Pharmafile Logo
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • - PMLiVE
  • Communiqué 53 Cover Image

US President Biden

- PMLiVE

FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

- PMLiVE

Biogen agrees $850m felzartamab rights deal with TJ Biopharma

The agreement gives Biogen full global rights to the investigational antibody across immune-mediated diseases

- PMLiVE

myTomorrows partners with Rare Disease Research to boost rare disease referrals

The collaboration aims to improve patient identification and access to clinical trials

- PMLiVE

AstraZeneca reports positive phase 3 data for tozorakimab in COPD

COPD affects nearly 400 million people globally and is the third leading cause of death worldwide

- PMLiVE

Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

Site Factor report illustrations

Why patient-centricity depends on site-centricity in clinical trials

Explore why patient-centricity in clinical trials relies on site-centricity, emphasising the need to design trials that empower site teams to better support patients.

Cuttsy + Cuttsy

Before and after

From cloudy to clear: visualising complex data privacy information

Discover how clear communication principles can transform complex clinical research topics, as we conclude our Health Literacy series with a practical example on participant data transparency.

Cuttsy + Cuttsy

Carer with patient

The invisible influence on trial retention: why caregivers must be part of the plan

Caregivers are vital to clinical trial retention, yet often overlooked—this article highlights their challenges, impact, and how to better support them.

Cuttsy + Cuttsy

The Site Factor - printed report

Where it all began: why we wrote ‘The Site Factor’

Discover the story behind ‘The Site Factor’ and how understanding site teams’ experiences can transform clinical trials for participants and researchers alike.

Cuttsy + Cuttsy

- PMLiVE

BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

- PMLiVE

Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment

The rare neurodegenerative disorder has no cure and no approved treatments

- PMLiVE

US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links