Manuscript deep dive

Jan 27, 2025

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Accelerating hope? Optimising rare disease MHRA/FDA approval pathways and access strategy considerations

Accelerating hope? Optimising rare disease MHRA/FDA approval pathways and access strategy considerations

Written by Jo Wallis, Head of Commercialisation and Pricing Strategy   The recent developments put forward by the Medicines and Healthcare products Regulatory Agency (MHRA) and the Food and Drug Administration (FDA) to streamline the ... Read more

The role of orphan drug designations in driving research and innovation for rare diseases

The role of orphan drug designations in driving research and innovation for rare diseases

Written by Emma Bartram, Associate Systematic Review Analyst   A rare disease is one that affects fewer than 1 in 2,000 individuals, and yet 3.5–5.9% of people will experience a rare disease over the course of their lifetime (1,2). ... Read more

HTA monthly update

HTA monthly update

By Toby Hobbs   Changes to NICE ICER thresholds confirmed NICE has announced that from April 2026 there will be an increase to its cost-effectiveness threshold. This marks one of the biggest shifts in UK Health technology assessment ... Read more

Evaluating Health Inequalities: NICE’s Modular Update

Evaluating Health Inequalities: NICE’s Modular Update

Written by Paloma Charlesworth (Assistant Project Manager)   Background  Health inequalities are systematic, avoidable, and unjust differences in health outcomes between patient groups. Despite decades of policy and research, they not ... Read more