DIA: Driving Insights to Action

Regulatory and Clinical Science Align to Fight COVID-19

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.”

DIA Keynote Calls for Expanded COVID-19 Trial Parameters

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.”

Impact Philanthropy Accelerating Cures Within Reach

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.”

FDA Guidance: Flexibility But No Compromise on Patient Safety

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."

Digital Tools Powering More and Safer Clinical Trials

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's COVID-19 Regulatory Response

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.

Partnerships Essential to SAHPRA Pandemic Response

The South African Health Products Regulatory Authority (SAHPRA) is responsible for regulating health products intended for human and animal use, the conduct of clinical trials, and other duties in South Africa. “Around the COVID-19 pandemic, as regulators, we are having to work very closely with each other because in some jurisdictions clinical trials are ongoing, and we need to access that data. In some jurisdictions, products have been registered and we need to access that data,” explains SAHPRA CEO Dr. Boitumelo Semete-Makokotlela. “It's been humbling to be part of the broader health sector where we contribute to not only saving the lives of South Africans but contribute to the knowledge base across the world and to averting this pandemic.”

EFPIA Shares Industry View of EMA Regulatory Framework

Scientific advancements in pharmaceutical R&D are simultaneously benefiting patients and challenging industry and regulatory frameworks throughout Europe. “The pharmaceutical R&D of today is driven by groundbreaking innovation which has the potential to transform patient treatments and improve patient outcomes, as we move more and more from treating symptoms to really treating the underlying cause of diseases,” explains Dr. Nathalie Moll, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA). “Developing this new generation of treatments raises new and challenging questions of our regulatory system, underlying the need for evolution to adapt to such advanced technologies.”

Time to Press the Pause Button on Speedy Drug Approvals?

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?’”

Reach More Patients While Improving Data Quality?

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.