Rare Disease

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

Rare Disease

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,…

Rare Disease

Daniel Zamfir, MBA

Rare Disease

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

Rare Disease

Operationalizing Gene Therapy Trials

Rare Disease

Adam Bloomfield, M.D.

Rare Disease

Angi Robinson

Rare Disease

In Silico Trial Design in Development of Rare Disease Cell and Gene Therapies

Rare Disease

In Silico Modeling Unveils a New Era in Rare Disease Drug Development

Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of…

Rare Disease

In Silico Trial Design in Development of Rare Disease Cell and Gene Therapies

There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease.