cases in Japan,” explains E....

“We're moving toward an era of truly personalized medicine where research is uncovering the genetic basis for disease as well as mutations and biomarkers that can be targeted with drug and biologic therapies. We need to continue to identify the right patients for these innovative therapies, and this is where companion diagnostics has a foothold,” explains Tiffany Levin (55th Parallel). “One of the key challenges for drug sponsors is the timing of the development and review of companion...

The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body appointed by the Australian Government to recommend new medicines for listing on the Pharmaceutical Benefits Scheme. How have advanced therapies impacted the cost and cost effectiveness of pharmaceuticals, and the work of PBAC, in Australia? “Our decision making is becoming harder because we're being asked to make decisions with substantially more uncertainty about the benefits and safety and the value...

In April 2021, the Pan American Health Organization (PAHO) issued Regulatory System Strengthening in the Americas: Lessons Learned from the National Regulatory Authorities of Regional Reference to overview the Americas’ regulatory landscape and regulatory responses to COVID-19. “The pandemic has highlighted the crucial role that the national regulatory authorities need to play in a public health emergency,” explains PAHO Assistant Director Jarbas Barbosa to Cammilla Gomes, regulatory policy...

Electronic or digital documents helped manage and disseminate clinical research data and information during the pandemic and are now being explored for potential use at the other end of this pipeline: the product safety and other information in the (paper) package insert. “Whether we are speaking of paper or electronic formats, this is the basic right, at the end of the day, for patients: to allow them that access and informed decision making about the use of their product,” suggests Aimad...

In April 2021, the Regulatory Information Management (RIM) Working Group of DIA’s Regulatory Affairs Community issued Version 2.0 of the RIM Whitepaper that provides insights relating to eleven key regulatory capability areas including RIM implementation considerations, processes, and best practices. The Whitepaper also provides context for the RIM Reference Model in development. “One of the key aspects of the model is standardizing terminology and relationships between information,” explains...

DIA Global Forum Australia/New Zealand Regional Editor Richard Day (University of New South Wales, Medicine, St. Vincent’s Hospital) and John Skeritt, Head of Australia’s Therapeutic Goods Administration (TGA), discuss regulatory strategies and other initiatives designed to help nurture clinical research and product development in Australia post-pandemic. With “Australia having had many fewer cases than comparable countries, we've actually become in very short time a very attractive global...

Japan, the world’s third largest pharmaceutical market, has long been engaged in clinical research in every therapeutic area. “COVID-19 totally changed the world. The remote connection is the only way for the CRAs and the sites to communicate and work together, so both the sponsors and the sites started using remote communication or remote monitoring gradually,” explains Eri Sekine, Region Head of Trial Monitoring Japan, Global Development Operations, Novartis Pharma KK, Japan. “So now it's...

How has DIA responded to its communities’ educational and informational needs during the pandemic? “We've always gotten our inspiration and our motivation and our energy from working together, whether it's with our own team on projects or with subject matter experts and key opinion leaders in our stakeholder community. So that reuniting of the community is really central in our minds right now,” explains DIA Global Chief Executive Barbara Lopez Kunz. “One of the things that you're going to...

In 2015, Japan's Minister of Health, Labour and Welfare established an expert advisory panel to focus on Japan Vision: Healthcare 2035 and plan ways to meet the challenges facing the country’s healthcare system over the next two decades. “Japan has been facing an unprecedented situation with fewer children and our aging society. The Japan policy of Healthcare 2035 actually focuses on this problem,” explains Kanmuri Kazuhiro, Ascent Development Services. “The balance of demand and supply will...

The HMA-EMA Big Data Task Force has proposed ten priority actions to help the EU’s medicines regulatory network make best use of big data to support public health. “The first one we mention is the establishment of a Data Analytics Real-World Interrogation Network, also called DARWIN EU, which we see as a natural evolution of what regulators have been doing for decades now,” explains Nikolai Brun, Co-Chair, HMA-EMA Steering Group on Big Data, in this conversation with Thomas Kühler, Head of...

“How can we find drugs to treat COVID as fast as possible? The master protocol is to me the obvious example,” suggests Lisa LaVange, Professor and Chair of the Department of Biostatistics at UNC Chapel Hill and former Director of the Office of Biostatistics at CDER, FDA. “They do require quite a bit more upfront planning but those that are launched and have been running for a while have been able to study a large number of drugs in a really smart way.” Lisa serves on the Therapeutics Clinical...

Global Forum Translational Science Co-Editors Gary Kelloff and David Parkinson look back at 2020 and discuss milestones in the early detection and targeted therapy of cancer and other diseases as well as the impact of the coronavirus pandemic on clinical research.

“As biosimilars receive FDA approval and more and more become available to patients, we're beginning to see a greater understanding and appreciation for these medicines as lower cost options,” explains Dr. Hillel Cohen, executive director of scientific affairs in the Sandoz division of Novartis and Sandoz representative to the education committee of the US Biosimilars Council. “You are not being switched to a new medication. You're being kept on the same medication that's being made by a...

In 2017, Health Canada launched the Regulatory Review of Drugs and Devices (R2D2) initiative with the goal of creating a regulatory system that provides greater and faster access to therapeutic products aligned with Canada's healthcare system needs. “One of our greatest achievements was setting up an aligned review process where a manufacturer can make a drug submission to the regulator and make a submission to health technology assessment at about the same time,” explains Megan Bettle,...

For racial and ethnic minorities in the United States, health disparities take on many forms, including higher rates of chronic disease and premature death. Gaps persist even after differences in health insurance, socioeconomic status, state and severity of the disease, comorbidities, and medical facility are taken into account, says Michelle Durham, Director, Psychiatry Residency Training at Boston Medical Center (BMC). The COVID-19 pandemic has highlighted, and in many cases widened, the...

David Mukanga, Senior Program Officer Regulatory Affairs, Africa Systems, Bill & Melinda Gates Foundation (and Africa Regional Editor for DIA Global Forum) explores the East African Community’s Medicines Regulatory Harmonization (EAC MRH) initiative with Margareth Sigonda, Head of Health Programs for The New Partnership for Africa's Development, the African Union development agency. “Before this initiative was launched in the EAC, each country had different requirements, different formats...

2020 marks the tenth anniversary of the FDA’s Biosimilars Price Competition & Innovation Act, which created a regulatory approval pathway in the US for biosimilars designed to increase access to safe, effective and cost-effective biological treatment options for patients. “I have absolute confidence in these products as a scientist and seeing them work clinically, but we've got to make it work commercially,” explains Gillian Woollett (Avalere Health) in this interview with Anna Rose Welch...

“How do you do the process of regulation? Not the science of regulation, but how do you do the process of regulation more flexibly and agilely?” asks Murray Lumpkin, Deputy Director Integrated Development and Lead for Global Regulatory Systems Initiatives, Bill & Melinda Gates Foundation. “We certainly hope that the openness that regulators around the world have to this concept of reliance, of referencing, will evolve and strengthen because of what we're all going through now,” says John...

The focus on clinical trials to discover therapies that alleviate suffering caused by COVID-19 also spotlights the regulators responsible for monitoring patient safety and scientific rigor in these trials. “The common goal is to define better therapies for patients and needs. At the same time, we are driven by science, so we need to do what is right on a scientific perspective in order to not create additional burden for patients, for physicians, for communities, for payers, but to really...