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 for dossier submission,” Margareth explains. “Therefore, adopting the CTD was really a game changer in terms of how now the countries were in a position to receive applications that were kind of in a similar format.”
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 (Chief Editor, Biosimilar Development, Life Science Connect). “I always say biosimilars offer savings–but be careful with that statement. That means somebody is not receiving the money they were previously receiving, and they don't want to forego that money.” Anna Rose will moderate the Short Course on Current Biosimilars Policy at DIA’s 2020 Biosimilars Conference.
“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 Lim, founding Executive Director of the Center of Regulatory Excellence at Duke-NUS Medical School in Singapore. (Listen to our previous Pandemic Vaccine Preparedness podcast with Dr. Lumpkin.)
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 deliver innovative products and medicines,” explains Dr. Max Wegner, Senior Vice President of Regulatory Affairs at Bayer. “What is the desire of the pharmaceutical industry? I think the desire is to have new products reaching patients. What is the desire of the regulators? I think it's the same.”
After her stage four metastatic cancer diagnosis in 2011, writer Mary Elizabeth Williams was selected to participate in one of the world’s first immunotherapy clinical trials. Twelve weeks later, she showed a complete response. Williams will explore the clinical trial process in her DIA 2020 Keynote Address. “If we are creating protocols for patients who, like me, are otherwise perfectly healthy and have no comorbidities, you're not in any way serving the people who are the most vulnerable,” she explains. “Look at who is most directly impacted by this virus: It's people who have pre-existing conditions and yet people with pre-existing conditions are routinely shut out of the clinical trial process.”
Cures Within Reach (CWR) was founded as a not-for-profit dedicated to repurposing drugs and other treatments to drive more treatments to more patients more quickly. “If you really think about the difference between de novo and drug repurposing, we think it falls into three key areas: the speed, the cost, and the success rate,” explains President and COO Barbara Goodman. “From de novo research, it takes about 13 to 15 years, it can easily take up to two billion dollars, and some would say it's five to ten percent success rate. In the drug repurposing side, it can be significantly shorter in five to eleven years, it can be $300 million, $350 million, with perhaps a 30% success rate. This happens because the safety, toxicity, pharmacology and other related studies are completed from the original indication.”
In late March 2020, FDA issued Guidance on conduct of clinical trials of medical products during COVID-19 public health emergency: Guidance for industry investigators and institutional review boards. In the agency statement accompanying its release, FDA Deputy Commissioner for Medical and Scientific Affairs Anand Shah stated, "FDA released this guidance to emphasize that at all times patient safety should continue to be at the forefront of considerations. We want to support the continuance of these clinical trials in compliance with good clinical practice and minimizing risks to trial integrity, while also safeguarding the health and well-being of study participants."
The global COVID-19 pandemic has brought new urgency and challenges to reporting drug adverse events in clinical trials and the consumer market. "It may well be the case in the months to come that we learn of adverse events which took place during the lockdown but which are only reported at a much later stage," suggests Dr. Thomas Leigh, an Executive Medical Director and Head of the Medical Group within Patient Safety Solutions & Adjudication for Covance. "The pandemic and its effects are significant, and it will be very important for all of us involved in the assessment of safety of medicines to look out for evidence of safety events which perhaps have not previously come to light." This podcast is sponsored by Covance Patient Safety as part of DIA’s White Paper Library. Download and listen to this podcast.
Even under COVID-19 quarantine, patients wearing remote sensors keep clinical research moving forward digitally. “Now suddenly, very suddenly, we're faced with an intense patient safety issue where we really are risking patients' lives by having them come into these clinical sites,” explains Paul O’Donohoe, Medidata Solutions. "I suspect we're going to see a significant uptake in the acceptability and use of technology as a way of allowing patients to now provide us that data in the safety of their own homes.” Bill Byrom, Signant Health, moderates this podcast with Paul and Jennifer Goldsack, Digital Medicine Society, and serves on the Steering Committee for the August 2020 virtual Digital Technology in Clinical Trials conference co-sponsored by DIA and the ePRO Consortium of the Critical Path Institute.
Latin America, a region of more than 640 million people, is experiencing significant outbreaks of COVID-19 since its first confirmed case on February 25, 2020. Health regulatory authorities are required to adapt to new challenges as the regions face medical supply shortages and halted clinical trials. “In our region, governments will learn even more the value of an independent regulatory authority that does regulation based on science and scientific evidence,” says Cammilla Horta Gomes, Latin America Regulatory Policy Lead at Roche, Brazil. She discusses the outbreak’s impact on the region’s regulatory landscape and the solutions that have been implemented to improve regulatory efficiencies in the face of the outbreak.