18th Edition July 2023

Clinical Drug Interaction Studies with Combined Oral Contraceptives?

The FDA has issued a final guidance on "Clinical Drug Interaction Studies with Combined Oral Contraceptives." The guidance is aimed to help the IND and NDA sponsors evaluate potential interactions between their drugs and oral contraceptives, design respective studies, and communicate its results and risk mitigation strategies of COCs exposure in labeling. It emphasizes the importance of assessing interactions that may affect the effectiveness and safety of contraceptives. The guidance focuses on metabolism-based interactions and highlights the need to evaluate and communicate such interactions during drug development. This guidance takes place of a draft guidance issued on November 23, 2020.

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EMA Opines on the Use of Real-World Evidence in Regulatory Decision Making

A recent report by the European Medicines Agency (EMA) highlights the value of real-world evidence (RWE) in regulatory decision-making. RWE studies conducted by regulators can complement clinical trial data and support assessments by EMA's scientific committees. Efforts are still needed to improve the anticipation and initiation of these studies to ensure timely access to RWE. The report summarizes EMA's experience with RWE studies, identifies research opportunities, and provides recommendations for enhancing RWE generation and collaboration with decision-makers. The findings will inform the ongoing work of the EMA-HMA Big Data Steering Group and the development of the DARWIN EU? platform.

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Screening Newborns for Deadly Immune Disease Saves Lives

Newborn screening for severe combined immunodeficiency (SCID), a deadly rare disease, has significantly increased the survival rate of affected children, according to a study by researchers at the National Institute of Allergy and Infectious Diseases (NIAID). The study found that screening followed by early treatment raised the five-year survival rate from 73% to 87% overall, and 92.5% for children identified through screening rather than symptoms or family history. The adoption of newborn screening for SCID has allowed for prompt treatment before life-threatening infections occur, leading to significantly better outcomes.

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Insights from ASCO 2023

Cromos Pharma's CEO, Vlad Bogin, recently attended the ASCO Annual Meeting and shared his key takeaways. ASCO 2023 showcased remarkable breakthroughs and significant advances in various areas of oncology. From immunotherapy to precision medicine, targeted therapies, liquid biopsies, and patient-centric care, the conference underscored the potential for transformative changes in cancer treatment and patient outcomes.?

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CAR-T Therapy?as a Promising Treatment for Myasthenia Gravis

A small-scale clinical trial has provided evidence that a modified version of CAR-T therapy, a type of immunotherapy used to treat blood cancers, could be adapted to treat myasthenia gravis, an autoimmune disorder of the nervous system. The modified CAR-T therapy, called Descartes-08, showed potential for longer-lasting reduction of myasthenia gravis symptoms and was well-tolerated by patients without significant side effects. The study, supported by a grant from the National Institute of Neurological Disorders and Stroke (NINDS), involved fourteen participants with generalized myasthenia gravis who received varying doses of Descartes-08. Early data from the study showed promising effectiveness, with three patients experiencing complete or near-complete elimination of their symptoms that persisted for at least six months after treatment.

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First Treatment of Obstructive Hypertrophic Cardiomyopathy Approved by NICE

The UK's National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending the use of mavacamten, also known as Camzyos (Bristol-Myers Squibb), as an add-on treatment for obstructive hypertrophic cardiomyopathy (HCM). Clinical trials have shown that mavacamten, when combined with standard care, is more effective in controlling symptoms and may delay or prevent the need for invasive surgery. Obstructive HCM is a chronic condition often caused by genetic mutations, leading to thickening and stiffening of the heart muscle, resulting in symptoms such as fatigue, chest pain, and shortness of breath. Mavacamten, a novel small-molecule modulator of cardiac myosin, offers a targeted treatment for HCM, addressing its underlying cause and potentially improving quality of life for affected individuals.

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Poland: A Thriving Hub for Biotech Advancements and Clinical Trials

Poland has emerged as a leading destination for biotech professionals seeking a dynamic and conducive environment for conducting clinical trials. With its robust healthcare infrastructure, highly skilled workforce, supportive regulatory framework, and favorable economic conditions, the country offers a compelling landscape for biotech advancements.

One of Poland's major strengths is its access to a broad range of potential study participants. The country's population of over 38 million represents a diverse genetic profile, allowing for comprehensive clinical research. Additionally, the country's centralized healthcare system ensures efficient patient recruitment and follow-up, thereby expediting trial timelines.

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