Clinical Trial News
Clinical Trials Evolution: New Data Management and Design Approaches Set to Transform Drug Development in 2020
• Clinical trial designs are evolving to incorporate broader patient populations, with new approaches including longitudinal studies, synthetic data integration, and combined results from both physical and virtual sites.
• Up to 90% of US clinical trials face significant enrollment delays, prompting the adoption of hybrid trials, community-based sites, and digital engagement strategies to improve patient recruitment.
• The industry is moving towards reusable data models, combining EHR data with trial information to create comprehensive repositories, while synthetic data emerges as a solution for trial efficiency and GDPR compliance.
Rare Disease Drug Development Faces Complex Challenges Despite Growing Pipeline
• Despite affecting 30 million Europeans, approximately 90% of the 7,000 known rare diseases still lack effective treatments, highlighting a significant unmet medical need.
• Orphan Drug Designations have declined by 49% at EMA and 8% at FDA between 2014-2019, while pricing pressures and clinical trial complexities continue to challenge development.
• Industry remains committed to rare disease research with nearly one-third of pipeline drugs targeting orphan conditions, supported by patient advocacy groups and regulatory incentives.
Novel Neural Biomarker ERNA Shows Promise for Optimizing Parkinson's Disease Deep Brain Stimulation
• Researchers have identified evoked resonant neural activity (ERNA) as a reliable biomarker that could improve deep brain stimulation electrode placement and programming for Parkinson's disease patients.
• ERNA demonstrates key advantages including large amplitude signals, localization to the dorsal subthalamic nucleus, and persistence under general anesthesia, making it potentially valuable for both awake and asleep DBS procedures.
• Clinical studies show ERNA amplitude correlates with therapeutic benefits, suggesting its utility for optimizing electrode configurations and potentially enabling more precise, personalized DBS treatment approaches.
Psychedelics and Psychedelic-Assisted Psychotherapy: A Review of Clinical Applications
This article reviews the clinical application of psychedelic drugs in psychiatric disorders, focusing on MDMA, psilocybin, LSD, and ayahuasca. It highlights the resurgence of research into their therapeutic potential, supported by organizations like MAPS and the Heffter Research Institute, and discusses their efficacy in treating conditions such as PTSD, depression, and anxiety.
Roche and Bicycle Therapeutics Forge $1.7B Cancer Immunotherapy Alliance
• Roche's Genentech unit partners with Bicycle Therapeutics in a $1.7 billion deal to leverage innovative bicyclic peptide platform for cancer immunotherapy development, with a $30 million upfront payment.
• Bicycle's proprietary technology combines antibody-like targeting with small-molecule drug properties, offering unique advantages in manufacturing, dosing, and tissue penetration for cancer treatment.
• The collaboration focuses on discovering novel cancer immunotherapy targets, with Bicycle handling early development and Roche taking over clinical-stage programs.
NIH Initiates Clinical Trial of Remdesivir for COVID-19 in the U.S.
• The National Institute of Health (NIH) has begun a clinical trial of Gilead Sciences' remdesivir to treat COVID-19 in U.S. hospitals.
• The first participant is an American patient at the Nebraska Medical Center, who was quarantined from the Diamond Princess cruise ship.
• Clinical trials are also underway in China to evaluate remdesivir's efficacy against SARS-CoV-2, the virus causing COVID-19.
• U.S. health officials are preparing for potential significant disruptions due to the expected spread of COVID-19.
Clinical Trial Sponsors Face Mandate to Publish Decade of Missing Data
• A federal judge has ruled that government research agencies misinterpreted a law requiring clinical trial data publication, creating a 10-year gap.
• Universities, drug companies, and medical device manufacturers may need to release previously unpublished data from trials of unapproved drugs and devices.
• The ruling affects trials conducted between 2007 and 2017, potentially impacting hundreds to over a thousand noncompliant trials.
• Experts assert the decision enhances public access to clinical trial results, making it harder to conceal unfavorable outcomes.
Digital Technologies Poised to Reverse Declining ROI in Pharmaceutical R&D
• Pharmaceutical R&D costs have nearly doubled from $1.1 billion in 2010 to $2.1 billion in 2018, while ROI has plummeted from 10.1% to 1.9%, prompting industry-wide concerns.
• AI, Big Data, and advanced analytics are emerging as key technologies to improve R&D productivity, with 80% of biopharma companies now implementing these solutions compared to 36% in 2017.
• Digital transformation is reshaping organizational structures, with 70% of surveyed firms reporting breakdown of internal silos and formal reorganization to accommodate new technologies.
Vifor Pharma and Fresenius Kabi Form Strategic Alliance to Address Iron Deficiency in China
• Vifor Pharma and Fresenius Kabi establish a joint venture with 55/45 ownership split to market intravenous iron products across China, targeting over 2,000 tier 3 hospitals.
• The partnership aims to address China's significant iron deficiency anemia burden, affecting an estimated 20% of the population, while reducing reliance on blood transfusions.
• The collaboration will reach over 45,000 healthcare professionals, including anesthesiologists and surgeons, focusing on patient blood management and nephrology care.
FDA Grants Fast Track Status to BBO-8520 for KRAS G12C-Mutated NSCLC
• The FDA has granted fast track designation to BBO-8520, an oral agent under investigation for treating KRAS G12C-mutated metastatic non-small cell lung cancer.
• This designation aims to expedite the development and review of BBO-8520, addressing an unmet need in previously treated NSCLC patients.
• The decision was based on the drug's potential to improve outcomes in this specific genetic subgroup of lung cancer, where treatment options are limited.