News By: Gary Cramer

Research within the rare diseases space brings its own set of challenges associated with clinical care, regulatory compliance, and commercial activities within the clinical trials environment. Managing each of these realms requires a deep knowledge of the disease state, the development space, and what data and evidence the regulatory authorities and payers will demand.

The global landscape for medical technology, biotechnology, and life sciences is on the cusp of seismic change. The confluence of geopolitical tensions, economic uncertainty, and evolving social values has created a complex web of challenges and opportunities for these sectors around the world.

Transgender and gender diverse individuals face an increased risk of cardiovascular disease (CVD). There is an unmet need to identify the root causes of CVD health disparities in this population. More research and evidence-based strategies are needed.

This article elucidates the multifaceted challenges impeding clinical trials, including patient recruitment, funding, and data management issues, while also highlighting the transformative potential of emerging technologies such as artificial intelligence (AI), blockchain, and genomic sequencing.

Clinical trials are critical for assessing new treatments in terms of their potential benefits for patients’ health and for their likelihood of financial success if marketed. Yet, the traditional approach focusing on a single primary endpoint may mean lengthy trials that often fail to capture the full spectrum of the treatment effects that are crucial to patient well-being