Rare Disease

Successful Rare Disease Development: 9 Best Practices to Optimize Clinical Trial Efficiency and Quality

Rare disease development is complex, characterized by distinctive challenges due to small patient populations spread across many geographies, limited disease data, and narrow site pools. These hurdles compound the universal difficulties inherent to recruiting and retaining eligible participants for clinical trials. For sponsors, successful studies involve striking a balance among cost, quality, and innovating approaches...

Where Innovation Meets Compassion: A Checklist for Implementing DCTs in Pediatric Rare Diseases

Decentralized clinical trials (DCTs), and hybrid iterations of them, redefine clinical research by shifting critical trial components – recruitment, informed consent, data collection, and monitoring – beyond the confines of traditional clinical trial sites like hospitals and research centers. This paradigm shift holds immense promise for transforming the landscape of pediatric rare disease research, offering...

WODC EU

October 23 – 25, 2024 Barcelona, Spain Booth #48 Join Premier Research at the World Orphan Congress EU for groundbreaking solutions, exclusive insights, and unmatched networking. Elevate your approach to rare diseases – visit our exhibit and be part of the future of orphan drug development!

Endpoint Protection in Rare Disease Trials: Safeguarding Data Integrity for Reliable Outcomes

The integrity of clinical trials depends on accurate and reliable data, making the protection of endpoints a critical aspect of the entire process. Endpoints, which are specific outcomes used to measure the effectiveness of an intervention, must be safeguarded to ensure the validity and credibility of the trial results. Endpoint protection is particularly important in...

Endpoint Protection in Rare Disease Trials: Safeguarding Data Integrity for Reliable Outcomes

Speaker: Angi RobinsonSenior Vice President, Specialty Areas, Premier Research

Why Rare Disease Day Matters to Us

An estimated 300 million people globally suffer from a rare disease. Further still, 25 million people in the U.S. alone are living with an undiagnosed rare disease. Limitations in disease understanding and lack of approved treatments can mean countless challenges and diminished quality of life for many battling a rare disease. For all those looking...

Premier Insight 285 | When Regulatory Meets Operations: FIH Gene Therapy Study Hits Milestone Despite IND Challenges

Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus (AAV)-based gene silencing technology to conduct a natural history study, with a goal to identify...

WODC US

April 23 – 25, 2024 Boston, Massachusetts Booth #325 Join Premier Research at the World Orphan Congress US for groundbreaking solutions, exclusive insights, and unmatched networking. Elevate your approach to rare diseases – visit our exhibit and be part of the future of orphan drug development! Adam Bloomfield, M.D., Executive Director, Medical Affairs, Rare Diseases...

Endpoint Protection in Rare Disease Trials: Safeguarding Data Integrity for Reliable Outcomes

February 22, 202411 a.m. EST The integrity of clinical trials depends on accurate and reliable data, making the protection of endpoints a critical aspect of the entire process. Endpoints, which are specific outcomes used to measure the effectiveness of an intervention, must be safeguarded to ensure the validity and credibility of the trial results. Endpoint...

Embracing the Intricacies of Rare Disease Cell and Gene Therapy Trials: A Guide to Overcoming Obstacles

Cell and gene therapies offer great promise for rare diseases, where patients—many of them children—may be severely ill or debilitated and treatment options are often limited. Development of these advanced therapeutics is complex due to both disease-specific and treatment-related factors and executing clinical trials that meet the expectations of regulators requires careful attention to study...