December 29, 2020 | This year clinicians everywhere have worked to understand COVID-19, its prognosis for different groups, and the progression of illness. At Clinical Research News, we began posting weekly literature roundups on Fridays early in the pandemic and those roundups alone are a fascinating capsule of our growing understanding of disease management and treatment.

But COVID-19 wasn’t the only disease we were concerned about this year. When much of regular life shut down, clinical trials were no exception. Crucial research on cancer, rare diseases, and many other indications was put on hold while sponsors and CROs re-envisioned clinical trials virtually and worked to reassure participants that they were safe at sites. All of the tentative forays into decentralized and virtual trials were fast tracked to get us back on track. This year we covered the best way to use real-world evidence, improving ROI in clinical research, applying AI to clinical data, and much more. —The Editors

Using Real-World Evidence To Gauge Drug Effectiveness: FDA released a framework for its Real-World Evidence Program: what makes RWD fit for use, how to generate RWE to provide adequate scientific evidence to address regulatory questions of interest, and how to ensure the conduct of the study meets regulatory requirements (e.g., study monitoring and data collection). The Agency is scheduled to issue a draft guidance by the end of 2021.

Trends in Drug Development: Improving ROI on R&D: According to a report released early this year, a surge in innovative pharmaceutical drug discovery has coincided with a decline in a return on investment (ROI) in pharmaceutical R&D. The leading approaches to stop the decline are address the cost of clinical trial development, to collaborate with other companies, to supplement traditional research methods with artificial intelligence (AI), and to be willing to change company paradigms.  

Applying AI To Clinical Data: Pfizer’s Approach to Identifying Use Cases: Presenting at the Summit for Clinical Ops Executives, Demetris Zambas, head of data monitoring and management for biometrics and data management in Global Product Development at Pfizer, outlined how the big pharma used a modified hackathon to validate machine learning tools.

Making the (Regulatory) Grade With Real-World Data: Assessing the quality of your real-world data is a crucial step in applying the findings to real-world scenarios. So how do you know if your data are any good? Relevant data are robust, representative of the population of interest and determined by the question at hand.

Could Plasma Dilution Be The Answer To Age-Related Diseases—and Cytokine Storm?: Therapeutic plasma exchange (TPE) is currently being used to treat more than 50 relatively uncommon diseases, primarily autoimmune disorders, and a pair of bioengineering experts at UC Berkeley, have been studying its impact on systemic aging.  

Research Ethics Knocked Sideways By The Pandemic: A growing number of health researchers and biomedical ethicists have begun speaking out with the message that “there are no shortcuts” to defeating the SARS-CoV-2 virus. Circumventing basic principles of science and fairness will do more harm than good, adding to the death toll and enlarging inequities, they warn.

COVID-19: A Catalyst For Innovation In Clinical Research?: Clinical trials not dedicated to the pandemic are struggling to remain operational. Guidance from FDA has attempted to provide clarity to the situation, offering solutions to challenges such as quarantines, site closures, travel limitations, and possible interruptions to the supply chain. But the pandemic may be what finally forces the industry to change.

Technological Ways To Make Clinical Trials Really Global: Although clinical trials are meant to cover global populations, the pool of patients currently enrolled in clinical trials is not representative of world populations. In a contributed commentary, Estelle Haenel at Kayentis, argues that in order to broaden the recruitment of patients enrolled in clinical trials, clinical research opportunities in remote areas must be increased. The inclusion/exclusion criteria should be made more flexible and regulatory requirements ought to be harmonized by the competent authorities.

‘Hybrid’ Trials Will Be Post-Pandemic Norm: For Medable, a leading advocate of decentralized clinical trials (DCTs), COVID-19 attracted surprising if welcome attention to the idea of making studies more patient-centric. The pandemic helped grow company revenues 400% in the first half of 2020 and, in the estimation of CEO and co-founder Dr. Michelle Longmire (a Stanford-trained physician), accelerated DCT adoption by five years.