As four major blockbuster drugs losing patent protection in 2028, the pharmaceutical industry faces one of the most commercially significant exclusivity cliffs in recent years, with more than $56 billion in annual revenue at stake. For generic and biosimilar sponsors, this is not simply a future market event but a present strategic window that demands […]
Read More →Sanfilippo Syndrome, caused by a deficiency in one of four enzymes responsible for breaking down heparan sulfate in the brain, presents across four biochemically distinct subtypes, each with its own genetic origin, rate of progression, and clinical nuance. Despite affecting an estimated 1 in 70,000 births globally, the condition remains without a single approved disease-modifying […]
Read More →Two gene therapy approvals. Twelve months. One disease that had almost no treatment options just three years ago. That is where Epidermolysis Bullosa (EB) stands today. The field has moved fast. But the work is far from over, and the most important opportunities may still lie ahead. What Is Epidermolysis Bullosa? Epidermolysis Bullosa is a […]
Read More →Key Takeaways from this article — Amyotrophic Lateral Sclerosis (ALS) affects approximately 30,000 Americans at any given time, with about 5,000 new diagnoses each year in the US alone. — The global ALS treatment market is in an active growth phase, driven by genetic stratification, biomarker-enabled trials, and a first genotype-specific approval in 2023. — […]
Read More →Japan has one of the most tightly regulated pharmaceutical landscapes, which necessitates a seamless supply of laboratory kits (for clinical trials) to its sites. The process requires strict adherence to the guidelines of the Pharmaceuticals and Medical Devices Agency (PMDA). For sponsors and CROs, this raises the question of how to supply laboratory kits to […]
Read More →Applying AI in clinical trials is no longer a futuristic concept; it’s a practical solution gaining traction across the industry. As clinical trials become increasingly complex—with growing data volumes, escalating costs, and stricter regulatory expectations—AI offers timely support. It is being used to optimize trial design, enhance site and patient selection, identify operational risks early, […]
Read More →Japan is quickly becoming one of the most important countries in Asia for clinical research, not just because of its strong healthcare system and regulatory structure, but also due to its growing focus on innovation and patient-centered studies. But running a successful trial in Japan takes more than just choosing a country; it requires selecting […]
Read More →Running a clinical trial for rare diseases is very different from trials in more common conditions. Small patient numbers, limited data, and complicated rules make every part of the process more careful and more creative. Instead of going over the basics, this article focuses on advanced but practical things that experienced sponsors need to think […]
Read More →Stepping into a first-in-human (FIH) clinical trial is a defining moment in drug development. It’s when a new drug that’s been years in the making finally transitions from lab bench to bedside. This isn’t about proving efficacy—that comes later. FIH clinical trials are all about safety, tolerability, and understanding how the drug behaves in the […]
Read More →In clinical trials, patient engagement has a direct impact on retention, which affects how well medical research turns out overall. Keeping patients in the study is key to making sure the trial results are valid and reliable. When people stick with the study, researchers can gather complete data and get accurate results. However many trials […]
Read More →