Development Strategy

The Path to Acceleration: In Silico Modeling Launches New Wave of Rare Disease Drug Development

Advancements in artificial intelligence, machine learning, and computing technologies have fueled progress in the use of sophisticated computational modeling and simulation as a tool for facilitating preclinical and clinical research in rare diseases. In this white paper, we explore the promise of in silico approaches to rare disease drug development and share real world scenarios...

Clinical Research: Phase 1 - Phase 4

8 Cost Containment Strategies for Rare Disease Clinical Trials

In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany these trials, such as small patient populations, limited disease data, and patient engagement challenges.  Here...

Clinical Research: Phase 1 - Phase 4

5 Stepping Stones to the Future State of Rare Disease Clinical Development

Rare diseases are a distinctive and complex field within clinical development, characterized by significant hurdles from start to finish. Addressing these complexities requires innovative approaches to achieve a future of adaptability, quality, and efficiency. Here are 5 key steps to bringing rare disease clinical development forward. Predicting the Future of Rare Disease Research As we...

The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

Pediatric rare disease trials combine rarity of disease with a restricted and geographically-dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution. Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation. Incorporating decentralized clinical trial (DCT) strategies...

The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

Meet Our Speakers: Betsy ReidSenior Vice President, Pediatrics Adam Bloomfield, MD, FAAPExecutive Director, Medical Affairs, Rare Diseases and Pediatrics

Development Strategy

Beginning With the Patient in Mind: 6 Ways to Accommodate the True Rare Disease Patient Journey

Designing patient-centric programs can have a resoundingly positive impact on clinical research and the health and life of patients. Consider these six ways to “reverse engineer” development strategy to accommodate the true rare disease patient journey. To read more, download the complete white paper.

Consulting

Orphan Drug Designation: When Is It Appropriate, and How Does It Apply to Orphan Subsets?

It is an unfortunate reality that many diseases and conditions affect such small numbers of patients that, when a sponsor develops a drug or biological product to treat them, relatively little return on investment is generated to offset the high development costs. As a result, the pharmaceutical industry has neglected many rare diseases in the past. However,...

The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

September 28th, 202311:00 a.m. EDT Pediatric rare disease trials combine rarity of disease with a restricted and geographically-dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution. Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation. Incorporating...

In Vitro Diagnostics for Early Cancer Detection: The Evolution and Promise of Liquid Biopsy Technology

As liquid biopsies become more commonplace, gaining wider FDA acceptance and even becoming standard of care in some instances, the technology is giving rise to important new questions. In this webinar, we will explore the technique’s growing importance in early cancer detection and address common questions about its use. Topics will include: Speakers: Charlie Chrisawn,...

In Vitro Diagnostics for Early Cancer Detection: The Evolution and Promise of Liquid Biopsy Technology

Speakers: Charlie Chrisawn, Executive Director, Program Strategy, In Vitro Diagnostics Michael Edwards, Senior Director, Regulatory Affairs, MedTech