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September 30, 2022: CTTI’s Digital Health Trials Hub Recommendations and Resources to Run Your Digital Health Trial (Marianne Chase, MGH, CTTI Team Lead, Jörg Goldhahn, ETH Zurich, CTTI Team Lead)

Rethinking Clinical Trials

Digital Health Trials can help research obtain better and more reliable information, conduct more patient-centric research, and move at higher speed and efficiency. We need to work together to find ways to evaluate the potential benefits and share lessons learned broadly. Find CTTI’s recommendations and resources. pctGR, @Collaboratory1.

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June 24, 2022: FDA Draft Guidance on Real-World Evidence (John Concato, MD, MS, MPH)

Rethinking Clinical Trials

Big Data, a term first used in the 1990s, leverages modern technology to increase the quantity, forms, speed, and capability to manipulate large-scale data. Big data; Real-word evidence; Real-world data; 21st Century Cures Act; FDA Draft Guidance. Key Points. and the FDA Draft Guidance for RWD/RWE. pctGR, @Collaboratory1.

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Grand Rounds July 15, 2022: Overview of Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Public-Private Partnership and Lessons Learned (Stacey J. Adam, PhD)

Rethinking Clinical Trials

ACTIV was stood up in about a month, launching on April 17, 2020, to develop a coordinated research response to speed COVID-19 treatment and vaccine options. The normal timeline for public-private partnerships is around 9 months of planning. In the therapeutics arm, the most promising therapeutic agents for COVID-19 were prioritized. .

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Epigenetics discovery could lead to new class of cancer drugs

Drug Discovery World

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. But, until now, scientists lacked an understanding of what the chemical tag does, despite many years of research.

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Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

According to one report, the orphan drug market is now growing at twice the speed of the non-orphan market, and a rise in the number of patients being diagnosed with rare diseases makes continued growth highly likely. Go-to-market strategies. The choice of where to launch an orphan drug is an important and difficult decision.

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Grand Rounds June 30, 2023: Decentralized Trials – From Guidance to Reality & What’s Left (Adrian Hernandez, MD, MHS; Pamela Tenaerts, MD, MPH; Craig Lipset, MPH)

Rethinking Clinical Trials

Over the past several years, a variety of factors have accelerated the need for decentralized trials, including the push to make trials more accessible, the increased speed of science, the possibility of environmentally conscious trials, and the need to be flexible in a rapidly changing world.

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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

The newly inserted IDS gene produces an IDS enzyme that contains a proprietary ApoEII-tagged sequence, which can bind to ApoE-dependent receptors on the blood brain barrier, and move enzyme into the brain more efficiently, thus potentially normalising brain pathology.