Disease Presentation Trials 
Drug Target Review
JULY 1, 2025
Clinical trials are expensive, slow and often limited by outdated design constraints. Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. At Unlearn, he is focused on bringing digital twin technology into active use across both early and late-phase trials.
Drug Target Review
JUNE 9, 2025
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
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FDA Law Blog: Drug Discovery
JANUARY 30, 2025
However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.
Drug Target Review
APRIL 7, 2025
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.
FDA Law Blog: Drug Discovery
NOVEMBER 10, 2024
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
The Pharma Data
JUNE 23, 2025
These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.
Drug Target Review
JULY 30, 2025
The journey of cell and gene therapies from preclinical discovery to clinical trials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. These digital platforms are designed to manage and automate critical trial processes, especially related to drug management.
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